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What changed in Marker Therapeutics, Inc.'s 10-K — 2023 vs 2024

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Paragraph-level year-over-year comparison of Marker Therapeutics, Inc.'s 2023 and 2024 10-K annual filings, covering the Business, Risk Factors, Legal Proceedings, Cybersecurity, MD&A and Market Risk sections. Every new, removed and edited paragraph is highlighted side-by-side so you can see exactly what management changed in the 2024 report.

+443 added−377 removedSource: 10-K (2025-03-31) vs 10-K (2023-12-31)

Top changes in Marker Therapeutics, Inc.'s 2024 10-K

443 paragraphs added · 377 removed · 292 edited across 7 sections

Item 1. Business

Business — how the company describes what it does

86 edited+44 added−15 removed214 unchanged
Biggest changeIn addition, upon a liquidity event (as defined in the BCM License Agreement) of the Company, BCM will receive a liquidity incentive payment of 0.5% of the liquidity event proceeds (as defined in the BCM License Agreement). 15 Table of Contents We have agreed to indemnify BCM and certain persons affiliated with BCM against claims and liabilities directly or indirectly related to or arising out of the design, process, manufacture or use by any third party of the licensed products, even though such claims and liabilities result in whole or in part from the negligence of the BCM indemnified parties or are based upon doctrines of strict liability or product liability, but not claims or liabilities arising from the gross negligence or intentional misconduct of any such BCM indemnified parties.
Biggest changeWe have agreed to indemnify BCM and certain persons affiliated with BCM against claims and liabilities directly or indirectly related to or arising out of the design, process, manufacture or use by any third party of the licensed products, even though such claims and liabilities result in whole or in part from the negligence of the BCM indemnified parties or are based upon doctrines of strict liability or product liability, but not claims or liabilities arising from the gross negligence or intentional misconduct of any such BCM indemnified parties. 15 Table of Contents Unless terminated sooner, the license will expire on a licensed product-by-product basis and country-by-country basis, on the later of (1) the date of expiration of the last valid claim of patent rights to expire that covers the sale of such licensed product in such country, or (2) the first date following the tenth anniversary of the first commercial sale of first licensed product by us in such country.
Our ability to compete successfully will depend, in part, on our ability to: ● develop proprietary products; ● develop and maintain products that reach the market first, are technologically superior to and/or are of lower cost than other products in the market; ● attract and retain scientific, product development and sales and marketing personnel; 14 Table of Contents ● obtain patent or other proprietary protection for our products and technologies; ● obtain required regulatory approvals; and ● manufacture, market, distribute and sell any products that we develop.
Our ability to compete successfully will depend, in part, on our ability to: ● develop proprietary products; ● develop and maintain products that reach the market first, are technologically superior to and/or are of lower cost than other products in the market; ● attract and retain scientific, product development and sales and marketing personnel; ● obtain patent or other proprietary protection for our products and technologies; ● obtain required regulatory approvals; and 14 Table of Contents ● manufacture, market, distribute and sell any products that we develop.
Such laws include, without limitation: the U.S. federal Anti-Kickback Statute, which prohibits, among other things, persons and entities from knowingly and willfully soliciting, receiving, offering or paying remuneration, to induce, or in return for, either the referral of an individual, or the purchase or recommendation of an item or service for which payment may be made under any federal healthcare program; federal civil and criminal false claims laws and civil monetary penalty laws, which prohibit, among other things, individuals or entities from knowingly presenting, or causing to be presented, claims for payment to the federal government, including federal healthcare programs, that are false or fraudulent; the Health Insurance Portability and Accountability Act, or HIPAA, which created additional federal criminal statutes which prohibit, among other things, executing a scheme to defraud any healthcare benefit program and making false statements relating to healthcare matters, and which, as amended by Health Information Technology for Economic and Clinical Health Act, or HITECH, also imposes certain requirements on HIPAA covered entities and their business associates and covered subcontractors relating to the privacy, security and transmission of individually identifiable health information; the U.S. federal Physician Payments Sunshine Act, which requires certain manufacturers of drugs, devices, biologics and medical supplies for which payment is available under Medicare, Medicaid or the Children’s Health Insurance Program, with specific exceptions, to annually report to the federal government, information related to payments or other transfers of value made to physicians (as defined by the Physician Payments Sunshine Act), other covered physicians and teaching hospitals, as well as ownership and investment interests held by health care professionals and their immediate family members; and U.S. state and foreign law equivalents of each of the above federal laws, which, in some cases, differ from each other in significant ways, and may not have the same effect, thus complicating compliance efforts.
Such laws include, without limitation: the U.S. federal Anti-Kickback Statute, which prohibits, among other things, persons and entities from knowingly and willfully soliciting, receiving, offering or paying remuneration, to induce, or in return for, either the referral of an individual, or the purchase or recommendation of an item or service for which payment may be made under any federal healthcare program; federal civil and criminal false claims laws and civil monetary penalty laws, which prohibit, among other things, individuals or entities from knowingly presenting, or causing to be presented, claims for payment to the federal government, including federal healthcare programs, that are false or fraudulent; the Health Insurance Portability and Accountability Act, or HIPAA, which created additional federal criminal statutes which prohibit, among other things, executing a scheme to defraud any healthcare benefit program and making false statements relating to healthcare matters, and which, as amended by Health Information Technology for Economic and Clinical Health Act, or HITECH, also imposes certain 24 Table of Contents requirements on HIPAA covered entities and their business associates and covered subcontractors relating to the privacy, security and transmission of individually identifiable health information; the U.S. federal Physician Payments Sunshine Act, which requires certain manufacturers of drugs, devices, biologics and medical supplies for which payment is available under Medicare, Medicaid or the Children’s Health Insurance Program, with specific exceptions, to annually report to the federal government, information related to payments or other transfers of value made to physicians (as defined by the Physician Payments Sunshine Act), other covered physicians and teaching hospitals, as well as ownership and investment interests held by health care professionals and their immediate family members; and U.S. state and foreign law equivalents of each of the above federal laws, which, in some cases, differ from each other in significant ways, and may not have the same effect, thus complicating compliance efforts.
The dose administered in this multicenter trial was up to 200 million cells every two weeks for up to three doses. ● In April 2020, the Orphan Product Development Office of the United States Food and Drug Administration, or the FDA, granted orphan drug designation to MT-401 (zedenoleucel), a multiTAA-specific T cell therapy that targets four TAAs, for the treatment of AML. ● The same multiTAA-specific T cell therapy has been well tolerated in an ongoing Phase 1 clinical trial in AML and myelodysplastic syndrome, or MDS, conducted by our strategic partner Baylor College of Medicine, or BCM. 2 Table of Contents ● As reported in a 2021 publication by Lulla et al., 11 of the 17 patients in the adjuvant disease setting dosed with the multiTAA-specific T cell therapy after receiving an allogeneic HSCT were relapse free [median leukemia-free survival, or LFS, not reached at a median follow-up of 1.9 years], with 11 of 15 patients (two patients were each treated during two different remissions) remaining alive (estimated two-year overall survival of 77%) at a median follow-up of 1.9 years post-infusion, which compares favorably with HSCT outcomes for risk-matched AML/MDS patients post-HSCT [median LFS of nine to 15 months and two-year survival probability of 42%]. ● Additionally, eight patients were treated for active disease that was resistant to salvage therapy post-HSCT with a median of five prior lines of therapy (range: four to 10). o One of the eight patients crossed over from the adjuvant group, while two patients enrolled twice, but all three patients had active AML that failed another line of salvage therapy after their first multiTAA-specific T cell infusion. o Two of the eight patients achieved objective responses, with one complete response and one partial response, with six patients continuing with stable disease. o We have observed evidence of a patient’s natural immune system participating in cancer killing (epitope spreading) after infusion of our multiTAA-specific T cell therapy.
The dose administered in this multicenter trial was up to 200 million cells every two weeks for up to three doses. ● In April 2020, the Orphan Product Development Office of the United States Food and Drug Administration, or the FDA, granted orphan drug designation to MT-401 (zedenoleucel), a MAR-T cell therapy that targets four TAAs, for the treatment of AML. ● The same MAR-T cell therapy has been well tolerated in an ongoing Phase 1 clinical trial in AML and myelodysplastic syndrome, or MDS, conducted by our strategic partner Baylor College of Medicine, or BCM. ● As reported in a 2021 publication by Lulla et al., 11 of the 17 patients in the adjuvant disease setting dosed with the MAR-T cell therapy after receiving an allogeneic HSCT were relapse free [median leukemia-free survival, or LFS, not reached at a median follow-up of 1.9 years], with 11 of 15 patients (two patients were each treated during two different remissions) remaining alive (estimated two-year overall survival of 77%) at a median follow-up of 1.9 years post-infusion, which compares favorably with HSCT outcomes for risk-matched AML/MDS patients post-HSCT [median LFS of nine to 15 months and two-year survival probability of 42%]. 3 Table of Contents ● Additionally, eight patients were treated for active disease that was resistant to salvage therapy post-HSCT with a median of five prior lines of therapy (range: four to 10). o One of the eight patients crossed over from the adjuvant group, while two patients enrolled twice, but all three patients had active AML that failed another line of salvage therapy after their first MAR-T cell infusion. o Two of the eight patients achieved objective responses, with one complete response and one partial response, with six patients continuing with stable disease. o We have observed evidence of a patient’s natural immune system participating in cancer killing (epitope spreading) after infusion of our MAR-T cell therapy.
As reported in a 2021 publication by Lulla et al., 11 of the 17 patients in the adjuvant disease setting dosed with the multiTAA-specific T cell therapy after receiving an allogeneic HSCT did not relapse during the follow-up period of the study [median LFS not reached at a median follow-up of 1.9 years], with 11 of 15 patients (two patients were each treated during two different remissions) remaining alive (estimated two-year overall survival of 77%) at a median follow-up of 1.9 years post-infusion which compares favorably with HSCT outcomes for risk-matched AML/MDS patients post-HSCT [median LFS of nine to 15 months and two-year survival probability of 42%].
As reported in a 2021 publication by Lulla et al., 11 of the 17 patients in the adjuvant disease setting dosed with the MAR-T cell therapy after receiving an allogeneic HSCT did not relapse during the follow-up period of the study [median LFS not reached at a median follow-up of 1.9 years], with 11 of 15 patients (two patients were each treated during two different remissions) remaining alive (estimated two-year overall survival of 77%) at a median follow-up of 1.9 years post-infusion which compares favorably with HSCT outcomes for risk-matched AML/MDS patients post-HSCT [median LFS of nine to 15 months and two-year survival probability of 42%].
Significantly, no patient with a CR has subsequently 7 Table of Contents relapsed with disease, whereas typically 30% or more of patients with CR in reported CAR-T studies relapse within one year. In patient results to date in this trial, observed therapeutic responses appear to be highly durable, with some patients being relapse-free beyond five years.
Significantly, no patient with a CR has subsequently relapsed with disease, whereas typically 30% or more of patients with CR in reported CAR-T studies relapse within one year. In patient results 8 Table of Contents to date in this trial, observed therapeutic responses appear to be highly durable, with some patients being relapse-free beyond five years.
Upon release of the final patient product, the cells are frozen and transported to the site where the cells will be administered. The standard dose for patients with lymphoma ranges from 100 to 400 million cells per adult patient. Manufacturing The manufacturing process was originally developed at Baylor College of Medicine, where we also conducted our clinical trials.
Upon release of the final patient product, the cells are frozen and transported to the site where the cells will be administered. The standard dose for patients with lymphoma ranges from 100 to 400 million cells per adult patient. Manufacturing Our manufacturing process was originally developed at Baylor College of Medicine, where we initially conducted our clinical trials.
In 2021, the ARTEMIS trial conducted by Marker has completed the safety lead-in portion, which tested the comparability of MT-401 or zedenoleucel, the multiTAA-specific T cell product manufactured using peptides from two different vendors and enrolled six patients with active disease: one measurable residual disease (MRD) positive patient and five frank relapse patients. ● Consistent with the results of the BCM Phase 1 trial, there were no dose-limiting toxicities, cytokine release syndrome or neurotoxicity observed in this stage of the trial\.
In 2021, the ARTEMIS trial conducted by Marker has completed the safety lead-in portion, which tested the comparability of MT-401 or zedenoleucel, the MAR-T cell product manufactured using peptides from two different vendors and enrolled six patients with active disease: one measurable residual disease (MRD) positive patient and five frank relapse patients. ● Consistent with the results of the BCM Phase 1 trial, there were no dose-limiting toxicities, cytokine release syndrome or neurotoxicity observed in this stage of the trial.
In 2020, we reported that in this trial, BCM administered multiTAA-specific T cells to a total of 31 patients with advanced or borderline resectable pancreatic adenocarcinoma in three arms: 13 patients in Arm A, which included patients with unresectable/metastatic disease who were responding to standard first-line chemotherapy; 12 patients in Arm B, which included patients with progressive disease or therapy intolerance; and eight patients in Arm C, which includes patients with surgically resectable disease.
In 2020, we reported that in this trial, BCM administered MAR-T cells to a total of 31 patients with advanced or borderline resectable pancreatic adenocarcinoma in three arms: 13 patients in Arm A, which included patients with unresectable/metastatic disease who were responding to standard first-line chemotherapy; 12 patients in Arm B, which included patients with progressive disease or therapy intolerance; and eight patients in Arm C, which includes patients with surgically resectable disease.
At full scale production, we estimate a single donor could provide treatment for approximately 40 patients, and the current stability program indicates that OTS multiTAA-specific T cell products are stable for more than a year in liquid nitrogen, which we expect will permit future on-demand availability for broad-scale implementation.
At full scale production, we estimate a single donor could provide treatment for approximately 40 patients, and the current stability program indicates that OTS MAR-T cell products are stable for more than a year in liquid nitrogen, which we expect will permit future on-demand availability for broad-scale implementation.
BCM Exclusive License Agreement On March 16, 2018, we entered into an exclusive license agreement, or the BCM License Agreement, with BCM, under which we received a worldwide, exclusive license to BCM’s rights in and to certain intellectual property rights, including European patent EP 2470644 (estimated expiration date August 24, 2030), to develop and commercialize multiTAA-specific T cell product candidates.
BCM Exclusive License Agreement On March 16, 2018, we entered into an exclusive license agreement, or the BCM License Agreement, with BCM, under which we received a worldwide, exclusive license to BCM’s rights in and to certain intellectual property rights, including European patent EP 2470644 (estimated expiration date August 24, 2030), to develop and commercialize MAR-T cell product candidates.
We believe the improved manufacturing process enables products with increased antigen specificity and diversity, both of which have a strong linear correlation to anti-tumor activity and has resulted in a four-fold increase in potency in vitro. ​ · Invest in our platform to maximize the beneficial outcomes for cancer patients.
We believe the improved manufacturing process enables products with increased antigen specificity and diversity, both of which have a strong linear correlation to anti-tumor activity and has resulted in a four-fold increase in potency in vitro. 5 Table of Contents ● Invest in our platform to maximize the beneficial outcomes for cancer patients.
When infused into a patient with cancer, the multiTAA-specific T cells are designed to kill cancer cells expressing the TAA and potentially recruit the patient’s immune system to participate in the cancer killing process. We licensed the underlying technology for multiTAA-specific T cell therapy from Baylor College of Medicine, or BCM, in March 2018.
When infused into a patient with cancer, the MAR-T cells are designed to kill cancer cells expressing the TAA and potentially recruit the patient’s immune system to participate in the cancer killing process. We licensed the underlying technology for MAR-T cell therapy from Baylor College of Medicine, or BCM, in March 2018.
While the blood source and the antigens for stimulation differ between the autologous and OTS therapies, the manufacturing process for each product is identical. 6 Table of Contents Cancers are heterogeneous in their expression of antigens.
While the blood source and the antigens for stimulation differ between the autologous and OTS therapies, the manufacturing process for each product is identical. 7 Table of Contents Cancers are heterogeneous in their expression of antigens.
The five-year mortality rate for patients who receive an allogeneic HSCT exceeds 50%, and patients who relapse after a transplant have a survival expectation of approximately 4.5 months. BCM recently completed a Phase 1 AML/MDS clinical trial of the multiTAA-specific T cell therapy for the treatment of patients with post-transplant AML.
The five-year mortality rate for patients who receive an allogeneic HSCT exceeds 50%, and patients who relapse after a transplant have a survival expectation of approximately 4.5 months. BCM recently completed a Phase 1 AML/MDS clinical trial of the MAR-T cell therapy for the treatment of patients with post-transplant AML.
This allows a 90% decrease in the number of interventions during production and an improved final T cell product compared to the original product used in previous clinical trials on multiTAA-specific T cells. ● After completing the safety lead-in portion, we initiated the remainder of the Phase 2 trial in July 2021.
This allows a 90% decrease in the number of interventions during production and an improved final T cell product compared to the original product used in previous clinical trials on MAR-T cells. ● After completing the safety lead-in portion, we initiated the remainder of the Phase 2 trial in July 2021.
With respect to both licensed and company-owned intellectual property, we cannot be sure that patents will be granted with respect to any of our pending patent applications or with respect to any patent applications filed in the future, nor can we be sure that any of our existing 16 Table of Contents patents or any patents that may be granted in the future will be commercially useful in protecting our commercial products and methods of manufacturing the same.
With respect to both licensed and company-owned intellectual property, we cannot be sure that patents will be granted with respect to any of our pending patent applications or with respect to any patent applications filed in the future, nor can we be sure that any of our existing patents or any patents that may be granted in the future will be commercially useful in protecting our commercial products and methods of manufacturing the same.
Based on our observations in clinical trials in AML, pancreatic cancer, lymphoma, ALL and MM, we believe that the multiTAA-specific T cell therapies have the potential to mediate a meaningful anti-tumor effect, as well as significant in vivo expansion of T cells.
Based on our observations in clinical trials in AML, pancreatic cancer, lymphoma, ALL and MM, we believe that the MAR-T cell therapies have the potential to mediate a meaningful anti-tumor effect, as well as significant in vivo expansion of T cells.
In the OTS program, we intend to dose patients using “banked” products based on partially human leukocyte antigen (HLA) matching. The U.S. FDA has cleared our clinical protocol to investigate MT-401-OTS as a treatment in patients with AML. We 3 Table of Contents already established a cellular inventory manufactured from healthy donors, with ongoing efforts to further expand the inventory.
In the OTS program, we intend to dose patients using “banked” products based on partially human leukocyte antigen (HLA) matching. The U.S. FDA has cleared our clinical protocol to investigate MT-401-OTS as a treatment in patients with AML. We already established a cellular inventory manufactured from healthy donors, with ongoing efforts to further expand the inventory.
None of the patients in CR had relapsed, and the range for the duration of CR in these patients was between two and over five years after being infused with the multiTAA-specific T cell therapy with the exception of one patient who died of an unrelated pneumonia while in a CR.
None of the patients in CR had relapsed, and the range for the duration of CR in these patients was between two and over five years after being infused with the MAR-T cell therapy with the exception of one patient who died of an unrelated pneumonia while in a CR.
To achieve this objective, a strategic focus for us has been identifying and licensing key patents and patent applications that serve to enhance our intellectual property and technology position. Currently, all of our multiTAA-specific T cell intellectual property rights are licensed from BCM.
To achieve this objective, a strategic focus for us has been identifying and licensing key patents and patent applications that serve to enhance our intellectual property and technology position. Currently, all of our MAR-T cell intellectual property rights are licensed from BCM.
We are developing a portfolio of highly differentiated T cell therapies utilizing the multiTAA-specific T cell platform that we believe has the potential to significantly disrupt the current cell therapy landscape, while substantially improving survival and quality of life for patients with cancers.
We are developing a portfolio of highly differentiated T cell therapies utilizing the MAR-T cell platform that we believe has the potential to significantly disrupt the current cell therapy landscape, while substantially improving survival and quality of life for patients with cancers.
FDA to investigate MT-601 in a Phase 1 trial in patients with pancreatic cancer in combination with first-line chemotherapy. · Off-the-Shelf (OTS) multiTAA-specific T cell therapies – The OTS product targets WT1, NY-ESO-1, PRAME, and Survivin antigens (MT-401-OTS).
FDA to investigate MT-601 in a Phase 1 trial in patients with pancreatic cancer in combination with first-line chemotherapy. · Off- the-Shelf (OTS) MAR-T cell therapies – The MT-401-OTS product targets WT1, NY-ESO-1, PRAME, and Survivin antigens (MT-401-OTS).
We believe our multiTAA-specific T cell therapy represent a safe alternative to CAR-T cells and can be manufactured at a fraction of the cost of a gene-modified T cell product, with substantially reduced complexity of manufacturing. · Low incidence rate of adverse events.
We believe our MAR-T cell therapy represent a safe alternative to CAR-T cells and can be manufactured at a fraction of the cost of a gene-modified T cell product, with substantially reduced complexity of manufacturing. ● Low incidence rate of adverse events.
If existing or future patents held by third parties and containing broad claims over technology used by us were upheld by a court or other authority of competent jurisdiction, the holders of such patents could require us to obtain licenses to use such technology.
If existing or future patents held by third parties and containing broad claims over technology used by us were upheld 17 Table of Contents by a court or other authority of competent jurisdiction, the holders of such patents could require us to obtain licenses to use such technology.
When a CAR-T cell product is applied to a specific antigen of a heterogeneous disease, CAR-T cells may leave behind tumor cells that do not express the target antigen, which can lead to tumor relapse due to immune escape.
When a CAR-T cell product is applied to a 6 Table of Contents specific antigen of a heterogeneous disease, CAR-T cells may leave behind tumor cells that do not express the target antigen, which can lead to tumor relapse due to immune escape.
One of the eight patients crossed over from the adjuvant group while two patients enrolled twice, but all three patients had active AML that failed another line of salvage therapy after their first multiTAA-specific T cell infusion.
One of the eight patients crossed over from the adjuvant group while two patients enrolled twice, but all three patients had active AML that failed another line of salvage therapy after their first MAR-T cell infusion.
We plan to explore new product opportunities by increasing and/or customizing the antigens we target to expand the indications in which the multiTAA-specific T cell products will be efficacious, including solid tumors or other hematologic malignancies.
We plan to explore new product opportunities by increasing and/or customizing the antigens we target to expand the indications in which the MAR-T cell products will be efficacious, including solid tumors or other hematologic malignancies.
Process Development and Manufacturing of the MultiTAA-Specific T Cell Therapies In the manufacturing process, blood is drawn from either the individual patient (in the case of the autologous T cells) or from a healthy donors/commercially available leukapheresis material (in the case of the OTS program).
Process Development and Manufacturing of the MAR-T Cell Therapies In the manufacturing process, blood is drawn from either the individual patient (in the case of the autologous T cells) or from a healthy donors/commercially available leukapheresis material (in the case of the OTS program).
Pancreatic Cancer In May 2020, we reported interim data from an ongoing Phase 1/2 clinical trial of the multiTAA-specific T cell therapy for the treatment of pancreatic adenocarcinoma being conducted by BCM.
Pancreatic Cancer In May 2020, we reported interim data from an ongoing Phase 1/2 clinical trial of the MAR-T cell therapy for the treatment of pancreatic adenocarcinoma being conducted by BCM.
The MultiTAA-Specific T Cell Therapies We are advancing two multiTAA-specific T cell therapies through clinical development: · Autologous multiTAA-specific T cell therapies – The autologous product targets the NY-ESO-1, PRAME, MAGE - A4, Survivin, WT1 and SSX2 antigens (MT-601).
MAR-T Cell Therapies We are advancing two MAR-T cell therapies through clinical development: · Autologous MAR-T cell therapies – The autologous product targets the NY-ESO-1, PRAME, MAGE - A4, Survivin, WT1 and SSX2 antigens (MT-601).
Unlike CAR and TCR-based approaches, the multiTAA-specific T cell therapy does not require genetic modification of T cells, a costly and complex process that significantly complicates the manufacturing of a patient product.
Unlike CAR and TCR-based approaches, the MAR-T cell therapy does not require genetic modification of T cells, a costly and complex process that significantly complicates the manufacturing of a patient product.
Compared to current gene-modified T cell therapies, the multiTAA-specific T cell product candidates are characterized by the following: · Clinical benefits observed in early-stage clinical trials in multiple cancer indications.
Compared to current gene-modified T cell therapies, the MAR-T cell product candidates are characterized by the following: ● Clinical benefits observed in early-stage clinical trials in multiple cancer indications.
These patients in the chemo-responsive arm have completed at least three months of standard-of-care chemotherapy (gemcitabine/nab-paclitaxel or FOLFIRINOX), which is the period during which a response to chemotherapy would typically occur, before receiving up to six administrations of multiTAA-specific T cell therapy in conjunction with chemotherapy. ● Out of the 13 evaluable patients (best overall response): o four patients experienced objective responses after administration of multiTAA-specific T cells; o one patient experienced a radiographic complete response occurring at month nine after starting chemotherapy; o three patients experienced partial responses per RECIST occurring at six-nine months after starting chemotherapy; o six patients experienced stable disease; o one patient experienced a mixed response. ● Patients had durable cancer control with nine of the 13 patients exceeding historical control of overall survival; ● Five patients enrolled in the study were not administered multiTAA-specific T cells, either because of disease progression (four patients) which made them ineligible for treatment, or because insufficient starting material from the patient was available for manufacturing (one patient); ● Evidence of epitope-spreading was observed in all responders, suggesting that the multiTAA-specific T cell therapy triggered the recruitment of a broader endogenous immune system response for improved anti-tumor activity; ● No infusion-related reactions, cytokine release syndrome or neurotoxicity was observed; In patients responding to therapy, significant expansion of the infused multiTAA-specific T cell therapy was observed. 10 Table of Contents Lymphoma BCM evaluated the multiTAA-specific T cell therapy (5 TAA product) in a Phase 1 clinical trial for the treatment of patients with lymphoma.
These patients in the chemo-responsive arm have completed at least three months of standard-of-care chemotherapy (gemcitabine/nab-paclitaxel or FOLFIRINOX), which is the period during which a response to chemotherapy would typically occur, before receiving up to six administrations of MAR-T cell therapy in conjunction with chemotherapy. ● Out of the 13 evaluable patients (best overall response): o four patients experienced objective responses after administration of MAR-T cells; o one patient experienced a radiographic complete response occurring at month nine after starting chemotherapy; o three patients experienced partial responses per RECIST occurring at six-nine months after starting chemotherapy; o six patients experienced stable disease; o one patient experienced a mixed response. ● Patients had durable cancer control with nine of the 13 patients exceeding historical control of overall survival; ● Five patients enrolled in the study were not administered MAR-T cells, either because of disease progression (four patients) which made them ineligible for treatment, or because insufficient starting material from the patient was available for manufacturing (one patient); ● Evidence of epitope-spreading was observed in all responders, suggesting that the MAR-T cell therapy triggered the recruitment of a broader endogenous immune system response for improved anti-tumor activity; ● No infusion-related reactions, cytokine release syndrome or neurotoxicity was observed; In patients responding to therapy, significant expansion of the infused MAR-T cell therapy was observed.
Two of the eight patients achieved objective responses with one complete response and one partial response, with six patients continuing with stable disease. ​ In this trial, the multiTAA-specific T cell therapy was well tolerated, with no drug-related serious adverse events and no instances of greater than Grade 2 acute graft-versus-host disease or moderate-severe chronic GVHD.
Two of the eight patients achieved objective responses with one complete response and one partial response, with six patients continuing with stable disease. 9 Table of Contents In this trial, the MAR-T cell therapy was well tolerated, with no drug-related serious adverse events and no instances of greater than Grade 2 acute graft-versus-host disease or moderate-severe chronic GVHD.
We, along with third-party contractors, will be required to navigate the various preclinical, clinical and commercial approval requirements of the governing regulatory agencies of the countries in which we wish to conduct studies or seek approval or licensure of our product candidates. 18 Table of Contents The process required by the FDA before biologic product candidates may be marketed in the United States generally involves the following: ● completion of preclinical laboratory tests and animal studies performed in accordance with the FDA’s current Good Laboratory Practices, or GLP, regulation; ● submission to the FDA of an IND, which must become effective before clinical trials may begin and must be updated annually or when significant changes are made; ● approval by an independent Institutional Review Board, or IRB, or ethics committee at each clinical site before the trial is commenced; ● performance of adequate and well-controlled human clinical trials to establish the safety, purity and potency of the proposed biologic product candidate for its intended purpose; ● preparation of and submission to the FDA of a biologics license application, or BLA, after completion of all pivotal clinical trials; ● a determination by the FDA within 60 days of its receipt of a BLA to file the application for review; ● satisfactory completion of an FDA Advisory Committee review, if applicable; ● satisfactory completion of an FDA pre-approval inspection of the manufacturing facility or facilities at which the proposed product is produced to assess compliance with cGMP and to assure that the facilities, methods and controls are adequate to preserve the biological product’s continued safety, purity and potency, and of selected clinical investigation sites to assess compliance with Good Clinical Practices, or GCP; and ● FDA review and approval of the BLA to permit commercial marketing of the product for particular indications for use in the United States. 19 Table of Contents Preclinical and Clinical Development Prior to beginning the first clinical trial with a product candidate, we must submit an IND to the FDA.
The process required by the FDA before biologic product candidates may be marketed in the United States generally involves the following: ● completion of preclinical laboratory tests and animal studies performed in accordance with the FDA’s current Good Laboratory Practices, or GLP, regulation; ● submission to the FDA of an IND, which must become effective before clinical trials may begin and must be updated annually or when significant changes are made; ● approval by an independent Institutional Review Board, or IRB, or ethics committee at each clinical site before the trial is commenced; ● performance of adequate and well-controlled human clinical trials to establish the safety, purity and potency of the proposed biologic product candidate for its intended purpose; ● preparation of and submission to the FDA of a biologics license application, or BLA, after completion of all pivotal clinical trials; ● a determination by the FDA within 60 days of its receipt of a BLA to file the application for review; ● satisfactory completion of an FDA Advisory Committee review, if applicable; ● satisfactory completion of an FDA pre-approval inspection of the manufacturing facility or facilities at which the proposed product is produced to assess compliance with cGMP and to assure that the facilities, methods and controls are adequate to preserve the biological product’s continued safety, purity and potency, and of selected clinical investigation sites to assess compliance with Good Clinical Practices, or GCP; and ● FDA review and approval of the BLA to permit commercial marketing of the product for particular indications for use in the United States. 19 Table of Contents Preclinical and Clinical Development Prior to beginning the first clinical trial with a product candidate, we must submit an IND to the FDA.
After blood is drawn, PBMCs are isolated and used to manufacture a patient-specific product. These cells are placed inside a G-Rex manufacturing device and combined with an experimentally optimized mix of GMP-grade cytokines that is used to restore and enhance the functional capability of the cultured T cells.
After blood is drawn, PBMCs are isolated and used to manufacture a patient-specific product. These cells are placed inside a cell culture device and combined with an experimentally optimized mix of GMP-grade cytokines that is used to restore and enhance the functional capability of the cultured T cells.
Correlative studies showed that the patient saw significant expansion of infused multiTAA-specific T cells. There were no objective responses from the frank relapse patients.
Correlative studies showed that the patient saw significant expansion of infused MAR-T cells. There were no objective responses from the frank relapse patients.
We are advancing two product candidates for 3 clinical indications as part of our multiTAA-specific T cell program for: ● Autologous multiTAA product for the treatment of lymphoma and pancreatic cancer (MT-601) ● Off-the-Shelf (OTS) product in various indications (e.g., MT-401-OTS) We do not genetically engineer our multiTAA-specific T cell therapies and we believe that our product candidates are superior to T cells engineered with chimeric antigen receptors, or CAR-T, for several reasons including: ● Multiple targets → enhanced tumoricidal effect→ minimized tumor immune escape ● Clinical safety → no treatment-related side effects, including cytokine release syndrome (CRS) or other severe adverse effects (SAEs), were attributed to the use of multiTAA-specific T cell therapies to date ● Non-genetically engineered T cell products → selective expansion of tumor-specific T cells from a patient’s or donor’s blood capable of recognizing a broad range of tumor antigens → no risk of mutagenesis and reduced manufacturing complexity → lower cost ​ For these reasons, we believe our endogenous T cell receptor-based therapies may provide meaningful clinical benefit and safety to patients with both hematological and solid tumors. 1 Table of Contents We believe that the simplicity of our manufacturing process allows additional modifications to expand multiTAA-specific T cell recognition of cancer targets.
We are advancing two product candidates for 3 clinical indications as part of our MAR-T cell program for: ● Autologous MAR-T cell product for the treatment of lymphoma and pancreatic cancer (MT-601) ● Off-the-Shelf (OTS) product in various indications (e.g., MT-401-OTS) We do not genetically engineer our MAR-T cell therapies and we believe that our product candidates are superior to T cells engineered with chimeric antigen receptors, or CAR-T, for several reasons including: ● Multiple targets → enhanced tumoricidal effect→ minimized tumor immune escape ● Clinical safety → no treatment-related side effects, including immune effector cell-associated neurotoxicity syndrome (ICANS) or other severe adverse effects (SAEs), were attributed to the use of MAR-T cell therapies to date ● Non-genetically engineered T cell products → selective expansion of tumor-specific T cells from a patient’s or donor’s blood capable of recognizing a broad range of tumor antigens → no risk of mutagenesis and reduced manufacturing complexity → lower cost ​ ​ 1 Table of Contents For these reasons, we believe our endogenous T cell receptor-based therapies may provide meaningful clinical benefit and safety to patients with both hematological and solid tumors.
There is significant interest in promoting changes in healthcare systems with the stated goals of containing healthcare costs, improving quality or expanding access.
There is significant interest in promoting changes in healthcare systems with the stated goals of containing healthcare costs, 25 Table of Contents improving quality or expanding access.
As a result, the FDA concurrently released a final rule and guidance in September 2020 providing pathways for states to build and submit importation 26 Table of Contents plans for drugs from Canada.
As a result, the FDA concurrently released a final rule and guidance in September 2020 providing pathways for states to build and submit importation plans for drugs from Canada.
No legislation or administrative actions have been finalized to implement these principles. However, it is unclear whether these or similar policy initiatives will be implemented in the future.
No legislation or administrative actions have been finalized to implement these principles. However, it is 26 Table of Contents unclear whether these or similar policy initiatives will be implemented in the future.
All funding agencies have agreed to continue their financial support and to shift funds to the MT-401-OTS program. Off-the-Shelf MT-401 (MT-401-OTS) for the Treatment of AML: Marker previously announced that it intends to focus on the advancement of the MT-401-OTS program in patients with AML.
All funding agencies have agreed to continue their financial support and to shift funds to the MT-401-OTS program. Off- the-Shelf MT-401 (MT-401-OTS) for the Treatment of AML and MDS: Marker previously announced that it intends to focus on the advancement of the MT-401-OTS program in patients with Acute Myeloid Leukemia (AML) and Myelodysplastic Syndromes (MDS).
A total of 32 patients received two protocol-specified infusions of multiTAA-specific T cells, 14 with Hodgkin lymphoma, or HL, and 18 with aggressive non-Hodgkin lymphoma, or NHL, [diffuse large B-cell lymphoma, or DLBCL, (n=12), mantle cell lymphoma, or MCL, (n=2), T-cell lymphoma (n=3) and composite lymphoma (HL and DLBCL, n=1)].
A total of 32 patients received two protocol-specified infusions of MAR-T cells, 14 with Hodgkin lymphoma, or HL, and 18 with aggressive 10 Table of Contents non-Hodgkin lymphoma, or NHL, [diffuse large B-cell lymphoma, or DLBCL, (n=12), mantle cell lymphoma, or MCL, (n=2), T-cell lymphoma (n=3) and composite lymphoma (HL and DLBCL, n=1)].
Our website and the information contained therein or connected thereto are not intended to be incorporated into this Annual Report on Form 10-K.
Our website and the information contained therein or connected thereto are not intended to be incorporated into this Annual Report on Form 10-K. 27 Table of Contents
As illustrated below, in the adjuvant lymphoma group, all 17 patients had entered CR, with 14 patients in continued complete remission, or CCR, without relapsing. The duration of response ranged from approximately nine months to over five years. ​ ​ In both treatment groups, the multiTAA-specific T cell therapy was well tolerated, with no drug-related serious adverse events.
As illustrated below, in the adjuvant lymphoma group, all 17 patients had entered CR, with 14 patients in continued complete remission, or CCR, without relapsing. The duration of response ranged from approximately nine months to over five years. ​ ​ In both treatment groups, the MAR-T cells were well tolerated, with no drug-related serious adverse events.
As of January 2024, the multiTAA-specific T cell therapy was generally well tolerated by the patients across clinical trials in hematological and solid tumor indications, and no treatment-related adverse events, including CRS or neurotoxicity, were attributed to the use of multiTAA-specific T cell therapies to date.
As of January 2025, the MAR-T cell therapy was generally well tolerated by the patients across clinical trials in hematological and solid tumor indications, and no treatment-related adverse events, including neurotoxicity, were attributed to the use of MAR-T cell therapies to date.
There can be no assurance that liability claims will not exceed such insurance coverage limits, which could have a materially adverse effect on our business, financial condition or results of operations or that such insurance will continue to be available on commercially reasonable terms, if at all. Human Resources Employees As of December 31, 2023, we had 8 full-time employees.
There can be no assurance that liability claims will not exceed such insurance coverage limits, which could have a materially adverse effect on our business, financial condition or results of operations or that such insurance will continue to be available on commercially reasonable terms, if at all.
Some of these infused T cells may remain in the body for long periods, providing immunological memory, thus leading to longer and more durable responses. TCRs and CARs have distinct signaling properties and antigen sensitivities.
This leads to a more effective treatment platform with fewer side effects. Some of these infused T cells may remain in the body for long periods, providing immunological memory, thus leading to longer and more durable responses. TCRs and CARs have distinct signaling properties and antigen sensitivities.
In addition, recent government proposals have sought to reduce the 12-year reference product exclusivity period. Other aspects of the BPCIA, some of which may impact the BPCIA exclusivity provisions, have also been the subject of recent litigation.
In addition, recent government proposals have sought to reduce the 12-year reference product exclusivity period. Other aspects of the BPCIA, some of which may impact the BPCIA exclusivity provisions, have also been the subject of recent litigation. As a result, the ultimate impact, implementation, and impact of the BPCIA is subject to significant uncertainty.
The coverage claimed in a patent application can be significantly reduced before the patent is issued. Accordingly, we do not know whether any of the patent applications we own, will acquire, license, or will license will issue as patents, or, if any patents are issued, whether they will provide significant proprietary protection or will be challenged, circumvented or invalidated.
Accordingly, we do not know whether any of the patent applications we own, will acquire, license, or will license will issue as patents, or, if any patents are issued, whether they will provide significant proprietary protection or will be challenged, circumvented or invalidated.
The six antigen targets can be recognized by a very wide range of T cells, which we believe facilitates robust killing of targeted cancer cells. Clinical Development of Our multiTAA-Specific T Cell Therapies by BCM The following clinical trials were conducted by BCM pursuant to our strategic alliance.
The six antigen targets can be recognized by a very wide range of T cells, and we believe that this multi antigen approach facilitates robust killing of targeted cancer cells and minimizes tumor escape. Clinical Development of Our MAR-T Cell Therapies by BCM The following clinical trials were conducted by BCM pursuant to our strategic alliance.
For example, we are assessing the potential of combining multiTAA-specific T cell products with other products. Company-Sponsored Clinical Development of MultiTAA Specific T Cell Therapies MT-601 for the Treatment of Lymphoma We developed MT-601, a multiTAA-specific autologous T cell product capable of recognizing multiple target antigens expressed by the tumor, thereby limiting tumor adaptation by negative antigen selection or downregulation.
Company-Sponsored Clinical Development of MAR-T Cell Therapies MT-601 for the Treatment of Lymphoma We developed MT-601, a MAR-T cell product capable of recognizing multiple target antigens expressed by the tumor, thereby limiting tumor adaptation by negative antigen selection or downregulation.
Our multiTAA-specific T cell drug candidates may compete with product candidates from a number of companies, which are developing various types of immunotherapies to treat cancer, including non-CD19 targeting CAR T cells that target different antigens beyond CD19, multi-targeted CAR T cells as well as NK-CAR therapies.
This highlights a significant unmet medical need for alternative and more effective treatments. Our MAR-T cell drug candidates may compete with product candidates from a number of companies, which are developing various types of immunotherapies to treat cancer, including non-CD19 targeting CAR-T cells that target different antigens beyond CD19, multi-targeted CAR-T cells as well as NK-CAR therapies.
Overall, we have observed a clinical benefit correlated with the detection of tumor-reactive T cells in patient peripheral blood (Arms A, B and C) and within tumor biopsy samples (Arm C) post-infusion.
Overall, we have observed a clinical benefit correlated with the detection of tumor-reactive T cells in patient peripheral blood (Arms A, B and C) and within tumor biopsy samples (Arm C) post-infusion. No cytokine release syndrome or neurotoxicity has been observed in any arm of the trial to date.
Based on the results of the Phase 1 clinical trials of the multiTAA-specific T cell therapies conducted at BCM and the positive clinical data from the Phase 1 APOLLO study, we plan to prioritize the advancement of MT-601 in patients with lymphoma and to advance the MT-401-OTS program in patients with AML. 4 Table of Contents We intend to initiate future additional clinical trials in other tumor types based on emerging data.
Based on the results of the Phase 1 clinical trials of the MAR-T cell therapies conducted at BCM and the positive clinical data from the Phase 1 APOLLO study, we plan to prioritize the advancement of MT-601 in patients with lymphoma and to advance the MT-401-OTS program in patients with AML and MDS.
Each agreement provides that all confidential information developed or made known to the individual during the course of the relationship will be kept confidential and not be disclosed to third parties except in specified circumstances. In the case of employees, the agreements provide that all inventions conceived by an employee shall be our exclusive property.
Each agreement provides that all confidential information developed or made known to the individual during the course of the relationship will be kept confidential and not be disclosed to third parties except in specified circumstances.
Our solution to the problem of tumor heterogeneity is the development of T cell products that are intended to simultaneously attack multiple tumor-expressed antigens and thereby enable more complete initial tumor targeting, thus minimizing the subsequent opportunity for the cancer to engage escape mechanisms. ​ ​ We believe our proprietary multiTAA-specific T cell platform may have meaningful advantages over current CAR and TCR-engineered cell therapy approaches.
Our solution to the problem of tumor heterogeneity is the development of T cell products that are intended to simultaneously attack multiple tumor-expressed antigens and thereby enable more complete initial tumor targeting, thus minimizing the subsequent opportunity for the cancer to engage escape mechanisms.
There were 6 in clinical and 2 were in administrative support. None of our employees is subject to a collective bargaining agreement. We consider our relationship with our employees to be good. Consultants We have consulting agreements with a number of leading academic scientists, clinicians and regulatory experts.
Human Resources Employees As of December 31, 2024, we had 5 full-time employees: 1 clinical and 4 in administrative support. None of our employees is subject to a collective bargaining agreement. We consider our relationship with our employees to be good. Consultants We have consulting agreements with a number of leading academic scientists, clinicians and regulatory experts.
Pursuant to the Cell Ready Purchase Agreement, effective as of the Closing Date, we (i) assigned to Cell Ready the leases for our two manufacturing facilities in Houston, Texas, or the Manufacturing Facilities, (ii) sold to Cell Ready all of the equipment and leasehold improvements at the Manufacturing Facilities and (iii) assigned to Cell Ready our rights, title and interest in any contracts related to the equipment and Manufacturing Facilities (collectively referred to as the “Purchased Assets”).
Subsequently, on June 26, 2023, we completed a transaction with Cell Ready, LLC, or Cell Ready, pursuant to a Purchase Agreement, or the Cell Ready Purchase Agreement, dated May 1, 2023, by and between us and Cell Ready, pursuant to which we (i) assigned to Cell Ready the leases for our two manufacturing facilities in Houston, Texas, or the Manufacturing Facilities, (ii) sold to Cell Ready all of the equipment and leasehold improvements at the Manufacturing Facilities and (iii) assigned to Cell Ready our rights, title and interest in any contracts related to the equipment and Manufacturing Facilities (collectively referred to as the “Purchased Assets”).
As a result, the ultimate impact, implementation, and impact of the BPCIA is subject to significant uncertainty. 24 Table of Contents Other Healthcare Laws and Compliance Requirements Pharmaceutical companies are subject to additional healthcare regulation and enforcement by the federal government and by authorities in the states and foreign jurisdictions in which they conduct their business.
Other Healthcare Laws and Compliance Requirements Pharmaceutical companies are subject to additional healthcare regulation and enforcement by the federal government and by authorities in the states and foreign jurisdictions in which they conduct their business.
Our intellectual property portfolio currently includes patent applications having: (1) claims directed to methods of generating multi-antigen specific T cell products; and (2) claims directed to therapeutic uses of such multi-antigen specific T cell products. We believe our patent portfolio, together with our efforts to develop and patent next-generation technologies, provides us with a substantial intellectual property position.
Our intellectual property portfolio currently includes patent applications having: (1) claims directed to methods of generating multi-antigen specific T cell products; and (2) claims directed to therapeutic uses of such multi-antigen specific T cell products.
We are contracting with Cell Ready to perform a wide variety of services to ensure the continuation of our research and development efforts and process development to optimize our manufacturing process, product quality and commercial scalability.
We continue to contract and collaborate with BCM and others to perform a wide variety of services to ensure the continuation of our research and development efforts, with the goal of optimizing our manufacturing process, product quality and commercial scalability.
The effect of patent expiration on our product candidates also depends upon many other factors such as the nature of the market and the position of the product in it, the growth of the market, the complexities and economics of the process for manufacture of the active ingredient of the product and the requirements of new drug provisions of the Federal Food, Drug and Cosmetic Act or similar laws and regulations in other countries. 17 Table of Contents Our pending patent applications cover a range of technologies, including specific embodiments and applications for treatment of various medical indications, improved application methods and adjunctive utilization with other therapeutic modalities.
The effect of patent expiration on our product candidates also depends upon many other factors such as the nature of the market and the position of the product in it, the growth of the market, the complexities and economics of the process for manufacture of the active ingredient of the product and the requirements of new drug provisions of the Federal Food, Drug and Cosmetic Act or similar laws and regulations in other countries.
For products administered under the supervision of a physician, obtaining coverage and adequate reimbursement may be particularly difficult because of the higher prices often associated with such drugs.
For products administered under the supervision of a physician, obtaining coverage and adequate reimbursement may be particularly difficult because of the higher prices often associated with such drugs. Additionally, separate reimbursement for the product itself or the treatment or procedure in which the product is used may not be available, which may impact physician utilization.
We are evaluating the safety and efficacy of MT-601 in a Phase 1, multicenter, open-label study (APOLLO) in participants with relapsed or refractory lymphoma who either failed or are ineligible for anti-CD19 CAR T cell therapy.
We are evaluating the safety and efficacy of MT-601 in a Phase 1, multicenter, open-label study (APOLLO) in participants with relapsed or refractory lymphoma who either failed or are ineligible for anti-CD19 CAR-T cell therapy. MT-601 is a MAR-T cell product that specifically targets six different tumor antigens upregulated in lymphoma cells (Survivin, PRAME, WT1, NY-ESO-1, SSX-2, MAGEA-4).
Our policy is to seek appropriate patent protection both in the United States and abroad for our proprietary technologies and product candidates.
We protect our technology through various United States and foreign patent filings and maintain trade secrets that we own. Our policy is to seek appropriate patent protection both in the United States and abroad for our proprietary technologies and product candidates.
MT-601 for the Treatment of Pancreatic Cancer We reported interim data for an ongoing Phase 1/2 clinical trial (TACTOPS) of the multiTAA-specific T cell therapy targeting five TAAs for the treatment of pancreatic adenocarcinoma being conducted by BCM.
Data as of September 10, 2024. To further validate these observations, additional patients are currently being enrolled in the Phase 1 study. MT-601 for the Treatment of Pancreatic Cancer We reported interim data for an ongoing Phase 1/2 clinical trial (TACTOPS) of the MAR-T cell therapy targeting five TAAs for the treatment of pancreatic adenocarcinoma being conducted by BCM.
Additionally, separate reimbursement for the product itself or the treatment or procedure in which the product is used may not be available, which may impact physician utilization. 25 Table of Contents In addition, the U.S. government, state legislatures and foreign governments have continued implementing cost-containment programs, including price controls, restrictions on coverage and reimbursement and requirements for substitution of generic products.
In addition, the U.S. government, state legislatures and foreign governments have continued implementing cost-containment programs, including price controls, restrictions on coverage and reimbursement and requirements for substitution of generic products.
No cytokine release syndrome or neurotoxicity has been observed in any arm of the trial to date. ​ 9 Table of Contents Arm A was designed to evaluate the safety and potential efficacy of using multiTAA-specific T cell therapy as part of first-line treatment for patients with pancreatic cancer.
Arm A was designed to evaluate the safety and potential efficacy of using MAR-T cell therapy as part of first-line treatment for patients with pancreatic cancer.
Trademarks We currently have pending with the USPTO applications for registration of the trademarks “Marker Therapeutics.” We also have rights to use other names essential to our business.
In the case of employees, the agreements provide that all inventions conceived by an employee shall be our exclusive property. 18 Table of Contents Trademarks We currently have pending with the USPTO applications for registration of the trademarks “Marker Therapeutics.” We also have rights to use other names essential to our business.
The previous ARTEMIS study was investigating MT-401 (zedenoleucel), in post-allogeneic hematopoietic stem cell transplant (HSCT) patients with AML. The study had three treatment arms, including patients with measurable residual disease (MRD), as well as patients with MRD negative complete remission or active disease.
The study had three treatment arms, including patients with measurable residual disease (MRD), as well as patients with MRD negative complete remission or active disease.
In each trial, correlative studies showed significant expansion of multiTAA-specific T cells, as well as evidence of epitope spreading against tumor-associated antigens that were not targeted by the multiTAA-specific T cell therapy. 8 Table of Contents Acute Myeloid Leukemia To date, available treatments for post-transplant AML patients are limited and include donor lymphocyte infusion, which has an approximately 15% overall response rate but a 30% to 50% risk of severe and debilitating graft-versus-host disease.
Acute Myeloid Leukemia To date, available treatments for post-transplant AML patients are limited and include donor lymphocyte infusion, which has an approximately 15% overall response rate but a 30% to 50% risk of severe and debilitating graft-versus-host disease.
This overlapping structure allows us to isolate, activate and expand any T cell that is specific for any segment of the antigens that we target in the unique genetic background of every patient. 12 Table of Contents The G-Rex is a cell culture device manufactured by Wilson Wolf Manufacturing Corporation, or Wilson Wolf, used by many cell therapy developers, both in commercial and academic settings.
This overlapping structure allows us to isolate, activate and expand any T cell that is specific for any segment of the antigens that we target in the unique genetic background of every patient. 12 Table of Contents Inside the cell culture device, PBMCs, including T cells and antigen-presenting cells, are exposed to the stimulating peptide pools.
Unlike chemotherapy, which is unable to distinguish between healthy and malignant cells, T cells produced for immunotherapy can selectively attack cancer cells that express the target antigen(s). This leads to a more effective treatment platform with fewer side effects.
The cells are then cultured to proliferate, and the proliferated cells are infused into the patient. Upon infusion, the cells can target and eliminate cancerous cells. Unlike chemotherapy, which is unable to distinguish between healthy and malignant cells, T cells produced for immunotherapy can selectively attack cancer cells that express the target antigen(s).
However, the area of patent and other intellectual property rights in biotechnology is an evolving one with many risks and uncertainties. Patents Patents and other proprietary rights are vital to our business operations. We protect our technology through various United States and foreign patent filings and maintain trade secrets that we own.
We believe our patent portfolio, together with our efforts to develop and patent next-generation technologies, provides us with a substantial intellectual property position. However, the area of patent and other intellectual property rights in biotechnology is an evolving one with many risks and uncertainties. Patents Patents and other proprietary rights are vital to our business operations.
However, up to 60% of CD19 CAR T cell treated patients will relapse, particularly in the third line setting (Chong EA et al, N Engl J Med, 2021). This highlights a significant unmet medical need for alternative and more effective treatments.
To date, there are four CD19-directed CAR-T cell therapies (Yescarta, Kymriah, Tecartus, and Bryanzi) 13 Table of Contents approved for patients with relapsed lymphoma. However, up to 60% of CD19 CAR-T cell treated patients will relapse, particularly in the third line setting (Chong EA et al, N Engl J Med, 2021).
T cell therapy, we believe has evolved as one of the most promising branches of immunotherapy. T cell immunotherapy involves the infusion of T cells into a patient. Immune cells used for immunotherapy treatments can either be collected from the patient (autologous) or harvested from a donor (allogeneic).
T cell immunotherapy involves the infusion of T cells into a patient. Immune cells used for immunotherapy treatments can either be collected from the patient (autologous) or harvested from a donor (allogeneic). The cells are retrieved and either genetically modified to express tumor-specific CARs or TCRs or stimulated with specific antigens.
Clinical advancement will be pending additional financial support from non-dilutive grant activities. MT-401 for the Treatment of Patients with AML and MT-401-OTS Program MT-401 ARTEMIS Study: We previously announced discontinuation of the Phase 2 ARTEMIS study to prioritize the MT-401-OTS program in patients with Acute Myeloid Leukemia (AML) and Myelodysplastic Syndromes (MDS).
MT-401 for the Treatment of Patients with AML and MT-401-OTS Program MT-401 ARTEMIS Study: In January 2024, we announced discontinuation of the Phase 2 ARTEMIS study to prioritize the MT-401-OTS program in patients with Acute Myeloid Leukemia (AML) and Myelodysplastic Syndromes (MDS). The previous ARTEMIS study was investigating MT-401 (zedenoleucel), in post-allogeneic hematopoietic stem cell transplant (HSCT) patients with AML.
We strive to create a diverse environment, and our commitment to diversity, equity and inclusion begins with our leadership team of diverse backgrounds and experiences. 100% of our executive officers are women or self-identify as a member of an underrepresented minority group. 27 Table of Contents Corporate Information We were incorporated under the laws of the State of Nevada in 1991 under the name “TapImmune, Inc.” and reincorporated in Delaware in October 2018 under the name “Marker Therapeutics, Inc.” Our principal executive offices are located at 9350 Kirby Drive, Suite 300, Houston, Texas 77054, and our telephone number is (713) 400-6400.
Corporate Information We were incorporated under the laws of the State of Nevada in 1991 under the name “TapImmune, Inc.” and reincorporated in Delaware in October 2018 under the name “Marker Therapeutics, Inc.” Our principal executive offices are located at 2450 Holcombe Blvd, Suite BCM-A, MS: BCM251, Houston, Texas, and our telephone number is (713) 400-6400.

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Item 1A. Risk Factors

Risk Factors — what could go wrong, per management

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Biggest changeIf the FDA or a comparable foreign regulatory authority finds deficiencies with or does not approve these facilities for the manufacture of our product candidates or if it withdraws any such approval in the future, we may need to find alternative manufacturing facilities, which would significantly impact our ability to develop, obtain regulatory approval for or market our product candidates, if approved. ​ Our failure, or the failure of our third-party manufacturers, to comply with applicable regulations could result in sanctions being imposed on us, including clinical holds, fines, injunctions, civil penalties, delays, suspension or withdrawal of approvals, license revocation, seizures or recalls of product candidates or drugs, operating restrictions, and criminal prosecutions, any of which could significantly and adversely affect supply of our products. ​ These factors could cause the delay of clinical trials, regulatory submissions, required approvals or commercialization of our product candidates, cause us to incur higher costs and prevent us from commercializing our products successfully, if approved.
Biggest changeOur failure, or the failure of our third-party manufacturers, to comply with applicable regulations could result in sanctions being imposed on us, including clinical holds, fines, injunctions, civil penalties, delays, suspension or withdrawal of approvals, license revocation, seizures or recalls of product candidates or drugs, operating restrictions, and criminal prosecutions, any of which could significantly and adversely affect supply of our products.
Those factors that could cause fluctuations include, but are not limited to, the following: ● price and volume of fluctuations in the overall stock market from time to time; ● fluctuations in stock market prices and trading volumes of similar companies; ● the thinly traded nature of our common stock; ● actual or anticipated changes in our net loss or fluctuations in our operating results or in the expectations of securities analysts; ● results of our preclinical studies and clinical trials or delays in anticipated timing; ● the issuance of new equity securities pursuant to a future offering, including issuances of preferred stock , or sales of large blocks of our stock and sales by insiders and our institutional investors; ● announcements of new collaboration agreements with strategic partners or developments by our existing collaboration partners; ● announcements of acquisitions, mergers or business combinations; ● competitive developments, including announcements by competitors of new products or services or significant contracts, acquisitions, strategic partnerships, joint ventures or capital commitments; ● general economic conditions and trends, including changes in interest rates, and other national and global conditions, including pandemics and related global economic uncertainty; ● major catastrophic events; ● departures of key personnel; ● events affecting BCM, Cell Ready or any future collaborators; ● announcements of new product candidates or technologies, commercial relationships or other events , including the results of clinical trials, or variations in our quarterly operating results; ● regulatory developments in the United States and other countries , including changes in the structure of healthcare payment systems, or other positive and negative events relating to healthcare and the overall pharmaceutical and biotechnology sectors; ● failure of our common stock to maintain listing requirements on Nasdaq; ● the outcome of any litigation to which we are a party; ● changes in accounting principles; and 62 Table of Contents ● discussion of our company or our stock price by the financial and scientific press and in online investor communities.
Those factors that could cause fluctuations include, but are not limited to, the following: ● price and volume of fluctuations in the overall stock market from time to time; ● fluctuations in stock market prices and trading volumes of similar companies; ● the thinly traded nature of our common stock; ● actual or anticipated changes in our net loss or fluctuations in our operating results or in the expectations of securities analysts; ● results of our preclinical studies and clinical trials or delays in anticipated timing; ● the issuance of new equity securities pursuant to a future offering, including issuances of preferred stock , or sales of large blocks of our stock and sales by insiders and our institutional investors; ● announcements of new collaboration agreements with strategic partners or developments by our existing collaboration partners; ● announcements of acquisitions, mergers or business combinations; ● competitive developments, including announcements by competitors of new products or services or significant contracts, acquisitions, strategic partnerships, joint ventures or capital commitments; ● general economic conditions and trends, including changes in interest rates, and other national and global conditions, including pandemics and related global economic uncertainty; ● major catastrophic events; ● departures of key personnel; ● events affecting BCM, Cell Ready or any future collaborators; 60 Table of Contents ● announcements of new product candidates or technologies, commercial relationships or other events , including the results of clinical trials, or variations in our quarterly operating results; ● regulatory developments in the United States and other countries , including changes in the structure of healthcare payment systems, or other positive and negative events relating to healthcare and the overall pharmaceutical and biotechnology sectors; ● failure of our common stock to maintain listing requirements on Nasdaq; ● the outcome of any litigation to which we are a party; ● changes in accounting principles; and ● discussion of our company or our stock price by the financial and scientific press and in online investor communities.
These risks and uncertainties include the following: ● the USPTO and various foreign governmental patent agencies require compliance with a number of procedural, documentary, fee payment and other provisions during the patent process, the noncompliance with which can result in abandonment or lapse of a patent or patent application, and partial or complete loss of patent rights in the relevant jurisdiction; ● patent applications may not result in any patents being issued; ● patents that may be issued or in-licensed may be challenged, invalidated, modified, revoked, circumvented, found to be unenforceable or otherwise may not provide any competitive advantage; 48 Table of Contents ● our competitors, many of whom have substantially greater resources than us, and many of whom have made significant investments in competing technologies, may seek or may have already obtained patents that will limit, interfere with or eliminate our ability to make, use and sell our potential product candidates; ● there may be significant pressure on the U.S. government and international governmental bodies to limit the scope of patent protection both inside and outside the United States for disease treatments that prove successful, as a matter of public policy regarding worldwide health concerns; and ● countries other than the United States may have patent laws less favorable to patentees than those upheld by U.S. courts, allowing foreign competitors a better opportunity to create, develop and market competing product candidates.
These risks and uncertainties include the following: ● the USPTO and various foreign governmental patent agencies require compliance with a number of procedural, documentary, fee payment and other provisions during the patent process, the noncompliance with which can result in abandonment or lapse of a patent or patent application, and partial or complete loss of patent rights in the relevant jurisdiction; ● patent applications may not result in any patents being issued; ● patents that may be issued or in-licensed may be challenged, invalidated, modified, revoked, circumvented, found to be unenforceable or otherwise may not provide any competitive advantage; ● our competitors, many of whom have substantially greater resources than us, and many of whom have made significant investments in competing technologies, may seek or may have already obtained patents that will limit, interfere with or eliminate our ability to make, use and sell our potential product candidates; ● there may be significant pressure on the U.S. government and international governmental bodies to limit the scope of patent protection both inside and outside the United States for disease treatments that prove successful, as a matter of public policy regarding worldwide health concerns; and ● countries other than the United States may have patent laws less favorable to patentees than those upheld by U.S. courts, allowing foreign competitors a better opportunity to create, develop and market competing product candidates.
Market acceptance of our product candidates, if we receive approval, depends on a number of factors, including the: ● efficacy and safety of our product candidates as demonstrated in clinical trials and post-marketing experience; ● clinical indications for which our product candidates may be approved; ● acceptance by physicians and patients of our product candidates as safe and effective; ● potential and perceived advantages of our product candidates over alternative treatments; ● safety of our product candidates seen in a broader patient group, including our use outside the approved indications should physicians choose to prescribe for such uses; 42 Table of Contents ● prevalence and severity of any side effects; ● product labeling, or product insert requirements of the FDA or other regulatory authorities; ● timing of market introduction of our product candidates as well as competitive products; ● cost in relation to alternative treatments; ● pricing and the availability of coverage and adequate reimbursement by third-party payors and government authorities; ● relative convenience and ease of administration; and ● effectiveness of any sales and marketing efforts.
Market acceptance of our product candidates, if we receive approval, depends on a number of factors, including the: ● efficacy and safety of our product candidates as demonstrated in clinical trials and post-marketing experience; ● clinical indications for which our product candidates may be approved; ● acceptance by physicians and patients of our product candidates as safe and effective; ● potential and perceived advantages of our product candidates over alternative treatments; ● safety of our product candidates seen in a broader patient group, including our use outside the approved indications should physicians choose to prescribe for such uses; ● prevalence and severity of any side effects; ● product labeling, or product insert requirements of the FDA or other regulatory authorities; ● timing of market introduction of our product candidates as well as competitive products; ● cost in relation to alternative treatments; ● pricing and the availability of coverage and adequate reimbursement by third-party payors and government authorities; ● relative convenience and ease of administration; and ● effectiveness of any sales and marketing efforts.
Such laws include: ● the federal Anti-Kickback Statute which prohibits, among other things, persons and entities from knowingly and willfully soliciting, offering, receiving or providing remuneration, directly or indirectly, in cash or in kind, to induce or reward, or in return for, either the referral of an individual for, or the purchase, order or recommendation of, any good or service, for which payment may be made under a federal healthcare program such as Medicare and Medicaid; ● the federal civil and criminal false claims laws, including the federal civil False Claims Act, and civil monetary penalties laws, which impose criminal and civil penalties against individuals or entities for, among other things, knowingly presenting, or causing to be presented, to the federal government, claims for payment that are false or fraudulent or making a false statement to avoid, decrease or conceal an obligation to pay money to the federal government; ● the federal Health Insurance Portability and Accountability Act of 1996, or HIPAA, which imposes criminal and civil liability for, among other things, executing a scheme to defraud any healthcare benefit program or making false statements relating to healthcare matters; ● HIPAA, as amended by the Health Information Technology for Economic and Clinical Health Act, or HITECH, and its implementing regulations, which also imposes obligations, including mandatory contractual terms, on covered entities, including certain healthcare providers, health plans, and healthcare clearinghouses, and their respective business associates that create, receive, maintain or transmit individually identifiable health information for or on behalf of a covered entity as well as their covered subcontractors, with respect to safeguarding the privacy, security and transmission of individually identifiable health information; ● the federal Physician Payments Sunshine Act, which requires certain manufacturers of drugs, devices, biologics and medical supplies for which payment is available under Medicare, Medicaid or the Children’s Health Insurance Program, with specific exceptions, to report annually to CMS, information related to payments or other transfers of value made to physicians (defined to include doctors, dentists, optometrists, podiatrists and chiropractors), other health care professionals)(such as physician assistants and nurse practitioners) and teaching hospitals, as well as information regarding ownership and investment interests held by physicians and their immediate family members; and ● analogous state, local, and foreign laws and regulations, such as state anti-kickback and false claims laws, which may apply to sales or marketing arrangements and claims involving healthcare items or services reimbursed by non-governmental third - party payors, including private insurers; state laws that require pharmaceutical companies to comply with the pharmaceutical industry’s voluntary compliance guidelines and the relevant compliance guidance promulgated by the federal government; state laws that require drug manufacturers to report information related to payments and other transfers of value to physicians and other healthcare providers or marketing expenditures or drug pricing; state and local laws that require the registration of pharmaceutical sales representatives; state and local “drug takeback” laws and regulations; and state and foreign laws governing the privacy and security of health information in some circumstances, many of which differ from each other in significant ways and often are not preempted by HIPAA, thus complicating compliance efforts. 58 Table of Contents Efforts to ensure that our business arrangements will comply with applicable healthcare laws may involve substantial costs.
Such laws include: ● the federal Anti-Kickback Statute which prohibits, among other things, persons and entities from knowingly and willfully soliciting, offering, receiving or providing remuneration, directly or indirectly, in cash or in kind, to induce or reward, or in return for, either the referral of an individual for, or the purchase, order or recommendation of, any good or service, for which payment may be made under a federal healthcare program such as Medicare and Medicaid; ● the federal civil and criminal false claims laws, including the federal civil False Claims Act, and civil monetary penalties laws, which impose criminal and civil penalties against individuals or entities for, among other things, knowingly presenting, or causing to be presented, to the federal government, claims for payment that are false or fraudulent or making a false statement to avoid, decrease or conceal an obligation to pay money to the federal government; 56 Table of Contents ● the federal Health Insurance Portability and Accountability Act of 1996, or HIPAA, which imposes criminal and civil liability for, among other things, executing a scheme to defraud any healthcare benefit program or making false statements relating to healthcare matters; ● HIPAA, as amended by the Health Information Technology for Economic and Clinical Health Act, or HITECH, and its implementing regulations, which also imposes obligations, including mandatory contractual terms, on covered entities, including certain healthcare providers, health plans, and healthcare clearinghouses, and their respective business associates that create, receive, maintain or transmit individually identifiable health information for or on behalf of a covered entity as well as their covered subcontractors, with respect to safeguarding the privacy, security and transmission of individually identifiable health information; ● the federal Physician Payments Sunshine Act, which requires certain manufacturers of drugs, devices, biologics and medical supplies for which payment is available under Medicare, Medicaid or the Children’s Health Insurance Program, with specific exceptions, to report annually to CMS, information related to payments or other transfers of value made to physicians (defined to include doctors, dentists, optometrists, podiatrists and chiropractors), other health care professionals)(such as physician assistants and nurse practitioners) and teaching hospitals, as well as information regarding ownership and investment interests held by physicians and their immediate family members; and ● analogous state, local, and foreign laws and regulations, such as state anti-kickback and false claims laws, which may apply to sales or marketing arrangements and claims involving healthcare items or services reimbursed by non-governmental third - party payors, including private insurers; state laws that require pharmaceutical companies to comply with the pharmaceutical industry’s voluntary compliance guidelines and the relevant compliance guidance promulgated by the federal government; state laws that require drug manufacturers to report information related to payments and other transfers of value to physicians and other healthcare providers or marketing expenditures or drug pricing; state and local laws that require the registration of pharmaceutical sales representatives; state and local “drug takeback” laws and regulations; and state and foreign laws governing the privacy and security of health information in some circumstances, many of which differ from each other in significant ways and often are not preempted by HIPAA, thus complicating compliance efforts.
Later discovery of previously unknown problems with our product candidates, including adverse events of unanticipated severity or frequency, or with our third-party manufacturers or manufacturing processes, or failure to comply with regulatory requirements, may result in, among other things: ● restrictions on the marketing or manufacturing of our product candidates, withdrawal of the product from the market, or voluntary or mandatory product recalls; ● fines, warning letters or holds on clinical trials; ● refusal by the FDA to approve pending applications or supplements to approved applications filed by us or suspension or revocation of license approvals; ● product seizure or detention, or refusal to permit the import or export of our product candidates; and ● injunctions or the imposition of civil or criminal penalties.
Later discovery of previously unknown problems with our product candidates, including adverse events of unanticipated severity or frequency, or with our third-party manufacturers or manufacturing processes, or failure to comply with regulatory requirements, may result in, among other things: ● restrictions on the marketing or manufacturing of our product candidates, withdrawal of the product from the market, or voluntary or mandatory product recalls; ● fines, warning letters or holds on clinical trials; ● refusal by the FDA to approve pending applications or supplements to approved applications filed by us or suspension or revocation of license approvals; ● product seizure or detention, or refusal to permit the import or export of our product candidates; and 55 Table of Contents ● injunctions or the imposition of civil or criminal penalties.
If any of our third-party vendors experience disruptions, or otherwise cease or substantially reduce their operations, our business and operations could be adversely affected. ● Our strategic relationship with BCM is dependent, in part, upon our ongoing relationship with key medical and scientific personnel and advisors. 28 Table of Contents ● Our commercial success depends upon attaining significant market acceptance of our product candidates, if approved, among physicians, patients, healthcare payors and the medical community. ● The biotechnology and immunotherapy industries are characterized by rapid technological developments and a high degree of competition.
If any of our third-party vendors experience disruptions, or otherwise cease or substantially reduce their operations, our business and operations could be adversely affected. ● Our strategic relationship with BCM is dependent, in part, upon our ongoing relationship with key medical and scientific personnel and advisors. ● Our commercial success depends upon attaining significant market acceptance of our product candidates, if approved, among physicians, patients, healthcare payors and the medical community. ● The biotechnology and immunotherapy industries are characterized by rapid technological developments and a high degree of competition.
We and the third parties upon which we rely may be subject to a variety of threats, including, but not limited to, malicious code (such as viruses and worms), social engineering attacks (including through phishing attacks), malware (including as a result of advanced persistent threat intrusions), denial of service attacks (such as credential stuffing), credential harvesting, software bugs, server malfunctions, software or hardware failures, unauthorized access, natural disasters, fire, terrorism, successful breaches, personnel misconduct or error, or human or technological error, war and telecommunication and electrical failures.
We and the third parties upon which we rely may be subject to a variety of threats, including, but not limited to, malicious code (such as viruses and worms), social engineering attacks (including through phishing attacks), malware (including as a result of advanced persistent threat intrusions), denial of service attacks (such as credential stuffing), credential harvesting, software bugs, server malfunctions, software or hardware failures, unauthorized access, natural disasters, 62 Table of Contents fire, terrorism, successful breaches, personnel misconduct or error, or human or technological error, war and telecommunication and electrical failures.
In the event of a major hurricane or other serious weather event or catastrophic event such as fire, power loss, cyberattack, war, terrorist attack or epidemic or pandemic that impacts the facilities of any third parties on which we may rely, we may be unable to continue our operations and may experience delays in our manufacturing process and shipment of clinical supply to trial sites or interruptions in our clinical trials and research activities, all of which could delay our development plans and materially harm our business, results of operations and prospects.
In the event of a major hurricane or other serious weather event or catastrophic event such as fire, power loss, cyberattack, war, terrorist attack or epidemic or pandemic that impacts the facilities of any third parties on which we may rely, we may be unable to continue our 64 Table of Contents operations and may experience delays in our manufacturing process and shipment of clinical supply to trial sites or interruptions in our clinical trials and research activities, all of which could delay our development plans and materially harm our business, results of operations and prospects.
Although we have an exclusive license agreement with BCM under which we received a worldwide, exclusive license to BCM’s rights in and to three patent families to develop and commercialize the multiTAA-specific T cell product candidates, we will need to enter into additional agreements with BCM with respect to (i) a strategic alliance to advance preclinical research, early stage clinical trials, and clinical trials with respect to our product candidates, as well as continued access to our clinical data, and (ii) support, including personnel and space at the institution for the foreseeable future.
Although we have an exclusive license agreement with BCM under which we received a worldwide, exclusive license to BCM’s rights in and to three patent families to develop and commercialize the MAR-T cell product candidates, we will need to enter into additional agreements with BCM with respect to (i) a strategic alliance to advance preclinical research, early stage clinical trials, and clinical trials with respect to our product candidates, as well as continued access to our clinical data, and (ii) support, including personnel and space at the institution for the foreseeable future.
In cooperation with our current contract manufacturers and suppliers, we developed and have implemented a new nine-day multiTAA-specific T cell manufacturing process for our current as well as future clinical trials using a patient-specific manufacturing approach or using products manufactured from healthy donors (“Off-the-Shelf”).
In cooperation with our current contract manufacturers and suppliers, we developed and have implemented a new nine-day MAR-T cell manufacturing process for our current as well as future clinical trials using a patient-specific manufacturing approach or using products manufactured from healthy donors (“Off-the-Shelf”).
Any of the previously identified or similar threats could cause a security incident or other incident during which our information technology systems or data could be compromised, which could result in unauthorized, unlawful, or accidental acquisition, modification, destruction, loss, alteration, encryption, disclosure 64 Table of Contents of, or access to our data; it could also disrupt our ability (and that of third parties upon which we rely) to operate our business, including conducting our clinical trials.
Any of the previously identified or similar threats could cause a security incident or other incident during which our information technology systems or data could be compromised, which could result in unauthorized, unlawful, or accidental acquisition, modification, destruction, loss, alteration, encryption, disclosure of, or access to our data; it could also disrupt our ability (and that of third parties upon which we rely) to operate our business, including conducting our clinical trials.
If we are unable to conclude that our internal control over financial reporting is effective, or if our independent registered public accounting firm determines we have a material weakness or significant deficiency in our internal control over financial reporting, we could lose investor confidence in the accuracy and completeness of our financial reports, the market price of our common 65 Table of Contents stock could decline, and we could be subject to sanctions or investigations by the Nasdaq, the SEC or other regulatory authorities.
If we are unable to conclude that our internal control over financial reporting is effective, or if our independent registered public accounting firm determines we have a material weakness or significant deficiency in our internal control over financial reporting, we could lose investor confidence in the accuracy and completeness of our financial reports, the market price of our common stock could decline, and we could be subject to sanctions or investigations by the Nasdaq, the SEC or other regulatory authorities.
Generally, a product that has orphan drug designation and subsequently receives the first FDA approval for the disease for which it has such designation is entitled to orphan drug exclusive approval (or exclusivity), which means that the FDA may not approve any other applications to market the same drug or biologic for the same indication for seven years, except in limited circumstances, such as a showing of clinical superiority to the product with orphan drug exclusivity.
Generally, a product that has orphan drug designation and subsequently receives the first FDA approval for the disease for which it has such designation is entitled to orphan drug exclusive approval (or exclusivity), which means that the FDA may not approve any other applications to market the same drug or biologic for the same indication for seven years, except 35 Table of Contents in limited circumstances, such as a showing of clinical superiority to the product with orphan drug exclusivity.
General Risk Factors If our information technology systems or data, or those of third parties upon which we rely, are or were compromised, or are perceived to have been compromised, we could experience adverse consequences, including but not limited to regulatory investigations or actions; litigation; fines and penalties; disruptions of our business operations; reputational harm; loss of revenue or profits; loss of customers or sales; and other adverse consequences.
If our information technology systems or data, or those of third parties upon which we rely, are or were compromised, or are perceived to have been compromised, we could experience adverse consequences, including but not limited to regulatory investigations or actions; litigation; fines and penalties; disruptions of our business operations; reputational harm; loss of revenue or profits; loss of customers or sales; and other adverse consequences.
In addition, certain of our immunotherapies may be subject to competition from investigational new drugs and/or products developed using other technologies, some of which have completed numerous clinical trials. 45 Table of Contents The market opportunities for our product candidates may be limited to those patients who are ineligible for or have failed prior treatments and may be small.
In addition, certain of our immunotherapies may be subject to competition from investigational new drugs and/or products developed using other technologies, some of which have completed numerous clinical trials. The market opportunities for our product candidates may be limited to those patients who are ineligible for or have failed prior treatments and may be small.
Additionally, these obligations may be subject to differing applications and interpretations, which may be inconsistent or conflict among jurisdictions. Preparing for and complying with these obligations requires us to devote significant resources, which may necessitate changes to our services, information technologies, systems, and practices and to those of any third parties that process personal data on our behalf.
Additionally, these obligations may be subject to differing applications and interpretations, which may be inconsistent or conflict among jurisdictions. 59 Table of Contents Preparing for and complying with these obligations requires us to devote significant resources, which may necessitate changes to our services, information technologies, systems, and practices and to those of any third parties that process personal data on our behalf.
In addition, it is uncertain if and to what extent various states will conform to federal tax legislation. The impact of such changes or future legislation could increase our U.S. tax expense and could have a material adverse impact on our business and financial condition. ​ ITEM 1B. UNRESOLVED STAFF COMMENTS None. ​ 67 Table of Contents
In addition, it is uncertain if and to what extent various states will conform to federal tax legislation. The impact of such changes or future legislation could increase our U.S. tax expense and could have a material adverse impact on our business and financial condition. ​ ITEM 1B. UNRESOLVED STAFF COMMENTS None.
Because our autologous multiTAA-specific T cell therapy-based product candidates, MT-601, is manufactured for each particular patient, we will be required to maintain a chain of identity with respect to the patient’s blood cells as it moves from the patient to the manufacturing facility, through the manufacturing process, and back to the patient.
Because our autologous MAR-T cell therapy-based product candidates, MT-601, is manufactured for each particular patient, we will be required to maintain a chain of identity with respect to the patient’s blood cells as it moves from the patient to the manufacturing facility, through the manufacturing process, and back to the patient.
If we are unsuccessful in securing such strategic collaborations, we may be unable to commercialize any approved products as we have not yet licensed, marketed or sold any of our immunotherapies or entered into successful collaborations for these services in order to ultimately commercialize our immunotherapies. Establishing strategic collaborations is difficult and time- 41 Table of Contents consuming.
If we are unsuccessful in securing such strategic collaborations, we may be unable to commercialize any approved products as we have not yet licensed, marketed or sold any of our immunotherapies or entered into successful collaborations for these services in order to ultimately commercialize our immunotherapies. Establishing strategic collaborations is difficult and time-consuming.
In addition, regulatory agencies may lack experience with our technologies and product candidates, which may lengthen the regulatory review process, increase our development costs and delay or prevent their commercialization. No adoptive T cell therapy using multiTAA-specific T cells has been approved for marketing in the U.S. by the FDA.
In addition, regulatory agencies may lack experience with our technologies and product candidates, which may lengthen the regulatory review process, increase our development costs and delay or prevent their commercialization. No adoptive T cell therapy using MAR-T cells has been approved for marketing in the U.S. by the FDA.
The combination of healthcare cost containment measures, increased health insurance costs, reduction of the number of people with health insurance coverage, as well as future legislation and regulations focused on reducing healthcare costs by reducing the cost of, or reimbursement and access to, pharmaceutical products, may limit or delay our ability to commercialize our products, generate revenue or attain profitability.
The combination of healthcare cost containment measures, increased health insurance costs, reduction of the number of people with health insurance coverage, as well as future legislation and regulations focused on reducing healthcare costs by reducing the cost of, or 58 Table of Contents reimbursement and access to, pharmaceutical products, may limit or delay our ability to commercialize our products, generate revenue or attain profitability.
Moreover, any of these events could prevent us from achieving or maintaining market acceptance of the particular product candidate, if approved, and could significantly harm our business, results of operations and prospects. 35 Table of Contents We may not obtain or maintain the benefits associated with orphan drug designation, including market exclusivity.
Moreover, any of these events could prevent us from achieving or maintaining market acceptance of the particular product candidate, if approved, and could significantly harm our business, results of operations and prospects. We may not obtain or maintain the benefits associated with orphan drug designation, including market exclusivity.
If government and other health care payors were not to provide adequate coverage and reimbursement levels for any of our products if approved, market acceptance and commercial success would be reduced. Our multiTAA-specific T cell therapy may be provided to patients in combination with other agents provided by third parties.
If government and other health care payors were not to provide adequate coverage and reimbursement levels for any of our products if approved, market acceptance and commercial success would be reduced. Our MAR-T cell therapy may be provided to patients in combination with other agents provided by third parties.
For products administered under the supervision of a physician, obtaining coverage and adequate reimbursement may be particularly difficult because of the higher prices 43 Table of Contents often associated with such drugs. Additionally, separate reimbursement for the product itself or the treatment or procedure in which the product is used may not be available, which may impact physician utilization.
For products administered under the supervision of a physician, obtaining coverage and adequate reimbursement may be particularly difficult because of the higher prices often associated with such drugs. Additionally, separate reimbursement for the product itself or the treatment or procedure in which the product is used may not be available, which may impact physician utilization.
In addition, the loss of any current or future licenses or the exclusivity rights provided therein could materially harm our business financial condition and our operations. Risks Related to Government Regulation We are subject to extensive regulation, which can be costly, time consuming and can subject us to unanticipated delays.
In addition, the loss of any current or future licenses or the exclusivity rights provided therein could materially harm our business financial condition and our operations. 54 Table of Contents Risks Related to Government Regulation We are subject to extensive regulation, which can be costly, time consuming and can subject us to unanticipated delays.
Our multiTAA-specific T cell drug candidates may compete with product candidates from a number of companies, which are developing various types of immunotherapies to treat cancer, including non-CD19 targeting CAR T cells that target different antigens beyond CD19, multi-targeted CAR T cells as well as NK-CAR therapies.
Our MAR-T cell drug candidates may compete with product candidates from a number of companies, which are developing various types of immunotherapies to treat cancer, including non-CD19 targeting CAR-T cells that target different antigens beyond CD19, multi-targeted CAR-T cells as well as NK-CAR therapies.
Certain of our technologies are in-licensed from third parties, and the protection of those technologies is not entirely within our control. We have a world-wide exclusive license from BCM of the rights in and to three patent families to develop and commercialize multiTAA-specific T cell product candidates in the field of oncology.
Certain of our technologies are in-licensed from third parties, and the protection of those technologies is not entirely within our control. We have a world-wide exclusive license from BCM of the rights in and to three patent families to develop and commercialize MAR-T cell product candidates in the field of oncology.
There have been judicial, Congressional and executive branch challenges to certain aspects of the ACA. For example, Congress considered legislation that would repeal or repeal and replace all or part of the ACA. While Congress has not passed repeal legislation, several bills affecting the implementation of certain taxes under the ACA have been signed into law.
There have been judicial, Congressional and executive branch challenges to certain aspects of the ACA. For example, Congress considered legislation that would 57 Table of Contents repeal or repeal and replace all or part of the ACA. While Congress has not passed repeal legislation, several bills affecting the implementation of certain taxes under the ACA have been signed into law.
The success of our multiTAA product candidates or any other product candidates that we develop or otherwise may acquire will depend on several factors, including: ● timely and successful completion of preclinical studies, including toxicology studies, biodistribution studies and minimally efficacious dose studies in animals, where applicable, and clinical trials; ● effective investigational new drug applications, or INDs, from the FDA or comparable foreign applications that allow commencement of our planned clinical trials or future clinical trials for our product candidates; ● sufficiency of our financial and other resources to complete the necessary preclinical studies and clinical trials; 30 Table of Contents ● successful enrollment and completion of clinical trials, including under the FDA’s current Good Clinical Practices, or GCPs, and current Good Laboratory Practices; ● successful development of, or making arrangements with third-party manufacturers for, our commercial manufacturing processes for any of our product candidates that receive regulatory approval; ● receipt of timely marketing approvals from applicable regulatory authorities; ● launching commercial sales of products, if approved, whether alone or in collaboration with others; ● acceptance of the benefits and use of our products, including method of administration, if approved, by patients, the medical community and third-party payors, for their approved indications; ● the prevalence and severity of adverse events experienced by our product candidates; ● the availability, perceived advantages, cost, safety and efficacy of alternative therapies for any product candidate, and any indications for such product candidate, that we develop; ● our ability to produce any product candidates we develop on a commercial scale; ● obtaining and maintaining patent, trademark and trade secret protection and regulatory exclusivity for our product candidates and otherwise protecting our rights in our intellectual property portfolio; ● maintaining compliance with regulatory requirements, including the FDA’s current Good Manufacturing Practices, or cGMPs, and complying effectively with other procedures; ● obtaining and maintaining third-party coverage and adequate reimbursement and patients’ willingness to pay out-of-pocket in the absence of such coverage and adequate reimbursement; and ● maintaining a continued acceptable safety, tolerability and efficacy profile of the products following approval.
The success of our MAR-T cell product candidates or any other product candidates that we develop or otherwise may acquire will depend on several factors, including: ● timely and successful completion of preclinical studies, including toxicology studies, biodistribution studies and minimally efficacious dose studies in animals, where applicable, and clinical trials; ● effective investigational new drug applications, or INDs, from the FDA or comparable foreign applications that allow commencement of our planned clinical trials or future clinical trials for our product candidates; ● sufficiency of our financial and other resources to complete the necessary preclinical studies and clinical trials; ● successful enrollment and completion of clinical trials, including under the FDA’s current Good Clinical Practices, or GCPs, and current Good Laboratory Practices; ● successful development of, or making arrangements with third-party manufacturers for, our commercial manufacturing processes for any of our product candidates that receive regulatory approval; 30 Table of Contents ● receipt of timely marketing approvals from applicable regulatory authorities; ● launching commercial sales of products, if approved, whether alone or in collaboration with others; ● acceptance of the benefits and use of our products, including method of administration, if approved, by patients, the medical community and third-party payors, for their approved indications; ● the prevalence and severity of adverse events experienced by our product candidates; ● the availability, perceived advantages, cost, safety and efficacy of alternative therapies for any product candidate, and any indications for such product candidate, that we develop; ● our ability to produce and manufacture our product candidates, which is dependent on third-party vendors and their willingness to support our manufacturing and cell processing; ● obtaining and maintaining patent, trademark and trade secret protection and regulatory exclusivity for our product candidates and otherwise protecting our rights in our intellectual property portfolio; ● maintaining compliance with regulatory requirements, including the FDA’s current Good Manufacturing Practices, or cGMPs, and complying effectively with other procedures; ● obtaining and maintaining third-party coverage and adequate reimbursement and patients’ willingness to pay out-of-pocket in the absence of such coverage and adequate reimbursement; and ● maintaining a continued acceptable safety, tolerability and efficacy profile of the products following approval.
Although we have patents and patent applications in other countries, we cannot be certain that the claims in other pending U.S. or European patent applications, international patent applications, and patent applications in certain other foreign territories directed to methods of generating multi-antigen specific T cell product candidates, or our other product candidates, will be considered patentable by the USPTO, courts in the United States or by the patent offices and courts in foreign countries, nor can we be certain that the claims in our issued licensed European patent will not be found invalid or unenforceable if challenged.
Although we have patents and patent applications in other countries, we cannot be certain that the claims in other pending U.S. or European patent applications, international patent applications, and patent applications in certain other foreign territories directed to methods of generating MAR-T cell product candidates, or our other product candidates, will be considered patentable by the USPTO, courts in the United States or by the patent offices and courts in foreign countries, nor can we be certain that the claims in our issued licensed European patent will not be found invalid or unenforceable if challenged.
Any such delay may lower our revenues and potential profitability. If any 36 Table of Contents third-party breaches or terminates its agreement with us or fails to conduct its activities in a timely manner, the commercialization of our product candidates could be slowed down or blocked completely.
Any such delay may lower our revenues and potential profitability. If any third-party breaches or terminates its agreement with us or fails to conduct its activities in a timely manner, the commercialization of our product candidates could be slowed down or blocked completely.
Our product candidates are biologics, and the process of manufacturing our product candidates is complex, highly regulated and subject to multiple risks. For example, the manufacture of our multiTAA-specific T cell therapy-based product candidates involves complex processes, including drawing blood from patients/donors, manufacturing the clinical product, and ultimately infusing the product into a patient.
Our product candidates are biologics, and the process of manufacturing our product candidates is complex, highly regulated and subject to multiple risks. For example, the manufacture of our MAR-T cell therapy-based product candidates involves complex processes, including drawing blood from patients/donors, manufacturing the clinical product, and ultimately infusing the product into a patient.
In addition, upon a liquidity event (as defined in our BCM license agreement with BCM) of the licensee under the BCM license agreement (which, the licensee shall be the Company), BCM will receive a liquidity incentive payment of 0.5% of the liquidity event 52 Table of Contents proceeds (as defined in the BCM license agreement) received by such licensee or its stockholders in the liquidity event, thereby diluting the amount of proceeds available to the licensee or its stockholders in a liquidity event.
In addition, upon a liquidity event (as defined in our BCM license agreement with BCM) of the licensee under the BCM license agreement (which, the licensee shall be the Company), BCM will receive a liquidity incentive payment of 0.5% of the liquidity event proceeds (as defined in the BCM license agreement) received by such licensee or its stockholders in the liquidity event, thereby diluting the amount of proceeds available to the licensee or its stockholders in a liquidity event.
If our stock price rises, the holders may exercise their warrants and options and sell a large number of shares. This could cause the market price of our common stock to decline and cause existing stockholders to experience significant further dilution. 63 Table of Contents We do not intend to pay cash dividends.
If our stock price rises, the holders may exercise their warrants and options and sell a large number of shares. This could cause the market price of our common stock to decline and cause existing stockholders to experience significant further dilution. We do not intend to pay cash dividends.
The lengthy approval process, as well as the unpredictability of future clinical trial results, may result in us failing to obtain regulatory approval for our product candidates, which would materially harm our business, results of operations and prospects. 33 Table of Contents The results of earlier preclinical and clinical trials may not be predictive of future clinical trial results.
The lengthy approval process, as well as the unpredictability of future clinical trial results, may result in us failing to obtain regulatory approval for our product candidates, which would materially harm our business, results of operations and prospects. The results of earlier preclinical and clinical trials may not be predictive of future clinical trial results.
A conflict of interest also may arise concerning the timing and scope of the parties’ planned and ongoing clinical trials, investigational new drug application filings and the parties’ opportunities for marketing their respective product candidates, as well as 47 Table of Contents our intellectual property rights with those of Allovir.
A conflict of interest also may arise concerning the timing and scope of the parties’ planned and ongoing clinical trials, investigational new drug application filings and the parties’ opportunities for marketing their respective product candidates, as well as our intellectual property rights with those of Allovir.
The cost of such combination therapy may increase the overall cost of multiTAA-specific T cell therapy and may result in issues regarding the allocation of reimbursements between our therapy and the other agents, all of which may adversely affect our ability to obtain reimbursement coverage for the combination therapy from third-party medical insurers.
The cost of such combination therapy may increase the overall cost of MAR-T cell therapy and may result in issues regarding the allocation of reimbursements between our therapy and the other agents, all of which may adversely affect our ability to obtain reimbursement coverage for the combination therapy from third-party medical insurers.
While these universities and public and private research institutions primarily have educational objectives, they may develop proprietary technologies that lead to other FDA approved therapies or that secure patent protection that we may need for the development of our technologies and product candidates.
While these universities and public and private research institutions 44 Table of Contents primarily have educational objectives, they may develop proprietary technologies that lead to other FDA approved therapies or that secure patent protection that we may need for the development of our technologies and product candidates.
However, damage and claims arising from such incidents may not be covered or may exceed the amount of any insurance available. Additionally, we cannot be sure that such coverage will continue to be available on commercially reasonable terms or at all, or that such coverage will pay future claims.
However, damage and claims arising from such incidents may not be covered or may exceed the amount of any insurance available. Additionally, we cannot 63 Table of Contents be sure that such coverage will continue to be available on commercially reasonable terms or at all, or that such coverage will pay future claims.
For example, on March 10, 2023, Silicon Valley Bank, or SVB, was closed by the California Department of Financial Protection and Innovation, which appointed the Federal Deposit Insurance Corporation, or the FDIC, as receiver. Although a statement by the U.S.
For example, on March 10, 2023, Silicon 29 Table of Contents Valley Bank, or SVB, was closed by the California Department of Financial Protection and Innovation, which appointed the Federal Deposit Insurance Corporation, or the FDIC, as receiver. Although a statement by the U.S.
In addition, these side effects may not be appropriately recognized or managed by the treating medical staff, as toxicities resulting from personalized cell therapy, as with our multiTAA-specific T cell therapy products, are not normally encountered in the general patient population and by medical personnel.
In addition, these side effects may not be appropriately recognized or managed by the treating medical staff, as toxicities resulting from personalized cell therapy, as with our MAR-T cell therapy products, are not normally encountered in the general patient population and by medical personnel.
Our inability to license the rights and technologies that we have identified, or newly developed multiTAA-specific T cell technology that we may in the future identify, could have a material adverse impact on our ability to complete the development of our product candidates or to develop additional product candidates.
Our inability to license the rights and technologies that we have identified, or newly developed MAR-T cell technology that we may in the future identify, could have a material adverse impact on our ability to complete the development of our product candidates or to develop additional product candidates.
The patent application process is subject to numerous risks and uncertainties, and there can be no assurance that we or any of our potential future collaborators will be successful in protecting our product candidates by obtaining and defending patents.
The patent application process is subject to numerous risks and uncertainties, and 47 Table of Contents there can be no assurance that we or any of our potential future collaborators will be successful in protecting our product candidates by obtaining and defending patents.
Under the unitary patent system, European applications will have the option, upon grant of a patent, of becoming a Unitary Patent which will be subject to the jurisdiction of the Unitary Patent Court, or UPC. As the UPC is a new court system, there is no precedent for the court, increasing the uncertainty of any litigation.
Under the unitary patent system, European applications will have the option, upon grant of a patent, of 50 Table of Contents becoming a Unitary Patent which will be subject to the jurisdiction of the Unitary Patent Court, or UPC. As the UPC is a new court system, there is no precedent for the court, increasing the uncertainty of any litigation.
We will have to conduct larger, well-controlled trials in our proposed indications at multiple sites to verify the results obtained to date and to support any regulatory submissions for further clinical development of our product candidates.
We will have 33 Table of Contents to conduct larger, well-controlled trials in our proposed indications at multiple sites to verify the results obtained to date and to support any regulatory submissions for further clinical development of our product candidates.
The new manufacturing process marks additional manufacturing 38 Table of Contents improvements compared to the processes used in the BCM Phase 1 and 2 trials (36-day manufacturing time) and our previous AML trial (20-day manufacturing time). The new nine-day manufacturing process enables increased antigen specificity and diversity, which has exhibited a strong linear correlation to anti-tumor activity in vitro.
The new manufacturing process marks additional manufacturing improvements compared to the processes used in the BCM Phase 1 and 2 trials (36-day manufacturing time) and our previous AML trial (20-day manufacturing time). The new nine-day manufacturing process enables increased antigen specificity and diversity, which has exhibited a strong linear correlation to anti-tumor activity in vitro.
It is possible that third parties relied upon by us will change their strategic focus, pursue alternative technologies, or develop alternative product candidates, either on their own or in collaboration with others, as a means for developing treatments for the diseases targeted by our collaborative programs, or for other reasons.
It is possible that third parties relied upon by us will change their strategic focus, pursue alternative technologies, or develop alternative product candidates, either on their own or in collaboration 39 Table of Contents with others, as a means for developing treatments for the diseases targeted by our collaborative programs, or for other reasons.
It is also unclear how such challenges and the healthcare reform measures of the Biden administration will impact the ACA and our business. 59 Table of Contents In addition, other federal health reform measures have been proposed and adopted in the United States.
It is also unclear how such challenges and the healthcare reform measures of the Biden administration will impact the ACA and our business. In addition, other federal health reform measures have been proposed and adopted in the United States.
In particular, because our product candidates are subject to regulation as biological products, 32 Table of Contents we will need to demonstrate that they are safe, pure and potent for use in their target indications. Each product candidate must demonstrate an adequate risk versus benefit profile in its intended patient population and for its intended use.
In particular, because our product candidates are subject to regulation as biological products, we will need to demonstrate that they are safe, pure and potent for use in their target indications. Each product candidate must demonstrate an adequate risk versus benefit profile in its intended patient population and for its intended use.
We depend on a limited number of vendors for supply of certain materials and equipment used in the manufacture of our multiTAA-specific T cell therapy-based product candidates. For example, in the past, we purchased equipment and reagents critical for the manufacture of our product candidates from Wilson Wolf (a company controlled by our director John Wilson), Almac and other suppliers.
We depend on a limited number of vendors for supply of certain materials and equipment used in the manufacture of our MAR-T cell therapy-based product candidates. For example, in the past, we purchased equipment and reagents critical for the manufacture of our product candidates from Wilson Wolf (a company controlled by our former director, John Wilson), Almac and other suppliers.
If our contract manufacturing organizations, or CMOs, ,including Cell Ready, are unable to reliably produce products to specifications acceptable to the FDA or other regulatory authorities, we may not obtain or maintain the approvals we need to commercialize any approved products.
If our contract manufacturing organizations, or CMOs, are unable to reliably produce products to specifications acceptable to the FDA or other regulatory authorities, we may not obtain or maintain the approvals we need to commercialize any approved products.
If we are unable to raise additional funds through equity or debt financings when needed, we may be required to delay, limit, reduce or terminate our drug development or future commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves.
If we are unable to raise additional funds through equity or debt 61 Table of Contents financings when needed, we may be required to delay, limit, reduce or terminate our drug development or future commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves.
In particular, we have made the strategic decision to dispose of our manufacturing facilities and related assets in order to focus on clinical development of the multiTAA-specific T cell therapy-based product candidates in our pipeline.
In particular, we have made the strategic decision to dispose of our manufacturing facilities and related assets in order to focus on clinical development of the MAR-T cell therapy-based product candidates in our pipeline.
If we are unable to contract for a sufficient supply of needed materials on acceptable terms, or if we should encounter delays or difficulties in our relationships with manufacturers, our clinical trials may be delayed, thereby delaying the submission of product candidates for regulatory approval or the market introduction and subsequent sales of any approved products.
If we are unable to contract for a sufficient supply of needed materials on acceptable terms, or if we should encounter delays, difficulties or disputes with regard to in our relationships with manufacturers, our clinical trials may be delayed, 36 Table of Contents thereby delaying the submission of product candidates for regulatory approval or the market introduction and subsequent sales of any approved products.
Dr. Vera is a co-founder and director of Allovir Inc., or Allovir. Allovir has technology which is being developed under a license agreement with BCM by the same research group at BCM. Allovir is a clinical-stage biopharmaceutical company that is investigating and developing virus-specific T cell therapy technology for the prevention and/or treatment of viral infections. Further, Dr.
Allovir has technology which is being developed under a license agreement with BCM by the same research group at BCM. Allovir is a clinical-stage biopharmaceutical company that is investigating and developing virus-specific T cell therapy technology for the prevention and/or treatment of viral infections. Further, Dr.
No assurance can be given that we will be able to develop a new, FDA-compliant, more efficient, lower cost manufacturing process upon which our business plan to commercialize multiTAA-based product candidates is dependent.
No assurance can be given that we will be able to develop a new, FDA-compliant, more efficient, lower cost manufacturing process upon which our business plan to commercialize MAR-T cell-based product candidates is dependent.
Even if we are successful in defending against these claims, litigation could result in substantial cost and be a distraction to our management and employees.
Litigation may be necessary to defend against these claims. Even if we are successful in defending against these claims, litigation could result in substantial cost and be a distraction to our management and employees.
Under our license agreement with BCM for our multiTAA-specific T cell therapy technologies, we are currently required to pay both substantial milestone payments and royalties to BCM based on our revenues from sales of any approved products utilizing the licensed technologies, and these payments could adversely affect the overall profitability for us of any products that we may seek to commercialize.
Under our license agreement with BCM for our MAR-T cell therapy technologies, we are currently required to pay both substantial milestone payments and royalties to BCM based on our revenues from sales of any approved products utilizing the licensed technologies, 51 Table of Contents and these payments could adversely affect the overall profitability for us of any products that we may seek to commercialize.
If these facilities cannot pass a pre-approval plant inspection, the FDA premarket approval of our product candidates will not be granted.
If these facilities cannot pass a pre-approval plant inspection, the FDA 38 Table of Contents premarket approval of our product candidates will not be granted.
We own or hold licenses to a number of issued patents and U.S. pending patent applications, as well as foreign patents and foreign counterparts.
We own or hold licenses to a number of 52 Table of Contents issued patents and U.S. pending patent applications, as well as foreign patents and foreign counterparts.
Without an internal commercial organization or the support of a third - party to perform sales and marketing functions, we may be unable to compete successfully against these more established companies. 44 Table of Contents The biotechnology and immunotherapy industries are characterized by rapid technological developments and a high degree of competition.
Without an internal commercial organization or the support of a third - party to perform sales and marketing functions, we may be unable to compete successfully against these more established companies. The biotechnology and immunotherapy industries are characterized by rapid technological developments and a high degree of competition. We may be unable to compete with more substantial enterprises.
We are highly dependent on our management, scientific, and medical personnel and consultants, including Juan Vera, M.D., our President, Chief Executive Officer, and Principal Financial and Accounting Officer, as well as others.
We are highly dependent on our management, scientific, and medical personnel and consultants, including Juan Vera, M.D., our President, Chief Executive Officer, and Principal Financial and 43 Table of Contents Accounting Officer, as well as others.
Third-party manufacturers may not be able to meet our needs concerning timing, quantity, or quality. Additionally, Cell Ready may engage, be engaged by, or otherwise enter into arrangements with our competitors.
Third-party manufacturers may not be able to meet our needs concerning timing, quantity, or quality. Additionally, our third-party manufacturing partners may engage, be engaged by, or otherwise enter into arrangements with our competitors.
Even if we generate revenue in the future, we may not be able to become profitable or sustain profitability in subsequent periods. These and other factors raise substantial doubt regarding our ability to continue as a going concern, which may create negative reactions to the price of our common stock.
Even if we generate revenue in the future, we may not be able to become profitable or sustain profitability in subsequent periods. The substantial doubt regarding our ability to continue as a going concern may create negative reactions to the price of our common stock.
Clinical trials are expensive and difficult to design and implement, in part because they are subject to rigorous regulatory requirements. Because our product candidates are based on new technologies and manufactured on a patient-by-patient basis for our multiTAA-specific T cell product candidates we expect that they will have substantial manufacturing costs.
Clinical trials are expensive and difficult to design and implement, in part because they are subject to rigorous regulatory requirements. Because our product candidates are based on new technologies and manufactured on a patient-by-patient basis for our MAR-T cell 32 Table of Contents product candidates we expect that they will have substantial manufacturing costs.
If any of our third-party suppliers, including Cell Ready, encounter such difficulties, the supply of our product candidates for clinical trials, or our product candidates for patients, if approved, could be delayed or stopped, or we may be unable to maintain a commercially viable cost structure.
If any of our third-party suppliers encounter such difficulties, the supply of our product candidates for clinical 37 Table of Contents trials, or our product candidates for patients, if approved, could be delayed or stopped, or we may be unable to maintain a commercially viable cost structure.
The listing of our common stock on Nasdaq does not assure that a meaningful, consistent and liquid trading market currently exists or will exist in the future. The trading price of our common stock may fluctuate substantially.
Risks Related to our Securities The price of our stock may be volatile. The listing of our common stock on Nasdaq does not assure that a meaningful, consistent and liquid trading market currently exists or will exist in the future. The trading price of our common stock may fluctuate substantially.
Efforts to ensure that our business arrangements comply with applicable healthcare laws may involve substantial costs. It is possible that governmental and enforcement authorities will conclude that our business practices may not comply with current or future statutes, regulations or case law interpreting applicable fraud and abuse or other healthcare laws and regulations.
It is possible that governmental and enforcement authorities will conclude that our business practices may not comply with current or future statutes, regulations or case law interpreting applicable fraud and abuse or other healthcare laws and regulations.
The effectiveness of these third parties in marketing their own products may also affect our revenues and earnings. We intend to continue to enter into additional third-party agreements in the future. However, we may not be able to negotiate any additional agreements successfully.
The effectiveness of these third parties in marketing their own products may also affect our revenues and earnings. We intend to continue to enter into additional third-party agreements in the future. However, we may not be able to negotiate any additional agreements successfully. Even if established, these relationships may not be scientifically or commercially successful.
Any of these events could have a material adverse effect on our reputation, business, or financial condition, including but not limited to: interruptions or stoppages in our business operations (including clinical trials); inability to process personal data or to operate in certain jurisdictions; limited ability to develop or commercialize our products; expenditure of time and resources to defend any claim or inquiry; adverse publicity; or substantial changes to our business model or operations. ​ 61 Table of Contents Risks Related to our Securities The price of our stock may be volatile.
Any of these events could have a material adverse effect on our reputation, business, or financial condition, including but not limited to: interruptions or stoppages in our business operations (including clinical trials); inability to process personal data or to operate in certain jurisdictions; limited ability to develop or commercialize our products; expenditure of time and resources to defend any claim or inquiry; adverse publicity; or substantial changes to our business model or operations.
Such changes carry the risk that they will not achieve these intended objectives, and any of these changes could cause our product candidates to perform differently and affect the results of planned clinical trials or other future clinical trials. We currently have our clinical supply manufactured at Cell Ready’s manufacturing facility, and are working to develop commercially viable processes.
Such changes carry the risk that they will not achieve these intended objectives, and any of these changes could cause our product candidates to perform differently and affect the results of planned clinical trials or other future clinical trials. We are working to develop commercially viable processes for manufacturing our products.
For example, the FDA has limited experience with commercial development of cell therapies for cancer. Accordingly, the regulatory approval pathway for our product candidates may be uncertain, complex, expensive and lengthy, and approval may not be obtained, and the FDA or non-U.S. regulatory authorities may disagree with the design or implementation of our clinical trials or study endpoints.
Accordingly, the regulatory approval pathway for our product candidates may be uncertain, complex, expensive and lengthy, and approval may not be obtained, and the FDA or non-U.S. regulatory authorities may disagree with the design or implementation of our clinical trials or study endpoints.
As of December 31, 2023, we had 8.9 million shares of our common stock issued and outstanding (as adjusted for the Reverse Stock Split). Those outstanding shares represent a minority of our authorized shares, meaning that the ownership position of the current stockholders could be diluted significantly were we to issue a large number of additional shares.
As of December 31, 2024, we had 10.7 million shares of our common stock issued and outstanding. Those outstanding shares represent a minority of our authorized shares, meaning that the ownership position of the current stockholders could be diluted significantly were we to issue a large number of additional shares.
We are a clinical-stage immunotherapy company with a history of losses, and we may always operate at a loss. We expect that we will continue to operate at a loss throughout our development stage, and as a result, we may exhaust our financial resources and be unable to complete the development of our product candidates.
We expect that we will continue to operate at a loss throughout our development stage, and as a result, we may exhaust our financial resources and be unable to complete the development of our product candidates.
An inability to continue to source product from any of these suppliers, which could be due to regulatory actions or requirements affecting the supplier, adverse financial, or other strategic developments experienced by a supplier, labor disputes or shortages, unexpected demands, or quality issues, could adversely affect our ability to satisfy demand for our product candidates, which could adversely and materially affect our operating results or our ability to conduct clinical trials, either of which could significantly harm our business. 37 Table of Contents In the future, we may need to obtain rights to and supplies of specific materials and equipment to be used for the development of our product candidates.
An inability to continue to source product from any of these suppliers, which could be due to regulatory actions or requirements affecting the supplier, adverse financial, or other strategic developments experienced by a supplier, labor disputes or shortages, unexpected demands, or quality issues, could adversely affect our ability to satisfy demand for our product candidates, which could adversely and materially affect our operating results or our ability to conduct clinical trials, either of which could significantly harm our business.
Following the closing of the transaction, we no longer operate our own cGMP manufacturing facility and must rely on Cell Ready and other third parties for the clinical and, if approved, commercial manufacture of our product candidates.
Following the closing of the Cell Ready Purchase Agreement, we no longer operate our own cGMP manufacturing facility and instead rely on third parties for the clinical and, once approved, commercial manufacture of our product candidates.
Any significant increases in inflation and related increase in interest rates could have a material adverse effect on our business, results of operations and financial condition. Changes in tax laws or regulations could materially adversely affect our company.
Increased inflation rates can adversely affect us by increasing our costs, including labor and employee benefit costs. Any significant increases in inflation and related increase in interest rates could have a material adverse effect on our business, results of operations and financial condition. Changes in tax laws or regulations could materially adversely affect our company.
A number of jurisdictions outside of the United States have also established abbreviated pathways for regulatory approval of biological products that are biosimilar to earlier versions of biological products. For example, the European Union has had an established regulatory pathway for biosimilars since 2005. The increased likelihood of biosimilar competition has increased the risk of loss of innovators’ market exclusivity.
A number of jurisdictions outside of the United States have also established abbreviated pathways for regulatory approval of biological products that 45 Table of Contents are biosimilar to earlier versions of biological products. For example, the European Union has had an established regulatory pathway for biosimilars since 2005.
Any future determination as to the payment of cash dividends on our common stock will be at our board of directors’ discretion and depends on our financial condition, operating results, capital requirements and other factors that our board of directors considers to be relevant.
Any future determination as to the payment of cash dividends on our common stock will be at our board of directors’ discretion and depends on our financial condition, operating results, capital requirements and other factors that our board of directors considers to be relevant. General Risk Factors From time to time, we may become involved in legal proceedings.

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Item 1C. Cybersecurity

Cybersecurity — threats and controls disclosure

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Biggest changeOur cybersecurity risk assessment and management processes are implemented and maintained by various members of our management team, third party service providers and other employees, who we believe have a combination of relevant expertise, experience, education and training. ​ Certain members of our management team are responsible for hiring appropriate personnel and third-party IT service providers, helping to integrate cybersecurity risk considerations into our overall risk management strategy, communicating key priorities to relevant personnel, approving budgets, helping prepare for cybersecurity incidents, approving cybersecurity processes, and reviewing security assessments and other security-related reports. ​ Our cybersecurity incident response processes are designed to escalate certain cybersecurity incidents to members of management depending on the circumstances, including in some cases to our executive team.
Biggest changeCertain members of our management team are responsible for hiring appropriate personnel and third-party IT service providers, helping to integrate cybersecurity risk considerations into our overall risk management strategy, communicating key priorities to relevant personnel, approving budgets, helping prepare for cybersecurity incidents, approving cybersecurity processes, and reviewing security assessments and other security-related reports.
Such a breach could expose us to business interruption, lost revenue, ransom payments, remediation costs, liabilities to affected parties, cybersecurity protection costs, lost assets, litigation, regulatory scrutiny and actions, reputational harm, customer dissatisfaction, harm to our vendor relationships, or loss of market share. ​ Cybersecurity Governance and Oversight ​ Our board of directors addresses our cybersecurity risk management as part of its general oversight function.
Such a breach could expose us to business interruption, lost revenue, ransom payments, remediation costs, liabilities to affected parties, cybersecurity protection costs, lost assets, litigation, regulatory scrutiny and actions, reputational harm, customer dissatisfaction, harm to our vendor relationships, or loss of market share.
Our cybersecurity incident management team, and other individuals as needed, work to help us mitigate and remediate cybersecurity incidents of which we are notified. In addition, our incident response processes include a procedure for reporting certain cybersecurity incidents to the board of directors. ​ The board of directors receives regular reports from management concerning our cybersecurity risk management program.
In addition, our incident response processes include a procedure for reporting certain cybersecurity incidents to the board of directors. The board of directors receives regular reports from management concerning our cybersecurity risk management program. The board also receives various summaries and/or presentations related to cybersecurity threats, risks and mitigation. ​
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The board also receives various summaries and/or presentations related to cybersecurity threats, risks and mitigation. ​ 68 Table of Contents
Added
Cybersecurity Governance and Oversight Our board of directors addresses our cybersecurity risk management as part of its general oversight function. Our cybersecurity risk assessment and management processes are implemented and maintained by various members of our management team, third party service providers and other employees, who we believe have a combination of relevant expertise, experience, education and training.
Added
Our cybersecurity incident response processes are designed to escalate certain cybersecurity incidents to members of management depending on the circumstances, including in some cases to our executive team. Our cybersecurity incident management team, and other 66 Table of Contents individuals as needed, work to help us mitigate and remediate cybersecurity incidents of which we are notified.

Item 2. Properties

Properties — owned and leased real estate

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Biggest changeITEM 2. PROPERTIES We do not own any real estate or other properties. We do not directly lease any property but utilize office space through our service agreements with Cell Ready at 9350 Kirby Drive, Suite 300, Houston, Texas 77054, which is also our principal business office. ​
Biggest changeITEM 2. PROPERTIES We do not own any real estate or other properties. We utilize office space through Baylor College of Medicine at 2450 Holcombe Blvd, Suite BCM-A, MS: BCM251, Houston, TX 77021, which is also our principal business office. ​

Item 3. Legal Proceedings

Legal Proceedings — active lawsuits and investigations

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Biggest changeOther than as described below, we are not currently a party to any material legal proceedings, and we are not aware of any pending or threatened legal proceeding against us that we believe could have an adverse effect on our business, operating results or financial condition. ​ ITEM 4.
Biggest changeITEM 3. LEGAL PROCEEDINGS From time to time, we may become involved in legal proceedings, including those arising in the ordinary course of our business. We are not currently a party to any material legal proceedings that we believe could have an adverse effect on our business, operating results or financial condition. ​ ITEM 4.
MINE SAFETY DISCLOSURE Not Applicable ​ 69 Table of Contents PART II
MINE SAFETY DISCLOSURE Not Applicable ​ 67 Table of Contents PART II
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ITEM 3. LEGAL PROCEEDINGS From time to time, we may become involved in legal proceedings arising in the ordinary course of our business.

Item 5. Market for Registrant's Common Equity

Market for Common Equity — stock, dividends, buybacks

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Biggest changeRecent Sales of Unregistered Securities We did not record any issuances of unregistered securities during the fourth quarter of 2023. Issuer Repurchase Of Equity Securities We did not repurchase any equity securities. ITEM 6. [RESERVED] ​ 70 Table of Contents
Biggest changeIssuer Repurchase Of Equity Securities We did not repurchase any equity securities. ITEM 6. [RESERVED] ​
ITEM 5. MARKET FOR REGISTRANT’S COMMON EQUITY, RELATED STOCKHOLDER MATTERS AND ISSUER PURCHASES OF EQUITY SECURITIES Market Information Our common stock is listed for trading on the Nasdaq Capital Market under the symbol “MRKR”. As of March 18, 2024, we had 36 stockholders of record, whom are holding shares.
ITEM 5. MARKET FOR REGISTRANT’S COMMON EQUITY, RELATED STOCKHOLDER MATTERS AND ISSUER PURCHASES OF EQUITY SECURITIES Market Information Our common stock is listed for trading on the Nasdaq Capital Market under the symbol “MRKR”. As of March 26, 2025, we had 38 stockholders of record, whom are holding shares.
The price of our common stock on March 18, 2024 was $4.43 per share. Dividend Policy No dividends have been declared or paid on our common stock. We have incurred recurring losses and do not currently intend to pay any cash dividends in the foreseeable future.
The price of our common stock on March 26, 2025 was $1.16 per share. Dividend Policy No dividends have been declared or paid on our common stock. We have incurred recurring losses and do not currently intend to pay any cash dividends in the foreseeable future.
Added
Recent Sales of Unregistered Securities As previously disclosed, on December 19, 2024, we entered into a securities purchase agreement (the “ Securities Purchase Agreement ”) with various investors for the issuance and sale in a private placement (the “ Private Placement ”) of (i) 1,783,805 shares of Common Stock par value $0.001 per share (the “ Common Stock ”), (ii) Series A Warrants to acquire 5,031,250 shares of Common Stock (the “ Private Placement Warrants ”) and (iii) partially prepaid warrants to acquire 3,247,445 additional shares of Common Stock (the “ Pre-Funded Warrants ”, and together with the Private Placement Warrants, the “ Warrants ”).
Added
The Private Placement closed on December 23, 2024. The gross proceeds to the Company from the Private Placement were approximately $16.1 million, before deducting placement agent fees and offering expenses payable by the Company. The Company intends to use the net proceeds received from the Private Placement for general corporate purposes and working capital.
Added
Pursuant to the Securities Purchase Agreement, the Company is obligated to call a meeting of its stockholders to obtain stockholder approval (the “ Stockholder Approval ”) for the issuance of the shares issuable upon exercise of the Warrants within 90 days of the closing date.
Added
If the Stockholder Approval is not obtained at such meeting, the Company is required to cause an additional stockholder meeting to be held on or prior to the 180 th day after the closing date, and, if despite the Company’s reasonable best efforts the Stockholder Approval is not obtained after such subsequent stockholder meeting, the Company is required to cause an additional stockholder meeting to be held semi-annual thereafter until such Stockholder Approval is obtained.
Added
In connection therewith, the Company entered into voting agreements with certain stockholders pursuant to which such stockholders, who collectively own approximately 27% of the shares of common stock outstanding immediately prior to the closing of the Private Placement, agreed to vote in favor of the Private Placement.
Added
The Warrants are exercisable at any time on or after the date that the Stockholder Approval is obtained and have a term of five years commencing upon the date that the Stockholder Approval is obtained. The Pre-Funded warrants are exercisable at a price of $0.001 per share and the Private Placement Warrants have an exercise price of $4.00 per share.
Added
None of the issuances of the shares of Common Stock, the Pre-Funded Warrants, the Private Placement Warrants, or the shares of Common Stock issuable upon exercise of the Warrants (the “ Warrant Shares ”) have been registered under the Securities Act of 1933, as amended (the “ Securities Act ”), or any state securities laws.
Added
The shares of Common Stock, the Pre-Funded Warrants and the Private Placement Warrants, were issued, and Warrant Shares will be issued, in reliance on the exemptions from registration provided by Section 4(a)(2) under the Securities Act and/or Regulation D promulgated thereunder.
Added
The investors who entered into to a Securities Purchase Agreement have represented that they are each an accredited investor, as defined in Rule 501 of Regulation D promulgated under the Securities Act.
Added
In connection with the Private Placement, the Company entered into a registration rights agreement (the “ Registration Rights Agreement ”), dated as of December 19, 2024, with the investors in the Private Placement, pursuant to which the Company agreed to prepare and file a registration statement with the Securities and Exchange Commission (the “ SEC ”) registering the resale of the shares of Common Stock issued in the Private Placement along with the shares of Common Stock underlying the Warrants no later than January 14, 2025, and to use its best efforts to have the registration statement declared effective as promptly as practical thereafter, and in any 68 Table of Contents event no later than the earlier of (A) February 14, 2024 and (B) the second business day after the date the Company is notified by the SEC that the Registration Statement will not be reviewed or will not be subject to further review Canaccord Genuity LLC acted as the Company’s lead placement agent and Brookline Capital Markets, a division of Arcadia Securities, LLC, acted as co-placement agent (collectively, the “ Agents ”) in connection with the Private Placement, pursuant to that certain engagement letter, dated as of June 14, 2024, between the Company and Canaccord Genuity LLC (the “ Engagement Letter ”).
Added
Pursuant to the Engagement Letter, the Company paid or agreed to pay the Agents a cash fee equal to (i) 6% of the aggregate gross proceeds of the Private Placement other than from certain identified investors (the “ Reduced Fee Investors ”), (ii) 3% of the first $5 million of aggregate gross proceeds of the Private Placement form the Reduced Fee Investors, and (iii) 6% of the aggregate gross proceeds of the Private Placement form the Reduced Fee Investors in excess of $5 million.
Added
In addition, the Company agreed to pay Wainwright certain expenses. The Engagement Letter and the Securities Purchase Agreement contain customary representations and warranties, agreements and obligations, conditions to closing and termination provisions.
Added
The foregoing descriptions of terms and conditions of the Securities Purchase Agreement, the Pre–Funded Warrants, the Private Placement Warrants, and the Registration Rights Agreement do not purport to be complete and are qualified in their entirety by the full text of the form of the Purchase Agreement, the form of the Pre–Funded Warrant, the form of Private Placement Warrant, and the form of the Registration Rights Agreement, which are attached hereto as Exhibits 10.7, 4.2, 4.3 and 10.8, respectively.

Item 7. Management's Discussion & Analysis

Management's Discussion & Analysis (MD&A) — revenue / margin commentary

62 edited+63 added−53 removed31 unchanged
Biggest changeThe decrease of $1.6 million in 2023 was primarily attributable to the following: ● decrease of $2.1 million in process development expenses, ● decrease of $0.8 million in AML Phase 2 clinical trial expenses, offset by ● increase of $1.3 million in Cell Ready (outsourced) clinical manufacturing costs and process development expenses.
Biggest changeThe increase of $3.1 million in 2024 was primarily attributable to the following: ● increase of $4.5 million in Cell Ready (outsourced) clinical manufacturing costs and process development expenses, ● increase of $1.8 million in clinical trial expenses, offset by ● decrease of $1.2 million in process development expenses, ● decrease of $1.5 million in headcount-related expenses, including stock-based compensation expense and ● decrease of $0.5 million in other expenses. 74 Table of Contents General and Administrative Expenses General and administrative expenses decreased by 43% to $4.2 million for the year ended December 31, 2024 from $7.5 million during the prior period.
Our future funding requirements will depend on many factors, as we: ● initiate or continue clinical trials of our product candidates; ● continue the research and development of our product candidates and seek to discover additional product candidates; seek regulatory approvals for our product candidates if they successfully complete clinical trials; ● continue development of our manufacturing capabilities and our manufacturing facility; ● establish sales, marketing and distribution infrastructure and scale-up manufacturing capabilities to commercialize any product candidates that may receive regulatory approval; ● evaluate strategic transactions we may undertake; and ● enhance operational, financial and information management systems and hire additional personnel, including personnel to support development of our product candidates and, if a product candidate is approved, our commercialization efforts.
Our future funding requirements will depend on many factors, as we: ● initiate or continue clinical trials of our product candidates; ● continue the research and development of our product candidates and seek to discover additional product candidates; seek regulatory approvals for our product candidates if they successfully complete clinical trials; ● continue development of our manufacturing capabilities; ● establish sales, marketing and distribution infrastructure and scale-up manufacturing capabilities to commercialize any product candidates that may receive regulatory approval; ● evaluate strategic transactions we may undertake; and ● enhance operational, financial and information management systems and hire additional personnel, including personnel to support development of our product candidates and, if a product candidate is approved, our commercialization efforts.
Costs and timing of clinical trials and development of our product candidates will depend on a variety of factors that include, but are not limited to, the following: ● per patient clinical trial costs; ● the number of patients that participate in the clinical trials; ● the number of sites included in the clinical trials; ● the length of time required to enroll eligible patients; ● the number of doses that patients receive; ● the drop-out or discontinuation rates of patients; ● potential additional safety monitoring or other studies requested by regulatory agencies; ● the duration of patient follow-up; ● the efficacy and safety profile of the product candidates; and ● the ability to successfully manufacture patient doses.
Costs and timing of clinical trials and development of our product candidates will depend on a variety of factors that include, but are not limited to, the following: ● per patient clinical trial costs; ● the number of patients that participate in the clinical trials; ● the number of sites included in the clinical trials; ● the length of time required to enroll eligible patients; ● the number of doses that patients receive; ● the drop-out or discontinuation rates of patients; ● potential additional safety monitoring or other studies requested by regulatory agencies; 72 Table of Contents ● the duration of patient follow-up; ● the efficacy and safety profile of the product candidates; and ● the ability to successfully manufacture patient doses.
MANAGEMENT’S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS The following discussion of our financial condition, changes in financial condition, plan of operations and results of operations should be read in conjunction with (i) our audited consolidated financial statements as of December 31, 2023 and December 31, 2022 and (ii) the section entitled “Business”, included in this annual report.
MANAGEMENT’S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS The following discussion of our financial condition, changes in financial condition, plan of operations and results of operations should be read in conjunction with (i) our audited consolidated financial statements as of December 31, 2024 and December 31, 2023 and (ii) the section entitled “Business”, included in this annual report.
We do not believe that inflation has had a material impact on our results of operations for the periods presented, except with respect to payroll-related costs and other costs arising from or related to government-imposed regulations.
We do not believe that inflation has had a material impact on our results of operations for the periods presented, except with respect to payroll-related costs and other costs arising from or related to government-imposed regulations. ITEM 7A.
FINANCIAL STATEMENTS The Financial Statements are incorporated herein by reference to pages beginning on page F-1 at the end of this report and the supplementary data is not applicable. ITEM 9. CHANGES IN AND DISAGREEMENTS WITH ACCOUNTANTS ON ACCOUNTING AND FINANCIAL DISCLOSURE We have had no changes in, or disagreements with our principal independent accountants. 83 Table of Contents
FINANCIAL STATEMENTS The Financial Statements are incorporated herein by reference to pages beginning on page F-1 at the end of this report and the supplementary data is not applicable. ITEM 9. CHANGES IN AND DISAGREEMENTS WITH ACCOUNTANTS ON ACCOUNTING AND FINANCIAL DISCLOSURE We have had no changes in, or disagreements with our principal independent accountants.
When infused into a patient with cancer, the multiTAA-specific T cells are designed to kill cancer cells expressing the TAA and potentially recruit the patient’s immune system to participate in the cancer killing process. We licensed the underlying technology for multiTAA-specific T cell therapy from Baylor College of Medicine, or BCM, in March 2018.
When infused into a patient with cancer, the MAR-T cells are designed to kill cancer cells expressing the TAA and potentially recruit the patient’s immune system to participate in the cancer killing process. We licensed the underlying technology for MAR-T cell therapy from Baylor College of Medicine, or BCM, in March 2018.
The incurrence of indebtedness would result in increased fixed payment obligations and could involve certain restrictive covenants, such as limitations on our ability to incur additional debt, limitations on our ability to acquire or license intellectual property rights and other operating restrictions that could adversely impact our ability to conduct our business.
The incurrence of indebtedness would result in increased fixed payment obligations and could involve certain restrictive covenants, such as limitations on our ability to incur additional debt, limitations on our ability to acquire or license intellectual property rights and other operating restrictions that could adversely impact 78 Table of Contents our ability to conduct our business.
We anticipate that we will continue to incur net losses in the future as we continue to invest in research and development activities, including clinical development of our multiTAA T cell product candidates.
We anticipate that we will continue to incur net losses in the future as we continue to invest in research and development activities, including clinical development of our MAR-T cell product candidates.
The Sales Agents will be entitled to compensation under the Sales Agreement at a commission 78 Table of Contents rate equal to 3.0% of the gross sales price per share sold under the ATM Agreement, and we have provided each of the Sales Agents with indemnification and contribution rights.
The Sales Agents will be entitled to compensation under the Sales Agreement at a commission rate equal to 3.0% of the gross sales price per share sold under the ATM Agreement, and we have provided each of the Sales Agents with indemnification and contribution rights.
Recently Issued Accounting Standards Not Yet Adopted Improvements to Reportable Segment Disclosures In November 2023, the Financial Accounting Standards Board (the “FASB”) issued Accounting Standards Update (“ASU”) No. 2023-07, Segment Reporting (Topic 280): Improvements to Reportable Segment Disclosures. The ASU requires disclosures to include significant segment expenses that are regularly provided to the chief operating decision maker, among other provisions.
Improvements to Reportable Segment Disclosures In November 2023, the Financial Accounting Standards Board (the “FASB”) issued Accounting Standards Update (“ASU”) No. 2023-07, Segment Reporting (Topic 280): Improvements to Reportable Segment Disclosures. The ASU requires disclosures to include significant segment expenses that are regularly provided to the chief operating decision maker, among other provisions.
We believe that the simplicity of our manufacturing process allows additional modifications to expand multiTAA-specific T cell recognition of cancer targets. For example, we are assessing the potential of combining multiTAA-specific T cell products with other products.
We believe that the simplicity of our manufacturing process allows additional modifications to expand MAR-T cell recognition of cancer targets. For example, we are assessing the potential of combining MAR-T cell products with other products.
At December 31, 2023 and 2022, we recorded a 100% valuation allowance against our deferred tax assets of approximately $39.7 million and $41.3 million, respectively, as our management believes it is uncertain that they will be fully realized.
At December 31, 2024 and 2023, we recorded a 100% valuation allowance against our deferred tax assets of approximately $41.0 million and $39.7 million, respectively, as our management believes it is uncertain that they will be fully realized.
The federal net operating loss carryforwards of $93.8 million generated in 2018 and thereafter are subject to an 80% limitation on taxable income, do not expire and will carry forward indefinitely. The state net operating loss carryforwards of $21.7 million, if not utilized, will begin to expire in 2035.
The federal net operating loss carryforwards of $99.9 million generated in 2018 and thereafter are subject to an 80% limitation on taxable income, do not expire and will carry forward indefinitely. The state net operating loss carryforwards of $21.9 million, if not utilized, will begin to expire in 2035.
Tax Loss and Credit Carryforwards As of December 31, 2023, we have approximately $131.8 million of federal and $38.4 million of state net operating loss carryforwards that may be available to offset future taxable income, if any. The federal net operating loss carryforwards of $38.0 million, if not utilized, will expire between 2030 and 2037.
Tax Loss and Credit Carryforwards As of December 31, 2024, we have approximately $138.3 million of federal and $38.8 million of state net operating loss carryforwards that may be available to offset future taxable income, if any. The federal net operating loss carryforwards of $38.4 million, if not utilized, will expire between 2030 and 2037.
In May 2023, the Company announced that it had received a $2.0 million grant from the National Institutes of Health Small Business Innovation Research (“SBIR”) program to support the development and investigation of MT-401 for the treatment of AML patients following standard-of-care therapy with hypomethylating agents.
In May 2023, the Company announced that it had received a $2.0 million grant from the National Institutes of Health Small Business Innovation Research (“SBIR”) program to support the development and investigation of MT-401 for the treatment of AML patients following standard-of-care therapy with hypomethylating agents. Through the date of this filing, the Company has received $1.2 million from SBIR.
Improvements to Income Tax Disclosures In December 2023, the FASB issued ASU No. 2023-09, Income Taxes (Topic 740): Improvements to Income Tax Disclosures (ASU 2023-09), which improves the transparency of income tax disclosures by requiring consistent categories and greater disaggregation of information in the effective tax rate reconciliation and income taxes paid disaggregated by jurisdiction.
Recently Issued Accounting Standards Not Yet Adopted Improvements to Income Tax Disclosures In December 2023, the FASB issued ASU No. 2023-09, Income Taxes (Topic 740): Improvements to Income Tax Disclosures (ASU 2023-09), which improves the transparency of income tax disclosures by requiring consistent categories and greater disaggregation of information in the effective tax rate reconciliation and income taxes paid disaggregated by jurisdiction.
In May 2023, the Company announced that it had received a $2.0 million grant from the National Institutes of Health Small Business Innovation Research (“SBIR”) program to support the development and investigation of MT-401 for the treatment of AML patients following standard-of-care therapy with hypomethylating agents.
In May 2023, we received notice of a $2.0 million grant from the National Institutes of Health Small Business Innovation Research (“SBIR”) program to support the development and investigation of MT-401 for the treatment of AML patients following standard-of-care therapy with hypomethylating agents.
The Company recorded $0.2 million of grant income related to the SBIR grant as revenue for the year ended December 31, 2023 and at December 31, 2023, the Company recorded $0.2 million of grant income receivable. In February 2024, the Company received $0.2 million of funds from the SBIR grant.
The Company recorded $1.0 million of grant income related to the SBIR grant as revenue during the year ended December 31, 2024 and at December 31, 2024, the Company recorded $0.2 million of grant income receivable. In February 2025, the Company received $0.2 million of funds from the SBIR grant.
Interest Income Interest income was $0.5 million and $0.2 million for the years ended December 31, 2023 and 2022, respectively, and was attributable to interest income relating to funds that are held in U.S. Treasury notes and U.S. government agency-backed securities.
Other Income (Expense) Interest Income Interest income was $0.4 million and $0.5 million for the years ended December 31, 2024 and 2023, respectively, and was attributable to interest income relating to funds that are held in U.S. Treasury notes and U.S. government agency-backed securities.
Financing Activities Net cash provided by financing activities was $1.1 million and $0.2 million during the years ended December 31, 2023 and 2022, respectively, primarily due to the net proceeds received from sale of common stock through the ATM Agreement as well as the exercise of stock options.
Financing Activities Net cash provided by financing activities was $15.0 million and $1.1 million during the years ended December 31, 2024 and 2023, respectively, primarily due to the net proceeds received from sale of common stock through the Private Placement in 2024 and ATM Agreement in 2023, as well as the exercise of stock options.
Forfeitures are accounted for as incurred. ​ Grant Income Grant Income represents funding under cost reimbursement programs from government agencies and non-profit foundations for qualified research and development activities performed by the Company.
Grant Income Grant Income represents funding under cost reimbursement programs from government agencies and non-profit foundations for qualified research and development activities performed by the Company.
Further, the COVID-19 pandemic, decades-high inflation and concerns about an economic recession in the United States or other major markets has resulted in, among other things, volatility in the capital markets that may have the effect of reducing our ability to access capital, which could in the future negatively affect our liquidity.
In addition to the foregoing, high inflation and concerns about an economic recession in the United States or other major markets have resulted in, among other things, volatility in the capital markets that may have the effect of reducing our ability to access capital, which could in the future negatively affect our liquidity.
Lurier provided consulting services to the Company pursuant to a consulting between the Company and Danforth Advisors, LLC (“Danforth”) and received no compensation directly from the Company. On November 17, 2023, the Company terminated the consulting agreement between the Company and Danforth, effective January 16, 2024. On November 17, 2023, Mr.
Lurier as the Company’s Interim Chief Financial Officer, whereby Mr. Lurier provided consulting services to the Company pursuant to a consulting between the Company and Danforth Advisors, LLC (“Danforth”) and received no compensation directly from the Company. On November 17, 2023, the Company terminated the consulting agreement between the Company and Danforth, effective January 16, 2024.
Significant changes and expenditures in operating expenses are outlined as follows: Research and Development Expense Research and development expenses or clinical trial costs decreased by 13% to $10.4 million for the year ended December 31, 2023, compared to $12.0 million for the year ended December 31, 2022, mainly as a result of the Cell Ready transaction.
Significant changes in operating expenses are outlined as follows: Research and Development Expense Research and development expenses or clinical trial costs increased by 29% to $13.5 million for the year ended December 31, 2024, compared to $10.4 million for the year ended December 31, 2023, mainly as a result of the Cell Ready transaction.
Operating expenses incurred during the fiscal year ended December 31, 2023 were $17.9 million compared to $23.3 million in the prior year.
Operating Expenses Operating expenses incurred during the fiscal year ended December 31, 2024 were $17.7 million compared to $17.9 million in the prior year.
Lurier ceased serving as the Company’s Interim Chief Financial Officer and Dr. Vera was appointed as the Company’s Principal Financial and Accounting Officer.
On November 17, 2023, Mr. Lurier ceased serving as the Company’s Interim Chief Financial Officer and Dr. Vera was appointed as the Company’s Principal Financial and Accounting Officer.
In September 2022, the Company received notice from the FDA that it had awarded the Company a $2.0 million grant from the FDA’s Orphan Products Grant program to support the Company’s Phase 2 clinical trial of MT-401 for the treatment of post-transplant AML.
In September 2022, the Company received notice from the FDA that it had awarded the Company a $2.0 million grant from the FDA’s Orphan Products Grant program to support the clinical investigation of MT-401 for the treatment of post-transplant AML. Through the date of this filing, the Company has received $1.0 million from the FDA grant.
The use of cash primarily related to our net loss from continuing operations of $14.0 million, partially offset by a $2.8 million increase from changes in assets and liabilities and $0.9 million of stock-based compensation. Net cash used in operating activities from continuing operations during the year ended December 31, 2022 was $21.5 million.
The use of cash primarily related to our net loss from continuing operations of $10.7 million and a $0.5 million decrease from changes in assets and liabilities, offset by $0.2 million of stock-based compensation. Net cash used in operating activities from continuing operations during the year ended December 31, 2023 was $10.3 million.
Discontinued Operations Operating Activities Net cash used in operating activities from discontinued operations during the year ended December 31, 2023 was $6.1 million, which primarily related to our net loss from discontinued operations of $3.0 million, which is net of $2.5 million in revenue for which cash had been received in the prior period.
Net cash used in operating activities from discontinued operations during the year ended December 31, 2023 was $6.1 million, which primarily related to our net loss from discontinued operations of $5.8 million, which is net of $2.5 million in revenue for which cash had been received in the prior period. 76 Table of Contents Investing Activities There was no cash flow activity related to discontinued operations investing activity during the year ended December 31, 2024.
We are advancing two product candidates for 3 clinical indications as part of our multiTAA-specific T cell program for: ● Autologous multiTAA product for the treatment of lymphoma and pancreatic cancer (MT-601) ● Off-the-Shelf (OTS) product in various indications (e.g., MT-401-OTS) We do not genetically engineer our multiTAA-specific T cell therapies and we believe that our product candidates are superior to T cells engineered with chimeric antigen receptors, or CAR-T, for several reasons including: ● Multiple targets → enhanced tumoricidal effect→ minimized tumor immune escape ​ ● Clinical safety → no treatment-related side effects, including cytokine release syndrome (CRS) or other severe adverse effects (SAEs), were attributed to the use of multiTAA-specific T cell therapies to date ​ ● Non-genetically engineered T cell products → no risk of mutagenesis and reduced manufacturing complexity → lower cost For these reasons, we believe our endogenous T cell receptor-based therapies may provide meaningful clinical benefit and safety to patients with both hematological and solid tumors.
In those studies, BCM saw evidence of clinical benefit, expansion of infused cells, and decreased toxicity compared to other cellular therapies. 69 Table of Contents We are advancing two product candidates for 3 clinical indications as part of our MAR-T cell program for: ● Autologous MAR-T cell product for the treatment of lymphoma and pancreatic cancer (MT-601) ● Off-the-Shelf (OTS) product in various indications (e.g., MT-401-OTS) We do not genetically engineer our MAR-T cell therapies and we believe that our product candidates are superior to T cells engineered with chimeric antigen receptors, or CAR-T, for several reasons including: ● Multiple targets → enhanced tumoricidal effect→ minimized tumor immune escape ● Clinical safety → no treatment-related side effects, including immune effector cell-associated neurotoxicity syndrome (ICANS) or other severe adverse effects (SAEs), were attributed to the use of MAR-T cell therapies to date ● Non-genetically engineered T cell products → selective expansion of tumor-specific T cells from a patient’s or donor’s blood capable of recognizing a broad range of tumor antigens→ no risk of mutagenesis and reduced manufacturing complexity → lower cost For these reasons, we believe our endogenous T cell receptor-based therapies may provide meaningful clinical benefit and safety to patients with both hematological and solid tumors.
We do not expect the adoption of this guidance to have a material impact on its consolidated financial statements. 81 Table of Contents Off-Balance Sheet Arrangements We have not entered into any off-balance sheet arrangements that have or are reasonably likely to have a current or future effect on our financial condition, changes in financial condition, revenues, expenses, results of operations, liquidity, capital expenditures or capital resources that is material to investors.
Off-Balance Sheet Arrangements We have not entered into any off-balance sheet arrangements that have or are reasonably likely to have a current or future effect on our financial condition, changes in financial condition, revenues, expenses, results of operations, liquidity, capital expenditures or capital resources that is material to investors.
During the second quarter of 2023, the Company recorded $0.9 million of severance and termination-related costs. The payments of these costs were completed in July of 2023. Effective May 1, 2023, the Company’s board of directors appointed Dr.
During the second quarter of 2023, the Company recorded $0.9 million of severance and termination-related costs. The payments of these costs were completed in July of 2023. Effective May 1, 2023, the Company’s board of directors appointed Dr. Juan Vera as the Company’s President and Chief Executive Officer. Effective June 30, 2023, the board of directors appointed Eliot M.
In August 2021, the Company received notice of a Product Development Research award totaling approximately $13.1 million from the CPRIT to support the Company’s Phase 2 clinical trial of MT-401.
In August 2021, we received notice of a Product Development Research award totaling approximately $13.1 million from the Cancer Prevention and Research Institute of Texas, or CPRIT, to support our Phase 2 clinical trial of MT-401.
Therefore, the Purchased Assets, related party revenue, service revenue and related expenses are classified as discontinued operations for all periods presented herein. See Note 6 to the accompanying financial statements for further information.
Therefore, the Purchased Assets, related party revenue, service revenue and related expenses are classified as discontinued operations for all periods presented herein. See Note 6 to the accompanying financial statements for further information. Critical Accounting Policies and Estimates The consolidated financial statements are prepared in conformity with U.S.
Our financial statements do not include any adjustments to the recoverability and classification of recorded asset amounts and classification of liabilities that might be necessary should we be unable to continue as a going concern. Critical Accounting Policies and Estimates The consolidated financial statements are prepared in conformity with U.S.
Our financial statements do not include any adjustments to the recoverability and classification of recorded asset amounts and classification of liabilities that might be necessary should we be unable to continue as a going concern.
The purchase agreement does not exhibit any of the characteristics for liability classification under ASC Topic 480, Distinguishing Liabilities from Equity. During the year ended December 31, 2023, we sold 12,500 shares of our stock under the Purchase Agreement. In January 2023, Lincoln Park was issued 180,410 shares of stock as a commitment fee at a value of $0.5 million.
The purchase agreement does not exhibit any of the characteristics for liability classification under ASC Topic 480, Distinguishing Liabilities from Equity. In January 2023, Lincoln Park was issued 180,410 shares of stock as a commitment fee at a value of $0.5 million. On February 29, 2024, we terminated the Purchase Agreement with Lincoln Park effective March 1, 2024.
The use of cash primarily related to our net loss from continuing operations of $19.8 million and a $4.7 million decrease from changes in assets and liabilities. This decrease was partially offset by $3.3 million of stock-based compensation.
The use of cash primarily related to our net loss from continuing operations of $14.0 million, partially offset by a $2.8 million increase from changes in assets and liabilities and $0.9 million of stock-based compensation.
The Company recorded $2.7 million of grant income related to the CPRIT grant as revenue for the year ended December 31, 2023 and at December 31, 2023, the Company recorded $0.5 million of grant income receivable.
The Company recorded $0.5 million of grant income related to the FDA grant as revenue during the year ended December 31, 2024 and at December 31, 2024, the Company had no grant income receivable recorded.
During the year ended December 31, 2023, we recognized $0.2 million of revenue associated with the SBIR grant. All funding agencies have agreed to continue their financial support and to shift funds to the MT-401-OTS program. Operating Expenses Operating expenses incurred during the fiscal year ended December 31, 2023 were $17.9 million compared to $23.3 million in the prior year.
During the years ended December 31, 2024 and 2023, we recognized $1.0 million and $0.2 million of revenue associated with the SBIR grant, respectively. The above funding agencies have agreed to continue their financial support and to shift funds to the MT-401-OTS program.
However, our ability to pursue our longer term planned business activities is dependent upon our successful efforts to raise additional capital and obtain grant funding. Our consolidated financial statements have been prepared on a going concern basis, which implies that we will continue to realize our assets and discharge our liabilities in the normal course of business.
Our consolidated financial statements have been prepared on a going concern basis, which implies that we will continue to realize our assets and discharge our liabilities in the normal course of business.
The decrease in general and administrative expenses of $3.8 million mainly comprised the following: ● decrease of $3.2 million in headcount-related expenses, including stock-based compensation expense and net of severance expense, ● decrease of $0.6 million in rent and utilities expense, primarily as a result of the Cell Ready transaction, including its assumption of facility leases, ● decrease of $0.6 million in legal and professional fees, ● decrease of $0.2 million in insurance expense, offset by ● increase of $0.8 million in consulting expenses.
The decrease in general and administrative expenses of $3.2 million is mainly comprised of the following: ● decrease of $1.8 million in headcount-related expenses, including stock-based compensation expense and net of severance expense, ● decrease of $0.8 million in legal and professional fees and insurance expense, ● decrease of $0.6 million in consulting and other expenses.
On September 13, 2022, we received notice from the FDA that we had been awarded a $2.0 million grant from the FDA’s Orphan Products Grant program to support our Phase 2 clinical trial of MT-401 for the treatment of post-transplant AML.
During the years ended December 31, 2024 and 2023, respectively, we recognized $4.4 million and $2.7 million of revenue associated with the CPRIT grant. 73 Table of Contents In September 2022, we received notice from the FDA that we had been awarded a $2.0 million grant from the FDA’s Orphan Products Grant program to support our Phase 2 clinical trial of MT-401 for the treatment of post-transplant AML.
The Company accounts for this arrangement as a contract to perform research and development for others and applies ASC 606 by analogy. The Company recognizes grant income when amounts eligible for reimbursement are determinable and have been incurred, the applicable conditions under the grant arrangements have been met, and collectability of amounts due is reasonably assured or already received.
The Company recognizes grant income when amounts eligible for reimbursement are determinable and have been incurred, the applicable conditions under the grant arrangements have been met, and collectability of amounts due is reasonably assured or already 80 Table of Contents received.
Results of Operations For the Years Ended December 31, 2023 and 2022 The following table summarizes the results of our continuing operations (rounded to the thousand except for per share amounts) for the years ended December 31, 2023 and 2022, together with the changes to those items: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ For the Years Ended ​ ​ ​ ​ ​ ​ ​ ​ December 31, ​ ​ ​ ​ ​ 2023 2022 Change Revenues: ​ ​ ​ ​ ​ Grant income ​ $ 3,311,000 ​ $ 3,514,000 ​ $ (203,000) (6) % Total revenues ​ 3,311,000 ​ 3,514,000 ​ (203,000) (6) % Operating expenses: ​ ​ ​ ​ ​ ​ ​ Research and development ​ 10,417,000 ​ 11,968,000 ​ (1,551,000) (13) % General and administrative ​ 7,476,000 ​ 11,336,000 ​ (3,860,000) (34) % Total operating expenses ​ 17,893,000 ​ 23,304,000 ​ (5,411,000) (23) % Loss from operations ​ (14,582,000) ​ (19,790,000) ​ 5,208,000 (26) % Other income (expenses): ​ ​ ​ ​ ​ ​ ​ ​ Arbitration settlement ​ ​ — ​ ​ (233,000) ​ ​ 233,000 ​ (100) % Interest income ​ 539,000 ​ 248,000 ​ 291,000 117 % Loss from continuing operations before income taxes ​ ​ (14,042,000) ​ ​ (19,775,000) ​ ​ 5,733,000 ​ (29) % ​ Revenue We did not generate any revenue during the years ended December 31, 2023 and 2022, respectively, from the sales or licensing of our product candidates.
Results of Operations for the Years Ended December 31, 2024 and 2023 The following table summarizes the results of our continuing operations (rounded to the thousands except for per share amounts) for the years ended December 31, 2024 and 2023, together with the changes to those items: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ For the Years Ended ​ ​ ​ ​ ​ ​ ​ ​ December 31, ​ ​ ​ ​ ​ 2024 2023 Change Revenues: ​ ​ ​ ​ ​ Grant income ​ $ 6,591,000 ​ $ 3,311,000 ​ $ 3,280,000 99 % Total revenues ​ 6,591,000 ​ 3,311,000 ​ 3,280,000 99 % Operating expenses: ​ ​ ​ ​ ​ ​ ​ Research and development ​ 13,468,000 ​ 10,417,000 ​ 3,051,000 29 % General and administrative ​ 4,241,000 ​ 7,475,000 ​ (3,234,000) (43) % Total operating expenses ​ 17,709,000 ​ 17,892,000 ​ (183,000) (1) % Loss from operations ​ (11,118,000) ​ (14,581,000) ​ 3,463,000 (24) % Other income (expenses): ​ ​ ​ ​ ​ ​ ​ ​ Interest income ​ 437,000 ​ 539,000 ​ (102,000) (19) % Loss from continuing operations before income taxes ​ ​ (10,681,000) ​ ​ (14,042,000) ​ ​ 3,361,000 ​ (24) % ​ Revenue We did not generate any revenue during the years ended December 31, 2024 and 2023, respectively, from the sales or licensing of our product candidates.
The Company recorded $0.4 million of grant income related to the FDA grant as revenue for the year ended December 31, 2023 and at December 31, 2023, the Company recorded $0.3 million of grant income receivable. In February 2024, the Company received $0.3 million of funds from the FDA grant.
Through the date of this filing, the Company has received approximately $7,000 of funds from this grant. The Company recorded approximately $7,000 of grant income related to this grant as revenue for the year ended December 31, 2024 and at December 31, 2024, the Company recorded $7,000 of grant income receivable.
Net cash used in operating activities from discontinued operations during the year ended December 31, 2022 was $5.5 million, which primarily related to our net loss from discontinued operations of $10.2 million, partially offset by cash received in advance of related party revenue recorded. 76 Table of Contents Investing Activities Net cash provided by investing activities from discontinued operations for the year ended December 31, 2023 was $18.7 million primarily due to the proceeds from the Cell Ready transaction, net of transaction costs.
Net cash provided by investing activities from discontinued operations for the year ended December 31, 2023 was $18.7 million primarily due to the proceeds from the Cell Ready transaction, net of transaction costs.
Net Loss from continuing operations The decrease in our net loss from continuing operations during the year ended December 31, 2023 compared to the year ended December 31, 2022 was due to higher grant income and related party service revenue, cost reductions in our research and development activities and moderate stabilization of our clinical trial activities.
Net Loss from continuing operations The decrease in our net loss from continuing operations during the year ended December 31, 2024 compared to the year ended December 31, 2023 was due to cost reductions in our general and administrative expenses, as well as higher grant income.
The state net operating loss carryforwards of $16.6 million generated in 2018 and thereafter are subject to an 80% limitation on taxable income, do not expire and will carry forward indefinitely. Any change in ownership greater than 50% under Section 382 of the Internal Revenue Code places significant annual limitations on the use of such net operating loss carryforwards.
The state net operating loss carryforwards of $16.9 million 81 Table of Contents generated in 2018 and thereafter are subject to an 80% limitation on taxable income, do not expire and will carry forward indefinitely.
We have based this estimate on assumptions that may prove to be wrong, and we could utilize our available capital resources sooner than we currently expect. Furthermore, our operating plan may change, and we may need additional funds sooner than planned in order to meet operational needs and capital requirements for 77 Table of Contents product development and commercialization.
Furthermore, our operating plan may change, and we may need additional funds sooner than planned in order to meet operational needs and capital requirements for product development and commercialization.
We have financed our operations primarily through public and private offerings of our stock and debt including warrants and the exercise thereof, grants, and more recently through the cash proceeds received from the Cell Ready transaction and additional grants to fund research. 75 Table of Contents The following table sets forth our cash and cash equivalents and working capital as of December 31, 2023 and 2022: ​ ​ ​ ​ ​ ​ ​ ​ December 31, 2023 December 31, 2022 Cash and cash equivalents ​ $ 15,111,000 ​ $ 11,782,000 Working capital ​ $ 14,053,000 ​ $ 8,837,000 ​ Cash Flows The following table summarizes our cash flows for the years ended December 31, 2023 and 2022: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ For the Years Ended ​ ​ December 31, ​ 2023 2022 Continuing operations: ​ ​ ​ ​ ​ ​ Net cash used in operating activities $ (10,341,000) $ (21,513,000) Net cash provided by financing activities ​ ​ 1,105,000 ​ ​ 202,000 Discontinued operations ​ ​ ​ ​ ​ ​ Net cash used in operating activities ​ (6,099,000) ​ (5,459,000) Net cash provided by (used in) investing activities ​ $ 18,664,000 ​ $ (4,945,000) Net increase (decrease) in cash and cash equivalents ​ $ 3,329,000 ​ $ (31,715,000) ​ Continuing Operations Operating Activities Net cash used in operating activities from continuing operations during the year ended December 31, 2023 was $10.3 million.
Cash and Working Capital The following table sets forth our cash and cash equivalents and working capital as of December 31, 2024 and 2023: ​ ​ ​ ​ ​ ​ ​ ​ December 31, 2024 December 31, 2023 Cash and cash equivalents ​ $ 19,192,000 ​ $ 15,111,000 Working capital ​ $ 18,558,000 ​ $ 14,053,000 ​ Cash Flows The following table summarizes our cash flows for the years ended December 31, 2024 and 2023: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ For the Years Ended ​ ​ December 31, ​ 2024 2023 Continuing operations: ​ ​ ​ ​ ​ ​ Net cash used in operating activities $ (10,910,000) $ (10,341,000) Net cash provided by financing activities ​ ​ 14,991,000 ​ ​ 1,105,000 Discontinued operations ​ ​ ​ ​ ​ ​ Net cash used in operating activities ​ — ​ (6,099,000) Net cash provided by (used in) investing activities ​ $ — ​ $ 18,664,000 Net increase (decrease) in cash and cash equivalents ​ $ 4,081,000 ​ $ 3,329,000 ​ Continuing Operations Operating Activities Net cash used in operating activities from continuing operations during the year ended December 31, 2024 was $10.9 million.
If we are unable to raise capital when needed or on attractive terms, we could be forced to delay, reduce or eliminate our research and development programs or future commercialization efforts.
If we are unable to raise capital when needed or on attractive terms, we could be forced to delay, reduce or eliminate our research and development programs or future commercialization efforts. In August 2021, the Company received notice of a Product Development Research award totaling approximately $13.1 million from the CPRIT to support the Company’s clinical investigation of MT-401.
In August 2021, we received notice of a Product Development Research award totaling approximately $13.1 million from the Cancer Prevention and Research Institute of Texas, or CPRIT, to support our Phase 2 clinical trial of MT-401. During the years ended December 31, 2023 and 2022, respectively, we recognized $2.7 million and $3.4 million of revenue associated with the CPRIT grant.
In August 2021, we received notice of a Product Development Research award totaling approximately $13.1 million from CPRIT to support our Phase 2 clinical trial of MT-401. In December 2024, we announced that the Company has been awarded a $9.5 million grant from CPRIT to support the clinical investigation of MT-601 in patients with metastatic pancreatic cancer.
We anticipate our clinical trial costs will continue to increase over the next several years due to increased spending on the clinical development and manufacturing of our product candidates. ​ 72 Table of Contents General and Administrative Expenses General and administrative expenses consist primarily of personnel costs, including share-based compensation, legal fees relating to patent and corporate matters, insurance costs, consulting and professional fees, audit and investor relations.
We anticipate our clinical trial costs will continue to increase over the next several years due to increased spending on the clinical development and manufacturing of our product candidates.
All funding agencies have agreed to continue their financial support and to shift funds to the MT-401-OTS program. As of December 31, 2023, we had working capital of $14.1 million, compared to working capital of $8.8 million as of December 31, 2022.
The above funding agencies have agreed to continue their financial support and to shift funds to the MT-401-OTS program.
GAAP, which require the use of estimates, judgments and assumptions that affect the reported amounts of assets and liabilities, the disclosure of contingent liabilities at the date of the financial statements, and the reported amounts of expenses in the periods presented. On an ongoing basis, we evaluate our estimates and judgments, including those related to accrued expenses and stock-based compensation.
GAAP, which require the use of estimates, judgments and assumptions that affect the reported amounts of assets and liabilities, the disclosure of contingent liabilities at the date of the financial statements, and the reported amounts of expenses in the periods presented. The Company’s critical accounting policies include grant income, which is discussed further below.
Recent Developments On February 22, 2024, we entered into a Master Services Agreement for Product Supply (the “MSA”) with Cell Ready LLC (“Cell Ready”) to provide services previously performed by the company until the disposition of its contract development and manufacturing operations. Cell Ready, which is owned by one of our directors and shareholders, Mr.
As such, on February 22, 2024, we entered into a Master Services Agreement for Product Supply (the “MSA”) with Cell Ready for the provision of various products and services by Cell Ready pursuant to work orders that may be entered into from time to time. Cell Ready, which is owned by one of our former directors and current shareholders, Mr.
Qualifying grant income earned in advance of cash received from grants is recognized as revenue and recorded as other receivable.
Qualifying grant income earned in advance of cash received from grants is recognized as revenue and recorded as other receivable. New Accounting Standards From time to time, new accounting pronouncements are issued by the FASB or other standard setting bodies that the Company adopts as of the specified effective date.
With respect to each grant or award, the Company determines if it has a collaboration in accordance with ASC Topic 808, Collaborative Arrangements (“ASC 808”). To the extent the grant or award is within the scope of ASC 808, the Company recognizes the award upon achievement of certain milestones as credits to research and development expenses.
With respect to each grant or award, the Company determines if it has a collaboration in accordance with ASC Topic 808, Collaborative Arrangements (“ASC 808”). For grant and awards outside the scope of ASC 808, the Company applies either ASC 606 or IAS 20 by analogy depending on if the arrangement is considered an exchange or non-exchange transaction, respectively.
If the services involve the supply of product, Cell Ready is required to supply such product in conformance with the product requirements set forth in the applicable work order(s). The MSA contains customary representations, warranties and indemnification provision. The initial term of the MSA is three years and may be extended upon the mutual written agreement of the parties.
John Wilson, is a contract development and manufacturing organization (CDMO). The MSA contains customary representations, warranties and indemnification provision. The initial term of the MSA is three years and may be extended upon the mutual written agreement of the parties. On March 27, 2025, we mutually agreed with Cell Ready to terminate the MSA.
Based on our clinical plans and our timing expectations related to the progress of our programs, we expect that, together with drawdowns of available grant funds, our cash and cash equivalents as of December 31, 2023 will enable us to fund our operating expenses and capital expenditure requirements into the fourth quarter of 2025.
Based on our lack of recurring revenues, anticipated uses of cash and historical recurring cash losses from operating activities, and cash and cash equivalents as of December 31, 2024, we anticipate that we will be able to fund our operating expenses and capital expenditure requirements into the first quarter of 2026, assuming no additional grant funds are received.
The Company is currently evaluating the standard to determine the impact of adoption to its consolidated financial statements and disclosures.
The Company does not expect the adoption of this guidance to have a material impact on its consolidated financial statements.
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We developed our lead product candidates from our multiTAA-specific T cell technology, which is based on the manufacture of non-engineered, tumor-specific T cells that recognize multiple tumor-associated antigens, or TAAs. MultiTAA-specific T cells are able to recognize multiple tumor targets to produce broad spectrum anti-tumor activity.
Added
Harnessing millions of years of immunologic evolution, Marker’s multi antigen recognizing (“MAR”)-T cell technology is designed to recognize and kill highly heterogeneous tumors without the need for genetic modifications. This approach selectively expands natural tumor-specific T cells from a patient’s/donor’s blood that are capable of recognizing a broad range of tumor associated antigens, or TAAs.
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In those studies, BCM saw evidence of clinical benefit, expansion of infused cells, and decreased toxicity compared to other cellular therapies.
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Unlike other T cell therapies, MAR-T cells are able to recognize hundreds of different epitopes within up to six tumor-specific antigens to produce broad spectrum anti-tumor activity. Targeting multiple antigens simultaneously exploits the natural capacity of T cells to recognize and kill tumor targets via native T cell receptors (“TCR”), while limiting tumor adaptation/escape by antigen-negative selection or antigen down-regulation.
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Reverse Stock Split On January 24, 2023, our board of directors approved the filing of a certificate of amendment to our amended and restated certificate of incorporation (the “Amendment”) with the Secretary of State of the State of Delaware to effect the one-for-ten (1:10) Reverse Stock Split of our outstanding common stock and a reduction in the total number of authorized shares of our common stock from 300,000,000 to 30,000,000 (the “Shares Reduction”).
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On December 19, 2024, we issued a press release providing an update on the progress and clinical observations from the Phase 1 APOLLO study, with a data cutoff date of September 10, 2024.
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Eastern Time on January 26, 2023. 71 Table of Contents ​ Pursuant to the Amendment, at the effective time of the Amendment, every ten (10) shares of our issued and outstanding common stock was automatically combined into one (1) issued and outstanding share of common stock and the authorized shares of our common stock was reduced from 300,000,000 to 30,000,000, without any change in par value per share.
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Our Phase 1 APOLLO study is investigating MT-601, a MAR-T cell product, in patients with lymphoma who have relapsed after anti-CD19 chimeric antigen receptor (CAR) T cell therapy or where anti-CD19 CAR-T cells are not an option.
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The Reverse Stock Split affected all shares of our common stock outstanding immediately prior to the effective time of the Amendment. No fractional shares were issued as a result of the Reverse Stock Split. Stockholders of record who would otherwise be entitled to receive a fractional share received a cash payment in lieu thereof.
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A total of 10 patients have been treated in the study, for which clinical data is currently available for 9 patients from 5 clinical sites across the United States. Study participants showed early objective responses with and without lymphodepletion.
Removed
As a result of the Reverse Stock Split, proportionate adjustments were made to the per share exercise price and/or the number of shares issuable upon the exercise or vesting of all stock options and warrants issued by us and outstanding immediately prior to the effective time of the Amendment, which resulted in a proportionate decrease in the number of shares of our common stock reserved for issuance upon exercise or vesting of such stock options and warrants and a proportionate increase in the exercise price of all such stock options and warrants.
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However, immunomonitoring data confirmed that lymphodepletion enhanced the expansion and persistence of MAR-T cell clones in vivo. 70 Table of Contents Pipeline Our clinical-stage pipeline is set forth below: Manufacturing Our manufacturing process was originally developed at Baylor College of Medicine, where we initially conducted our clinical trials.
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In addition, the number of shares reserved for issuance under our equity compensation plans immediately prior to the effective time of the Amendment were reduced proportionately. All share and per share amounts of common stock presented in this Annual Report on Form 10-K have been retroactively adjusted to reflect the one-for-10 (1:10) Reverse Stock Split.
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We continue to contract and collaborate with BCM and others to perform a wide variety of services to ensure the continuation of our research and development efforts, with the goal of optimizing our manufacturing process, product quality and commercial scalability.
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Income Taxes We recognized $4,000 in state tax expense for the year ended December 31, 2023 and none for the year ended December 31, 2022. Other Income (Expense) Other income (expense), net consists of interest income and arbitration settlement expenses.
Added
In July 2021, we opened an in-house cGMP manufacturing facility in Houston, Texas, where we manufactured the clinical supply of our product candidates.
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During the years ended December 31, 2023 and 2022, we recognized $0.4 million and $0.1 million of revenue associated with the FDA grant, respectively. 73 Table of Contents In May 2023, we received notice of a $2.0 million grant from the National Institutes of Health Small Business Innovation Research (“SBIR”) program to support the development and investigation of MT-401 for the treatment of AML patients following standard-of-care therapy with hypomethylating agents.
Added
Subsequently, on June 26, 2023, we completed a transaction with Cell Ready, LLC, or Cell Ready, pursuant to a Purchase Agreement, or the Cell Ready Purchase Agreement, dated May 1, 2023, by and between us and Cell Ready, pursuant to which we (i) assigned to Cell Ready the leases for our two manufacturing facilities in Houston, Texas, or the Manufacturing Facilities, (ii) sold to Cell Ready all of the equipment and leasehold improvements at the Manufacturing Facilities and (iii) assigned to Cell Ready our rights, title and interest in any contracts related to the equipment and Manufacturing Facilities (collectively referred to as the “Purchased Assets”).

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