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What changed in Moleculin Biotech, Inc.'s 10-K — 2024 vs 2025

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Paragraph-level year-over-year comparison of Moleculin Biotech, Inc.'s 2024 and 2025 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 2025 report.

+619 added−415 removedSource: 10-K (2026-03-18) vs 10-K (2025-03-21)

Top changes in Moleculin Biotech, Inc.'s 2025 10-K

619 paragraphs added · 415 removed · 297 edited across 7 sections

Item 1. Business

Business — how the company describes what it does

168 edited+95 added−46 removed284 unchanged
Biggest changeConcluded 10 subjects enrolled and treated over 3 dose levels up to 8 mg/kg No drug related serious adverse events noted 1 DIPG subject had a temporary clinical response WP1220 MB-201 / CTCL / Poland 1B / Proof of Concept Concluded and CSR completed Believe results warrant a Phase 2 study Met safety endpoints 60% of subjects documented PR WP1122 MB-301 / COVID-19 / UK 1A Completed; Established RP2D Drug at RP2D was tolerable and safe Met safety endpoints N/A as in healthy volunteer subjects WP1122 IND approved for GBM 1B/2 External Exploring for an investigator to lead a study Trial has not begun Trial has not begun Trial has not begun 4 Table of Contents Notes for Table 1 : 1) This is a summary of the detailed clinical discussion below and does not include compassionate use/right-to-try usage of our drug candidates; 2) Complete Response Composite (CRc) includes CRs and CRi’s; 3) Overall Response Rate (ORR) includes CRc and Partial Response (PR); 4) “Met safety endpoints” means that no drug-related serious and no unexpected adverse event (only one serious in MB-105) occurred as defined in the trial protocol 5) All data presented are preliminary (and subject to change) unless a CSR or an investigator's final report has been issued for the trial referenced; 6) With regard to safety and human activity summaries please see the detailed discussion below; and, 7) MB-106 Phase 1 included “all-comers” or subjects with unlimited lines of prior therapy while Phase 2 included only subjects as 1 st thru 3 rd line of therapy.
Biggest changeWP1066 IND cleared for GBM 1 Open to an investigator to lead a study WP1066 in combination w/ radiation therapy 2 nd protocol under IND allowed for Adult GBM / US 2 Open to an investigator to lead a study Used as reference for next trial WP1066 in combination with radiation therapy IND allowed for IIT led Adult GBM / US 1B/2 External Enrolling and treating 4 subjects enrolled and treated with 1 subject currently on active treatment No safety data reported to date No efficacy data reported to date WP1066 in combination with radiation therapy IIT led Pediatric Brain Tumors / US 1B/2 External Planned 2026 start WP1066 IIT / Pediatric Brain Tumors / US 1 External Concluded in February 2023 with a dose level at 8mg/kg; Concluded 10 subjects enrolled and treated over 3 dose levels up to 8 mg/kg No drug related serious adverse events noted 1 DIPG subject had a temporary clinical response WP1220 MB-201 / CTCL / Poland 1B / Proof of Concept Concluded and CSR completed Believe results warrant a Phase 2 study Met safety endpoints 60% of subjects documented PR WP1122 MB-301 / COVID-19 / UK 1A Completed; Established RP2D Drug at RP2D was tolerable and safe Met safety endpoints N/A as in healthy volunteer subjects WP1122 IND allowed for GBM; Inactive 1B/2 External Open to an investigator to lead a study 5 Table of Contents Notes for Table 1 : 1) This is a summary of the detailed clinical discussion below and does not include compassionate use/right-to-try usage of our drug candidates; 2) Complete Remission ("Response" for STS) Composite includes Complete Remission and CR with incomplete hematologic recovery (CRi or CRh); 3) Overall Response Rate (ORR) includes CRc and Partial Response (PR); 4) “Met safety endpoints” means that the studies achieved their safety objectives and refers to the overall safety profile observed in the study, which did not preclude continued clinical development.
The following discussion, which includes estimates based on current literature and our discussions with key opinion leaders, suggests that approximately 60% of AML patients continue to have a significant unmet need for new therapies. This is based on the reality that effective treatment options are limited.
The following discussion, which includes estimates based on current literature and our discussions with key opinion leaders, suggests that approximately 60% of AML patients continue to have a significant unmet need for new and effective therapies. This is based on the reality that effective treatment options are limited.
Of those subjects that, upon Venetoclax regimen failure and received salvage therapy, only 12.5% and 4% achieved a CRc and a CR, respectively (A. Maiti, C. Rausch, J. Cortes, Et al, “Outcomes of relapsed or refractory acute myeloid leukemia after frontline hypomethylating agent and Venetoclax regimens, Haematologica online, vol. 106 No.3 (2021)).
Of those subjects, that, upon venetoclax regimen failure received salvage therapy, only 12.5% and 4% achieved a CRc and a CR, respectively (A. Maiti, C. Rausch, J. Cortes, Et al, “Outcomes of relapsed or refractory acute myeloid leukemia after frontline hypomethylating agent and venetoclax regimens, Haematologica online, vol. 106 No.3 (2021)).
WP1220. We have rights to three issued US patents which claim compositions of WP1220, as well as foreign counterparts. These patents have an international filing date in June 2009. In addition, we have rights to an issued US patent for the treatment of skin disorders using WP1220, with a filing date in September 2009.
We have rights to three issued US patents which claim compositions of WP1220, as well as foreign counterparts. These patents have an international filing date in June 2009. In addition, we have rights to an issued US patent for the treatment of skin disorders using WP1220, with a filing date in September 2009.
Products that are approved to treat rare diseases that are serious or life-threatening and where the serious or life-threatening manifestations primarily affect patients under the age of 19 years of age may qualify for the Rare Pediatric Disease Priority Review Voucher (RPDPRV) program, in which the product sponsor receives upon approval a voucher for priority review of another product.
Products that are approved to treat rare diseases that are serious or life-threatening and where the serious or life-threatening manifestations primarily affect patients under 19 years of age may qualify for the Rare Pediatric Disease Priority Review Voucher (RPDPRV) program, in which the product sponsor receives upon approval a voucher for priority review of another product.
Any agency or judicial enforcement action could have a material adverse effect on us. 18 Table of Contents Development and Approval The process required by the FDA before a pharmaceutical product may be marketed in the US generally involves the following: • Completion of preclinical laboratory tests, animal studies and formulation studies; • Submission to the FDA of an Investigational New Drug application, or IND, which must become effective before human clinical trials may begin; • Performance of adequate and well-controlled human clinical trials according to the FDA’s regulations commonly referred to as good clinical practices (GCP) and additional requirements for the protection of human research subjects and their health information, to establish the safety and effectiveness of the proposed product; • Submission to the FDA of an NDA seeking marketing approval that includes substantial evidence of safety and effectiveness from results of clinical trials, as well as the results of preclinical testing, detailed information about the chemistry, manufacturing and controls, and proposed labeling and packaging for the product; • Review of the product candidate by an FDA advisory committee, if applicable; • Satisfactory completion of an FDA inspection of the manufacturing facility or facilities where the pharmaceutical product is produced, to assess compliance with current good manufacturing practice, or cGMP, requirements, to assure that the facilities, methods and controls are adequate to preserve the pharmaceutical product’s identity, strength, quality and purity; • Potential FDA audit of the preclinical and clinical trial sites that generated the data in support of the NDA; and • FDA review and approval of the NDA, including agreement on post-marketing commitments, if applicable.
Any agency or judicial enforcement action could have a material adverse effect on us. 20 Table of Contents Development and Approval The process required by the FDA before a pharmaceutical product may be marketed in the US generally involves the following: • Completion of preclinical laboratory tests, animal studies and formulation studies; • Submission to the FDA of an Investigational New Drug application, or IND, which must become effective before human clinical trials may begin; • Performance of adequate and well-controlled human clinical trials according to the FDA’s regulations commonly referred to as good clinical practices (GCP) and additional requirements for the protection of human research subjects and their health information, to establish the safety and effectiveness of the proposed product; • Submission to the FDA of an NDA seeking marketing approval that includes substantial evidence of safety and effectiveness from results of clinical trials, as well as the results of preclinical testing, detailed information about the chemistry, manufacturing and controls, and proposed labeling and packaging for the product; • Review of the product candidate by an FDA advisory committee, if applicable; • Satisfactory completion of an FDA inspection of the manufacturing facility or facilities where the pharmaceutical product is produced, to assess compliance with current good manufacturing practice, or cGMP, requirements, to assure that the facilities, methods and controls are adequate to preserve the pharmaceutical product’s identity, strength, quality and purity; • Potential FDA audit of the preclinical and clinical trial sites that generated the data in support of the NDA; and • FDA review and approval of the NDA, including agreement on post-marketing commitments, if applicable.
Although preliminary data from physician-sponsored brain tumor trials indicate that the oral administration of WP1066 results in detectable levels of WP1066 in plasma, we believe our opportunity for successful development of a p-STAT3 inhibitor would be expanded if we were able to develop a compound capable of a different oral delivery or intravenous (IV) administration.
Although preliminary data from physician-sponsored brain tumor trials indicate that the oral administration of WP1066 results in detectable levels of WP1066 in plasma, we believe our opportunity for successful development of a p-STAT3 inhibitor would be expanded if we were able to develop a compound capable of a different oral delivery or intravenous (IV) administration (WP1066IV).
The FDA also wanted to see the durability of response (DoR) as a secondary endpoint and overall survival as an exploratory endpoint, as well as data for patients beyond 2nd line, which is why our plan includes a follow-on MIRACLE2 trial in 3rd line patients starting once the optimum dose is established in the MIRACLE trial.
The FDA also wanted to see overall survival (OS) as a secondary endpoint and durability of response (DoR) as an exploratory endpoint, as well as data for patients beyond 2nd line, which is why our plan includes a follow-on MIRACLE2 trial in 3rd line patients starting once the optimum dose is established in the MIRACLE trial.
Based on these data, they filed and received clearance to proceed with an IND for a trial to treat children with recurrent or refractory malignant brain tumors with WP1066. This trial is being conducted at the Aflac Cancer & Blood Disorders Center at Children's Healthcare of Atlanta.
Based on these data, they filed and received clearance to proceed with an IND for a trial to treat children with recurrent or refractory malignant brain tumors with WP1066. This trial is being conducted at the Aflac Cancer & Blood Disorders Center at Children's Healthcare of Atlanta (Emory).
Clinical Trial with WP1122 In 2021, we received authorization from the MHRA to commence a Phase 1a clinical trial of WP1122 in the United Kingdom. The Phase 1a study in healthy human volunteers investigated the effects of a single ascending dose (SAD) and multiple days of ascending dosing (MAD) of WP1122 administered as an oral solution.
Activities with WP1122 In 2021, we received authorization from the MHRA to commence a Phase 1a clinical trial of WP1122 in the United Kingdom. The Phase 1a study in healthy human volunteers investigated the effects of a single ascending dose (SAD) and multiple days of ascending dosing (MAD) of WP1122 administered as an oral solution.
Additionally, the FDA has granted ODD for Annamycin for the treatment of soft tissue sarcoma. Such designation means, in part, these agencies believe we have established a medically plausible basis for using the drug for those indications. The FDA also granted Fast Track-Designation (FTD) for Annamycin for both the treatment of AML and Soft Tissue Sarcoma.
Additionally, the FDA has granted ODD for Annamycin for the treatment of soft tissue sarcoma. Such designation means, in part, these agencies believe we have established a medically plausible basis for using the drug for those indications. The FDA also granted Fast Track-Designation for Annamycin for both the treatment of AML and Soft Tissue Sarcoma.
In August 2023, we successfully completed the second cohort at 230 mg/m 2 of Annamycin in this combination study. Four subjects were treated in this cohort, one is believed to be relapsed from one or more prior therapies and three are believed to be refractory to up to three prior therapies.
In August 2023, we successfully completed the second cohort at 230 mg/m 2 of Annamycin in this combination study. Of the four subjects treated in this cohort, one is believed to be relapsed from one or more prior therapies and three are believed to be refractory to up to three prior therapies.
The FDA and certain state agencies also conduct periodic unannounced inspections to re-inspect equipment, facilities, and processes for compliance with cGMP and other laws. Accordingly, manufacturers must continue to expend time, money and effort in the area of production and quality control to maintain cGMP compliance.
The FDA and certain state agencies also conduct periodic inspections to re-inspect equipment, facilities, and processes for compliance with cGMP and other laws. Accordingly, manufacturers must continue to expend time, money and effort in the area of production and quality control to maintain cGMP compliance.
Our Intellectual Property and FDA Designations We have obtained worldwide, exclusive licenses or options to license from MD Anderson to issued US patents and pending US patent applications for each of our drug candidates, as well as pending foreign patent applications or issued foreign patents.
Our Intellectual Property, FDA Designations and License Agreements We have obtained worldwide, exclusive licenses or options to license from MD Anderson to issued US patents and pending US patent applications for each of our drug candidates, as well as pending foreign patent applications or issued foreign patents.
A "Paragraph IV" certification is an assertion that the patent does not block approval of the later product, either because the patent is invalid or unenforceable or because the patent, even if valid, is not infringed by the new product. 21 Table of Contents Once the FDA accepts for filing an ANDA or 505(b)(2) application containing a Paragraph IV certification, the applicant must within 20 days provide notice to the RLD or listed drug NDA holder and patent owner that the application has been submitted, and provide the factual and legal basis for the applicant's assertion that the patent is invalid or not infringed.
A "Paragraph IV" certification is an assertion that the patent does not block approval of the later product, either because the patent is invalid or unenforceable or because the patent, even if valid, is not infringed by the new product. 23 Table of Contents Once the FDA accepts for filing an ANDA or 505(b)(2) application containing a Paragraph IV certification, the applicant must within 20 days provide notice to the RLD or listed drug NDA holder and patent owner that the application has been submitted, and provide the factual and legal basis for the applicant's assertion that the patent is invalid or not infringed.
Working Environment Our headquarters and laboratory are in Houston, Texas, and our workforce, as of year-end 2024, consisted of 17 full and part-time employees, in the US which are leveraged with other service providers and contractors worldwide working in a primarily virtual environment. We do not have manufacturing facilities and all manufacturing activities are contracted out to third parties.
Working Environment Our headquarters and laboratory are in Houston, Texas, and our workforce, as of year-end 2025, consisted of 17 full and part-time employees in the US, which are leveraged with other service providers and contractors worldwide working in a primarily virtual environment. We do not have manufacturing facilities, and all manufacturing activities are contracted out to third parties.
A total of 2.0 million new cancer cases and 618,120 deaths from cancer are expected to occur in the US in 2025 , which is about 1,693 d eaths a day. These statistics do not include either basal cell or squamous cell skin cancers because US cancer registries are not required to collect information on these cancers.
A total of 2.0 million new cancer cases and 618,120 deaths from cancer are expected to occur in the US in 2025, which is about 1,693 deaths a day. These statistics do not include either basal cell or squamous cell skin cancers because US cancer registries are not required to collect information on these cancers.
In some instances, an NDA approval may be obtained based on Phase 2 clinical data, often with the understanding that the approved drug can be sold subject to a confirmatory trial to be conducted post-approval. 19 Table of Contents Additionally, post-approval studies, also referred to as Phase 4 clinical trials, may be conducted after initial marketing approval.
In some instances, an NDA approval may be obtained based on Phase 2 clinical data, often with the understanding that the approved drug can be sold subject to a confirmatory trial to be conducted post-approval. 21 Table of Contents Additionally, post-approval studies, also referred to as Phase 4 clinical trials, may be conducted after initial marketing approval.
We estimate that only about 21% of those 2nd line patients who happen to have the requisite genetic profile will achieve a durable CR, which means only another 11% of the AML population is given a chance to beat their disease with a successful bone marrow transplant or lasting remission.
We estimate that only about 21% of those 2nd line patients who happen to have the requisite genetic profile will achieve a durable CR, which means only another 18% of the AML population is given a chance to beat their disease with a successful bone marrow transplant or lasting remission.
Within 90 days of receiving the applications and assessment report, each member state must decide whether to recognize approval. 23 Table of Contents In addition to regulations in Europe and the United States, we will be subject to a variety of foreign regulations governing clinical trials and commercial distribution of our future drugs.
Within 90 days of receiving the applications and assessment report, each member state must decide whether to recognize approval. 25 Table of Contents In addition to regulations in Europe and the United States, we will be subject to a variety of foreign regulations governing clinical trials and commercial distribution of our future drugs.
All of the clinical trials are or were in the Phase 1 or 2 stage with the exception of MB-108 which is a Phase 2B/3. Starting in 2021 through 2024, there have been eight "right-to-try" (or their foreign equivalent) uses of Annamycin and WP1066.
All of the clinical trials are or were in the Phase 1 or 2 stage with the exception of MB-108 which is a Phase 2B/3. Starting in 2021 through 2024, there have been eight "right-to-try" or “compassionate use” (or their foreign equivalent) uses of Annamycin and WP1066.
This would be consistent with the current practice to use Ara-C in combination with other anthracyclines in AML patients.
This would also be consistent with the current practice to use Ara-C in combination with other anthracyclines in AML patients.
This leaves, based on our estimates, about 58% of all AML patients who will ultimately succumb to their disease. We believe this is not an acceptable outcome and are advancing Annamycin for the treatment of AML via our clinical trials.
This leaves, based on our estimates, about 60% of all AML patients who will ultimately succumb to their disease. We believe this is not an acceptable outcome and are advancing Annamycin for the treatment of AML via our clinical trials.
We expect that changes or additions to the ACA, IRA or their implementing regulations or guidance, changes to the Medicare and Medicaid programs, changes regarding the federal government’s authority to directly negotiate drug prices and changes stemming from other healthcare reform measures, especially with regard to healthcare access or financing or other legislation in individual states, could have a material adverse effect on the healthcare industry and our business.
We expect that changes or additions to the ACA, IRA OBBBA, MFN pricing or their implementing regulations or guidance, changes to the Medicare and Medicaid programs, changes regarding the federal government’s authority to directly negotiate drug prices and changes stemming from other healthcare reform measures, especially with regard to healthcare access or financing or other legislation in individual states, could have a material adverse effect on the healthcare industry and our business.
In multiple clinical studies, subjects treated with Annamycin have shown no signs of cardiotoxicity, allowing physicians to dose higher than the currently set limits for other anthracyclines or potentially treat traditionally "Unfit" subjects.
In multiple clinical studies, subjects treated with Annamycin have shown no signs of drug-related cardiotoxicity, allowing physicians to dose higher than the currently set limits for other anthracyclines or potentially treat traditionally "Unfit" subjects.
Although we periodically monitor the FDA compliance of our third-party manufacturers, we cannot be certain that our present or future third-party manufacturers will consistently comply with cGMP and other applicable FDA regulatory requirements. 22 Table of Contents Discovery of problems with a product after approval may result in restrictions on a product, manufacturer or NDA sponsor, including withdrawal of the product from the market.
Although we periodically monitor the FDA compliance of our third-party manufacturers, we cannot be certain that our present or future third-party manufacturers will consistently comply with cGMP and other applicable FDA regulatory requirements. Discovery of problems with a product after approval may result in restrictions on a product, manufacturer or NDA sponsor, including withdrawal of the product from the market.
An applicant receiving a complete response letter may either revise and resubmit the NDA or withdraw the application. 20 Table of Contents FDA approval of an NDA may impose significant limitations that could weaken the commercial value of the product.
An applicant receiving a complete response letter may either revise and resubmit the NDA or withdraw the application. 22 Table of Contents FDA approval of an NDA may impose significant limitations that could weaken the commercial value of the product.
Thus, we focused our efforts on a clinical trial studying AnnAraC for the treatment of AML in Europe. MB-106 : Below in Table 2 is a summary of the preliminary responses in the MB-106 combination therapy trial to date.
Thus, we focused our efforts on a clinical trial studying AnnAraC for the treatment of AML in Europe. MB-106 : Below in Table 2 is a summary of the responses demonstrated in the MB-106 combination therapy trial to date.
Given this backdrop, we believe the best initial pathway for Annamycin is to pursue the second-line treatment of STS lung metastasis. Per the American Cancer Society, in 2025, an estimated 67,440 new cases of pancreatic cancer will be diagnosed in the US and 51,980 people will die from the disease.
Given this backdrop, we believe the best initial pathway for Annamycin is to pursue the second-line treatment of STS lung metastasis. 18 Table of Contents Per the American Cancer Society, in 2025, an estimated 67,440 new cases of pancreatic cancer will be diagnosed in the US and 51,980 people will die from the disease.
Five such drugs have been approved to date, but each is only relevant to a subset of AML patients who happen to have the requisite genetic mutation and response rates are relatively low.
Six such drugs have been approved to date, but each is only relevant to a subset of AML patients who happen to have the requisite genetic mutation and response rates are relatively low.
These also stimulate a natural immune response to tumors by inhibiting the errant activity of Regulatory T-Cells (TRegs). 3 Table of Contents c) Our WP1122 Portfolio contains compounds (including WP1122, WP1096, and WP1097) designed to exploit the potential uses of inhibitors of glycolysis such as 2-deoxy-D-glucose (2-DG).
These also stimulate a natural immune response to tumors by inhibiting the errant activity of Regulatory T-Cells (TRegs). c) Our WP1122 Portfolio contains compounds (including WP1122, WP1096, and WP1097) designed to exploit the potential uses of inhibitors of glycolysis such as 2-deoxy-D-glucose (2-DG).
Food and Drug Administration (FDA) in July 2024. In July 2024, we announced the completion of our EOP2 meeting with the FDA for our Phase 1B/2 clinical trial evaluating Annamycin in combination with Cytarabine for the treatment of subjects with AML as both first line therapy and for subjects who are refractory to or relapsed after induction therapy (MB-106).
In July 2024, we announced the completion of our EOP2 meeting with the FDA for our Phase 1B/2 clinical trial evaluating Annamycin in combination with Cytarabine for the treatment of subjects with AML as both first line therapy and for subjects who are refractory to or relapsed after induction therapy (MB-106).
Based on our discussions with the FDA, we amended in September 2024 our MB-104 investigational new drug application or IND for MB-108. In the amendment with the new MIRACLE protocol, the trial will, for the first time ever in the US for an AML trial, allow dosing above the lifetime maximum allowable dose (LTMAD) for currently prescribed anthracyclines.
Based on our discussions with the FDA, we amended in September 2024 our MB-104 investigational new drug application or IND for MB-108. In the amendment with the new MIRACLE protocol, the trial, for the first time ever in the US, allowed for dosing in an AML trial above the lifetime maximum allowable dose (LTMAD) for currently prescribed anthracyclines.
In 2020, we began developing IV formulation methods for WP1066 and/or its analogs that might address these issues. Recently, we have succeeded in identifying a promising candidate for IV formulation and we have begun IND-enabling preclinical work, however there can be no assurance that this effort will be successful.
In 2020, we began developing IV formulation methods for WP1066 and/or its analogs that might address these issues. Recently, we have succeeded in identifying a promising candidate for IV formulation, filed appropriate patents, and we have begun IND-enabling preclinical work. However, there can be no assurance that this effort will be successful.
Animal Life Sciences Licensing Agreement On February 19, 2019, we sublicensed certain intellectual property rights, including rights to Annamycin, our WP1122 portfolio, and our WP1066 portfolio in the field of non-human animals to Animal Life Sciences, LLC (ALI) (the “ALI Agreement”). ALI is affiliated with Dr. Waldemar Priebe, our founder.
Animal Life Sciences Licensing Agreement On February 19, 2019, we sublicensed certain intellectual property rights, including rights to Annamycin, our WP1122 portfolio, and our WP1066 portfolio in the field of non-human animals to Animal Life Sciences, LLC (ALI) (the “ALI Agreement”). ALI is affiliated with Dr. Waldemar Priebe, one of our founders.
Annamycin was dosed at 190 mg/m 2 , along with Cytarabine at 2.0 g/m 2 /day for five days (total dose of 10g/m 2 ). 8 Table of Contents At the recommendation of the safety review committee, we deemed the first cohort dose as safe and opened the second cohort with the Annamycin dose being increased to 230 mg/m 2 .
Annamycin was dosed at 190 mg/m 2 , along with Cytarabine at 2.0 g/m 2 /day for five days (total dose of 10g/m 2 ). At the recommendation of the safety review committee, we deemed the first cohort dose as safe and opened the second cohort with the Annamycin dose being increased to 230 mg/m 2 .
For Part B of the trial, approximately 220 additional subjects will be randomized to receive either HiDAC plus placebo or HiDAC plus the optimum dose of Annamycin (randomized 1:1). The selection of the optimum dose will be based on the overall balance of safety, pharmacokinetics and efficacy, consistent with the FDA’s new Project Optimus initiative.
For Part B of the trial, 222 additional subjects will be randomized to receive either HiDAC plus placebo or HiDAC plus the optimum dose of Annamycin (randomized 1:1). The selection of the optimum dose will be based on the overall balance of safety, pharmacokinetics and efficacy, consistent with the FDA’s new Project Optimus initiative.
The particular animal models used in our testing are considered to represent very aggressive forms of cancer. Furthermore, a poster entitled, "Liposomal annamycin inhibition of lung localized breast cancer," was presented at the San Antonio Breast Cancer Symposium held in December 2019.
The particular animal models used in our testing are considered to represent very aggressive forms of cancer. 8 Table of Contents Furthermore, a poster entitled, "Liposomal annamycin inhibition of lung localized breast cancer," was presented at the San Antonio Breast Cancer Symposium held in December 2019.
The MIRACLE study, subject to appropriate future filings with and potential additional feedback from the FDA and their foreign equivalents, utilizes an adaptive design whereby the first 75 to 90 subjects will be randomized (1:1:1) in Part A of the trial to receive high dose cytarabine (HiDAC) combined with either placebo, 190 mg/m2 of Annamycin, or 230 mg/m2 of Annamycin, which Annamycin doses were specifically recommended by the FDA in the Company’s end of Phase 1B/2 meeting.
The MIRACLE study, subject to appropriate future filings with and potential additional feedback from the FDA and their foreign equivalents, utilizes an adaptive design whereby the first 90 subjects will be randomized (1:1:1) in Part A of the trial to receive HiDAC (or AraC) combined with either placebo, 190 mg/m2 of Annamycin, or 230 mg/m2 of Annamycin, which Annamycin doses were specifically recommended by the FDA in the Company’s end of Phase 1B/2 meeting.
Additionally, war, terrorism, geopolitical uncertainties (such as the current war in Ukraine and in Israel) and other business interruptions could cause damage to, disrupt or cancel the conduct of our clinical trials on a global or regional basis, which could have a material adverse effect on our business, clinical sites, drug suppliers or vendors with which we do business.
War, terrorism, pandemics or the like, geopolitical uncertainties (such as the current war in Ukraine) and other business interruptions could cause damage to, disrupt or cancel the conduct of our clinical trials on a global or regional basis, which could have a material adverse effect on our business, clinical sites, drug suppliers or vendors with which we do business.
Additionally, recruiting and treatment concluded in 2024 for the externally funded Phase 1B/2 clinical trial studying an alternative dosing schedule of Annamycin for the treatment of STS lung mets in Poland. The investigator is expected to issue a publication in 2025 on this study.
Additionally, recruiting and treatment concluded in 2024 for the externally funded Phase 1B/2 clinical trial studying an alternative dosing schedule of Annamycin for the treatment of STS lung mets in Poland. The investigator is expected to issue a publication or CSR in 2026 on this study.
Table 1 - Clinical Summary as of this filing Unless CSR Completed – Data are as of March 1, 2025 - preliminary and subject to change Drug Candidate Trial / Indication / Location Phase (Funding Source: Internal unless noted as External) Status Comments Safety Summary Human Activity Summary Annamycin MB-104 / R/R AML / US 1 P1 Concluded; P2 replaced with MB-105 Maximum Dose allowed per protocol 120 mg/m 2 Met safety endpoints; no cardiotoxicity reported 14.3% MLFS (subtherapeutic dose level when compared to MB-105) Annamycin MB-105 / R/R AML / Poland 1/2 P1 concluded; P2 replaced with MB-106 Maximum Dose and RP2D 240 mg/m 2 Met safety endpoints; no cardiotoxicity reported 80% ORR in last cohort Annamycin in Combination with Cytarabine MB-106 / R/R AML / Europe 1B/2 22 subjects intent to treat. 2 subjects had an allergic reaction and did not receive full dose.
Table 1 - Clinical Summary as of this filing Unless CSR Completed – Data are as of March 1, 2026 - preliminary and subject to change (1) (5) Drug Candidate Trial / Indication / Location Phase (Funding Source: Internal unless noted as External) Status Comments Safety Summary (6) Human Activity Summary (2) (3) (4) Annamycin MB-104 / R/R AML / US 1 P1 Concluded; P2 replaced with MB-105 Maximum Dose allowed per protocol 120 mg/m 2 Met safety endpoints; no cardiotoxicity reported 14.3% MLFS (subtherapeutic dose level when compared to MB-105) Annamycin MB-105 / R/R AML / Poland 1/2 P1 concluded; P2 replaced with MB-106 Maximum Dose and RP2D 240 mg/m 2 Met safety endpoints; no cardiotoxicity reported 80% ORR in last cohort Annamycin in Combination with Cytarabine MB-106 / AML / Europe (7) 1B/2 22 subjects' safety evaluable. 2 subjects had an allergic reaction and did not receive full dose.
The lack of cardiotoxicity and the potential for efficacy of Annamycin have been demonstrated in 84 subjects treated to date.
The lack of cardiotoxicity and the potential for efficacy of Annamycin have been demonstrated in 90 subjects treated to date.
The Australian government provides an aggressive incentive for research and development carried out in their country. We believe having an Australian subsidiary could provide a great opportunity for quality, pre-clinical and clinical development and reduce the overall cost of our continued drug development efforts.
The Australian government provides an aggressive incentive for research and development carried out in their country. We believe having an Australian subsidiary has provided a great opportunity for quality, pre-clinical and clinical development and reduce the overall cost of our continued drug development efforts.
The MIRACLE study, subject to appropriate future filings with and potential additional feedback from the FDA and their foreign equivalents, utilizes an adaptive design whereby the first 75 to 90 subjects will be randomized (1:1:1) in Part A of the trial to receive high dose cytarabine (HiDAC) combined with either placebo, 190 mg/m2 of Annamycin, or 230 mg/m2 of Annamycin, which Annamycin doses were specifically recommended by the FDA in the Company’s end of Phase 1B/2 meeting.
The MIRACLE study, subject to appropriate future filings with and potential additional feedback from the FDA and their foreign equivalents, utilizes an adaptive design whereby the first 90 subjects will be randomized (1:1:1) in Part A of the trial to receive five days of HiDAC combined with three days of either placebo, 190 mg/m2 of Annamycin, or 230 mg/m2 of Annamycin, which Annamycin doses were specifically recommended by the FDA in the Company’s end of Phase 1B/2 meeting.
With respect to certain patents or patent applications, we are co-owners with MD Anderson, in which instances we have exclusively licensed MD Anderson’s rights in those patents or patent applications.
With respect to certain patents or patent applications, we are co-owners (due to being coinventors) with MD Anderson, in which instances we have exclusively licensed MD Anderson’s rights in those patents or patent applications.
We have rights to four issued US patents for WP1066. These patents claim WP1066 and other molecules, as well as methods of treating disease using WP1066. Foreign counterparts to the US patents are issued outside the US including in Europe. These patents have an international filing date in December 2004, and in certain instances have had the patent term adjusted.
These patents claim WP1066 and other molecules, as well as methods of treating disease using WP1066. Foreign counterparts to the US patents are issued outside the US including in Europe. These patents have an international filing date in December 2004, and in certain instances have had the patent term adjusted. WP1220.
Based upon the FDA minutes, we designed and began implementation of a Phase 2B/3 pivotal trial for the treatment of AML patients who are refractory to or relapsed after induction therapy. This MIRACLE trial will be a global trial, including sites in the US, Europe, Western Asia and the Middle East.
Based upon the FDA minutes, we designed and began implementation of a Phase 2B/3 pivotal trial, dubbed the MIRACLE trial, for the treatment of AML patients who are refractory to or relapsed after a single induction therapy. MIRACLE is a global trial, including sites in the US, Europe, Western Asia and the Middle East.
Anthracyclines are an important class of first line tools for physicians and while effective, their maximum lifetime dose in patients is limited due to concerns over cardiotoxicity.
Anthracyclines are an important class of first line tools for physicians and while effective, their rate of relapse is very high and their maximum lifetime dose in patients is limited due to concerns over cardiotoxicity.
Additionally, we will rely on external collaborations for testing other molecules in the WP1122 portfolio against other hard to treat viruses such as HIV, Dengue fever, and Zika. 13 Table of Contents Funding Strategy By “internally funded” we mean that the primary costs of the preclinical activity and clinical trials are funded and sponsored by us.
As the opportunity arises, we expect to rely on external collaborations for testing other molecules in the WP1122 portfolio against other hard to treat viruses such as HIV, Dengue fever, and Zika. 15 Table of Contents Funding Strategy By “internally funded” we mean that the primary costs of the preclinical activity and clinical trials are funded and sponsored by us.
Our Drug Candidate Programs Overview In the US and Europe, since our inception, we or independent investigators have approval to begin, are currently conducting or have completed fourteen internally or externally funded clinical trials for four of our drug candidates – Annamycin, WP1066, WP1220, and WP1122, as listed above.
Our Drug Candidate Programs Overview In the US and Europe, since our inception, we or independent investigators are actively planning, have approval to begin, are currently conducting or have completed eighteen internally or externally funded clinical trials for our drug candidates – Annamycin, WP1066, WP1220, and WP1122, as listed above.
We believe WP1122 should be well suited as a treatment for highly glycolytic cancers such as GBM and pancreatic cancer. 16 Table of Contents In addition to the market for GBM described above, pancreatic cancer is a rare and difficult to treat form of cancer.
We believe WP1122 could be well suited as a treatment for highly glycolytic cancers such as GBM and pancreatic cancer. In addition to the market for GBM described above, pancreatic cancer is a rare and difficult to treat form of cancer.
Consistent with the FDA’s recommendations, in the adaptive MIRACLE trial we plan to utilize a double-blind, placebo-controlled design, where we will compare AnnAraC versus a control arm of high dose cytarabine (HiDAC) plus placebo and we will rely solely on CR (complete remission) at approximately one month as the primary endpoint.
Consistent with the FDA’s recommendations, in the adaptive MIRACLE trial we plan to utilize a double-blind, placebo-controlled design, where we will compare AnnAraC versus the control arm of HiDAC plus placebo and we will rely solely on CR (complete remission) after a single cycle of treatment (at approximately one month) as the primary endpoint.
Based on our discussions with the FDA, we amended in November 2024 our MB-104 investigational new drug application or IND for MB-108. In the amendment with the new MIRACLE protocol, the trial will allow, for the first time in the US for AML subjects, dosing above the lifetime maximum allowable dose for currently prescribed anthracyclines.
Based on our discussions with the FDA, we amended in November 2024 our MB-104 investigational new drug application or IND for MB-108. In the amendment with the new MIRACLE protocol, the trial allows, for the first time in the US, for AML subjects to be dosed above the lifetime maximum allowable dose for currently prescribed anthracyclines.
WP1066 WP1066 is our flagship Immune/Transcription Modulator. It has been the subject of over 50 peer-reviewed articles and its activity against p-STAT3 has now been validated in independent labs around the world. This discovery was inspired by a naturally occurring compound (caffeic acid) in propolis (from honeybees).
It has been the subject of over 50 peer-reviewed articles and its activity against p-STAT3 has now been validated in independent labs around the world. This discovery was inspired by a naturally occurring compound (caffeic acid) in propolis (from honeybees).
The preliminary data to date are 63% (5 of 8) have received greater at least two cycles (approximately 2 months) of therapy where we have assumed stable disease (SD) through two cycles and 38% (3 of 8) subjects received 4 cycles (with us assuming SD through four cycles). This is based on a preliminary data.
The preliminary data to date are 63% (5 of 8) have received greater at least two cycles (approximately 2 months) of therapy where we have assumed stable disease (SD) through two cycles and 38% (3 of 8) subjects received 4 cycles (with us assuming SD through four cycles). The data are preliminary and subject to change.
However, in two of the PRs, as noted by the site, subjects' bone marrow blast counts were successfully decreased to below 5%, however these subjects were still designated as a PR by the sub-investigator at that site.
However, in two of the PRs, as noted by the site, subjects' bone marrow blast counts were successfully decreased to below 5%, however these subjects were still designated as a PR by the sub-investigator at that site. Our Expert noted no cardiotoxicity in subjects in this trial.
This first line regimen is often referred to as “7+3.” We estimate that only about 36% of these patients, or approximately 18% of overall AML patients, will have a durable CR as a result of first line therapy, meaning the cancerous cells in their bone marrow have been reduced to 5% or less.
This first line regimen is often referred to as “7+3.” We estimate that only about 36% of these patients, or approximately 18% of overall AML patients, will have a durable CR as a result of first line therapy, meaning the cancerous cells in their bone marrow have been reduced to 5% or less and that this remission lasts long enough to have a meaningful impact on their overall survival.
In the case of acute leukemia, anthracyclines are typically used in “induction therapy,” where the goal is often to induce sufficient remission of patients’ bloodborne tumor cells to allow for a potentially curative bone marrow transplant. Two key factors limit the safety and effectiveness of anthracyclines: cardiotoxicity and multidrug resistance.
In the case of acute leukemia, anthracyclines are typically used in “induction therapy,” where the goal is often to induce sufficient remission of patients’ bloodborne tumor cells to allow for a potentially curative bone marrow transplant. 7 Table of Contents Several key factors limit the safety and effectiveness of anthracyclines: cardiotoxicity, multidrug resistance and tissue/organ distribution and uptake.
The FDA also wanted to see the durability of response (DoR) and overall survival as secondary endpoints, as well as data for patients beyond 2nd line, which is why our plan includes a follow-on MIRACLE2 trial in 3rd line patients starting once the optimum dose is established in the MIRACLE trial.
The FDA also wanted to see overall survival as a secondary endpoint and the DoR as an exploratory endpoint. The FDA also wanted data for patients beyond 2nd line, which is why our plan includes a follow-on MIRACLE2 trial in 3rd line patients starting once the optimum dose is established in the MIRACLE trial.
Our pharmaceutical product candidates may not be considered medically necessary or cost-effective. To the extent other drugs or therapies are found to be more effective than our products, payers may elect to cover such therapies in lieu of our products and/or reimburse our products at a lower rate. Different pricing and reimbursement schemes exist in other countries.
To the extent other drugs or therapies are found to be more effective than our products, payers may elect to cover such therapies in lieu of our products and/or reimburse our products at a lower rate. Different pricing and reimbursement schemes exist in other countries.
The subjects treated to date have included those who were initially deemed unfit for intensive chemotherapy and the initial, preliminary data suggest that Annamycin’s safety and tolerability profile may make the product suitable for those patients, too. Annamycin Clinical Trials – AML We have studied Annamycin in three internally funded AML clinical trials. These trials are MB-104, MB-105, and MB-106.
The subjects treated to date have included those who were initially deemed unfit for intensive chemotherapy and the initial, preliminary data suggest that Annamycin’s safety and tolerability profile may make the product suitable for those patients, too. 9 Table of Contents Annamycin Clinical Trials – AML We have studied Annamycin in three internally funded AML clinical trials.
Each of our three core technologies is based substantially on discoveries made at and licensed from the University of Texas MD Anderson Cancer Center (MD Anderson) in Houston, Texas, and features one or more drugs that have successfully completed a Phase 1 clinical trial.
Each of our three core technologies is based substantially on discoveries made at, made in conjunction with, and/or licensed from the University of Texas MD Anderson Cancer Center (MD Anderson) in Houston, Texas. For each of the core technologies, one or more drug candidates have successfully completed a Phase 1 or greater clinical trial.
We estimate that only around 40% of AML patients are afforded an opportunity to overcome their disease through a curative bone marrow transplant or through lasting remission. We believe this aligns with the published statistic that the 5-year survival rate for AML is only 29%.
We estimate that only around 40% of AML patients are afforded an opportunity to overcome their disease through a curative bone marrow transplant or through lasting remission. We believe this aligns with the published statistic that the 5-year survival rate for AML is roughly 30% (https://seer.cancer.gov/statfacts/html/amyl.html).
In 2022, we determined with the data from the Phase 1a trial that the maximum tolerated dose (MTD) for WP1122 is a daily cumulative dose of 32 mg/kg in two divided doses for seven days, and we concluded the Phase 1a study of WP1122. We believe this will advance future studies of WP1122 in antiviral and oncology indications.
In 2022, we determined with the data from the Phase 1a trial that the maximum tolerated dose (MTD) for WP1122 is a daily cumulative dose of 32 mg/kg in two divided doses for seven days, and we concluded the Phase 1a study of WP1122.
Subsequently, in February 2025 we received FDA feedback and guidance on our IND amendment, noted above, that allowed a reduction in the size of our Phase 3 pivotal trial protocol to 220. With their feedback and our response, all the major aspects of the trial remain unchanged.
Subsequently, in February 2025 we received FDA feedback and guidance on our IND amendment, noted above, that allowed a reduction in the size of Part B of the trial to 222 subjects. With their feedback and our response, all the major aspects of the trial remain unchanged.
The data are preliminary and subject to change. 10 Table of Contents Along with the results in STS lung metastases, our animal models have shown activity in other lung metastases, including osteosarcoma, colorectal and triple negative breast cancer, as well as meaningful concentration levels of Annamycin in the liver, spleen and pancreas.
Along with the results in STS lung metastases, our animal models have shown activity in other lung metastases, including osteosarcoma, colorectal and triple negative breast cancer, as well as meaningful concentration levels of Annamycin in the liver, spleen and pancreas.
The voucher can be used by the sponsor for a subsequent application that would not in its own right qualify for priority review, or it may be sold to another company for that use. In either case, a RPDPRV may have significant value.
The voucher can be used by the sponsor for a subsequent application that would not in its own right qualify for priority review, or it may be sold to another company for that use. In either case, a RPDPRV may have significant value. In February 2026, the statute was amended to continue the RPDPRV program through September 2029.
Other milestone payments for submission of an NDA to the FDA and receipt of first marketing approval for sale of a license product can be as high as $0.6 million.
Milestone payments for the commencement of phase II and phase III clinical trials can cost as high as $0.5 million. Other milestone payments for submission of an NDA to the FDA and receipt of first marketing approval for sale of a license product can be as high as $0.6 million.
Simply substituting Annamycin for the currently used anthracycline in a similar 7+3 (or as is the case in MB-106, 5+3) regimen would therefore represent a familiar and well-practiced treatment modality.
Simply substituting Annamycin for the currently used anthracycline in a similar 7+3 regimen would therefore represent a familiar and well-practiced treatment modality.
Royalty payments can range in the single digits as a percent of net sales on drug products or flat fees as high as $0.6 million, depending upon certain terms and conditions. Not all of these payments are applicable to every drug.
Certain agreements contain a milestone payment for the initiation of Phase 3 clinical trials. Royalty payments can range in the single digits as a percent of net sales on drug products or flat fees as high as $0.6 million, depending upon certain terms and conditions. Not all payments are applicable to every drug.
Consistent with the FDA’s recommendations, in the adaptive MIRACLE trial we plan to utilize a double-blind, placebo-controlled design, where we will compare AnnAraC versus the control arm of high dose cytarabine (HiDAC) plus placebo and we will rely solely on CR (complete remission) at approximately one month as the primary endpoint.
Consistent with the FDA’s recommendations, in the adaptive MIRACLE trial we are utilizing a double-blind, placebo-controlled design, where we will compare two arms of AnnAraC, with two different strengths of Annamycin, versus a control arm of high dose cytarabine (HiDAC) plus placebo and we will rely solely on CR (complete remission) after a single cycle of treatment (at approximately one month) as the primary endpoint.
Improvement was noted within seven days of treatment initiation and maintained 1 month after discontinuation. Of the eleven lesions, 45% exhibited a CR or a 50% or more reduction in CAILS and 55% exhibited stable disease with 100% showing a clinical benefit. Independent dermatologic review based on photographic documentation was conducted and corroborated these findings.
Of the eleven lesions, 45% exhibited a CR or a 50% or more reduction in CAILS and 55% exhibited stable disease with 100% showing a clinical benefit. Independent dermatologic review based on photographic documentation was conducted and corroborated these findings.
The MIRACLE trial will be a global trial, including sites in the US, Europe, Western Asia and the Middle East. The FDA’s Divisions of Hematologic Malignancies I and Cardiology and Nephrology, as well as related divisions, were involved in the review of the data showing no cardiotoxicity in MB-106 and prior clinical trials.
The MIRACLE trial is a global trial, including sites in the US, Europe or the European Union (EU), and Eastern Europe. The FDA’s Divisions of Hematologic Malignancies I and Cardiology and Nephrology, as well as related divisions, were involved in the review of the data showing no cardiotoxicity in MB-106 and prior clinical trials.
Moleculin’s novel candidate for the treatment of acute myeloid leukemia (AML) and soft tissue sarcoma lung metastases (STS lung mets) uses a unique lipid-based delivery technology. In addition to the issued ‘118 and expected ‘634 U.S. patents, we have additional patent applications pending in the US and in major jurisdictions worldwide. p-STAT3 Inhibitors WP1066.
Moleculin’s novel candidate for the treatment of AML and STS uses a unique lipid-based delivery technology. In addition to the four issued U.S. patents, we have additional patent applications pending in the US and in major jurisdictions worldwide. p-STAT3 Inhibitors WP1066. We have rights to four issued US patents for WP1066.
Accordingly, we also intend to pursue approval for pediatric use in these conditions when practicable. Soft tissue sarcoma is a broad term for cancers that start in soft tissues (muscle, tendons, fat, bone, lymph and blood vessels, and nerves).
Accordingly, we also intend to pursue approval for pediatric use in these conditions when practicable. Soft tissue sarcoma is a broad term for cancers that start in soft tissues (muscle, tendons, fat, bone, lymph and blood vessels, and nerves). These cancers can develop anywhere in the body but are found mostly in the arms, legs, chest, and abdomen.
We believe this overcomes the limitations of many other drugs designed to inhibit STAT3 activity by blocking upstream receptors. 11 Table of Contents Another important attribute of WP1066 (unlike some of our other Immune/Transcription Modulators) is its apparent ability in pre-clinical testing to cross the blood brain barrier, which we believe makes it a good candidate for potentially treating brain tumors and other malignancies of the central nervous system.
Another important attribute of WP1066 (unlike some of our other Immune/Transcription Modulators) is its apparent ability in pre-clinical testing to cross the blood brain barrier, which we believe makes it a good candidate for potentially treating brain tumors and other malignancies of the central nervous system.
We believe the FDA minutes, reflect a positive discussion and the meeting resulted in the design and implementation of a Phase 2B/3 pivotal trial for the treatment of AML patients who are refractory to or relapsed after induction therapy. This MIRACLE trial will be a global trial, including sites in the US, Europe, Western Asia and the Middle East.
We believe the FDA minutes reflect a positive discussion and the meeting resulted in the design and implementation of a Phase 2B/3 pivotal trial for the treatment of AML patients who are refractory to or relapsed after induction therapy.
In our MB-106 Phase 1B/2 all-comers clinical trial using Annamycin in combination with Cytarabine for the treatment of AML, we concluded recruitment and treatment with 22 subjects recruited on an Intent-To-Treat (ITT) basis.
The CSR was published in 2025. In our MB-106 Phase 1B/2 all-comers clinical trial using Annamycin in combination with Cytarabine for the treatment of AML, we concluded recruitment and treatment with 23 subjects recruited on an Intent-To-Treat (ITT) basis (22 subjects' safety evaluable with one subject enrolled but never treated).
In part, because of the emphasis placed on alternatives to chemotherapy, we believe that not enough has been done to improve chemotherapeutic agents to make them safer, especially with regard to cardiotoxicity (damage to the heart), and more effective.
Despite the progress made with immunotherapy and precision medicine, the first-line treatment for many cancers continues to include chemotherapy. In part, because of the emphasis placed on alternatives to chemotherapy, we believe that not enough has been done to improve chemotherapeutic agents to make them safer, especially with regard to cardiotoxicity (damage to the heart), and more effective.

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Item 1A. Risk Factors

Risk Factors — what could go wrong, per management

78 edited+172 added−24 removed339 unchanged
Biggest changeFast Track designation may also be rescinded if the FDA believes the designation is no longer supported by data from our clinical development program. • Interim or preliminary data from our clinical trials that we announce or publish from time to time may change as more patient data become available and are subject to audit and verification procedures that could result in material changes in the final data. • We may not be able to conduct, or contract others to conduct, animal testing in the future, which could harm our research and development activities. • We, or our third-party manufacturers, may be unable to successfully scale-up manufacturing of our product candidates in sufficient quality and quantity, which would delay or prevent us from developing our product candidates and commercializing approved products, if any. • Even if we obtain regulatory approvals for our product candidates, they will remain subject to ongoing regulatory oversight. • Recently enacted legislation, future legislation and healthcare reform measures may increase the difficulty and cost for us to obtain marketing approval for and commercialize Annamycin and any future product candidates and may affect the prices we may set. 24 Table of Contents Risks Related to Our Intellectual Property • The composition of matter patent for Annamycin has expired, and other patents have not yet been issued, and may not be issued. • The intellectual property rights we have licensed from MD Anderson are subject to the rights of the US government. • We may incur substantial costs as a result of litigation or other proceedings relating to patent and other intellectual property rights. • We may be subject to claims that our employees have wrongfully used or disclosed alleged trade secrets of their former employers. • If we are not able to adequately prevent disclosure of trade secrets and other proprietary information, the value of our technology and products could be significantly diminished. • If we breach any of the agreements under which we license patent rights or if we fail to meet certain development deadlines, pay certain fees including extension fees or exercise certain rights to technology, we could lose or fail to obtain license rights that are important to our business. • We will not be able to protect our intellectual property rights throughout the world.
Biggest changeFast Track designation may also be rescinded if the FDA believes the designation is no longer supported by data from our clinical development program. • Interim or preliminary data from our clinical trials that we announce or publish from time to time may change as more patient data become available and are subject to audit and verification procedures that could result in material changes in the final data. • We may not be able to conduct, or contract others to conduct, animal testing in the future, which could harm our research and development activities. • We, or our third-party manufacturers, may be unable to successfully scale-up manufacturing of our product candidates in sufficient quality and quantity, which would delay or prevent us from developing our product candidates and commercializing approved products, if any. • Even if we obtain regulatory approvals for our product candidates, they will remain subject to ongoing regulatory oversight. • Recently enacted legislation, future legislation and healthcare reform measures may increase the difficulty and cost for us to obtain marketing approval for and commercialize Annamycin and any future product candidates and may affect the prices we may set. 26 Table of Contents Risks Related to Our Intellectual Property • We rely on patents and other intellectual property rights to protect our product candidates, the attainment, enforcement, defense and maintenance of which may be challenging and costly and failure to do so could impair our business. • We enjoy only limited geographical protection with respect to certain patents and may not be able to protect our intellectual property rights throughout the world. • The intellectual property rights we have licensed from MD Anderson are subject to the rights of the US government. • Our patents and other proprietary rights may not adequately protect our technologies and product candidates and may not necessarily address all potential threats to our competitive advantage. • We may become subject to third parties’ claims alleging infringement of third-party patents and proprietary rights, or we may be involved in lawsuits to protect or enforce our patents and other proprietary rights, which could be costly and time consuming, delay or prevent the development and commercialization of our product candidates, or put our patents and other proprietary rights at risk. • We may not identify relevant third-party patents or may incorrectly interpret the relevance, scope or expiration of a third-party patent which might adversely affect our ability to develop, manufacture and market our product candidates. • If we breach any of the agreements under which we license patent rights or if we fail to meet certain development deadlines, pay certain fees including extension fees or exercise certain rights to technology, we could lose or fail to obtain license rights that are important to our business. • We may not be successful in maintaining necessary rights to our product candidates or obtaining patent or other intellectual property rights important to our business through acquisitions and in-licenses. • Obtaining and maintaining our patent protection depends on compliance with various procedural, document submission, fee payment, and other requirements imposed by governmental patent agencies, and our patent protection could be reduced or eliminated for non-compliance with these requirements. • We may be subject to claims challenging the inventorship of our patents and other intellectual property. • Changes in patent laws or patent jurisprudence could diminish the value of patents in general, thereby impairing our ability to protect our product candidates. • If we do not obtain protection under the Hatch-Waxman Amendments and similar non-U.S. legislation for extending the term of patents covering our product candidates, our ability to compete effectively could be impaired. • If our trademarks and trade names are not adequately protected, we may not be able to build name recognition in our markets of interest and our competitive position may be adversely affected. • If we are unable to protect the confidentiality of our trade secrets and know-how, our business and competitive position would be harmed. • We may be subject to claims by third parties asserting that we or our employees have misappropriated third-party intellectual property, or claiming ownership of what we regard as our own intellectual property.
If the FDA or its EU equivalent requires that we perform additional nonclinical studies or clinical trials, our expenses would further increase beyond what we currently expect and the anticipated timing of any potential approval of Annamycin would likely be delayed.
If the FDA or its EU equivalent requires that we perform additional nonclinical studies or clinical trials, our expenses would further increase beyond what we currently expect and the anticipated timing of any potential approval of Annamycin would likely be delayed.
Risks Related to Regulatory Approval and the Development and Commercialization of our Drug Candidates • We are developing our drugs to treat patients who are extremely or terminally ill, and patient deaths that occur in our clinical trials could negatively impact our business even if such outcomes are not shown to be related to our drugs. • We are conducting important clinical trials in the US and Europe, and assessing additional countries in which to perform preclinical studies and clinical trials and the risks associated with conducting research and clinical trials abroad could materially adversely affect our business. • There are limited suppliers for active pharmaceutical ingredients (API) used in in our drug candidates and we utilize a single source for such API for certain of our drug candidates.
Risks Related to Regulatory Approval and the Development and Commercialization of our Drug Candidates • We are developing our drug candidates to treat patients who are extremely or terminally ill, and patient deaths that occur in our clinical trials could negatively impact our business even if such outcomes are not shown to be related to our drug candidates. • We are conducting important clinical trials in the US and Europe, and assessing additional countries in which to perform preclinical studies and clinical trials and the risks associated with conducting research and clinical trials abroad could materially adversely affect our business. • There are limited suppliers for active pharmaceutical ingredients (API) used in our drug candidates and we utilize a single source for such API for certain of our drug candidates.
Subject enrollment is affected by a variety factors including, among others: • severity of the disease under investigation; • design of the trial protocol and size of the patient population required for analysis of the trial’s primary endpoints; • size of the patient population; • eligibility criteria for the trial in question; • perceived risks and benefits of the product candidate being tested; • willingness or availability of subjects to participate in our clinical trials; • willingness of the investigators to accept the trial design, including the control arm, of the study; • proximity and availability of clinical trial sites for prospective subjects; • our ability to recruit clinical trial investigators with the appropriate competencies and experience; • availability of competing vaccines and/or therapies and related clinical trials; 30 Table of Contents • efforts to facilitate timely enrollment in clinical trials; • our ability to obtain and maintain subject consents; • the risk that subjects enrolled in clinical trials will drop out of the trials before completion; • subject referral practices of physicians; and • ability to monitor subjects adequately during and after treatment.
Subject enrollment is affected by a variety factors including, among others: • severity of the disease under investigation; 32 Table of Contents • design of the trial protocol and size of the patient population required for analysis of the trial’s primary endpoints; • size of the patient population; • eligibility criteria for the trial in question; • perceived risks and benefits of the product candidate being tested; • willingness or availability of subjects to participate in our clinical trials; • willingness of the investigators to accept the trial design, including the control arm, of the study; • proximity and availability of clinical trial sites for prospective subjects; • our ability to recruit clinical trial investigators with the appropriate competencies and experience; • availability of competing vaccines and/or therapies and related clinical trials; • efforts to facilitate timely enrollment in clinical trials; • our ability to obtain and maintain subject consents; • the risk that subjects enrolled in clinical trials will drop out of the trials before completion; • subject referral practices of physicians; and • ability to monitor subjects adequately during and after treatment.
Factors relating to our business that may contribute to these fluctuations include: • any delays in regulatory review and approval of our product candidates in clinical development, including our ability to receive approval from the FDA or the Polish authorities for our drugs in clinical trials; • delays in the commencement, enrollment and timing of clinical trials; • difficulties in identifying subjects suffering from our target indications; • the success of our clinical trials through all phases of clinical development; • potential side effects of our product candidates that could delay or prevent approval or cause an approved drug to be taken off the market; • our ability to obtain additional funding to develop drug candidates; • our ability to identify and develop additional drug candidates beyond Annamycin and our WP1066 and WP1122 Portfolios; • competition from existing products or new products that continue to emerge; • the ability of subjects or healthcare providers to obtain coverage or sufficient reimbursement for our products; • our ability to adhere to clinical trial requirements directly or with third parties such as contract research organizations (CROs); • our dependency on third-party manufacturers to manufacture our products and key ingredients; • our ability to establish or maintain collaborations, licensing or other arrangements, particularly with MD Anderson; • our ability to defend against any challenges to our intellectual property including, claims of patent infringement; • our ability to enforce our intellectual property rights against potential competitors; • our ability to secure additional intellectual property protection for our developing drug candidates and associated technologies; • our ability to attract and retain key personnel to manage our business effectively; and • potential product liability claims.
Factors relating to our business that may contribute to these fluctuations include: • any delays in regulatory review and approval of our product candidates in clinical development, including our ability to receive approval from the FDA or the Polish authorities for our drugs in clinical trials; • delays in the commencement, enrollment and timing of clinical trials; • difficulties in identifying subjects suffering from our target indications; • the success of our clinical trials through all phases of clinical development; • potential side effects of our product candidates that could delay or prevent approval or cause an approved drug to be taken off the market; • our ability to obtain additional funding to develop drug candidates; • our ability to identify and develop additional drug candidates beyond Annamycin and our WP1066 and WP1122 Portfolios; • competition from existing products or new products that continue to emerge; • the ability of subjects or healthcare providers to obtain coverage or sufficient reimbursement for our products; • our ability to adhere to clinical trial requirements directly or with third parties such as contract research organizations (CROs); • our dependency on third-party manufacturers to manufacture our products and key ingredients; • our ability to establish or maintain collaborations, licensing or other arrangements, particularly with MD Anderson; • our ability to defend against any challenges to our intellectual property including, claims of patent infringement; • our ability to enforce our intellectual property rights against potential competitors; • our ability to secure additional intellectual property protection for our developing drug candidates and associated technologies; 44 Table of Contents • our ability to attract and retain key personnel to manage our business effectively; and • potential product liability claims.
We have in the past, and intend in the future, to publicly disclose preliminary data from our clinical trials, which is based on a preliminary analysis of then-available data, and the results and related findings and conclusions are subject to change following a full analyses of all data related to the particular trial.
We have in the past, and intend in the future, to publicly disclose preliminary data from our clinical trials, which is based on a preliminary analysis of then-available data, and the results and related findings and conclusions are subject to change following a full analysis of all data related to the particular trial.
Finally, a data breach affecting sensitive personal information, including health information, could result in significant legal and financial exposure and reputational damages that could potentially have an adverse effect on our business. 40 Table of Contents EU Member States, Switzerland and other countries have also adopted data protection laws and regulations, which impose significant compliance obligations.
Finally, a data breach affecting sensitive personal information, including health information, could result in significant legal and financial exposure and reputational damages that could potentially have an adverse effect on our business. 47 Table of Contents EU Member States, Switzerland and other countries have also adopted data protection laws and regulations, which impose significant compliance obligations.
If we fail to meet our payment obligations, our license agreements could be terminated, which would materially and adversely affect our business operations and financial condition. 41 Table of Contents New tax laws or regulations that are enacted or existing tax laws and regulations that are interpreted, modified or applied adversely to us or our customers may have a material adverse effect on our business and financial condition.
If we fail to meet our payment obligations, our license agreements could be terminated, which would materially and adversely affect our business operations and financial condition. 48 Table of Contents New tax laws or regulations that are enacted or existing tax laws and regulations that are interpreted, modified or applied adversely to us or our customers may have a material adverse effect on our business and financial condition.
If subjects are unwilling to participate in our trials because of negative publicity from adverse events in the biotechnology industries, public perception of vaccine safety issues or for other reasons, including competitive clinical trials for similar patient populations, the timeline for recruiting subjects, conducting studies and obtaining regulatory approval of potential products may be delayed.
If subjects are unwilling to participate in our trials because of negative publicity from adverse events in the biotechnology industry, public perception of vaccine safety issues or for other reasons, including competitive clinical trials for similar patient populations, the timeline for recruiting subjects, conducting studies and obtaining regulatory approval of potential products may be delayed.
The amount and timing of our future funding requirements will depend on many factors, including but not limited to: • whether our plan for clinical trials will be completed on a timely basis and, if completed, whether we will be able to publicly announce results from our phase I/II clinical trials in accordance with our announced milestones; • whether the results of our clinical trials will be announced on a timely basis and, when announced, whether such results are in accordance with our expectations or our announced milestones; • whether we are successful in obtaining the benefits of FDA’s expedited development and review programs related to Annamycin or our other drug candidates; • the progress, costs, results of and timing of our clinical trials and also of our preclinical studies; • the outcome, costs and timing of seeking and obtaining FDA and any other regulatory approvals; • the costs associated with securing and establishing commercialization and manufacturing capabilities; • market acceptance of our product candidates; • the costs of acquiring, licensing or investing in businesses, products, product candidates and technologies; • our ability to maintain, expand and enforce the scope of our intellectual property portfolio, including the amount and timing of any payments we may be required to make, or that we may receive, in connection with the licensing, filing, prosecution, defense and enforcement of any patents or other intellectual property rights; • our need and ability to hire additional management and scientific and medical personnel; • the effect of competing drug candidates and new product approvals; • our need to implement additional internal systems and infrastructure, including financial and reporting systems; and • the economic and other terms, timing of and success of our existing licensing arrangements and any collaboration, licensing or other arrangements into which we may enter in the future. 36 Table of Contents Some of these factors are outside of our control.
The amount and timing of our future funding requirements will depend on many factors, including but not limited to: • whether our plan for clinical trials will be completed on a timely basis and, if completed, whether we will be able to publicly announce results from our phase I/II clinical trials in accordance with our announced milestones; • whether the results of our clinical trials will be announced on a timely basis and, when announced, whether such results are in accordance with our expectations or our announced milestones; • whether we are successful in obtaining the benefits of FDA’s expedited development and review programs related to Annamycin or our other drug candidates; • the progress, costs, results of and timing of our clinical trials and also of our preclinical studies; • the outcome, costs and timing of seeking and obtaining FDA and any other regulatory approvals; • the costs associated with securing and establishing commercialization and manufacturing capabilities; 43 Table of Contents • market acceptance of our product candidates; • the costs of acquiring, licensing or investing in businesses, products, product candidates and technologies; • our ability to maintain, expand and enforce the scope of our intellectual property portfolio, including the amount and timing of any payments we may be required to make, or that we may receive, in connection with the licensing, filing, prosecution, defense and enforcement of any patents or other intellectual property rights; • our need and ability to hire additional management and scientific and medical personnel; • the effect of competing drug candidates and new product approvals; • our need to implement additional internal systems and infrastructure, including financial and reporting systems; and • the economic and other terms, timing of and success of our existing licensing arrangements and any collaboration, licensing or other arrangements into which we may enter in the future.
We may not be successful in our efforts to establish and implement collaborations or other alternative arrangements for the development of our product candidates. 38 Table of Contents When we collaborate with a third party for development and commercialization of a product candidate, we can expect to relinquish some or all of the control over the future success of that product candidate to the third party.
We may not be successful in our efforts to establish and implement collaborations or other alternative arrangements for the development of our product candidates. 45 Table of Contents When we collaborate with a third party for development and commercialization of a product candidate, we can expect to relinquish some or all of the control over the future success of that product candidate to the third party.
As a result of the accounting for our acquisition of Moleculin, LLC and the agreement we, on Moleculin, LLC’s behalf, entered into with Houston Pharmaceuticals, Inc., we have carried on our balance sheet within intangible assets in-process research and development (IPR&D) of $11.1 million as of December 31, 2024.
As a result of the accounting for our acquisition of Moleculin, LLC and the agreement we, on Moleculin, LLC’s behalf, entered into with Houston Pharmaceuticals, Inc., we have carried on our balance sheet within intangible assets in-process research and development (IPR&D) of $11.1 million as of December 31, 2025.
We have obtained a royalty-bearing, worldwide, exclusive license to intellectual property rights, including patent rights related to our WP1066 Portfolio and WP1122 Portfolio drug product candidates from MD Anderson. Some of our licensed intellectual property rights from MD Anderson have been developed in the course of research funded by the US government.
We have obtained or have rights to a royalty-bearing, worldwide, exclusive license to intellectual property rights, including patent rights related to our Annamycin, WP1066 Portfolio and WP1122 Portfolio drug product candidates from MD Anderson. Some of our licensed intellectual property rights from MD Anderson have been developed in the course of research funded by the US government.
If we submit a NDA to the FDA, the FDA must decide whether to accept or reject the submission for filing. We cannot be certain that any submissions will be accepted for filing and review by the FDA. Regulators in other jurisdictions have their own procedures for approval of product candidates.
If we submit an NDA to the FDA, the FDA must decide whether to accept or reject the submission for filing. We cannot be certain that any submissions will be accepted for filing and review by the FDA. Regulators in other jurisdictions have their own procedures for approval of product candidates.
We are pursuing additional patents with claims directed to Annamycin drug product formulations and the methods of use of Annamycin to treat relapsed or refractory AML and other conditions, and methods for its synthesis, as the composition of matter patent protection for Annamycin has expired.
We are pursuing additional patents with claims directed to Annamycin drug product formulations and the methods of use of Annamycin to treat relapsed or refractory AML and other conditions, and methods for its manufacture, as the composition of matter patent protection for Annamycin has expired.
However, ODD may enable market exclusivity of 7 years from the date of approval of a NDA in the United States. During that period the FDA generally could not approve another product containing the same drug for the same designated indication.
However, ODD may enable market exclusivity of 7 years from the date of approval of an NDA in the United States. During that period the FDA generally could not approve another product containing the same drug for the same designated indication.
Our board of directors has the authority, without action or vote of the stockholders, to issue all or any part of our authorized but unissued shares of common or preferred stock. Our certificate of incorporation authorizes us to issue up to 100,000,000 shares of common stock and 5,000,000 shares of preferred stock.
Our board of directors has the authority, without action or vote of the stockholders, to issue all or any part of our authorized but unissued shares of common or preferred stock. Our certificate of incorporation authorizes us to issue up to 500,000,000 shares of common stock and 5,000,000 shares of preferred stock.
Any claims for indemnification made by our directors or officers could impact our cash resources and our ability to fund the business. 44 Table of Contents We have no intention of declaring dividends in the foreseeable future.
Any claims for indemnification made by our directors or officers could impact our cash resources and our ability to fund the business. 51 Table of Contents We have no intention of declaring dividends in the foreseeable future.
Our failure to retain key personnel or consultants could materially harm our business. 39 Table of Contents In addition, we have scientific and clinical advisors and consultants who assist us in formulating our research, development, and clinical strategies.
Our failure to retain key personnel or consultants could materially harm our business. 46 Table of Contents In addition, we have scientific and clinical advisors and consultants who assist us in formulating our research, development, and clinical strategies.
Our drug product candidate, Annamycin, is currently in a physician-sponsored Phase 1b/2 clinical trial in Poland for the treatment of STS lung metastases. These physician-sponsored trials are an important part of our clinical development plan. Although we provide drug product and other minor supporting activities for these clinical trials, we are not otherwise directly involved in these physician-sponsored trials.
Our drug product candidate, Annamycin, was in a physician-sponsored Phase 1b/2 clinical trial in Poland for the treatment of STS lung metastases. These physician-sponsored trials are an important part of our clinical development plan. Although we provide drug product and other minor supporting activities for these clinical trials, we are not otherwise directly involved in these physician-sponsored trials.
Accordingly, the results of any historical quarterly or annual periods should not be relied upon as indications of future operating performance. 37 Table of Contents We have in the past completed related party transactions that were not conducted on an arm’s length basis.
Accordingly, the results of any historical quarterly or annual periods should not be relied upon as indications of future operating performance. We have in the past completed related party transactions that were not conducted on an arm’s length basis.
The exercise of the options and warrants will dilute the voting interest of the owners of presently outstanding shares by adding a substantial number of additional shares of our common stock. 42 Table of Contents As a biotechnology company, we are at increased risk of securities class action litigation.
The exercise of the options and warrants will dilute the voting interest of the owners of presently outstanding shares by adding a substantial number of additional shares of our common stock. As a biotechnology company, we are at increased risk of securities class action litigation.
Our existing capital resources are not sufficient to enable us to complete the development and commercialization of our product candidates, or to initiate any clinical trials or additional development work needed for any other drug candidates. Accordingly, we will need to raise additional funds in the future.
Some of these factors are outside of our control. Our existing capital resources are not sufficient to enable us to complete the development and commercialization of our product candidates, or to initiate any clinical trials or additional development work needed for any other drug candidates. Accordingly, we will need to raise additional funds in the future.
Responding to proxy contests and other actions by such activist investors or others in the future could be costly and time-consuming, disrupt our operations and divert the attention of our Board of Directors and senior management from the pursuit of our business strategies, which could adversely affect our results of operations and financial condition. ITEM 1B. UNRESOLVED STAFF COMMENTS None.
Responding to proxy contests and other actions by such activist investors or others in the future could be costly and time-consuming, disrupt our operations and divert the attention of our Board of Directors and senior management from the pursuit of our business strategies, which could adversely affect our results of operations and financial condition.
We have never been profitable and do not expect to be profitable in the foreseeable future. We have not yet submitted any drug candidates for approval by regulatory authorities in the United States or elsewhere. For the year ended December 31, 2024, we incurred a net loss of $21.8 million.
We have never been profitable and do not expect to be profitable in the foreseeable future. We have not yet submitted any drug candidates for approval by regulatory authorities in the United States or elsewhere. For the year ended December 31, 2025, we incurred a net loss of $33.6 million.
We had an accumulated deficit of $153.4 million as of December 31, 2024. To date, we have devoted most of our financial resources to research and development, including our drug discovery research, preclinical development activities and clinical trial preparation, as well as corporate overhead. We have not generated any revenues from product sales.
We had an accumulated deficit of $206.0 million as of December 31, 2025. To date, we have devoted most of our financial resources to research and development, including our drug discovery research, preclinical development activities and clinical trial preparation, as well as corporate overhead. We have not generated any revenues from product sales.
Over the past two years, our stock price has ranged from a high o f $30.00 to a low of $0.42 (t aking into account the reverse stock splits we have completed), and the market price of our common stock is likely to continue to be highly volatile and could fluctuate widely in response to various factors, many of which are beyond our control.
Over the past two years, our stock price has ranged from a high o f $356.19 to a low of $3.31 (t aking into account the reverse stock splits we have completed), and the market price of our common stock is likely to continue to be highly volatile and could fluctuate widely in response to various factors, many of which are beyond our control.
Biotechnology companies have experienced greater than average stock price volatility in recent years, and our common stock price has been particularly volatile ranging from a high o f $30.00 to a low of $0.42 over the past two years (taking into a ccount the reverse stock splits we have completed).
Biotechnology companies have experienced greater than average stock price volatility in recent years, and our common stock price has been particularly volatile ranging from a high o f $356.19 to a low of $3.31 over the past two years (taking into a ccount the reverse stock splits we have completed).
For our lead product candidate, Annamycin, we currently utilize a single source to manufacture API, and if we were to lose this supplier, it could cause delays while we located a new supplier.
For our lead product candidate, Annamycin, we currently utilize a single source to manufacture API and a single source for drug product. If we were to lose either of these suppliers, it could cause delays while we located a new supplier.
The financial markets, energy prices, and the global economy may also be adversely affected by the current or anticipated impact of military conflict, including the conflict between Russia and Ukraine, terrorism or other geopolitical events.
The financial markets, energy prices, and the global economy may also be adversely affected by the current or anticipated impact of military conflict, including the conflicts in Ukraine and the Middle East, terrorism or other geopolitical events.
Proceedings to enforce our intellectual property and other proprietary rights in foreign jurisdictions could result in substantial costs and divert our efforts and attention from other aspects of our business, could put our patents or the patents of our licensors at risk of being invalidated or interpreted narrowly, could put our patent applications or the patent applications of our licensors at risk of not issuing and could provoke third parties to assert claims against us.
Proceedings to enforce our patent rights in other jurisdictions, whether or not successful, could result in substantial costs and divert our efforts and attention from other aspects of our business, could put our patents at risk of being invalidated or interpreted narrowly and our patent applications at risk of not issuing as patents, and could provoke third parties to assert claims against us.
We are conducting important clinical trials in the US and Europe, and assessing additional countries in which to perform preclinical studies and clinical trials, and the risks associated with conducting research and clinical trials abroad could materially adversely affect our business. We have approved Clinical Trial Authorizations in Poland and Italy.
We are conducting important clinical trials in the US and Europe, and additional countries in which to perform preclinical studies and clinical trials, and the risks associated with conducting research and clinical trials abroad could materially adversely affect our business.
Rates of subject enrollment are affected by many factors, including the size of the patient population, the eligibility criteria for the clinical trial, that include the age and condition of the subjects and the stage and severity of disease, the nature of the protocol, the proximity of subjects to clinical sites and the availability of effective treatments and/or availability of investigational treatment options for the relevant disease. 28 Table of Contents A product candidate can unexpectedly fail at any stage of preclinical and clinical development.
Rates of subject enrollment are affected by many factors, including the size of the patient population, the eligibility criteria for the clinical trial, that include the age and condition of the subjects and the stage and severity of disease, the nature of the protocol, the proximity of subjects to clinical sites and the availability of effective treatments and/or availability of investigational treatment options for the relevant disease.
If any of our product candidates receives marketing approval and we or others later identify undesirable or unacceptable side effects caused by such products: • regulatory authorities may require the addition of labeling statements, specific warnings, a contraindication or field alerts to physicians and pharmacies; • we may be required to change instructions regarding the way the product is administered, conduct additional clinical trials or change the labeling of the product; • we may be subject to limitations on how we may promote the product; • sales of the product may decrease significantly; • regulatory authorities may require us to take our approved product off the market; 31 Table of Contents • we may be subject to litigation or product liability claims; and • our reputation may suffer.
If any of our drug candidates cause undesirable or unacceptable side effects in the future, this could interrupt, delay or halt clinical trials and result in the failure to obtain or suspension or termination of marketing approval from the FDA and other regulatory authorities or result in marketing approval from the FDA and other regulatory authorities only with restrictive label warnings or other limitations. 33 Table of Contents If any of our product candidates receives marketing approval and we or others later identify undesirable or unacceptable side effects caused by such products: • regulatory authorities may require the addition of labeling statements, specific warnings, a contraindication or field alerts to physicians and pharmacies; • we may be required to change instructions regarding the way the product is administered, conduct additional clinical trials or change the labeling of the product; • we may be subject to limitations on how we may promote the product; • sales of the product may decrease significantly; • regulatory authorities may require us to take our approved product off the market; • we may be subject to litigation or product liability claims; and • our reputation may suffer.
These and other risks associated with our international operations may materially adversely affect our ability to attain or maintain profitable operations. 26 Table of Contents There are limited suppliers for active pharmaceutical ingredients (API) used in our drug candidates and we utilize a single source for such API for certain of our drug candidates.
These and other risks associated with our international operations may materially adversely affect our ability to attain or maintain profitable operations. 28 Table of Contents There are limited suppliers for active pharmaceutical ingredients (API) used in our drug candidates and/or in manufacturing the final drug product.
Even if the reverse stock split has a positive effect on the market price for shares of our common stock, performance of our business and financial results, general economic conditions and the market perception of our business, and other adverse factors which may not be in our control could lead to a decrease in the price of our common stock following the reverse stock split.
Even if the reverse stock split has a positive effect on the market price for shares of our common stock, performance of our business and financial results, general economic conditions and the market perception of our business, and other adverse factors which may not be in our control could lead to a decrease in the price of our common stock following the reverse stock split. 50 Table of Contents Even if the reverse stock split does result in an increased market price per share of our common stock, the market price per share following the reverse stock split may not increase in proportion to the reduction of the number of shares of our common stock outstanding before the implementation of the reverse stock split.
As such, we are dependent on the institutions conducting the trials to proceed with such trials on a timely basis, and we have encountered unforeseen delays in our physician-sponsored trials.
As such, we are dependent on the institutions conducting the trials to proceed with such trials on a timely basis, and we have in the past encountered unforeseen delays in our physician-sponsored trials. We can provide no assurance that we will not encounter future delays with our physician-sponsored trials.
In addition, others, including regulatory agencies, may not accept or agree with our assumptions, estimates, calculations, conclusions or analyses or may interpret or weigh the importance of data differently, which could impact the approvability of the particular drug candidate and our business in general.
Further, disclosure of preliminary or interim data by us could result in volatility in the price of our common stock. 35 Table of Contents In addition, others, including regulatory agencies, may not accept or agree with our assumptions, estimates, calculations, conclusions or analyses or may interpret or weigh the importance of data differently, which could impact the approvability of the particular drug candidate and our business in general.
We can provide no assurance that we will not encounter future delays with our physician-sponsored trials. If any of our drug product candidates are found to be unsafe or lack sufficient efficacy, we will not be able to obtain regulatory approval for it and our business would be harmed.
If any of our drug product candidates are found to be unsafe or lack sufficient efficacy, we will not be able to obtain regulatory approval for it and our business would be harmed.
If the interim data that we report differ from actual results, or if others, including regulatory authorities, disagree with the conclusions reached, our ability to obtain approval for and commercialize our current or any our future drug candidate, our business, operating results, prospects or financial condition may be materially harmed. 33 Table of Contents We may not be able to conduct, or contract others to conduct, animal testing in the future, which could harm our research and development activities.
If the interim data that we report differ from actual results, or if others, including regulatory authorities, disagree with the conclusions reached, our ability to obtain approval for and commercialize our current or any of our future drug candidates, our business, operating results, prospects or financial condition may be materially harmed.
Competitors may use our technologies in jurisdictions where we have not obtained patent protection to develop their own products and, further, may export otherwise infringing products to territories where we have patent protection, but enforcement is not as strong as that in the United States.
Competitors may use our and our licensors’ technologies in jurisdictions where we have not obtained patent protection to develop their competitor’s own product candidates and, further, may export otherwise infringing product candidates to territories where we and our licensors have patent protection, but enforcement rights are not as strong as that in the U.S.
In addition, a portion of our supply chain is located in countries that may be subject to new or increased tariffs, which could lead to increased costs to us. This exposure to international trade policies poses a risk to our operations and our financial condition.
In addition, a portion of our supply chain is located in countries that may be subject to new or increased tariffs, which could lead to increased costs to us.
We are a party to a number of license agreements with MD Anderson under which we are granted rights to intellectual property that are critical to our business and we expect that we will need to enter into additional license agreements in the future with MD Anderson based on development work we are pursuing under a sponsored research agreement.
However, our failure to meet any financial or other obligations under our license agreements in a timely manner could result in the loss of our rights to our core technologies. 40 Table of Contents We are a party to a number of license agreements with MD Anderson under which we are granted rights to intellectual property that are critical to our business and we expect that we will need to enter into additional license agreements in the future with MD Anderson based on development work we are pursuing under a sponsored research agreement.
Certain laws and regulations relating to drug development require us to test our product candidates on animals before initiating clinical trials involving humans. Animal testing activities have been the subject of controversy and adverse publicity.
We may not be able to conduct, or contract others to conduct, animal testing in the future, which could harm our research and development activities. Certain laws and regulations relating to drug development require us to test our product candidates on animals before initiating clinical trials involving humans. Animal testing activities have been the subject of controversy and adverse publicity.
If other banks and financial institutions enter receivership or become insolvent in the future in response to financial conditions affecting the banking system and financial markets, our ability to access our existing cash, cash equivalents and investments may be threatened and could have a material adverse effect on our business and financial condition. 43 Table of Contents We cannot predict the effect that our reverse stock split will have on the market price for shares of our common stock .
We have no exposure to SVB. If other banks and financial institutions enter receivership or become insolvent in the future in response to financial conditions affecting the banking system and financial markets, our ability to access our existing cash, cash equivalents and investments may be threatened and could have a material adverse effect on our business and financial condition.
If we are unable to maintain compliance with the listing requirements of The Nasdaq Capital Market, our common stock may be delisted from The Nasdaq Capital Market which could have a material adverse effect on our financial condition and could make it more difficult for you to sell your shares.
If any of our stockholders were to bring such a lawsuit against us, we could incur substantial costs defending the lawsuit and the attention of management would be diverted from the operation of our business. 49 Table of Contents If we are unable to maintain compliance with the listing requirements of The Nasdaq Capital Market, our common stock may be delisted from The Nasdaq Capital Market which could have a material adverse effect on our financial condition and could make it more difficult for you to sell your shares.
Failure to commence or complete, or delays in, our planned clinical trials, could prevent us from or delay us in commercializing our product candidates. 29 Table of Contents We may expend significant resources to pursue certain product candidates for specific indications and fail to capitalize on the potential of such product candidates for the potential treatment of other indications that may be more profitable or for which there is a greater likelihood of success.
We may expend significant resources to pursue certain product candidates for specific indications and fail to capitalize on the potential of such product candidates for the potential treatment of other indications that may be more profitable or for which there is a greater likelihood of success.
General Risks Your ownership may be diluted if additional capital stock is issued to raise capital, to finance acquisitions or in connection with strategic transactions. We intend to seek to raise additional funds, finance acquisitions or develop strategic relationships by issuing equity or convertible debt securities, which would reduce the percentage ownership of our existing stockholders.
We intend to seek to raise additional funds, finance acquisitions or develop strategic relationships by issuing equity or convertible debt securities, which would reduce the percentage ownership of our existing stockholders.
Effective internal controls are necessary for us to produce reliable financial reports and are important to help prevent financial fraud. Because we are a smaller reporting company and a non-accelerated filer, we are not required to comply with the auditor attestation requirements of Section 404 of the Sarbanes-Oxley Act.
Because we are a smaller reporting company and a non-accelerated filer, we are not required to comply with the auditor attestation requirements of Section 404 of the Sarbanes-Oxley Act.
Because the legal systems of many foreign countries do not favor the enforcement of patents and other intellectual property protection, it could be difficult for us to stop the infringement, misappropriation or violation of our patents or our licensors’ patents or marketing of competing products in violation of our proprietary rights.
The legal systems of certain countries, particularly certain developing countries, do not favor the enforcement of patents, trade secrets and other intellectual property protection, which could make it difficult for us to stop the infringement of our patents or marketing of competing product candidates in violation of our proprietary rights generally.
Failure of Congress to reauthorize the program will limit our ability to obtain an RPDPRV if WP1066 is approved for the treatment any of the four pediatric cancer indications for which WP1066 received rare pediatric disease designation and may limit our ability to obtain future rare pediatric disease designations for our product candidates. 32 Table of Contents The regulatory approval processes of the FDA and comparable foreign authorities are lengthy, time consuming and inherently unpredictable, and even if we obtain approval for a product candidate in one country or jurisdiction, we may never obtain approval for or commercialize it in any other jurisdiction, which would limit our ability to realize our full market potential.
The regulatory approval processes of the FDA and comparable foreign authorities are lengthy, time consuming and inherently unpredictable, and even if we obtain approval for a product candidate in one country or jurisdiction, we may never obtain approval for or commercialize it in any other jurisdiction, which would limit our ability to realize our full market potential.
Delays in the commencement, enrollment and completion of clinical trials could result in increased costs to us and delay or limit our ability to obtain regulatory approval for any of our product candidates. Delays in the commencement, enrollment and completion of clinical trials could increase our product development costs or limit the regulatory approval of our product candidates.
This exposure to international trade policies poses a risk to our operations and our financial condition. 30 Table of Contents Delays in the commencement, enrollment and completion of clinical trials could result in increased costs to us and delay or limit our ability to obtain regulatory approval for any of our product candidates.
We do not know whether any future trials or studies of our other product candidates will begin on time or will be completed on schedule, if at all.
Delays in the commencement, enrollment and completion of clinical trials could increase our product development costs or limit the regulatory approval of our product candidates. We do not know whether any future trials or studies of our other product candidates will begin on time or will be completed on schedule, if at all.
Even if either Annamycin or WP1066 is approved and ODE is granted, we cannot know that the exclusivity will prevent approval of another product containing Annamycin and intended to treat AML or soft tissue sarcomas, or WP1066 and intended to treat glioblastoma, because any such subsequent product could be demonstrated to be clinically superior to Annamycin or WP1066.
Even if either Annamycin or WP1066 is approved and ODE is granted, we cannot know that the exclusivity will prevent approval of another product containing Annamycin and intended to treat AML or soft tissue sarcomas, or WP1066 and intended to treat glioblastoma, because any such subsequent product could be demonstrated to be clinically superior to Annamycin or WP1066. 34 Table of Contents We received four rare pediatric disease designations for WP1066, but may not be able to obtain a rare pediatric disease priority review voucher if WP1066 is approved or obtain future rare pediatric disease designations for our product candidates, which could adversely affect our potential profitability.
Carrying out pivotal clinical trials is a complicated process. As an organization, we have limited experience in successfully executing earlier-stage clinical trials, and, prior to the MIRACLE trial currently underway, we had not previously conducted any later stage or pivotal clinical trials.
As an organization, we have limited experience in successfully executing earlier-stage clinical trials, and, prior to the MIRACLE trial currently underway, we had not previously conducted any later stage or pivotal clinical trials. In order to do so, we are expanding our clinical development and regulatory capabilities, and we may be unable to retain qualified personnel.
We expect to continue to depend on third parties to supply the API for our current and future product candidates and to supply the API in commercial quantities. We are ultimately responsible for confirming that the APIs used in our product candidates are manufactured in accordance with applicable regulations.
We expect to continue to depend on third parties to supply the API for our current and future product candidates and to supply the API in commercial quantities in a timely manner.
Although off-label prescriptions may infringe our method of use patents, the practice is common across medical specialties and such infringement is difficult to prevent or prosecute.
Although off-label prescriptions may infringe our method of use patents, the practice is common across medical specialties and such infringement is difficult to prevent or prosecute. Off-label sales would limit our ability to generate revenue from the sale of Annamycin, if approved for commercial sale. The patent prosecution process is expensive and time-consuming.
Risks Relating to Our Common Stock • Our stock price has been and may continue to be volatile, which could result in substantial losses for investors. • Shares issuable upon the exercise of outstanding options or warrants may substantially increase the number of shares available for sale in the public market and depress the price of our common stock. • As a biotechnology company, we are at increased risk of securities class action litigation. • If we are unable to maintain compliance with the listing requirements of The Nasdaq Capital Market, our common stock may be delisted from The Nasdaq Capital Market which could have a material adverse effect on our financial condition and could make it more difficult for you to sell your shares. • Failure to maintain our accounting systems and controls could impair our ability to comply with the financial reporting and internal controls requirements for publicly traded companies. • Unstable market and economic conditions may have serious adverse consequences on our business, financial condition and stock price. • We cannot predict the effect that our reverse stock split will have on the market price for shares of our common stock. 25 Table of Contents General Risks • Your ownership may be diluted if additional capital stock is issued to raise capital, to finance acquisitions or in connection with strategic transactions. • Negative research about our business published by analysts or journalists could cause our stock price to decline.
Audits of our compliance with these rules may result in additional liabilities for taxes, duties, interest and penalties related to our international operations which would reduce our profitability. • Errors in our assumptions, estimates and judgments related to tax matters, including those resulting from regulatory reviews, could adversely affect our financial results. • We will require significant additional funding to complete the MIRACLE trial, which may not be available to us on acceptable terms, or at all, and, if not so available, may require us to delay, limit, reduce or cease our operations. 27 Table of Contents Risks Relating to Our Common Stock • Our stock price has been and may continue to be volatile, which could result in substantial losses for investors. • Shares issuable upon the exercise of outstanding options or warrants may substantially increase the number of shares available for sale in the public market and depress the price of our common stock. • As a biotechnology company, we are at increased risk of securities class action litigation. • If we are unable to maintain compliance with the listing requirements of The Nasdaq Capital Market, our common stock may be delisted from The Nasdaq Capital Market which could have a material adverse effect on our financial condition and could make it more difficult for you to sell your shares. • Failure to maintain our accounting systems and controls could impair our ability to comply with the financial reporting and internal controls requirements for publicly traded companies. • Unstable market and economic conditions may have serious adverse consequences on our business, financial condition and stock price. • We cannot predict the effect that our reverse stock split will have on the market price for shares of our common stock. • There are provisions in certain of our outstanding warrants that could discourage an acquisition of us by a third party. • If our estimates or judgments relating to our critical accounting policies prove to be incorrect, or if financial reporting standards or interpretations change, our results of operations could be adversely affected.
As a public company, we operate in an increasingly demanding regulatory environment, which requires us to comply with the Sarbanes-Oxley Act of 2002, and the related rules and regulations of the SEC. Company responsibilities required by the Sarbanes-Oxley Act include establishing corporate oversight and adequate internal control over financial reporting and disclosure controls and procedures.
Failure to maintain our accounting systems and controls could impair our ability to comply with the financial reporting and internal controls requirements for publicly traded companies. As a public company, we operate in an increasingly demanding regulatory environment, which requires us to comply with the Sarbanes-Oxley Act of 2002, and the related rules and regulations of the SEC.
However, regardless of such option to negotiate, we may be unable to negotiate a license within the specified time frame or under terms that are acceptable to us.
However, regardless of such option to negotiate, we may be unable to negotiate a license within the specified time frame or under terms that are acceptable to us. If we are unable to do so, the institution may offer the intellectual property rights to other parties, potentially blocking our ability to pursue a program based on that technology.
In addition, many of the factors that cause, or lead to, a delay in the commencement or completion of clinical trials may also ultimately lead to the denial of regulatory approval of our product candidates.
In addition, many of the factors that cause, or lead to, a delay in the commencement or completion of clinical trials may also ultimately lead to the denial of regulatory approval of our product candidates. 31 Table of Contents As an organization, we have never before conducted pivotal clinical trials, and we may be unable to do so for any product candidates we may develop.
If our third-party drug suppliers fail to achieve and maintain high manufacturing standards in compliance with cGMP regulations, we could be subject to certain product liability claims in the event such failure to comply resulted in defective products that caused injury or harm. 27 Table of Contents We cannot guarantee how long it will take regulatory agencies to review our applications for product candidates, and we may fail to obtain the necessary regulatory approvals to market our product candidates.
If our third-party drug suppliers fail to achieve and maintain high manufacturing standards in compliance with cGMP regulations, we could be subject to certain product liability claims in the event such failure to comply resulted in defective products that caused injury or harm. 29 Table of Contents We are required to order the API used in our drug candidates from our manufacturers significantly in advance of our delivery needs, and problems with our forecasts related to the manufacturer of the API used in our drug candidates may delay our clinical trials.
Additionally, from time to time, we perform studies to determine if there are additional countries in which we should hold current and future clinical and preclinical studies.
We have approved INDs, Clinical Trial Authorizations or their equivalent in the US, Ukraine, Georgia, Spain, Romania, Czechia, Lithuania, Poland and Italy. Additionally, from time to time, we perform studies to determine if there are additional countries in which we should hold current and future clinical and preclinical studies.
In addition, there is a risk that the court will decide that such patents are not valid and that we do not have the right to stop the other party from using the inventions.
There is a risk that in connection with such proceedings, a court will decide that a patent of ours is invalid or unenforceable, in whole or in part, and that we do not have the right to stop the other party from using the invention at issue.
The historical failure rate for product candidates is high due to scientific feasibility, safety, efficacy, changing standards of medical care and other variables. The results from preclinical testing or early clinical trials of a product candidate may not predict the results that will be obtained in later phase clinical trials of the product candidate.
A product candidate can unexpectedly fail at any stage of preclinical and clinical development. The historical failure rate for product candidates is high due to scientific feasibility, safety, efficacy, changing standards of medical care and other variables.
As an organization, we have never before conducted pivotal clinical trials, and we may be unable to do so for any product candidates we may develop. We will need to successfully complete pivotal clinical trials in order to obtain the approval of the FDA, EMA or other regulatory agencies to market our product candidates.
We will need to successfully complete pivotal clinical trials in order to obtain the approval of the FDA, EMA or other regulatory agencies to market our product candidates. Carrying out pivotal clinical trials is a complicated process.
Consequently, we may be unable to successfully and efficiently execute and complete necessary clinical trials in a way that leads to NDA submission and approval of our product candidates. We may require more time and incur greater costs than our competitors and may not succeed in obtaining regulatory approvals of product candidates that we develop.
We also expect to continue to rely on third parties to conduct our pivotal clinical trials. Consequently, we may be unable to successfully and efficiently execute and complete necessary clinical trials in a way that leads to NDA submission and approval of our product candidates.
Delisting also could have other negative results, including the potential loss of employee confidence, the loss of institutional investors or interest in business development opportunities. Failure to maintain our accounting systems and controls could impair our ability to comply with the financial reporting and internal controls requirements for publicly traded companies.
Delisting also could have other negative results, including the potential loss of employee confidence, the loss of institutional investors or interest in business development opportunities.
Problems with the third parties that manufacture the API used in our drug candidates may delay our clinical trials or subject us to liability. We do not currently own or operate manufacturing facilities for clinical or commercial production of the API used in any of our product candidates.
We utilize a single source for such API and/or final drug product for certain of our drug candidates. We do not currently own or operate manufacturing facilities for clinical or commercial production of the API used in any of our product candidates.
There is also the risk that, even if the validity of such patents is upheld, the court will refuse to stop the other party on the ground that such other party's activities do not infringe our intellectual property rights.
There is also a risk that, even if the validity of such patents is upheld, the court will construe the patent’s claims narrowly or decide that we do not have the right to stop the other party from using the invention at issue on the grounds that our patent claims do not cover the invention.
Any reduction in reimbursement from Medicare or other government programs may result in a similar reduction in payments from third-party payors. The implementation of cost containment measures or other healthcare reforms may prevent us from being able to generate revenue, attain profitability or commercialize Annamycin, if approved.
Any reduction in reimbursement from Medicare or other government programs may result in a similar reduction in payments from third-party payors.
As of December 31, 2024, we had warrants and options outstanding to purchase an aggregate of 8,594,561 shares of common stock at an average exercise price of $4.33 per share (taking into account the reverse stock splits we have completed).
As of December 31, 2025 and February 28, 2026, we had outstanding warrants and options to purchase an aggregate of 7,868,686 and 12,719,348 shares of common stock, respectively, at weighted average exercise prices of $6.36 and $4.11 per share, respectively, in each case after giving effect to the reverse stock splits we completed.
To date, we have been able meet such milestones, pay certain fees or have been able to enter into extensions with MD Anderson related to such milestones. However, our failure to meet any financial or other obligations under our license agreements in a timely manner could result in the loss of our rights to our core technologies.
To date, we have been able to meet such milestones, pay certain fees or have been able to enter into extensions with MD Anderson related to such milestones, or have obtained options to acquire the technology.
We may be subject to claims that these employees, or we, have used or disclosed trade secrets or other proprietary information of their former employers. Litigation may be necessary to defend against these claims. Even if we are successful in defending against these claims, litigation could result in substantial costs and be a distraction to management.
If we fail in defending any such claims, in addition to paying monetary damages, we may lose valuable intellectual property rights, such as exclusive ownership of, or right to use, valuable intellectual property. Even if we are successful in defending against such claims, litigation could result in substantial costs and be a distraction to management and other employees.
Filing, prosecuting and defending patents in all countries throughout the world would be prohibitively expensive, and our intellectual property rights in some countries outside the United States may be less extensive than those in the United States.
We enjoy only limited geographical protection with respect to certain patents and may not be able to protect our intellectual property rights throughout the world. Filing and prosecuting patent applications and maintaining and defending patents covering our product candidates in all countries throughout the world would be prohibitively expensive.
We rely in part on confidentiality agreements with our employees, consultants, outside scientific collaborators, sponsored researchers and other advisors to protect our trade secrets and other proprietary information. These agreements may not effectively prevent disclosure of confidential information and may not provide an adequate remedy in the event of unauthorized disclosure of confidential information.
In addition, current or former employees, consultants, contractors, and advisers may unintentionally or willfully disclose our confidential information to competitors, and confidentiality agreements may not provide an adequate remedy in the event of unauthorized disclosure of confidential information.
We received four rare pediatric disease designations for WP1066, but may not be able to obtain a rare pediatric disease priority review voucher if WP1066 is approved or obtain future rare pediatric disease designations for our product candidates, which could adversely affect our potential profitability.
Failure of Congress to reauthorize the program will limit our ability to obtain an RPDPRV if WP1066 is approved for the treatment any of the four pediatric cancer indications for which WP1066 received rare pediatric disease designation and may limit our ability to obtain future rare pediatric disease designations for our product candidates.
Risks Related to our Intellectual Property The composition of matter patent for Annamycin has expired, and other patents have not yet been issued, and may not be issued.
We may be subject to claims challenging the inventorship of our patents and other intellectual property. We may be subject to claims challenging the inventorship of our patents and patent applications or ownership of our intellectual property.
Removed
Audits of our compliance with these rules may result in additional liabilities for taxes, duties, interest and penalties related to our international operations which would reduce our profitability. • Errors in our assumptions, estimates and judgments related to tax matters, including those resulting from regulatory reviews, could adversely affect our financial results. • We will require significant additional funding to complete the MIRACLE trial, which may not be available to us on acceptable terms, or at all, and, if not so available, may require us to delay, limit, reduce or cease our operations.
Added
These claims may be costly to defend and if we do not successfully do so, we may be required to pay monetary damages and lose valuable intellectual property rights or personnel. • Our business and operations may suffer, and proprietary information may be lost, in the event of information technology system failures, cyberattacks or deficiencies in our cybersecurity.

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Item 1C. Cybersecurity

Cybersecurity — threats and controls disclosure

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Biggest changeFor additional information regarding risks from cybersecurity threats, please refer to Item 1A, “Risk Factors,” in this Annual report on Form 10-K. 45 Table of Contents Governance One of the key responsibilities of our board of directors is informed oversight of our risk management process, including risks from cybersecurity threats.
Biggest changeFor additional information regarding risks from cybersecurity threats, please refer to Item 1A, “Risk Factors,” in this Annual report on Form 10-K. Governance One of the key responsibilities of our board of directors is informed oversight of our risk management process, including risks from cybersecurity threats.
As of December 31, 2024, and through the date of the filing of this report, we are not aware of any cybersecurity incidents that have materially affected or are reasonably likely to materially affect us, including our business strategy, results of operations or financial condition.
As of December 31, 2025, and through the date of the filing of this report, we are not aware of any cybersecurity incidents that have materially affected or are reasonably likely to materially affect us, including our business strategy, results of operations or financial condition.
Our Head of IT and Cybersecurity has served as an information technology professional for over nine years and has held senior IT positions at multiple companies, where his primary responsibilities included maintaining direct oversight over his companies’ cybersecurity risks.
Our Head of IT and Cybersecurity has served as an information technology professional for over ten years and has held senior IT positions at multiple companies, where his primary responsibilities included maintaining direct oversight over his companies’ cybersecurity risks.
In this regard, our Chief Executive Officer and Head of IT and Cybersecurity have assistance from service providers, other consultants and third parties. Our Chief Financial Officer has served as an executive officer for over twenty-five years, including over fifteen years as an executive officer of public companies.
In this regard, our Chief Financial Officer and Head of IT and Cybersecurity have assistance from service providers, other consultants and third parties. Our Chief Financial Officer has served as an executive officer for over twenty-six years, including over sixteen years as an executive officer of public companies.

Item 3. Legal Proceedings

Legal Proceedings — active lawsuits and investigations

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Biggest changeWe have insurance policies covering potential losses where such coverage is cost effective. We are not at this time involved in any legal proceedings that we believe could have a material effect on our business, financial condition, results of operations or cash flows. ITEM 4. MINE SAFETY DISCLOSURE Not applicable. PART II
Biggest changeWe have insurance policies covering potential losses where such coverage is cost effective. We are not at this time involved in any legal proceedings that we believe could have a material effect on our business, financial condition, results of operations or cash flows.

Item 5. Market for Registrant's Common Equity

Market for Common Equity — stock, dividends, buybacks

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Biggest changeITEM 5. MARKET FOR REGISTRANT’S COMMON EQUITY, RELATED STOCKHOLDER MATTERS AND ISSUER PURCHASES OF EQUITY SECURITIES Market Information Our common stock is listed on the NASDAQ Capital Market under the symbol “MBRX”. Holders As o f March 13, 2025, there were approximately 139 holders of record of our common stock.
Biggest changeITEM 5. MARKET FOR REGISTRANT’S COMMON EQUITY, RELATED STOCKHOLDER MATTERS AND ISSUER PURCHASES OF EQUITY SECURITIES Market Information Our common stock is listed on the NASDAQ Capital Market under the symbol “MBRX”. Holders As of March 11, 2026, there were approximately 316 holders of r ecord of our common stock.
Purchases of Equity Securities by the Issuer and Affiliated Purchasers We did not repurchase any of our equity securities during the year ended December 31, 2024. ITEM 6. [RESERVED]
Purchases of Equity Securities by the Issuer and Affiliated Purchasers We did not repurchase any of our equity securities during the year ended December 31, 2025. ITEM 6. [RESERVED]
I n addition, we believe that a significant number of beneficial owners of our common stock hold their shares in nominee or in “street name” accounts through brokers. 46 Table of Contents Dividends We have never paid any dividends on our common stock.
I n addition, we believe that a significant number of beneficial owners of our common stock hold their shares in nominee or in “street name” accounts through brokers. Dividends We have never paid any dividends on our common stock.
Recent Sales of Unregistered Securities Except as set forth below, all information related to equity securities sold by us during the period covered by this report that were not registered under the Securities Act have been included in our Form 10-Q filings or in a Form 8-K filing.
Recent Sales of Unregistered Securities All information related to equity securities sold by us during the period covered by this report that were not registered under the Securities Act have been included in our Form 10-Q filings or in a Form 8-K filing.
Removed
During the quarter ended December 31, 2024, we issued ten-year warrants to purchase an aggregate of 36,000 shares of our common stock at an exercise price of $2.36 per share to four entities that provided services to the Company.
Removed
The warrants and the shares issuable upon exercise of the warrants are being sold and issued without registration under the Securities Act in reliance on the exemptions provided by Section 4(a)(2) of the Securities Act as transactions not involving a public offering, and in reliance on similar exemptions under applicable state laws.

Item 6. [Reserved]

Selected Financial Data — reserved (removed by SEC in 2021)

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Biggest changeItem 6. [Reserved] 47 Item 7. Management's Discussion and Analysis of Financial Condition and Results of Operations 47 Item 7A. Quantitative and Qualitative Disclosures About Market Risk 53 Item 8. Financial Statements and Supplementary Data 53 Item 9. Change in and Disagreements with Accountants on Accounting and Financial Disclosures 53 Item 9A. Controls and Procedures 53 Item 9B.
Biggest changeItem 6. [Reserved] 54 Item 7. Management's Discussion and Analysis of Financial Condition and Results of Operations 54 Item 7A. Quantitative and Qualitative Disclosures About Market Risk 61 Item 8. Financial Statements and Supplementary Data 61 Item 9. Change in and Disagreements with Accountants on Accounting and Financial Disclosures 61 Item 9A. Controls and Procedures 61

Item 7. Management's Discussion & Analysis

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

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Biggest changeWe believe that our lead drug candidate Annamycin has summarily: ● Demonstrated significant efficacy in Phase 1 and 2 cancer clinical trials – specifically relapsed and refractory (R/R) AML with complete remission rates significantly above currently approved second line therapies and STS lung mets with overall survival as a median seventh line therapy comparable to overall survival seen with approved first line monotherapies; ● Shown encouraging activity in five different clinical trials (most of which are now complete), funded both internally and externally (through investigator initiated trials); ● Shown in its clinical trials to be non-cardiotoxic (N=84) with some patients being safely dosed at five times the typical lifetime maximum allowed anthracycline dose, which potentially enables repeated dosing and consolidation; ● Demonstrated in preclinical models, to be more potent than and to avoid cross resistance with some of the most common drugs used to treat AML (as well as many other cancers), including currently approved anthracyclines, Cytarabine and Venetoclax; ● Composition of matter patent protection thru 2040 and has orphan drug and fast track status; ● No vesicant activity, making it safer to handle and administer as compared to other anthracyclines; and, ● Shown a complete response (CR) rate of 50%, a composite complete response (CRc) rate of 60%, durability of CRc’s of approximately 9 months (and climbing), and an overall survival (OS) rate of approximately 11 months (and climbing) (N=10) in combination with Cytarabine for the treatment of R/R AML as second line therapy to date, which is higher than other currently approved second line therapies for R/R AML One of our core management beliefs is that anthracyclines represent one of the most important treatments for AML and Advanced STS, and we believe Annamycin may, for the first time ever, allow a majority of these patients to benefit from this treatment.
Biggest changeWe have also sponsored two Phase 1B/2 clinical trials of Annamycin for treating Soft Tissue Sarcoma metastasized to the lungs (STS lung metastases, STS lung mets, or Advanced STS), the results of which we believe supports the initiation of a pivotal approval trial in this additional indication. 54 Table of Contents We believe that our lead drug candidate Annamycin has: ● The following advantages over early-generation anthracyclines which in their totality support Annamycin’s classification as a “next-generation” anthracycline: 1) demonstrated across five clinical trials to be non-cardiotoxic (n=90) with some patients being safely dosed at five times the typical lifetime maximum allowable anthracycline dose, (which potentially enables repeated dosing, consolidation and even use as a maintenance therapy); 2) exhibits no vesicant activity as well as greater tolerability, making it safer to handle and administer and easier on patients; 3) shown in preclinical models to circumvent multidrug resistance by avoiding MDR1 mechanisms and also to avoid cross-resistance with some of the most commonly used drugs for the treatment of AML (as well as many other cancers), including currently approved early-generation anthracyclines, cytarabine and venetoclax and, 4) exhibited in preclinical models substantially improved tissue/organ distribution; ● Demonstrated a complete remission (CR) rate of 50% and a composite complete remission (CRc) rate of 60% in combination with Cytarabine for the treatment of R/R AML as second line therapy (n=10).
We estimate the fair value of options granted using the Black-Scholes option valuation model, and the fair value of restricted stock units using the closing price of our common stock as reported on the date of grant. The Black-Scholes estimate uses assumptions regarding a number of inputs that require us to make significant estimates and judgments.
We estimate the fair value of options granted using the Black-Scholes option valuation model (BSM), and the fair value of restricted stock units using the closing price of our common stock as reported on the date of grant. The Black-Scholes estimate uses assumptions regarding a number of inputs that require us to make significant estimates and judgments.
Beginning in 2020, only the volatility of our stock was used in the BSM as we now have sufficient historic data in our stock price. 52 Table of Contents Income taxes We account for income taxes using ASC 740, Income Taxes.
Beginning in 2020, only the volatility of our stock was used in the BSM as we now have sufficient historic data in our stock price. 60 Table of Contents Income taxes We account for income taxes using ASC 740, Income Taxes.
This takes into account cash outlays for preparations for clinical trials beyond the current active trials. The continuation of our Company as a going concern is depe ndent upon our ability to obtain necessary financing to continue operations and the attainment of profitable operations.
This takes into account cash outlays for preparations for clinical trials beyond the current active trials. The continuation of our Company as a going concern is dependent upon our ability to obtain necessary financing to continue operations and the attainment of profitable operations.
The forward-looking statements included in this discussion and elsewhere in this Form 10-K involve risks and uncertainties, including those set forth under “Cautionary Statement About Forward-Looking Statements.” Actual results and experience could differ materially from the anticipated results and other expectations expressed in our forward-looking statements as a result of a number of factors, including but not limited to those discussed in this Item and in Item 1A - “Risk Factors.” Our Business Summary We are a late-stage pharmaceutical development company currently conducting a pivotal Phase 3 trial evaluating Annamycin, a non-cardiotoxic anthracycline, in combination with Cytarabine for the treatment of subjects with relapsed/refractory acute myeloid leukemia (AML).
The forward-looking statements included in this discussion and elsewhere in this Form 10-K involve risks and uncertainties, including those set forth under “Cautionary Statement About Forward-Looking Statements.” Actual results and experience could differ materially from the anticipated results and other expectations expressed in our forward-looking statements as a result of a number of factors, including but not limited to those discussed in this Item and in Item 1A - “Risk Factors.” Our Business Summary We are a late-stage pharmaceutical development company currently evaluating Annamycin, also known as L-Annamycin and by its generic name “naxtarubicin”, which we believe is a “next-generation” anthracycline and, with it, are conducting a pivotal Phase 3 trial in combination with cytarabine for the treatment of subjects with relapsed/refractory (R/R) acute myeloid leukemia (AML).
ASC 740 also requires the recognition of liabilities created by differences between tax positions taken in a tax return and amounts recognized in the financial statements. Recent accounting pronouncements See Note 2 to the Notes to Consolidated Financial Statements in "Item 8 - Financial Statements and Supplementary Data" in this Annual Report for discussion regarding recent accounting pronouncements.
ASC 740 also requires the recognition of liabilities created by differences between tax positions taken in a tax return and amounts recognized in the financial statements. Recent accounting pronouncements See Note 2 of our consolidated financial statements in Item 8 in this Annual Report on Form 10-K for discussion regarding recent accounting pronouncements.
Each of our three core technologies is based substantially on discoveries made at and licensed from the University of Texas MD Anderson Cancer Center (MD Anderson) in Houston, Texas, and features one or more drugs that have successfully completed a Phase 1 clinical trial.
Each of our three core technologies is based substantially on discoveries made at, made in conjunction with, and/or licensed from the University of Texas MD Anderson Cancer Center (MD Anderson) in Houston, Texas. For each of the core technologies, one or more drug candidates have successfully completed a Phase 1 or greater clinical trial.
For the years ended December 31, 2024 and 2023, we used approxima tely $23.9 million and $23.6 million of cash in operating activities, respectively, which represents cash outlays for research and development and general and administrative expenses in such periods. The slightly increased cash outflows in 2024 was primarily due to timing of payments for sponsored research and other expenses.
For the years ended December 31, 2025 and 2024, we used approximately $22.7 million and $23.9 million of cash in operating activities, respectively, which represents cash outlays for research and development and general and administrative expenses in such periods. The slightly decreased cash outflows in 2025 was primarily due to timing of payments for sponsored research and other expenses.
Refer to Note 2 for additional discussion. 50 Table of Contents Critical Accounting Policies and Significant Judgments and Estimates The accompanying consolidated financial statements and related notes have been prepared in accordance with accounting principles generally accepted in the United States of America (US GAAP) for financial information, and in accordance with the rules and regulations of the SEC.
Critical Accounting Policies and Significant Judgments and Estimates The accompanying consolidated financial statements and related notes have been prepared in accordance with accounting principles generally accepted in the United States of America (US GAAP) for financial information, and in accordance with the rules and regulations of the SEC.
We expect our general and administrative expense to increase due to the anticipated growth of our business and related infrastructure as well as accounting, insurance, investor relations and other costs associated with being a public company. 51 Table of Contents Depreciation and Amortization. Depreciation and amortization expense consists of depreciation on our property and equipment.
We expect our general and administrative expense to increase due to the anticipated growth of our business and related infrastructure as well as accounting, insurance, investor relations and other costs associated with being a public company. Depreciation and Amortization. Depreciation and amortization expense consists of depreciation on our property and equipment. We depreciate our assets over their estimated useful life.
The Company received gross proceeds of $3.5 million. 49 Table of Contents In February 2025, the Company entered into a warrant exercise inducement offer letter with a holder of certain existing warrants to receive new warrants to purchase up to a number of shares of common stock equal to 200% of the number of warrant shares issued pursuant to the exercise of such existing warrants to purchase up to 5,828,570 shares of common stock pursuant to which the warrant holder agreed to exercise for cash their existing warrants at a reduced exercise price of $1.00 in exchange for the Company's agreement to issue the inducement warrants to purchase up to 11,657,140 shares of the Company's common stock.
We received gross proceeds of $3.5 million. 57 Table of Contents On February 13, 2025, we entered into a warrant exercise inducement offer letter with a holder of certain existing warrants to receive new warrants to purchase up to a number of shares of common stock equal to 200% of the number of warrant shares issued pursuant to the exercise of such existing warrants to purchase up to 233,143 shares of common stock pursuant to which the warrant holder agreed to exercise for cash their existing warrants at a reduced exercise price of $25.00 in exchange for our agreement to issue the inducement warrants to purchase up to 466,286 shares of our common stock.
In combination with Cytarabine (also known as Ara-C, the combination with Annamycin of which is referred to as AnnAraC) for the treatment of R/R AML, and b. For the treatment of STS metastasized to the lungs. 2.
Internally funded – In combination with Cytarabine (also known as Ara-C, the combination with Annamycin of which is referred to as AnnAraC) for the treatment of R/R AML, and b. Externally funded – Advancing beyond our initial Phase 1B/2 internally funded trial for the treatment of STS metastasized to the lungs. c.
We believe that our cash resources as of December 31, 2024, along with $9.3 m illion in gross proceeds received via our financing activities in the first quarter of 2025 (see Recent Stock Offerings below) will be sufficient to fund our planned operations into the third quarter of 2025, without the issuance of additional equity for cash.
Cash used in investing activities for the years ended December 31, 2025 and 2024 was approximately $0 and $13,000, respectively. 56 Table of Contents We believe that our cash resources as of December 31, 2025, along with $8.3 million in gross proceeds received via our financing activities in the first quarter of 2026 (see Recent Stock Offerings below) will be sufficient to fund our planned operations into the third quarter of 2026, without the issuance of additional equity for cash.
The combined purchase price for the securities was $2.23 per share of common stock (or pre-funded warrant in lieu thereof) and accompanying warrants. Each pre-funded warrant is exercisable for one share of common stock at an exercise price of $0.001 per share.
The combined purchase price for the securities was $26.75 per share of common stock (or pre-funded warrant in lieu thereof). Each pre-funded warrant was exercisable for one share of common stock at an exercise price of $0.025 per share. All pre-funded warrants were exercised in June 2025.
We also had $2.0 million of accounts payable and $3.3 million of accrued expenses and other current liabilities. A significant portion of the accounts payable and accrued expenses are due to work performed in relation to our preclinical activities and our clinical trials.
A significant portion of the accounts payable and accrued expenses were due to work performed in relation to our preclinical activities and our clinical trials.
Cash used in investing activities Net cash used in investing activities w as de minimis f or the years ended December 31, 2024 and December 31, 2023, respectively. Cash provided by financing activities Net cash provided by financing activities was $4.6 million f or the year ended December 31, 2024, consisting of the proceeds from the August 2024 stock offering.
Net cash provided by financing activities was $4.6 million for the year ended December 31, 2024, consisting of proceeds from the August 2024 stock offering.
Results of Operations for the Year Ended December 31, 2024 as Compared to the Year Ended December 31, 2023 The following table is data derived from the Consolidated Statement of Operations (in thousands) and the discussions that follow are in approximate amounts: Year ended December 31, 2024 2023 Revenue $ — $ — Operating expenses: Research and development 17,729 19,487 General and administrative 8,786 10,017 Depreciation and amortization 126 127 Total operating expense 26,641 29,631 Loss from operations (26,641 ) (29,631 ) Other income: Gain (loss) from change in fair value of warrant liability 6,125 (1,044 ) Transaction costs allocated to warrant liabilities (993 ) (510 ) Loss on issuance of warrant liabilities (847 ) — Other income, net 43 48 Interest income, net 550 1,368 Net loss $ (21,763 ) $ (29,769 ) 48 Table of Contents Research and Development Expense Research and development (R&D) expense w as $17.7 million an d $19.5 million for the years ended December 31, 2024 and 2023, respectively.
Results of Operations for the Year Ended December 31, 2025 as Compared to the Year Ended December 31, 2024 The following table is data derived from the Consolidated Statement of Operations (in thousands) and the discussions that follow are in approximate amounts: Year ended December 31, 2025 2024 Revenue $ — $ — Operating expenses: Research and development 15,891 17,729 General and administrative 9,122 8,786 Depreciation and amortization 90 126 Total operating expense 25,103 26,641 Loss from operations (25,103 ) (26,641 ) Other income: Gain from change in fair value of warrant liabilities 24,441 — Loss on issuance of warrant liabilities (30,962 ) — Transaction costs allocated to warrant liabilities (1,407 ) — Loss on extinguishment of warrant liabilities (614 ) — Other (expense) income, net (20 ) 43 Interest income, net 105 550 Net loss $ (33,560 ) $ (26,048 ) Research and Development Expense Research and development (R&D) expense was $15.9 million and $17.7 million for the years ended December 31, 2025 and 2024, respectively.
We believe that the following accounting policies are the most critical to aid in fully understanding and evaluating our reported financial results, and they require our most difficult, subjective or complex judgments, resulting from the need to make estimates about the effect of matters that are inherently uncertain .
We believe that the following accounting policies are the most critical to aid in fully understanding and evaluating our reported financial results, and they require our most difficult, subjective or complex judgments, resulting from the need to make estimates about the effect of matters that are inherently uncertain . 58 Table of Contents Research and Development Costs We record accrued expenses for estimated costs of our research and development activities conducted by third-party service providers, which include the conduct of pre-clinical and clinical trials and preparation for clinical trials and contract manufacturing activities.
Cash used in operating activities Net cash used in operating activities was $23.9 million for the year ended December 31, 2024 compared to $23.6 million for the year ended December 31, 2023. This slight increase in use of cash for operations was mainly due to timing of payments related to sponsored research and other expenses.
This slight decrease in use of cash for operations was mainly due to timing of payments related to sponsored research and other expenses. Cash used in investing activities Net cash used in investing activities w as de minimis f or the years ended December 31, 2025 and December 31, 2024, respectively.
We believe that such a benefit would be disruptive to the competitive landscape for these markets. This belief, coupled with our limited resources, leads us to currently focus mainly on the development of Annamycin.
We believe that such benefits will be disruptive to the competitive landscape for these markets. This belief, coupled with our limited resources, leads us to currently focus mainly on the development of Annamycin. We seek to advance our other drug candidates via investigator led studies, both clinically and preclinically, and as available resources will allow.
The following table sets forth the primary sources and uses of cash for the years indicated (in thousands): For the Year Ended December 31, 2024 2023 Net cash used in operating activities $ (23,862 ) $ (23,591 ) Net cash used in investing activities (13 ) (124 ) Net cash provided by financing activities 4,635 4,141 Effect of exchange rate changes on cash and cash equivalents (32 ) (21 ) Net change in cash and cash equivalents $ (19,272 ) $ (19,595 ) As of December 31, 2024, there was $0.6 million of cas h on hand in a bank account in Australia and we know of no related limitations impacting our liquidity in Australia.
The following table sets forth the primary sources and uses of cash for the years indicated (in thousands): For the Year Ended December 31, 2025 2024 Net cash used in operating activities $ (22,735 ) $ (23,862 ) Net cash used in investing activities — (13 ) Net cash provided by financing activities 27,284 4,635 Effect of exchange rate changes on cash and cash equivalents 51 (32 ) Net change in cash and cash equivalents $ 4,600 $ (19,272 ) Cash used in operating activities Net cash used in operating activities was $22.7 million for the year ended December 31, 2025 compared to $23.9 million for the year ended December 31, 2024.
Our core technologies consist of the following programs: a) Annamycin or L-Annamycin is a “next generation” anthracycline (one of the most widely used classes of chemotherapy), designed to be different than currently approved anthracyclines, which are limited in utility because of cardiotoxicity risks and their susceptibility to multidrug resistance mechanisms.
Core Technologies and Focus Our core technologies consist of the following programs: a) Annamycin is what we believe to be a “next-generation” anthracycline (one of the most widely used classes of chemotherapy), designed to be different than currently approved anthracyclines, which are limited in utility because of cardiotoxicity risks, tolerability, their susceptibility to multidrug resistance mechanisms and a lack of efficacy in certain tumor models. b) Our WP1066 Portfolio includes WP1066, WP1193 and WP1220, three of several Immune/Transcription Modulators in the portfolio designed to inhibit p-STAT3 (phosphorylated signal transducer and activator of transcription) among other transcription factors associated with tumor activity.
Recent Stock Offerings In February 2025, the Company entered into a securities purchase agreement with an institutional investor for the sale by the Company of 1,150,000 shares of common stock, and 2,121,029 pre-funded warrants to purchase shares of common stock, and common warrants to purchase up to 6,543,058 shares of common stock.
On February 25, 2025, we entered into a securities purchase agreement with an institutional investor for the sale of 46,000 shares of common stock, and 84,841 pre-funded warrants to purchase shares of common stock, and Series D warrants to purchase up to 261,722 shares of common stock.
Each inducement warrant has an exercise price of $0.75, and was immediately exercisable as of the date of issuance and may be exercised for a period of five years therefrom. The Company received gross proceeds of $5.8 million. This brings the total gross proceeds received in February 2025 to $9.3 million.
Each inducement warrant is exercisable as of the date of issuance and may be exercised for a period of five years from the date of issuance. We received gross proceeds of $5.8 million.
We intend to advance our other drug candidates via investigator led studies – both clinically and preclinically. 47 Table of Contents Focus and Core Technologies We are focused on internally funded (“internally” and “externally” funded trials are defined in the Funding Strategy section below) development of our core technologies: 1. Annamycin: a.
Our Current Clinical Trial Focus We are focused on Annamycin and WP1066 program’s internally and externally funded (“internally” and “externally” funded trials are defined in the Funding Strategy section below) development of our core technologies as follows: 1. Annamycin: a.
F or the year ended December 31, 2024, there were $4.6 million in net proceeds from financing activities. In 2023, there were $4.1 million in net proceeds from financing activities. Cash used in investing activities for the years ended December 31, 2024 and 2023 was approximatel y $0.0 million, and $0.1 million, respectively.
For the year ended December 31, 2025, there were $27.3 million in net proceeds from financing activities. In 2024, there were $4.6 million in net proceeds from financing activities.
Three of our six drug candidates have shown human activity in clinical trials and are currently or have been in Phase 1B/2 or Phase 2 clinical trials. One is currently beginning a Phase 2B/3 trial. Since our inception, our drugs have completed, are currently in, or have been permitted to proceed in, fourteen clinical trials.
Three of our drug candidates have shown human activity in clinical trials and are currently or have been in Phase 1B/2, Phase 2, Phase 2B/3 clinical trials. One of those drug candidates is Annamycin, which is currently in the MIRACLE trial. We believe Annamycin is the first of its kind DNA binding agent, a “next-generation” anthracycline.
In October 2024, the Company’s shareholders approved the issuance of both the August 2024 warrants, as well as the warrant amendment. The Company received gross proceeds of $5.5 million, before deducting the placement agent's fees and other offering expenses payable by the Company.
We received gross proceeds of $5.5 million, before deducting the placement agent's fees and other offering expenses.
Additionally, we have two portfolios of technologies for hard-to-treat cancers and viruses with clinical and preclinical research funded by investigators at academic institutions.
We believe such early visibility for a pivotal registration-enabling trial is unique in that stakeholders will receive preliminary safety and efficacy data long before the conclusion of the trial. We have two additional portfolios of technologies for hard-to-treat cancers and viruses with clinical and preclinical research funded by investigators at academic institutions.
Interest income, net Interest income, net decreased by approximately $0.8 million for the comparable period due to a decreasing cash balance, coupled with decreasing interest rates during the past year. Liquidity and Capital Resources As of December 31, 2024 , we had cash and cash equivalents of $4.3 million and prepaid expenses and other current assets of $0.9 million.
Interest Income, Net Interest income, net decreased by approximately $0.4 million for the year ended December 31, 2025 from the comparable period in 2024 due to a decreasing cash balance during the past year. Net Loss Net loss for the year ended December 31, 2025 was $33.6 million, compared to a net loss of $26.0 million for the prior year.
Proceeds of offerings are allocated between common shares and warrants first by allocating to the warrants classified as a liability based on their fair value and then allocating the residual to the equity instruments, which would include pre-funded warrants.
Allocation of Warrant Proceeds For equity offerings during 2025 that included both common stock, equity-classified warrants, and warrants classified as liabilities, we allocated the proceeds first to the warrants based on their fair value at issuance, with the residual proceeds allocated to the equity instruments, including common stock and pre-funded warrants.
Gain (Loss) from Change in Fair Value of Warrant Liability We recorded a gain o f $6.1 million durin g the year ended December 31, 2024 as compared to a loss of $1.0 million, during the year ended December 31, 2023, for the change in fair value on revaluation of our warrant liability associated with our warrants issued in conjunction with our stock offerings.
Gain from Change in Fair Value of Warrant Liabilities, Loss on Issuance of Warrant Liabilities and Loss on Extinguishment of Warrant Liabilities During the year ended December 31, 2025, we recognized a non-cash gain of $24.4 million related to the change in fair value of our warrant liabilities, a non-cash loss of $31.0 million associated with the issuance of warrant liabilities in connection with our equity offerings, and a $0.6 million non-cash loss related to the extinguishment of warrant liabilities that were reclassified to equity.
The fair value was estimated using the Black-Scholes option pricing model, based on the market value of the underlying common stock at the measurement date, the contractual term of the warrant, risk-free interest rates, expected dividends and expected volatility of the price of the underlying common stock.
The valuation model incorporates assumptions related to: • the market price of our common stock at the measurement date; • the contractual term of the warrants; • expected volatility of our common stock; • risk-free interest rates; and • expected dividend yield. The risk-free interest rate is based on observed interest rates of zero-coupon U.S.
General and Administrative Expense General and administrative (G&A) expense was $8.8 million a nd $10.0 million for the years ended December 31, 2024 and 2023, respectively. Th e decrease in G&A of $1.2 million wa s mainly attributable to a decrease in regulatory and legal services, and consulting & advisory fees.
The decrease in R&D of $1.8 million is primarily attributable to a reduction in sponsored research activities during the current year compared to the prior year. General and Administrative Expense General and administrative (G&A) expense was $9.1 million and $8.8 million for the years ended December 31, 2025 and 2024, respectively.
Transaction costs related to the offering were correspondingly fully allocated to warrant liabilities, and $1.0 million in related transaction costs were expensed during the year ended December 31, 2024.
Transaction Costs Allocated to Warrant Liabilities During the year ended December 31, 2025, we recognized $1.4 million of transaction costs associated with the issuance of Series E and Series F warrant liabilities in June and August 2025, respectively.
We estimate leasehold improvements to have an estimated useful life over the term of the lease or the estimated useful life, whichever is shorter; computer equipment to have a 2-year life; software to have a 3-year life, machinery and equipment to have a 2 to 5 year life and furniture and office equipment to have a 2 to 7 year life.
We estimate leasehold improvements to have an estimated useful life over the term of the lease or the estimated useful life, whichever is shorter; computer equipment to have a 2-year life; software to have a 3-year life, machinery and equipment to have a 2 to 5 year life and furniture and office equipment to have a 2 to 7 year life. 59 Table of Contents Accounting for warrants When the Company enters into arrangements pursuant to which warrants are issued, modified, or amended, we evaluate the terms of the warrants to determine the appropriate accounting and classification in accordance with FASB Accounting Standards Codification (“ASC”) Topic 480, Distinguishing Liabilities from Equity (“ASC 480”), ASC Topic 505, Equity (“ASC 505”), ASC Topic 815, Derivatives and Hedging (“ASC 815”), and ASC Topic 718, Compensation — Stock Compensation (“ASC 718”).
The combined purchase price for the securities was $1.07 per share of common stock (or pre-funded warrant in lieu thereof) and accompanying common warrant. Each pre-funded warrant is exercisable for one share of common stock at an exercise price of $0.001 per share.
In June 2025, we completed a public offering for the sale of 398,881 shares of common stock, and 244,319 pre-funded warrants to purchase shares of common stock, and Series E warrants to purchase up to 1,929,600 shares of common stock. The combined purchase price for the securities was $9.25 per share of common stock (or pre-funded warrant in lieu thereof).
We are required to revalue certain of the warrants at the time of each warrant exercise and at the end of each reporting period and reflect in the statement of operations a gain or loss from the change in fair value of the warrant in the period in which the change occurred.
The warrants were previously remeasured at fair value at the end of each reporting period, with the related liability reflected on our balance sheet. The changes in fair value during each previous reporting period were recognized as a gain (loss) from change in fair value of warrant liability in our consolidated statement of operations.
WP1066 IV: A better formulation for delivery intravenously of a molecule from the WP1066 portfolio to possibly further support future externally funded oncology clinical trials. Such a formulation will require additional preclinical work prior to a clinical trial. We have established a Recommended Phase 2 Dose for WP1122 to potentially enable future externally funded oncology and virology trials.
Externally funded – MB-110 utilizing AnnAraC for R/R AML as 2 nd line therapy in pediatrics is planned for 2027. We are researching potential sites for a grant funded trial. 2. WP1066: a. Externally funded - An improved formulation for delivery intravenously (IV) of a molecule from the WP1066 portfolio to possibly further support future externally funded oncology clinical trials.
Beyond this, we support development of our core technologies through several externally funded clinical trials and primarily externally funded non-clinical research, with the potential for further studies in the future.
We have established a Recommended Phase 2 Dose for WP1122 to potentially enable future externally funded oncology and virology trials. Beyond this, we support development of our core technologies through sponsored (internally funded) non-clinical research at MD Anderson. 55 Table of Contents Moleculin Biotech, Inc.
Annamycin, in a unique multilamellar lipid formulation, is our lead molecule and we have recently concluded one Phase 1B/2 clinical trial for treating Acute Myeloid Leukemia (AML) and are embarking on a Phase 3 clinical trial for the treatment of AML, which we believe will be pivotal.
The result is a next-generation anthracycline that we believe effectively addresses critical problems associated with currently used anthracyclines. It is our lead drug candidate, and we have concluded a Phase 1B/2 clinical trial for treating AML and are now conducting a Phase 3 clinical trial for R/R AML, which we believe will be registration enabling.
Removed
This Phase 3 trial should have an interim unblinding of data by the end of 2025, less than a year from its commencement, and an additional unblinding in the first half of 2026.
Added
We call this the “MIRACLE” trial (derived from Moleculin R/R AML AnnAraC Clinical Evaluation). A blinded preliminary data readout has already been released for the MIRACLE trial, and we expect to have an interim unblinding of data in mid-2026 and an additional unblinding in the second half of 2026, thereby concluding Part A of the two-part trial.
Removed
We believe such early visibility for a pivotal registration-enabling trial is highly unique in that stakeholders will receive preliminary safety and efficacy data in the “MIRACLE” trial (derived from Moleculin R/R AML AnnAraC Clinical Evaluation) within one year of dosing the first subject.
Added
One of these portfolios has an ongoing investigator-initiated trial, another one planned and additional active preclinical testing. Since our inception, drug candidates from each of the three core portfolios are in active planning for, have approval to begin, are currently in or have successfully completed eighteen clinical trials.
Removed
Annamycin is in a class of drugs referred to as Anthracyclines, which are an inhibitor of topoisomerase II, enabling them to cause DNA damage in rapidly replicating tumor cells.
Added
Whereas first- and second-generation anthracyclines are currently used to treat approximately half of all cancers, they carry with them the burden of dangerous cardiotoxicity and efficacy limitations associated with multidrug resistance mechanisms and poor tissue/organ distribution.
Removed
Annamycin is also in two Phase 1B/2 clinical trials for treating Soft Tissue Sarcoma metastasized to the lungs (STS lung metastases, STS lung mets, or Advanced STS).
Added
Later generation anthracyclines are comprised mainly of attempts to reduce cardiotoxicity or enhance anticancer activity but we believe have failed to deliver the necessary safety and efficacy profiles to win approval or gain market acceptance.
Removed
Annamycin was designed to avoid multidrug resistance and to be non-cardiotoxic and, with intensive cardiac monitoring, has shown no cardiotoxicity in subjects treated in our five Annamycin clinical trials to date.
Added
Annamycin is an entirely new chemical entity integrating a new molecular structure intended to enhance its ability to selectively kill cancer cells and reduce toxic side effects. Annamycin possesses a unique and innovative multilamellar lipid-based delivery system designed to improve bioavailability and to increase therapeutic window.
Removed
Furthermore, we have demonstrated safe dosing significantly beyond the dose limitations imposed by regulatory authorities upon commonly prescribed anthracyclines due to their inherent cardiotoxicity. b) Our WP1066 Portfolio includes WP1066, WP1193 and WP1220, three of several Immune/Transcription Modulators in the portfolio designed to inhibit p-STAT3 (phosphorylated signal transducer and activator of transcription) among other transcription factors associated with tumor activity.
Added
On an all comer’s basis (no limit on prior lines of therapy), the population of subjects who achieved a CR (n=8) demonstrated durability of approximately 13 months with median overall survival of 15 months and developing at the time of analysis, with 4 of 8 subjects (50%) censored; ● Shown promising blinded safety and efficacy data for the first 30 subjects treated in the MIRACLE trial as we head towards the planned interim unblinding of data for the first 45 subjects treated with efficacy data in Part A of the MIRACLE trial in mid-2026; ● Shown encouraging activity in a total of five different clinical trials (most of which are now complete), funded both internally and externally (through investigator-initiated trials); specifically including relapsed and refractory AML with complete remission rates significantly above currently approved second line therapies and STS lung mets with overall survival as a median seventh line therapy comparable to overall survival seen with approved first line monotherapies; and ● Obtained composition of matter patent protection through 2040 with the potential to extend as far as 2045 as well as Orphan Drug and Fast Track designation.
Removed
In all of our discussions, clinical data, where a CSR or its equivalent has not been published, are considered preliminary and subject to change. Clinical Trials Summary We have multiple active INDs/CTAs (Investigational New Drug authorization in the US or Clinical Trial Authorization in Europe).
Added
One of our core management beliefs is that anthracyclines represent one of the most important treatments available for AML and Advanced STS, as well as many other cancers, and we believe Annamycin may, for the first time ever, allow a majority of these patients to benefit from Annamycin’s improved safety and efficacy by allowing increased dosing (and even maintenance dosing), improved tolerability and allowing access for patients who otherwise would not be eligible for an anthracycline (including the elderly and those with impaired cardiac function).
Removed
These INDs/CTAs are under development, approved, in progress, or completed and total fourteen clinical trials, internally and externally funded.
Added
Externally funded – Based on sponsored research, we intend to begin in 2026 a Phase 1B/2 clinical trial with Annamycin as a monotherapy treating 3 rd line pancreatic cancer. d. Internally funded – MB-109 utilizing AnnAraC for R/R AML as 3 rd line therapy in adults is planned for 2027. e.
Removed
With Annamycin, we currently have active two AML clinical trials MB-106 which is a Phase 1B/2 treating AML with AnnAraC (recruitment is closed and is in subject follow-up) and MB-108 which is a Phase 2B/3 pivotal clinical trial treating R/R AML as 2 nd line therapy and is just starting.
Added
Such a formulation requires additional preclinical work prior to a clinical trial. b. Externally funded - Phase 1B/2 trial for WP1066 oral formulation in combination with radiation treating glioblastoma (GBM) both in adults and pediatrics. An adult clinical trial is in process and a pediatric trial is in the planning stage.
Removed
Additionally, there are one externally funded Phase 1B/2 trial treating STS Lung Mets with Annamycin as monotherapy. This trial is closed and is in follow-up on the trial’s subjects. With WP1066, we have an externally funded phase 1B/2 in combination with radiation treating GBM at Northwestern University that is actively recruiting. Moleculin Biotech, Inc.
Added
The increase in G&A of $0.3 million was primarily attributable to higher regulatory and legal services, as well as increased consulting and investor relations expenses.
Removed
The decrease in R&D of $1.8 million is mainly related to the $1.5 million WPD sublicense termination in 2023, which enabled the reacquisition of our intellectual property rights in certain territories, including parts of the European Union.
Added
Certain of our warrants are classified as liabilities and are remeasured at fair value upon issuance and at each reporting date, as well as upon modification or inducement. Changes in the fair value of these instruments are recognized in the consolidated statements of operations in the period in which they occur.
Removed
We calculated the fair value of the warrants outstanding using the Black-Scholes model. Generally, a gain results principally from a decline in our share price during the period and a loss results principally from an increase in our share price.
Added
The fair value of these warrant liabilities is estimated using a Monte Carlo simulation model, which incorporates significant assumptions including expected volatility, expected term, risk-free interest rate, and the contractual terms of the warrants, including path-dependent features.
Removed
Net Loss The net loss for the year ended December 31, 2024 wa s $21.8 million, which included a non-cash gain of $6.1 million on wa rrants in 2024 as compared to a loss of $1.0 million in the prior year and approximatel y $1.7 million o f stock-based compensation expense in 2024 as compared to $2.0 million in 2023.
Added
Because the valuation is sensitive to changes in our stock price and other inputs, the resulting gains or losses can be significant and may fluctuate materially from period to period.
Removed
In March 2022, we received a subpoena from the SEC requesting information and documents, including materials related to certain individuals (none of which are our officers or directors) and entities, and materials related to the development of and statements regarding our drug candidate for the treatment of COVID-19.
Added
In general, decreases in our stock price during a reporting period result in non-cash gains, while increases in our stock price result in non-cash losses related to the remeasurement of warrant liabilities.
Removed
We have received, and expect to continue to receive, periodic further requests from the SEC staff with respect to this matter.
Added
The current-year net loss includes a $31.0 million non-cash loss related to the issuance of warrant liabilities, a $24.4 million non-cash gain from the change in the fair value of warrant liabilities, and a $0.6 million non-cash loss on the extinguishment of warrant liabilities that were reclassified to equity.
Removed
We are not aware of the specific nature of the underlying investigation by the SEC, and to the extent that this investigation relates to prior public disclosures that we have made, we believe in the accuracy and adequacy of such prior disclosures.
Added
Liquidity and Capital Resources As of December 31, 2025, we had cash and cash equivalents of $8.9 million and prepaid expenses and other current assets of $0.8 million. We also had $3.5 million of accounts payable and $3.3 million of accrued expenses and other current liabilities.
Removed
The correspondence from the SEC transmitting the subpoena to us states that the SEC is trying to determine whether there have been any violations of federal securities laws, but that its investigation does not mean that the SEC has concluded that anyone has violated the law or that the SEC has a negative opinion of any person, entity, or security.
Added
Recent Stock Offerings We have historically funded our operations through a combination of at-the-market equity sales, public offerings, and warrant exercises, including warrant exercise inducement transactions, and we expect to continue to rely on these sources of capital to support our operations.
Removed
We cannot predict when this matter will be resolved or what, if any, action the SEC may take following the conclusion of the investigation. During the years ended December 31, 2024 and 2023, we have expensed approximately $0.2 million and $1.5 million, respectively, in related legal fees and expenses, which has impacted and may continue to impact our liquidity.
Added
On February 19, 2026, we entered into warrant inducement agreements with certain holders of our existing equity-classified Series E and Series F warrants. Pursuant to these agreements, the holders exercised warrants to purchase an aggregate of 2,122,652 shares of our common stock at an exercise price of $3.90 per share, resulting in gross proceeds of approximately $8.3 million.
Removed
The pre-funded warrants are exercisable immediately and may be exercised at any time until all of the pre-funded warrants are exercised in full, subject to the beneficial ownership limitation. Each common warrant will be exercisable upon the receipt of shareholder approval, will have an exercise price of $1.07 per share, and expire five years from the initial exercise date.
Added
In consideration for such exercises, we issued new Series H warrants to purchase up to 6,367,956 shares of common stock. The Series H warrants are exercisable upon shareholder approval, have a term of five years from such approval, and an exercise price of $2.3976 per share. The Series H warrants contain customary anti-dilution adjustments and beneficial ownership limitations.
Removed
In August 2024, the Company entered into a securities purchase agreement with an institutional investor for the sale by the Company of 283,000 shares of common stock, and 2,183,368 pre-funded warrants to purchase shares of common stock, series A warrants to purchase up to 2,466,368 shares of common stock, series B warrants to purchase up to 2,466,368 shares of common stock, and placement agent warrants.

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