On November 13, 2025 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), reported an enrollment update with 60% of the target number of subjects for the first planned interim unblinding of data having consented to its pivotal Phase 2B/3 "MIRACLE" study of Annamycin in combination with cytarabine for the treatment of adult patients with acute myeloid leukemia (AML) who are refractory to or relapsed (R/R) after induction therapy (R/R AML). The targeted number for the first unblinding of data is 45 subjects. Additional subjects continued to be identified by site investigators. This update is as of November 4, 2025, as identification and recruitment are ongoing. The Company expects to complete treatment of the first 45 subjects in the first quarter of 2026 with the initial unblinding of data thereafter.

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Walter Klemp, Chairman and CEO of Moleculin, commented, "We’re very encouraged by the strong momentum in recruitment and enthusiasm I’ve personally heard from investigators around Europe and the US. To date, we are seeing blinded response activity tracking within our expected range, based on historical responses of the trial arm equivalents. Although enrollment at some sites in Europe has been impacted by bed shortages, the MIRACLE study continues to progress as planned. As we move toward our first unblinding milestone, we are excited about Annamycin’s potential to fill a major gap in AML treatment. We believe we’re well on our way to determining if Annamycin has the potential to offer a much-needed, safer, and more effective option for patients facing this devastating disease."

MIRACLE Trial Progress and Next Steps

The MIRACLE study (derived from Moleculin R/R AML AnnAraC Clinical Evaluation) is a Phase 2B/3, global multi-center, randomized, double-blind, placebo-controlled, adaptive designed clinical trial whereby data from the 2B (Part A) portion will be combined with the Phase 3 (Part B) portion for purposes of measuring its primary efficacy endpoint. The protocol for the MIRACLE trial allows for the unblinding of preliminary primary efficacy data (Complete Remission or CR) and safety/tolerability of the three arms at 45 subjects, in addition to the conclusion of Part A (at 75 to 90 subjects). The first early unblinding will yield 30 subjects treated with Annamycin (190mg/m2 and 230 mg/m2) in combination with HiDAC and 15 subjects treated with just HiDAC plus placebo.

The Company expects to reach the recruitment and treatment of the first 45 subjects in the first quarter of 2026 with unblinding thereafter, in addition to the second unblinding, which is expected in the first half of 2026. This accelerated estimated timeline is due in part to the positive response the Company received in meetings during December with potential investigators regarding recruitment for the trial.

The currently enrolled subjects are from sites across five countries, providing a diverse base of subjects. With the upcoming holidays in addition to unexpected bed shortages at certain EU sites, the Company expects treatment of enrolled subjects to slow, pushing the completion of the first 45 subjects into the first quarter of 2026. Such data will be audited, locked, and reviewed prior to release. The release of the unblinded data will be thereafter.

For more information about the MIRACLE trial, visit clinicaltrials.gov and reference identifier NCT06788756. Additionally, the clinical trial in the EU is on euclinicaltrials.eu and the reference identifier there is 2024-518359-47-00.

Annamycin, also known by its non-proprietary name of naxtarubicin, currently has Fast Track Status and Orphan Drug Designation from the FDA for the treatment of relapsed or refractory acute myeloid leukemia, in addition to Orphan Drug Designation for the treatment of soft tissue sarcoma. Annamycin also benefits from composition of matter patent protection through 2040 with the potential to extend that protection as far as 2045. Furthermore, Annamycin has Orphan Drug Designation for the treatment of relapsed or refractory acute myeloid leukemia from the EMA.

(Press release, Moleculin, NOV 13, 2025, View Source [SID1234659907])

On November 13, 2025 Taiho Oncology, Inc., a company developing and commercializing novel treatments for hematologic malignancies and solid tumors, reported the presentation of new data from 14 studies across company-sponsored and company-funded externally led studies at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, to be held Dec. 6-9, 2025, in Orlando, Florida.

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Taiho Oncology will share findings from clinical trials in two oral presentations and evidence from real-world studies in two online abstracts.

The oral presentations will highlight results from the Phase 2 part of ASTX030-01, a randomized, open-label, crossover trial comparing oral combination of azacitidine and cedazuridine to subcutaneous azacitidine and the Phase 2 portion of the Phase 1/2 trial ASTX727-03 of low-dose versus standard-dose oral decitabine and cedazuridine in patients with lower-risk myelodysplastic syndromes (MDS).
The two online posters will present real-world data on U.S. adults with acute myeloid leukemia (AML), describing treatment patterns and outcomes with hypomethylating agent (HMA) plus venetoclax, and evaluating the time burden of initiating parenteral HMA-based therapy in routine practice.
Additionally, 10 presentations – including four oral presentations – led by independent investigators will feature data from Taiho-funded investigator-initiated or collaborative studies. These presentations will highlight:

Results from a UK multicenter Phase 2 randomized trial comparing ASTX727 with hydroxycarbamide or best supportive care in advanced MDS/ myeloproliferative neoplasm (MPN).
Data from a Phase 2 study evaluating the all-oral combination of ASTX727, venetoclax and revumenib in newly diagnosed AML patients with NPM1m, KMT2Ar or NUP98r AML mutations.
Results of a Phase 1/2 clinical trial of the combination of oral decitabine/cedazuridine with the venetoclax in patients with untreated high-risk MDS and chronic myeloid leukemia (CMML).
Safety and efficacy results from a Phase 1/2 trial evaluating an all-oral combination of ASTX727, venetoclax and gilteritinib in both relapsed refractory and newly diagnosed FLT3-mutated AML, high-risk MDS or MPN.
"As the leader of oral HMA development, we’re excited that these data will be featured in six oral presentations at the ASH (Free ASH Whitepaper) Annual Meeting," said Harold Keer, MD, PhD, Taiho Oncology Chief Medical Officer. "These data highlight our increasing understanding of novel oral regimens in hematology. We are invested in developing treatments that have the potential to improve flexibility and lower the treatment burden for patients, and collaborating with leading institutions, evidenced by our continued support of independent clinical research to advance cancer care. We look forward to participating in the essential scientific exchange that contributes to progress in hematology at the ASH (Free ASH Whitepaper) Annual Meeting."

Data being presented by Taiho Oncology and its partners at the 2025 ASH (Free ASH Whitepaper) Annual Meeting and Exposition include:

Company-Sponsored Studies

Oral Presentations

Title: A phase 2 dose confirmation trial of oral ASTX030, a combination of oral azacitidine with cedazuridine among patients with myelodysplastic syndromes, chronic myelomonocytic leukemia, and acute myeloid leukemia
Abstract Number: 491
Session Name: 637. Myelodysplastic Syndromes: Clinical and Epidemiological: Moving the Needle Through Novel Approaches in MDS and CMML
Session Date: Dec. 7, 2025
Session Time: 9:30 a.m. to 11a.m.
Presentation Time: 10:15 to 10:30 a.m.
Location: OCCC – Valencia Room W415D
Presenter: Guillermo Garcia-Manero, MD

Title: Low-dose oral decitabine and cedazuridine among patients with low-risk myelodysplastic syndromes
Abstract Number: 790
Session Name: 637. Myelodysplastic Syndromes: Clinical and Epidemiological: Treatment Advances in Lower risk Myelodysplastic Syndromes and Myelodysplastic/Myeloproliferative Neoplasms
Session Date: Dec. 8, 2025
Session Time: 10:30 a.m. to 12 p.m.
Presentation Time: 11:15 to 11:30 a.m.
Location: OCCC – Valencia Room W415A
Presenter: Guillermo Garcia-Manero, MD

Online Abstracts

Title: Time burden of treatment with parenteral HMAs in combination with venetoclax among patients with newly diagnosed AML
Abstract Number: 6990
Lead Author: Amer Zeidan, MD

Title: Real-World Characteristics, Treatment Modifications, and Outcomes for AML Patients Receiving HMAs + Venetoclax (1L)
Abstract Number: 8176
Lead Author: Amer Zeidan, MD

Company-Funded Externally Led Studies

Oral Presentations

Title: Phase II Study of the All-Oral Combination of Revumenib (SNDX-5613) with Decitabine/Cedazuridine (ASTX727) and Venetoclax (SAVE) in Newly Diagnosed AML
Abstract Number: 47
Session Name: 617. Acute Myeloid Leukemias: Commercially Available Therapies: Frontline treatment with intensive or lower-intensity regimens
Session Date: Dec. 6, 2025
Session Time: 9:30 to 11 a.m.
Presentation Time: 10:30 to 10:45 a.m.
Location: OCCC – Valencia Room W415A
Presenter: Wei-Ying Jen, BM BCh, FRCPath

Title: Oral decitabine/cedazuridine in combination with venetoclax in treatment-naïve high-risk myelodysplastic syndrome or chronic myelomonocytic leukemia: Updates of a Phase 1/2 clinical trial
Abstract Number: 237
Session Name: 637. Myelodysplastic Syndromes: Clinical and Epidemiological: Treatment Advances in Higher Risk Myelodysplastic Syndromes
Session Date: Dec. 6, 2025
Session Time: 2 to 3:30 p.m.
Presentation Time: 2:30 to 2:45 p.m.
Location: OCCC – Chapin Theater W415BC
Presenter: Alex Bataller, MD, PhD

Title: ASTX727 delivers superior response rates and associated survival benefit versus hydroxycarbamide/best supportive care in CMML and other MDS/MPN overlap syndromes: First results from the Phase 2 UK multicenter randomized ammo trial
Abstract Number: 488
Session Name: 637. Myelodysplastic Syndromes: Clinical and Epidemiological: Moving the Needle Through Novel Approaches in MDS and CMML
Session Date: Dec. 7, 2025
Session Time: 9:30 to 11 a.m.
Presentation Time: 10:45 to 11 a.m.
Location: OCCC – Valencia Room W415D
Presenter: Daniel H. Wiseman, MB, ChB, PhD

Title: Phase I/II Study of Decitabine/Cedazuridine (ASXT727), Venetoclax, and Gilteritinib for Patients with FLT3-Mutated Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome
Abstract Number: 997
Session Name: 617. Acute Myeloid Leukemias: Commercially Available Therapies: Frontline treatment with intensive or lower-intensity regimens
Session Date: Dec. 8, 2025
Session Time: 4:30 to 6 p.m.
Presentation Time: 4:30 to 4:45 p.m.
Location: OCCC – Chapin Theater W320
Presenter: Roberta S. Azevedo, MD

Poster Presentations

Title: Translational Investigation of Tolinapant (ASTX660) in Acute Myeloid Leukemia Using Integrated Clinical, Bioinformatic, and Pharmacological Approaches
Abstract Number: 2552
Session Name: 802. Chemical Biology and Experimental Therapeutics: Poster I
Session Date: Dec. 6, 2025
Session Time: 5:30 to 7:30 p.m.
Location: OCCC – West Halls B3-B4

Title: A Randomized Phase II Trial of ASTX727 and Venetoclax With or Without Enasidenib for Newly Diagnosed Older Adults With IDH2 Mutant Acute Myeloid Leukemia: A MyeloMATCH Substudy (MM1OA-S03)
Abstract Number: 3466
Session Name: 617. Acute Myeloid Leukemias: Commercially Available Therapies: Poster II
Session Date: Dec. 7, 2025
Session Time: 6 to 8 p.m.
Location: OCCC – West Halls B3-B4

Title: Trial in progress: A Phase I Study Evaluating the Safety of cirtuvivint (CIRT) as Monotherapy and in Combination with ASTX727 in Patients with MDS and AML
Abstract Number: 3431
Session Name: 616. Acute Myeloid Leukemias: Investigational Drug and Cellular Therapies: Poster II
Session Date: Dec. 7, 2025
Session Time: 6 to 8 p.m.
Location: OCCC – West Halls B3-B4

Title: Trial in Progress – A Randomized Study of ASTX727 With or Without Ladademstat in Accelerated/Blast-Phase Myeloproliferative Neoplasms
Abstract Number: 3827
Session Name: 634. Myeloproliferative Syndromes: Clinical and Epidemiological: Poster II
Session Date: Dec. 7, 2025
Session Time: 6 to 8 p.m.
Location: OCCC – West Halls B3-B4

Title: A Comparison of Overall Survival and Quality of Life in MDS Patients Treated with Azacitidine vs Decitabine: A Propensity-Matched Registry Study
Abstract Number: 3862
Session Name: 637. Myelodysplastic Syndromes: Clinical and Epidemiological: Poster II
Session Date: Dec. 7, 2025
Session Time: 6 to 8 p.m.
Location: OCCC – West Halls B3-B4

Title: A Multiarm Phase 1b Study of Personalized Oral Maintenance Therapy with Decitabine/Cedazuridine (ASTX727) Plus Physician’s Choice of Venetoclax, Gilteritinib, Enasidenib, or Ivosidenib in Acute Myeloid Leukemia
Abstract Number: 5228
Session Name: 617. Acute Myeloid Leukemias: Commercially Available Therapies: Poster III
Session Date: Dec. 8, 2025
Session Time: 6 to 8 p.m.
Location: OCCC – West Halls B3-B4

(Press release, Taiho, NOV 13, 2025, View Source [SID1234659936])

On November 13, 2025 Immutep Limited (ASX: IMM; NASDAQ: IMMP) ("Immutep" or "the Company"), a late-stage immunotherapy company targeting cancer and autoimmune diseases, reported positive data from the EFTISARC-NEO trial that were detailed in an oral presentation by Paweł Sobczuk, M.D., Ph.D., Maria Sklodowska-Curie National Research Institute of Oncology, Warsaw, Poland, at the Connective Tissue Oncology Society (CTOS) 2025 Annual Meeting held in Boca Raton, Florida.

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The investigator-initiated Phase II study evaluating eftilagimod alfa (efti) with radiotherapy plus KEYTRUDA (pembrolizumab) in the neoadjuvant setting for resectable soft tissue sarcoma (STS) significantly exceeded the study’s prespecified level of pathologic response rates. In the evaluable patient population (N=38), the novel combination with efti reached a median 51.5% tumour hyalinization/fibrosis (p<0.001), meeting the study’s primary endpoint.1

These promising results were achieved in patients with ten different STS subtypes including rare and/or highly aggressive tumours with poor prognosis such as myxofibrosarcoma (N=16), undifferentiated pleomorphic sarcoma (N=10) and malignant peripheral nerve sheath tumor (N=2).

Early translational data from the initial twenty patients who underwent surgery in the trial show a strong immune system activation in line with efti’s mode of action, with statistically significant increases in the expression of key cytokines and chemokines in peripheral blood — specifically CXCL9, CXCL10, IL-23, and IFN-g.

Immune Response Biomarkers (fold change from week 1 through pre-surgery)

Serum Biomarker Fold change (p-value)
C-X-C motif chemokine ligand 9 (CXCL9)

2.5x (p<0.01)
C-X-C motif chemokine ligand 10 (CXCL10)

1.8x (p<0.0001)
Interleukin-23 (IL-23)

2.2x (p<0.05)
Interferon-gamma (IFN-g)

2.5x (p<0.05)
The increase on treatment of immune response biomarkers like IFN-gamma correlated with pathologic responses in this study, meaning patients with a biomarker increase during treatment also had a higher probability of a good clinical response at surgery.

The promising tumour hylanization/fibrosis rate achieved (over 3X greater than standard-of-care radiotherapy based on historical data)1 may hold significance in terms of future outcomes as it serves as an early surrogate endpoint correlated with enhanced overall survival and recurrence-free survival in STS patients.2,3 Disease-free survival and overall survival data are immature at this stage and will be presented in the future. Further correlative translational studies are also ongoing.

Dr. Paweł Sobczuk, one of the trial’s principal investigators, stated: "We are excited to share these strong results in resectable soft tissue sarcoma, a challenging indication with a high unmet medical need. This level of efficacy, observed across ten different STS subtypes including rare, highly aggressive tumours with poor prognosis, further supports the hypothesis that efti’s distinctive activation of antigen-presenting cells—and consequent induction of both adaptive and innate immunity—plays a key role in driving a coordinated immune response to fight cancer. This novel combination with neoadjuvant efti warrants further investigation in registrational settings."

"Our team was delighted to have recently been awarded the distinguished Golden Scalpel Award (Złoty Skalpel)* for EFTISARC-NEO. This honour is reserved for projects that demonstrate exceptional innovation and impact in medical research and clinical practice," added Dr. Sobczuk.

Dr. Frédéric Triebel, CSO of Immutep, said: "We are pleased to see a confirmation of earlier promising data on now 38 patients in this difficult-to-treat cancer. The prolonged increase in serum immune response biomarkers, observed two weeks after efti subcutaneous injection, is indicative of a robust adaptive and innate immune response. This enhanced immune activity is crucial because it means the body’s own defences are being mobilised to target and destroy cancer cells more effectively, further supporting the positive impact of the observed pathologic responses. The recent results suggest that efti may have potential applications beyond advanced or metastatic cancer, extending into earlier-stage disease."

As neoadjuvant immunotherapy becomes more established in the treatment of early-stage cancers, the findings from EFTISARC-NEO highlight the possibility for efti to be used in patients who have a lower tumor burden at diagnosis. This could expand the range of patients who might benefit from efti, potentially increasing its role in the treatment landscape for cancers that are still localized and resectable.

STS is an orphan disease with high unmet medical need and a poor prognosis for patients. The incidence of STS varies in different regions globally. In the United States, the number of new STS cases in 2025 is estimated to be ~13,520 with ~5,420 deaths, according to the American Cancer Society.4

For more information on EFTISARC-NEO, visit clinicaltrials.gov (NCT06128863). The CTOS 2025 oral presentation slides can be found on the Posters & Publications page of Immutep’s website.

*About the Golden Scalpel Award in Poland

The Golden Scalpel Award in Poland is recognized as a benchmark of excellence within the medical community. It is presented by independent experts to initiatives that set new standards in advancing healthcare. This year, EFTISARC NEO was the only oncology project to receive this accolade, underscoring its leadership and breakthrough potential in cancer treatment.

About Eftilagimod Alfa (Efti)

Efti is a novel immunotherapy that directly activates antigen-presenting cells or APCs (e.g. dendritic cells, monocytes) via the MHC Class II pathway to fight cancer. As an MHC Class II agonist, its activation of APCs engages the adaptive and innate immune system to initiate a broad anti-cancer immune response. This includes priming and activating cytotoxic T cells as well as generating important co-stimulatory signals & cytokines that further boost the immune system’s ability to combat cancer.

Efti is under evaluation for a variety of solid tumours including non-small cell lung cancer (NSCLC) in a pivotal Phase III trial called TACTI-004 (KEYNOTE-F91), as well as head and neck squamous cell carcinoma (HNSCC), soft tissue sarcoma, and breast cancer. Its favourable safety profile enables various combinations like with anti-PD-[L]1 immunotherapy, radiotherapy, and/or chemotherapy. Efti has received Fast Track designation in first line HNSCC and in first line NSCLC from the United States Food and Drug Administration (FDA).

(Press release, Immutep, NOV 13, 2025, View Source [SID1234659971])

On November 13, 2025 Aura Biosciences, Inc. (NASDAQ: AURA), a clinical-stage biotechnology company developing precision therapies for solid tumors designed to preserve organ function, reported financial results for the third quarter ended September 30, 2025, and provided recent business highlights.

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"In the third quarter, we remained focused on clinical execution in both our global Phase 3 CoMpass trial in early choroidal melanoma and our Phase 1b/2 trial in NMIBC," said Elisabet de los Pinos, Ph.D., Chief Executive Officer of Aura Biosciences. "Enrollment has taken longer than we expected in early choroidal melanoma due to the requirement to document active tumor growth prior to patient enrollment and other unique enrollment challenges with the first Phase 3 trial in this rare indication. In 2025, we implemented measures to address these operational challenges and have experienced improved enrollment in recent months. We believe we are now in a position to provide guidance regarding the topline data readout for the study."

"Our NMIBC trial remains on track, with data expected in mid-2026. Recently presented Phase 1 trial immune profiling data reinforce bel-sar’s distinct dual mechanism, driving focal anti-tumor immune activation. We believe these findings highlight bel-sar’s potential as a differentiated frontline, organ-preserving treatment to deliver durable responses across the bladder cancer spectrum and in other solid tumors."

Recent Pipeline Developments

Early Choroidal Melanoma

Ongoing Phase 3 CoMpass Trial: CoMpass is the first registration-enabling trial in early choroidal melanoma. The trial is a global, Phase 3, randomized trial evaluating bel-sar treatment against a sham control arm utilizing an enrichment strategy to enroll approximately 100 patients with documented tumor growth. We believe the study enrollment has been slower than expected due to the operational challenges of implementing the first Phase 3 trial in this rare indication across global sites and the requirement of active growth in our inclusion criteria, which often requires sites to follow patients for variable periods of time until they demonstrate required growth.

Approximately 90% of targeted clinical sites are now activated, and we have implemented measures to address multiple operational challenges. Our patient identification tool has shown strong growth, with more than 400 patients having been entered since June 2024, and the current number of potentially eligible patients having increased to 280. We believe this highlights the enrollment opportunity and the significant unmet need.

Based on trial activity in recent months and the growing number of potentially eligible patients, the Company currently expects that it can complete CoMpass enrollment in 2026 and provide topline data readout for the 15-month primary endpoint in the fourth quarter of 2027. This estimate assumes enrollment rates that are generally consistent with the rates experienced in recent months.

Currently, there are no approved vision-preserving therapies for patients with early choroidal melanoma. The standard of care remains radiotherapy, which frequently results in irreversible vision loss. Bel-sar has the potential to become the first frontline vision-preserving therapy in this setting. The Company previously received Orphan Drug Designation from the FDA and the European Medicines Agency and Fast Track designation from the FDA for the treatment of early choroidal melanoma. The CoMpass trial is under a Special Protocol Assessment agreement with the FDA.

Bladder Cancer

Additional Phase 1 Biological Activity Data Presented at the 45th Congress of the Société Internationale d’Urologie (SIU): Multiplex immune fluorescence data from the Phase 1 trial of bel-sar demonstrating robust induction of adaptive immune memory in patients with non-muscle invasive bladder cancer (NMIBC) were presented by Seth P. Lerner, M.D., Professor of Urology at Baylor College of Medicine at SIU, held October 29–November 1, 2025, in Edinburgh, United Kingdom: link. These multiplex immune fluorescence data (n=5 patients from the Phase 1 trial) further characterize the previously announced results from the Company’s completed Phase 1 clinical trial of bel-sar in patients with NMIBC.

These data revealed that a single focal administration of bel-sar induced adaptive immune memory through generation of de novo mature tertiary lymphoid structures (TLS) in 3/5 participants evaluated. Bel-sar also generated innate and adaptive effectors regardless of immune environment and converted immune-cold or exhausted lesions into active, immune-hot microenvironments. In treated lesions, natural killer cell density increased up to 40x, CD4+ cytolytic T cell density increased up to 7x, and CD4+ and CD8+ memory T cells were observed after bel-sar treatment.

This biological activity profile reinforces bel-sar’s potential as a frontline therapy designed to treat the tumor, activate durable anti-tumor immunity, and reduce recurrence risk across the bladder cancer spectrum.

Ongoing Phase 1b/2 Trial: This trial will evaluate additional doses and cycles of bel-sar in approximately 26 intermediate and high-risk NMIBC patients. Patients will be monitored for response assessments and recurrence at 3, 6, 9, and 12 months. This trial is actively enrolling and remains on track, with initial three-month clinical data expected in mid-2026.

Metastases to the Choroid

The Company has dosed the first patient in an ongoing Phase 2 clinical trial in metastases to the choroid. A protocol amendment has now been implemented to expand the entry criteria to include all metastases arising from different solid tumors to facilitate enrollment into the study and broaden the proof of concept for bel-sar in this indication. This approach is supported by preclinical models that demonstrate robust efficacy across multiple primary solid tumors. The Company is evaluating further expansion of the inclusion criteria for this trial to address the high unmet medical need. With a four-week efficacy endpoint, the Company expects early proof of concept data from this trial in 2026.

Metastases to the choroid is an indication with high unmet medical need and no approved therapies with an incidence of 20,000 patients annually in the United States and Europe. Bel-sar has the potential to treat a wide variety of tumor types in the choroid that metastasize from various primary tumors. The Company previously received FDA Fast Track designation for bel-sar in this indication.

Cancers of the Ocular Surface

The Company is planning to initiate a Phase 1 proof-of-concept trial to assess safety, feasibility and tumor response through histopathologic evaluation at a 2–4-week time point. Development activities for this program remain on track, with early proof of concept data from Australian clinical sites expected in 2026.

Cancers of the ocular surface affect approximately 35,000 patients in the United States and Europe annually and are associated with a particularly high incidence in regions such as Australia. There are currently no approved therapies for these tumors.

Third Quarter 2025 Financial Results

•
As of September 30, 2025, the Company had cash and cash equivalents and marketable securities totaling $161.9 million. The Company believes its current cash and cash equivalents and marketable securities are sufficient to fund its operations into the first half of 2027. The Company remains focused on driving a cash-efficient operation to deliver data across its pipeline.

•
Research and development expenses increased to $22.2 million for the three months ended September 30, 2025 from $17.0 million for the three months ended September 30, 2024, primarily due to ongoing clinical and clinical research organization (CRO) costs associated with the progression of our global Phase 3 trial of bel-sar in early choroidal melanoma and higher personnel expenses related to growth of our Company.

•
General and administrative expenses decreased to $5.7 million for the three months ended September 30, 2025 from $6.2 million for the three months ended September 30, 2024. General and administrative expenses include $1.7 million and $1.6 million of stock-based compensation for the three months ended September 30, 2025 and 2024, respectively. The decrease was primarily driven by reduced professional fees.

•
Net loss for the three months ended September 30, 2025 was $26.1 million compared to $21.0 million for the three months ended September 30, 2024.

(Press release, Aura Biosciences, NOV 13, 2025, View Source [SID1234659892])

On November 13, 2025 Monopar Therapeutics Inc. ("Monopar" or the "Company") (Nasdaq: MNPR), a clinical‐stage biopharmaceutical company developing innovative treatments for patients with unmet medical needs, reported third quarter 2025 financial results and recent developments.

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Recent Developments

ALXN1840 for Wilson Disease

On September 14-15, 2025, the Company presented new data on the long-term neurological efficacy and safety of ALXN1840 (tiomolybdate choline) at the 150th American Neurological Association (ANA) Annual Meeting. Matthew Lorincz, M.D., Ph.D., Professor of Neurology and Co-Director of the Wilson Disease Center of Excellence at the University of Michigan delivered the poster and oral presentations. The new findings presented at ANA highlight the long-term neurological benefit of ALXN1840, and follow the European Association for the Study of the Liver (EASL) International Liver Congress presentation in May on the long-term hepatic and systemic safety and efficacy data. Together, these findings underscore the potential of ALXN1840 to favorably impact both neurological and hepatic manifestations of Wilson disease.

On November 9, 2025, the Company presented new data and analyses from the Phase 2 ALXN1840-WD-204 copper balance study at the American Association for the Study of Liver Diseases (AASLD) – The Liver Meeting 2025. In an oral presentation titled "Rapidly Improved Cu Balance in Wilson Disease Patients on Tiomolybdate Choline," Professor Aftab Ala, MBBS, M.D., FRCP, Ph.D., Professor of Hepatology and Consultant Hepatologist at the Institute of Liver Studies at King’s College Hospital in London, shared results showing that treatment with ALXN1840 led to a rapid and sustained improvement in daily copper balance in patients with Wilson disease, primarily through increased fecal copper excretion.

Monopar is preparing to submit a New Drug Application ("NDA") to the FDA in early 2026.

MNPR‐101 for Radiopharmaceutical Use

On September 26, 2025, Monopar received FDA clearance on its IND application for MNPR-101-Lu, which covers the protocol titled "Phase 1, Open-Label, Multicenter, Dosimetry and Dose-Escalation Trial to Characterize the Safety, Tolerability, and Anti-Tumor Activity of Fractionated MNPR-101-Lu Dosing in the Treatment of uPAR-Expressing Advanced or Metastatic Solid Tumors." This IND incorporates the Company’s proprietary linker technology, which has been designed to enhance the stability and biodistribution of its therapeutic radiopharmaceuticals.

Recent Financing Capital Raise and Share Repurchase

On September 23, 2025, the Company priced an underwritten public offering (the "Offering") consisting of (i) 1,034,433 shares of its common stock and (ii) pre-funded warrants to purchase 960,542 shares of common stock, pursuant to an underwriting agreement (the "Underwriting Agreement") with Morgan Stanley & Co. LLC, Leerink Partners LLC, and Barclays Capital Inc. (the "Underwriters"). The public offering price was $67.67 per share and $67.669 per pre-funded warrant, which represents the per share offering price less a $0.001 per share exercise price. The aggregate net proceeds from the Offering were approximately $126.9 million, after deducting underwriting discounts and commissions but before offering expenses and the Share Repurchase (as defined below).

On September 24, 2025, the Company entered into a share purchase agreement (the "Share Purchase Agreement") with Tactic Pharma LLC ("Tactic Pharma"), an existing significant stockholder that held approximately 13.4% of the Company’s common stock prior to the Offering and Share Repurchase. Pursuant to the Share Purchase Agreement, the Company used $35 million of the Offering proceeds to repurchase 550,229 shares of its common stock from Tactic Pharma at a purchase price of $63.6098 per share, which equals the public offering price per share less underwriting discounts and commissions (the "Share Repurchase"). After giving effect to the Share Repurchase, the Company’s net proceeds from the Offering were approximately $91.9 million, before estimated offering expenses.

Financial Results for the Third Quarter Ended September 30, 2025, Compared to the Third Quarter Ended September 30, 2024

Cash and Net Loss

Cash, cash equivalents and investments as of September 30, 2025, were $143.7 million. Monopar expects that its current funds will be sufficient to continue operations at least through December 31, 2027, in order to: (1) assemble a regulatory package and file an NDA for the ALXN1840 investigational drug candidate for Wilson disease; (2) continue to conduct and conclude our first-in-human imaging and dosimetry clinical trial with MNPR-101-Zr, continue to conduct our first-in-human therapeutic clinical trial of MNPR-101-Lu, and advance our preclinical MNPR-101-Ac program into the clinic; and (3) invest in internal R&D projects to expand our radiopharmaceutical pipeline.

Net loss for the third quarter of 2025 was $3.4 million or $0.48 per share compared to net loss of $1.3 million or $0.37 per share for the third quarter of 2024.

Research and Development ("R&D") Expenses

R&D expenses for the third quarter of 2025 were $2,589,749, compared to $984,278 for the third quarter of 2024. This represents an increase of $1,605,471 attributed to (1) a $937,582 increase in manufacturing activities related to ALXN1840, (2) a $617,667 increase in R&D personnel expenses including stock-based compensation and (3) a net increase of $50,223 in other R&D expenses.

General and Administrative ("G&A") Expenses

G&A expenses for the third quarter of 2025 were $1,503,326, compared to $590,624 for the third quarter of 2024. This represents an increase of $912,702 primarily attributed to (1) a $369,959 increase in Board compensation resulting from the grant of stock options in March 2025 (no stock options were granted to the Board in 2024), (2) a $287,749 increase in G&A personnel expenses including stock-based compensation, and (3) a net increase of $254,993 in other G&A expenses.

Interest Income

Interest income for the third quarter of 2025 increased by $556,129 compared to the same period in 2024. The increase is attributed to interest earned on U.S. Treasury securities and higher bank balances in 2025, as a result of the approximately $91.9 million raised in the Offering after giving effect to the Share Repurchase, deducting underwriting discounts and commissions but before offering expenses.

(Press release, Monopar Therapeutics, NOV 13, 2025, View Source [SID1234659908])