On September 28, 2026 AstraZeneca reported that it has entered into an agreement to invest $2 billion in newly issued equity in Summit Therapeutics Inc. to accelerate the development of ivonescimab, a first-in-class bispecific antibody targeting PD-1 and VEGF, in combination with antibody drug conjugates (ADCs) across tumour types.

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Summit and AstraZeneca have also entered into a clinical collaboration agreement to evaluate sonesitatug vedotin (Sone-Ve) in combination with ivonescimab. Sone-Ve is a potential global first-in-class CLDN18.2-targeting ADC with a monomethyl auristatin E (MMAE) payload with several ongoing trials underway in gastrointestinal (GI) cancers. The companies intend to initiate trials in GI cancers under the collaboration imminently.

Under the terms of this agreement, each company will contribute their respective medicine for the planned combination trials and jointly contribute to trial costs. Each company will retain development and commercial rights to their respective medicines.

The two companies have also executed a Memorandum of Understanding (MOU) with the intent to enter into an agreement initiating a global development programme combining ivonescimab with AstraZeneca’s cancer medicines, including additional ADCs.

Susan Galbraith, Executive Vice President, Oncology Haematology R&D, AstraZeneca, said: "A core pillar of our oncology strategy is to broaden the reach of our ADC portfolio as the backbone of treatment across tumour types with combinations alongside next-generation immunotherapies. Bispecifics targeting PD-1 and VEGF are rapidly advancing in development and have the potential to improve on current immunotherapies, particularly in lung, breast and gastrointestinal cancers. This opportunity to combine ivonescimab with AstraZeneca’s ADC portfolio, including with Sone-Ve, could enable new regimens that raise the bar for patients with cancer across the treatment landscape."

Dr. Maky Zanganeh, President and Co-Chief Executive Officer of Summit Therapeutics, said: "The developments announced today with AstraZeneca open an exciting new chapter in the advancement of ivonescimab. With a growing body of evidence supporting ivonescimab’s differentiated PD-1 / VEGF bispecific approach, we look forward to further broadening the development plan of ivonescimab and initiating new clinical trials exploring the potential to combine ivonescimab with promising novel anti-cancer compounds, including ADCs, to bring together complementary approaches to tumour-cell killing, antitumour immunity, and the tumour microenvironment."

AstraZeneca recently reported positive high-level results from the CLARITY-Gastric01 trial for Sone-Ve in 2nd and later-line Claudin18.2-positive advanced gastric cancers demonstrating a statistically significant and highly clinically meaningful improvement in overall survival (OS) versus investigator’s choice of therapy. Results from this trial will be presented at the European Society for Medical Oncology Congress 2026 in a Presidential Symposium.

Ivonescimab is a novel, potential first-in-class bispecific antibody combining the effects of immunotherapy via a blockade of PD-1 with the anti-angiogenesis effects associated with blocking VEGF into a single molecule. Ivonescimab was engineered by Akeso Inc. Summit has exclusive rights to develop and commercialise ivonescimab in major territories outside China, while its partner Akeso Inc. retains rights in China and other regions.

Ivonescimab is approved to treat certain patients with non-small cell lung cancer (NSCLC) in China, and a Biologics License Application (BLA) seeking approval in EGFR-mutated NSCLC is under review by the US Food and Drug Administration (FDA). Ivonescimab is currently being studied in Phase III clinical trials in NSCLC, small cell lung cancer, BTC, bladder cancer, triple-negative breast cancer, head and neck squamous cell carcinoma, colorectal cancer, and pancreatic cancer.

Financial considerations
Summit Therapeutics will receive an equity investment of $2 billion from AstraZeneca. AstraZeneca will purchase an aggregate of approximately 109 thousand shares of preferred stock convertible into shares of common stock of Summit at a 1:1,000 ratio. Following completion of the investment, AstraZeneca will hold rights equivalent to approximately 12.0% of Summit Therapeutics outstanding common stock (approximately 10.6% on a fully diluted basis).

Closing of the investment in preferred stock is anticipated within one week. Conversion of AstraZeneca’s preferred stock to common stock in the future would be subject to customary regulatory clearances.

Notes

Sonesitatug vedotin (Sone-Ve)
Sone-Ve is a novel ADC targeting CLDN18.2, a protein found in the stomach lining and a validated therapeutic target in oncology, particularly for GI cancers. Sone-Ve consists of an anti-CLDN18.2 monoclonal antibody, a protease-degradable linker and a cytotoxic small molecule MMAE payload.

AstraZeneca entered into a global exclusive licence agreement with KYM Biosciences to develop and commercialise Sone-Ve in March 2023.

In addition to CLARITY-Gastric01, Sone-Ve is being evaluated in the CLARITY-Gastric02 Phase III trial in combination with capecitabine, with or without rilvegostomig, as a 1st-line treatment for advanced or metastatic gastric cancer, GEJ cancer and EAC. In Phase II development, Sone-Ve is being evaluated in patients with advanced solid tumours in multiple combinations across settings, including in CLDN18.2-positive pancreatic and BTC.

(Press release, AstraZeneca, SEP 28, 2026, View Source [SID1234671118])

On September 28, 2026 Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with RAS/MAPK pathway-driven cancers, reported that Gynecologic Oncology, the official journal of the Society of Gynecologic Oncology (SGO), has published two-year long-term results from the Phase 2 RAMP 201 study evaluating AVMAPKI FAKZYNJA CO-PACK (avutometinib capsules; defactinib tablets) in women with recurrent low-grade serous ovarian cancer (LGSOC). The manuscript, titled "Long-Term Efficacy and Safety of Avutometinib + Defactinib in Recurrent Low-Grade Serous Ovarian Cancer: Results of a 2-Year Follow Up of ENGOT-OV60/GOG-3052/RAMP 201​" has been published online and will appear in the October 2026 print issue of Gynecologic Oncology.

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"Data published in this analysis of RAMP 201 accounts for more than two years of follow-up, further reinforcing the clinically meaningful and durable activity observed with the combination of avutometinib and defactinib in recurrent low-grade serous ovarian cancer," said Professor Susana Banerjee, M.B.B.S., M.A., Ph.D., F.R.C.P, Consultant Medical Oncologist at The Royal Marsden NHS Foundation Trust and Team Leader in Women’s Cancers at The Institute of Cancer Research, London and Global Lead Principal Investigator of ENGOTov60/GOG3052/NCRI/RAMP201. "These results strengthen our confidence in the consistency of response and disease control seen across patients and highlight the important role this combination treatment should play for this rare ovarian cancer."

"Low-grade serous ovarian cancer is a rare and distinct form of ovarian cancer with a unique biology and clinical behavior different from other ovarian cancers. Patients with this disease often experience delays in diagnosis and have historically had limited treatment options," said John Hayslip, M.D., chief medical officer at Verastem Oncology. "Data from this longer-term analysis, also presented during a scientific plenary session at the Society of Gynecologic Oncology 2026 Annual Meeting on Women’s Cancers, further underscore the durable responses, safety, and tolerability of avutometinib in combination with defactinib across patients with KRAS wild-type or KRAS-mutated LGSOC."

In the updated analysis, with ongoing patients presenting a median follow-up of 24.9 months, efficacy measures, including median duration of response (mDoR) and median progression free survival (mPFS), and safety, are consistent with the primary analysis, which was conducted more than 13 months prior:

Long-Term Median Follow-Up
(data cutoff of August 12, 2025)
(N=17)

Primary Analysis*
(data cutoff of June 30, 2024)
(N=109)

Total
(N=17)

KRAS mt
(N=12)

KRAS wt
(N=5)

Total
(N=109)

KRAS mt
(N=57)

KRAS wt
(N=52)

mDoR

31.1
months

31.1
months

12
months

31.1
months

31.1
months

9.2
months

mPFS

12.9
months

19.6
months

12.7
months

12.9
months

22
months

12.8
months

*The primary analysis was published in the Journal of Clinical Oncology (Banerjee et al, July 11, 2025).

Fifty percent of patients with KRAS-mutated and 30% with KRAS wild-type LGSOC remained on therapy for more than one year. Importantly, the median DoR for patients with KRAS wild-type LGSOC has increased from 9.2 to 12 months. Adverse events (AEs) were consistent with the primary analysis with no new safety signals observed, and a 12% discontinuation rate due to AEs.

About RAMP 201

RAMP 201 (ENGOTov60/GOG3052/NCRI) (NCT04625270) was an adaptive, two-part multicenter, parallel cohort, randomized, open-label Phase 2 registration-directed trial evaluating the efficacy and safety of avutometinib alone and in combination with defactinib in patients with recurrent low-grade serous ovarian cancer (LGSOC). The first part of the trial (Part A) determined the selection of the go-forward regimen, which was the combination of avutometinib and defactinib versus avutometinib alone, based on overall response rates. The expansion phases of the trial (Parts B and C) evaluated the safety and efficacy of the go-forward regimen of avutometinib 3.2 mg twice weekly and defactinib 200 mg twice daily. The Part D portion of the trial evaluated a low dose of the combination to inform individualized dose reduction.

About Low-Grade Serous Ovarian Cancer (LGSOC)

LGSOC is a rare ovarian cancer that is insidious and persistent. LGSOC is distinct and different from high-grade serous ovarian cancer (HGSOC) and requires different treatment. LGSOC is highly recurrent and less sensitive to chemotherapy compared to HGSOC. Approximately 6,000-8,000 women in the U.S. and 80,000 worldwide are living with this disease. LGSOC affects younger women with bimodal peaks of diagnosis at ages between 20-30 and 50-60 and has a median survival of approximately ten years. Approximately 70 percent of LGSOC shows RAS pathway-associated mutations, and 30 percent of people with LGSOC have a KRAS mutation. The majority of patients report a negative impact of LGSOC on their mental and physical health, fertility, and long-term quality of life.

About AVMAPKI and FAKZYNJA Combination Therapy

AVMAPKI (avutometinib) inhibits MEK kinase activity while also blocking the compensatory reactivation of MEK by upstream RAF. RAF and MEK proteins are regulators of the RAS/RAF/MEK/ERK (MAPK) pathway. Blocking RAF and/or MEK activates FAK, a key mediator of drug resistance. FAKZYNJA (defactinib) is a FAK inhibitor and together, the avutometinib and defactinib combination was designed to provide a more complete blockade of the signaling that drives the growth and drug resistance of RAS/MAPK pathway-dependent tumors.

The U.S. Food and Drug Administration (FDA) approved AVMAPKI FAKZYNJA CO-PACK (avutometinib capsules; defactinib tablets) for the treatment of adult patients with KRAS-mutated recurrent LGSOC who have received prior systemic therapy on May 8, 2025. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. Verastem is conducting RAMP 301 (GOG-3097/ENGOT-ov81/GTG-UK) (NCT06072781), an international Phase 3 confirmatory trial evaluating the combination of avutometinib and defactinib versus standard chemotherapy or hormonal therapy for the treatment of recurrent low-grade serous ovarian cancer (LGSOC) with and without a KRAS mutation. Verastem is also evaluating avutometinib plus defactinib with standard-of-care chemotherapy as a potential treatment in the first-line for patients with advanced pancreatic cancer (RAMP 205; NCT05669482). Avutometinib and defactinib are not approved by the FDA or any other regulatory authority, either in combination or with other therapies, for any of these investigative uses. Neither avutometinib nor defactinib are approved by the FDA or any other regulatory authority on a stand-alone basis for any use.

AVMAPKI FAKZYNJA CO-PACK U.S. Indication

Indication

AVMAPKI FAKZYNJA CO-PACK is indicated for the treatment of adult patients with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC) who have received prior systemic therapy. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

Important Safety Information

Warnings and Precautions

Ocular Toxicities: Ocular toxicities, including visual impairment and vitreoretinal disorders, occurred. Perform comprehensive ophthalmic evaluation at baseline, prior to cycle 2, every three cycles thereafter, and as clinically indicated. Withhold AVMAPKI FAKZYNJA CO-PACK for ocular toxicities until improvement at the same or reduced dose. Permanently discontinue AVMAPKI FAKZYNJA CO-PACK for any grade 4 toxicity.
Serious Skin Toxicities: Skin toxicities, including photosensitivity and severe cutaneous adverse reactions (SCARSs) occurred. Adhere to concomitant medications. Monitor for skin toxicities and interrupt, reduce or permanently discontinue AVMAPKI FAKZYNJA CO-PACK based on severity, tolerability and duration.
Hepatotoxicity: Monitor liver function tests prior to each cycle, on day 15 of the first 4 cycles, and as clinically indicated. Withhold, reduce or discontinue AVMAPKI FAKZYNJA CO-PACK based on severity and persistence of abnormality.
Rhabdomyolysis: Monitor creatine phosphokinase prior to the start of each cycle, on day 15 of the first four cycles, and as clinically indicated. If increased CPK occurs, evaluate patients for rhabdomyolysis or other causes. Withhold, reduce or permanently discontinue AVMAPKI FAKZYNJA CO-PACK based on severity and duration of the adverse reaction.
Embryo-Fetal Toxicity: AVMAPKI FAKZYNJA CO-PACK can cause fetal harm. Advise patients of the potential risk to a fetus and to use effective contraception.
Adverse Reactions

The most common (≥ 25%) adverse reactions, including laboratory abnormalities, were increased creatine phosphokinase, nausea, fatigue, increased aspartate aminotransferase, rash, diarrhea, musculoskeletal pain, edema, decreased hemoglobin, increased alanine aminotransferase, vomiting, increased blood bilirubin, increased triglycerides, decreased lymphocyte count, abdominal pain, dyspepsia, dermatitis acneiform, vitreoretinal disorders, increased alkaline phosphatase, stomatitis, pruritus, visual impairment, decreased platelet count, constipation, dry skin, dyspnea, cough, urinary tract infection, and decreased neutrophil count.

Drug Interactions

Strong and moderate CYP3A4 inhibitors: Avoid concomitant use with AVMAPKI FAKZYNJA CO-PACK.
Strong and moderate CYP3A4 inducers: Avoid concomitant use with AVMAPKI FAKZYNJA CO-PACK.
Warfarin: Avoid concomitant use of AVMAPKI FAKZYNJA CO-PACK with warfarin and use an alternative to warfarin.
Gastric acid reducing agents: Avoid concomitant use of AVMAPKI FAKZYNJA CO-PACK with proton pump inhibitors (PPIs) or H2 receptor antagonists. If use of an acid-reducing agent cannot be avoided, administer FAKZYNJA 2 hours before or 2 hours after the administration of a locally acting antacid.
Use in Specific Populations

Lactation: Advise not to breastfeed.
Fertility: May impair fertility in males and females.

(Press release, Verastem, SEP 28, 2026, View Source [SID1234671117])

On September 28, 2026 TransCode Therapeutics, Inc. (NASDAQ: RNAZ), a clinical stage company pioneering immuno-oncology and RNA-based therapeutics for the treatment of high risk and advanced cancers, reported updated clinical data from its Phase 1a clinical trial of TTX-MC138, its novel anti-miR-10b therapeutic candidate, in patients with relapsed or refractory, unresectable locally advanced, or metastatic solid tumors.

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At the data cutoff date of September 1, 2026, 10 of 14 efficacy-evaluable patients, or 71.4%, achieved stable disease as their best overall response by investigator assessment. In the context of a first-in-human, dose-escalation study enrolling patients with advanced, heavily pretreated cancers,

the Company believes the consistency, breadth, and durability of disease control observed thus far are promising and warrant continued clinical evaluation.

The disease control rate was 57.1% (95% confidence interval, 28.9% to 82.3%) at Cycle 3 Day 1 and 35.7% (95% confidence interval, 12.8% to 64.9%) at Cycle 6 Day 1.

The durability of responses was also notable. Six of 14 efficacy-evaluable patients were progression-free at three months, three of 14 at six months, and three of 14 at nine and 12 months. Together, these findings show that the efficacy signal was not limited to a single assessment and included prolonged disease control in a subset of patients.

As of the September 1, 2026, a total 98 doses have been administered. In this pre-specified data snapshot, TTX-MC138 demonstrated an acceptable tolerability profile in subjects with advanced solid tumors. No dose-limiting toxicities were reported, no treatment-emergent adverse events led to treatment discontinuation, including the highest evaluated dose level of 4.8 mg/kg. Of note, administration of TTX-MC138 for over a year was well tolerated and not associated with any dose-limiting toxicities.

"The updated efficacy findings are highly positive and clinically meaningful for an early dose-escalation study in a heavily pretreated, heterogeneous advanced solid-tumor population," said Daniel Vlock, M.D., Medical Consultant for TransCode Therapeutics. "A 71% stable disease rate, disease control extending through six months, and three patients remaining progression-free at twelve months collectively demonstrate a compelling and durable signal of antitumor activity. Importantly, these efficacy findings were observed alongside continued tolerability and no reported dose-limiting toxicities, supporting further evaluation of TTX-MC138."

Post-hoc Growth Modulation Index (GMI) Analysis Further Supports Clinical Benefit

The Growth Modulation Index (GMI) was calculated to further document the activity of TTX-MC138. GMI compares the progression-free survival achieved on study treatment to that achieved on the patient’s most recent prior systemic anticancer therapy. A GMI greater than 1.3 is generally considered evidence of meaningful clinical benefit. In this analysis, 6 of 15 evaluable patients (40%) achieved a GMI greater than 1.3, indicating that treatment with TTX-MC138 resulted in at least a 30% longer progression-free interval than that achieved with the patient’s immediately preceding therapy. Notably, several patients demonstrated substantially greater extensions of disease control relative to prior treatment, providing additional evidence of antitumor activity in this heavily pretreated population.

Exploratory Biomarker Findings Support Biological Activity

Exploratory RNA-sequencing analysis of peripheral blood identified treatment-associated, target-specific immune effects without major dysregulation of the global immune landscape. The analysis showed encouraging on-treatment increases in an antitumor cytokine signature and dose-dependent immune effects, including cytotoxic effector activity and increased monocyte and M1-associated inflammatory activity. Patients with stable disease showed higher B-cell and monocyte fractions and enrichment of cytotoxic, phagocytic, and Type I interferon programs compared with patients with progressive disease. These exploratory findings provide orthogonal biological support for the clinical efficacy observations and are consistent with TTX-MC138 engaging antitumor immune pathways. Given the small sample size, the biomarker findings are hypothesis-generating and require confirmation in larger studies. "The alignment between the highly positive clinical disease-control findings and the exploratory transcriptomic evidence is particularly encouraging," said Zdravka Medarova, Ph.D., Co-founder and Chief Scientific Officer of TransCode Therapeutics. "The data show treatment-associated activation of multiple arms of antitumor immunity and biological features that distinguish patients with stable disease from those with progressive disease. This convergence of clinical and biomarker evidence strengthens the mechanistic rationale for targeting miR-10b and supports advancement of TTX-MC138."

"These updated findings substantially strengthen our confidence in TTX-MC138," said Philippe P. Calais, Pharm.D., Ph.D., Chairman and Chief Executive Officer of TransCode Therapeutics. "The breadth and durability of disease control, together with an acceptable tolerability profile and supportive evidence of biological activity, provide a strong foundation for the next stage of development. We are evaluating dose-expansion strategies designed to confirm activity in selected cancers and molecularly defined patient populations."

TransCode expects to incorporate the updated clinical findings into a clinical study report addendum and to continue evaluating safety, pharmacokinetics, pharmacodynamics, and antitumor activity.

(Press release, TransCode Therapeutics, SEP 28, 2026, View Source [SID1234671115])

On September 28, 2026 Oncolytics Biotech Inc. (Nasdaq: ONCY) ("Oncolytics" or the "Company"), a clinical-stage immunotherapy company developing pelareorep, reported continued clinical and operational progress in REO 033, its randomized study evaluating pelareorep in second-line RAS-mutant, microsatellite-stable ("MSS") metastatic colorectal cancer ("mCRC").

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Nearly all planned clinical sites for Part A of REO 033 have now been activated, and multiple patients are currently enrolled (link to study on ClinicalTrials.gov). With the site activation process largely complete, the Company is focused on accelerating enrollment and generating randomized clinical data evaluating the contribution of pelareorep to the current standard-of-care regimen.

REO 033 is a randomized controlled study evaluating pelareorep in combination with folinic acid, fluorouracil and irinotecan ("FOLFIRI") and bevacizumab versus FOLFIRI and bevacizumab in patients with second-line RAS-mutant MSS mCRC. Part A is expected to enroll approximately 60 patients randomized between the two treatment arms, with objective response rate ("ORR") as the primary endpoint, and progression-free survival ("PFS"), overall survival ("OS"), safety, and biomarker analyses among the additional endpoints.

"We are seeing strong momentum in REO 033 now that substantially all of our planned sites are open and multiple patients are on study," said John McAdory, Chief Operating Officer of Oncolytics. "Given the current pace of enrollment, we expect to have the ability to report interim data from Part A by year-end, based on the number of evaluable patients enrolled by the end of October. Importantly, with site activation substantially behind us, our focus is now squarely on enrollment, execution, and generating the randomized clinical data that can inform the next stage of the program."

REO 033 Builds on Encouraging REO 022 Clinical Data

REO 033 was designed to prospectively evaluate the efficacy signals previously observed in REO 022 in a randomized setting. In REO 022, pelareorep in combination with FOLFIRI and bevacizumab demonstrated encouraging ORR, PFS, and OS compared with historical second-line benchmarks:

Efficacy Measure
REO 022: Pelareorep + FOLFIRI + Bevacizumab1
Historical Second-Line Benchmark
Objective Response Rate 33%
~6–11%2, 3
Median Progression-Free Survival 16.6 months
~5.7 months2

Median Overall Survival 27.0 months
~11.2 months2
Median Duration of Response 19.5 months
~4–6 months4

The REO 022 results were generated in a small, non-randomized study, and cross-study comparisons have inherent limitations. REO 033 is designed to test the pelareorep regimen prospectively against a concurrent control arm and determine whether the efficacy signals observed in REO 022 can be replicated in a randomized study.

Potential Registration Path Aligned with the Food and Drug Administration

The Company recently aligned with the U.S. Food and Drug Administration (the "FDA") on the concept for a potential pivotal Part B expansion of REO 033.

Under the proposed regulatory strategy, Part B would build directly upon the ongoing randomized REO 033 study and could support a potential accelerated approval submission based on objective response rate, with progression-free survival providing the basis for potential full approval.

The Company expects data from Part A to inform the final size and execution of the potential pivotal Part B expansion.

About Pelareorep
Pelareorep is an intravenously delivered, systemically active, investigational immunotherapy with a dual mechanism of action that selectively replicates in tumor cells while activating both innate and adaptive anti-tumor immune responses, including the upregulation of key inflammatory cytokines resulting in the formation of tertiary lymphoid structures and the expansion of tumor-infiltrating lymphocytes. It has been administered to over 1,200 patients, and clinical studies have demonstrated pelareorep’s potential to enhance the activity of checkpoint inhibitors and other anti-cancer therapies across multiple solid tumor types.

(Press release, Oncolytics Biotech, SEP 28, 2026, View Source [SID1234671114])

On September 28, 2026 Moleculin Biotech, Inc. (Nasdaq: MBRX) ("Moleculin" or the "Company") reported the publication of a peer-reviewed analysis of the cardiac safety of Annamycin (also known as L-Annamycin or naxtarubicin) in Frontiers in Cardiovascular Medicine. The publication, titled "Cardiac safety of L-Annamycin: a pooled analysis of five clinical trials," reports cardiac safety findings from 90 patients treated across five clinical trials in acute myeloid leukemia (AML) and metastatic soft tissue sarcoma (STS).

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The analysis was independently evaluated by a cardio-oncology laboratory at Cleveland Clinic and found no clinical or subclinical evidence of treatment-related cardiotoxicity, including at cumulative Annamycin exposures exceeding traditional lifetime limits for conventional anthracyclines.

"Publication of this comprehensive pooled analysis in a peer-reviewed cardiovascular medicine journal provides additional validation of the cardiac safety profile we have observed with Annamycin across our clinical development program," said Walter Klemp, Chairman and Chief Executive Officer of Moleculin. "For decades, the clinical utility of anthracyclines has been limited by cumulative cardiotoxicity and associated lifetime dose limits. The absence of detectable cardiac toxicity in patients receiving cumulative doses of Annamycin beyond those traditional thresholds supports our continued development of Annamycin and its potential to address an important limitation of the anthracycline class."

Pooled Analysis Evaluates Cardiac Safety Across Five Clinical Trials

The publication evaluated cardiac safety data from five sponsor- and investigator-initiated clinical trials, including three trials in AML and two trials in metastatic STS. Across the five studies, 90 patients received Annamycin, with paired echocardiographic data available for 78 patients.

Key findings from the pooled analysis include:

No significant change in cardiac function: Mean LVEF was 60.6% at baseline compared with 60.0% following treatment, with no statistically significant difference (p=0.84).
No relationship between cumulative dose and change in LVEF: The analysis found no association between cumulative Annamycin exposure and changes in LVEF.
No relationship between age and change in LVEF: Patient age was not associated with changes in LVEF.
No treatment-related cardiotoxicity identified: Serial ECGs, cardiac biomarkers including troponin I/T, and global longitudinal strain assessments showed no evidence of treatment-related cardiotoxicity.
High cumulative exposure: Patients received a median cumulative Annamycin dose of 660 mg/m², with findings observed despite cumulative exposures exceeding conventional anthracycline lifetime limits.
The authors concluded that Annamycin was not associated with clinical or subclinical evidence of cardiotoxicity at cumulative doses exceeding traditional anthracycline thresholds and that the findings support continued clinical evaluation of Annamycin as a potentially safer anthracycline platform.

Anthracyclines are among the most widely used and effective classes of cancer medicines, but their use can be limited by cumulative, dose-dependent cardiotoxicity. These cardiac risks can restrict treatment intensity, limit retreatment options and prevent some patients from receiving additional anthracycline therapy.

Annamycin is a fundamentally re-engineered anthracycline designed to maintain the antitumor activity of the anthracycline class while addressing the cardiotoxicity associated with conventional agents. Its liposomal formulation was developed to optimize tissue distribution and reduce cardiac exposure.

The newly published article builds upon previously presented clinical data demonstrating a lack of detectable cardiotoxicity with Annamycin despite cumulative exposure levels substantially exceeding conventional anthracycline limits. Moleculin previously reported that the pooled analysis included 90 patients across five completed clinical trials, with independent cardiac review conducted through the Cleveland Clinic Division of Cardiovascular Medicine.

Annamycin is currently being evaluated in Moleculin’s MIRACLE clinical development program for patients with relapsed or refractory AML.

(Press release, Moleculin, SEP 28, 2026, View Source [SID1234671113])