Takeda’s MIMRYLO Wins U.S. FDA Approval for Polycythemia Vera; Lilly’s Mounjaro Gains FDA Approval for Cardiovascular Risk Reduction in Type 2 Diabetes; Teva Expands Neuroscience Portfolio Through Proposed Acquisition; BioNTech Ends Phase 2 Study of Autogene Cevumeran in Colorectal Cancer; AusperBio Secures $120 Million Series C to Advance AHB-137 and Next-Generation Hepatitis B Therapies

Takeda Gains FDA Approval for MIMRYLO

Takeda announced that the U.S. Food and Drug Administration (FDA) has approved the New Drug Application (NDA) for MIMRYLO™ (rusfertide) to treat erythrocytosis in adults with polycythemia vera (PV), a type of blood cancer. MIMRYLO is a first-in-class hepcidin mimetic developed to regulate iron distribution and excessive red blood cell production, thereby helping maintain hematocrit levels. Hematocrit refers to the proportion of red blood cells relative to the total volume of blood. Keeping hematocrit below 45% is a key treatment objective for patients with PV.1

According to Andrew T. Kuykendall, M.D., lead investigator of the VERIFY study and Associate Member in the Department of Hematology at Moffitt Cancer Center, poorly controlled hematocrit levels can have serious implications for people with PV, including an increased risk of potentially life-threatening thrombotic events. He noted that existing approaches such as phlebotomy can be challenging for many patients and may interfere with their daily routines. The approval of MIMRYLO provides physicians and patients with a novel first-in-class treatment specifically targeting erythrocytosis, a major driver of excessive red blood cell production in PV. He added that the consistent results from the VERIFY study support the potential of MIMRYLO to improve routine PV management and help patients maintain hematocrit control.

The FDA approval was based on findings from the global, randomized Phase 3 VERIFY trial (NCT05210790), which enrolled 293 patients with PV. MIMRYLO achieved all prespecified efficacy endpoints and showed a favorable safety profile. Patients treated with MIMRYLO in combination with standard of care achieved higher response rates than those receiving placebo plus standard of care. Benefits included improved hematocrit control, reduced dependence on phlebotomy, and improvements in fatigue, as assessed using the PROMIS Fatigue Short Form 8a.

MIMRYLO was generally well tolerated through 52 weeks of treatment in the VERIFY study. The most frequently reported treatment-emergent adverse events among patients receiving MIMRYLO were injection-site reactions and anemia.

Lilly’s Mounjaro Wins FDA Approval for Reducing Major Cardiovascular Events in Type 2 Diabetes

Eli Lilly and Company announced that the U.S. Food and Drug Administration (FDA) has approved Mounjaro (tirzepatide), a dual GIP and GLP-1 receptor agonist, for reducing the risk of major adverse cardiovascular events (MACE), including cardiovascular death, non-fatal myocardial infarction, and non-fatal stroke, in adults with type 2 diabetes who are at elevated cardiovascular risk. Mounjaro is also approved, alongside diet and exercise, to help improve blood glucose levels in adults and children aged 10 years and older with type 2 diabetes.

Kenneth Custer, Ph.D., executive vice president and president of Lilly Cardiometabolic Health, stated that cardiovascular disease remains the leading cause of death among people with type 2 diabetes. He noted that Mounjaro, described by Lilly as the most-prescribed branded type 2 diabetes medication for U.S. adults, now provides an established option for reducing cardiovascular risk while also offering benefits in A1C control and weight management. He added that Lilly raised the evidentiary standard by evaluating Mounjaro directly against a GLP-1 medicine with demonstrated cardiovascular benefits, reflecting the company’s expanding body of clinical evidence.

The FDA’s decision was supported by findings from SURPASS-CVOT, the first cardiovascular outcomes study to directly compare two incretin-based therapies rather than evaluating treatment against placebo. The trial also represents the largest and longest study of tirzepatide conducted to date, involving more than 13,000 participants from 30 countries over a period exceeding four and a half years. Mounjaro achieved non-inferiority compared with Trulicity (dulaglutide), a GLP-1 therapy with established cardiovascular benefits, and was associated with an 8% lower incidence of cardiovascular death, heart attack, or stroke (MACE-3). The estimated hazard ratio for the time to first MACE was 0.92 (95.3% CI: 0.83–1.01) for Mounjaro versus Trulicity.

David A. D’Alessio, M.D., co-author of the study and director of the Division of Endocrinology and Metabolism at Duke University School of Medicine, emphasized that cardiovascular health should remain a treatment priority throughout the management of type 2 diabetes, rather than becoming a focus only after a major cardiovascular event. He noted that although glucose management remains central to diabetes care, addressing cardiovascular risk is equally important and may sometimes receive less attention. According to D’Alessio, the approval provides patients with a treatment option that can reduce cardiovascular event risk while simultaneously supporting metabolic health.

The safety and tolerability profile of Mounjaro in SURPASS-CVOT was broadly consistent with its previously established safety profile. Gastrointestinal adverse events were the most frequently reported side effects and were generally mild to moderate in severity, occurring mainly during the dose-escalation phase.

Teva Announces Proposed Acquisition of Novel Neuroscience Asset to Advance Its Pivot to Growth Strategy

Teva Pharmaceuticals International GmbH, a subsidiary of Teva Pharmaceutical Industries Ltd., has submitted a bid to acquire an innovative neuroscience asset from BioXcel Therapeutics, Inc. as part of a court-supervised sale process. The asset, a novel sublingual dexmedetomidine film, is currently under review by the U.S. Food and Drug Administration (FDA) for potential at-home use in the acute management of agitation associated with schizophrenia or bipolar I or II disorder in adults. If approved, the therapy could represent the first FDA-approved at-home treatment for agitation related to these conditions. Completion of the transaction would further strengthen Teva’s psychiatry portfolio and, subject to regulatory approval, could provide an important treatment option for individuals with complex mental health disorders where significant unmet needs remain.

Evan Lippman, Executive Vice President of Business Development at Teva, stated that business development is an important component of the company’s Pivot to Growth strategy. He noted that the proposed acquisition would enhance Teva’s neuroscience portfolio and align with its strategy of selectively pursuing innovative assets that offer strong strategic relevance, meaningful potential patient benefits, and long-term growth opportunities while maintaining a balanced approach to risk and value creation.

Agitation episodes among individuals with schizophrenia or bipolar I or II disorder can occur unpredictably and may be highly disruptive and distressing for patients, their families, and caregivers. Severe episodes can sometimes require intensified medical intervention, including emergency department care or hospitalization. Despite the considerable impact of these episodes, treatment options specifically intended for the acute management of agitation remain limited, especially therapies that could potentially be administered outside traditional healthcare or hospital environments.

BioNTech Announces Termination of Autogene Cevumeran Phase 2 Trial in Colorectal Cancer

BioNTech SE announced its decision to discontinue the Phase 2 BNT122-01 clinical trial (NCT04486378), which was investigating autogene cevumeran (BNT122/RO7198457), an experimental individualized mRNA-based cancer immunotherapy used as adjuvant monotherapy in patients with circulating tumor DNA (ctDNA)-positive, surgically resected Stage II (high-risk) or Stage III colorectal cancer (CRC). The therapy is being co-developed by BioNTech and Genentech, a member of the Roche Group. The decision to end the study was reached in consultation with Genentech.

The decision comes after a new assessment by the trial’s independent Data Safety Monitoring Board (DSMB), which oversees patient safety and the scientific integrity of the study. In October 2025, the trial crossed its predefined futility boundary. At that point, the DSMB determined that the available data were not mature enough to draw dependable conclusions about efficacy and that the follow-up period was inadequate to assess the primary endpoint. Because there were no safety concerns and the DSMB did not recommend stopping the study, BioNTech continued the trial according to the established protocol.

During its latest review, the DSMB observed a numerical difference in overall survival between the treatment groups within the specific patient population being studied. Based on the available evidence, the board concluded that continuing the trial was unlikely to alter the efficacy findings and recommended stopping treatment and terminating the study. The review did not identify any new safety concerns associated with autogene cevumeran. BioNTech has subsequently notified the clinical investigators and applicable regulatory authorities of the decision.

The BNT122-01 study was designed to determine whether individualized mRNA cancer immunotherapy administered as a standalone adjuvant treatment, without the addition of a checkpoint inhibitor, could reduce the risk of disease recurrence in high-risk CRC patients compared with watchful waiting, which represents the current standard of care. Colorectal cancer is a biologically heterogeneous and immunologically “cold” tumor that has historically demonstrated limited responsiveness to immunotherapies. The disease also carries a substantial risk of metastatic recurrence, highlighting the significant unmet need for new treatment approaches.

Prof. Özlem Türeci, M.D., Co-Founder and Chief Medical Officer of BioNTech, said that decisions involving clinical trials are made carefully, with patient well-being remaining the company’s primary consideration. Although the outcome was not consistent with the company’s expectations for mRNA monotherapy in colorectal cancer, she noted that the findings offer valuable scientific information about the difficulties of treating tumors that are relatively insensitive to immunotherapy and characterized by immunosuppressive tumor microenvironments. According to Türeci, these insights may support the future development of investigational mRNA-based cancer treatments. BioNTech remains committed to mRNA technology as a central component of its oncology strategy, including the development of novel combination approaches. The company also expressed its appreciation to the patients, families, investigators, and clinical site teams who contributed to the study.

In line with standard clinical development procedures, BioNTech plans to conduct a comprehensive evaluation of the trial data to better understand patient selection and identify implications for the clinical development of other potential mRNA cancer immunotherapies. The company intends to communicate the study findings to the broader scientific and medical community at an appropriate time.

The discontinuation of BNT122-01 does not affect the Phase 2 IMcode003 trial (NCT05968326), which is evaluating autogene cevumeran in combination with checkpoint inhibition and chemotherapy as an adjuvant treatment for pancreatic ductal adenocarcinoma (PDAC). That study is continuing according to plan.

AusperBio Raises $120 Million Series C to Propel AHB-137 Through Phase 3 and Expand HBV Therapeutics

AusperBio Therapeutics, Inc. and Ausper Biopharma Co., Ltd., a near-commercial-stage biopharmaceutical company developing targeted oligonucleotide therapies for chronic hepatitis B (CHB) and other diseases, announced the successful completion of a $120 million Series C financing round.

The financing was led by a prominent strategic investor and included participation from new investor RA Capital Management, L.P. (“RA Capital”), as well as continued backing from existing investors, including HanKang Capital, Sherpa Capital, InnoPinnacle Fund, Qiming Venture Partners, YuanBio Venture Capital, and CDH Investments. Following the Series C round, AusperBio has secured a total of $360 million in funding since 2024, underscoring sustained investor confidence in the Company’s lead development programs and differentiated oligonucleotide technology platforms.

The proceeds will primarily fund the ongoing development of AHB-137, AusperBio’s lead investigational antisense oligonucleotide (ASO) therapy for CHB, including its Phase 3 registrational program and preparations for potential commercialization. The funding will also support the accelerated development of AHB-171, an investigational HBV siRNA candidate based on the Company’s proprietary Au-HALO™ targeted delivery platform. In addition, AusperBio plans to advance next-generation combination therapies for CHB and broaden its pipeline of targeted oligonucleotide therapeutics aimed at addressing significant unmet medical needs.

Dr. Guofeng Cheng, co-founder and CEO of AusperBio, said the financing marks a significant milestone for the Company as it works to advance AHB-137 toward potential commercialization while expanding its next-generation CHB treatment portfolio. He expressed appreciation for the continued support from existing investors and welcomed RA Capital as a new investor. The Company intends to leverage this momentum to pursue its strategy of achieving a functional cure for CHB and capitalize on additional opportunities across its oligonucleotide technology platforms.