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Clinical Trial News

Boehringer Ingelheim Partners with AnGes for HGF Gene Therapy Manufacturing Ahead of FDA Filing

FDA Approval
  • Boehringer Ingelheim has signed a contract development and manufacturing agreement with AnGes for the drug substance of their investigational hepatocyte growth factor gene therapy targeting peripheral arterial disease.
  • The partnership follows AnGes' completion of clinical trials and plans to submit a biologics license application to the FDA for their HGF gene therapy product.
  • Peripheral arterial disease affects 200 million people worldwide and can lead to severe complications including ulceration, infection, and limb amputation with mortality rates comparable to cancer.
  • The plasmid DNA molecule has been manufactured using proprietary microbial technology in E. coli at Boehringer's Vienna facility for nearly 20 years since the collaboration began.

Krystal Biotech Halts Melanoma Study of KB707 Following FDA Rejection of Similar Viral Immunotherapy

  • Krystal Biotech has discontinued its Phase I/II OPAL-1 study of KB707, a viral immunotherapy for melanoma, citing heightened regulatory uncertainty following the FDA's rejection of Replimune's similar therapy RP1.
  • Both KB707 and RP1 use modified herpes simplex virus type 1 vectors to trigger immune responses against tumors, but the FDA's controversial rejection of RP1 has complicated accelerated approval pathways for this drug class.
  • The company is now focusing on an inhaled version of KB707 for non-small cell lung cancer, which showed a 36% objective response rate in heavily pretreated patients and has secured an FDA End of Phase II meeting for October.
  • Over 20 melanoma experts have challenged the FDA's rejection of RP1 in an open letter, arguing that the agency's concerns about patient population heterogeneity actually better represent real-world treatment scenarios.

Molecular Partners Appoints Martin Steegmaier as Chief Scientific Officer to Advance DARPin Therapeutics Pipeline

Oncology
  • Molecular Partners has appointed Martin Steegmaier, Ph.D. as Chief Scientific Officer effective October 1, 2025, bringing extensive oncology drug development experience from senior roles at Roche, MorphoSys, Boehringer Ingelberg, and SOTIO Biotech.
  • Steegmaier will lead the research organization and advance the company's pipeline of targeted DARPin therapeutics for cancer patients, including Radio-DARPins and Switch-DARPins for logic-gated immune cell activation.
  • The appointment strengthens Molecular Partners' leadership team as the clinical-stage biotech company continues developing its proprietary DARPin platform for medical challenges that other drug modalities cannot readily address.
  • Steegmaier previously served as CSO at SOTIO Biotech and Head of Research at MorphoSys, with particular expertise in antibody-based therapeutics in immuno-oncology and hematology-oncology.

CVS Health Declines Coverage for Gilead's HIV Prevention Drug Yeztugo Despite 99.9% Efficacy

  • CVS Health, the largest U.S. pharmacy benefit manager, will not add Gilead's new HIV prevention drug Yeztugo to its commercial plans, citing clinical, financial, and regulatory factors despite the drug's proven 99.9% effectiveness.
  • The decision reflects concerns over Yeztugo's $28,000 annual list price, with CVS stating it's inappropriate for manufacturers to manipulate guidelines with clinically similar products priced far higher than existing options.
  • AIDS activists call the decision a "grave disappointment" and missed opportunity, as Yeztugo could be transformative in ending the HIV epidemic that infects 1.3 million people annually.
  • Gilead remains confident in securing 75% U.S. insurer coverage by year-end and 90% by June 2026, with government programs and several state Medicaid plans already providing coverage.

Trethera Secures $5.3 Million in NIH Funding to Advance First-in-Class dCK Inhibitor TRE-515 Across Cancer and Autoimmune Indications

  • Trethera Corporation received two separate NIH SBIR grants totaling $5.3 million to advance TRE-515, a first-in-class deoxycytidine kinase inhibitor, for treating metastatic castration-resistant prostate cancer and systemic lupus erythematosus.
  • The $2.3 million grant will evaluate TRE-515 combined with radiation therapy in prostate cancer models, targeting a disease that claims 70% of patients within 5 years of diagnosis.
  • The $3 million grant will support advanced studies for lupus treatment, building on preclinical data showing TRE-515 can stop disease progression in mouse models.
  • TRE-515 works by inhibiting the nucleoside salvage pathway, depriving abnormal cells of DNA building blocks while maintaining a favorable safety profile in ongoing clinical trials.

Ascentage Pharma Reports Strong Growth with 93% Increase in Olverembatinib Sales and Historic Lisaftoclax Approval

Drug Approval
  • Ascentage Pharma reported a 93% year-over-year increase in Olverembatinib sales to US$30.3 million for the first half of 2025, driven by expanded National Reimbursement Drug List coverage in China.
  • The company achieved a historic milestone with Lisaftoclax becoming the first Bcl-2 inhibitor to receive conditional approval for chronic lymphocytic leukemia/small lymphocytic lymphoma treatment in China on July 10, 2025.
  • Nine registrational Phase III clinical trials are currently ongoing, including three cleared by the FDA, demonstrating the company's robust pipeline advancement.
  • The company strengthened its financial position by completing a top-up placement in July 2025, raising US$190.1 million in net proceeds to support commercialization and development programs.

Xoma Royalty Acquires Mural Oncology for $35.2-38.8M in Latest Biotech Liquidation Deal

Oncology
  • Xoma Royalty Corp. agreed to acquire Mural Oncology for $2.035-2.240 per share, valuing the deal at $35.2-38.8 million depending on closing cash balance.
  • The acquisition follows Mural's discontinuation of lead IL-2 therapy nemvaleukin alfa after failed Artistry-6 and 7 trials, leading to 90% workforce reduction in April.
  • Xoma will wind down Mural's operations as part of a growing trend where investment firms acquire struggling biotechs trading below cash value for liquidation.
  • The deal represents Xoma's fourth oncology acquisition this year, following purchases of Turnstone Biologics, Lava Therapeutics, and HilleVax.

Steel Therapeutics Advances Fizurex Topical Wipe for Anal Fissures with $2M Funding and Pivotal Toxicology Study

  • Steel Therapeutics secured $2 million in strategic investment to advance development of Fizurex, a single-use topical wipe for treating anal fissures.
  • The company launched a pivotal toxicology study with Altasciences using minipigs to support an Investigational New Drug application planned for early 2026.
  • Fizurex aims to bridge the gap between compounded therapies and FDA-regulated products, offering more consistent treatment for a condition with limited therapeutic options.
  • The product leverages a commonly compounded combination therapy in a standardized format to improve consistency, safety, and patient access.

KROMATID Secures $8 Million Series C to Advance Genomic Analysis Platform for Gene and Cell Therapy

  • KROMATID successfully closed an $8 million Series C funding round led by BroadOak Capital Partners, surpassing its targeted capital goals with the most recent tranche contributing $3.8 million.
  • The funding will fuel expansion of KROMATID's proprietary platforms for detecting chromosomal structural rearrangements and scale operations to meet growing global demand in gene and cell therapy.
  • Over the next 12-18 months, the company plans to accelerate commercial expansion, enhance laboratory automation and throughput, and invest in strategic hiring across scientific and operational teams.
  • KROMATID's differentiated platform provides accurate, high-resolution analysis of genomic integrity, addressing one of the most pressing needs in the rapidly expanding gene and cell therapy industry.

Savara's Inhaled Molgramostim Shows 9.8% Lung Function Improvement in Phase 3 Autoimmune PAP Trial

Rare Disease
  • Savara's Phase 3 IMPALA-2 trial demonstrated that inhaled molgramostim significantly improved lung function by 9.8% at 24 weeks compared to 3.8% with placebo in autoimmune pulmonary alveolar proteinosis patients.
  • The treatment showed sustained benefits through 48 weeks with 11.6% improvement in gas transfer, while also enhancing quality of life and exercise capacity in the largest clinical trial conducted in this rare disease.
  • Results from the 43-site global study will be published in the New England Journal of Medicine, marking a potential breakthrough for patients with this chronic rare lung condition.

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