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Clinical Trial News

VASTHERA Receives FDA Clearance for Phase 1 Trial of Novel PAH Drug VTB-10

Orphan Drug
  • VASTHERA received FDA IND clearance on August 6, 2025, to initiate Phase 1 clinical trials for VTB-10, a first-in-class drug candidate targeting pulmonary arterial hypertension.
  • VTB-10 is developed using VASTHERA's proprietary Redoxizyme™ platform and functions as a small molecule enzyme that replicates peroxiredoxin function to address enzyme deficiency in PAH lesions.
  • Preclinical studies demonstrated VTB-10's ability to reverse abnormal vascular remodeling and restore functional endothelium, differentiating it from existing PAH therapies.
  • The drug candidate previously received FDA Orphan Drug Designation in November 2024 and is supported by funding from the Korea Drug Development Fund.

Teleflex Launches Barrigel Rectal Spacer in Japan Following PMDA Approval for Prostate Cancer Radiation Protection

  • Teleflex has launched Barrigel rectal spacer in Japan following regulatory approval from the Japanese Pharmaceuticals and Medical Devices Agency (PMDA), marking a key milestone in the company's global expansion strategy.
  • The hyaluronic acid-based device is the first and only sculptable non-animal stabilized hyaluronic acid rectal spacer proven to significantly reduce radiation reaching the rectum during prostate cancer treatment.
  • Clinical data shows 98 percent of treated men achieved at least a 25 percent reduction in rectal radiation exposure, with patients averaging an 85 percent reduction in rectal V54 Gy radiation.
  • The launch addresses a significant medical need in Japan, where prostate cancer was the most common cancer among men in 2022 with 104,318 new cases accounting for 18 percent of all cancer diagnoses nationwide.

CSL to Spin Off Seqirus Vaccine Unit and Launch $487M Share Buyback in Major Restructuring

  • Australian biotech giant CSL announced plans to separate its vaccine business Seqirus into a standalone company by June 2025, allowing the unit greater autonomy in the dynamic vaccines market.
  • The restructuring includes workforce cuts of up to 15% and is expected to generate $500-550 million in annual cost savings over three years, despite a one-time charge of $700-770 million.
  • CSL will launch a $750 million share buyback program as part of the strategic pivot, reflecting strong cash flow performance with operations generating $3.56 billion in fiscal 2025.
  • Seqirus contributed $2.2 billion to CSL's total revenue of $15.6 billion, with the vaccine unit facing competitive pressures in the U.S. influenza market despite overall 2% revenue growth.

jCyte Initiates Phase 2 Trial of Famzeretcel Cell Therapy for Retinitis Pigmentosa

Rare Disease
  • jCyte has dosed the first patients in the JC02-88 phase 2 trial evaluating famzeretcel, an allogeneic retinal progenitor cell therapy for retinitis pigmentosa.
  • The study tests a single injection of 8.8 million cells, representing a 50% dose increase from previous trials, with patients monitored for safety and vision changes over 6 months.
  • Retinitis pigmentosa affects approximately 2 million people worldwide and 100,000 in the United States, with patients typically becoming legally blind by middle age.
  • The therapy requires sufficient living cone photoreceptors to achieve visual function restoration through its neurotrophic mechanism.

BioMed X and Novo Nordisk Partner to Advance Oral Peptide Drug Delivery Technology

  • BioMed X and Novo Nordisk have launched a joint research collaboration to develop novel oral formulation technologies for therapeutic peptides, targeting site-specific retention in the lower small intestine.
  • The partnership aims to overcome critical challenges in conventional oral peptide formulations by enhancing bioavailability and enabling continuous drug release while maintaining gastrointestinal safety.
  • The initiative includes a global call for scientists to submit project proposals by October 12, 2025, to join the Heidelberg-based research team working on this breakthrough drug delivery platform.
  • This collaboration addresses one of the most significant challenges in modern pharmaceutical development, potentially transforming how peptide-based therapies are administered to patients.

ORIC Pharmaceuticals Appoints Kevin Brodbeck as Chief Technical Officer Ahead of Phase 3 Trials

Oncology
  • ORIC Pharmaceuticals has appointed Kevin Brodbeck, PhD, as Chief Technical Officer to support the company's transition into late-stage clinical development.
  • The appointment reflects ORIC's preparation for potential Phase 3 trials of ORIC-944 and enozertinib starting in 2026.
  • Brodbeck brings over 25 years of experience in technical operations, CMC, and regulatory activities across pharmaceutical development and commercialization.
  • ORIC's clinical pipeline includes ORIC-944 for prostate cancer and enozertinib for multiple genetically defined cancers targeting resistance mechanisms.

RemeGen and Santen Strike $1.3 Billion Licensing Deal for Dual-Target Ophthalmic Drug RC28-E

  • RemeGen has entered into an exclusive licensing agreement with Santen Pharmaceutical for RC28-E, a VEGF/FGF dual-target fusion protein drug for ocular neovascular diseases, with potential payments totaling up to RMB 1.295 billion ($180 million USD).
  • RC28-E demonstrated significant improvements in visual acuity and reduced central subfield thickness in Phase II trials for diabetic macular edema, with Phase III trials currently underway for both DME and wet age-related macular degeneration.
  • The agreement grants Santen exclusive rights to develop and commercialize RC28-E in Greater China and eight Asian countries, while RemeGen retains global rights outside these territories.
  • RemeGen plans to submit a Biologics License Application for the DME indication in China in the second half of 2025, followed by a wAMD application in mid-2026.

Molecular Testing and Targeted Therapies Transform Treatment Landscape for Pediatric Low-Grade Gliomas

Targeted TherapyPrecision Medicine
  • Comprehensive molecular testing using NGS panels and methylation profiling has become foundational for accurately diagnosing pediatric low-grade gliomas and identifying actionable mutations like BRAF alterations.
  • Targeted therapies including BRAF and MEK inhibitors are emerging as compelling alternatives to traditional chemotherapy, offering oral administration and better tolerability for children.
  • The treatment approach has evolved from cytotoxic chemotherapy to precision medicine strategies that balance tumor control with quality of life over the chronic, multi-year treatment course.
  • Molecular diagnostics typically return within 2-4 weeks for full results, with initial pathology and specific mutations like BRAF V600E detectable within one week through targeted immunohistochemistry stains.

Ozempic Faces $2 Billion in Lawsuits Over Severe Side Effects Including Stomach Paralysis and Vision Loss

  • Over 1,800 lawsuits have been filed against Novo Nordisk's Ozempic in US federal courts, with legal analysts estimating total liability could exceed $2 billion.
  • Patients report severe complications including gastroparesis (stomach paralysis), intestinal blockages, gallbladder disease, and sudden vision loss linked to NAION.
  • The litigation has been consolidated into multidistrict litigation in Pennsylvania, with bellwether trials expected to begin in early 2026.
  • A 2023 JAMA Internal Medicine study found increased gastroparesis risk among GLP-1 drug users, while FDA adverse event reports show thousands of gastrointestinal and vision complaints.

Invivyd's PEMGARDA Maintains Neutralizing Activity Against Dominant XFG COVID-19 Variant

Monoclonal Antibody
  • Invivyd announced that PEMGARDA (pemivibart) demonstrates continued neutralizing activity against the currently dominant XFG SARS-CoV-2 variant, maintaining efficacy as COVID-19 infections rise across 34 U.S. states.
  • The company's next-generation monoclonal antibody candidate VYD2311 showed similarly consistent and highly potent neutralization results against XFG and other circulating variants.
  • Both antibodies target structurally stable epitopes on the SARS-CoV-2 spike protein, with Invivyd estimating that every clinical variant since Omicron BA.2 has remained susceptible to pemivibart.
  • The neutralization data will be provided to the FDA imminently for potential inclusion in PEMGARDA's healthcare provider fact sheet, supporting continued use in immunocompromised patients.

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