Clinical Trial News

FDA approved Cosentyx® (secukinumab) for treating moderate to severe hidradenitis suppurativa (HS) in adults, based on Phase III trials showing symptom relief as early as Week 2. Cosentyx, the only IL-17A inhibitor for HS, demonstrated significant reductions in inflammatory nodules and abscesses, offering a new treatment option for this chronic, painful disease affecting 1 in 100 people globally.

Sarepta Therapeutics Inc. reported its $3.2M gene therapy for Duchenne muscular dystrophy didn't clearly slow the disease in a trial with 125 children, leading to a 37% stock drop and a $3.76B market value loss.

4SC AG received EMA's Orphan Drug Designation for resminostat, treating CTCL, a rare disease affecting 5,000 Europeans annually. Resminostat showed significant efficacy in the RESMAIN study, improving progression-free survival. ODD grants 10 years' EU market exclusivity, aiding 4SC's commercialization efforts in major markets.

Catalyst Pharmaceuticals announced FDA approval for Santhera Pharmaceuticals' DMD treatment, Agamree, for patients aged two and older. The drug, similar to corticosteroids but with fewer side effects, is set for U.S. launch in Q1 2024. Catalyst plans a financial assistance program to enhance drug accessibility.

Amivantamab plus lazertinib showed superior progression-free survival over osimertinib alone in advanced EGFR+ NSCLC, with a 30% reduced risk of disease progression or death. The combination also reduced extracranial progression risk by 32%. Safety profiles were manageable, with higher rates of EGFR- and MET-related adverse events.

The Phase III PSMAfore trial demonstrated Pluvicto™ significantly improved radiographic progression-free survival (rPFS) in PSMA-positive mCRPC patients, doubling median rPFS to 12.0 months. Pluvicto also enhanced quality of life and showed favorable safety. OS data collection continues, with next analysis expected in 2024.

Verve Therapeutics announced FDA clearance of its IND application for VERVE-101, a gene-editing treatment targeting PCSK9 to lower LDL-C in HeFH patients. The heart-1 trial, evaluating VERVE-101's safety and efficacy, progresses with interim data to be presented at AHA's 2023 Scientific Sessions.

Ciltacabtagene autoleucel (cilta-cel) showed significant progression-free survival (PFS) benefits in multiple myeloma patients with poor prognostic features, including high-risk cytogenetics and triple-class refractoriness, in the CARTITUDE-4 trial. 12-month PFS rates exceeded 82% across various high-risk subgroups, affirming cilta-cel's effectiveness in a broad patient population.

Sequana Medical has announced additional positive data from the POSEIDON study, showing that the alfapump maintains a strong safety and efficacy profile at 12 months post-implantation for patients with recurrent or refractory ascites. The data also indicates a strong preference among US patients for the alfapump over standard care, with the company on track for a PMA submission by year-end.

Rise Therapeutics received FDA clearance for its IND application to start a Phase 1 clinical trial of R-2487, a novel oral immunotherapy for rheumatoid arthritis. R-2487 aims to induce regulatory T cells and reset immune balance, targeting the disease's root cause. The trial will assess safety, tolerability, and clinical activity in up to 36 participants.