Clinical Trial News

PTC Therapeutics plans to resubmit an NDA for Translarna™ by mid-2024, following FDA feedback. The therapy targets nonsense mutation Duchenne. PPMD supports FDA's review for informed patient decisions, hopeful for approval.

Bayer plans to seek regulatory approval for elinzanetant, a drug aimed at alleviating menopause symptoms, after positive results from a third Phase 3 trial. The drug has shown efficacy in reducing hot flashes and improving sleep in menopausal women, positioning it as a competitor to Astellas Pharma's Veozah.

Petros Pharmaceuticals integrates AI into a Human Factors Study for STENDRA (avanafil), aiming for OTC status. The AI tool, developed with a software developer, assesses safe drug administration. The study, following FDA guidance, evaluates the web app's effectiveness for patient self-screening, supporting STENDRA's potential as the first OTC erectile dysfunction medication.

Vertex Pharmaceuticals' VX-548, a non-opioid pain medication, showed safety and efficacy in trials but offered modest pain relief compared to opioids. Despite the opioid crisis driven by illicit drugs, prescription opioids remain widely used due to their effectiveness and low cost. Non-opioid treatments face clinical and reimbursement challenges, with limited success in development. VX-548 aims to address chronic pain, an area with higher addiction risks. Reimbursement hurdles persist for non-opioid therapies, complicating their adoption despite the need for alternatives.

HepaRegeniX's HRX-215, a first-in-class MKK4 inhibitor, shows promise in treating liver diseases by enhancing liver regeneration and preventing liver failure, as evidenced by clinical and preclinical data. Safe and well-tolerated in healthy volunteers, it aims to revolutionize liver surgery and transplantation, with Phase II studies planned.

The NIH, through NIDA, invites SBIR grant applications for developing tools for decentralized clinical trials (DCT) for substance use disorder (SUD). The goal is to enhance participation, diversity, and retention in SUD trials by creating tools and software for remote data collection, transfer, handling, and storage. Applications can be for Phase I, Fast-Track, or Direct to Phase II, with budgets up to $306,872 for Phase I and $2,045,816 for Phase II. The initiative aims to address the limitations of traditional SUD trials by leveraging DCT to reduce stigma, increase accessibility, and improve trial efficiency.

CAP-1002, an investigational cell therapy, has shown to slow declines in upper limb and heart function in Duchenne muscular dystrophy (DMD) patients over two years in the HOPE-2 study. A Phase 3 trial is ongoing, with potential FDA approval pathways being explored. CAP-1002 aims to benefit late-stage DMD patients by slowing skeletal and cardiac muscle function decline, demonstrating disease-modifying potential and a favorable safety profile.

Gilgamesh Pharmaceuticals received a $14M NIDA grant for GM-3009, a cardiac-safe ibogaine analog for opioid use disorder treatment, aiming to eliminate ibogaine's cardiovascular risks and prove efficacy in Phase 1/1b trials, marking a potential breakthrough in addiction therapy.

Dimerix Limited announced successful interim Phase 3 trial results for DMX-200, a treatment for Focal Segmental Glomerulosclerosis (FSGS), a rare kidney disease. The trial involved 72 patients, with plans to expand to more countries and include children over 12. DMX-200 was co-invented by Professor Kevin Pfleger, Dr James Williams, and Liddy McCall, aiming to address the high unmet need in FSGS treatment.

Rezdiffra, the first FDA-approved medication for NASH, targets liver fibrosis and NASH resolution in noncirrhotic patients with moderate to advanced liver fibrosis. Approved based on Phase 3 MAESTRO-NASH trial results, it offers a new treatment paradigm, with ongoing studies to confirm clinical benefits and support full approval.