Clinical Trial News

Elixir Medical's DynamX® BTK System, a novel bioadaptor platform for treating chronic limb-threatening ischemia (CLTI) in below-the-knee vessels, received FDA Breakthrough Device Designation. It uniquely supports and restores vessel function post-intervention, addressing a critical unmet need in vascular disease treatment.

ACELYRIN announced positive Phase 1/2 trial results for lonigutamab, a subcutaneous anti-IGF-1R treatment for Thyroid Eye Disease (TED), showing rapid improvements in proptosis and clinical activity score within 3 weeks. The treatment, well-tolerated with no serious adverse events, demonstrated potential for longer-term dosing to enhance clinical response depth and durability. A Phase 2b/3 trial is planned for 2024.

FDA approved Lenmeldy, a $4.25M gene therapy for MLD, the most expensive drug. Acelyrin reported positive results for a thyroid eye disease treatment. Clasp Therapeutics raised $150M for a cancer treatment targeting body's defenses with minimal risk to healthy cells.

The FDA has accepted Journey Medical’s new drug application for DFD-29, a minocycline hydrochloride modified release capsule for rosacea treatment, setting a PDUFA date of Nov. 4. Supported by positive phase 3 trial data, DFD-29 aims to address both inflammatory lesions and erythema, potentially becoming a preferred treatment option.

Acelyrin, an immunology biotech company, has announced positive early-stage clinical trial results for its experimental treatment targeting thyroid eye disease, potentially positioning it as a competitor to Tepezza.

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.