Clinical Trial News

Stoke Therapeutics reported positive data from STK-001 studies in Dravet syndrome patients, showing significant seizure reduction and cognitive improvements. The FDA approved a dosing regimen, and the company plans regulatory discussions for a registrational study. STOK stock surged following the update.

Syndax Pharmaceuticals announces FDA Priority Review for revumenib, a menin inhibitor for relapsed/refractory KMT2Ar acute leukemia, with a PDUFA target action date of September 26, 2024, under the FDA's Real-Time Oncology Review Program.

IntraBio's NDA for IB1001, a treatment for Niemann-Pick Disease Type C, has been accepted by the FDA with a PDUFA date of September 24, 2024. IB1001 showed improvement in neurological signs, functioning, and quality of life in Phase 3 and IIb trials. IntraBio raised $40 million to support IB1001's commercialization.

Gemtuzumab ozogamicin (GO), the first ADC drug approved globally, targets CD33 in AML patients. Despite initial safety concerns leading to its withdrawal, reevaluation at lower doses showed improved safety and efficacy, leading to FDA reapproval in 2017. GO, combined with chemotherapy, enhances prognosis for AML patients, demonstrating its value in treating CD33-positive AML.

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.

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.

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.

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.

The UNIFAI Natural History Study of Friedreich’s Ataxia (FA) is a global, multicenter research initiative designed to deepen the understanding of this rare genetic disorder's progression and support the development of effective treatments. By collecting data from individuals worldwide over up to 25 years, the study aims to inform clinical trial designs and improve outcomes for those affected by FA.