Clinical Trial News

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.

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.

Zacks.com highlights biotech stocks, focusing on Q4 earnings and regulatory updates. Moderna beat earnings estimates, reaffirmed 2024 sales guidance, and expects RSV vaccine approval. RAPT Therapeutics faced a clinical hold due to a liver failure case. Iovance surged after FDA approval for melanoma treatment. Sarepta seeks label expansion for DMD drug. Regeneron's myeloma drug gets Priority Review.

Clearside Biomedical discussed its Q4 2023 financial results and corporate updates, highlighting progress in its CLS-AX clinical development program for wet AMD, including the initiation and completion of the Phase IIb trial, Odyssey. The company emphasized its leadership in suprachoroidal drug delivery technology, collaborations, and anticipated data readouts in 2024. Financial updates included a cash balance sufficient to fund operations into Q3 2025.

Tyra Biosciences Inc reported a Q4 net loss of $22.8M, up from $12.9M last year, with R&D expenses doubling to $20.7M. The company's cash position is strong at $403.5M, supporting operations through 2026. Progress includes advancing TYRA-300 for mUC treatment and SURF301 Phase 1 study, with key milestones expected in 2024.

PMV Pharmaceuticals ended 2023 with $228.6M in cash, reporting a $69.0M net loss, improved from $73.3M. R&D expenses rose to $55.9M for rezatapopt development, while G&A costs fell to $24.2M. The company progresses in the PYNNACLE trial, with Phase 2 starting Q1 2024, and strengthens leadership for clinical advancements.

Amylyx Pharmaceuticals' shares fell after its ALS treatment failed in a phase III study. Acadia Pharmaceuticals also saw a decline due to a failed schizophrenia treatment study. Regulus Therapeutics reported positive results for a kidney disease treatment, boosting its shares. Moderna initiated a cancer therapy study, increasing its stock value. Regeneron received FDA approval to extend its cholesterol drug's label to pediatric patients.

BioNTech reported strong 2023 financials with €3.8B in revenues and advanced its oncology and infectious disease pipelines, aiming for multiple product approvals. The company plans to initiate 10+ registration trials by 2024 end, targeting a first oncology product launch by 2026. Despite COVID-19 vaccine revenue challenges, BioNTech remains profitable, focusing on innovative therapies and strategic collaborations to drive long-term growth.

Vaxart discussed its 2023 achievements, including progress in oral vaccine development for norovirus and COVID-19, highlighted by a $9.27M BARDA contract for a Phase IIb COVID-19 trial. The company emphasized the potential of its oral pill vaccines to offer advantages over injectables, such as ease of administration and mucosal immunity. Vaxart also announced the appointment of a new CEO, Steve Lo, and shared updates on financial results and future clinical trial plans.

A phase 1 clinical trial evaluating a new CAR-T therapy approach for glioblastoma has shown dramatic tumor regression in patients, with one achieving near-complete regression. Despite eventual tumor progression, the results are promising for treating this deadly brain cancer.