Serina Therapeutics’ SER-252 for advanced Parkinson's gains FDA support for a registrational study via the 505(b)(2) NDA pathway. The POZ Platform enables continuous dopaminergic stimulation, aiming to improve motor fluctuations.
MannKind Corporation acquires scPharmaceuticals, significantly expanding its presence in cardiometabolic medicine with FUROSCIX, an FDA-approved treatment for heart and kidney disease. This strategic move aims to diversify revenue and accelerate growth.
Rocket Pharma's RP-A501 Danon disease trial resumed after FDA lifted hold due to patient death. Shares surged. It will use a lower dose & modified regimen. Signals FDA support for gene therapy despite investor wariness & other patient deaths, like Sarepta's and Neurogene's.
Celldex stopped barzolvolimab for eosinophilic esophagitis after it failed to improve symptoms, despite depleting mucosal mast cells. The company will now explore other GI diseases driven by mast cells where the antibody might be effective.
The FDA declined PTC Therapeutics' vatiquinone for Friedreich's Ataxia, citing insufficient efficacy data and requiring more studies.
Viking Therapeutics' oral VK2735 achieved up to 12.2% weight loss in a Phase 2 trial, which was below expectations. It is safe, well-tolerated, and has mild gastrointestinal effects.
