GTX-102

Ultragenyx reported Q3 2024 revenue of $139 million, up 42% YoY, with Crysvita® at $98 million and Dojolvi® at $21 million. The company reaffirmed 2024 revenue guidance of $530-$550 million and announced Breakthrough Designation for setrusumab (UX143) in osteogenesis imperfecta. DTX401 Phase 3 data showed a 62% mean reduction in cornstarch in crossover patients with glycogen storage disease type Ia (GSDIa).

Acasti Pharma Inc. rebrands to Grace Therapeutics, Inc., trading on Nasdaq as 'GRCE' from Oct 28, 2024. The company focuses on GTx-104, an injectable nimodipine for aneurysmal subarachnoid hemorrhage (aSAH), with a $300M U.S. market potential. A Key Opinion Leader event on GTx-104 is scheduled for Nov 20, 2024.

FDA grants Ultragenyx's setrusumab Breakthrough Therapy designation for osteogenesis imperfecta (OI) Type I, III, or IV, targeting fracture risk reduction in patients aged two and older. Setrusumab, a sclerostin inhibitor, is being evaluated in late-stage studies for OI, with preliminary data showing significant fracture rate reduction.

Acasti Pharma completes patient enrollment in Phase 3 STRIVE-ON trial for GTX-104, an injectable nimodipine for aSAH, with data readout expected early 2025 and NDA submission planned for 1H 2025.

Acasti Pharma Inc. completed patient enrollment in its Phase 3 STRIVE-ON safety trial for GTX-104, an injectable nimodipine for aneurysmal subarachnoid hemorrhage (aSAH). The trial, involving 100 patients across 25 U.S. hospitals, compares GTX-104 with oral nimodipine. Acasti anticipates trial data in early 2025 and plans to submit a New Drug Application to the FDA in the first half of 2025.