Business
Scholar Rock to Present 24-Month Data from the Phase 2 TOPAZ trial at the 2023 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference
Oral presentation to include patient-reported outcomes data at 24 months CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Scholar Rock (NASDAQ: SRRK), a Phase 3,
About this update from Scholar Rock Holding Corporation
[{"type":"text","content":"\nOral presentation to include patient-reported outcomes data at 24 months\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nScholar Rock (NASDAQ: SRRK), a Phase 3, clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today announced it will present data from its Phase 2 TOPAZ trial extension period evaluating patient outcomes after 24 months of treatment with apitegromab, an investigational selective inhibitor of the activation of myostatin for spinal muscular atrophy (SMA). An analysis of quality-of-life measures such as fatigue, mobility, and activities of daily living, which indicates consistency with motor function improvements observed in nonambulatory SMA patients, will be presented as an oral presentation by Thomas O. Crawford, M.D., at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference on March 22.\n\n“We’re pleased to have this opportunity to share additional 24-month data from the TOPAZ trial with the neuromuscular community at the Muscular Dystrophy Association Conference,” said Jing L. Marantz, M.D., Ph.D., Chief Medical Officer of Scholar Rock. “This analysis of our 24-month data continues to provide insights into apitegromab’s potential effects on motor function and quality of life measures over time.”\n\nDetails of the presentation are as follows:\n\nTitle: Apitegromab in SMA (TOPAZ trial): Covariates of Multiple Efficacy Endpoints From 24 Month Data\nPresentation type: Oral presentation\nPresenter: Thomas O. Crawford, M.D., Professor of Neurology and Pediatrics, Johns Hopkins University\nDate and time: Wednesday, March 22, 2023, 10:15 – 10:30 a.m. CDT\nLocation: Hilton Anatole Dallas; Chantilly Ballroom West\n\nFor conference information, visit https://www.mdaconference.org/.\n\nThe presentations will be made available in the Publications & Posters section of Scholar Rock’s website following the conference.\n\nAbout the Phase 2 TOPAZ Trial\n\nThe TOPAZ trial is an ongoing proof-of-concept, open-label Phase 2 trial evaluating the safety and efficacy of apitegromab in patients with Types 2 and 3 SMA. In the main treatment period, patients were dosed intravenously every four weeks as monotherapy or with nusinersen, an approved SMN therapy. The trial enrolled 58 patients in the U.S. and Europe. The primary efficac...