Benzinga | Jun 7, 2021 07:37AM EDT

Scholar Rock to Present Apitegromab Clinical Data from the TOPAZ Phase 2 Trial in Patients with Type 2 and 3 Spinal Muscular Atrophy at the 2021 Virtual SMA Research & Clinical Care Meeting

Scholar Rock (NASDAQ:SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today announced an oral presentation of apitegromab TOPAZ Phase 2 clinical trial results at the 2021 Virtual SMA Research and Clinical Care Meeting, being held in conjunction with Cure SMA's Annual SMA Conference June 7-11, 2021.

Details for the virtual Cure SMA oral presentation are as follows:

* Title: TOPAZ: A Phase 2 Study to Evaluate the Efficacy and Safety of Apitegromab (SRK-015) in Patients with Later-Onset Spinal Muscular Atrophy (Type 2 and Type 3 SMA): Topline Results

* Presenter: Thomas Crawford, M.D., Co-Director, Muscular Dystrophy Association Clinic and Professor of Neurology, Johns Hopkins Medicine (Lead TOPAZ Principal Investigator)

* Clinical Drug Development Session: Virtual oral presentation on June 11, 2021 at 2:00pm CST

Scholar Rock recently announced positive 12-month top-line data from the TOPAZ Phase 2 trial (NCT03921528), which evaluated apitegromab, a selective inhibitor of myostatin activation, in patients with Type 2 and 3 SMA. The results show the transformative potential of apitegromab to further improve motor function in patients with non-ambulatory Type 2 and 3 SMA who were already receiving chronic maintenance therapy with a background SMN upregulator (nusinersen). A Phase 3 registrational trial in patients with non-ambulatory Type 2 and Type 3 SMA is anticipated to initiate by the end of 2021.