Benzinga | Jun 18, 2021 08:01AM EDT

Scholar Rock To Present Apitegromab TOPAZ Phase 2 Trial Results Highlighting Pharmacokinetic And Pharmacodynamic Data At 2021 European Academy Of Neurology Congress Jun. 19-22

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 e-presentation of clinical results from the TOPAZ Phase 2 trial (NCT03921528) that evaluated apitegromab in patients with Type 2 and Type 3 spinal muscular atrophy (SMA) at the Virtual 7th Congress of the European Academy of Neurology (EAN) being held June 19-22, 2021.

Details for the virtual EAN oral e-presentation are as follows:

* Title: Apitegromab, an Investigational Anti-proMyostatin Monoclonal Antibody for Spinal Muscular Atrophy: Phase 2 Results

* Muscle and Neuromuscular Junction Disease 2 Session:Virtual e-presentation #EPR-184 on Saturday, June 19 from 14:00 to 14:45 CEST (8:00-8:45am ET) in Room Paris

* Presenter:Amy Place, PhD, MBA, MS, RD, CLT on behalf of the apitegromab development team

The e-presentation will highlight the 12-month top-line TOPAZ clinical results, including PK/PD data as these relate to the efficacy of apitegromab, an investigational selective inhibitor of myostatin activation. In the TOPAZ trial, apitegromab showed therapeutic potential 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). Two doses of apitegromab (20 mg/kg and 2 mg/kg) were evaluated in the TOPAZ trial and a dose response was observed based upon clinical efficacy (improvements in Hammersmith Functional Motor Scale Expanded scores) and pharmacodynamics (target engagement). Scholar Rock plans to initiate a Phase 3 trial to further evaluate apitegromab in patients with non-ambulatory Type 2 and Type 3 SMA by the end of 2021.