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 two poster presentations as part of the World Muscle Society (WMS) Virtual Congress taking place September 20-24, 2021.
Details for the virtual posters at the WMS meeting are as follows:
- Title (late breaker): Apitegromab in Spinal Muscular Atrophy (SMA): An Analysis of Multiple Efficacy Endpoints in the TOPAZ Trial (LBP.10)
- This late breaking poster presentation will feature multiple efficacy endpoints, including Hammersmith scales and Revised Upper Limb Module (RULM) function, from the 12-month TOPAZ Phase 2 trial evaluating apitegromab in patients with Type 2 and 3 Spinal Muscular Atrophy (SMA).
- An additional exploratory analysis evaluating time to achieving various thresholds of improvement in Hammersmith Functional Motor Scale Expanded (HFMSE) scores further support the dose response in clinical efficacy.
- Increases in RULM were also observed in both non-ambulatory cohorts.
- Greater increases in HFMSE (non-ambulatory) and Revised Hammersmith Scale (ambulatory) scores were seen in patients who were not limited by scoliosis or joint contractures.
- Title: Insights into the Potential Pharmacological Effects of Apitegromab in Health and Disease: Data from Preclinical and Clinical Studies (EP.277)
- The poster will showcase insights into the target engagement and exposure levels of apitegromab in the TOPAZ trial as well as from Phase 1 healthy volunteer and preclinical studies.
- Virtual poster presentations (LBP.10 and EP.277) on September 23, 2021, at 16:30-18:30 BST
- E-Posters available to view on demand and exhibition area open starting Monday, September 20, 2021.
“These additional analyses from the TOPAZ Phase 2 trial further reinforce our enthusiasm for the potential of apitegromab to improve motor function for patients with SMA, and provide exploratory insights in both the ambulatory and non-ambulatory populations,” said Yung Chyung, M.D., Chief Medical Officer of Scholar Rock. “We anticipate initiating a Phase 3 trial to evaluate apitegromab in patients with non-ambulatory Type 2 and Type 3 SMA by the end of 2021 as we work towards our aim of establishing apitegromab as the potential first muscle-directed therapy to treat SMA.”