Scholar Rock Holding Corp. (SRRK) Announces Additional Data Analyses from the Apitegromab TOPAZ Phase 2 Trial

September 23, 2021 11:31 AM EDT

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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.”

About Apitegromab

Apitegromab is a selective inhibitor of the activation of myostatin and is an investigational product candidate for the treatment of patients with spinal muscular atrophy (SMA). Myostatin, a member of the TGFβ superfamily of growth factors, is expressed primarily by skeletal muscle cells, and the absence of its gene is associated with an increase in muscle mass and strength in multiple animal species, including humans. Scholar Rock believes that inhibiting myostatin activation with apitegromab may promote a clinically meaningful improvement in motor function in patients with SMA. The U.S. Food and Drug Administration (FDA) has granted Fast Track (FTD), Orphan Drug (ODD) and Rare Pediatric Disease (RPD) designations, and the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) and Orphan Medicinal Product designations, to apitegromab for the treatment of SMA. The efficacy and safety of apitegromab have not been established and apitegromab has not been approved for any use by the FDA or any other regulatory agency.



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