Scholar Rock Holding Stock Price, News & Analysis

Scholar Rock (NASDAQ: SRRK) announced promising results from its TOPAZ Phase 2 trial for apitegromab, showing significant motor function improvements in Type 2 and 3 SMA patients. The trial revealed a 74% clinical improvement rate in non-ambulatory patients on nusinersen, with notable Hammersmith Functional Motor Scale Expanded (HFMSE) score increases. Apitegromab, a myostatin inhibitor, may complement existing SMA therapies. A Phase 3 trial is expected to begin by the end of 2021, further evaluating apitegromab's efficacy when combined with other treatments.

Scholar Rock (NASDAQ: SRRK) announced positive results from the Phase 2 TOPAZ trial of apitegromab for Type 2 and Type 3 spinal muscular atrophy (SMA). The trial demonstrated improved motor function in non-ambulatory patients already on SMN therapy. An oral presentation will occur on June 11, 2021, at the Cure SMA Annual Conference. With Fast Track and Orphan Drug designations from the FDA, Scholar Rock anticipates launching a Phase 3 trial by the end of 2021 to further evaluate apitegromab's efficacy in SMA patients.

Scholar Rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical firm, announced participation in key investor conferences. Management will present at the Jefferies Virtual Healthcare Conference on June 2, 2021, at 11:00 a.m. ET, and the Goldman Sachs Annual Global Healthcare Conference on June 9, 2021, at 2:10 p.m. ET. Additionally, they'll be part of the BMO Biopharma Day: Rare Disease Panel on June 22, 2021, at 10:30 a.m. ET. Webcasts of these presentations can be accessed via the Scholar Rock website, with replays available for 90 days.

Scholar Rock (NASDAQ: SRRK) announced that the FDA has granted Fast Track designation for apitegromab, targeting Spinal Muscular Atrophy (SMA). This designation aims to expedite drug development for serious conditions. Scholar Rock plans to submit a rolling Biologic License Application if criteria are met. The company sees apitegromab as a potential first muscle-directed therapy for SMA, with promising results from the TOPAZ Phase 2 trial. The drug has also received other crucial designations from both the FDA and EMA, highlighting an urgent medical need for effective SMA treatments.

Scholar Rock (NASDAQ: SRRK) announced a presentation of its DRAGON Phase 1 trial for SRK-181 at the 2021 ASCO Annual Meeting, focusing on patients with solid tumors resistant to checkpoint inhibitors. The trial aims to assess the efficacy of SRK-181, a selective TGFβ1 inhibitor, in overcoming this resistance. The poster will detail the trial design and biomarker analysis. The presentation is scheduled for June 4, 2021, showcasing SRK-181's potential to enhance tumor response when combined with anti-PD-(L)1 therapy.

Scholar Rock (NASDAQ: SRRK) announced its Q1 2021 financial results and progress on pipeline programs. Revenue decreased to $4.7 million from $5.0 million year-over-year, while net loss increased to $27.7 million, or $0.76 per share. The company highlighted positive top-line data from the TOPAZ Phase 2 trial for apitegromab, showing significant motor function improvements in SMA patients. Scholar Rock plans to initiate a Phase 3 trial by year-end 2021. With $315 million in cash, the company is well-positioned to fund operations into 2023.

Scholar Rock (NASDAQ: SRRK) announced the publication of Phase 1 trial results for apitegromab in the journal Advances in Therapy. Apitegromab, a selective myostatin inhibitor, aims to treat spinal muscular atrophy (SMA). Key findings include safety at doses up to 30 mg/kg and a 24-31 day half-life. Following positive Phase 2 results indicating improved motor function in Type 2 and Type 3 SMA, a Phase 3 trial is expected to start by the end of 2021. The FDA has granted Orphan Drug Designation and Rare Pediatric Disease designation for apitegromab.

Scholar Rock (NASDAQ: SRRK) announced the issuance of U.S. Patent No. 10,981,981, expiring in May 2034, covering methods for inhibiting myostatin activation. This patent supports the company’s proprietary platform that targets precursor growth factors. CEO Tony Kingsley highlighted the recent positive results from the TOPAZ Phase 2 trial for apitegromab, which aims to enhance motor function in spinal muscular atrophy (SMA) patients. The trial demonstrated significant potential for apitegromab as a first muscle-directed therapy for SMA.

Scholar Rock (NASDAQ:SRRK) announced a poster presentation at the AACR Annual Meeting 2021, detailing biomarker assays for its DRAGON Phase 1 trial of SRK-181, a selective TGFβ1 inhibitor. This investigational candidate targets patients with advanced solid tumors resistant to checkpoint inhibitors. The e-poster, titled “Development of a Comprehensive Biomarker Strategy to Support the Latent TGFβ1 Inhibitor SRK-181 Phase 1 Clinical Trial, DRAGON,” will be available for on-demand viewing starting April 10, 2021.