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Scholar Rock Holding Corporation develops biologic therapies for spinal muscular atrophy and other rare, severe, and debilitating neuromuscular diseases through its myostatin biology platform. Its principal program, apitegromab, is an anti-myostatin therapy being developed for children and adults with SMA, and its pipeline also includes subcutaneous apitegromab and SRK-439.
Recurring company updates include FDA regulatory activity for apitegromab, manufacturing and fill-finish matters tied to the program, quarterly financial results, cash and financing activity such as debt and at-the-market equity programs, and Nasdaq inducement equity grants for new employees. Scholar Rock also reports investor-conference participation and pipeline commentary related to anti-myostatin biology.
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.
Scholar Rock (NASDAQ:SRRK) has announced positive top-line results from the 12-month TOPAZ Phase 2 trial for apitegromab, aimed at treating Type 2 and Type 3 spinal muscular atrophy (SMA). Key findings include a 0.3-point decline in the Revised Hammersmith Scale (RHS) for ambulatory patients, with 57% maintaining or improving motor function. Non-ambulatory patients showed a 0.6-point improvement in the Hammersmith Functional Motor Scale Expanded (HFMSE). Cohort 3 reported a 7.1-point mean improvement in HFMSE. No major safety concerns were raised, with ongoing studies planned for additional indications.
Scholar Rock (NASDAQ: SRRK) announced the publication of preclinical data for SRK-181 in the International Journal of Toxicology. SRK-181 is a potent inhibitor of latent TGFβ1, aimed at overcoming resistance to checkpoint inhibitor therapy, and is currently in the DRAGON Phase 1 trial for solid tumors. The publication details SRK-181’s pharmacology, pharmacokinetics, and safety, supporting its clinical evaluation. Promising preclinical findings indicate a potentially improved safety profile compared to non-selective TGFβ inhibitors, with further clinical results expected by year-end.
Scholar Rock (NASDAQ: SRRK) has appointed Joshua Reed as an independent director on its Board of Directors. Reed, with over two decades of biopharmaceutical experience, will also chair the audit committee. His background includes significant roles at Aldeyra Therapeutics and Bristol-Myers Squibb. The company emphasizes that Reed's operational and strategic expertise is crucial at this stage of its growth, particularly as it aims to advance innovative treatments for serious diseases such as spinal muscular atrophy and cancer. Reed expressed enthusiasm about joining the team to drive transformative milestones.
Scholar Rock (NASDAQ: SRRK) announced the issuance of U.S. Patent No. 10,946,036, expiring June 2037, which covers myostatin inhibitor and neuronal corrector therapy for Spinal Muscular Atrophy (SMA). CEO Tony Kingsley expressed enthusiasm for this patent, anticipating top-line results from the apitegromab TOPAZ Phase 2 trial next quarter. The patent claims are broad and also granted in Japan and Europe. SMA affects 30,000 to 35,000 patients in the U.S. and Europe, highlighting the need for new therapies addressing muscle atrophy.
Scholar Rock (NASDAQ: SRRK) has announced the presentation of interim data from the TOPAZ Phase 2 clinical trial of apitegromab at the 2021 MDA Virtual Clinical Conference. The study focuses on apitegromab, a selective myostatin inhibitor for treating Type 2 and Type 3 Spinal Muscular Atrophy (SMA). Interim data from the trial will be shared in both an oral presentation on March 18 and a virtual poster available from March 15-18. Full 12-month data is anticipated in Q2 2021. Apitegromab has received Orphan Drug Designation from the FDA and the European Commission.