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Scholar Rock Holding Corporation (SRRK) is a biotechnology company dedicated to pioneering a novel class of biologic therapies known as 'niche modulators'. These therapies selectively target the activation of growth factors within the disease microenvironment, offering a groundbreaking approach to treating serious medical conditions. Scholar Rock's initial proprietary and partnered drug discovery programs focus on specific growth factors, particularly members of the TGF-beta superfamily, which are critical in regulating cell growth and differentiation. These growth factors are notably present in the microenvironments of significant diseases like fibrosis, musculoskeletal disorders, and autoimmune diseases.
The company’s innovative therapeutic strategy stems from the foundational scientific discoveries of its founders, Professors Timothy Springer, PhD, and Leonard Zon, MD, from Children's Hospital Boston and Harvard Medical School. These discoveries illuminate the molecular mechanisms of growth factor activation, empowering Scholar Rock to advance its ground-breaking treatments.
Scholar Rock is backed by a consortium of top-tier life sciences investors, including Polaris Partners and Arch Venture Partners. Its flagship product candidate, Apitegroma, is a selective, fully human, monoclonal antibody that inhibits the activation of the growth factor myostatin in skeletal muscle. This innovative mechanism holds promise for treating neuromuscular disorders. Another key product in the company's pipeline is SRK-181, which targets the activation of latent transforming growth factor beta-1 (TGFβ1) and is being developed to treat cancers that are resistant to existing anti-PD-(L)1 antibody therapies.
With its operations based in the United States, Scholar Rock continues to make significant strides in the biopharmaceutical industry, pushing the envelope of medical science and offering hope for effective treatments against some of the most challenging diseases.
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.
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.
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