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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 its participation in two upcoming investor conferences. The first is the Guggenheim 4th Annual Neuro/Immunology Conference on November 15, 2022, from 2:10-2:35 p.m. ET. The second is the Piper Sandler 34th Annual Healthcare Conference on November 29, 2022, from 8:00-8:25 a.m. ET. Live webcasts can be accessed on the Scholar Rock website, with archived replays available for 90 days post-event. Scholar Rock focuses on innovative treatments for serious diseases involving protein growth factors.
Scholar Rock (NASDAQ: SRRK) has announced new quality-of-life data from its Phase 2 TOPAZ trial extension, indicating sustained improvements in daily activities, fatigue, and muscle endurance for non-ambulatory patients with spinal muscular atrophy (SMA) over 24 months. Data presented in Barcelona suggest apitegromab may offer significant benefits, particularly for patients receiving nusinersen. No safety concerns were reported, with the majority of patients opting to continue treatment into a 36-month extension.
Scholar Rock (NASDAQ: SRRK) announced the upcoming presentation of data from the DRAGON Phase 1 clinical trial of SRK-181 at the SITC Annual Meeting in Boston, from November 8-12. The study focuses on patients with advanced solid tumors, showcasing safety, efficacy, and biomarker results from Part A and an update on Part B.
SRK-181 is designed to overcome resistance to checkpoint inhibitors, with potential benefits in tumor response and survival rates. The trial continues to enroll patients across multiple cancer types, demonstrating a promising path for future cancer therapies.
Scholar Rock (NASDAQ: SRRK) announced that new data from the Phase 2 TOPAZ trial extension for apitegromab will be presented at two upcoming conferences. The presentations, scheduled for October 12 in Halifax and October 22 in Barcelona, will focus on multiple efficacy endpoints after 24 months of treatment for spinal muscular atrophy (SMA). Apitegromab is the first selective myostatin inhibitor showing positive proof of concept in SMA. The FDA has granted several designations to apitegromab, highlighting its potential in treating this serious condition.
Scholar Rock (NASDAQ: SRRK) announced the granting of a stock option to Jay Backstrom, M.D., M.P.H., as an employment inducement. The award, part of the 2022 Inducement Plan, involves 1,000,000 shares at an exercise price of $8.85, which will vest over four years. This plan, approved on June 16, 2022, allows for equity awards to new hires or those returning after a break in employment. Scholar Rock focuses on biopharmaceutical developments targeting serious diseases influenced by protein growth factors.
Scholar Rock has appointed Dr. Jay Backstrom as CEO-Elect, effective
Scholar Rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical company, announced two upcoming presentations at major conferences on September 25-29, focusing on SRK-181, a selective inhibitor aimed at overcoming cancer therapy resistance. At the ACCP Annual Meeting, new pharmacokinetic data will be presented, while the World Clinical Biomarkers & CDx Summit will discuss biomarker strategies and early clinical data. These presentations will be available on Scholar Rock's website post-conference.
Scholar Rock (NASDAQ: SRRK) reported positive 24-month data from its Phase 2 TOPAZ trial, supporting apitegromab's potential for Spinal Muscular Atrophy (SMA) patients. The company is actively enrolling for its pivotal Phase 3 SAPPHIRE trial. Financially, Scholar Rock completed a $205 million direct offering, strengthening its balance sheet, while net loss widened to $44 million for Q2 2022, with revenues dropping to $0 from $4.6 million a year prior. R&D expenses increased due to higher clinical costs for apitegromab.
Scholar Rock (NASDAQ: SRRK) has appointed Dr. Srinivas Akkaraju, Founder and Managing General Partner at Samsara BioCapital, to its Board of Directors as an independent director. With over 20 years in biotechnology investment, Dr. Akkaraju brings extensive experience to the company as it develops its late-stage pipeline and discovery programs. His addition is anticipated to enhance Scholar Rock's strategic direction, particularly in advancing its therapeutic candidate for spinal muscular atrophy amid ongoing Phase 3 trials.
Scholar Rock (NASDAQ: SRRK) announced its participation in the ‘Muscle Beach’ panel at the 2022 Wedbush PacGrow Healthcare Conference on August 9, 2022, at 2:20 PM ET. This clinical-stage biopharmaceutical company focuses on developing treatments for serious diseases reliant on protein growth factors. A live webcast of the event will be available on their website, with an archived replay accessible for 90 days post-event.