Full Press Release Details
CAMBRIDGE, Mass., Feb. 06, 2020 (GLOBE NEWSWIRE) -- 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 on SRK-015, a highly specific inhibitor of the activation of latent myostatin, at the SMA Europe 2ndInternational Scientific Congress being held in Paris-Evry, France. The two posters will highlight key preclinical and Phase 1 clinical data for SRK-015 as well as preliminary demographics, baseline characteristics, and pharmacokinetic (PK)/pharmacodynamic (PD) data from the TOPAZ Phase 2 proof-of-concept trial evaluating SRK-015 for the treatment of patients with Type 2 and Type 3 Spinal Muscular Atrophy (SMA).
“We are pleased to be presenting preclinical and clinical data for SRK-015 at the SMA Europe Congress as we continue to advance the development of SRK-015 towards a goal of improving motor function in patients with SMA,” said Yung Chyung, M.D., Chief Medical Officer of Scholar Rock. “With the recent completion of enrollment in the TOPAZ Phase 2 trial, we now await the interim safety and efficacy results that are expected mid-year for an early look at the potential of SRK-015 in addressing the continued functional deficits experienced by patients with SMA despite treatment with SMN upregulators.”
TOPAZ Phase 2 Proof-of-Concept Trial Details and Preliminary PK/PD Data
A Phase 2 Study to Evaluate the Efficacy and Safety of SRK-015 in Patients with Later-Onset Spinal Muscular Atrophy (TOPAZ): An Introduction
Preliminary TOPAZ demographic and baseline characteristics:
Preliminary PK/PD analysis (as of data cutoff in November 2019):
Preclinical and Phase 1 Healthy Volunteer Clinical Data(1)
Clinical Development of SRK-015, a Fully Human Anti-proMyostatin Monoclonal Antibody, for the Treatment of Later-Onset Spinal Muscular Atrophy
Preclinical studies(2):
Phase 1 healthy volunteer trial(3):
Both posters can be accessed at:https://scholarrock.com/platform/publications/
About Scholar RockScholar Rockis a clinical-stage biopharmaceutical company focused on the discovery and development of innovative medicines for the treatment of serious diseases in which signaling by protein growth factors plays a fundamental role. Scholar Rock is creating a pipeline of novel product candidates with the potential to transform the lives of patients suffering from a wide range of serious diseases, including neuromuscular disorders, cancer, fibrosis and anemia. Scholar Rock’s newly elucidated understanding of the molecular mechanisms of growth factor activation enabled it to develop aproprietary platformfor the discovery and development of monoclonal antibodies that locally and selectively target these signaling proteins at the cellular level. By developing product candidates that act in the disease microenvironment, the Company intends to avoid the historical challenges associated with inhibiting growth factors for therapeutic effect. Scholar Rock believes its focus on biologically validated growth factors may facilitate a more efficient development path. For more information, please visitwww.ScholarRock.comor follow Scholar Rock on Twitter (@ScholarRock) and LinkedIn (https://www.linkedin.com/company/scholar-rock/).
Scholar Rock® is a registered trademark of Scholar Rock, Inc.
Forward-Looking StatementsThis press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding Scholar Rock’s future expectations, plans and prospects, including without limitation, Scholar Rock’s expectations regarding the timing of its clinical trials for SRK-015; the potential of SRK-015 to address certain patient unmet needs; and the ability of any product candidate to perform in humans in a manner consistent with nonclinical or preclinical study data. The use of words such as “may,” “might,” “will,” “should,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “project,” “intend,” “future,” “potential,” or “continue,” and other similar expressions are intended to identify such forward-looking statements. All such forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include Scholar Rock’s ability to provide the financial support, resources and expertise necessary to identify and develop product candidates on the expected timeline; preclinical data and results may not be predictive of clinical results; Scholar Rock’s dependence on third parties for development and manufacture of product candidates including to supply any clinical trials; and those risks more fully discussed in the section entitled "Risk Factors" in Scholar Rock’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2019, as well as discussions of potential risks, uncertainties, and other important factors in Scholar Rock’s subsequent filings with the Securities and Exchange Commission. Any forward-looking statements represent Scholar Rock’s views only as of today and should not be relied upon as representing its views as of any subsequent date. All information in this press release is as of the date of the release, and Scholar Rock undertakes no duty to update this information unless required by law.