Scholar Rock to Present Preclinical and Phase 1 Clinical Data on SRK-015 at the World Muscle Society Congress
CAMBRIDGE, Mass., Oct. 02, 2019 (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 that a poster presentation of preclinical and Phase 1 healthy volunteer data for SRK-015 will be presented at the World Muscle Society Congress being held October 1-5, 2019 in Copenhagen, Denmark. SRK-015 is a highly selective inhibitor of the activation of myostatin, a negative regulator of muscle mass, and is being evaluated in the TOPAZ Phase 2 clinical trial for the treatment of patients with Type 2 and Type 3 spinal muscular atrophy (SMA).
“Collectively, these preclinical and Phase 1 healthy volunteer data provided the basis for advancing SRK-015 to our TOPAZ Phase 2 clinical trial,” said Nagesh Mahanthappa, Ph.D, President and CEO of Scholar Rock. “We are excited for the potential role of SRK-015 as the first muscle-directed therapy to address the continued functional deficits experienced by some patients with SMA. We look forward to data from the upcoming clinical read-outs of the TOPAZ trial, starting with the preliminary PK/PD results at the end of 2019 and the interim efficacy and safety data in the first half of 2020.”
Consistent with previously reported results, the preclinical and Phase 1(1) clinical data to be presented at World Muscle Society show:
Phase 1 healthy volunteer trial:
The details for the SRK-015 poster at the World Muscle Society Congress are as follows:
(1) Preclinical and Phase 1 clinical data are not predictive of clinical outcomes in Phase 2 and Phase 3 clinical trials
(1) Lally, C. et al. Indirect estimation of the prevalence of spinal muscular atrophy Type I, II, and III in the United States. Orphanet Journal of Rare Diseases. (2017) 12:175.
About Scholar Rock
Scholar Rock® is a registered trademark of Scholar Rock, Inc.
This 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 future expectations, plans and prospects, including without limitation, expectations regarding the potential of SRK-015 as a therapy in SMA and the timeline for and progress in developing SRK-015. 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 the risks that earlier preclinical and clinical data and testing of SRK-015 may not be predictive of the results or success of additional clinical trials, the development of SRK-015 will take longer and/or cost more than planned, SRK-015 will not receive regulatory approval 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 June 30, 2019, which is on file with the Securities and Exchange Commission, 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.
Scholar Rock Contact: