Scholar Rock Announces Issuance of U.S. Patent Providing Composition of Matter Protection for SRK-015
“We are at the forefront in the development of antibodies that target the latent form of many growth factors. This composition of matter patent further protects SRK-015, which targets the latent form of myostatin and is being evaluated in the TOPAZ Phase 2 trial for patients with Spinal Muscular Atrophy,” said
The composition of matter patent is directed to the full-length amino acid sequence of SRK-015 that specifically binds to pro/latent myostatin while avoiding binding to mature myostatin and related growth factors, such as GDF11 and Activin A. The patent also covers methods of treating myopathy, muscle atrophy and metabolic disorders. The ongoing TOPAZ Phase 2 trial is evaluating SRK-015’s potential in improving motor function in patients with Type 2 and Type 3 Spinal Muscular Atrophy. A 6-month interim readout is anticipated in the fourth quarter of 2020 with top-line data for the 12-month treatment period expected in the first half of 2021.
SRK-015 is a selective inhibitor of the activation of myostatin and is an investigational product candidate for the treatment of patients with spinal muscular atrophy (SMA). Myostatin, a member of the TGFβ superfamily of growth factors, is expressed primarily by skeletal muscle cells, and the absence of its gene is associated with an increase in muscle mass and strength in multiple animal species.
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 Scholar Rock’s future expectations, plans and prospects, including without limitation, Scholar Rock’s expectations regarding its growth, strategy, progress and timing of its clinical trials for SRK-015, SRK-181, and other product candidates and development timing, the potential of its proprietary platform, and its intellectual property protection. 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, the data generated from Scholar Rock’s nonclinical and preclinical studies and clinical trials, competition from third parties that are developing products for similar uses, Scholar Rock’s ability to obtain, maintain and protect its intellectual property, the success of Scholar Rock’s current and potential future collaborations, including its collaboration with Gilead, Scholar Rock’s dependence on third parties for development and manufacture of product candidates including to supply any clinical trials, Scholar Rock’s ability to manage expenses and to obtain additional funding when needed to support its business activities and establish and maintain strategic business alliances and new business initiatives, and the impacts of public health pandemics such as COVID-19 on business operations and expectations, as well as 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
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