Scholar Rock Provides Corporate Update and Highlights Priorities for 2023
- Pivotal Phase 3 SAPPHIRE trial enrollment completion expected in 2023
- Phase 1 DRAGON trial of SRK-181 continues to advance with presented data that showed early indications of efficacy; ongoing clinical data updates planned in 2023
- Anticipated cash runway into 2025
Apitegromab is a selective inhibitor of myostatin activation being developed as the potential first muscle-targeted therapy for the treatment of spinal muscular atrophy (SMA).
- Complete enrollment of Phase 3 SAPPHIRE clinical trial in 2023. SAPPHIRE is a randomized, double-blind, placebo-controlled clinical trial evaluating apitegromab for patients with nonambulatory Types 2 and 3 SMA on either nusinersen or risdiplam. The last patient is expected to be enrolled in SAPPHIRE in 2023, with the top-line data readout expected in 2024. If successful, the company expects to initiate a commercial product launch in 2025.
Progress TOPAZ long-term extension to 36-month data readout. The company expects to report 36-month extension data in the first half of 2023. As of
December 31, 2022, approximately 90 percent of patients (51/57) remained enrolled in the trial’s long-term extension period.
SRK-181 is an investigational selective inhibitor of latent TGFβ-1 activation and is being developed with the aim of overcoming resistance to checkpoint therapy in patients with advanced cancer.
Advance Progress in DRAGON Phase 1 trial.
Scholar Rockis expecting to provide biomarker and clinical updates from the DRAGON Phase 1 trial in 2023.
Preclinical fibrosis and iron-restricted anemia assets
Advance the fibrosis program towards IND-enabling studies.
Scholar Rockplans to advance a highly potent, anti-latent TGFβ-1 antibody that selectively inhibits TGFβ1 activation within the extracellular matrix by targeting latent TGFβ-1 associated with latent TGFβ-binding proteins (LTBPs), thus enabling specific inhibition of TGFβ-1 in fibrotic tissue.
Advance the iron-restricted anemia program towards IND-enabling studies.
Scholar Rockplans to advance a highly selective, RGMc/HJV antibody that targets the signaling of BMP6, a key regulator of iron availability in the body. Utilizing Scholar Rock’s unique structural biology insights into BMP6 and its co-receptors and leveraging its novel antibody discovery and optimization platform, the company generated an anti-RGMc antibody that can modulate iron release and has the potential to address anemia.
- TOPAZ 24-month extension trial data showed sizeable and sustained gains in Hammersmith Functional Motor Scale Expanded (HFMSE), increased Revised Upper Limb Module (RULM), and positive trends in quality-of-life data for nonambulatory patients with Types 2 and 3 SMA receiving an SMN-targeted therapy.
Completed equity financing of
$205 millionin June. As of December 31, 2022, Scholar Rockhad cash, cash equivalents, and marketable securities of approximately $315 million, which is expected to fund the company’s operations into 2025.
Phase 1 DRAGON trial data presented at the
Society for Immunotherapy of Cancer'sAnnual Meeting in November showed that SRK-181 continued to be generally well tolerated with early indications of efficacy (as of the data cut-off date of August 29, 2022).
Presented new data on LTBP showing reduction of TGFβ-1 signaling and fibrosis in relevant in vivo preclinical models. The findings were presented at the 2022
FASEB Science Research Conferencein July and the American College of ToxicologyAnnual Meeting in November.
Jay Backstrom, M.D., M.P.H. was appointed President & CEO in October, bringing an exceptional range of research and development, regulatory, and leadership experience spanning several decades in the biopharmaceutical industry.
Jing L. Marantz, M.D., Ph.D., M.B.A., was appointed Chief Medical Officer in November. Dr. Marantzis an accomplished biopharmaceutical executive with over 20 years of industry experience spanning multiple specialties, including neurology, hematology/oncology, and rare diseases.
“With our strong balance sheet and two well established clinical programs, both of which we expect to generate data in 2023,
Availability of Other Information About Scholar Rock
Investors and others should note that we communicate with our investors and the public using our company website www.scholarrock.com, including, but not limited to, company disclosures, investor presentations and FAQs,
Scholar Rock® is a registered trademark of
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 apitegromab, SRK-181, and other product candidates and indication selection and development timing, its cash runway, the ability of any product candidate to perform in humans in a manner consistent with earlier nonclinical, preclinical or clinical trial data, and the potential of its product candidates and proprietary platform. The use of words such as “may,” “might,” “could,” “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, without limitation, that preclinical and clinical data, including the results from the Phase 2 clinical trial of apitegromab, or Part A of the Phase 1 clinical trial of SRK-181, are not predictive of, may be inconsistent with, or more favorable than, data generated from future clinical trials of the same product candidate, including, without limitation, the Phase 3 clinical trial of apitegromab in SMA or Part B of the Phase 1 clinical trial of SRK-181, 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, information provided or decisions made by regulatory authorities, competition from third parties that are developing products for similar uses, Scholar Rock’s ability to obtain, maintain and protect its intellectual property, Scholar Rock’s dependence on third parties for development and manufacture of product candidates including, without limitation, 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 current macroeconomic and geopolitical events, including changing conditions from the COVID-19 pandemic, hostilities in