On January 9, 2023 Scholar Rock (NASDAQ: SRRK), a Phase 3 clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, reported its recent corporate updates and highlighted upcoming priorities for its pipeline programs in 2023 (Press release, Scholar Rock, JAN 9, 2023, View Source [SID1234626106]).
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"In 2022, Scholar Rock made significant progress in advancing its clinical programs, notably with the 24-month extension data from the Phase 2 TOPAZ trial, which reinforces our conviction behind apitegromab and the Phase 3 SAPPHIRE trial, and with early data readouts from the Phase 1 DRAGON trial. The company also strengthened its financials in June 2022 with a substantial equity raise. We are excited by the potential coming from our highly differentiated platform targeting growth factors like TGFβ, as we advance our spinal muscular atrophy and oncology programs to address critical unmet needs for patients," said Dr. Jay Backstrom, M.D., M.P.H., President & CEO of Scholar Rock. "In 2023, we see continued momentum for our growing pipeline, including completing enrollment of our pivotal SAPPHIRE trial, disclosing 36-month extension data from the Phase 2 TOPAZ trial and clinical and biomarker updates from the SRK-181 Phase 1 DRAGON trial, and advancing two preclinical assets towards IND-enabling studies in fibrosis and iron-restricted anemia."
2023 Priorities:
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 Rock is 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 Rock plans 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 Rock plans 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.
2022 Highlights:
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 million in June. As of December 31, 2022, Scholar Rock had 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 (SITC) (Free SITC Whitepaper)’s Annual 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 Conference in July and the American College of Toxicology Annual Meeting in November.
Announced 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.
Announced Jing L. Marantz, M.D., Ph.D., M.B.A., was appointed Chief Medical Officer in November. Dr. Marantz is 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, Scholar Rock is uniquely positioned to bring differentiated therapies to patients suffering from serious diseases in which protein growth factors play a fundamental role," said Ted Myles, Chief Operating Officer and Chief Financial Officer.