On November 14, 2022 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 financial results and corporate updates for the third quarter ended September 30, 2022 (Press release, Scholar Rock, NOV 14, 2022, View Source [SID1234624024]).
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"We continue to build conviction in our clinical programs with recent TOPAZ data indicating apitegromab’s potential for sustained improvements of quality-of-life measures over 24 months, and with the most recently available SRK-181 data from DRAGON that we presented at SITC (Free SITC Whitepaper)," said Jay Backstrom, M.D., M.P.H., Chief Executive Officer & President of Scholar Rock. "I am excited to strengthen our experienced executive team with the addition of Scholar Rock’s new CMO, Dr. Jing Marantz. I am confident the continued momentum of the Phase 3 SAPPHIRE trial and the DRAGON trial will be further bolstered under her leadership."
Company Highlights and Upcoming Milestones
Pipeline Updates
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).
Presented new Phase 2 TOPAZ trial data indicating positive trends in quality-of-life measures over 24 months with apitegromab for nonambulatory patients with Types 2 and 3 SMA. Scholar Rock presented these data, which indicate the potential for sustained improvement in quality-of-life measures for patients with symptomatic SMA and offer further signs of possible durable effects of apitegromab, at the World Muscle Society (WMS) Hybrid Congress and the International Scientific Congress on Spinal Muscular Atrophy in October.
Continued enrollment of the Phase 3 SAPPHIRE clinical trial. SAPPHIRE, a randomized, double-blind, placebo-controlled clinical trial evaluating apitegromab for patients with non-ambulatory Types 2 and 3 SMA on either nusinersen or risdiplam, is actively enrolling SMA patients across 55 sites in the U.S. and Europe. The anticipated enrollment is approximately 156 patients aged 2-12 in the main efficacy population.
SRK-181 is an investigational selective inhibitor of latent TGFβ1 activation and is being developed with the aim of overcoming resistance in patients with advanced cancer.
Data from the Phase 1 DRAGON trial presented at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s Annual Meeting show that SRK-181 continues to be generally well tolerated with early indications of efficacy (as of the data cut-off date of August 29, 2022).
No dose-limiting toxicities were observed in patients participating in Part A, and all dose levels were generally well tolerated, including the recommended SRK-181 dose of 1500 mg q3w or 1000 mg q2w in combination with anti‑PD-(L)1 for Part B.
In Part A2 of the trial (n=15), there was one confirmed partial response ("PR") in a patient with anti-PD-1 resistant clear cell renal cell carcinoma at 800mg who remained in the study for 30 weeks. Response was assessed based on RECIST 1.1 by principal investigators. One ongoing patient in the 2400 mg dose group of Part A2 with head and neck cancer experienced a 29.4% tumor reduction.
Enrollment in DRAGON Part B is ongoing. Across the cohorts, 14 patients have been dosed as of the data cut-off date. The recommended dose of 1500mg q3w or 1000mg q2w SRK-181 in combination with anti-PD-(L)1 was generally well tolerated. There was a confirmed PR in one ongoing patient with anti-PD-1 resistant clear cell renal cell carcinoma. Scholar Rock will continue to provide updates at future scientific meetings.
Corporate Updates
Announced key leadership appointments:
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. He has been instrumental in organizing and executing development strategies and has led programs in a broad range of therapeutic areas through regulatory approvals. Dr. Backstrom most recently served as Executive Vice President, Research and Development at Acceleron Pharma, which was acquired by Merck in 2021.
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. Most recently, she served as the Executive Vice President and Chief Business Officer at Krystal Biotech. Previously, she was Senior Vice President and Head of Medical Affairs at Acceleron Pharma, where she also was a member of the R&D leadership team.
Amended debt facility with Oxford Finance and Silicon Valley Bank. Scholar Rock amended the existing debt facility with Oxford Finance and Silicon Valley Bank. Under the agreement, an additional $25 million tranche is available at the company’s discretion and subject to meeting certain criteria. The amendment also extends both the interest-only period for an additional 24 months to November 2024, and the loan maturity date to November 2027.
Third Quarter 2022 Financial Results
For the quarter ended September 30, 2022, net loss was $43.3 million or $0.55 per share compared to a net loss of $37.5 million or $1.02 per share for the quarter ended September 30, 2021.
Revenue was $0 for the quarter ended September 30, 2022, compared to $5.5 million for the quarter ended September 30, 2021.
Research and development expense was $33.4 million for the quarter ended September 30, 2022, compared to $31.3 million for the quarter ended September 30, 2021. The increase was primarily attributable to expenses related to non-cash equity-based compensation expense, a component of employee compensation and benefits costs.
General and administrative expense was $10.5 million for the quarter ended September 30, 2022, compared to $11.3 million for the quarter ended September 30, 2021. The decrease was attributable to the reduction of employee severance and benefits expenses that were recognized during the same period in the prior year.
As of September 30, 2022, Scholar Rock had cash, cash equivalents, and marketable securities of approximately $343.7 million, which is expected to fund the Company’s anticipated operating and capital expenditure requirements into 2025.
"With the successful equity financing that we completed in the second quarter and the added flexibility provided by the amendment of our debt facility, we are well funded to advance our pipeline through key milestones," said Ted Myles, Chief Operating Officer and Chief Financial Officer of Scholar Rock. "We believe the company is well positioned to deliver value to patients with unmet medical needs. With sufficient capital to complete the topline data readout from the SAPPHIRE study, progress Part B of the DRAGON trial, and continue advancing select preclinical programs, we are laser focused on the successful execution of these priorities."