On May 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 financial results and corporate updates for the first quarter ended March 31, 2023 (Press release, Scholar Rock, MAY 9, 2023, View Source [SID1234631292]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are executing across our business and leveraging Scholar Rock’s differentiated platform, which selectively targets latent forms of growth factors, such as myostatin and TGFβ1, to develop highly selective and potentially transformative therapies for patients who have high unmet medical need," said Jay Backstrom, M.D., M.P.H., President & Chief Executive Officer of Scholar Rock. "We continue to advance our two clinical programs in spinal muscular atrophy and oncology, and we look forward to sharing updates on our progress as we approach several key milestones, including presenting 36-month data on apitegromab from the Phase 2 TOPAZ trial, completing enrollment of our apitegromab Phase 3 SAPPHIRE trial, and providing biomarker and clinical updates from our SRK-181 Phase 1 DRAGON trial."

Recent Company Highlights and Upcoming Milestones

Apitegromab is an investigational antibody inhibiting myostatin activation by selectively binding the pro- and latent forms of myostatin and is being developed as the potential first muscle-targeted therapy for the treatment of spinal muscular atrophy (SMA).

Continued progress towards completion of enrollment for Phase 3 SAPPHIRE clinical trial. The randomized double-blind, placebo-controlled clinical trial evaluating apitegromab for patients with nonambulatory Types 2 and 3 SMA on either nusinersen or risdiplam, is actively enrolling SMA patients across sites in the U.S. and Europe. Enrollment completion is expected in 2023, with the top-line data readout expected in 2024. If successful and if approved, the company expects to initiate a commercial product launch in 2025.

36-month data from Phase 2 TOPAZ trial to be presented at Cure SMA conference in June. The company announced two upcoming oral presentations at the Cure SMA Research & Clinical Conference, taking place June 28-30 in Orlando, Florida.

24-month data from the TOPAZ trial presented at the 2023 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference in March. The company shared data evaluating outcomes after 24 months of treatment with apitegromab, demonstrating sustained improvements in motor function in patients with nonambulatory Types 2 and 3 SMA.

SRK-181 is an investigational selective inhibitor of latent TGFβ1 activation and is being developed with the aim of overcoming resistance to checkpoint therapy in patients with advanced cancer.

Advancing Phase 1 DRAGON proof-of-concept trial. In the second half of 2023, the company expects to provide biomarker and clinical updates from Part B of the DRAGON trial. In March 2023, Scholar Rock presented encore data at the European Society for Medical Oncology Targeted Anticancer Therapies (ESMO TAT) Congress, which showed that SRK-181 continued to be generally well tolerated with early indications of efficacy (as of the data cut-off date of December 2, 2022).
Corporate

Announced the addition of Richard Brudnick to the Board of Directors. In April 2023, Mr. Brudnick joined Scholar Rock’s Board of Directors. As an accomplished biotechnology executive, he brings an extensive background in corporate development and strategy, with over 30 years of industry experience across multiple specialties, stages and therapeutic areas.

First Quarter 2023 Financial Results

For the quarter ended March 31, 2023, net loss was $39.4 million or $0.49 per share compared to a net loss of $8.0 million or $0.21 per share for the quarter ended March 31, 2022.

Revenue was $0 for the quarter ended March 31, 2023, compared to $33.2 million for the quarter ended March 31, 2022. The prior year revenue was related to the Gilead fibrosis-focused research collaboration, which was executed in December 2018 and concluded in December 2021.

Research and development expense was $29.7 million for the quarter ended March 31, 2023, compared to $29.4 million for the quarter ended March 31, 2022. The increase was primarily attributable to costs associated with clinical trials, including the Phase 3 SAPPHIRE pivotal trial for apitegromab in SMA, and the DRAGON trial for SRK-181. These increases were offset by decreases in employee compensation and benefits costs, resulting from the restructuring in May 2022.

General and administrative expense was $10.8 million for the quarter ended March 31, 2023, compared to $10.8 million for the quarter ended March 31, 2022.
As of March 31, 2023, Scholar Rock had cash, cash equivalents, and marketable securities of approximately $275 million, which is expected to fund the company’s anticipated operating and capital expenditure requirements into 2025.

"Scholar Rock is in a strong financial position, and we are relentlessly focused on achieving our goal of improving the lives of patients who may potentially benefit from our medicines," said Ted Myles, Chief Operating Officer and Chief Financial Officer of Scholar Rock. "We continue to execute on our plan by advancing our clinical programs towards key inflection points, while diligently managing our resources."