Scholar Rock Announces Q2 2024 Financial Results and Business Progress

Scholar Rock, a biopharmaceutical company based in Cambridge, Massachusetts, announced its financial results and corporate updates for the second quarter of 2024. Focused on developing groundbreaking treatments for spinal muscular atrophy (SMA), cardiometabolic disorders, and other severe diseases, the company has made significant strides in its research programs.

According to Dr. Jay Backstrom, CEO and President, Scholar Rock has made considerable progress in its myostatin inhibition programs. The company's confidence in apitegromab, their lead drug candidate, remains strong due to consistent clinical data over the past four years. Apitegromab is a muscle-targeted therapy demonstrating clinical proof-of-concept in SMA. Dr. Backstrom shared that over 90% of patients in the TOPAZ study with nonambulatory SMA have continued on apitegromab treatment, showing sustained clinical benefits.

In the SMA program, the company is on track to release topline data from the Phase 3 SAPPHIRE trial by the fourth quarter of 2024. If successful, Scholar Rock aims to launch apitegromab commercially in 2025. Data from the 48-month study of apitegromab indicate significant improvements in motor function. Specifically, nonambulatory patients aged 2-21 showed a mean change of 5.3 points on the Hammersmith Functional Motor Scale, while patients aged 2-12 showed a mean change of 6.4 points. Additionally, the Revised Upper Limb Module (RULM) scores improved by 3.6 points for the 2-21 age group and 4.5 points for the 2-12 age group.

The ongoing ONYX extension study continues to evaluate the long-term safety and efficacy of apitegromab in patients with Types 2 and 3 SMA. More than 90% of patients on combination therapy in the TOPAZ study have completed four years of treatment and enrolled in the ONYX study.

In the cardiometabolic program, Scholar Rock's SRK-439, a preclinical myostatin inhibitor, has shown promising results. The company presented new preclinical data in May, comparing SRK-439 to an anti-activin receptor II antibody, indicating its potential to preserve lean muscle mass in patients on GLP-1 receptor agonists. Scholar Rock initiated the Phase 2 EMBRAZE trial in May, which is a randomized, double-blind, placebo-controlled study evaluating apitegromab in combination with GLP-1 receptor agonists for obesity. Enrollment is ahead of schedule, and topline data are expected in the second quarter of 2025.

Scholar Rock also shared updates on other pipeline projects. The DRAGON Phase 1 trial of SRK-181, aimed at overcoming resistance to checkpoint therapy in advanced cancer patients, showed encouraging results. Additionally, published data on SRK-373, a selective inhibitor for treating fibrosis, was featured in Science Signaling.

Financially, Scholar Rock reported a net loss of $58.5 million for the quarter ended June 30, 2024, compared to a net loss of $37.9 million for the same period in 2023. Research and development expenses increased to $42.4 million, up from $26.9 million in the previous year, primarily due to clinical trial and employee compensation costs. General and administrative expenses also rose to $17.1 million from $12.2 million. As of June 30, 2024, the company had cash, cash equivalents, and marketable securities totaling approximately $190.5 million, anticipated to fund operations into the second half of 2025.

Ted Myles, Chief Operating Officer and Chief Financial Officer, emphasized the company's robust foundation for growth, citing progress across their myostatin inhibition programs and the experienced team in place. Scholar Rock is advancing towards multiple milestones, potentially evolving into a commercial-stage company.

Scholar Rock is dedicated to discovering and developing therapies for severe diseases with high unmet needs, leveraging its expertise in the TGF-beta superfamily of cell proteins. The company continues to innovate in areas such as neuromuscular disease, cardiometabolic disorders, and cancer, aiming to transform treatment standards and improve patient outcomes.