Scholar Rock Announces Q1 2024 Financial Results and Business Updates

Scholar Rock, a biopharmaceutical company based in Cambridge, Massachusetts, has shared its financial results and corporate updates for the first quarter ending March 31, 2024. The company, which is advancing treatments for spinal muscular atrophy (SMA), cardiometabolic disorders, and other severe diseases, has provided significant updates on its clinical trials and financial health.

According to Jay Backstrom, M.D., MPH, the President and CEO of Scholar Rock, the company is nearing the release of topline data from the pivotal Phase 3 SAPPHIRE trial for SMA, expected in the fourth quarter of 2024. If successful, this could lead to the commercial launch of apitegromab, a novel muscle-targeted therapy for SMA, in 2025. Apitegromab has shown clinical proof-of-concept as a treatment for SMA and is designed to inhibit the activation of myostatin in skeletal muscles, thereby preserving muscle mass.

Additionally, Scholar Rock is advancing its cardiometabolic program. The company is set to begin a Phase 2 proof-of-concept trial in June 2024 to evaluate apitegromab in combination with a GLP-1 receptor agonist for the treatment of obesity. This trial aims to assess the effectiveness of this combination therapy in preserving lean muscle mass as part of weight management strategies. New preclinical data on SRK-439, another investigational myostatin inhibitor, will be presented at the American Diabetes Association's annual meeting in June 2024.

In the field of immuno-oncology, Scholar Rock continues to develop SRK-181, a selective inhibitor targeting latent TGFβ1 activation. The company will present new data from the Phase 1 DRAGON proof-of-concept trial at the American Society of Clinical Oncology (ASCO) annual meeting in June 2024. This trial examines the effectiveness of SRK-181 combined with pembrolizumab in patients with advanced solid tumors who have shown resistance to anti-PD1 therapies.

Financially, Scholar Rock reported a net loss of $56.9 million for the first quarter of 2024, compared to a net loss of $39.4 million for the same period in 2023. Research and development expenses rose to $43.1 million from $29.7 million, mainly due to clinical trial costs. General and administrative expenses also increased to $15.3 million, driven by higher employee-related costs. As of March 31, 2024, the company had cash, cash equivalents, and marketable securities totaling $238 million, expected to support operational needs into the second half of 2025.

Ted Myles, Chief Operating Officer and Chief Financial Officer of Scholar Rock, emphasized the company's focus on achieving key milestones in 2024, including the pivotal Phase 3 readout of apitegromab for SMA and the progression of their cardiometabolic program. The company's strong cash position is seen as a critical enabler for reaching these goals.

The Phase 3 SAPPHIRE trial is a randomized, double-blind, placebo-controlled study evaluating apitegromab's safety and efficacy in nonambulatory patients with Types 2 and 3 SMA who are already receiving SMN-targeted therapies. The trial aims to enroll around 156 patients aged 2-12 years in the main efficacy group and an additional 48 patients aged 13-21 years in an exploratory group.

Scholar Rock is also making strides with SRK-439, targeting obesity by preserving lean muscle mass through selective myostatin inhibition. Preclinical data suggests a promising profile for this investigational therapy, though its efficacy and safety have yet to be established in clinical trials.

In summary, Scholar Rock is making significant progress across multiple therapeutic areas with several upcoming milestones that could have a substantial impact on the treatment of SMA and other serious diseases. The company's robust financial position provides a solid foundation for continued innovation and clinical advancement.