As of September 30, 2024, Scholar Rock Holding Corporation reported total assets of $179.1 million, a significant decrease from $311.0 million at the end of 2023. Current assets also fell to $152.9 million from $288.2 million, with cash and cash equivalents declining to $76.4 million from $101.9 million. Marketable securities saw a sharp drop to $62.7 million from $178.1 million. Total liabilities increased to $99.9 million from $85.8 million, while stockholders' equity decreased to $79.2 million from $225.2 million. The accumulated deficit rose to $(856.3 million) from $(676.4 million).

For the three months ending September 30, 2024, operating expenses surged to $64.8 million, up 48.3% from $43.7 million in the same period of 2023. Research and development expenses rose significantly to $48.7 million from $30.3 million, reflecting a 60.6% increase. The loss from operations for this quarter was $(64.8 million), compared to $(43.7 million) in 2023. The net loss for the quarter was $(64.5 million), an increase from $(42.4 million) in the prior year, marking a 52.2% rise.

For the nine months ended September 30, 2024, total operating expenses reached $182.7 million, up from $123.3 million in 2023, while net loss increased to $(179.8 million) from $(119.7 million), reflecting a 50.3% rise. Research and development expenses for this period totaled $134.2 million, compared to $86.9 million in 2023, driven by increased costs associated with clinical trials, particularly for the apitegromab program.

Scholar Rock's primary focus remains on the development of innovative therapies, particularly apitegromab, which targets spinal muscular atrophy (SMA). The pivotal Phase 3 SAPPHIRE trial for apitegromab completed enrollment in 2023, with positive top-line results announced in October 2024. The company plans to submit a U.S. Biologics License Application and a European Union marketing authorization application in early 2025, with a commercial launch expected later that year.

In addition to apitegromab, Scholar Rock is expanding its portfolio with new candidates, including SRK-439 for cardiometabolic disorders and SRK-181 for certain cancers. The company initiated a Phase 2 trial for SRK-439 in May 2024, with results anticipated in Q2 2025.

Scholar Rock's financial strategy includes a follow-on offering completed in October 2024, which is expected to provide approximately $324.4 million in net proceeds, aimed at funding operations through at least Q4 2026. The company anticipates continued significant operating losses as it advances its clinical programs.