Scholar Rock submits FDA application for SMA treatment

CAMBRIDGE, Mass. - Scholar Rock (NASDAQ: SRRK), a biopharmaceutical company with a market capitalization of $3.96 billion and an impressive 362.73% stock price surge over the past six months, announced the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for apitegromab. According to InvestingPro analysis, the company maintains a strong liquidity position with a current ratio of 3.88, though it currently appears overvalued based on Fair Value calculations. This muscle-targeted therapy is designed to improve motor function for people with spinal muscular atrophy (SMA) who are concurrently receiving SMN-targeted treatments.

The BLA for apitegromab is underpinned by results from the Phase 3 SAPPHIRE trial, which demonstrated a statistically significant 1.8-point improvement in motor function in patients receiving the treatment over those given a placebo. With the company’s next earnings report due on March 11, 2025, and analyst price targets ranging from $39 to $57, InvestingPro subscribers can access 13 additional key insights about Scholar Rock’s financial health and market position through the comprehensive Pro Research Report. This was measured by the Hammersmith Functional Motor Scale-Expanded at week 52.

The FDA has previously awarded apitegromab Fast Track, Orphan Drug, and Rare Pediatric Disease designations. Scholar Rock is also on track to submit a Marketing Authorisation Application to the European Medicines Agency in the first quarter of 2025, supported by PRIME and Orphan Medicinal Product designations already granted by the EMA.

In addition to the SAPPHIRE trial, the BLA submission includes data from the Phase 2 TOPAZ trial. Further details will be presented at the MDA Clinical & Scientific Conference in March 2025.

Scholar Rock has requested Priority Review for apitegromab, which, if granted, could reduce the FDA review period to six months. The company also plans to initiate the Phase 2 OPAL trial in mid-2025 to study apitegromab in SMA patients under two years of age.

Apitegromab is a fully human monoclonal antibody that inhibits myostatin activation by selectively binding to its pro- and latent forms in skeletal muscle. It is the first muscle-targeted therapy to show a clinically meaningful and statistically significant improvement in motor function in a pivotal Phase 3 trial for SMA. For investors following this development, InvestingPro offers detailed financial analysis and real-time updates on Scholar Rock’s market performance, including exclusive access to advanced metrics and expert insights through their Pro Research Reports available for over 1,400 US stocks.

This announcement is based on a press release statement from Scholar Rock. The company is known for its work in developing therapies for diseases with high unmet need, leveraging its proprietary platform to create selective monoclonal antibodies that modulate protein growth factors. Apitegromab has not yet been approved by the FDA or any other regulatory agency.

In other recent news, Scholar Rock, a biopharmaceutical company, has been making significant strides in the development of its potential treatment for spinal muscular atrophy (SMA), apitegromab. The company plans to submit regulatory applications in the first quarter of 2025, with a U.S. launch anticipated in the fourth quarter of the same year. This comes after the successful Phase 3 SAPPHIRE study, which demonstrated significant improvements in motor function among SMA patients.

Furthermore, Scholar Rock is expanding its efforts beyond SMA. The company is advancing its cardiometabolic program, with top-line data from the Phase 2 EMBRAZE trial in obesity expected in the second quarter of 2025. Another candidate, SRK-439, also targeting obesity, is on track for an Investigational New Drug (IND) application filing in the third quarter of 2025.

In terms of analyst coverage, H.C. Wainwright, Truist Securities, and BMO Capital Markets have all increased their price targets for Scholar Rock. These adjustments are based on the anticipated market performance and potential of apitegromab, with peak sales estimates ranging from $1.12 billion to $1.5 billion.

Finally, Scholar Rock’s financial position remains robust, with sufficient funds to support operations and capital expenditure requirements into the fourth quarter of 2026. The company maintains a healthy current ratio of 3.88, indicating strong liquidity, while operating with moderate debt levels. These recent developments highlight Scholar Rock’s consistent efforts to address unmet needs in the medical field.

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