Scholar Rock NASDAQ: SRRK said its resubmitted biologics license application for apitegromab remains on track for a U.S. Food and Drug Administration decision by its Sept. 30 PDUFA date, as the company prepares for a potential launch of the muscle-targeted treatment for spinal muscular atrophy, or SMA.
The company’s application includes two fill-finish manufacturing facilities, providing what management described as independent potential paths to approval. The original approvability issue cited in a complete response letter last September concerned observations from a routine inspection at the Catalent Indiana fill-finish facility, which is owned and operated by Novo Nordisk.
Scholar Rock resubmitted its application on March 30 after discussions with the FDA, adding a second fill-finish facility. The application was accepted in April. The FDA’s inspection classification from an April 2026 reinspection of the Catalent Indiana facility remains pending.
Two Potential Manufacturing Paths
Chairman and Chief Executive Officer David Hallal said the company and FDA agreed during a March Type C meeting that including two fill-finish sites would offer the fastest potential route to approval. The company said it has submitted the data package needed for FDA review of the second facility and that the review is progressing.
“We have significant optionality with two independent paths to approval, either through Catalent, Indiana or through our second fill-finish facility or both,” Hallal said.
Hallal said Scholar Rock now has more apitegromab vials available from the second facility than from Catalent Indiana. Vials from both sites are at a third-party provider awaiting packaging and labeling pending approval.
During the question-and-answer session, Hallal said the second facility could support a fully independent approval path. If the FDA were to maintain the Catalent Indiana site’s current official action indicated, or OAI, status, Scholar Rock said it could remove the site from its application after receiving an agency signal, without expecting an effect on the Sept. 30 review timeline.
Akshay Vaishnaw, Scholar Rock’s president of research and development, said the company sees no reason the FDA could not complete its review by the PDUFA date. The resubmission included new information relating to the second facility and an updated safety database, according to management.
European Review and Commercial Preparation
In Europe, Scholar Rock’s marketing authorization application currently lists Catalent Indiana as its sole fill-finish facility. The European Medicines Agency is awaiting the FDA’s inspection classification for that site, according to the company.
Scholar Rock is also discussing with European regulators how its second fill-finish facility could be included in the application. Hallal said the facility has recently undergone successful FDA and EMA inspections and is in good standing with European regulators. The company did not provide a timetable for a Committee for Medicinal Products for Human Use opinion, saying it would update investors after further alignment with regulators.
The company is preparing for an initial European launch in Germany, followed by other countries as it establishes a planned 50-country operating platform. Management said its ambition is to reach the estimated 35,000 people globally with SMA who have received an SMN-targeted therapy.
Chief Operating Officer Keith Woods said the company’s U.S. commercial organization is ready to launch upon FDA approval. Scholar Rock is engaging approximately 140 SMA treatment centers, 2,600 prescribing physicians and multidisciplinary care teams, he said.
The company has also trained its Scholar Rock Supports patient-services team to assist eligible patients and families with insurance coverage, financial and copay assistance, and treatment logistics. Woods said the company expects prior authorization requirements for apitegromab, similar to those used for existing SMN-targeted SMA treatments.
He said patients in the company’s early-access program would be among the first considered for a transition to commercial product, followed by participants in its open-label extension and ONYX programs. However, Woods noted that access may take time, particularly while the company lacks a J-code and payers establish policies. He said that in comparable launches, the average time between prescription and infusion can exceed 60 days during the first six months.
Pipeline Expands Into FSHD
Scholar Rock said enrollment remains robust in its Phase 2 OPAL trial of apitegromab in infants and toddlers with SMA under age two. The study includes participants who have received an SMN1-targeted gene therapy or are receiving an SMN2-targeted treatment.
The company also announced initiation of FORGE, a randomized, double-blind, placebo-controlled Phase 2 trial of apitegromab in facioscapulohumeral muscular dystrophy, or FSHD. The trial is planned to enroll 60 patients.
Vaishnaw said Scholar Rock selected FSHD because of the absence of approved treatments, the disease’s impact on function, and preclinical and clinical evidence suggesting that gains in muscle mass may translate into functional benefits. The study will focus on mild-to-moderate patients, including those with Ricci scores between 1.5 and 3, he said.
Scholar Rock is also advancing a high-concentration subcutaneous formulation of apitegromab. The company plans to engage U.S. and European regulators on its development path after apitegromab is approved. Management said Phase 1 data presented in January showed favorable bioavailability and a pharmacodynamic profile comparable with intravenous administration.
For SRK-439, a high-potency subcutaneous anti-myostatin antibody, the company said dosing is progressing in a Phase 1 healthy-volunteer study. Top-line data are expected later this year.
Second-Quarter Financial Position
Scholar Rock reported second-quarter operating expenses of $108.9 million, including $19.7 million in non-cash stock-based compensation. Excluding stock-based compensation, operating expenses were $89.2 million.
The company ended the quarter with $492 million in cash equivalents and marketable securities, including $63 million in net proceeds from its at-the-market equity program during the quarter.
Chief Financial Officer Vikas Sinha said Scholar Rock could draw an additional $150 million from its existing debt facility upon FDA approval of apitegromab. The company also plans to monetize a priority review voucher to further strengthen its balance sheet.
Management said its 2026 financial priorities include supporting a U.S. and European commercial launch, advancing its clinical pipeline, strengthening its supply chain, and maintaining financial flexibility.
About Scholar Rock (NASDAQ:SRRK)
Scholar Rock is a clinical-stage biotechnology company focused on the discovery and development of protein therapeutics that selectively target growth factors involved in disease processes. The company's research platform is designed to modulate endogenous growth factor activation and signaling with high specificity, aiming to restore normal biological function across a range of disorders. Scholar Rock's approach is distinguished by its emphasis on engineering antibodies and biologics that interact with growth factor precursors or latent complexes rather than the active form, potentially offering improved safety and efficacy profiles.
The company's lead program, SRK-015 (appercept), is an investigational monoclonal antibody targeting the activation of latent myostatin proproteins and is being evaluated for the treatment of spinal muscular atrophy (SMA).
This instant news alert was generated by narrative science technology and financial data from MarketBeat in order to provide readers with the fastest reporting and unbiased coverage. Please send any questions or comments about this story to contact@marketbeat.com.
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