Vice President - Head of Regulatory Strategy

Scholar Rock (NASDAQ:SRRK) shares up 5.8% - still a Buy? August 26, 2026 Key points. * Scholar Rock shares rose 5.8% to about $61.62, lifting the company's market capitalization to approximately $7.42 billion. * Analyst sentiment remains broadly positive: the stock has an average "Buy" rating, with 15 of 16 analysts rating it Buy or Strong Buy. However, the consensus price target of $58.77 is below the current share price. * The company reported quarterly EPS of negative $0.84, in line with estimates, while institutional investors own a substantial 91.08% of outstanding shares. Scholar Rock is developing protein therapeutics, including its lead SMA drug candidate, appercept. * MarketBeat previews top five stocks to own in September. Scholar Rock Holding Corporation (NASDAQ:SRRK - Get Free Report)'s share price was up 5.8% on Wednesday. The company traded as high as $61.28 and last traded at $61.6150. Approximately 1,244,564 shares traded hands during mid-day trading, a decline of 6% from the average session volume of 1,327,546 shares. The stock had previously closed at $58.25. Analysts set new price targets. SRRK has been the subject of a number of analyst reports. BMO Capital Markets reaffirmed an "outperform" rating and issued a $76.00 price target on shares of Scholar Rock in a report on Friday, August 7th. Weiss Ratings reaffirmed a "sell (d-)" rating on shares of Scholar Rock in a report on Friday, July 17th. Cantor Fitzgerald restated an "overweight" rating on shares of Scholar Rock in a research report on Thursday, June 18th. JPMorgan Chase & Co. upped their target price on Scholar Rock from $56.00 to $63.00 and gave the stock an "overweight" rating in a research note on Thursday, August 13th. Finally, Citigroup lifted their price target on Scholar Rock from $58.00 to $60.00 and gave the company a "buy" rating in a research report on Friday, August 7th. Four research analysts have rated the stock with a Strong Buy rating, eleven have assigned a Buy rating and one has given a Sell rating to the company's stock. Based on data from MarketBeat.com, the company currently has an average rating of "Buy" and a consensus target price of $58.77. Scholar Rock trading up 4.6%. The stock has a 50 day moving average of $52.36 and a 200 day moving average of $48.64. The company has a debt-to-equity ratio of 0.77, a current ratio of 7.02 and a quick ratio of 7.02. The firm has a market cap of $7.42 billion, a P/E ratio of -17.67 and a beta of 0.69. Scholar Rock (NASDAQ:SRRK - Get Free Report) last announced its quarterly earnings data on Thursday, August 6th. The company reported ($0.84) EPS for the quarter, meeting the consensus estimate of ($0.84). During the same period in the prior year, the business earned ($0.98) EPS. As a group, equities research analysts predict that Scholar Rock Holding Corporation will post -3.23 EPS for the current year. Insider activity at Scholar Rock. In other Scholar Rock news, Director Kristina Burow sold 3,053 shares of the business's stock in a transaction on Wednesday, July 1st. The shares were sold at an average price of $53.74, for a total value of $164,068.22. Following the completion of the sale, the director directly owned 114,657 shares of the company's stock, valued at $6,161,667.18. The trade was a 2.59% decrease in their ownership of the stock. The transaction was disclosed in a document filed with the SEC, which is available at the SEC website. The sale was made to cover tax withholding obligations related to the vesting of equity awards. Also, Director Jeffrey S. Flier sold 3,053 shares of the firm's stock in a transaction dated Wednesday, July 1st. The shares were sold at an average price of $53.88, for a total value of $164,495.64. Following the transaction, the director directly owned 27,856 shares in the company, valued at $1,500,881.28. This trade represents a 9.88% decrease in their ownership of the stock. The SEC filing for this sale provides additional information. The sale was made to cover tax withholding obligations related to the vesting of equity awards. Insiders have sold 17,149 shares of company stock worth $923,108 in the last 90 days. Corporate insiders own 10.30% of the company's stock. Institutional inflows and outflows. A number of hedge funds have recently bought and sold shares of the business. Jennison Associates LLC boosted its position in shares of Scholar Rock by 74.0% during the fourth quarter. Jennison Associates LLC now owns 2,795,188 shares of the company's stock valued at $123,128,000 after purchasing an additional 1,188,676 shares in the last quarter. Nicholas Investment Partners LP bought a new position in Scholar Rock in the second quarter worth $4,968,000. iA Global Asset Management Inc. purchased a new position in Scholar Rock during the fourth quarter valued at $5,430,000. Geode Capital Management LLC boosted its holdings in shares of Scholar Rock by 4.1% during the 4th quarter. Geode Capital Management LLC now owns 2,034,024 shares of the company's stock valued at $89,616,000 after buying an additional 79,214 shares in the last quarter. Finally, Norges Bank purchased a new stake in shares of Scholar Rock in the 4th quarter worth about $91,603,000. Hedge funds and other institutional investors own 91.08% of the company's stock. Scholar Rock company profile. 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. Discover more Stock Split Calculator 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 [email protected]. Continue following MarketBeat Before you consider Scholar Rock, you'll want to hear this. MarketBeat keeps track of Wall Street's top-rated and best performing research analysts and the stocks they recommend to their clients on a daily basis. MarketBeat has identified the five stocks that top analysts are quietly whispering to their clients to buy now before the broader market catches on... and Scholar Rock wasn't on the list. While Scholar Rock currently has a Buy rating among analysts, top-rated analysts believe these five stocks are better buys. MarketBeat just released its list of the 7 hottest IPOs expected to hit Wall Street in 2026. See which companies are preparing to go public and why investors are watching closely.

SMA therapy stays on track for FDA decision after manufacturing change. Scholar Rock withdraws Europe filing, plans Japan submission by year-end * Scholar Rock removed a flagged manufacturing site from apitegromab's FDA application and still expects a decision by Sept. 30. * Apitegromab targets myostatin to strengthen muscle and improve motor function alongside existing SMA treatments. * Scholar Rock withdrew its European application, while Japan agreed no additional clinical studies are required for the planned submission. Scholar Rock has successfully removed a manufacturing facility from its application to the U.S. Food and Drug Administration (FDA) seeking approval of apitegromab, an experimental muscle-strengthening treatment for spinal muscular atrophy (SMA). The move follows an announcement earlier this month that the company planned to remove Catalent Indiana, part of Novo Nordisk, after the FDA classified it as Official Action Indicated (OAI), meaning significant problems were identified that may warrant regulatory action. Following FDA guidance, Scholar Rock proceeded with a second, U.S.-based fill-finish facility instead. Recommended Reading FDA decision remains on track after manufacturing change. Scholar Rock continues to expect an FDA decision by Sept. 30. The company says it is prepared to launch apitegromab in the U.S. immediately if approved, with a supply of commercial vials produced at the alternate facility. The manufacturing change is also reshaping apitegromab's regulatory path in Europe. Scholar Rock has withdrawn its Marketing Authorisation Application (MAA) from review and plans to resubmit it using the alternate fill-finish facility. "We are very pleased with the continued constructive and collaborative engagement we have with regulators both in the U.S. and Europe and look forward to commencing our US launch... pending FDA approval," David L. Hallal, Scholar Rock's board chair and CEO, said in a press release. Meanwhile, regulators in Japan have agreed that no additional clinical studies are required to support Scholar Rock's planned Japanese New Drug Application for apitegromab. Clinical studies involving Japanese patients are typically required to support such applications. However, recent guidelines allow certain rare disease medicines to be submitted without domestic clinical data under specific circumstances, including when a pivotal trial has already been successfully conducted outside Japan and recruiting enough local patients would be difficult because of the rarity of the disease. Scholar Rock plans to submit its Japanese application by year-end. "In line with our ambition to serve patients with SMA in up to 50 countries around the world, we are also thrilled to announce that we now have alignment with [Japan's Pharmaceuticals and Medical Devices Agency] in Japan to submit our apitegromab [application] for the treatment of children and adults with SMA by year end. Together, our regulatory advancements reinforce our commitment to act with urgency on behalf of the SMA community globally," Hallal said. Apitegromab takes a different approach to treating SMA. SMA is a genetic disease characterized by symptoms such as muscle weakness and wasting. While existing disease-modifying treatments target the underlying shortage of survival motor neuron (SMN) protein, which is essential for the health of nerve cells that control movement, apitegromab is being developed through a different approach. The antibody-based therapy is designed to selectively block the activation of myostatin, a protein that normally limits muscle growth. By targeting the inactive, or pro- and latent, forms of myostatin before the protein becomes active, apitegromab is intended to improve muscle strength and improve motor function. Scholar Rock is developing it as a muscle-targeted treatment that could be used alongside SMN-targeting therapies. The application seeking its approval is supported mainly by data from the Phase 3 clinical trial SAPPHIRE (NCT05156320), which enrolled 188 children and young adults with SMA type 2 or type 3, who were already taking Spinraza (nusinersen) or Evrysdi (risdiplam). After about a year, apitegromab improved motor function, with Hammersmith Functional Motor Scale Expanded (HFMSE) scores averaging 1.8 points higher than with a placebo. Patricia holds her PhD in cell biology from the University Nova de Lisboa, Portugal, and has served as an author on several research projects and fellowships, as well as major grant applications for European agencies. She also served as a PhD student research assistant in the Department of Microbiology & Immunology, Columbia University, New York, for which she was awarded a Luso-American Development Foundation (FLAD) fellowship.

European review of SMA therapy awaits upcoming results from US facility check. Scholar Rock prepares a secondary manufacturing site for apitegromab * EU approval for apitegromab, an experimental SMA therapy, awaits US FDA inspection results of a manufacturing facility. * Apitegromab is an add-on therapy for SMA, shown to improve motor function. * Scholar Rock is preparing a second manufacturing facility to ensure supply and address regulatory concerns. An official decision on whether to approve apitegromab for spinal muscular atrophy (SMA) in the European Union may hinge on upcoming inspection results from U.S. regulators, biopharmaceutical company Scholar Rock has announced. The European Medicines Agency (EMA) is currently reviewing the experimental muscle-strengthening therapy. However, European authorities are awaiting an official inspection update from the U.S. Food and Drug Administration (FDA) about one of the drug's key manufacturing facilities before rendering a final opinion. "We are very pleased with the EMA's review of the apitegromab [application] and with the level of engagement we have from the Agency," David L. Hallal, board chair and CEO of Scholar Rock, said in a company press release. SMA is a neuromuscular disorder that causes progressive muscle weakness. Apitegromab is an antibody-based therapy designed to boost muscle strength by blocking the activity of myostatin, a protein that normally prevents excess muscle growth. Scholar Rock is developing apitegromab as an add-on to disease-modifying treatments that slow progression by targeting SMA's root cause, with the aim of helping patients achieve better motor function. Recommended Reading Phase 3 trial and regulatory path. A Phase 3 clinical trial called SAPPHIRE (NCT05156320) tested apitegromab against a placebo in children and young adults with SMA type 2 or type 3 who were taking the disease-modifying therapies Spinraza (nusinersen) or Evrysdi (risdiplam). The study met its main goal, demonstrating that motor function scores improved significantly in patients receiving apitegromab. Based on those positive Phase 3 results, Scholar Rock submitted an application seeking U.S. approval for apitegromab in early 2025. However, later that year, the FDA declined to approve the initial submission. The agency raised no concerns about apitegromab's safety or efficacy data, but it cited compliance issues at a third-party fill-finish facility, a specialized manufacturing center responsible for final drug packaging and product sterility. Since receiving the FDA's decision, Scholar Rock has been working with the facility, known as Catalent Indiana (part of Novo Nordisk), to address regulators' concerns. To ensure a stable medication supply, Scholar Rock also enlisted a second fill-finish facility. Scholar Rock has since resubmitted its U.S. application, and an FDA decision is expected in late September. Meanwhile, Scholar Rock is also seeking approval of apitegromab in the EU. Notably, while the company's resubmitted U.S. application details both fill-finish facilities, the European application currently lists only the Catalent Indiana site. When experimental therapies are reviewed in the EU, a key step is evaluation by the Committee for Medicinal Products for Human Use (CHMP), an arm of the EMA. The committee makes recommendations for or against approval, which are almost always followed by the European Commission, the body with final decision-making authority for EU drug approvals. Scholar Rock prepares dual-facility backup. The FDA completed an inspection of the Catalent Indiana facility in April, and according to Scholar Rock, the CHMP is withholding its recommendation on apitegromab until the FDA's inspection results are available. If the FDA gives the facility the green light, an opinion from the CHMP is expected before the end of 2026. If not, Scholar Rock said it will work with European regulators to update its European filing to incorporate the second fill-finish facility, which the company said is already in good standing with both the FDA and EMA. "We expect clarity from the FDA on the inspection classification of Catalent Indiana in the near term, and we remain confident in our ability to advance our [application for apitegromab] through the EMA review process either with Catalent Indiana or by taking the necessary steps to include our second fill-finish facility," Hallal said. Which best describes you? Marisa holds a Master of Science in cellular and molecular pathology from the University of Pittsburgh, where she studied novel genetic drivers of ovarian cancer. Her areas of expertise include cancer biology, immunology, and genetics, and she has worked as a science writing and communications intern for the Genetics Society of America.