Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on developing and commercializing apitegromab for patients with spinal muscular atrophy (SMA) and other severe and debilitating neuromuscular diseases, today reported financial results and updates for the first quarter ended March 31, 2025.
“Scholar Rock is at an inflection point as we evolve to a commercial-stage biopharmaceutical company, and our team remains focused on key priorities in preparation for the potential U.S. launch of apitegromab in Q3 2025,” said David L. Hallal, Chief Executive Officer of Scholar Rock. “With the FDA granting our BLA priority review and review of the MAA underway in Europe, I am delighted we have bolstered our leadership with the appointments of Akshay Vaishnaw, Keith Woods and Vikas Sinha as we scale our operations to serve patients with SMA in the U.S., Europe and in many additional countries around the world.”
Company Highlights and Upcoming Milestones
Spinal Muscular Atrophy (SMA) Program
Apitegromab is an investigational monoclonal antibody that inhibits myostatin activation by selectively binding the pro- and latent forms of myostatin in skeletal muscle. Apitegromab is the only muscle-targeted therapeutic candidate for patients with SMA to demonstrate statistically significant improvement and clinically meaningful benefit in a pivotal Phase 3 (SAPPHIRE) clinical trial.
Apitegromab in Additional Rare, Severe and Debilitating Neuromuscular Disorders
Cardiometabolic Program
Advancing Our Portfolio of Highly Innovative and Selective Latent Myostatin Inhibitors
SRK-439 is a novel, investigational, preclinical, subcutaneous myostatin inhibitor that binds to pro- and latent myostatin with high affinity and is selective for myostatin (i.e., no GDF11 or Activin A binding). Based on preclinical data, SRK-439 has the potential to potently inhibit myostatin and increase muscle mass.
Corporate
First Quarter 2025 Financial Results
For the quarter ended March 31, 2025, net loss was $74.7 million or $0.67 per share compared to a net loss of $56.9 million or $0.59 per share for the quarter ended March 31, 2024.
Conference Call Information
Management will provide an update on the Company and discuss first quarter 2025 results via conference call on Wednesday, May 14 at 8:15 am ET. To access the live conference call, participants may register here. The live audio webcast of the call will be available under “Events and Presentations” in the Investor Relations section of the Scholar Rock website at http://investors.scholarrock.com. To participate via telephone, please register in advance here. Upon registration, all telephone participants will receive a confirmation email detailing how to join the conference call, including the dial-in number along with a unique passcode and registrant ID that can be used to access the call. An archived replay of the webcast will be available on the Company’s website for approximately 90 days.
About Scholar Rock
Scholar Rock is a biopharmaceutical company that discovers, develops, and delivers life-changing therapies for people with serious diseases that have high unmet need. As a global leader in the biology of the transforming growth factor beta (TGFβ) superfamily, the company is named for the visual resemblance of a scholar rock to protein structures. Over the past decade, Scholar Rock has created a pipeline with the potential to advance the standard of care for neuromuscular disease, cardiometabolic disorders, cancer, and other conditions where growth factor-targeted drugs can play a transformational role.
This commitment to unlocking fundamentally different therapeutic approaches is powered by broad application of a proprietary platform, which has developed novel monoclonal antibodies to modulate protein growth factors with extraordinary selectivity. By harnessing cutting-edge science in disease spaces that are historically under-addressed through traditional therapies, Scholar Rock works every day to create new possibilities for patients. Learn more about our approach at ScholarRock.com and follow @ScholarRock and on LinkedIn.
The efficacy and safety of apitegromab, SRK-181, and SRK-439 have not been established and apitegromab, SRK-181, and SRK-439 have not been approved for any use by the FDA or any other regulatory agency.
Scholar Rock® is a registered trademark of Scholar Rock, Inc.
Availability of Other Information About Scholar Rock
Investors and others should note that we communicate with our investors and the public using our company website www.scholarrock.com, including, but not limited to, company disclosures, investor presentations and FAQs, Securities and Exchange Commission filings, press releases, public conference call transcripts and webcast transcripts, as well as on X (formerly known as Twitter) and LinkedIn. The information that we post on our website or on X (formerly known as Twitter) or LinkedIn could be deemed to be material information. As a result, we encourage investors, the media and others interested to review the information that we post there on a regular basis. The contents of our website or social media shall not be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended.
Forward-Looking Statements
This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding Scholar Rock’s future expectations, plans and prospects, including without limitation, Scholar Rock’s expectations regarding its growth, strategy, progress and timing of its clinical trials for apitegromab and its preclinical programs, including SRK-439, and indication selection and development timing, including the timing of any regulatory submissions, the therapeutic potential, clinical benefits and safety of any product candidates, expectations regarding timing, success and data announcements of current ongoing preclinical and clinical trials, its cash runway, expectations relating to commercial launch timing in the US and in Europe, expectations regarding the achievement of important milestones, the ability of any product candidate to perform in humans in a manner consistent with earlier nonclinical, preclinical or clinical trial data, and the potential of its product candidates and proprietary platform. The use of words such as “may,” “might,” “could,” “will,” “should,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “project,” “intend,” “future,” “potential,” or “continue,” and other similar expressions are intended to identify such forward-looking statements. All such forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, without limitation, that preclinical and clinical data, including whether the results from the Phase 3 SAPPHIRE trial will be sufficient to support regulatory approval, that the full results from the Phase 3 SAPPHIRE trial may differ from the topline data, that preclinical and clinical data, including the results from the Phase 2 or Phase 3 clinical trial of apitegromab, or Part A or Part B of the Phase 1 clinical trial of SRK-181, are not predictive of, may be inconsistent with, or more favorable than, data generated from future or ongoing clinical trials of the same product candidates; Scholar Rock’s ability to manage expenses or provide the financial support, resources and expertise necessary to identify and develop product candidates on the expected timeline; information provided or decisions made by regulatory authorities; competition from third parties that are developing products for similar uses; Scholar Rock’s ability to obtain, maintain and protect its intellectual property; and Scholar Rock’s dependence on third parties for development and manufacture of product candidates including, without limitation, to supply any clinical trials as well as those risks more fully discussed in the section entitled "Risk Factors" in Scholar Rock’s Quarterly Report on Form 10-Q for the quarter ended March 31, 2025, as well as discussions of potential risks, uncertainties, and other important factors in Scholar Rock’s subsequent filings with the Securities and Exchange Commission. Any forward-looking statements represent Scholar Rock’s views only as of today and should not be relied upon as representing its views as of any subsequent date. All information in this press release is as of the date of the release, and Scholar Rock undertakes no duty to update this information unless required by law.
Financial Statements
| Scholar Rock Holding Corporation | |||||||
| Condensed Consolidated Statements of Operations | |||||||
| (unaudited) | |||||||
| (in thousands, except share and per share data) | |||||||
| Three Months Ended March 31, | |||||||
|
2025 |
2024 |
||||||
| Operating expenses | |||||||
| Research and development |
$ |
48,678 |
|
$ |
43,094 |
|
|
| General and administrative |
|
28,412 |
|
|
15,325 |
|
|
| Total operating expenses |
|
77,090 |
|
|
58,419 |
|
|
| Loss from operations |
|
(77,090 |
) |
|
(58,419 |
) |
|
| Other income (expense), net |
|
2,367 |
|
|
1,566 |
|
|
| Net loss |
$ |
(74,723 |
) |
$ |
(56,853 |
) |
|
| Net loss per share, basic and diluted |
$ |
(0.67 |
) |
$ |
(0.59 |
) |
|
| Weighted average common shares outstanding, basic and diluted |
|
111,838,272 |
|
|
95,892,601 |
|
|
|
Scholar Rock Holding Corporation |
|||||
|
Condensed Consolidated Balance Sheets |
|||||
|
(unaudited) |
|||||
|
(in thousands) |
|||||
| March 31, 2025 | December 31, 2024 | ||||
| Assets | |||||
| Cash, cash equivalents and marketable securities |
$ |
364,375 |
$ |
437,278 |
|
| Other current assets |
|
20,872 |
|
13,887 |
|
| Total current assets |
|
385,247 |
|
451,165 |
|
| Other assets |
|
22,306 |
|
23,757 |
|
| Total assets |
$ |
407,553 |
$ |
474,922 |
|
| Liabilities and Stockholders' Equity | |||||
| Current liabilities |
$ |
37,574 |
$ |
46,936 |
|
| Long-term liabilities |
|
57,646 |
|
59,352 |
|
| Total liabilities |
|
95,220 |
|
106,288 |
|
| Total stockholders' equity |
|
312,333 |
|
368,634 |
|
| Total liabilities and stockholders' equity |
$ |
407,553 |
$ |
474,922 |
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