Scholar Rock Highlights 2025 Strategic Priorities
Scholar Rock (SRRK) outlined its 2025 strategic priorities, focusing on three key areas: commercializing apitegromab in SMA, expanding its benefits, and advancing their anti-myostatin program in cardiometabolic disorders. The company plans to submit BLA and MAA applications for apitegromab in Q1 2025, with U.S. launch expected in Q4 2025.
Key upcoming milestones include initiating the Phase 2 OPAL trial in SMA patients under two years old in mid-2025, expecting data from Phase 2 EMBRAZE obesity trial in Q2 2025, and filing an IND application for SRK-439 in Q3 2025. The company reported positive topline data from the SAPPHIRE trial in October 2024, showing significant improvement in motor function for SMA patients.
Scholar Rock's financial position remains strong, with funding expected to last into Q4 2026, supported by a successful $345 million public offering completed in October.
Scholar Rock (SRRK) ha delineato le sue priorità strategiche per il 2025, concentrandosi su tre aree chiave: la commercializzazione dell'apitegromab per la SMA, l'espansione dei suoi benefici e l'avanzamento del loro programma anti-myostatina per i disturbi cardiometabolici. L'azienda prevede di presentare le domande BLA e MAA per l'apitegromab nel primo trimestre del 2025, con il lancio negli Stati Uniti previsto nel quarto trimestre del 2025.
I principali traguardi futuri includono l'avvio della sperimentazione di Fase 2 OPAL in pazienti SMA di età inferiore ai due anni a metà del 2025, l'aspettativa di dati dalla sperimentazione di Fase 2 EMBRAZE per l'obesità nel secondo trimestre del 2025 e la presentazione di una domanda IND per SRK-439 nel terzo trimestre del 2025. L'azienda ha riportato dati positivi dai risultati preliminari della sperimentazione SAPPHIRE nell'ottobre 2024, mostrando un miglioramento significativo della funzione motoria per i pazienti SMA.
La posizione finanziaria di Scholar Rock rimane solida, con i finanziamenti previsti per durare fino al quarto trimestre del 2026, supportati da una riuscita offerta pubblica di $345 milioni completata a ottobre.
Scholar Rock (SRRK) delineó sus prioridades estratégicas para 2025, enfocándose en tres áreas clave: comercializar el apitegromab en la SMA, expandir sus beneficios y avanzar en su programa anti-mioestatina para desórdenes cardiometabólicos. La compañía planea presentar solicitudes BLA y MAA para el apitegromab en el primer trimestre de 2025, con un lanzamiento en EE. UU. esperado para el cuarto trimestre de 2025.
Los hitos clave que se aproximan incluyen iniciar el ensayo Fase 2 OPAL en pacientes con SMA menores de dos años a mediados de 2025, espera de datos del ensayo de obesidad Fase 2 EMBRAZE en el segundo trimestre de 2025 y presentar una solicitud IND para SRK-439 en el tercer trimestre de 2025. La empresa reportó datos positivos de la fase preliminar del ensayo SAPPHIRE en octubre de 2024, mostrando una mejora significativa en la función motora de los pacientes con SMA.
La posición financiera de Scholar Rock sigue siendo fuerte, con fondos que se espera duren hasta el cuarto trimestre de 2026, respaldados por una exitosa oferta pública de $345 millones completada en octubre.
스칼라 록 (SRRK)은 2025년 전략적 우선 사항을 설명하며, SMA에서 아피테그로맙 상용화, 혜택 확대, 심혈관 대사 질환에 대한 항 미오스타틴 프로그램의 발전 등 세 가지 주요 영역에 집중하고 있습니다. 이 회사는 2025년 1분기 내에 아피테그로맙에 대한 BLA 및 MAA 신청서를 제출할 계획이며, 미국 출시가 2025년 4분기에 예상됩니다.
주요 향후 이정표로는 2025년 중반에 2세 미만 SMA 환자를 대상으로 Phase 2 OPAL 시험을 시작하고, 2025년 2분기에 Phase 2 EMBRAZE 비만 시험의 데이터 발표를 기대하며, 2025년 3분기에 SRK-439에 대한 IND 신청서를 제출하는 것입니다. 이 회사는 2024년 10월 SAPPHIRE 시험에서 긍정적인 톱라인 데이터를 보고했으며, SMA 환자의 운동 기능에서 상당한 개선을 보였습니다.
스칼라 록의 재무 상태는 여전히 강력하며, 2026년 4분기까지 자금이 지속될 것으로 예상되며, 10월에 완료된 3억4500만 달러 규모의 공모가 이를 지원합니다.
Scholar Rock (SRRK) a défini ses priorités stratégiques pour 2025, en se concentrant sur trois domaines clés : la commercialisation de l'apitegromab dans la SMA, l'expansion de ses avantages et l'avancement de leur programme anti-myostatine pour les troubles cardiométaboliques. L'entreprise prévoit de soumettre des demandes BLA et MAA pour l'apitegromab au premier trimestre 2025, avec un lancement attendu aux États-Unis au quatrième trimestre 2025.
Les principales étapes à venir incluent le lancement de l'essai OPAL de phase 2 chez les patients SMA de moins de deux ans à la mi-2025, l'attente de données de l'essai EMBRAZE sur l'obésité de phase 2 au deuxième trimestre 2025 et le dépôt d'une demande IND pour SRK-439 au troisième trimestre 2025. L'entreprise a rapporté des données préliminaires positives de l'essai SAPPHIRE en octobre 2024, montrant une amélioration significative de la fonction motrice chez les patients SMA.
La position financière de Scholar Rock reste solide, avec des financements prévus jusqu'au quatrième trimestre 2026, soutenus par une offre publique réussie de 345 millions de dollars réalisée en octobre.
Scholar Rock (SRRK) hat seine strategischen Prioritäten für 2025 umrissen, die sich auf drei Schlüsselbereiche fokussieren: die Kommerzialisierung von Apitegromab bei SMA, die Erweiterung seiner Vorteile und die Weiterentwicklung ihres Anti-Myostatin-Programms für kardiometabolische Erkrankungen. Das Unternehmen plant, im ersten Quartal 2025 BLA- und MAA-Anträge für Apitegromab einzureichen, mit einer erwarteten Markteinführung in den USA im vierten Quartal 2025.
Wichtige bevorstehende Meilensteine umfassen den Beginn der Phase-2-OPAL-Studie bei SMA-Patienten unter zwei Jahren Mitte 2025, die erwarteten Daten aus der Phase-2-EMBRAZE-Studie zur Fettleibigkeit im zweiten Quartal 2025 und die Einreichung eines IND-Antrags für SRK-439 im dritten Quartal 2025. Das Unternehmen berichtete im Oktober 2024 von positiven Topline-Daten aus der SAPPHIRE-Studie, die eine signifikante Verbesserung der motorischen Funktion bei SMA-Patienten zeigten.
Die finanzielle Lage von Scholar Rock bleibt stark, wobei die Finanzierung bis ins vierte Quartal 2026 reichen soll, unterstützt durch eine erfolgreiche Kapitalerhöhung in Höhe von 345 Millionen Dollar, die im Oktober abgeschlossen wurde.
- Positive SAPPHIRE trial results showing statistically significant improvement in motor function
- Strong cash position extending into Q4 2026
- Successful completion of $345 million public offering
- High patient retention with 98% of SAPPHIRE patients enrolling in ONYX extension study
- 90% of TOPAZ trial patients completed 4 years of treatment
- No commercial revenue expected until Q4 2025 at earliest
- Multiple regulatory approvals still pending
Insights
The upcoming BLA and MAA submissions for apitegromab in SMA represent a critical regulatory milestone for Scholar Rock. The positive Phase 3 SAPPHIRE trial data, showing statistically significant motor function improvements, strongly supports approval potential. With 98% patient retention in the ONYX extension study, the long-term safety profile appears favorable. The planned Q4 2025 U.S. launch could establish Scholar Rock as a key player in the
The expansion into obesity treatment through the EMBRAZE trial strategically positions the company in the rapidly growing GLP-1 market. The preclinical data for SRK-439 showing muscle preservation during GLP-1 therapy addresses a significant unmet need, as muscle loss is a major concern with current obesity treatments.
Scholar Rock's strong financial position is noteworthy, with cash runway extending into Q4 2026 following their successful
- Commercial launch preparations for apitegromab
- Phase 2 EMBRAZE trial completion
- SRK-439 development initiatives
The expansion into SMA patients under two years old through the OPAL trial represents a important market opportunity, as early intervention in SMA is known to produce better outcomes. The company's exploration of additional neuromuscular disorders like Duchenne and ALS demonstrates the broader potential of their myostatin inhibition platform. The preclinical data showing SRK-439's efficacy at doses as low as 0.3 mg/kg suggests an attractive therapeutic window, while the enhanced fat mass loss in combination with tirzepatide indicates potential synergistic benefits in obesity treatment.
- BLA and MAA submission for apitegromab in SMA on track for 1Q 2025; if approved,
- Initiating Phase 2 OPAL clinical trial in SMA patients under two years old in mid-2025
- Data from Phase 2 EMBRAZE trial evaluating apitegromab in obesity expected in 2Q 2025
- Filing of IND application for SRK-439 on track for 3Q 2025
- Presenting at the 43rd Annual J.P. Morgan Healthcare Conference on Tuesday, January 14, 2025 at 9:45 a.m. PT (12:45 p.m. ET)
“We are excited about the potential for apitegromab to help those living with SMA as the first and only muscle-targeted therapy for the treatment of this condition and are making excellent progress in finalizing our regulatory applications and preparing for commercial launch,” said Jay Backstrom, M.D., MPH, President and Chief Executive Officer of Scholar Rock. “We remain on track for BLA and MAA submission in the first quarter of 2025 and are laser focused on bringing apitegromab to people with SMA as soon as possible. Building on the success of our leading, highly selective anti-myostatin approach in SMA, we plan to expand development of apitegromab into additional rare neuromuscular disorders to serve more people with unmet medical needs and as the foundation of a growing neuromuscular franchise.”
Dr. Backstrom continued, “Additionally, we are making excellent progress to deliver value across our pipeline, including the next wave of innovation in cardiometabolic disorders. We look forward to reporting topline data from our Phase 2 EMBRAZE proof-of-concept trial of apitegromab in combination with a GLP-1 receptor agonist in obesity and submitting an IND for SRK-439, our highly selective myostatin inhibitor specifically designed for obesity and cardiometabolic disorders.”
2025 Strategic Priorities and Upcoming Milestones:
Scholar Rock plans to focus on three strategic priorities in 2025 as it transitions into a commercial-stage biopharmaceutical company, creating sustainable long-term value opportunities:
- Commercialize apitegromab in SMA.
- Expand the potential benefit of apitegromab.
- Advance Scholar Rock’s anti-myostatin program in cardiometabolic disorders.
Commercialize apitegromab in SMA
- On track to submit a biologics license application (BLA) to the FDA and a marketing authorisation application (MAA) to the European Medicines Agency in 1Q 2025 for apitegromab in SMA.
-
Preparing for
U.S. commercial launch in 4Q 2025, and European launch to follow. - Presenting detailed results from the pivotal Phase 3 SAPPHIRE trial at upcoming medical meetings in 2025.
Expand the potential benefit of apitegromab
- Initiating Phase 2 OPAL clinical trial in SMA patients under two years of age in mid-2025. The trial will evaluate apitegromab in patients under two years of age who have been or are continuing to be treated with any currently approved SMN therapy, including onasemnogene abeparvovec.
- Building upon the positive Phase 3 SAPPHIRE trial, the Company is exploring development of apitegromab in other rare neuromuscular disorders such as Duchenne muscular dystrophy, Becker muscular dystrophy, Facioscapulohumeral muscular dystrophy and amyotrophic lateral sclerosis to build a growing neuromuscular franchise.
Advance the anti-myostatin program in cardiometabolic disorders
- Topline data expected in the second quarter of 2025 from the Phase 2 EMBRAZE proof-of-concept trial, a randomized, double-blind, placebo-controlled, multi-center study evaluating the safety and efficacy of apitegromab, a highly selective investigational myostatin inhibitor, to preserve muscle mass in overweight and obese adults who are taking a GLP-1 receptor agonist (GLP-1 RA). Trial outcomes will be used to guide clinical development of SRK-439.
- Plan to file an IND for SRK-439 for the treatment of obesity on the background of standard of care GLP-1 RAs in 3Q 2025.
- Scholar Rock disclosed new preclinical data which showed that SRK-439 protected against tirzepatide-induced muscle loss in DIO mice. Lean mass preservation was seen with doses of SRK-439 as low as 0.3 mg/kg and lean mass gain was observed at higher doses. The addition of SRK-439 with tirzepatide improved overall body composition with dose-dependent enhancement of fat mass loss.
Scholar Rock believes that its existing cash, cash equivalents and marketable securities will be sufficient to fund the Company’s anticipated operating expenses and capital expenditure requirements into the fourth quarter of 2026.
“Scholar Rock is funded to scale up and drive forward our key priorities in 2025, including the commercialization of apitegromab, the EMBRAZE readout, the initiation of clinical development for SRK-439, and growing our pipeline,” said Ted Myles, Chief Operating Officer and Chief Financial Officer of Scholar Rock. “We believe in the transformative potential of our highly selective muscle-targeted approach and will advance our programs as we prepare to transition into a commercial company.”
2024 Highlights and Accomplishments:
- Reported positive topline data from the pivotal SAPPHIRE trial in October 2024. The trial achieved its primary endpoint, demonstrating a statistically significant and clinically meaningful improvement for apitegromab versus placebo in motor function as measured by the Hammersmith Functional Motor Scale – Expanded (HFMSE) in SMA patients on chronic dosing of standard of care therapies (either nusinersen or risdiplam).
- Continued high participation in the ONYX open-label extension study evaluating the long-term safety and efficacy of apitegromab in patients who completed the TOPAZ or SAPPHIRE trials. More than 90 percent of patients on combination therapy in the TOPAZ trial have completed 4 years of apitegromab treatment and enrolled into ONYX. Following trial completion, 98 percent of SAPPHIRE patients (185/188) enrolled in the ongoing ONYX open-label expansion study.
- Initiated Phase 2 EMBRAZE proof-of-concept trial with apitegromab in combination with a GLP-1 receptor agonist (GLP-1 RA) in obesity in May and completed enrollment in September.
- Presented new SRK-439 preclinical data at multiple conferences: ObesityWeek, American Diabetes Association’s 84th Scientific Sessions (ADA) and Keystone Symposia’s Obesity: Causes and Consequences meeting.
-
Successfully completed upsized
$345 million
J.P. Morgan Healthcare Conference Presentation and Webcast
Scholar Rock management will highlight these updates in a corporate presentation at the 43rd Annual J.P. Morgan Healthcare Conference on Tuesday, January 14, 2025, at 9:45 a.m. PT (12:45 p.m. ET). A live webcast of the presentation may be accessed by visiting the Investors & Media section of the Scholar Rock website at http://investors.scholarrock.com. An archived replay of the webcast will be available on the Company’s website for approximately 90 days following the presentation.
About Apitegromab
Apitegromab is an investigational fully human monoclonal antibody inhibiting myostatin activation by selectively binding the pro- and latent forms of myostatin in the skeletal muscle. It is the first muscle-targeted treatment candidate to demonstrate clinical proof-of-concept in spinal muscular atrophy (SMA). Myostatin, a member of the TGFβ superfamily of growth factors, is expressed primarily by skeletal muscle cells, and the absence of its gene is associated with an increase in muscle mass and strength in multiple animal species, including humans. Scholar Rock believes that its highly selective targeting of pro- and latent forms of myostatin with apitegromab may lead to a clinically meaningful improvement in motor function in patients with SMA. The
About the Phase 3 SAPPHIRE Trial
SAPPHIRE was a randomized, double-blind, placebo-controlled Phase 3 clinical trial that evaluated the safety and efficacy of apitegromab in nonambulatory patients with Types 2 and 3 SMA who are receiving current standard of care (either nusinersen or risdiplam). SAPPHIRE enrolled 156 patients aged 2-12 years old in the main efficacy population. These patients were randomized 1:1:1 to receive for 12 months either apitegromab 10 mg/kg, apitegromab 20 mg/kg, or placebo by intravenous (IV) infusion every 4 weeks. An exploratory population that enrolled 32 patients aged 13-21 years old was also evaluated. These patients were randomized 2:1 to receive either apitegromab 20 mg/kg or placebo.
About EMBRAZE
EMBRAZE is a randomized, double-blind, placebo-controlled, Phase 2 proof-of-concept trial evaluating the efficacy, safety and pharmacokinetics of apitegromab in adults with a body mass index (BMI) of >27 (overweight) or a BMI of >30 (obese) and taking a GLP-1 RA (tirzepatide or semaglutide). The target enrollment of EMBRAZE is 100 subjects aged 18-65 who are overweight or obese without diabetes. As part of the study design, the treatment period is 24 weeks, and all subjects will receive a GLP-1 RA. In addition, all subjects will be randomized 1:1 to receive either apitegromab or placebo by intravenous (IV) infusion every four weeks during the 24-week treatment period. The primary endpoint is change from baseline at Week 24 in lean mass assessed by dual-energy X-ray absorptiometry. Secondary endpoints include additional weight loss measures, safety and tolerability, and pharmacokinetic outcomes. Exploratory endpoints at Weeks 24 and 32 include cardiometabolic parameters (e.g. HbA1c), body composition, and physical function.
About SRK-439
SRK-439 is a novel, preclinical, investigational myostatin inhibitor that has high in vitro affinity for pro- and latent myostatin and maintains myostatin specificity (i.e., no GDF11 or Activin-A binding), and is initially being developed for the treatment of cardiometabolic disorders, including obesity. Based on preclinical data, SRK-439 has the potential to support healthier weight management by preserving lean mass during weight loss. The efficacy and safety of SRK-439 have not been established and SRK-439 has not been approved for any use by the FDA or any other regulatory agency.
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 and named for the visual resemblance of a scholar rock to protein structures, the clinical-stage company is focused on advancing innovative treatments where protein growth factors are fundamental. 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 Twitter and LinkedIn. The information that we post on our website or on 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, results, its product candidate selection and development timing, including timing for the initiation of and reporting results from its preclinical studies and clinical trials for apitegromab, SRK-439, linavonkibart and other product candidates and indication selection and development timing, its cash runway, 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,” “could,” “might,” “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 for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995. 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 the results from the Phase 3 trial of apitegromab or Part A or Part B of the Phase 1 trial of linavonkibart, are not predictive of, may be inconsistent with, or more favorable than, data generated from future or ongoing clinical trials of the same product candidate; Scholar Rock’s ability to provide the financial support, resources and expertise necessary to identify and develop product candidates on the expected timeline; the data generated from Scholar Rock’s nonclinical and preclinical studies and clinical trials, including from the EMBRAZE clinical trial; 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; the success of Scholar Rock’s current and potential future collaborations; Scholar Rock’s dependence on third parties for development and manufacture of product candidates including, without limitation, to supply any clinical trials; Scholar Rock’s ability to manage expenses and to obtain additional funding when needed to support its business activities; its ability to establish or maintain strategic business alliances; its ability to receive priority or expedited regulatory review or to obtain regulatory approval of apitegromab; its ability to expand globally and the anticipated commercial launch in
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Scholar Rock:
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ir@scholarrock.com
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Scholar Rock
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