logo
#

Latest news with #ScholarRock

Spinal Muscular Atrophy Pipeline Drugs Analysis 2025: Clinical Trials, EMA, PDMA, FDA Approvals, Medication, Treatment Market, NDA Approval, Therapies, ROA, MOA and Companies by DelveInsight
Spinal Muscular Atrophy Pipeline Drugs Analysis 2025: Clinical Trials, EMA, PDMA, FDA Approvals, Medication, Treatment Market, NDA Approval, Therapies, ROA, MOA and Companies by DelveInsight

Globe and Mail

time2 days ago

  • Business
  • Globe and Mail

Spinal Muscular Atrophy Pipeline Drugs Analysis 2025: Clinical Trials, EMA, PDMA, FDA Approvals, Medication, Treatment Market, NDA Approval, Therapies, ROA, MOA and Companies by DelveInsight

"Spinal Muscular Atrophy Pipeline" Spinal Muscular Atrophy companies are Amniotics, Biohaven Pharmaceuticals, Hoffmann-La Roche, Amylon Therapeutics, Voyager Therapeutics, Catalyst Pharmaceuticals, CANbridge Pharmaceuticals Inc., Astellas Pharma, Alcyone Therapeutics, Exicure, AndroScience Corporation, Scholar Rock, NMD Pharma, Aurimed Pharma, Biogen, Hanugen Theraputics, and others. (Albany, USA) 'Spinal Muscular Atrophy Pipeline Insight, 2025″ report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the Spinal Muscular Atrophy Market. The Spinal Muscular Atrophy clinical trials report embraces in-depth commercial and clinical assessment of the pipeline products from the pre-clinical developmental phase to the marketed phase. The report also covers a detailed description of the drug, including the mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, collaborations, mergers acquisition, funding, designations, and other product-related details. Spinal Muscular Atrophy pipeline constitutes 18+ key companies continuously working towards developing 20+ Spinal Muscular Atrophy treatment therapies, analysis of Clinical Trials, Therapies, Mechanism of Action, Route of Administration, and Developments analyzes DelveInsight. Some of the key takeaways from the Spinal Muscular Atrophy Pipeline Report: In March 2025, Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on advancing innovative treatments for neuromuscular diseases, cardiometabolic disorders, and other serious diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted its Biologics License Application (BLA) for apitegromab, an investigational muscle-targeted treatment that is being developed to provide clinically meaningful improvement in motor function for people living with spinal muscular atrophy (SMA) who are receiving an SMN-targeted treatment. The FDA will review the application under priority review and has assigned a Prescription Drug User Fee Act (PDUFA) target action date of September 22, 2025. The FDA priority review designation conveys that the FDA has determined that if apitegromab is approved, it could offer significant improvement in the safety or effectiveness of treatment of the serious condition of SMA Spinal Muscular Atrophy Companies across the globe are diligently working toward developing novel Spinal Muscular Atrophy treatment therapies with a considerable amount of success over the years. Leading Spinal Muscular Atrophy companies working in the market are Scholar Rock, Biogen, Astellas Pharma, Alcyone Therapeutics, AndroScience Corporation, Hanugen Theraputics, Voyager Therapeutics, Hoffmann-La Roche, Catalyst Pharmaceuticals, NMD Pharma, Biohaven Pharmaceuticals, CANbridge Pharmaceuticals Inc., Aurimed Pharma, Exicure, Amylon Therapeutics, Amniotics, and others, are developing therapies for the Spinal Muscular Atrophy treatment Spinal Muscular Atrophy emerging therapies such as – ACTX-401, Apitegromab, GYM329, and others are expected to have a significant impact on the Spinal Muscular Atrophy market in the coming years. In February 2025, Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today that the U.S. Food and Drug Administration (FDA) has approved a New Drug Application (NDA) for an Evrysdi® (risdiplam) tablet for people living with spinal muscular atrophy (SMA). Evrysdi is the only non-invasive disease-modifying treatment for SMA. The 5 mg Evrysdi tablet can either be swallowed whole or dispersed in water. In January 2025, Biogen Inc. (Nasdaq: BIIB) announced that the U.S. Food and Drug Administration (FDA) has accepted the company's supplemental New Drug Application (sNDA) and the European Medicines Agency (EMA) has validated the application for a higher dose regimen of nusinersen for spinal muscular atrophy (SMA). The higher dose regimen of nusinersen comprises a more rapid loading regimen, two 50 mg doses 14 days apart, and higher maintenance regimen, 28 mg, every 4 months, compared to the approved nusinersen regimen (SPINRAZA®). On April 2024, Hoffmann-La Roche announced a Phase IV Open-Label Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Patients With Spinal Muscular Atrophy After Gene Therapy On April 2024, Genentech announced results of a multi-center, longitudinal, prospective, non-comparative study to investigate the long-term safety and effectiveness of risdiplam, prescribed based on clinician judgment as per the Evrysdi® U.S. Package Insert (USPI) in adult and pediatric participants with spinal muscular atrophy (SMA). In this study, participants will be followed for up to 5 years from enrollment or until withdrawal of consent, loss to follow-up, or death. Participants who discontinue risdiplam may still remain in the study, if they agree to continue participating in the follow-up assessments. On April 2024, Biogen announced results of a study whose primary objective is to examine the clinical efficacy of nusinersen administered intrathecally at higher doses to participants with spinal muscular atrophy (SMA), as measured by change in Children's Hospital of Philadelphia-Infant Test of Neuromuscular Disorders (CHOP-INTEND) total score (Part B); to examine the safety and tolerability of nusinersen administered intrathecally at higher doses to participants with SMA (Parts A and C). On January 2024, Novartis announced results of a Phase IIIb, Open-label, Single-arm, Multi-center Study to Evaluate the Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally (1.2 x 10^14 Vector Genomes) to Participants 2 to < 18 Years of Age With Spinal Muscular Atrophy (SMA) Who Have Discontinued Treatment With Nusinersen (Spinraza®) or Risdiplam (Evrysdi®). On January 2024, NMD Pharma A/S announced results of a Phase 2, Randomised, Double-blind, Placebo-controlled, 2-way Crossover Study to Evaluate the Efficacy, Safety, and Tolerability of NMD670 in Ambulatory Adults With Type 3 Spinal Muscular Atrophy On December 2023, Novartis announced results of a Long-term Safety Study in Brazilian Patients With a Confirmed Diagnosis of Spinal Muscular Atrophy (SMA) Treated With Onasemnogene Abeparvovec (Zolgensma®) – ARISER Study. On May 2023, Biogen announced results of a study whose primary objective is to describe the natural history and utilization of disease modifying therapy (DMT) among adult Chinese participants with SMA linked to chromosome 5q (5q-SMA) Spinal Muscular Atrophy Overview Spinal Muscular Atrophy (SMA) is a rare genetic neuromuscular disorder that affects motor neurons in the spinal cord, leading to progressive muscle weakness and atrophy. It is caused by mutations in the SMN1 (Survival Motor Neuron 1) gene, which results in reduced levels of the SMN protein essential for motor neuron survival. The severity of SMA varies, and it is classified into different types (SMA 0–4) based on age of onset and disease progression. Spinal Muscular Atrophy primarily affects voluntary muscles, particularly those involved in movement, breathing, and swallowing. Spinal Muscular Atrophy Symptoms range from severe muscle weakness in infancy (SMA Type 1, the most common and severe form) to milder symptoms with later onset (SMA Type 3 and 4). In severe cases, respiratory complications can be life-threatening. Spinal Muscular Atrophy Diagnosis is confirmed through genetic testing. While there is no cure, disease-modifying therapies like nusinersen (Spinraza), risdiplam (Evrysdi), and gene therapy with onasemnogene abeparvovec (Zolgensma) can improve motor function and survival. Supportive care, including physical therapy, respiratory support, and nutritional management, plays a crucial role in improving the quality of life. Spinal Muscular Atrophy Advancements in treatment and early intervention, especially through newborn screening, have significantly improved outcomes for individuals with SMA. Spinal Muscular Atrophy Pipeline Therapeutics Assessment DelveInsight's Spinal Muscular Atrophy Report covers around 20+ products under different phases of clinical development like Late-stage products (Phase III) Mid-stage products (Phase II) Early-stage product (Phase I) Pre-clinical and Discovery stage candidates Discontinued & Inactive candidates Route of Administration Emerging Spinal Muscular Atrophy Drugs Under Different Phases of Clinical Development Include: ACTX-401: Novartis Apitegromab: Scholar Rock GYM329: Roche And Many Others Spinal Muscular Atrophy Pipeline Analysis: The Spinal Muscular Atrophy pipeline report provides insights into The report provides detailed insights about companies that are developing therapies for the Spinal Muscular Atrophy treatment with aggregate therapies developed by each company for the same. It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Spinal Muscular Atrophy Treatment. Spinal Muscular Atrophy key companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects. Spinal Muscular Atrophy Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type. Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Spinal Muscular Atrophy market. The report is built using data and information traced from the researcher's proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations, and featured press releases from company/university websites and industry-specific third-party sources, etc. Further Spinal Muscular Atrophy product details are provided in the report. Download the Spinal Muscular Atrophy pipeline report to learn more about the emerging Spinal Muscular Atrophy therapies @ Spinal Muscular Atrophy Companies and Drugs Scope of Spinal Muscular Atrophy Pipeline Drug Insight Coverage: Global Key Spinal Muscular Atrophy Companies: Scholar Rock, Biogen, Astellas Pharma, Alcyone Therapeutics, AndroScience Corporation, Hanugen Theraputics, Voyager Therapeutics, Hoffmann-La Roche, Catalyst Pharmaceuticals, NMD Pharma, Biohaven Pharmaceuticals, CANbridge Pharmaceuticals Inc., Aurimed Pharma, Exicure, Amylon Therapeutics, Amniotics, and others Key Spinal Muscular Atrophy Therapies: AS-202, HK001, Tegoprubart, AP-101, BLZ945, ANX005, MN-166, TW001, BIIB067, and others Spinal Muscular Atrophy Therapeutic Assessment: Spinal Muscular Atrophy current marketed and Spinal Muscular Atrophy emerging therapies Spinal Muscular Atrophy Market Dynamics: Spinal Muscular Atrophy market drivers and Spinal Muscular Atrophy market barriers Table of Contents Spinal Muscular Atrophy Report Introduction Spinal Muscular Atrophy Executive Summary Spinal Muscular Atrophy Overview Spinal Muscular Atrophy- Analytical Perspective In-depth Commercial Assessment Spinal Muscular Atrophy Pipeline Therapeutics Spinal Muscular Atrophy Late Stage Products (Phase II/III) Spinal Muscular Atrophy Mid Stage Products (Phase II) Spinal Muscular Atrophy Early Stage Products (Phase I) Spinal Muscular Atrophy Preclinical Stage Products Spinal Muscular Atrophy Therapeutics Assessment Spinal Muscular Atrophy Inactive Products Company-University Collaborations (Licensing/Partnering) Analysis Spinal Muscular Atrophy Key Companies Spinal Muscular Atrophy Key Products Spinal Muscular Atrophy Unmet Needs Spinal Muscular Atrophy Market Drivers and Barriers Spinal Muscular Atrophy Future Perspectives and Conclusion Spinal Muscular Atrophy Analyst Views Appendix About DelveInsight About DelveInsight DelveInsight is a leading Business Consultant, and Market Research firm focused exclusively on life sciences. Media Contact Company Name: DelveInsight Business Research LLP Contact Person: Ankit Nigam Email: Send Email Phone: +14699457679 Address: 304 S. Jones Blvd #2432 City: Albany State: New York Country: United States Website:

Scholar Rock to Participate in the 46 th Annual Goldman Sachs Global Healthcare Conference
Scholar Rock to Participate in the 46 th Annual Goldman Sachs Global Healthcare Conference

Business Wire

time7 days ago

  • Business
  • Business Wire

Scholar Rock to Participate in the 46 th Annual Goldman Sachs Global Healthcare Conference

CAMBRIDGE, Mass.--(BUSINESS WIRE)--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 announced that David L. Hallal, Chief Executive Officer, and members of management will participate in a fireside chat at the 46 th Annual Goldman Sachs Global Healthcare Conference on Wednesday, June 11 at 9:20 a.m. ET in Miami, Florida. A live webcast of the events may be accessed by visiting the Investors & Media section of the Scholar Rock website at Archived replays of the webcasts 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 and follow @ScholarRock and on LinkedIn. 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 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.

Scholar Rock to Participate in the 46th Annual Goldman Sachs Global Healthcare Conference
Scholar Rock to Participate in the 46th Annual Goldman Sachs Global Healthcare Conference

Yahoo

time7 days ago

  • Business
  • Yahoo

Scholar Rock to Participate in the 46th Annual Goldman Sachs Global Healthcare Conference

CAMBRIDGE, Mass., June 05, 2025--(BUSINESS WIRE)--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 announced that David L. Hallal, Chief Executive Officer, and members of management will participate in a fireside chat at the 46th Annual Goldman Sachs Global Healthcare Conference on Wednesday, June 11 at 9:20 a.m. ET in Miami, Florida. A live webcast of the events may be accessed by visiting the Investors & Media section of the Scholar Rock website at Archived replays of the webcasts 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 and follow @ScholarRock and on LinkedIn. 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 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. View source version on Contacts Scholar Rock:Investors & MediaRushmie NofsingerScholar Rockrnofsinger@ ir@ 857-259-5573

Scholar Rock Appoints Rebecca McLeod Chief Brand Officer and U.S. General Manager
Scholar Rock Appoints Rebecca McLeod Chief Brand Officer and U.S. General Manager

Yahoo

time02-06-2025

  • Business
  • Yahoo

Scholar Rock Appoints Rebecca McLeod Chief Brand Officer and U.S. General Manager

Rebecca McLeod brings exceptional U.S. operating experience; most recently served as argenx U.S. General Manager responsible for leading the launch of VYVGART® for gMG and CIDP CAMBRIDGE, Mass., June 02, 2025--(BUSINESS WIRE)--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 announced that Rebecca McLeod has been appointed to the newly created role of Chief Brand Officer and U.S. General Manager. In this role, she will be responsible for leading the anticipated U.S. commercial launch of apitegromab for patients with SMA – along with establishing the global apitegromab brand strategy and market positioning for Europe, Asia-Pacific and Latin America. "Rebecca's unprecedented experience in building and leading U.S. operations at argenx to launch VYVGART® successfully is invaluable as Scholar Rock accelerates planning toward our anticipated initial launch in SMA this year," said R. Keith Woods, Chief Operating Officer of Scholar Rock. "Launching apitegromab in the U.S. is vital in bringing the potentially transformative benefits of apitegromab to patients with SMA. I am confident that under her leadership, we will deliver for the SMA community in the U.S. and around the world as we become a global leader in developing and delivering innovative therapies to treat patients with rare, severe and debilitating neuromuscular disorders." Ms. McLeod is a 25-year pharmaceutical and biotechnology industry veteran who joins Scholar Rock from argenx, where she led market access, distribution, patient services, medical affairs, operations, marketing and sales for the U.S. organization. Under her leadership, argenx has delivered one of the most successful biopharmaceutical franchise launches with VYVGART. Earlier, she served in several commercial leadership positions across sales, marketing and product management at Alexion Pharmaceuticals and Takeda Pharmaceuticals. "I am thrilled to join Scholar Rock at this pivotal time of scale and growth as we prepare to become a global commercial biotechnology company," said Ms. McLeod. "I'm honored to leverage my leadership experience over the past seven years in the neuromuscular rare disease space to now build and lead Scholar Rock's U.S. operations as we plan to deliver apitegromab for children and adults with SMA, while also rolling out our global apitegromab brand strategy." 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 and follow @ScholarRock and on LinkedIn. 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 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. VYVGART® is a trademark of argenx. 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, the timing and results of regulatory submissions, the therapeutic potential of apitegromab, its transition to a fully integrated global commercial enterprise and planned launch of apitegromab, the establishment of its global brand strategy and the anticipated impact of the leadership appointment described herein. 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, whether the results from the Phase 3 SAPPHIRE trial will be sufficient to support regulatory approval, that preclinical and clinical data, including the results from the Phase 2 or Phase 3 clinical trial of apitegromab, 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; 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; Scholar Rock's dependence on third parties for development and manufacture of product candidates including, without limitation, to supply any clinical trials; and Scholar Rock's ability to manage expenses and to obtain additional funding when needed to support its business activities and establish and maintain strategic business alliances and new business initiatives, and Scholar Rock's ability to continue as a going concern; 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. View source version on Contacts Scholar Rock: Investors & Media Rushmie NofsingerScholar Rockrnofsinger@ ir@ 857-259-5573 Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data

Scholar Rock to Present at the Jefferies Global Healthcare Conference
Scholar Rock to Present at the Jefferies Global Healthcare Conference

Yahoo

time28-05-2025

  • Business
  • Yahoo

Scholar Rock to Present at the Jefferies Global Healthcare Conference

CAMBRIDGE, Mass., May 28, 2025--(BUSINESS WIRE)--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 announced that David L. Hallal, Chief Executive Officer, and members of the management team will participate in a fireside chat at the Jefferies Global Healthcare Conference on Wednesday, June 4 at 3:45 p.m. ET in New York City. A live webcast of the events may be accessed by visiting the Investors & Media section of the Scholar Rock website at Archived replays of the webcasts 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 and follow @ScholarRock and on LinkedIn. 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 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. View source version on Contacts Scholar Rock:Investors & MediaRushmie NofsingerScholar Rockrnofsinger@ ir@ 857-259-5573

DOWNLOAD THE APP

Get Started Now: Download the App

Ready to dive into the world of global news and events? Download our app today from your preferred app store and start exploring.
app-storeplay-store