Scholar Rock Holding Corp. is a biopharmaceutical company. It engages in the business of discovering, developing, and delivering innovative medicines for the treatment of serious diseases in which signaling by protein growth factors plays a fundamental role. The company was founded by Timothy A. Springer and Leonard I. Zon in October 2012 and is headquartered in Cambridge, MA.
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- The FDA approved Isembyld (apitegromab-mstn) for SMA patients aged two and older already receiving an SMN2-targeted therapy, marking Scholar Rock's first product approval. - Isembyld is the first and only muscle-targeted SMA therapy, blocking myostatin activation to increase muscle mass and strength rather than targeting motor neurons. - In the Phase 3 SAPPHIRE trial, Isembyld produced a statistically significant 2.2-point HFMSE motor function improvement over placebo after 52 weeks of treatment. - The approval follows a prior complete response letter tied to manufacturing compliance issues, and Scholar Rock plans European and Japanese filings plus development in FSHD and obesity-related muscle loss.
- The FSHD Society, SOLVE FSHD, and FSHD CTRN announced the launch of a first-of-its-kind multi-stakeholder Industry Collaborative to improve clinical trial design and execution for facioscapulohumeral muscular dystrophy. - The initiative brings together industry experts, academic researchers, and technology partners to analyze biomarker and clinical endpoint data, aiming to reduce drug development risk and improve patient selection strategies. - Founding sponsors include Scholar Rock developing apitegromab, with Fulcrum Therapeutics donating Phase 2 and Phase 3 losmapimod trial data after program suspension in 2024. - The collaborative operates as a pre-competitive effort across multiple datasets while protecting sponsor confidentiality, building on prior FDA engagements to address regulatory expectations for FSHD therapeutics.
- Linavonkibart, a first-in-class selective TGF-β1 inhibitor, demonstrated a manageable safety profile and encouraging antitumor activity when combined with pembrolizumab in heavily pretreated patients with immune checkpoint blockade-resistant solid tumors. - The Phase I DRAGON trial showed a 20% objective response rate in clear cell renal cell carcinoma patients, with responses also observed in melanoma, head and neck squamous cell cancer, and urothelial cancer despite prior anti-PD-1 therapy resistance. - Biomarker analyses revealed that patients with elevated baseline CD8+ T cells, regulatory T cells, or TGF-β1 expression had improved response rates, suggesting a potential patient selection strategy for future treatment approaches.
- Scholar Rock's manufacturing partner Catalent Indiana received an FDA warning letter in early December 2025, creating potential supply chain risks for the company's planned 2026 launch of apitegromab for spinal muscular atrophy. - The company plans to resubmit its biologics license application in 2026 and pursue U.S. approval despite the manufacturing challenges at its primary production facility. - Scholar Rock is accelerating technology transfer to a second commercial facility as a contingency plan to mitigate manufacturing risks and protect its 2026 launch timeline. - The manufacturing overhang represents a new execution risk for the company, which has no revenue and net losses exceeding $350 million over the last twelve months.
- Over 25 pharmaceutical companies are actively developing 30+ TGF-β inhibitors to address significant unmet needs in oncology and fibrosis where existing therapies show limited effectiveness. - Key pipeline advances include Keros Therapeutics initiating Phase III trials for elritercept in myelodysplastic syndromes and EpicentRx receiving FDA Fast Track designation for AdAPT-001 in soft tissue sarcoma. - Recent clinical milestones demonstrate promising efficacy across multiple indications, with companies like Isarna Therapeutics reporting positive Phase II results for retinal fibrosis treatment ISTH0036.
- The FDA issued a Complete Response Letter declining approval of Scholar Rock's apitegromab for spinal muscular atrophy treatment due to manufacturing facility inspection findings. - The rejection was based on routine inspection issues at third-party manufacturer Catalent Indiana LLC, not related to the drug's safety or efficacy profile. - Scholar Rock plans to resubmit the Biologics License Application once Catalent Indiana addresses the FDA's manufacturing observations. - Company shares dropped approximately 15% in premarket trading following the regulatory setback announcement.
- The FDA issued a Form 483 to Novo Nordisk's Bloomington, Indiana manufacturing facility following an inspection that identified contamination issues including cat and human hair in vial stoppers and pest infestations in classified areas. - The plant, acquired through Novo Nordisk's $16.5 billion purchase of Catalent facilities in December 2024, has faced recurring contamination complaints dating back several years with approximately 20 additional hair contamination deviations occurring since the initial investigation. - The facility produces therapies for Regeneron and Scholar Rock, and the FDA found that the company failed to determine root causes for contamination or implement adequate corrective actions despite ongoing equipment failures and client complaints since 2022.
- FDA inspection found cat hair, pests, bacteria, and equipment failures at a Novo Nordisk manufacturing plant in Bloomington, Indiana, that was acquired from Catalent last year. - The facility has a history of troubled inspections over the past three years, with some problems recurring despite previous regulatory oversight. - The contamination issues are raising concerns for pharmaceutical companies like Regeneron Pharmaceuticals and Scholar Rock that rely on the facility for manufacturing services. - Wall Street analysts are monitoring the situation closely for potential impacts on other drug companies that depend on the plant's operations.
- Scholar Rock's Phase 2 EMBRAZE trial demonstrated that apitegromab preserved 54.9% more lean mass compared to tirzepatide alone, adding 4.2 pounds of lean muscle during weight loss treatment. - Patients receiving the combination therapy achieved higher quality weight loss with 85% fat mass reduction versus 70% fat mass reduction with tirzepatide alone over 24 weeks. - The myostatin inhibitor apitegromab was well tolerated with no serious adverse events related to treatment, addressing concerns about muscle loss associated with GLP-1 obesity medications. - The positive results validate Scholar Rock's platform for developing selective myostatin inhibitors while the company remains focused on apitegromab's potential approval for spinal muscular atrophy.
• Scholar Rock has appointed David L. Hallal as CEO, transitioning from his role as Chairman of the Board, as the company prepares for the global launch of apitegromab for spinal muscular atrophy. • The company has bolstered its leadership team with three key appointments: Akshay Vaishnaw as President of R&D, R. Keith Woods as Chief Operating Officer, and Vikas Sinha as Chief Financial Officer. • Apitegromab, a first-in-class muscle-targeted treatment for SMA, has received FDA priority review following successful Phase 3 trials, with potential approval and launch expected later this year.
