apitegromab (SRK-015) / Scholar Rock - LARVOL DELTA

SAPPHIRE: Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam (clinicaltrials.gov) - P3 | N=188 | Completed | Sponsor: Scholar Rock, Inc. | Active, not recruiting ➔ Completed

Trial completion • CNS Disorders • Genetic Disorders • Movement Disorders • Muscular Atrophy • Rare Diseases

Apitegromab in other rare neuromuscular disorders (Businesswire) - "Presenting preclinical data on a combination treatment approach with apitegromab in DMD at the MDA Clinical and Scientific Conference. The poster presentation includes initial preclinical data on an investigational approach combining the murine equivalent of apitegromab and a dystrophin corrector for addressing muscle defects in a DMD mouse model."

Preclinical • Duchenne Muscular Dystrophy

Scholar Rock Reports Full Year 2024 Financial Results and Highlights Business Progress (Businesswire) - "Apitegromab: In anticipation of regulatory approvals, Scholar Rock is planning for a U.S. commercial launch in 4Q 2025, with European launch to follow....Plans to initiate Phase 2 OPAL clinical trial in SMA patients under two years of age in 3Q 2025....SRK-439: Topline data expected in the second quarter of 2025 from the Phase 2 EMBRAZE proof-of-concept trial....IND application for SRK-439 for the treatment of obesity in patients on standard of care GLP-1 RAs expected to be filed in 3Q 2025....The Company’s outstanding debt balance is unchanged at $50 million. The new facility provides the Company with access to an additional $50 million at its discretion through December 31, 2025. An additional $50 million would be available to the Company upon FDA approval of apitegromab in SMA..."

Commercial • IND • Launch US • New P2 trial • P2 data • CNS Disorders • Metabolic Disorders • Muscular Atrophy • Obesity

Scholar Rock to Present Additional Clinical Data from the Phase 3 SAPPHIRE Trial at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference (Businesswire) - "Scholar Rock...announced today that it will present additional data from its Phase 3 SAPPHIRE clinical trial (NCT05156320) at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference being held March 16-19 in Dallas, Texas."

P3 data • Muscular Atrophy

ONYX: Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab (clinicaltrials.gov) - P3 | N=238 | Active, not recruiting | Sponsor: Scholar Rock, Inc. | Trial completion date: Jan 2027 ➔ May 2029

Trial completion date • CNS Disorders • Genetic Disorders • Movement Disorders • Muscular Atrophy • Rare Diseases

Scholar Rock Submits Biologics License Application (BLA) to the U.S. FDA for Apitegromab as a Treatment for Patients with Spinal Muscular Atrophy (SMA) (Businesswire) - "Scholar Rock...today announced the submission of a Biologics License Application to the U.S. Food and Drug Administration (FDA) for apitegromab, a muscle-targeted therapy designed and developed to provide clinically meaningful improvement in motor function for people living with SMA who are receiving SMN-targeted treatments. The Company remains on track to file a Marketing Authorisation Application to the European Medicines Agency in 1Q 2025...The submission is supported by data from the Phase 3 SAPPHIRE trial and the Phase 2 TOPAZ trial...Scholar Rock plans to also study apitegromab in SMA patients under two years of age in the Phase 2 OPAL trial, with a planned initiation in mid-2025."

EMA filing • FDA filing • New P2 trial • Muscular Atrophy

Advance the anti-myostatin program in cardiometabolic disorders (Businesswire) - "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."

P2 data • Metabolic Disorders • Obesity

EMBRAZE: Efficacy and Safety of Apitegromab for the Treatment of Adults Who Are Overweight or Obese (clinicaltrials.gov) - P2 | N=100 | Active, not recruiting | Sponsor: Scholar Rock, Inc. | Recruiting ➔ Active, not recruiting

Enrollment closed • Genetic Disorders • Obesity

Scholar Rock Reports Second Quarter 2024 Financial Results and Highlights Business Progress (Businesswire) - "Remains on track to report topline data from pivotal Phase 3 SAPPHIRE trial in patients with Spinal Muscular Atrophy (SMA) in 4Q 2024...Phase 2 EMBRAZE proof-of-concept trial enrolling ahead of schedule, topline data expected in 2Q 2025...The Company plans to file an IND for SRK-439 for the treatment of obesity in 2025."

IND • P2 data • P3 data: top line • CNS Disorders • Metabolic Disorders • Muscular Atrophy • Obesity

Long-term efficacy, safety, and patient-reported outcomes of apitegromab in patients with spinal muscular atrophy: results from the 36-month TOPAZ study. (PubMed, Front Neurol) - P2 | "The most frequently reported treatment-emergent adverse events were pyrexia (48.6%), nasopharyngitis (45.7%), COVID-19 infection (40.0%), vomiting (40.0%), and upper respiratory tract infection (31.4%). The benefit of apitegromab treatment observed at 12 months was sustained at 36 months with no new safety findings."

Journal • Patient reported outcomes • Fatigue • Genetic Disorders • Infectious Disease • Movement Disorders • Muscular Atrophy • Novel Coronavirus Disease • Pediatrics • Rare Diseases • Respiratory Diseases

EMBRAZE: Efficacy and Safety of Apitegromab for the Treatment of Adults Who Are Overweight or Obese (clinicaltrials.gov) - P2 | N=100 | Recruiting | Sponsor: Scholar Rock, Inc.

New P2 trial • Genetic Disorders • Obesity

Safety and Efficacy of Apitegromab in Patients With Spinal Muscular Atrophy Types 2 and 3: The Phase 2 TOPAZ Study. (PubMed, Neurology) - No abstract available

Journal • P2 data • Genetic Disorders • Movement Disorders • Muscular Atrophy • Rare Diseases

TOPAZ: An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy (clinicaltrials.gov) - P2 | N=58 | Completed | Sponsor: Scholar Rock, Inc. | Active, not recruiting ➔ Completed

Trial completion • CNS Disorders • Genetic Disorders • Movement Disorders • Muscular Atrophy • Rare Diseases

Effect of apitegromab on motor function and patient-reported outcomes at 36 months in patients aged 2–21 years with spinal muscular atrophy (MDA 2024) - P2 | "In the 52-week treatment period, 3 cohorts of patients received either 2- or 20-mg/kg apitegromab, alone (n=11) or in combination with nusinersen (n=47). Treatment with apitegromab was associated with sustained clinical benefit and improvements in patient- and caregiver-reported outcomes of function and perceived fatigue in patients with Types 2 or 3 SMA for 36 months. These results support further development of apitegromab in SMA."

Clinical • Patient reported outcomes • Fatigue • Genetic Disorders • Movement Disorders • Muscular Atrophy • Rare Diseases

Safety and Efficacy of Apitegromab in Patients With Spinal Muscular Atrophy Types 2 and 3: The Phase 2 TOPAZ Study. (PubMed, Neurology) - P2 | "Apitegromab led to improved motor function in participants with later-onset types 2 and 3 SMA. These results support a randomized, placebo-controlled phase 3 trial of apitegromab in participants with SMA."

Journal • P2 data • CNS Disorders • Cough • Genetic Disorders • Infectious Disease • Movement Disorders • Muscular Atrophy • Pain • Rare Diseases • Respiratory Diseases

Scholar Rock Announces FDA Clearance of IND Application to Initiate Phase 2 Proof-of-Concept Trial with Apitegromab to Treat Obesity (Businesswire) - "Scholar Rock...announced that the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application for its Phase 2 proof-of-concept trial of apitegromab to treat obesity in patients taking a GLP-1 receptor agonist (GLP-1 RA)....Trial initiation is on track for mid-2024, and data from the apitegromab Phase 2 trial are expected in mid-2025."

IND • New P2 trial • P2 data • Metabolic Disorders • Obesity

SAPPHIRE: Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam (clinicaltrials.gov) - P3 | N=188 | Active, not recruiting | Sponsor: Scholar Rock, Inc. | Trial primary completion date: Jun 2024 ➔ Oct 2024

Trial primary completion date • CNS Disorders • Genetic Disorders • Movement Disorders • Muscular Atrophy • Rare Diseases

SAPPHIRE: Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam (clinicaltrials.gov) - P3 | N=188 | Active, not recruiting | Sponsor: Scholar Rock, Inc. | Recruiting ➔ Active, not recruiting

Enrollment closed • CNS Disorders • Genetic Disorders • Movement Disorders • Muscular Atrophy • Rare Diseases

Effect of Apitegromab on Motor Function at 36-months in patients with nonambulatory Spinal Muscular Atrophy aged 2-12 years old (WMS 2023) - No abstract available

Clinical • Genetic Disorders • Movement Disorders • Muscular Atrophy • Rare Diseases

Effect of Apitegromab on pedi-cat and promis-fatigue questionnaire at 36-months in patients with Spinal Muscular Atrophy (WMS 2023) - No abstract available

Clinical • Fatigue • Genetic Disorders • Movement Disorders • Muscular Atrophy • Rare Diseases

Apitegromab in SMA: An Analysis of Additional Efficacy Endpoints in the TOPAZ Study at 36 Months (EAN 2023) - P2 | "Patients received IV apitegromab Q4W, alone or with nusinersen. The ongoing TOPAZ extension B is ongoing and will provide further long-term efficacy and safety data of apitegromab in SMA patients. This information will deepen our understanding of latent myostatin dynamics and inform future studies."

Clinical • Muscular Atrophy

Apitegromab in SMA: Analysis of Correlates of Patient Reported Outcomes and Motor Function Increases in 24 Month TOPAZ Data (EPNS 2023) - P2 | "Apitegromab has the potential to be the first muscle-targeted therapy to address motor function impairment affecting patients with SMA and further exploration of its impact on Quality of Life (QoL) is warranted."

Clinical • Patient reported outcomes • Fatigue • Muscular Atrophy

Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab-ONYX (clinicaltrials.gov) - P3 | N=260 | Active, not recruiting | Sponsor: Scholar Rock, Inc. | Not yet recruiting ➔ Active, not recruiting

Enrollment closed • CNS Disorders • Genetic Disorders • Movement Disorders • Muscular Atrophy • Rare Diseases

Treatment Effects Among Patients with Type 2 and Type 3 SMA Directly Reported by Patients and Caregivers from the TOPAZ Clinical Trial (AAN 2023) - P2 | "Conclusions The symptoms and impacts of living with SMA as reported by 12 patients/caregivers provided insight related to meaningful treatment benefits experienced while receiving apitegromab during the TOPAZ trial, particularly around activities of daily living, fatigue, muscle weakness, physical functioning, independence, and balance. These data provide supplemental insights beyond what may be captured in a standardized questionnaire and give insight to the holistic improvement in the patient’s quality of life."

Clinical • Fatigue • SMA4

Long-Term Safety and Efficacy of Apitegromab in Patients with Type 2 and Type 3 Spinal Muscular Atrophy Who Completed Previous Investigational Trials of Apitegromab (clinicaltrialsregister.eu) - P3 | N=260 | Sponsor: Scholar Rock, Inc.

New P3 trial • Genetic Disorders • Movement Disorders • Muscular Atrophy • Rare Diseases