- Scholar Rock’s BLA submission is based on the Phase 3 SAPPHIRE trial that demonstrated a statistically significant improvement in motor function for patients receiving apitegromab compared to placebo, as measured by the Hammersmith Functional Motor Scale-Expanded at week 52
- The FDA has granted apitegromab Fast Track, Orphan Drug and Rare Pediatric Disease Designations in SMA
- Scholar Rock remains on track to submit the apitegromab Marketing Authorisation Application (MAA) to the European Medicines Agency in 1Q 2025, with PRIME and Orphan Medicinal Product Designations
CAMBRIDGE, MA, USA I January 29, 2025 I Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on advancing innovative treatments for spinal muscular atrophy (SMA), cardiometabolic disorders, and other serious diseases where protein growth factors play a fundamental role, 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.
“We are gratified that in patients already on a SMN-targeted treatment, the SAPPHIRE trial met its primary endpoint for the main efficacy population showing a statistically significant 1.8-point improvement for patients receiving apitegromab compared to placebo, as measured by the Hammersmith Functional Motor Scale-Expanded at week 52,” said Jing Marantz, M.D., Ph.D., Chief Medical Officer of Scholar Rock. “With the strength of our Phase 3 data as the foundation of our submission, we look forward to continuing to work closely with the FDA through the review of our BLA on behalf of patients and families living with SMA.”
The review of the apitegromab BLA submission will be conducted by the Division of Neurology Products in FDA’s Center for Drug Evaluation and Research. The submission is supported by data from the Phase 3 SAPPHIRE trial and the Phase 2 TOPAZ trial. The Company shared positive topline data from the SAPPHIRE trial in 2024, and additional data will be presented at the 2025 MDA Clinical & Scientific Conference being held March 16-19 in Dallas, Texas.
Scholar Rock has requested Priority Review which, if granted, would shorten the FDA’s review time to six months from the date of filing acceptance. The FDA has granted Fast Track, Orphan Drug, and Rare Pediatric Disease designations, and the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) and Orphan Medicinal Product designations to apitegromab for the treatment of SMA.
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. The trial will evaluate apitegromab in patients who have been or are continuing to be treated with any currently approved SMN therapy (nusinersen, risdiplam and onasemnogene abeparvovec).
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 clinically meaningful and statistically significant motor function improvement in a pivotal Phase 3 trial 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. The U.S. Food and Drug Administration (FDA) has granted Fast Track, Orphan Drug and Rare Pediatric Disease designations, and the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) and Orphan Medicinal Product designations, to apitegromab for the treatment of SMA. Apitegromab has not been approved for any use by the FDA or any other regulatory agency.
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 were 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 either apitegromab 10 mg/kg, apitegromab 20 mg/kg, or placebo by intravenous (IV) infusion every 4 weeks for 12 months. An exploratory population including 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 every 4 weeks for 12 months.
The SAPPHIRE trial met its primary endpoint for the main efficacy population with a statistically significant 1.8-point improvement for all patients receiving apitegromab 10 mg/kg and 20 mg/kg (with an SMN-targeted treatment) compared to placebo (SMN-targeted treatments), as measured by the Hammersmith Functional Motor Scale-Expanded at week 52.
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.
SOURCE: Scholar Rock
Post Views: 201
- Scholar Rock’s BLA submission is based on the Phase 3 SAPPHIRE trial that demonstrated a statistically significant improvement in motor function for patients receiving apitegromab compared to placebo, as measured by the Hammersmith Functional Motor Scale-Expanded at week 52
- The FDA has granted apitegromab Fast Track, Orphan Drug and Rare Pediatric Disease Designations in SMA
- Scholar Rock remains on track to submit the apitegromab Marketing Authorisation Application (MAA) to the European Medicines Agency in 1Q 2025, with PRIME and Orphan Medicinal Product Designations
CAMBRIDGE, MA, USA I January 29, 2025 I Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on advancing innovative treatments for spinal muscular atrophy (SMA), cardiometabolic disorders, and other serious diseases where protein growth factors play a fundamental role, 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.
“We are gratified that in patients already on a SMN-targeted treatment, the SAPPHIRE trial met its primary endpoint for the main efficacy population showing a statistically significant 1.8-point improvement for patients receiving apitegromab compared to placebo, as measured by the Hammersmith Functional Motor Scale-Expanded at week 52,” said Jing Marantz, M.D., Ph.D., Chief Medical Officer of Scholar Rock. “With the strength of our Phase 3 data as the foundation of our submission, we look forward to continuing to work closely with the FDA through the review of our BLA on behalf of patients and families living with SMA.”
The review of the apitegromab BLA submission will be conducted by the Division of Neurology Products in FDA’s Center for Drug Evaluation and Research. The submission is supported by data from the Phase 3 SAPPHIRE trial and the Phase 2 TOPAZ trial. The Company shared positive topline data from the SAPPHIRE trial in 2024, and additional data will be presented at the 2025 MDA Clinical & Scientific Conference being held March 16-19 in Dallas, Texas.
Scholar Rock has requested Priority Review which, if granted, would shorten the FDA’s review time to six months from the date of filing acceptance. The FDA has granted Fast Track, Orphan Drug, and Rare Pediatric Disease designations, and the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) and Orphan Medicinal Product designations to apitegromab for the treatment of SMA.
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. The trial will evaluate apitegromab in patients who have been or are continuing to be treated with any currently approved SMN therapy (nusinersen, risdiplam and onasemnogene abeparvovec).
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 clinically meaningful and statistically significant motor function improvement in a pivotal Phase 3 trial 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. The U.S. Food and Drug Administration (FDA) has granted Fast Track, Orphan Drug and Rare Pediatric Disease designations, and the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) and Orphan Medicinal Product designations, to apitegromab for the treatment of SMA. Apitegromab has not been approved for any use by the FDA or any other regulatory agency.
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 were 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 either apitegromab 10 mg/kg, apitegromab 20 mg/kg, or placebo by intravenous (IV) infusion every 4 weeks for 12 months. An exploratory population including 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 every 4 weeks for 12 months.
The SAPPHIRE trial met its primary endpoint for the main efficacy population with a statistically significant 1.8-point improvement for all patients receiving apitegromab 10 mg/kg and 20 mg/kg (with an SMN-targeted treatment) compared to placebo (SMN-targeted treatments), as measured by the Hammersmith Functional Motor Scale-Expanded at week 52.
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.
SOURCE: Scholar Rock
Post Views: 201
