Scholar Rock announced a significant leadership transition today, appointing David L. Hallal as Chief Executive Officer as the late-stage biopharmaceutical company prepares for the global launch of its lead candidate apitegromab for spinal muscular atrophy (SMA). Hallal, who has served as Chairman of the Board since 2017, succeeds Jay Backstrom, M.D., who will continue with the company as a strategic advisor.
The leadership changes come at a pivotal moment for Scholar Rock, with the FDA having accepted the Biologics License Application (BLA) for apitegromab under priority review, setting the stage for a potential commercial launch later this year.
"It has been an honor to lead the incredible team at Scholar Rock over the past several years. I am very proud of all we have achieved - from advancing apitegromab through a successful, pivotal Phase 3 study and on the path to becoming a commercial company," said Dr. Backstrom. "With the BLA now accepted under priority review and the anticipated launch later this year, this is the right moment for our leadership transition."
Strategic Leadership Expansion
In addition to Hallal's appointment, Scholar Rock has strengthened its executive team with three key leaders to support its transformation into a fully integrated global commercial enterprise:
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Akshay Vaishnaw, M.D., Ph.D., joins as President of R&D in a newly created role. Vaishnaw previously served as President of Alnylam, bringing extensive experience in RNA therapeutics development.
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R. Keith Woods has been appointed Chief Operating Officer, tasked with evolving the organization into a fully integrated global enterprise. Woods previously served as COO of argenx, a company that successfully navigated the transition from clinical to commercial operations.
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Vikas Sinha joins as Chief Financial Officer, bringing financial leadership experience from his previous roles as CFO of Alexion and ElevateBio.
Apitegromab: A First-in-Class Therapy
Apitegromab represents a potentially groundbreaking approach to treating SMA. As an investigational fully human monoclonal antibody, it inhibits myostatin activation by selectively binding the pro- and latent forms of myostatin in skeletal muscle. This makes it the first muscle-targeted treatment candidate in SMA to demonstrate clinical success in a pivotal Phase 3 trial.
The drug's development addresses a critical unmet need in SMA treatment. While existing therapies primarily address the loss of motor neurons, apitegromab directly targets the progressive muscle weakness that leads to loss of motor function in SMA patients.
"I look forward to continuing our collaboration on behalf of the SMA community to bring our highly innovative apitegromab, the world's first muscle targeted treatment, to patients and families living with the impact of this severe and progressive disease," Dr. Backstrom added.
Regulatory Support and Clinical Progress
Apitegromab has garnered significant regulatory support, with the U.S. Food and Drug Administration (FDA) granting Fast Track, Orphan Drug, and Rare Pediatric Disease designations. The European Medicines Agency (EMA) has also granted Priority Medicines (PRIME) and Orphan Medicinal Product designations for the treatment of SMA.
These designations reflect the promising clinical profile of apitegromab and the high unmet need in SMA, a rare genetic neuromuscular disease that affects an estimated 30,000 to 35,000 people in the United States and Europe.
SMA Disease Burden
Spinal Muscular Atrophy is characterized by the loss of motor neurons, atrophy of voluntary muscles in the limbs and trunk, and progressive muscle weakness. The disease can severely impact patients' quality of life and mobility, with varying degrees of severity depending on the type.
Scholar Rock's approach with apitegromab represents a novel scientific strategy. By targeting myostatin, a member of the TGFβ superfamily of growth factors expressed primarily by skeletal muscle cells, the company aims to improve motor function in SMA patients. The absence of the myostatin gene has been associated with increased muscle mass and strength in multiple animal species, including humans.
Looking to Commercialization
With the leadership team now in place and regulatory review underway, Scholar Rock is positioning itself for the next phase of growth as it transitions from a clinical-stage to a commercial-stage company. The anticipated launch of apitegromab later this year would mark a significant milestone not only for the company but potentially for the SMA community as well.
The company's focus on the biology of the transforming growth factor beta (TGFβ) superfamily has enabled it to develop a pipeline with potential applications across neuromuscular diseases, cardiometabolic disorders, and cancer. This scientific approach, combined with the strengthened leadership team, positions Scholar Rock to potentially deliver meaningful advances for patients with serious diseases that have high unmet needs.
