CHARLESTOWN, Mass. — Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation for SGT-003, the company’s next-generation Duchenne muscular dystrophy (Duchenne) gene therapy candidate.
“Solid’s receipt of Rare Pediatric Disease Designation for SGT-003 highlights the continuing need for transformational treatments for this devastating disease,” said Bo Cumbo, President and Chief Executive Officer at Solid Biosciences. “The key components of SGT-003 were rationally designed to improve on first generation gene therapies to provide skeletal muscle tropism, enhanced durability, and improved clinical outcomes. With site activation scheduled in April, and patient screening beginning shortly thereafter, we anticipate dosing patients in Q2 of this year.”
The planned Phase 1/2 trial, INSPIRE Duchenne, is a first-in-human, open-label, multicenter trial to determine the safety and tolerability of SGT-003 in pediatric patients with DMD at a dose of 1E14vg/kg. SGT-003 will be administered as a one-time intravenous infusion to patients in two cohorts with a minimum of three patients each, with the potential for cohort expansion. Cohort 1 will study patients with DMD ages 4 to < 6 and cohort 2 will study patients with DMD ages 6 to < 8. We anticipate providing an initial safety update for the first three to four patients enrolled in the INSPIRE Duchenne trial in mid-2024, and we anticipate providing initial expression and functional data from those patients in the fourth quarter of 2024.
“Preclinical data suggests that SGT-003 has potential to significantly improve on existing treatments for Duchenne by using a muscle tropic proprietary capsid to deliver a DNA sequence encoding a shortened form of the dystrophin protein which, importantly, includes the nNOS binding domain. nNOS is believed to play a crucial role in both muscular function and endurance,” said Dr. Gabriel Brooks, M.D., Chief Medical Officer at Solid Biosciences. “We look forward to rapidly bringing SGT-003 to the clinic and hope to all Duchenne patients in need.”
About Rare Pediatric Disease Designation
Rare Pediatric Disease Designation is granted by the FDA for serious or life-threatening diseases in which manifestations primarily affect children ages 18 years and younger. In addition, the disease must affect fewer than 200,000 people in the United States. The program is designed to encourage development of new drugs for the prevention and treatment of rare pediatric diseases. If a new biologics license application (BLA) is approved for SGT-003, Solid may be eligible to receive a priority review voucher. This voucher could be redeemed to obtain priority review for any subsequent marketing application for a different product and may be sold or transferred.
About DMD
Duchenne is a genetic muscle-wasting disease predominantly affecting boys, with symptoms usually appearing between three and five years of age. Duchenne is a progressive, irreversible, and ultimately fatal disease that affects approximately one in every 3,500 to 5,000 live male births and has an estimated prevalence of 5,000 to 15,000 cases in the United States alone.
About SGT-003
SGT-003 uses a proprietary, rationally designed capsid (AAV-SLB101) to deliver a DNA sequence encoding a shortened form of the dystrophin protein (microdystrophin), containing the R16-R17 nNOS binding domain. Preclinical data suggests this may be important for both muscular function and durability of benefit in patients.
About Solid Biosciences
Solid Biosciences is a life sciences company focused on advancing a portfolio of gene therapy candidates including SGT-003 for the treatment of Duchenne muscular dystrophy (Duchenne), SGT-501 for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT), AVB-401 for the treatment of BAG3-mediated dilated cardiomyopathy, and additional assets for the treatment of fatal cardiac diseases. Solid is advancing its diverse pipeline across rare neuromuscular and cardiac diseases, bringing together experts in science, technology, disease management, and care. Patient-focused and founded by those directly impacted, Solid’s mandate is to improve the daily lives of patients living with these devastating diseases.
Contact:
Leah Monteiro
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