Solid Biosciences Reports 1.5-Year Data from Patients in the Ongoing IGNITE DMD Phase I/II Clinical Trial of SGT-001

- Data support continued functional benefit 1.5 years post treatment compared with natural history data; assessed by North Star Ambulatory Assessment (NSAA), 6-Minute Walk Test (6MWT) and Forced Vital Capacity (FVC) -

- Patient reported outcomes showed sustained improvements at 1.5 years compared to patient baseline and natural history data -

- No new drug-related safety findings for a period of up to 3.5 years -

CAMBRIDGE, Mass., Sept. 23, 2021 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company focused on advancing meaningful therapies for Duchenne muscular dystrophy (Duchenne), will report positive 1.5-year functional data and patient-reported outcome measures (Pediatric Outcomes Data Collection Instrument, or PODCI) for Patients 4-6 in the ongoing IGNITE DMD Phase I/II clinical trial of SGT-001. Vamshi Rao, MD, Attending Physician, Neurology, Lurie Children’s Hospital of Chicago and Assistant Professor of Pediatrics (Neurology and Epilepsy), Northwestern University Feinberg School of Medicine, and an IGNITE DMD clinical investigator, will present the data today in an oral session at the World Muscle Society 2021 Virtual Congress.

“The data presented today demonstrate durable expression and function of microdystrophin protein in biopsy samples collected 12 to 24 months post-dosing of SGT-001,” said Dr. Rao. “Additionally, these data provide encouraging evidence of functional benefit at 1.5 years post-treatment compared with natural history data and show meaningful improvement in patient-reported outcomes. Data from additional patients should enhance our understanding of the role that SGT-001 may play in improving outcomes for patients with Duchenne.”

“We continue to believe that SGT-001 has the potential to provide differentiated benefit to patients with Duchenne and are on track to dose additional patients in IGNITE DMD,” said Ilan Ganot, Chief Executive Officer, President and Co-Founder of Solid Biosciences.

IGNITE DMD 1.5-Year Data
Today, the company will report 1.5-year functional data and patient-reported outcome measures for Patients 4-6, all of whom received 2E14 vg/kg of SGT-001 manufactured using Solid Biosciences’ first-generation manufacturing process. The company previously reported 1-year data for the same measures in March 2021.

Biopsy Data
As previously reported in May 2021, analyses of the long-term biopsy data collected from Patients 4-6 at the 2E14 vg/kg dose level, taken 2 years, 1.5 years and 1-year post-dosing, respectively, indicate evidence of durable and widespread expression of the microdystrophin protein.