SGT-001 Improves Pulmonary Function in Duchenne Muscular Dystrophy


Improvements in peak expiratory flow, forced expiratory volume, and forced vital capacity were observed in all evaluable treated patients in the IGNITE-DMD trial.

SGT-001 (Solid Biosciences) continues to show efficacy in Duchenne muscular dystrophy (DMD), with new data showcasing pulmonary improvements after treatment.1

The updated data from the phase 1/2 IGNITE-DMD trial (NCT03368742) were presented at the Child Neurology Society 50th Annual Meeting by Oscar H. Mayer, MD, attending pulmonologist and director, Pulmonary Function Laboratory, Children’s Hospital of Philadelphia.

“The improvements in pulmonary function endpoints seen in the IGNITE DMD study, from baseline to one year are very promising, especially given that loss of pulmonary function leads to respiratory failure and ultimately death and, to varying degrees, impacts all patients living with Duchenne muscular dystrophy,” Mayer said in a statement.1 “The potential to improve, stabilize or even slow the decline in pulmonary function would be clinically beneficial and meaningful to patients. Continued collection and analysis of pulmonary function data in IGNITE DMD should provide additional insight into the potential benefit that SGT-001 may provide for patients with DMD.” 

Peak expiratory flow (PEF% predicted) and forced expiratory volume (FEV1% predicted) improved in all evaluable patients treated with the adeno-associated viral gene therapy and declined in all evaluable control cohort patients. Improvements in forced vital capacity (FVC% predicted) were also observed, with a mean difference of +16.0% from natural history at 1.5 years from baseline.

READ MORE: Cardiac-Derived Cell Therapy Shows Efficacy in Duchenne Muscular Dystrophy

“DMD is ultimately a fatal disease, with patients often succumbing to cardiopulmonary failure as muscle cells in the diaphragm and heart deteriorate,” Roxana Donisa Dreghici, MD, senior vice president and head, clinical development, Solid Biosciences, added to the statement.1 “Consequently, the ability to improve pulmonary function in these patients, especially during a period when the untreated control cohort and natural history data indicate functional decline, is evidence of the potentially meaningful clinical benefit of SGT-001.” 

Four of 6 treated patients and 2 of 3 control patients were evaluable for PEF assessment. Two low-dose (5E13 vg/kg) cohort patients improved by 2.5% and 38.5% from baseline to 1 year and 2 high-dose (2E14 vg/kg) cohort patients improved by 15.9% and 26.7% from baseline to 1 year. The 2 control patients declined by 1.1% and 18.2% from baseline to 1 year.

Five of 6 treated patients and all 3 control patients were evaluable for FEV assessment. Two low-dose cohort patients improved by 13.4% and 4.3% from baseline to 1 year and 3 high-dose patients improved by 10.8%, 15.5%, and 2.8% from baseline to 1 year. Comparatively, the 3 control patients declined by 8.7%, 17.0%, and 12.0% from baseline to 1 year.

Previous 1.5-year data announced by the company demonstrated an average improvement in FEV of 4.1% (range, 0.6-9.2) from baseline for a mean difference of 11.6% from natural history data.2 The previous data included mobility improvements such as a mean difference of 2.8 points in north star ambulatory assessment (NSAA) scores from baseline to 1.5 years compared to natural history and a mean difference of 78.8 meters in 6-minute walk test distances from natural history data.

Meaningful improvements were also observed with the Pediatric Outcomes Data Collection Instrument (PODCI) global score (range of change from baseline, 7-18), sports score (range, 14-23), transfer score (range, –6 to 3), and upper extremity scores (range, 2-9). Natural history studies would suggest a decline in global (–7.6), sports (–4.7), and transfer (–14.9) scores over 1.5 years.

"DMD is a really challenging disease to measure in the clinic. It's easy to measure the biomarker; it's more difficult to correlate that with outcomes. But from what we have seen... we are certainly encouraged and excited that we have a treatment that may well be improving outcomes,” Ilan Ganot, co-founder, president, and chief executive officer, Solid Biosciences, told GeneTherapyLive in an earlier interview.

1. Solid Biosciences reports additional pulmonary function results from the ongoing IGNITE DMD phase I/II clinical trial of SGT-001. News release. Solid Biosciences. September 29, 2021.
2. Solid Biosciences Reports 1.5-Year Data from Patients in the Ongoing IGNITE DMD Phase I/II Clinical Trial of SGT-001. News release. Solid Biosciences.
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