Solid Biosciences Reports Additional Pulmonary Function Results from the Ongoing IGNITE DMD Phase I/II Clinical Trial of SGT-001
- Improvements in two additional assessments of pulmonary function in treated patients compared with untreated control patients further support potential functional benefit of SGT-001 one-year post administration -
- Data presented at the
“Duchenne is ultimately a fatal disease, with patients often succumbing to cardiopulmonary failure as muscle cells in the diaphragm and heart deteriorate,” said Roxana Donisa Dreghici, Senior Vice President, Head of Clinical Development for
“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,” said
IGNITE DMD Pulmonary Function Data
The pulmonary function results include percent predicted peak expiratory flow (PEF % predicted) and forced expiratory volume in one second (FEV1 % predicted) one year post SGT-001 administration. The data were collected from the first six patients dosed in IGNITE DMD (three at the low dose of 5E13 vg/kg and three at the high dose of 2E14 vg/kg) and three untreated control patients. Certain patients were not evaluable due to either missed patient assessments or not meeting the clinically acceptable criteria for the respective assessment.
- Four of these six patients dosed with SGT-001 and two of three patients in the untreated control cohort were evaluable for PEF % predicted assessment, with improvements noted in all dosed patients and declines noted in the untreated control patients. Patients in the low-dose cohort, Patients 1 and 3, demonstrated improvements in PEF % predicted from baseline to 1 year of 2.5% and 38.5%, respectively, and patients in the high-dose cohort, Patients 4 and 6, demonstrated improvements of 15.9% and 26.7%, respectively, at 1 year. Patients in the untreated control cohort, Control Patients 1 and 3 had declines of 1.1% and 18.2%, respectively, at 1 year.
- Five of these six patients dosed with SGT-001 and all three patients in the untreated control cohort were evaluable for FEV1 % predicted assessment, with improvements noted in all dosed patients and declines noted in the untreated control patients. Patients in the low-dose cohort, Patients 1 and 3, demonstrated improvements in FEV1 % predicted from baseline to 1 year of 13.4% and 4.3%, respectively, and patients in the high-dose cohort, Patients 4-6, demonstrated improvements of 10.8%, 15.5% and 2.8%, respectively, at 1 year. Patients in the untreated control cohort, Control Patients 1-3, reported declines of 8.7%, 17.0% and 12.0%, respectively, at 1 year.
These data, together with another pulmonary function endpoint, percent predicted forced vital capacity (FVC), provide further evidence that SGT-001 may benefit patients with respect to pulmonary function. Earlier this month, Solid reported positive 1.5-year data showing improvements in FVC at the
Natural history analyses of pulmonary function data suggest that patients would normally expect to exhibit a decline over the same time periods assessed.
“To our knowledge, Solid is the first company to report improvement in multiple assessments of pulmonary function following administration of a Duchenne gene therapy,” said
Solid’s SGT-001 is a novel adeno-associated viral (AAV) vector-mediated gene transfer therapy designed to address the underlying genetic cause of Duchenne. Duchenne is caused by mutations in the dystrophin gene that result in the absence or near absence of dystrophin protein. SGT-001 is a systemically administered candidate that delivers a synthetic dystrophin gene, called microdystrophin, to the body. This microdystrophin encodes for a functional protein surrogate that is expressed in muscles and stabilizes essential associated proteins, including neuronal nitric oxide synthase (nNOS). Data from Solid’s clinical program suggests that SGT-001 has the potential to slow or stop the progression of Duchenne, regardless of genetic mutation or disease stage.
SGT-001 is based on pioneering research in dystrophin biology by Dr.
This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the ability of the Company to continue dosing patients in the IGNITE DMD trial, the implication of interim clinical data, the safety or potential treatment benefits of SGT-001 in patients with DMD, the Company’s regulatory plans, the Company’s SGT-003 program, including the Company’s expectation for filing an IND, timelines, the sufficiency of the Company’s cash and cash equivalents to fund its operations, and other statements containing the words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “would,” “working” and similar expressions. Any forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with the Company’s ability to continue IGNITE DMD on the timeline expected or at all; obtain and maintain necessary approvals from the FDA and other regulatory authorities; obtain and maintain the necessary approvals from investigational review boards at IGNITE DMD clinical trial sites and the IGNITE DMD independent data safety monitoring board; enroll additional patients in IGNITE DMD and on the timeline expected; the Company’s dosing strategy; replicate in clinical trials positive results found in preclinical studies and earlier stages of clinical development; whether the interim data referenced in this release will be predicative of the final results of the trial or will demonstrate a safe or effective treatment benefit of SGT-001; whether the methodologies, assumptions and applications we utilize to assess particular safety or efficacy parameters will yield meaningful statistical results; advance the development of its product candidates under the timelines it anticipates in current and future clinical trials; successfully optimize and scale its manufacturing process; obtain, maintain or protect intellectual property rights related to its product candidates; compete successfully with other companies that are seeking to develop Duchenne treatments and gene therapies; manage expenses; and raise the substantial additional capital needed, on the timeline necessary, to continue development of SGT-001, SGT-003 and other product candidates, achieve its other business objectives and continue as a going concern. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company’s actual results to differ from those contained in the forward-looking statements, see the “Risk Factors” section, as well as discussions of potential risks, uncertainties and other important factors, in the Company’s most recent filings with the
Source: Solid Biosciences Inc.