DMD SGT-001 gene therapy improves lung function in boys in trial


Treatment with SGT-001 – Solid Biosciences’ gene therapy candidate for Duchenne muscular dystrophy (DMD) – improves lung function, according to data from the first six patients enrolled in the ongoing IGNITE DMD clinical trial.

The improvements, seen one year after a single infusion of SGT-001 gene therapy into the vein, included a better percentage of predicted peak expiratory flow, called PEF% predicted – a measure of how quickly air can be exhaled. of the lungs – and a forced exhalation volume in one second, or FEV1% predicts, a measure of the amount of air that can be expelled from the lungs in one second.

These data are presented in a poster at the 50th Annual Meeting of the Child Neurology Society, by Oscar H. Mayer, MD, attending pulmonologist and director of the Lung Function Laboratory at Children’s Hospital of Philadelphia. The meeting will be held in Boston from September 29 to October 2.

“Improvements in pulmonary function parameters [goals] seen in the IGNITE DMD study, from baseline [study start] at one year are very promising, especially since the loss of lung function leads to respiratory failure and ultimately death and, to varying degrees, affects all patients living with Duchenne muscular dystrophy, ”Mayer said in A press release.

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DMD is caused by mutations in the DMD gene, which provides instructions for making dystrophin, a protein found in muscles. Its absence or near absence leads to weakness of the muscles, including those involved in breathing. SGT-001 is designed to deliver a gene encoding a shorter but functional dystrophin, called microdystrophin, into the body via a viral vector.

IGNITE DMD (NCT03368742) is a phase 1/2 clinical trial that aims to test whether SGT-001 is safe, well tolerated and effective in boys with DMD. A total of eight participants have received SGT-001 to date.

The latest data were obtained from three patients who received a low dose of 5E13 vector genomes (vg) / kg, three boys who received a high dose of 2E14 vg / kg and three untreated patients (controls).

Predicted data of PEF% were available for two of the low dose SGT-001 participants, two high dose participants, and two controls. In those who received SGT-001, improvements ranged from 2.5% to 38.5% at one year. In the control group, both patients had decreases of 1.1% and 18.2%.

For the predicted FEV1, data were available for two boys who received a low dose of SGT-001, the three patients who received high dose treatment and the three controls. Among treated patients, improvements ranged from 2.8% to 15.5% at one year. All control patients had declines ranging from 8.7% to 17%.

“The ability to improve lung function in these patients, especially during a period when untreated control [group] and natural history data indicate functional decline, is evidence of the potentially significant clinical benefit of SGT-001, ”said Roxana Donisa Dreghici, senior vice president and head of clinical development at Solid.

Earlier this month, the company announced positive one-and-a-half-year data showing sustained production of microdystrophin in muscles, while supporting previously reported benefits in functional ability and patient-reported outcomes.

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These data were presented during the virtual congress of the World Muscle Society 2021, in an oral presentation entitled “IGNITE DMD Phase I / II ascending dose study of SGT-001 microdystrophin gene therapy for DMD: 1.5-yearfunctional results update”, by Vamshi K. Rao, MD. Rao is an attending physician in neurology at Lurie Children’s Hospital, Chicago, and assistant professor of pediatrics at Northwestern University Feinberg School of Medicine.

“These data provide encouraging evidence of a functional benefit at one and a half years after treatment compared to natural history data and show a significant improvement in patient-reported outcomes,” said Rao.

Data over a year and a half also showed improvement in lung function. Specifically, the predicted mean percent improvement in forced vital capacity – the total amount of air exhaled during the FEV test – since the start of the study for the three patients who received a high dose of SGT-001 was 8.5%, and the average improvement over controls was 16% over the same period.

“To our knowledge, Solid is the first company to report improvement in multiple pulmonary function assessments after administration of Duchenne gene therapy,” said Ilan Ganot, CEO, President and Co-Founder of Solid.

“This data is in addition to data we previously reported from the IGNITE DMD clinical trial,” Ganot said. “We believe that exploring various parameters will allow us to better understand the full potential benefits that SGT-001 can bring across the spectrum of Duchenne disease manifestations. “

Mayer said he looked forward to further lung function data – and analysis – from IGNITE DMD, saying these results “should provide additional information on the potential benefits SGT-001 could bring to patients. with Duchenne “.

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