Solid Biosciences says FDA lifts halt on gene therapy trial, shares soar

(Reuters) - Solid Biosciences Inc said on Thursday the U.S. Food and Drug Administration (FDA) has lifted the clinical halt placed on a trial of its experimental gene therapy for Duchenne muscular dystrophy (DMD), a muscle-wasting disorder.

The news sent the company’s shares 75% higher in premarket trading, with Solid Biosciences saying it expects to restart the trial of the drug, SGT-001, in patients with DMD in the first quarter of 2021.

The study was halted in November for the second time in less than two years after a seven-year-old boy experienced serious complications, such as a decrease in red blood cell count and acute kidney injury.

The U.S. health agency in July requested updated safety and efficacy data for all patients dosed in the trial and provided direction on total viral load to be administered per patient.

The halt had pushed Solid Biosciences further behind Sarepta Therapeutics Inc and Pfizer Inc in the race to develop the first gene therapy for DMD.

DMD is a progressive disorder that mainly affects men. It begins to cause symptoms at an early age, first by hampering the ability to walk and later by causing breathing difficulties and heart problems.

Some analysts have suggested that the Sarepta gene therapy appears to have a better safety profile in early trials than Pfizer’s.

Gene therapies use engineered viruses to carry healthy genetic material into a person’s cells to replace faulty or mutated genes that cause a disease or condition.

Pfizer said on Thursday its gene therapy candidate for the muscle disorder received “fast track” designation from the FDA.

Reporting By Mrinalika Roy in Bengaluru; Editing by Aditya Soni