Further safety issues lead to clinical hold for DMD gene therapy

By Ben Hargreaves contact

- Last updated on GMT

(Image: Getty/E J Rodriquez)
(Image: Getty/E J Rodriquez)

Related tags: Solid Biosciences, Gene therapy, Pfizer, Sarepta Therapeutics, Duchenne muscular dystrophy

Solid Biosciences announces that its gene therapy for DMD has been placed under clinical hold again, after a patient experienced a serious adverse event.

The patient had received Solid Bioscience’s SGT-001 gene therapy candidate for Duchenne muscular dystrophy (DMD). Solid stated that several days after dosing the patient was hospitalized.

The company revealed that the patient, a seven-year-old boy, was found to have decreased red blood cell count, acute kidney injury, and cardio-pulmonary injury. Since hospitalization, the child is now recovering and his condition continues to improve, the company added.

The child was one of three patients that received a higher dose of the gene therapy treatment, with the other two patients reportedly doing well.

As a result of the adverse event, the US Food and Drug Administration (FDA) placed the trial on clinical hold, which means that Solid will halt enrollment onto the Ignite DMD Phase I/II trial and the dosing of new patients.

For Solid, this follows a previous clinical hold that the trial was placed under in March 2018​, after another patient showed signs of a reduction in platelet and red blood cell count.

Following the announcement, Solid’s share price dropped by 74%, at the time of writing.

Rival’s progress

There are currently two advanced competitors to Solid in the area of gene therapy for DMD, with Sarepta Therapeutics and Pfizer also looking to bring their own investigational treatments through the clinic.

Pfizer announced earlier this year, in August​, that it would be ramping up the manufacture of its own gene therapy ahead of the beginning of Phase III trials. During a previous trial, Pfizer also had a patient admitted to hospital with acute kidney injury and reduced platelet count.

Sarepta also experienced a clinical hold, however, the issues for the company were not patient-related but were related to the manufacturing process for plasmids​ used in its therapy.

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