No respite for Solid Bio's troubled Duchenne program as FDA keeps it on hold

stop sign (knerri61/Pixabay)
Solid Bio's Duchenne muscular dystrophy gene therapy, SGT-001, has endured partial and full clinical holds over the past couple of years due to safety concerns and manufacturing issues. (knerri61/Pixabay)

Solid Biosciences didn’t exactly hit 2020 running, laying off one-third of its staff and “curtailing” most of its R&D in a bid to keep its Duchenne muscular dystrophy treatment afloat. Four months later, those efforts have yet to bear fruit.

The FDA is keeping the Duchenne program, a microdystrophin gene therapy, under a clinical hold it placed in November, despite Solid Bio’s efforts to appease the agency. It put the phase 1/2 study of the treatment, SGT-001, on hold after the company reported side effects in a sixth patient, including decreased red blood cell and platelet counts, acute kidney injury and complement activation, a type of innate immune system response.

And this isn’t Solid Bio’s first rodeo. SGT-001 has endured partial and full clinical holds over the past couple of years due to safety concerns and manufacturing issues.

RELATED: Solid Bio starts to break up as the ax swings down on one-third of workers, CMO

In April, the company submitted information to the FDA about improvements to the manufacturing of the treatment as well as changes to the clinical protocol designed to boost safety, the company said in a statement. But the FDA didn’t budge and requested more data and analyses on the treatment’s manufacturing process.

Solid Bio is working to generate those data with plans to submit them by the end of the third quarter. The company’s restructuring was designed to save it enough cash to keep going into 2021.

“We share the FDA’s commitment to patient safety and are working collaboratively with the agency to resolve the clinical hold,” said Solid Bio CEO Ilan Ganot in the statement. “We consider patient safety the utmost priority and believe the clinical development of SGT-001 could offer meaningful benefits to patients with this devastating disease.”