Regulatory, Safety

Full FDA letter in hand, Solid Bio says it’s working to reverse clinical hold on its DMD gene therapy

Faced with skeptical investors and low share prices ever since the FDA slapped a clinical hold on its gene therapy for Duchenne muscular dystrophy, Solid Biosciences put out a note today detailing how it’s addressing the agency’s concerns. The update also came with positive news: If the PhI/II trial resumes, the biotech would be able to test both the high and low doses of the treatment.

Solid Bio CEO Ilan Ganot

In a full clinical hold letter Solid Bio $SLDB received today, the FDA laid out what it would take to resume the trial: an assessment of the underlying causes of the adverse event, the patient’s clinical status and laboratory parameters, as well as any new measures to address patient safety.

Cambridge, MA-based Solid Bio says it’s working with the FDA to provide that information. Meanwhile, CEO Ilan Ganot cannot wait to share that the patient in question is doing well, according to the treating physician.

The clinical hold was handed down about a month ago, in the wake of a serious adverse event reported in the first patient dosed in the Ignite DMD trial. Several days after being treated with SGT-001 — Solid Bio’s lead AAV therapy — investigators observed a drop in platelet count and, subsequently, red blood cell count. There was also evidence of complement activation, leading to the patient’s hospitalization.

That followed a much-discussed warning from gene therapy pioneer James Wilson, who reported severe toxic reactions in a separate animal study that used a high-dose AAV delivery of a corrective gene for the survival of motor neuron protein. One monkey and three piglets in that study had to be euthanized due to liver and neurological damages, respectively.

As it turned out, the FDA refused to green-light the high dose portion of the trial back in November, apparently unsatisfied with the manufacturing processes and product attributes. Solid Bio disclosed the partial hold in January in an amendment to its IPO filing.

Having reviewed additional data submitted by Solid Bio, the FDA is now saying that part of the issue is resolved. But it is yet unclear how long it would take for the company to get back on track — or to regain investors’ trust. Its stock, which has plummeted almost 60% since announcement of the hold, largely stayed flat in pre-market trading.


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