Solid Biosciences showed signs Tuesday morning of facing serious setbacks for its Duchenne muscular dystrophy gene therapy, sending the biotechs shares down about 70% in early trading.
Solid (ticker: SLDB) said the Food and Drug Administration had put its Phase I/II study of its experimental gene therapy treatment for Duchenne muscular dystrophy on clinical hold after one of the six patients dosed with the treatment suffered acute kidney injury, among other serious side effects.
This is the second clinical hold placed on this study. In March 2018, the FDA held the study after a patient in the low-dose cohort was hospitalized.
We are encouraged that this patient is recovering, Solid Biosciences CEO Ilan Ganot said in a statement. We remain committed to bringing meaningful new therapies to the Duchenne community and continue to believe in the differentiated construct of SGT-001 and the potential benefits it may offer to patients.
Solid is one of a number of companies seeking to be the first to bring to market a gene therapy to cure Duchenne muscular dystrophy, a genetic disorder that produces muscle weakness and dramatically shortens the life expectancy of people who suffer from it. Wall Street has increasingly seen Sarepta Therapeutics (SRPT) as having the leading Duchenne muscular dystrophy gene therapy candidate, after Solid released disappointing data earlier this year.
Pfizer (PFE) is also developing a competing gene therapy.
Ganot founded the company after his son was diagnosed with the disease. Safety concerns have long dogged Solid. In January of 2018, gene therapy pioneer James Wilson resigned from the companys scientific advisory board, according to a company filing, over emerging concerns about the possible risks of high systemic dosing of AAV, the viral vector used to deliver the gene therapy.
In its Tuesday morning statement, Solid said three patients given a lower dose of the experimental gene therapy continue to do well, as do two of the three patients given a higher dose. But one of the patients who received the higher dose fell ill.
The third patient...dosed in late October, experienced a serious adverse event (SAE) deemed related to the study drug that was characterized by complement activation, thrombocytopenia [low blood platelet count], a decrease in red blood cell count, acute kidney injury, and cardio-pulmonary insufficiency, the company said.
In a note out Tuesday morning, SVB Leerink analyst Joseph P. Schwartz noted that the new adverse event resembles the one from March 2018.
The safety profile of SGT-001 will most likely be under increased scrutiny, Schwartz wrote.
Solid Biosciences stock was recently trading 68.6% lower at $3.45.
Write to Josh Nathan-Kazis at firstname.lastname@example.org
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