Why There May Be a Big Issue With This Duchenne Muscular Dystrophy Study

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Duchenne muscular dystrophy (DMD) is an inherited disorder of progressive muscular weakness, especially in boys. It affects one out of 3,500 to 5,000 males worldwide and is one of the most serious genetic diseases in children globally. A few companies have made strides in treating the disease; unfortunately, this isn’t one of those stories.

Solid Biosciences Inc. (NASDAQ: SLDB) shares crumbled on Tuesday after the firm announced its first-quarter results and an update to its midstage DMD trial. The results didn’t play as much of a factor in the stock sinking as the trial update. This isn’t the first time, as Solid Biosciences has faced issues with this trial before.

In simple terms, one patient had issues with blood flow and liver function as a result of the study drugs, and more drugs were needed to help correct this. Although these serious adverse effects were resolved, it puts a damper on the study.

As for the specifics, the firm announced that two patients have been randomized in the second cohort of the Phase 1/2 Ignite DMD study, including one patient dosed with 2E14 vg/kg of SGT-001 and another added to the control group.

Shortly after dosing, the patient in the treatment group was diagnosed with a gastrointestinal infection that was classified as a serious adverse event unrelated to study drug, as well as a transient decline in platelet count that was considered a non-serious adverse event related to study drug; these events have fully resolved.

Additionally, the patient experienced a transient elevation of transaminases, as well as a transient increase in bilirubin higher than two times the upper limit of normal, which was rapidly resolved with an increase in oral glucocorticoids. This was reported to the FDA as a serious adverse event related to study drug. The patient is doing well and has resumed normal activities. Solid Biosciences continues to enroll patients in Ignite DMD per the study protocol and anticipates providing additional data later this year.

Ilan Ganot, CEO, president and co-founder of Solid Biosciences, commented:

We have continued to advance our programs for Duchenne muscular dystrophy over the last few months, particularly our lead microdystrophin gene therapy candidate, SGT-001. Building on our findings from the preliminary Phase 1/2 clinical data that we announced in February, we initiated dosing at 2E14 vg/kg in the second cohort of patients. Now, we are working to progress as quickly as possible, and we look forward to providing additional data later this year.

Management previously stated that it believes that existing cash, cash equivalents and available-for-sale securities as of the end of September 2018 will be sufficient to fund its operations through the first quarter of 2020.

Shares of Solid Biosciences were last seen down 28% at $6.36, in a 52-week range of $5.23 to $54.84. The consensus price target is $10.83.


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