Solid Biosciences Tanks After Reporting Adverse Event In Duchenne Muscular Dystrophy Trial

The microcap biotech Solid Biosciences Inc SLDB's shares were losing more than one-quarter of their value Tuesday after the company issued a clinical trial update on its Duchenne muscular dystrophy gene therapy candidate with its first-quarter earnings release.

What Happened

Solid Biosciences said in the earnings release that, among the two patients randomized in the second cohort of the Phase 1/2 study dubbed IGNITE, one was dosed with 2E14 vg/kg of its DMD gene therapy candidate and another was added to the control group.

The patient in the treatment arm experienced a transient decline in platelet count, considered a serious adverse event, or SAE, related to the study.

The patient also 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 reported as an SAE related to the study, the company said.

The issue was rapidly resolved with an increase in oral glucocorticoids, according to Solid BIosciences. 

Solid also noted that the treated patient experienced a gastrointestinal infection, considered an SAE unrelated to the study.

The company also reported a wider-than-expected loss in the first quarter. Solid Biosciences reported a first-quarter earnings per share loss of 85 cents versus a consensus estimate calling for a 63-cents-per-share loss. 

Why It's Important

In March 2018, Solid's IGNITE study was clamped with a clinical hold by the FDA following the reporting of an SAE in the first patient dosed in the study; the clinical hold was lifted in June.

Subsequently, the company's shares plummeted a whopping 68 percent Feb. 7 when it released preliminary results from the IGNITE trial that evaluated three patients, with the study group showing low levels of microdystrophin protein expression.

DMD is caused by the absence of the dystrophin protein, leading to progressive deterioration of muscle fibers. It typically affects boys.

The company said at the time it was planning a dose escalation to test the effectiveness of the therapy.

What's Next

Solid said it is continuing to enroll patients in the IGNITE study per the study protocol. The company said it expects to provide additional data later this year.

Solid Biosciences shares were plunging by 29.38 percent to $6.25 at the time of publication Tuesday. 

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