Solid Biosciences Shares Crash After Data Release On Muscular Dystrophy Candidate

Solid Biosciences Inc SLDB shares were trading close to their historical lows Thursday following the release of data from a Phase 1/2 dose-escalating trial of its Duchenne muscular dystrophy candidate SGT-001.

DMD, caused by the absence of a protein called dystrophin that aids in keeping muscle cells intact, is a genetic disorder that causes progressive muscle weakness and degeneration, primarily in boys.

Unimpressive Efficacy Data

Solid Biosciences released preliminary findings from a study dubbed IGNITE DMD, a Phase 1/2 dose-escalating trial that evaluated the safety and efficacy of its gene therapy candidate SGT-001, showing low levels of microdystrophin expression.

Microdystrophin is a shorter but functional version of dystrophin protein and alleviates symptoms in DMD patients.

"We believe that SGT-001 will be a meaningful treatment for patients with DMD and are confident we have the right approach in place to evaluate its potential at higher doses," Solid Biosciences Ilan Ganot said in a statement. 

The preliminary findings were based on three-month data from the first three patients who were dosed with 5E13 vg/Kg of SGT-001, which is the lowest dose outlined in the study protocol.

Solid Biosciences said it is negotiating with appropriate parties for dose escalation as soon as possible.

Sarepta Moving In Sympathy

Sarepta Therapeutics Inc SRPT is seen as a frontrunner in the DMD gene therapy race. Solid Biosciences and Pfizer Inc. PFE are also in the fray.

"The 'first mover' is modeled to seize a 50-percent market share with peak sales near $4 billion and annual recurring revenue of $1.5 billion," Credit Suisse analyst Martin Auster said in a September 2018 note.

At last check Thursday, Solid Biosciences shares were sliding more than 64 percent to $8.

Sarepta shares were declining 2.51 percent to $137.94 and Pfizer was down 2.53 percent at $41.26. 

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