Solid Biosciences shares plunge on disappointing gene therapy trial results

Shares of Solid Biosciences Inc (NASDAQ:SLDB) plunged Thursday after unveiling a mixed set of results from a Phase ½ trial evaluating its SGT-001 microdystrophin gene transfer for the treatment of the degenerative disease Duchenne muscular dystrophy (DMD).

In a statement, Solid said initial three-month biopsy data from the Ignite DMD trial showed low levels of microdystrophin – the protein which bolsters muscles that people who suffer from this rare genetic disorder lack. DMD is a muscle-wasting disease that primarily affects boys and young men.

READ: US FDA lifts clinical hold on Solid Biosciences’ gene therapy SGT-001

In response, investors sent Solid shares down 73.5% to $6.05 before the opening bell.

Of the three patients dosed with 5E13 vg/kg of SGT-001, microdystrophin was detected via the western blot technique only in one patient at a level below 5% while very low levels of the protein were recorded in two patients, but they were undetectable via western blot.

Looking ahead, Solid CEO Ilan Ganot hopes that SGT-001 will be more effective at higher doses.

READ: Solid Biosciences plunges after FDA puts Phase I/II DMD trial on hold

“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. We have already begun working to expedite the planned dose escalation strategy outlined in our clinical trial protocol,” said Ganot in a statement.

Six patients have been enrolled in Ignite DMD. The main aim of the study is to assess the safety and tolerability of SGT-0001 as well as its efficacy as measured by microdystrophin expression.

SGT-001 is a type of gene therapy being investigated for its ability to address the underlying genetic cause of Duchenne muscular dystrophy – mutations in the dystrophin gene that result in the absence of dystrophin protein.

Contact Ellen Kelleher at [email protected]

Story by ProactiveInvestors