Solid Biosciences : FDA Removes Clinical Hold On SGT-001; Stock Surges

(RTTNews) - Solid Biosciences Inc. (SLDB) said that the U.S. Food and Drug Administration has lifted the clinical hold on IGNITE DMD, the Company's Phase I/II clinical trial for its investigational microdystrophin gene transfer, SGT-001, for the treatment of Duchenne muscular dystrophy or DMD.

In its letter, the FDA acknowledged that the company satisfactorily addressed all clinical hold questions. Solid has begun activities to resume the clinical trial and plans to reinitiate enrollment as quickly as possible.

In the Monday's pre-market, SLDB is trading at $31.15, up $4.65 or 17.55%.

As previously disclosed, the FDA placed a clinical hold on IGNITE DMD following the Company's report of a Serious Adverse Event (SAE) in the first patient dosed with SGT-001. The event was characterized by a decrease in platelet count followed by a reduction in red blood cell count, transient renal impairment and evidence of complement activation. There were no signs of bleeding or clotting abnormalities and no laboratory evidence of liver dysfunction.

The patient received standard medical care, a modified steroid regimen and a limited course of eculizumab for the observed complement activation. He remained clinically stable and generally asymptomatic throughout the event, which fully resolved.

In connection with the lifting of the clinical hold, Solid has made changes to the IGNITE DMD protocol, including the addition of IV glucocorticoids in the initial weeks post administration of SGT-001 and enhanced monitoring measures that include a panel for complement activation. The amended protocol also specifies that eculizumab will be available as a treatment option if complement activation is observed.

The company plans to enroll and dose several children prior to dosing additional adolescents. In addition, Solid now has the choice to obtain the intermediate muscle biopsy at 45 days post administration of SGT-001 to collect additional information about the time course of microdystrophin expression.

As a result of the clinical hold, Solid now expects to report initial data from a pre-specified interim analysis of IGNITE DMD in the second half of 2019.