Solid Biosciences Inc. (NASDAQ: SLDB) shares were absolutely crushed on Thursday after preliminary data from the firm’s midstage trial in Duchenne muscular dystrophy (DMD) was not up to par.
Specifically, the data came from Solid Bio’s Ignite DMD Phase 1/2 dose-ascending clinical trial evaluating the safety and efficacy of SGT-001 microdystrophin gene transfer for the treatment of DMD.
For some quick background: SGT-001 is a drug that delivers to the body a synthetic form of the dystrophin gene that codes for a replacement protein called microdystrophin.
These preliminary analyses are based on three-month biopsy data from the first three patients. In one patient, microdystrophin was detected via western blot below the 5% level of quantification of the assay and in approximately 10% of fibers via immunofluorescence. There were also signs of co-localization of neuronal nitric oxide synthase and beta-sarcoglycan associated with microdystrophin expression. In the second and third patients, microdystrophin was detected via immunofluorescence at very low levels, but it was undetectable via western blot.
In simpler terms, the initial three-month biopsy data showed low levels of microdystrophin protein expression, even after being treated. Management believes that higher dosing of SGT-001 can help solve the problem, but investors don’t seem to agree.
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.
Ilan Ganot, co-founder, CEO and president of Solid Bio, commented:
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. This strategy is further supported by our scalable manufacturing process, from which we have sufficient drug product available to dose escalate without delay. We have the financial resources to execute on our plan and look forward to communicating additional data later this year.
Shares of Solid Bio were last seen down about 65% at $7.76 on Thursday, in a 52-week range of $5.97 to $54.84. The stock has a consensus price target of $43.33.