What happened
Shares of Solid Biosciences (SLDB 0.22%) jumped over 32% today after the company announced changes to the clinical program evaluating its lead drug candidate, SGT-001, as a treatment for Duchenne muscular dystrophy (DMD). The changes are meant to address previous red flags experienced in the first patient given the highest dose of the gene therapy, as reported in May, and shorten the time required to generate meaningful clinical data.
Investors have good reason to cheer the announcement from the struggling biopharma. Solid Biosciences exited June with just $52 million in cash. While it raised $60 million through a private placement in July and says there's enough cash to fund operations into late 2020, the business reported an operating loss of $57 million in the first half of 2019. Therefore, expediting the time to results will help to mitigate the risk of dilution.
As of 12:03 p.m. EDT, the stock had settled to a 28.5% gain.
Image source: Getty Images.
So what
It would have been easy for investors to write off Solid Biosciences up to this point. The lowest dose of SGT-001 didn't appear to have any effect, while the highest dose resulted in serious side effects. The business is hoping its latest pivot can deliver meaningful clinical results and win back the trust of the markets.
Solid Biosciences announced that it has amended the protocol for the ongoing phase 1/2 trial for SGT-001 in DMD. For now, patients weighing more than 25 kilograms (about 55 pounds) will not be evaluated in the study. Gene therapy doses are determined by the mass of a patient, so instituting an upper weight limit will effectively reduce the total dose being administered. The company intends to allow larger patients into the study at a later date.
The biopharma also said it will get rid of the control arm of the study, which included a more cumbersome patient matching protocol for the experimental gene therapy. The second patient has been dosed in the high-dose cohort with the amended protocol.
Now what
The latest changes show that management is eager to continue developing SGT-001 in DMD. Investors are certainly more fond of that strategy than scrapping the clinical asset altogether. That said, the focus on smaller patients and axing the control arm may not yield the end results Solid Biosciences is hoping for. Investors will have to wait for the next data update promised before the end of 2019 (likely at the time of the next quarterly update in November) before getting too excited.
