- A bet by New Jersey biotech Amicus Therapeutics Inc. to bring the first treatment for a rare skin condition to market did not pay off as hoped, with a Phase 3 trial failure pushing the company to halt further development of the experimental drug.
- Topline results from the study showed Amicus' candidate for epidermolysis bullosa performed no better than placebo in speeding wound closure in patients with the condition, which is characterized by exceptionally fragile skin that frequently blisters and tears.
- Two years ago, Amicus paid nearly $230 million in cash and stock upfront to acquire Scioderm, Inc. and gain access to the drug. At the time, Amicus saw a needed treatment option for a devastating condition with no approved therapies and a potential billion dollar market in the making.
People born with epidermolysis bullosa (EB) — sometimes referred to as "butterfly children" for their easily wounded skin — lack the ability to make certain proteins which help bind the layers of skin together.
With no drugs approved, EB is usually managed through the bandaging and cleansing of open wounds to help stave off infections. Yet, the process takes hours and patients are still left vulnerable and in constant pain.
Amicus had not originally been interested in EB, but encouraging signs from two small studies conducted by ScioDerm led Amicus to acquire the company and push ScioDerm's candidate into Phase 3 study.
Amicus expanded the number of clinical trial sites and subsequently enrolled 169 patients into the study, which compared the time to target wound closure within three months between patients treated with the experimental drug and those on placebo.
Results released Wednesday showed no difference between the two groups. On another measure, 49% of treated patients achieved target wound closure by month three, compared to 54% of patients who received placebo.
"In seeking to develop novel, high quality therapies for those living with devastating rare diseases we may sometimes fail," said Amicus CEO John Crowley. "But we would rather be the first to fail, than the last to try."
Amicus does not plan to invest further in other clinical studies of the drug, although participants in ongoing extension studies can continue to receive treatment.
Between 30,000 and 40,000 patients globally have EB, according to Amicus, although patient groups put the population number higher.
Shares in Amicus initially dipped in value during pre-market trading Wednesday, but reversed course and rose higher by nearly 4% after markets open — suggesting investors have already turned the page and are focusing on the biotech's lead candidate and remaining pipeline.
Failure of the study means Amicus will no longer owe about $345 million in success-based milestone payments, another factor that could be behind the stock rebound.
Amicus plans to submit its Fabry disease drug migalastat for U.S. approval later this year, much sooner than had originally been expected after the Food and Drug Administration dropped a requirement for an additional study. Migalastat is already approved in Europe and marketed as Galafold.
A pipeline candidate for Pompe disease has also attracted investor attention, and results from a Phase 1/2 study are expected in the coming weeks. In an interview last month, Crowley called the drug the "crown jewel" in the biotech's portfolio, ratcheting up the importance of a successful readout from that trial.