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The Pulse of the Pharmaceutical Industry

Amicus Ends Lead Rare Disease Drug Program

Written by: | | Dated: Wednesday, September 13th, 2017


Amicus Dumps Lead Rare Disease Drug After Phase III Trial Failure


September 13, 2017
By Alex Keown, Breaking News Staff


CRANBURY, N.J. – Amicus Therapeutics (FOLD) is terminating a late-stage wound-treatment therapeutic after it failed to meet primary and secondary endpoints in a Phase III trial

New Jersey-based Amicus was developing SD-101 a topical wound-healing agent for patients with epidermolysis bullosa (EB), a rare genetic disorder that leads to severe skin blistering and open wounds often beginning at birth. In some cases, the blistering can occur internally along the lining of the mouth or intestines, according to the Mayo Clinic.

This morning, Amicus said SD0-101 did not differentiate itself from placebo in the 169-patient trial. In fact, on one key measure, closing wounds associated with EB within three months, SD-101 performed worse than placebo, the company said. According to data, 49 percent of patients on SD-101 saw closure within the three month treatment period compared to 54 percent of the placebo patients. Additionally, the company said the three month time frame to heal the EB wounds was no different between the SD-101 patients and the placebo group.

Amicus also noted that secondary endpoints failed to show a statistically significant difference than placebo. Tolerability was similar across both SD-101 and placebo, Amicus said.

John Crowley, chairman and chief executive officer of Amicus, called the Phase III results a disappointment. He lamented not being able to develop a treatment for EB, for which he said there is no approved treatment option. Current standard of care consists of pain management and the bandaging and cleaning of open wounds to prevent infection.

“In keeping with our Amicus mission, we have a strong commitment to the EB community and will work closely with investigators and other leading experts to understand and to share these data. We would like to sincerely thank the patients, families, clinical investigators, regulators and our Amicus team involved in this EB program. In seeking to develop novel, high quality therapies for those living with devastating rare diseases we may sometimes fail. But we would rather be the first to fail, than the last to try. Our vision at Amicus remains steadfast and is focused on building a leading global biotechnology company that delivers significant benefits for people living with rare diseases,” Crowley said.

Although the Amicus drug failed, investors have not dumped the stock in droves. In pre-market trading this morning, shares of Amicus fell about 14 percent, but then began climbing. As of 8:27 a.m. shares are actually up slightly over Tuesday’s closing price of $13.28.

Leerink analyst Joseph Schwartz said the negative EB news will be short-lived for Amicus. In a note, published on Endpoints News, Schwartz said Amicus investors are looking down the road at data from a Phase I/II for Pompe Disease.

Crowley is well known for his focus on rare diseases. His daughter was diagnosed with Pompe Disease when she was 15 months old. Crowley founded Novazyme Pharmaceuticals to develop a treatment for Pompe Disease.

Currently Amicus said it has no intention of investing in any additional clinical studies of SD-101. The company said it will share an analysis of Phase III data with key stakeholders in the EB community.

Although Amicus saw failure with SD-101, the company is still benefitting from a change in tune by the U.S. Food and Drug Administration for another drug. In July, the FDA reversed course on the company’s Phase III treatment for Fabry disease. The change paves the way for Amicus to seek regulatory approval later this year. The FDA originally rejected lead drug candidate migalastat in November 2016.



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