Why This Overlooked New Jersey Biotech Could be an Orphan Underdog

June 15, 2017
By Alex Keown, BioSpace.com Breaking News Staff

CRANBURY, N.J. – Rare disease-focused company Amicus Therapeutics might be a company to watch due to federal incentives to develop treatments for the orphan disease sector. At least, that’s how one analyst sees it.

Writing in The Motley Fool, David Liang speculated that New Jersey-based Amicus has the potential to offer “jaw-dropping returns” for investors. Shares of Amicus are currently trading at $8.63. Since the start of the year, Amicus stock has steadily climbed from a low of $4.53 that resulted after the U.S. Food and Drug Administration called for additional studies of its Phase III drug candidate migalastat for the treatment of Fabry disease. Migalastat, which is sold in Europe as Galafold, is designed to bind with high affinity to the active sites of certain mutant forms of alpha-Gal A, the genotypes of which are referred to as amenable mutations.

Here in the U.S., the FDA expressed concern over gastrointestinal issues in some patients who were on the Amicus drug. About half of Fabry disease patients show GI signs and symptoms, including diarrhea, abdominal pain, constipation, nausea, and vomiting. Fabry disease is a rare genetic disease caused by the lack of an enzyme that allows the body to break down lipids, which are fat-like substances that include oils, waxes and fatty acids. Without the ability to break down the enzyme, it can lead to kidney problems as well as heart attack or stroke. The disease affects about 10,000 people globally.

Amicus is conducting an additional trial for U.S. approval, with top-line results expected in 2019. Liang said the new trial is progressing, with more than 100 patients already enrolled.

While the drug has been delayed in the United States, Galafold has been a strong revenue driver overseas. Liang said Amicus has approval to sell Galafold in 11 countries, including Japan, which is the second largest market for Fabry disease. The U.S. market for migalastat is estimated to be approximately 25 percent of the global opportunity, the company said in December.

While Galafold is the only approved product, Liang said what will please Amicus investors is the number of other experimental treatments in the company’s pipeline. Liang pointed to SD-101, a treatment for the orphan disease epidermolysis bullosa, which is advancing into Phase III trials. If Phase III trials are successful, Liang said SD-101 could be the catalyst Amicus investors are hoping for as the patient pool is much larger than Fabry disease. Epidermolysis bullosa, a connective tissue disorder, affects between 30,000 and 40,000 patients globally. In May, the FDA granted SD-101 with the Rare Pediatric Disease designation. The experimental drug had already received Orphan Drug and Breakthrough Therapy designations.

If approved, Liang expects SD-101 to generate annual revenue of about $1 billion for Amicus. Current standard of care consists of pain management and the bandaging and cleaning of open wounds to prevent infection. Data is expected later this year, during the third quarter.

Additionally, Amicus is conducting a Phase I/II study of ATB200/AT2221 for patients with Pompe disease, an inherited lysosomal storage disorder.