Amicus Therapeutics, Inc. (NASDAQ:FOLD)’s lead drug candidate is migalastat, which targets Fabry disease. An estimated 1 in 40,000 to 60,000 males have this disease; it also occurs in females but on a less frequent scale. Currently Fabry disease patients are limited to treatments like Sanofi’s Fabrazyme or Shires’ Replagal, but these require biweekly infusions. Amicus’ migalastat is taken orally, giving it a huge advantage. Sales for Fabrazyme and Replagal stood at $993 million in 2013.
On October 2, shares fell 53% after Amicus Therapeutics, Inc. (NASDAQ:FOLD)announced it will not file the NDA for migalastat as previously planned. Amicus delayed its filing because the FDA wanted more information concerning migalastat’s gastrointestinal symptoms and wanted Amicus to better integrate its Phase 2 and Phase 3 data. Management has since said that they are finalizing the briefing documents/integrated data for FDA and anticipate meeting with FDA by mid-year to clarify the US regulatory pathway for migalastat. Management will provide an update on the US regulatory pathway following the receipt of formal minutes in the agency event meeting.
Amicus’ European filing is still on track with the CHMP recommending approval of migalastat for the treatment of Fabry disease for patients who have amenable genetic mutations on April 1. The European Commission should make a final decision to approve Migalastat by June 1.
Amicus Therapeutics, Inc. (NASDAQ:FOLD)’s other lead drug candidate, Zorblisa, is in Phase 3 trials for the treatment of epidermolysis bullosa. Management believes Zorblisa could be a first-to-market therapy to address a $1 billion dollar global opportunity. Amicus expects to have top-line data from the Phase 3 trials in the second half of 2016. In addition, Amicus has also initiated a clinical study concerning a novel ERT for Pompe Disease. So far the preclinical data for Amicus’ candidate for Pompe disease is encouraging.
Although Amicus Therapeutics, Inc. (NASDAQ:FOLD)’s shares have fallen, the company had $165.9 million of cash, cash equivalents, and marketable securities at the end of March, and management expects its cash position to be able to fund operations until mid-2017.
Among the funds tracked by Insider Monkey, Amicus Therapeutics is not very popular, although the company’s small size allowed these investors to amass over 40% of its outstanding stock, according to the last round of 13F filings. More specifically, 22 funds, out of the 766 tracked by Insider Monkey, held $428.60 million worth of Amicus’ stock at the end of March, compared to 23 funds with stakes worth $475.01 million in aggregate a quarter earlier. Joseph Edelman‘s Perceptive Advisors is the largest shareholder of Amicus Therapeutics, Inc. (NASDAQ:FOLD) in our list, holding 15.24 million shares, followed by Jeremy Green‘s Redmile Group with 10.72 million shares.