The stock price of Amicus Therapeutics, Inc. (NASDAQ:FOLD) surged 21% to $2.22 per share after the biopharmaceutical company disclosed positive results from the 12-month and 24-month data from its phase 3 study (Study 011) of migalastat, a treatment for Fabry patients with amenable mutation.

Amicus Therapeutics

Results of the study

According to Amicus Therapeutics, Inc. (NASDAQ:FOLD), patients who switched from placebo to migalastat after month 6 demonstrated a statistically significant reduction in kidney interstitial capillary GL-3 (p=0.013) on month 12. Those who remained on migalastat for 12 months experienced a durable reduction kidney interstitial capillary GL-3.

The biopharmaceutical company said reduction in disease substrate was observed in plasma lyso-Gb3 in patients who switched from placebo to migalstat (p< 0.0001).

Amicus Therapeutics, Inc. (NASDAQ:FOLD) said kidney function (estimated glomerular filtration rate (eGFR), iohexol mGFR) remained stable over 18-24 months and migalastat was well tolerated by patients and was generally safe. According to the company, 85% or 35 of the 41 subjects with GLP HEK amenable mutations who completed the Study 011 remained in the voluntary extension study.

Amicus expects to meet regulators

In a statement, John Frowly, chairman and executive officer of Amicus Therapeutics, Inc. (NASDAQ:FOLD) said the 12 and 24 months results of migalastat treatment from the Study 011 met the pre-defined criteria for success in terms of substrate reduction and clinical measures for kidney function.

He added, “We believe these data provide important validation that a small-molecule chaperone can restore the function of a patient’s own enzyme in patients with amenable mutations, and that our pharmacogenomic assays can identify these patients. Together these results demonstrate the power of personalized medicine in rare diseases and offer the prospect of a new treatment option that differs from traditional enzyme replacement therapy.”

Crowley said Amicus Therapeutics, Inc. (NASDAQ:FOLD) expects to meet with regulators to discuss the data and determine the fastest way for the registration of migalastat.

According to the National Institute of Health, Fabry disease is caused by the lack of or  faulty enzyme needed to metabolize lipid.  Its symptoms include small, raised reddish-purple blemishes on the skin and patients feel burning sensations in their hands. The disease could also lead to heart and kidney complications.