Health and Healthcare

Amicus Therapeutics Wins Big at World Muscle Society Conference

Chris Lange

Published: October 4, 2017 10:56 am

Last Updated: January 12, 2020 7:31 am

Amicus Therapeutics Inc. (NASDAQ: FOLD) is another biotech player that’s seeing a nice boost from the World Muscle Society Conference in France. In this case, the company announced additional positive results from its global Phase 1/2 clinical study (ATB200-02) to investigate ATB200/AT2221 in patients with Pompe disease.

24/7 Wall St. has already reported on Catabasis Pharmaceuticals Inc. (NASDAQ: CATB) and its incredible results from its MoveDMD trial in Duchenne muscular dystrophy (DMD). Keep in mind that Sarepta Therapeutics (NASDAQ: SRPT) is expected to present its DMD results on Thursday. There are even more biopharma companies that could see catalysts coming in October.

For some quick background: Pompe disease is an inherited disorder caused by the buildup of glycogen (disease substrate) in cells. Ultimately, the accumulation of glycogen does not allow muscles to function normally.

The Amicus trial proved consistent with previous results, meaning patients who completed six months of treatment with ATB200/AT2221 showed improvements in multiple measures of muscle function, including the six-minute walk test, stability or increases in forced vital capacity and further reductions in biomarkers of muscle damage.

Looking ahead, Amicus plans to continue a series of collaborative discussions with regulators in the United States and European Union, and it expects to provide an update in the first half of 2018.

John F. Crowley, board chair and chief executive of Amicus, commented:

These remarkable data from our Pompe clinical study of ATB200/AT2221 have once again exceeded our expectations. The consistency, durability and magnitude of the functional outcomes align with significant and continued reductions in key biomarkers of muscle damage and disease substrate, across patients, across cohorts and over significant periods of time. Taken together the strength of these results suggest the effect of ATB200/AT2221 may be very clinically meaningful for people living with Pompe disease. We are committed to working collaboratively with regulators to determine the fastest regulatory pathways that may be available to bring this new treatment paradigm to as many patients living with Pompe disease globally, as quickly as possible.

Shares of Amicus were last seen trading up more than 7% at $16.00, with a consensus analyst price target of $16.75 and a 52-week range of $4.41 to $16.60.