Amicus Rockets on Early-Stage Pompe Disease Data

Published: Oct 05, 2017

Amicus Rockets on Early-Stage Pompe Disease Data October 4, 2017
By Alex Keown, BioSpace.com Breaking News Staff

CRANBURY, N.J. – A few weeks after Amicus Therapeutics dumped a failed late-stage wound treatment drug, the company is making waves again with additional positive results from a Phase I/II treatment for Pompe disease.

Pompe disease is an enzyme deficiency that leads to muscle and respiratory issues in patients.

The company said Pompe disease patients who completed six months of treatment with ATB200/AT2221 showed improvements in six-minute walking test. Data shows that at six months in ERT-naive patients were able to walk 42 meters in six minutes, but by nine months they had increased that pace to 75 meters in six minutes. Data also showed that ERT-switch patients showed mean increases of 35 meters at six months and 37 meters at nine months.

Additionally, patients demonstrated improvements in measures of motor function, stability and increases in forced vital capacity, which is the pulmonary function. ERT-naïve patients saw improvement in breathing between months six and nine of 4.2 percent and 5 percent. Amicus said ERT-switch patients saw a slight decline.

Patients taking the treatment also showed reductions in biomarkers of muscle damage and disease substrate, the company said. With additional Phase I/II data in hand, Amicus said it plans to continue a series of collaborative discussions with regulators in the United States and Europe about ATB200/AT2221, and expects to provide an update in the first half of 2018.

John Crowley, Amicus’ chief executive officer, said the additional data points have exceeded the company’s expectations regarding ATB200/AT2221.

“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,” Crowley said in a statement.

Not only was Crowley excited about the additional data, but so too were Amicus investors. Share prices spiked by about 21 percent, rising from $14.78 to a high of $16.50 this morning. Prices have slid back a little bit to $15.86 as of 11:35 a.m.

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. Researchers at Novazyme developed an enzyme replacement therapy that has allowed Megan and her brother, who was also diagnosed with Pompe, to live long past their initial life expectancy. Novazyme was acquired by Genzyme in 2001.

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