Amicus Therapeutics, Inc. (NASDAQ:FOLD) closed Friday’s trading session at $11.40 after a slight uptick in its share price probably influenced by its announcement about positive results in its Pompe Disease Phase 1/2 Study.
The biopharma announced on February 5 revealed that its AT-GAA Phase 1/2 clinical study yielded positive results. The study was designed to evaluate AT-GAA’s performance in pompe disease patients. The latter is a lysosomal storage disorder that is inherited and one that occurs when an enzyme deficiency causes glycogen accumulation in cells. The AT-GAA patients treated for 24 months experienced improvements in the six-minute walk test (6MWT) as well as other motor function, stability and muscle strength functions.
“We are very pleased to report the latest data for AT-GAA, our investigational therapy for Pompe disease,” stated Amicus CEO John F. Crowley.
Crowly added that the treatment has continued to demonstrate consistent and compelling results during the 24 months that the patients underwent the treatment. The CEO also noted that positive long-term results indicate that AT-GAA can facilitate durable improvements. The Phase 2 study results and the PROPEL pivotal study which is currently ongoing will likely support the company’s AT-GAA advancement strategy. It will also possibly allow AT-GAA to become the new care standard for all Pompe disease patients.
Mark Roberts, ATB200-02’s lead investigator pointed out that Pompe disease patients require new treatment options that offer long-term relief. The recent clinical trial results have continued to produce strong and positive effects on Pompe patients in the trial.
The treatment achieved a positive safety and efficacy profile
Meanwhile, the clinical trial results also revealed that the treatment also demonstrated a better safety and tolerability profile. So far the adverse events that have occurred in the trial have been mild.
Pompe disease patients treated with AT-GAA also experienced minor infusion-associated reactions (IARs). More than 1,110 infusions were done, and only six patients experienced IARs. Key muscle damage biomarkers demonstrated a noteworthy and persistent reduction in the patients involved in the clinical trial. The ATB200-02 clinical study had a total of 25 patients enrolled across four groups.