Amicus Therapeutics, Inc. (NASDAQ:FOLD) reported a growth of 41% YoY to $62.4 million in Q2 2020. It is on the backdrop of strong patient demand worldwide in all of its businesses. The company added more than 1,000 mutations to its EU Galafold label.
On track to accomplish key milestones
Chairman of Amicus, John F. Crowley, said the company achieved significant Q2 2020 in advancing its mission for patients. The company is on track to achieve its key milestones that comprise the Pompe late-stage development program, global launch of Fabry, and advancing gene therapy pipeline.
Support for people with rare diseases
John further said the finances mobilized in July 2020 keep the company on a profitability path without touching the equity markets. The company is well-positioned to introduce innovative medicines and improve the lives of people with rare diseases.
Phase 3 Clinical trial of AT-GAA in LOPD is on track
Amicus’s Phase 3 clinical trial of AT-GAA in LOPD (late-onset Pompe disease) is on track and expects to publish topline results in H1 2021. It completed more than 97% of the 2,810 planned assessments and infusions for the PROPEL trial. The company would commence a rolling BLA in H1 2020 for AT-GAA for the final submission in H1 2021.
Amicus also commenced an EAP (expanded access program) for infantile-onset Pompe patients. Accordingly, young children with IOPD (infantile-onset Pompe disease) will get access to AT-GAA medicine under EAP.
Will present Phase 1/2 CLN6 data at CNSAM
Amicus will demonstrate Phase 1/2 CLN6 data CNSAM (Child Neurology Society Annual Meeting) in October 2020. It will provide feedback on the path early next year because regulatory interactions are underway.
Amicus will release initial data in early 2021 from the Phase 1/2 CLN3 clinical trial considering MCM changes (medical conference meetings). Feedback on the regulatory pathway is expected soon.
Amicus expects to post revenues of between $250 million and $260 million from Galafold global revenue this year. It will commence advance production to support MAA and BLA 2021. The company progresses an IND application for Pompe gene therapy.