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Amicus (FOLD) Provides Updates on AT-GAA for Pompe Disease

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Amicus Therapeutics FOLD announced clinical, regulatory and manufacturing advancements for AT-GAA, its investigational therapy for Pompe disease.The company is utilizing its biologics capabilities and CHART platform to develop AT-GAA to treat Pompe disease.

AT-GAA is a novel treatment paradigm that consists of ATB200, a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme to enhance uptake, co-administered with AT2221, which is a pharmacological chaperone. In February 2019, the FDA granted a Breakthrough Therapy designation (“BTD”) to AT-GAA. The candidate is the first-ever investigational product for Pompe disease to receive BTD.

The company completed and exceeded patient enrollment in the global phase III PROPEL study for Pompe disease. The study enrolled 123 patients globally. The study is intended to assess the superiority of AT-GAA compared to the current standard of care, alglucosidase alfa, in both enzyme replacement therapy (ERT) switch and ERT-naive patients.

Amicus plans to apply for and initiate a rolling submission of the Biologics License Application (BLA) for AT-GAA in late-onset Pompe disease and complete the final submission in the first half of 2021. 

 Shares of the company have lost 18.3% in the past year compared with the industry’s decline of 2.9%.

Further, the company has two gene-therapy programs in the pipeline for two different types of Batten disease.

Amicus entered into several partnerships for gene-therapy manufacturing and expansion. In July 2019, it inked partnerships with Catalent Biologics and Thermo Fisher for clinical and commercial gene-therapy manufacturing. Brammer Bio, a part of Thermo Fisher, will assist Amicus’ gene therapy programs in CLN6 and additional forms of Batten disease with clinical and commercial-scale capabilities. Paragon Gene Therapy, a unit of Catalent Biologics, will provide clinical manufacturing capabilities and capacity for multiple active preclinical lysosomal disorder programs in collaboration with Penn.

Amicus has exclusive worldwide rights to Penn’s next-generation gene therapy technologies for a majority of lysosomal disorders and 12 additional rare diseases, including Rett Syndrome, Angelman Syndrome, Myotonic Dystrophy and select other muscular dystrophies.

Amicus Therapeutics, Inc. Price

Zacks Rank and Stocks to Consider

Amicus currently has a Zacks Rank #3 (Hold).

Some better-ranked stocks in the biotech sector are Agenus Inc. AGEN, Amarin Corporation Plc. (AMRN - Free Report) and AquaBounty Technologioes Inc. AQB, all sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Alexion’s earnings per share estimates have increased from $9.95 to $10.35 for 2019 and from $10.87 to $11.32 for 2020 in the past 90 days. The company delivered a positive earnings surprise in the trailing four quarters by 12.50%, on average.  

Amarin’s loss per share estimates have narrowed from 9 cents to 2 cents for 2019 in the past 90 days. The company delivered a positive earnings surprise in three of the trailing four quarters by 109.17%, on average.

AquaBounty’s loss per share estimates have narrowed from 70 cents to 59 cents for 2019 and from 44 cents to 36 cents for 2020 in the past 60 days.

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