Amicus Progresses On Pipeline Development Amid Competition

We issued an updated report on Amicus Therapeutics, Inc. (NASDAQ:FOLD) on Jan 2, 2020.

The company's only marketed drug, Galafold, is approved for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene ("GLA") variant. The company realized revenues from sales Galafold in the third quarter of 2019.Owing to success in the first three quarters of the year and the strength in global Galafold launch metrics across all major geographies, Amicus raised the low end of the 2019 Galafold revenue guidance from $160-$180 million to $170-$180 million during the third quarter of 2019. The company expects to have almost double annual worldwide revenues, with more than 1,000 Fabry patients on Galafold, by the end of 2019.

Shares of the company have lost 12.3% in the past year against the industry’s growth of 3.5%.

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, 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 plans to enhance the body of clinical data for AT-GAA in ongoing clinical studies, including the pivotal phase III PROPEL study. Based on regulatory feedback from both the FDA and the European Medicines Agency (EMA), the PROPEL study is expected to support the approval of the candidate for a broad indication, including ERT-switch and treatment-naïve patients.

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 entered into 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.

However, the company faces substantial competition Several large pharmaceutical and biotechnology companies currently market and sell products for treating lysosomal storage disorders, including Fabry disease. These products include Sanofi (PA:SASY) Aventis’ (NASDAQ:SNY) Fabrazyme and Takeda's Replagal plus other Fabry treatment products in development. Additionally, Sanofi markets and sells Myozyme and Lumizyme for the treatment of Pompe disease.

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. (NASDAQ:AGEN) and Alexion Pharmaceuticals Inc. (NASDAQ:ALXN) . While Agenus sports a Zacks Rank #1 (Strong Buy), Alexion carries a Zacks Rank #2 (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.

Agenus’ loss per share estimates have narrowed from $1.44 to $1.41 for for 2020 in the past 60 days. The company delivered a positive earnings surprise in two of the trailing four quarters by 23.79%, on average.

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