Amicus Therapeutics Building Rare Disease Treatment Strategy; Bank Of America Initiates At Buy

Bank of America's Peter Stapor initiated a Buy rating on Amicus Therapeutics, Inc. (NASDAQ: FOLD) with a price target of $10.00.

The leading drug in the company's portfolio is migalastat (Galafold) for a rare disease (Fabry disease), which results in enzyme deficiency and can lead to organ failure. Migalastat is a convenient alternative to other treatments as it can be taken orally. It is pending approval from Europe's Committee for Medicinal Products for Human Use.

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The acquisition of Scioderm added SD-101 (Zorblisa) to the company's portfolio. Zorblisa is in its phase three trial. It is being developed to treat Epidermolysis Bullosa (EB) a rare disease that affects infants and results in fragile skin and blisters and open wounds. These symptoms increase risk for complications such as sepsis and even squamous cell carcinoma. There are no approved treatments, and Zorblisa would be able to corner this market quickly. Its "phase 2b data was encouraging" and "doctors indicate Zorblisa could have wide use," Stapor noted.

Amicus is developing a compound to treat a third rare genetic disease (Pompe). Phase I/II is expected this year. Amicus Therapeutics is building a strong platform in rare disease treatment and with few to no drugs to compete against the upsides of this strategy seem large.