6. Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE)
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) is one of the 10 best biotech stocks with highest upside potential.
On April 16, Morgan Stanley increased the price target for Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) from $50 to $67, resulting in an adjusted upside potential in excess of 178% at the prevailing level. The firm also reiterated its Outperform rating on the stock.
Morgan Stanley revised the price target upward based on a strict new probability-of-success approach to the forthcoming GTX-102 Phase 3 Angelman trial. This major clinical readout is anticipated in the second half of 2026. The resulting risk profile is rather asymmetrical. The firm has projected a 50% to 70% upside, which significantly offsets a 20% downside risk of a clinical failure.
Back on March 30, Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) announced clearance from the US Food & Drug Administration for the Investigational New Drug Application for UX016. It is a prodrug of sialic acid under investigation for use as a substrate replacement therapy for GNE Myopathy. GNEM is a rare neuromuscular disease characterized by severe disability due to mutations in the GNE gene, which impairs sialic acid biosynthesis.
The UX016 program is supported externally via proof-of-concept clinical trials, which include a Phase 1/2 trial scheduled for initiation in the latter half of 2026. Commenting on the progress of UX016, CEO Emil Kakkis observed that moving the drug into the clinic is a significant step for the GNE myopathy community, and it demonstrates the company’s effort to create a novel method for improving sialic acid utilization by the muscles. The first-in-human study will involve around 24 patients between 18 and 55 years of age suffering from GNEM, who will be recruited in the US.
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) develops novel therapies, with a focus on identifying, acquiring, and commercializing products for rare and ultra-rare genetic diseases. It has a strong emphasis on gene therapy and covers various stages of clinical trials. The company is currently going through high cash burn with the aim of turning profitable from 2027 onwards.
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