7. Ultragenyx Pharmaceutical, Inc. (NASDAQ:RARE)
Analyst Upside: 97.57%
Number of Hedge Fund Holders: 41
Ultragenyx Pharmaceutical, Inc. (NASDAQ:RARE) develops, acquires, and commercializes novel products to treat genetic diseases. Its product portfolio includes Crysvita, Mepsevii, Dojolvi, and Evkeeza. In 2024, the company expanded its business through four products in five indications worldwide, exceeding analyst expectations in terms of revenue. It estimates its total revenue for fiscal 2024 to be $555 million to $560 million, surpassing its guidance range and reflecting a 29% growth compared to 2023.
The company has plans to expand its commercial business base in 2025 and prepare for the potential launch of its first gene therapy for Sanfilippo syndrome. It is also planning to file a Biologics License Application (BLA) for its second gene therapy, in Glycogen Storage Disease Type Ia.
Ultragenyx Pharmaceutical, Inc., (NASDAQ:RARE) is executing a valuable late-stage pipeline in rare diseases. It anticipates pivotal Phase 3 results in osteogenesis imperfecta and is also completing the enrollment in its Phase 3 trial for Angelman syndrome. Progress across these avenues gives the company a competitive advantage and positions it to launch three to four new therapies over the next few years. This would translate to the company accumulating 8-9 approved products over a 10-year period, reflecting its solid operations.
Baron Health Care Fund stated the following regarding Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) in its Q3 2024 investor letter:
“We purchased Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on developing treatments for rare genetic diseases. Impressively, the company has gotten 4 drugs approved across 5 indications in 10 years, and it has a large clinical pipeline with several potential blockbuster opportunities in late stage development. While the company’s approved products continue to grow 20%-plus, we are most excited about the company’s new product pipeline. Setrusumab is in Phase 3 studies for Osteogenesis Imperfecta, a rare genetic disorder that causes bones to break easily. The drug helps patients increase bone mineral density and reduces the number of fractures patients experience. We think this could be transformative for patients and could be a $1 billion-plus peak sales drug. We are also excited about Ultragenyx’s GTX-102, an antisense oligonucleotide that treats Angelman Syndrome, a rare genetic disorder that affects the nervous system and causes severe development delay and intellectual disability. Early data showed dramatic improvement for patients across several behavioral and cognitive endpoints, and Ultragenyx just started a registrational study. The company is also working on a drug for Wilson disease, a rare genetic disorder that causes copper to build up in the body, where we will get proof-of-concept data soon. We think each of these programs has potential for significant value creation, transforming the company to one with significantly higher revenue and profits.”
