What Makes Ultragenyx Pharmaceutical (RARE) an Exciting Investment Opportunity?

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Baron Funds, an investment management company, released its “Baron Health Care Fund” third quarter 2024 investor letter. A copy of the letter can be downloaded here. The fund appreciated 5.81%% (Institutional Shares) in the quarter compared to a 6.74% gain for the Russell 3000 Health Care Index (benchmark) and a 6.23% gain for the Russell 3000 Index (the Index). Since inception, the fund appreciated 13.02% on an annualized basis compared to an 11.47% gain for the benchmark and a 14.03% gain for the index. In addition, please check the fund’s top five holdings to know its best picks in 2024.

Baron Health Care Fund highlighted stocks like Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) in the Q3 2024 investor letter. Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) is a biopharmaceutical company that focuses on the treatment of rare and ultra-rare genetic diseases. The one-month return of Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) was -4.98%, and its shares gained 34.93% of their value over the last 52 weeks. On October 31, 2024, Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) stock closed at $50.99 per share with a market capitalization of $4.7 billion.

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."