10 Best Mid Cap Biotech Stocks to Buy

3. Ultragenyx Pharmaceutical, Inc. (NASDAQ:RARE)

Market Cap: $3.58 billion

Number of Hedge Fund Holders: 57

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.

Ultragenyx Pharmaceutical, Inc. (NASDAQ:RARE) plans to expand its commercial business base in 2025, supported by several products in launch mode worldwide. This progress sets the company on a path to full-year GAAP profitability in 2027. It also exceeded expectations with its 2024 results, reporting a total revenue of $560 million. The company expects 2025 total revenue to be between $640 million and $670 million.

Piper Sandler analyst Allison Bratzel maintained a Buy rating on Ultragenyx Pharmaceutical, Inc. (NASDAQ:RARE) on March 17 and set a price target of $115.00. J.P. Morgan analyst Anupam Rama also maintained a Buy rating on the company on March 6, setting a price target of $104.00. Analysts are bullish on the stock, and its median price target of $38.69 implies an upside of 124.86% from current levels.

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.”