Ultragenyx Pharmaceutical, Inc. (RARE)

Ultragenyx Pharmaceutical's stock has been impacted by several events over the past six months. The company reported its second-quarter 2025 financial results, showing revenue growth and reaffirmed its 2025 revenue guidance. The Phase III Orbit study failed to achieve statistical significance, leading to a stock price drop. GTX-102 for Angelman syndrome received Breakthrough Therapy Designation from the FDA. Both Phase III Orbit and Cosmic Studies for UX143 did not achieve statistical significance, causing the stock price to fall. Ultragenyx announced preliminary unaudited total revenue for 2025, surpassing its previous guidance. The company resubmitted its BLA for UX111 gene therapy for Sanfilippo syndrome type A to the FDA. Ultragenyx announced positive longer-term data from clinical studies evaluating UX111. The company's CFO, Howard Horn, engaged in stock sales. As of early February 2026, the stock has seen a 4.88% increase over the past month and a 7.62% year-to-date return, but a 28.46% decrease over the last three months.

Information regarding specific demand seasonality for Ultragenyx Pharmaceutical, Inc.'s products and services is not explicitly available. Demand is typically driven by diagnosis and medical need rather than seasonal consumer patterns.

Ultragenyx Pharmaceutical, Inc. is a biopharmaceutical company focused on researching, developing, and commercializing treatments for rare and ultra-rare genetic diseases. The company operates within the Biotechnology and Life Sciences sector, utilizing drug modalities like biologics, small molecules, gene therapies, ASOs, and mRNAs. Its approved products include Crysvita for XLH and TIO, Mepsevii for MPSVII, Dojolvi for LC-FAOD, and Evkeeza for HoFH. The company's clinical pipeline includes UX111 for Sanfilippo syndrome type A, UX143 for Osteogenesis Imperfecta, DTX401, DTX301, UX701, and GTX-102 for Angelman syndrome.

Ultragenyx Pharmaceutical Inc. is headquartered in Novato and Brisbane, California, with a presence in the Boston area and a Latin American headquarters in Miami. The company serves international markets, with therapies approved in the U.S., Canada, Latin America, Europe, and Japan. Its focus extends to North America, Latin America, Europe, the Middle East, Africa, and the Asia-Pacific regions.

Ultragenyx has maintained a positive brand reputation over the past year. In 2024, Ultragenyx was named one of Barron's 100 Most Sustainable Companies and recognized as one of the Top Places to Work in the USA for the second consecutive year. The company also received accolades from The Boston Globe and the San Francisco Chronicle in 2024. Ultragenyx was honored with the MassEcon Gold Award for Economic Impact in the Northeast region and recognized as one of the Best Places for Working Parents in Massachusetts. In May 2025, Ultragenyx released its 2024 Impact Report, detailing its approach to corporate responsibility.

Approximately 44.39% of Ultragenyx Pharmaceutical Inc.'s stock is held by institutional investors, 3.99% by insiders, and 51.62% by public companies and individual investors. Major institutional owners include Vanguard Group Inc., Fmr Llc, BlackRock, Inc., JPMorgan Chase & Co, Sands Capital Management, Llc, State Street Corp, Rtw Investments, Lp, VTSMX - Vanguard Total Stock Market Index Fund Investor Shares, Baker Bros. Advisors Lp, and Price T Rowe Associates Inc /md/.