Ultragenyx Pharmaceutical Inc. develops and commercializes therapies for serious rare and ultra-rare genetic diseases. Company news commonly covers revenue from approved medicines such as Crysvita, Dojolvi, and Mepsevii, financial guidance, and updates to a portfolio of approved therapies and product candidates for diseases with high unmet medical need and clear biology.
Recurring developments include FDA interactions and clinical data for investigational programs such as UX111 for Sanfilippo syndrome type A, GTX-102 for Angelman syndrome, UX016 for GNE myopathy, and AAV gene therapy candidates for rare metabolic disorders. Updates also include healthcare conference participation, employment inducement equity grants under Nasdaq rules, and corporate restructuring or expense-management actions when disclosed.
Ultragenyx (NASDAQ: RARE) announced that Chief Medical Officer Eric Crombez, M.D. will participate in fireside chats at two investor conferences in early December 2025: Citi’s 2025 Global Healthcare Conference in Miami on December 2, 2025 and the 8th Annual Evercore ISI Healthcare Conference in Coral Gables on December 3, 2025.
The company said live and archived webcasts of both presentations will be available on its investor website at https://ir.ultragenyx.com/events-presentations. Contact for investors is Joshua Higa at ir@ultragenyx.com.
Ultragenyx (NASDAQ: RARE) granted 18,180 restricted stock units (RSUs) to 12 newly hired non-executive officers under the Ultragenyx Employment Inducement Plan.
The awards were approved by the compensation committee, granted with a November 16, 2025 grant date and issued as an inducement under Nasdaq Listing Rule 5635(c)(4). The RSUs vest over four years with 25% vesting on each anniversary subject to continued employment.
Ultragenyx (NASDAQ: RARE) reported third quarter 2025 results with $159.9M total revenue, a 15% increase year-over-year, including $111.9M from Crysvita and $24.3M from Dojolvi.
Operating expenses were $330.8M in Q3, driving a net loss of $180.4M (−$1.81 per share). Cash, cash equivalents and marketable securities were $447M as of Sept 30, 2025.
The company received $400M from selling an additional 25% U.S./Canada Crysvita royalty interest to OMERS (payments begin Jan 2028; total payments capped at 1.55x the 2025 purchase price). Ultragenyx reaffirmed 2025 revenue guidance of $640M–$670M and reiterated a path to GAAP profitability in 2027. Multiple late‑stage clinical readouts and BLA activities expected through 2026 are highlighted.
Ultragenyx (NASDAQ: RARE) sold an additional 25% of its North American Crysvita® (burosumab) royalty interest to OMERS for $400 million. OMERS will begin receiving the extra 25% of U.S. and Canada royalties starting January 2028. OMERS will continue to receive 30% of net sales after the 2022 cap is reached, and total payments to OMERS are capped at 1.55x the purchase price. Crysvita has treated >3,000 patients and generated >$4 billion cumulative U.S. and Canada sales. Proceeds are earmarked to fund four expected product launches and provide a payment holiday through January 2028 to bolster the balance sheet toward GAAP profitability in 2027.
Ultragenyx (NASDAQ: RARE) announced the first patient has been dosed in the Aurora study (NCT07157254) evaluating GTX-102 (apazunersen) in additional Angelman syndrome genotypes and age groups.
The global, open-label basket trial will enroll approximately 60 participants ages 1 to <65 across all genotypes, with four cohorts and a 48-week primary efficacy period; Cohort D uses a 2:1 randomization versus a No Treatment group that crosses over at Week 24. The fully enrolled Phase 3 Aspire study completed enrollment at 129 participants (ages 4–17) and Aspire data are expected in H2 2026.
Ultragenyx (NASDAQ: RARE) will host a conference call at 5:00 p.m. ET on Tuesday, November 4, 2025 to discuss third quarter 2025 financial results and a corporate update for the period ended September 30, 2025.
The live webcast and replay will be available at https://ir.ultragenyx.com/events-presentations. The replay will remain available for three months.
Ultragenyx (NASDAQ: RARE) reported an inducement grant of 20,317 restricted stock units (RSUs) to 17 newly hired non-executive officers, approved by the compensation committee and granted under the Ultragenyx Employment Inducement Plan.
The grant date was October 16, 2025 and the awards were disclosed October 24, 2025 as material inducements under Nasdaq Listing Rule 5635(c)(4). The RSUs vest over four years with 25% vesting on each anniversary, contingent on continuous employment.
Ultragenyx (NASDAQ: RARE) reported an inducement equity grant under Nasdaq Listing Rule 5635(c)(4) for a newly hired non-executive officer, with a grant date of September 30, 2025.
The company granted 62,232 non-qualified stock options and 34,564 restricted stock units. The options have a ten-year term and an exercise price of $30.08, equal to the closing price on September 30, 2025. RSUs vest 25% annually over four years; options vest 25% after one year then monthly over the remaining three years (1/48th monthly), subject to continued employment.
Ultragenyx Pharmaceutical (NASDAQ: RARE) has appointed Eric Olson as Chief Business Officer and Executive Vice President, effective September 22, 2025. Olson succeeds Thomas Kassberg, who is retiring after 14 years with the company.
Olson brings nearly two decades of biopharma industry experience, having led or supported transactions worth over $15 billion. His previous roles include CBO at Stoke Therapeutics, VP of Business Development at Alnylam Pharmaceuticals, and leadership positions at Takeda and Genzyme Corporation. Notable deals include Stoke's partnership with Biogen and Alnylam's $2.8 billion collaboration with Roche.
In his new role, Olson will lead Ultragenyx's business development, corporate development, and alliance management functions, supporting the company's mission to develop therapies for untreated rare diseases.
Ultragenyx Pharmaceutical (NASDAQ: RARE), a biopharmaceutical company specializing in rare and ultra-rare disease therapies, has announced an inducement grant of 48,561 restricted stock units to 17 newly hired non-executive officers. The grants were approved by the company's compensation committee under the Ultragenyx Employment Inducement Plan on September 16, 2025.
The restricted stock units feature a four-year vesting schedule, with 25% of shares vesting annually on the grant date anniversary, contingent on continued employment. This grant complies with Nasdaq Listing Rule 5635(c)(4) for employment inducement awards.
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