Welcome to our dedicated page for Ultragenyx Pharm news (Ticker: RARE), a resource for investors and traders seeking the latest updates and insights on Ultragenyx Pharm stock.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) is a biopharmaceutical company focused on novel therapies for serious rare and ultra-rare genetic diseases. The RARE news feed highlights company announcements on commercial performance, clinical development, and key corporate decisions that shape its rare disease portfolio.
Investors and followers of Ultragenyx can find updates on revenue trends from its approved products Crysvita, Dojolvi, Mepsevii, and Evkeeza in select territories, as well as guidance ranges and commentary on operating expenses and cash position. Earnings-related press releases and Form 8-K summaries detail product sales, royalty revenue, and royalty financing transactions, including agreements involving future Crysvita royalties with OMERS.
The news stream also covers Ultragenyx’s late-stage pipeline and regulatory milestones. Readers can track progress of AAV gene therapy programs such as DTX401 for glycogen storage disease type Ia, UX111 for Sanfilippo syndrome type A, and UX701 for Wilson disease, along with monoclonal antibody UX143 for osteogenesis imperfecta and antisense oligonucleotide GTX-102 for Angelman syndrome. Company releases describe Phase 3 study results, longer-term follow-up data, regulatory designations, rolling BLA submissions, and responses to FDA communications such as Complete Response Letters.
In addition, the RARE news page features items on conference presentations, investor events, and equity inducement grants under Nasdaq Listing Rule 5635(c)(4). By reviewing these updates, users can follow how Ultragenyx manages its commercial portfolio, advances its clinical pipeline, and executes financing and collaboration strategies in the rare and ultra-rare disease space.
Ultragenyx (NASDAQ: RARE) reported Phase 3 results for setrusumab (UX143) in osteogenesis imperfecta from the Orbit and Cosmic studies dated Dec 29, 2025. Neither trial met the primary endpoint of a statistically significant reduction in annualized clinical fracture rate versus placebo (Orbit) or bisphosphonates (Cosmic). Both studies did achieve the secondary endpoint of statistically significant improvements in bone mineral density (BMD) versus comparators. Safety findings showed no change in the safety profile. Ultragenyx said it will implement significant expense reductions while performing additional analyses of other bone-health and clinical endpoints to define next steps for the program.
Ultragenyx (NASDAQ: RARE) granted 13,144 restricted stock units to nine newly hired non-executive officers under the Ultragenyx Employment Inducement Plan.
The awards were approved by the compensation committee with a grant date of December 17, 2025 and reported December 19, 2025, as an inducement under Nasdaq Listing Rule 5635(c)(4). The RSUs vest over four years with 25% vesting each anniversary, subject to continuous employment on each vesting date.
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.