Ultragenyx Pharmaceutical Inc. filings document a rare-disease biopharmaceutical business with approved medicines, investigational genetic-disease therapies, and a Nasdaq-listed common stock capital structure. Form 8-K reports include operating results, financial guidance, FDA communications, clinical-study data, and risk language tied to drug development, regulatory review, manufacturing, funding needs, competition, and commercialization.
Proxy materials describe annual-meeting matters, board and compensation governance, equity compensation, and stockholder voting. Recent current reports also record restructuring and cost disclosures, employment-related equity plans, and material updates for programs including UX111, UX016, DTX301, DTX401, and GTX-102.
Ultragenyx Pharmaceutical Inc. (RARE) has an officer, Theodore Huizenga, filing a notice to sell common stock under Rule 144. The notice covers 1,283 shares of common stock, with an indicated aggregate market value of $17,461.63 as of September 15, 2026, to be sold through Morgan Stanley Smith Barney LLC on NASDAQ.
The shares derive from 252 shares issued as Performance Stock Units dated October 14, 2020, 817 shares from Restricted Stock Units dated May 25, 2020, and 214 shares from the Employee Stock Purchase Plan dated October 31, 2020. The filing also reports that Huizenga sold 161 shares of common stock in the past three months on September 3, 2026, for $2,252.39.
Ultragenyx Pharmaceutical Inc. (RARE) reports that Theodore Alan Huizenga, its SVP and Chief Accounting Officer, reported a sale of 161 shares of common stock on September 3, 2026 at $13.99 per share. The shares were sold to pay required tax withholdings from vesting RSUs, and he now holds 69,146 shares directly, including 396 shares acquired under the company’s Amended and Restated Employee Stock Purchase Plan and previously reported unvested RSUs. No Rule 10b5-1 trading plan is reported.
Ultragenyx Pharmaceutical Inc. (RARE) reported that its Phase 3 Aspire study of apazunersen (GTX-102) in Angelman syndrome did not meet the primary endpoint of change from Baseline in Bayley-4 cognitive raw score or the key secondary endpoint of net response in the Multidomain Responder Index. The safety profile in Aspire was consistent with the earlier Phase 1/2 study, and randomized groups were comparable at baseline. Ultragenyx plans to evaluate the future of the apazunersen program in light of these results and to assess its planned operations to define and implement significant expense reductions while continuing to support its growing commercial business.
Ultragenyx Pharmaceutical Inc. (RARE) reported that the U.S. Food and Drug Administration granted accelerated approval for GENGLYCOS™ (pariglasgene brecaparvovec-opnr, DTX401) to treat adult and pediatric patients eight years and older with glycogen storage disease type Ia (GSDIa). Approval is based on a 48-week randomized, double-blind, placebo-controlled Phase 3 study in 46 participants, which showed a statistically significant reduction in cornstarch requirements for DTX401-treated patients at a 1.0 x 10^13 GC/kg dose (p<0.001). Ultragenyx agreed to a post-marketing program that will follow 50 commercially treated patients and 20 control patients for at least 2 years, with broader disease monitoring over 10 years to further characterize safety, fasting tolerance, and other clinical outcomes.
RTW Investments, LP, as investment adviser to certain funds, and Roderick Wong, M.D. report beneficial ownership of Ultragenyx Pharmaceutical Inc. common stock. They report 7,796,895 Shares of common stock, representing 7.9% of the class, based on 98,489,224 Shares outstanding as of May 1, 2026.
Both RTW Investments and Dr. Wong report 0 Shares with sole voting or dispositive power and 7,796,895 Shares with shared voting and shared dispositive power. The RTW funds have the right to receive dividends and sale proceeds on these Shares. The reporting persons state that the filing is not an admission of beneficial ownership for Section 13 purposes.
AQR Capital Management, LLC and AQR Capital Management Holdings, LLC report passive ownership of Ultragenyx Pharmaceutical Inc. common stock. They beneficially own 6,560,809 shares, representing 6.66% of Ultragenyx’s common stock.
The AQR entities report shared voting power over 6,398,809 shares and shared dispositive power over 6,560,809 shares, with no sole voting or dispositive power. AQR Capital Management, LLC is a wholly owned subsidiary of AQR Capital Management Holdings, LLC, and the Schedule is filed on behalf of both entities.
Ultragenyx Pharmaceutical Inc. reports that it and Baylor Research Institute entered into a settlement agreement with Esjay Pharma regarding patent litigation over DOJOLVI® (triheptanoin) oral liquid. Esjay had filed an Abbreviated New Drug Application seeking approval to market a generic DOJOLVI in the United States.
Under the settlement, Ultragenyx will grant Esjay a non-exclusive, royalty-free license to market its generic version of DOJOLVI starting January 1, 2033, extendable to July 1, 2033 if pediatric exclusivity is granted, subject to customary conditions and exceptions. Similar litigation with other ANDA filers remains pending, and the agreement will be submitted to the FTC and Department of Justice.
State Street Corporation reported beneficial ownership of 6,158,766 shares of Ultragenyx Pharmaceutical Inc. common stock on a Schedule 13G. This represents 6.3% of the class.
State Street reports 0 shares with sole voting or dispositive power, and 5,871,222 shares with shared voting power and 6,158,766 shares with shared dispositive power through its investment management subsidiaries.
Ultragenyx Pharmaceutical Inc. reported Q2 2026 revenue of $214 million, up 28% from Q2 2025, driven by higher Crysvita product and royalty revenue and expanding sales of Dojolvi, Evkeeza, and Mepsevii. Product sales were $112 million and Crysvita royalty revenue was $102 million in the quarter.
Net loss for Q2 2026 narrowed to $92 million (basic and diluted net loss per share $0.90) from $115 million a year earlier as revenue growth outpaced operating expense increases. For the first six months of 2026, revenue was $350 million and net loss was $277 million. Cash, cash equivalents and marketable securities totaled $436 million as of June 30, 2026, with $294 million used in operating activities in the first half. Liabilities for sales of future Crysvita royalties were $1.20 billion, contributing to a stockholders’ deficit of $291 million. A February 2026 restructuring reduced headcount by about 10% and generated $30 million in charges, all settled by June 30, 2026. Key near-term catalysts include FDA Priority Review decisions for DTX401 (PDUFA August 23, 2026) and UX111 (PDUFA September 19, 2026), and pivotal GTX-102 data expected in late 2026; management states expectations for profitability in 2027.
Ultragenyx Pharmaceutical Inc. reported second quarter 2026 results, with total revenue of $214 million, up from $167 million a year earlier and the highest quarterly revenue in its history. Key contributors were Crysvita at $156 million, Dojolvi at $27 million, Evkeeza at $21 million and Mepsevii at $10 million.
The company recorded a net loss of $92 million, or $0.90 per share, compared with a net loss of $115 million, or $1.17 per share, in second quarter 2025. Cash, cash equivalents and marketable securities were $436 million as of June 30, 2026, with $97 million of net cash used in operations in the quarter.
Ultragenyx reaffirmed 2026 guidance for total revenue of $730 million to $760 million, Crysvita revenue of $500 million to $520 million, and Dojolvi revenue of $100 million to $110 million, and continues to target profitability in 2027. Upcoming catalysts include FDA PDUFA action dates for DTX401 on August 23, 2026 and UX111 on September 19, 2026, plus a pivotal Phase 3 GTX-102 data readout in Angelman syndrome.