Welcome to our dedicated page for Ultragenyx Pharm SEC filings (Ticker: RARE), a comprehensive resource for investors and traders seeking official regulatory documents including 10-K annual reports, 10-Q quarterly earnings, 8-K material events, and insider trading forms.
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.’s Chief Financial Officer, Howard Horn, sold 4,683 shares of common stock in an open-market transaction. The shares were sold at $20.97 each. After this sale, he directly holds 80,351 shares, which include previously reported RSU-based shares that vest over time.
Ultragenyx Pharmaceutical Inc. reported that the U.S. FDA has accepted for review its resubmitted Biologics License Application seeking accelerated approval of UX111 (rebisufligene etisparvovec), an AAV9 gene therapy for Sanfilippo syndrome Type A (MPS IIIA). The FDA set a Prescription Drug User Fee Act (PDUFA) action date of September 19, 2026 for this application.
Howard Horn reported multiple sales of Common stock under a Form 144 notice. The filing lists five dispositions dated between 01/02/2026 and 03/03/2026, with individual share amounts and gross proceeds for each sale.
The transactions are shown as sales of previously issued restricted or performance-based shares originating from the issuer, with each row listing the sale date, number of shares, and dollar proceeds.
Ultragenyx Pharmaceutical Inc. announced that the FDA has cleared its Investigational New Drug application for UX016, a small molecule prodrug of sialic acid being developed as a substrate replacement therapy for GNE myopathy, a rare inherited neuromuscular disorder.
The UX016 program is externally funded by a patient group through clinical proof-of-concept, including a planned first-in-human Phase 1/2 study in the United States. This trial, expected to start in the second half of 2026, will enroll about 24 adults with GNE myopathy to evaluate safety, efficacy, pharmacokinetics, and muscle delivery over up to 48 weeks of treatment.
Ultragenyx Pharmaceutical Inc. is asking stockholders to approve four key items at its virtual annual meeting on May 14, 2026. Investors will vote on re-electing three Class I directors, including founder and CEO Emil Kakkis, to serve until the 2029 meeting.
Stockholders are also asked to approve the Third Amended and Restated 2023 Incentive Plan, which adds 3.0 million shares and brings the plan’s share authorization to 14.5 million, extending it through May 14, 2036. The company reports a three-year average equity burn rate of 5.62% and an expected overhang of about 21% as of March 9, 2026.
Other proposals include ratifying Ernst & Young LLP as independent auditor for 2026 and an advisory say-on-pay vote on executive compensation, which emphasizes performance-based awards such as PSUs. The proxy highlights a largely independent, biopharma-experienced board and a range of governance practices, including a clawback policy, anti-hedging and anti-pledging rules, and director overboarding limits.
Ultragenyx Pharmaceutical Inc ownership disclosure: The Vanguard Group filed Amendment No. 10 to a Schedule 13G/A describing an internal realignment and reporting disaggregated holdings. The amendment states 0 shares beneficially owned and 0% of the class as of the filing, with subsidiaries reporting separately in reliance on SEC Release No. 34-39538.
Ultragenyx Pharmaceutical Inc. reported positive Phase 3 Enh3ance results for DTX301, its investigational AAV8 gene therapy for ornithine transcarbamylase (OTC) deficiency. At Week 36, DTX301-treated patients showed an 18% reduction in 24-hour plasma ammonia AUC0-24 versus placebo, with ammonia levels generally maintained in the normal range.
Treated patients reduced ammonia scavenger medications by a mean 27% and increased protein intake by about 13%, while still keeping ammonia controlled. Patient-reported outcomes at Week 24 showed notably higher rates of symptom improvement with DTX301 compared to placebo. DTX301 was described as well tolerated, with most adverse events being mild to moderate hepatic reactions managed with steroids.
The trial continues to a second primary endpoint assessing reduction in treatment burden through 64 weeks, with additional data expected in the first half of 2027. Ultragenyx states that the program fits within its 2026 spending guidance and its goal of achieving profitability in 2027.
Ultragenyx Pharmaceutical Inc.’s EVP and Chief Legal Officer, Karah Herdman Parschauer, reported open-market sales of company common stock. She sold 6,018 shares on March 6, 2026 at $21.40 per share and 3,508 shares on March 10, 2026 at $22.67 per share, for total reported sales of 9,526 shares. Following these transactions, she directly holds 66,820 shares of common stock, which include previously reported shares underlying restricted stock units that are subject to vesting conditions.
RARE filed a Form 144 reporting a proposed sale of 3,508 shares of Common Stock (restricted stock) by an issuer-affiliated holder. The filing also lists recent dispositions by Karah Parschauer, including sales of 6,018 shares on 03/06/2026 and 8,135 shares on 03/02/2026.