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.
The Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) SEC filings page provides access to the company’s official disclosures filed with the U.S. Securities and Exchange Commission. These documents include current reports on Form 8-K, quarterly and annual reports, and other filings that describe material agreements, financial results, and key developments in Ultragenyx’s rare disease business.
Form 8-K filings for Ultragenyx often cover topics such as royalty purchase agreements, clinical and regulatory milestones, and financial updates. For example, the company has reported a Royalty Purchase Agreement with an OMERS investment vehicle involving a portion of future Crysvita royalty payments in the United States and Canada, including details on purchase price, royalty percentages, and caps based on multiples of the purchase price. Other 8-Ks summarize press releases announcing quarterly financial results, revenue by product, operating expenses, and net loss.
Ultragenyx also uses Form 8-K to report clinical and regulatory events, including initiation and progress of rolling Biologics License Application submissions for DTX401 in glycogen storage disease type Ia, longer-term Phase 3 data for DTX401, Breakthrough Therapy Designation for GTX-102 in Angelman syndrome, enrollment status of the Phase 3 Aspire study, and FDA communications such as a Complete Response Letter for UX111 in Sanfilippo syndrome type A. These filings outline requested chemistry, manufacturing, and controls information and the company’s plans to address regulatory feedback.
Through Stock Titan, users can review Ultragenyx’s SEC filings and benefit from AI-powered summaries that explain the significance of complex documents, including 10-K and 10-Q reports when available, as well as Form 4 insider transaction reports. Real-time updates from EDGAR combined with AI-generated insights help readers understand how material agreements, clinical data, and regulatory interactions influence Ultragenyx’s operations and capital structure in the rare and ultra-rare genetic disease space.
Ultragenyx Pharmaceutical Inc. filed a current report to note that it has issued a press release with preliminary unaudited fiscal year 2025 results. The release covers total revenue for the year, as well as revenue contributions from its key products Crysvita® and Dojolvi®, plus the company’s cash and investment balances at the end of the 2025 fiscal year. Ultragenyx plans to report full audited results for the 2025 fiscal year in February 2026. The company specifies that this preliminary financial information and the accompanying press release are being furnished rather than filed under securities laws.
Ultragenyx Pharmaceutical (RARE) reported higher Q3 revenue and a wider loss. Total revenues were $159.9 million, up from $139.5 million a year ago, driven by product sales of $95.0 million and Crysvita royalties of $64.9 million. Crysvita revenue across product sales and royalties reached $111.9 million.
The company posted a net loss of $180.4 million (basic and diluted loss per share $1.81) versus a loss of $133.5 million last year, reflecting increased R&D and SG&A. For the first nine months, revenue was $465.7 million and net loss was $446.4 million.
Cash and cash equivalents were $202.5 million, with $244.8 million of marketable debt securities (current and non‑current). Total assets were $1.19 billion against total liabilities of $1.17 billion, leaving stockholders’ equity at $9.2 million. Liabilities for sales of future royalties totaled $829.3 million. As of October 30, 2025, 96,477,569 shares of common stock were outstanding.
Ultragenyx (RARE) entered a Royalty Purchase Agreement with an OMERS affiliate, receiving
The arrangement ends when OMERS has received 1.55 times the purchase price (
Ultragenyx reported that the first patient has been dosed in the global Aurora study (NCT07157254) evaluating GTX-102 (apazunersen), an investigational antisense oligonucleotide for Angelman syndrome.
Aurora plans to enroll approximately 60 participants ages one to less than 65 across all genotypes in four cohorts. Cohorts A–C are single-arm; Cohort D is randomized 2:1 to GTX-102 or No Treatment, with a 48-week primary efficacy period and crossover at Week 24. Participants may continue in a long-term extension after study completion.
The Phase 3 Aspire study (NCT06617429) is fully enrolled, with 129 participants ages four to 17 with full maternal UBE3A gene deletion randomized across 28 global sites. Data from Aspire are expected in the second half of 2026.
Ultragenyx Pharmaceutical (RARE) insider activity: The company’s Chief Financial Officer reported a sale of common stock on 10/13/2025. The filing shows 7,942 shares were sold at $31.51 per share.
According to the explanation, these shares were sold to pay required tax withholdings related to the vesting of restricted stock units (RSUs). Following the transaction, the reporting person beneficially owned 98,227 shares. The ownership is listed as direct.
The filing notes that the remaining amount includes previously reported shares underlying RSUs that are subject to vesting conditions.
Theodore Alan Huizenga, SVP and Chief Accounting Officer and director of Ultragenyx Pharmaceutical Inc. (RARE), reported a sale of 64 shares of common stock on 09/18/2025 at a price of $29.17 per share. The filing states the shares were sold to pay required tax withholdings related to the vesting of restricted stock units (RSUs). After the transaction, the reporting person beneficially owned 50,242 shares. The Form 4 was signed by an attorney-in-fact on 09/19/2025.
Theodore A. Huizenga, SVP and Chief Accounting Officer of Ultragenyx Pharmaceutical Inc. (RARE), reported a sale of company common stock on 09/05/2025. The Form 4 shows a sale (Code S) of 84 shares at $31.39 per share. After the reported transaction the reporting person beneficially owned 50,306 shares directly. The filing explains the sale represents shares sold to pay required tax withholdings upon vesting of restricted stock units, and also notes the reporting person acquired 266 shares under the company’s Employee Stock Purchase Plan on April 30, 2025. Previously reported RSU-related shares subject to vesting are included in the total.
Ultragenyx Pharmaceutical Inc. reported that it has begun a rolling submission of a Biologics License Application to the U.S. FDA for DTX401, an AAV gene therapy for Glycogen Storage Disease Type Ia. The company has submitted the non-clinical and clinical modules and plans to complete the full application, including the chemistry, manufacturing and controls module, in the fourth quarter of 2025.
The BLA will include 96-week data from a Phase 3 trial showing patients had greater reductions in total daily cornstarch from baseline at their last visit, with a -60% reduction in the ongoing DTX401 group and -64% in the Crossover Placebo to DTX401 group compared to the 48-week data. Ultragenyx also plans to incorporate updates in the CMC section to proactively address FDA observations from the UX111 complete response letter and at its gene therapy manufacturing facilities.
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