Ultragenyx Provides Financial and Business Updates at J.P. Morgan Annual Healthcare Conference

Rhea-AI Summary

Ultragenyx (NASDAQ: RARE) reported preliminary unaudited 2025 results and a corporate update at J.P. Morgan Healthcare Conference on January 12, 2026. Preliminary 2025 total revenue was $672M–$674M, roughly +20% vs 2024, with Crysvita revenue $480M–$482M (+17% vs 2024) and Dojolvi revenue $95M–$97M (+9% vs 2024). Cash and investments were ~ $735M at December 31, 2025. The company completed a rolling BLA submission for DTX401 and expects a PDUFA in Q3 2026; UX111 BLA resubmission is planned for early 2026 with up to a 6-month review. Key 2026 catalysts include potential approvals for two gene therapies and pivotal Phase 3 Aspire data for GTX-102 in Angelman syndrome in H2 2026.

Positive

• Preliminary total revenue of $672M–$674M (~+20% vs 2024)
• Crysvita revenue $480M–$482M (~+17% vs 2024)
• Cash and investments of approximately $735M as of Dec 31, 2025
• Completed rolling BLA for DTX401; anticipated PDUFA Q3 2026

Negative

• UX111 BLA requires resubmission and up to a 6-month FDA review
• Preliminary figures are unaudited and subject to adjustment
• No full 2026 guidance yet; company will issue guidance including expense reductions in Feb 2026

News Market Reaction – RARE

+8.26%

14 alerts

+8.26% News Effect

+$181M Valuation Impact

$2.38B Market Cap

0.8x Rel. Volume

On the day this news was published, RARE gained 8.26%, reflecting a notable positive market reaction. Our momentum scanner triggered 14 alerts that day, indicating notable trading interest and price volatility. This price movement added approximately $181M to the company's valuation, bringing the market cap to $2.38B at that time.

Data tracked by StockTitan Argus on the day of publication.

Key Figures

2025 total revenue: $672–$674M Revenue growth: 20% Crysvita revenue: $480–$482M +5 more

8 metrics

2025 total revenue $672–$674M Preliminary 2025, exceeding top end of guidance

Revenue growth 20% Approximate growth versus 2024 total revenue

Crysvita revenue $480–$482M Preliminary 2025, exceeds top end of guidance

Dojolvi revenue $95–$97M Preliminary 2025, around midpoint of guidance

Year-end cash & investments $735M Approximate cash and investments as of Dec 31, 2025

DTX401 PDUFA timing Q3 2026 Anticipated PDUFA date for DTX401 BLA

Aurora enrollment target 60 participants Planned enrollment for GTX-102 Aurora study

Aspire participants 129 participants Fully enrolled GTX-102 Phase 3 Aspire study

Market Reality Check

Price: $22.72 Vol: Volume 2,724,783 is below...

normal vol

$22.72 Last Close

Volume Volume 2,724,783 is below 20-day average 3,702,145 (relative volume 0.74). normal

Technical Price 22.51 is trading below 200-day MA at 32.75, reflecting a longer-term downtrend.

Peers on Argus

RARE was down 3.02% while momentum scanner peers were mixed: SLNO up 4.67% and A...

1 Up 1 Down

RARE was down 3.02% while momentum scanner peers were mixed: SLNO up 4.67% and APLS down 4.10%, suggesting today’s move was more stock-specific than a broad biotech rotation.

Common Catalyst Several biotech peers, including SLNO and SRRK, also issued business or results-focused updates on the same day, pointing to a cluster of company-specific catalysts rather than a unified sector driver.

Historical Context

5 past events · Latest: Jan 07 (Neutral)

Pattern 5 events

Date	Event	Sentiment	Move	Catalyst
Jan 07	Conference participation	Neutral	-4.1%	J.P. Morgan conference presentation and webcast availability announcement.
Dec 30	Regulatory submission	Positive	+1.0%	Completion of DTX401 BLA rolling submission to U.S. FDA.
Dec 29	Clinical trial results	Negative	-42.3%	UX143 Phase 3 Orbit and Cosmic studies missed primary fracture endpoints.
Dec 19	Equity compensation	Neutral	+3.7%	Grant of 13,144 RSUs to new non-executive officers.
Nov 24	Investor conferences	Neutral	+2.3%	Participation in December healthcare investor conferences with webcasts.

Pattern Detected

Recent major clinical setbacks have triggered sharp selloffs, while positive regulatory and routine corporate updates have seen modest or mixed price reactions.

Recent Company History

Over the last few months, Ultragenyx has combined clinical, regulatory, and corporate updates with varied market responses. A major setback came on Dec 29, 2025, when UX143 Phase 3 trials missed primary endpoints, and shares fell 42.32%. In contrast, completion of the DTX401 BLA submission on Dec 30, 2025 saw a modest 0.97% gain. Routine items like conference participation (Jan 7, 2026) and inducement grants (Dec 19, 2025) produced small, mixed moves. Today’s revenue beat and 2026 catalyst roadmap sit against this backdrop of heightened sensitivity to clinical outcomes.

Market Pulse Summary

The stock moved +8.3% in the session following this news. A strong positive reaction aligns with the...

Analysis

The stock moved +8.3% in the session following this news. A strong positive reaction aligns with the company’s preliminary 2025 revenue of $672–$674M exceeding guidance and highlighting roughly 20% growth versus 2024. The announcement also outlined multiple 2026 catalysts, including an anticipated DTX401 PDUFA decision in Q3 2026 and pivotal GTX-102 Phase 3 data. However, past trading shows sharp sensitivity to clinical outcomes, as seen with the 42.32% drop after UX143 results, so future data readouts could still drive significant volatility.

Key Terms

biologics license application, pdufa, aav gene therapy, antisense oligonucleotide, +3 more

7 terms

biologics license application regulatory

"Biologics License Application (BLA) rolling submission completed in December 2025"

A biologics license application is a formal request submitted to regulatory authorities seeking approval to market a new biological medicine, such as vaccines or treatments made from living organisms. It is a comprehensive review process that evaluates the safety, effectiveness, and manufacturing quality of the product. For investors, receiving approval signals that a biological therapy can be sold to the public, potentially leading to revenue growth and market success.

pdufa regulatory

"with an anticipated PDUFA date in the third quarter of 2026"

PDUFA, short for the Prescription Drug User Fee Act, is a law that allows drug companies to pay fees to the government to speed up the review process for new medicines. This helps bring important drugs to market more quickly, which can impact their availability and pricing. For investors, PDUFA timelines can influence the timing of a drug’s approval and potential market success.

aav gene therapy medical

"DTX401 (pariglasgene brecaparvovec) AAV gene therapy for glycogen storage"

AAV gene therapy uses a harmless adeno-associated virus as a delivery vehicle to carry a working copy of a gene into a patient’s cells, like a targeted mail carrier delivering a new instruction manual to fix a malfunctioning part. It matters to investors because these treatments can be one-time or long-lasting cures, driving high potential revenue and valuation but also carrying large development costs, regulatory hurdles and safety and manufacturing risks that affect returns.

antisense oligonucleotide medical

"GTX-102 (apazunersen) antisense oligonucleotide (ASO) for the treatment"

An antisense oligonucleotide is a small piece of synthetic genetic material designed to attach to specific molecules in the body’s cells, effectively blocking or modifying how genes are expressed. This technology is important because it can be used to develop targeted treatments for certain diseases, which may influence the value of biotech companies and the broader healthcare sector. Its development reflects advances in personalized medicine and gene-based therapies.

angelman syndrome medical

"GTX-102 ... for the treatment of Angelman syndrome (AS)"

A rare genetic disorder that causes severe developmental delays, problems with movement and balance, limited speech, and frequent seizures; it results from a missing or malfunctioning gene that acts like a broken instruction in the body’s manual for brain development. Investors watch it because treatments are a focus of drug development—successful therapies or clinical trial results can change the commercial outlook for biotech firms working on targeted genetic or neurological medicines.

osteogenesis imperfecta medical

"Phase 3 Orbit and Cosmic studies of UX143 (setrusumab) in osteogenesis imperfecta"

A genetic disorder that makes bones unusually fragile because the body produces faulty or too little of the protein that gives bone its strength; think of bones like a building made from weak bricks rather than strong ones. Investors track it because treatments, diagnostics, or therapies for the condition can create markets, affect clinical trial outcomes, regulatory approvals, and potential revenue for biotech and pharmaceutical companies developing interventions.

bone mineral density medical

"including data on bone mineral density, vertebral fractures, and patient reported"

A measure of how much mineral — mainly calcium — is packed into a given area of bone, usually obtained from a painless scan; higher values generally mean stronger bones and lower fracture risk. Investors care because changes in this measure are often used as proof that drugs, medical devices, or diagnostics work, so it can drive clinical approval, insurance coverage and sales prospects much like a car’s crash-test rating influences buyer confidence and market demand.

AI-generated analysis. Not financial advice.

Preliminary 2025 total revenue of $672 million to $674 million, exceeding top end of guidance 

Preliminary cash and investments of approximately $735 million as of December 31, 2025

Anticipated 2026 catalysts include two potential approvals and pivotal Phase 3 data from the GTX-102 Phase 3 Aspire study for Angelman syndrome

NOVATO, Calif., Jan. 12, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today reported preliminary unaudited 2025 revenue results, cash and investments at year end 2025, and provided a corporate update.

“In 2025, we delivered another year of significant revenue growth, with preliminary revenue exceeding the top end of guidance," said Emil Kakkis, chief executive officer and president of Ultragenyx. "The year ahead will be transformational for our company and the rare disease communities we serve, with two potential gene therapy approvals of first-ever treatments, and a pivotal data readout from our Phase 3 program in Angelman syndrome, setting the stage for our next phase of growth."

Financial Update
2025 Preliminary Revenue (unaudited)
Total revenue for 2025 is estimated to be $672 million to $674 million, which exceeds the top end of the guidance range, and represents approximately 20% growth versus 2024. Crysvita^® revenue for 2025 is estimated to be $480 million to $482 million, which exceeds the top end of the guidance range, and represents approximately 17% growth versus 2024. Dojolvi^® revenue for 2025 is estimated to be $95 million to $97 million, at the midpoint of the guidance range, and represents approximately 9% growth versus 2024.

2025 Ending Cash Position (unaudited)
Cash and investments were approximately $735 million as of December 31, 2025.

The 2025 revenues and cash position included in this release are preliminary and are therefore subject to adjustment. The preliminary revenue results are based on management’s initial analysis of operations for the year ended December 31, 2025. The Company expects to issue financial guidance for fiscal year 2026, including details on previously announced planned expense reductions, and full financial results for the fourth quarter and fiscal year 2025, in February 2026.

2026 Clinical and Regulatory Catalysts

• DTX401 (pariglasgene brecaparvovec) AAV gene therapy for glycogen storage disease type Ia (GSDIa): Biologics License Application (BLA) rolling submission completed in December 2025, with an anticipated PDUFA date in the third quarter of 2026.
• UX111 (rebisufligene etisparvovec) AAV gene therapy for Sanfilippo syndrome type A (MPS IIIA): Resubmission of the BLA is on track for early in 2026 and will be followed by an up to 6-month review per FDA regulations.
• GTX-102 (apazunersen) antisense oligonucleotide (ASO) for the treatment of Angelman syndrome (AS): Data from the fully enrolled, pivotal, Phase 3 Aspire study in patients with a genetically confirmed diagnosis of UBE3A deletion is expected in the second half of 2026. The Phase 2/3 Aurora study is also underway in other genotypes and ages, with first patient dosed in October 2025.
• UX701 (rivunatpagene miziparvovec) AAV gene therapy for Wilson disease: Enrollment is complete for the fourth cohort in the ongoing, dose-finding stage of the pivotal Cyprus2+ study. Data are expected in the first half of 2026.

Data from the Phase 3 Orbit and Cosmic studies of UX143 (setrusumab) in osteogenesis imperfecta, including data on bone mineral density, vertebral fractures, and patient reported outcomes on pain and physical function, will be presented at the J.P. Morgan Healthcare Conference.

The company presentation is scheduled for Monday, January 12, 2026 at 10:30 AM PT. The live and archived webcast of the presentation will be accessible from the company’s website at https://ir.ultragenyx.com/events-presentations.

About Ultragenyx

Ultragenyx is a biopharmaceutical company committed to bringing novel therapies to patients for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved medicines and treatment candidates aimed at addressing diseases with high unmet medical need and clear biology, for which there are typically no approved therapies treating the underlying disease.

The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

For more information on Ultragenyx, please visit the company's website at: www.ultragenyx.com.

Forward-Looking Statements and Use of Digital Media

Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations and projections regarding its future operating results and financial performance, anticipated cost or expense reductions, the timing, progress and plans for its clinical programs and clinical studies, future regulatory interactions, the components and timing of regulatory submissions and the timing of results from ongoing clinical studies are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause the company’s clinical development programs, commercial success of its products and product candidates, continued collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainty of clinical drug development and unpredictability and lengthy process for obtaining regulatory approvals, risks related to serious or undesirable side effects of our product candidates, the company’s ability to achieve its projected development goals in its expected timeframes, risks related to reliance on third party partners to conduct certain activities on the company’s behalf, our limited experience in generating revenue from product sales, risks related to product liability lawsuits, our dependence on Kyowa Kirin for the commercialization of Crysvita in certain major markets, including the U.S. and Canada, and for our commercial supply of Crysvita in those markets, fluctuations in buying or distribution patterns from distributors and specialty pharmacies, , smaller than anticipated market opportunities for the company’s products and product candidates, manufacturing risks, our ability to successfully manage the expansion of our company, competition from other therapies or products, regulatory scrutiny of the company’s products and product candidates, the company’s limited experience as a company in operating its own manufacturing facility, market acceptance of our products, uncertainty related to insurance coverage and reimbursement, and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations, the company’s future operating results and financial performance, the timing of clinical trial activities and reporting results from same, and the availability or commercial potential of Ultragenyx’s products and drug candidate. Ultragenyx undertakes no obligation to update or revise any forward-looking statements.

For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on November 4, 2025, and its subsequent periodic reports filed with the SEC.

In addition to its SEC filings, press releases and public conference calls, Ultragenyx uses its investor relations website and social media outlets to publish important information about the company, including information that may be deemed material to investors, and to comply with its disclosure obligations under Regulation FD. Financial and other information about Ultragenyx is routinely posted and is accessible on Ultragenyx’s Investor Relations website (https://ir.ultragenyx.com/) and LinkedIn website (https://www.linkedin.com/company/ultragenyx-pharmaceutical-inc-/).

Contacts Ultragenyx Pharmaceutical Inc.
Investors
Joshua Higa
ir@ultragenyx.com

Media
Jess Rowlands
media@ultragenyx.com

FAQ

What were Ultragenyx (RARE) preliminary total revenues for 2025?

Preliminary 2025 total revenue was $672M–$674M, about 20% growth versus 2024.

How much cash did Ultragenyx (RARE) report at year-end 2025?

Cash and investments were approximately $735M as of December 31, 2025.

When is the expected PDUFA date for DTX401 (RARE)?

The company anticipates a PDUFA date in Q3 2026 for DTX401.

When will Ultragenyx (RARE) report pivotal Phase 3 Aspire data for GTX-102 in Angelman syndrome?

Data from the fully enrolled Phase 3 Aspire study are expected in the second half of 2026.

What is the status of the UX111 (RARE) BLA for Sanfilippo A?

UX111 BLA resubmission is on track for early 2026 and may face an up to 6-month FDA review.

Will Ultragenyx (RARE) publish full 2025 financials and 2026 guidance soon?

Yes; the company expects to issue full Q4 and FY2025 results and 2026 guidance in February 2026.