Ultragenyx outlines 14%–20% 2025 revenue growth, advances late-stage pipeline with $400M OMERS financing

Earnings Call Insights: Ultragenyx Pharmaceutical Inc. (RARE) Q3 2025

MANAGEMENT VIEW

*
CEO Emil Kakkis indicated that Ultragenyx is at a “defining moment” with four commercial products showing consistent double-digit annual revenue growth and advancing two BLA submissions for ultra-rare diseases. He stated, “We announced earlier today that we took an important step to strengthen our financial position, receiving $400 million of nondilutive capital from OMERS through the cap sale of a portion of our Crysvita royalties.” Kakkis emphasized the timing of deferred payments, which “bolster our balance sheet as we approach pivotal data readouts for our most significant commercial opportunities.”

*
Kakkis highlighted clinical momentum, noting full enrollment of the pivotal Aspire study for GTX-102 in Angelman syndrome and ongoing Phase III trials for UX143 in osteogenesis imperfecta, with data expected around the end of the year.

*
Erik Harris, Chief Commercial Officer, reported that Crysvita in Latin America added approximately 50 new patients and now has 875 patients on commercial product in the region. He also stated, “We now have patients on reimbursed therapy from nearly all of the major countries” for Evkeeza in EMEA, with about 310 patients across 17 countries.

*
CFO Howard Horn stated, “In the third quarter of 2025, we reported $160 million, representing 15% growth over the quarter of 2024 and 18% growth for the first 9 months of 2025 over the first 9 months of 2024.” Horn explained, “We received $400 million through the sale of an additional 25% of our royalty interest on the future sales of Crysvita in the United States and Canada.” He added, “Adding $400 million to our balance sheet will help us deliver on our expected launches, setting us up for our next stage of growth and on our path to profitability in 2027.”

*
Chief Medical Officer Eric Crombez updated on UX111 and DTX401, noting constructive FDA interactions and positive long-term data, with a BLA resubmission for UX111 planned for early 2026 and DTX401’s rolling submission expected to complete next month.

OUTLOOK

*
Kakkis reaffirmed 2025 revenue guidance of $640 million to $670 million, representing 14% to 20% growth over 2024. Crysvita revenue is expected between $460 million and $480 million (12% to 17% growth). Dojolvi revenue is projected at $90 million to $100 million (2% to 14% growth).

*
Management reiterated the path to GAAP profitability in 2027 and highlighted continued focus on cash burn management and investment prioritization.

FINANCIAL RESULTS

*
Ultragenyx reported third quarter revenue of $160 million, with Crysvita contributing $112 million (North America $57 million, Latin America and Turkey $47 million, Europe $8 million). Dojolvi contributed $24 million, Evkeeza $17 million, and Mepsevii $7 million.

*
Operating expenses totaled $331 million, including $216 million in R&D and $87 million in SG&A. Net loss was $180 million, or $1.81 per share. As of September 30, cash, cash equivalents, and marketable debt securities stood at $447 million, with an additional $400 million from the Crysvita royalty transaction.

*
Net cash used in operations for the quarter was $91 million; for the nine months ended September 30, it totaled $366 million. Horn stated, “We do expect 2025 net cash used in operations to [indiscernible] increase compared to 2024, and we also reaffirm our path to full year GAAP profitability in 2027.”

Q&A

*
Huidong Wang, Barclays: Asked for timing and scope of the Orbit and Cosmic data release. Kakkis replied both studies’ data will be released together in December or January due to variability in database finalization.

*
Maurice Raycroft, Jefferies: Requested quantitative updates from open-label extension of Phase II and effect size expectations for fracture reduction. Kakkis said, “We haven't put out another cut of the Phase II data…what we've been observing in the trial is consistent with what we've put out before…anywhere between 40% and 70% reduction in fractures…would be a clinically meaningful change.”

*
Yigal Nochomovitz, Citi: Queried the sequencing of UX111 and DTX401 filings. Kakkis explained, “401 doesn't have some of those things because it is the new filing, and so we felt we can get the things that are uncommon done in time and kept 401 on track to file this next month.”

*
Tazeen Ahmad, BofA: Sought clarity on the OMERS transaction and its impact on financing needs for 2026. Horn responded, “We think that all of those levers will put us on our path to profitability in '27.”

*
Maxwell Skor, Morgan Stanley: Asked about setrusumab adoption by age and severity. Kakkis stated, “I would expect younger and more severe patients to get more immediate access compared to others.”

*
Jack Allen, Baird: Inquired about R&D expense increases and OI market opportunity. Horn attributed higher R&D to “investments in prelaunch inventory and getting ready for these launches.” Kakkis said OI opportunity is likely larger than XLH.

SENTIMENT ANALYSIS

*
Analysts focused on clinical data timing, regulatory path, and the impact of the OMERS transaction, showing a slightly positive but cautious tone, especially regarding pivotal trial outcomes and financing runway.

*
Management maintained a confident and proactive stance, emphasizing strong product growth, clinical pipeline momentum, and financial discipline. Kakkis used phrases like “we remain confident,” and Horn reiterated the company’s “path to profitability in 2027.”

*
Compared to the previous quarter, the tone remains confident but more focused on financial strategy given the new royalty transaction and cash runway discussions.

QUARTER-OVER-QUARTER COMPARISON

*
Guidance for 2025 revenue and segment targets remains unchanged from Q2. The company maintained its focus on advancing late-stage clinical programs and reiterated its profitability timeline.

*
The major new development is the $400 million OMERS royalty transaction, strengthening liquidity ahead of pivotal data readouts. Operating expenses and net loss increased compared to Q2, largely due to prelaunch investments.

*
Analyst questions shifted more toward financial runway and the implications of the royalty sale, while management’s confidence in upcoming clinical milestones and product growth was consistent.

RISKS AND CONCERNS

*
Management highlighted ongoing cash burn and noted that net cash used in operations is expected to increase in 2025 compared to 2024.

*
Risks related to pivotal trial readouts for UX143 and GTX-102 were discussed, as the timing and outcome of these studies are critical to near-term growth.

*
The OMERS royalty sale, while strengthening liquidity, reduces future royalty streams and may impact long-term recurring revenue from Crysvita.

*
Analyst concerns centered on the outcome of pivotal trials, timing of regulatory submissions, and the company’s ability to manage expenses and achieve profitability as projected.

FINAL TAKEAWAY

Ultragenyx emphasized its strengthened financial position following the $400 million OMERS royalty transaction, reaffirmed 2025 revenue guidance, and maintained a clear focus on advancing pivotal clinical programs with key data readouts expected by early 2026. Management projects the company will reach full year GAAP profitability in 2027 and continues to prioritize disciplined investment while supporting product launches and regulatory milestones in rare disease markets.

Read the full Earnings Call Transcript [https://seekingalpha.com/symbol/rare/earnings/transcripts]

MORE ON ULTRAGENYX PHARMACEUTICAL

* Ultragenyx Pharmaceutical Inc. (RARE) Q3 2025 Earnings Call Transcript [https://seekingalpha.com/article/4837880-ultragenyx-pharmaceutical-inc-rare-q3-2025-earnings-call-transcript]
* Ultragenyx: Rough Road Ahead Despite Growing Revenues And Catalysts [https://seekingalpha.com/article/4830620-ultragenyx-stock-rough-road-ahead-despite-growing-revenues-catalysts]
* Ultragenyx Pharmaceutical Inc. (RARE) Presents at Bank of America Global Healthcare Conference 2025 Transcript [https://seekingalpha.com/article/4825109-ultragenyx-pharmaceutical-inc-rare-presents-at-bank-of-america-global-healthcare-conference]
* Ultragenyx sells some royalties on Crysvita to OMERS Life Sciences [https://seekingalpha.com/news/4515046-ultragenyx-sells-some-royalties-on-crysvita-to-omers-life-sciences]
* Ultragenyx Pharmaceutical GAAP EPS of -$1.81 misses by $0.57, revenue of $160M misses by $6.79M [https://seekingalpha.com/news/4514959-ultragenyx-pharmaceutical-gaap-eps-of-1_81-misses-by-0_57-revenue-of-160m-misses-by-6_79m]