Ultragenyx Pharmaceutical Inc.: Q3 FY2026 earnings call

What management said about Q3 FY2026, from the documents filed with the SEC on 3 November 2026: press release. Guidance figures: 6 new, 3 kept. The quarter’s numbers.

Read the filing

Key takeaways

• Revenue rose to $173 million: Total revenue for the third quarter 2026 was $173 million, against $160 million a year earlier. Nine-month revenue was $523 million versus $466 million. Source
• Net loss lower than prior year: Net loss was $118 million, or $1.16 per share, compared with $180 million, or $1.81 per share, in the third quarter 2025. Source
• CRYSVITA revenue $109 million: CRYSVITA revenue was $109 million, against $112 million a year earlier. The company said this is consistent with expected ordering patterns. Source
• Operating expenses fell to $283 million: Total operating expenses were $283 million, against $331 million a year earlier, including $34 million of non-cash stock-based compensation. Source
• R&D spending lower, SG&A higher: R&D expense was $154 million versus $216 million. SG&A was $97 million versus $87 million. Cost of sales was $32 million versus $28 million. Source
• Headcount cut about 20%: The company is reducing headcount by about 20%, leaving about 1,000 employees, and reducing expenses across the company, including its real estate footprint. Source
• 2027 expense ceilings set: The company expects 2027 R&D expenses below $350 million and 2027 SG&A expenses below $375 million. Source
• GENGLYCOS approved for GSDIa: FDA granted accelerated approval for GENGLYCOS in August 2026 for patients eight years and older with GSDIa. The company received a priority review voucher on approval. Source
• FAYUVI approved for Sanfilippo type A: FDA granted standard full approval for FAYUVI in pediatric Sanfilippo syndrome type A in September 2026. The company received a priority review voucher on approval. Source
• Two PRVs sold for $413 million: Agreements to sell the GENGLYCOS and FAYUVI vouchers total $413 million, at $210 million and $203 million. Net proceeds are expected to be about $350 million. Source
• Cash balance declined to $401 million: Cash, cash equivalents and marketable securities were $401 million at September 30, 2026, against $436 million at June 30, 2026 and $737 million at December 31, 2025. Source
• R&D focused on DTX301 and UX143: R&D investment will focus on primary responder-endpoint data from the DTX301 Phase 3 study, a decision on any UX143 path forward, and measured spending on early-stage programs. Source

“We received two BLA approvals from the FDA within one month for our first gene therapy treatments and the launches are progressing well with growing demand for these products,”

Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx

Guidance

What changed from last quarter

• Guidance kept: 2026 total revenue guidance of $730 million to $760 million was reaffirmed, excluding new launches. Source
• Guidance kept: 2026 CRYSVITA revenue guidance of $500 million to $520 million was reaffirmed. Source
• Guidance kept: 2026 DOJOLVI revenue guidance of $100 million to $110 million was reaffirmed. Source
• New guidance: Specific 2027 caps on R&D and SG&A expenses were given. The prior release had guided to a combined 2027 decrease of at least 15% versus 2025.
• New guidance: A Fourth quarter 2026 charge of approximately $25 million for severance and restructuring was stated. Source
• New: GENGLYCOS received FDA accelerated approval in August 2026. Its INN, pariglasgene brecaparvovec, matches DTX401 in the prior release. Source
• New: FAYUVI received FDA standard full approval in September 2026. Its INN, rebisufligene etisparvovec, matches UX111, which had a September 19, 2026 PDUFA date. Source
• New: Two priority review vouchers were sold under agreements for a combined $413 million. Source
• New: Headcount is being reduced by about 20%, with expense reductions and a smaller real estate footprint. Source
• New: The company is moving into a stage focused on commercial execution and a smaller, more targeted R&D footprint. Source
• Ended: All ongoing GTX-102 studies for Angelman syndrome are being wound down. Source
• Ended: The UX701 program for Wilson disease is terminated after it did not meet study goals for advancing to Phase 3. Source

Results and growth drivers

• Revenue rose to $173 million: Total revenue for the third quarter 2026 was $173 million, against $160 million a year earlier. Nine-month revenue was $523 million versus $466 million. Source
• Net loss lower than prior year: Net loss was $118 million, or $1.16 per share, compared with $180 million, or $1.81 per share, in the third quarter 2025. Source

Products and customers

• CRYSVITA revenue $109 million: CRYSVITA revenue was $109 million, against $112 million a year earlier. The company said this is consistent with expected ordering patterns. Source
• CRYSVITA mix by region: Latin America and Türkiye product sales were $41 million (prior year $47 million). U.S. and Canada royalties were $61 million (prior year $57 million). Europe royalties were $7 million (prior year $8 million). Source
• EVKEEZA revenue up on launches: EVKEEZA revenue was $24 million, against $17 million, driven by increased demand from new country launches and early access. Source
• DOJOLVI and MEPSEVII revenue higher: DOJOLVI revenue was $30 million (prior year $24 million). MEPSEVII revenue was $10 million (prior year $7 million). Source

“With substantial reductions in headcount and expenses, combined with two launches and continued growth in our base business, we expect meaningful profitability in 2027 and going forward.”

Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx

Costs and margins

• Operating expenses fell to $283 million: Total operating expenses were $283 million, against $331 million a year earlier, including $34 million of non-cash stock-based compensation. Source
• R&D spending lower, SG&A higher: R&D expense was $154 million versus $216 million. SG&A was $97 million versus $87 million. Cost of sales was $32 million versus $28 million. Source
• Royalty financing interest higher: Non-cash interest on liabilities for sales of future royalties was $20 million, against $14 million. A $9 million income tax benefit was recorded in the quarter. Source
• No restructuring expense this quarter: Restructuring expense totalled $30 million for the nine months, with none recorded in the third quarter. Source

Restructuring and 2027 spending plan

• Headcount cut about 20%: The company is reducing headcount by about 20%, leaving about 1,000 employees, and reducing expenses across the company, including its real estate footprint. Source
• 2027 expense ceilings set: The company expects 2027 R&D expenses below $350 million and 2027 SG&A expenses below $375 million. Source
• GTX-102 studies being wound down: The company is completing the wind-down of all ongoing studies for GTX-102 in Angelman syndrome. Source
• Workforce reduction complete by year-end: The reduction in force is expected to be substantially completed by the end of 2026, with severance charges in the fourth quarter. Source

Pipeline and regulatory milestones

• GENGLYCOS approved for GSDIa: FDA granted accelerated approval for GENGLYCOS in August 2026 for patients eight years and older with GSDIa. The company received a priority review voucher on approval. Source
• FAYUVI approved for Sanfilippo type A: FDA granted standard full approval for FAYUVI in pediatric Sanfilippo syndrome type A in September 2026. The company received a priority review voucher on approval. Source
• UX701 did not meet advancement criteria: UX701 did not meet the criteria to advance to pivotal Phase 3, though it showed clinical activity in some patients and a favorable safety profile across cohorts. Source
• DTX301 Phase 3 showed improvement: The Phase 3 Enh3ance study showed improvement in ammonia control and a reduced need for scavenger medicines. Patients continue through 64 weeks of follow-up. Source
• UX143 path forward under review: Phase 3 setrusumab data were presented at ASBMR 2026. The company will continue engaging regulators on the data needed for a filing and whether a path forward exists. Source
• UX016 study externally funded: The UX016 Phase 1/2 study is fully funded through clinical proof-of-concept by an external venture philanthropy agreement. Dosing will be initiated soon. Source

Priority review vouchers and cash

• Two PRVs sold for $413 million: Agreements to sell the GENGLYCOS and FAYUVI vouchers total $413 million, at $210 million and $203 million. Net proceeds are expected to be about $350 million. Source
• GENGLYCOS voucher sale closed: The GENGLYCOS voucher sale closed the week before the release. The FAYUVI voucher sale is expected to close later in the month. Source
• Cash balance declined to $401 million: Cash, cash equivalents and marketable securities were $401 million at September 30, 2026, against $436 million at June 30, 2026 and $737 million at December 31, 2025. Source
• Operating cash use narrowed: Net cash used in operations was $43 million for the quarter, against $97 million in the second quarter of 2026. Source
• Working capital and stockholders' deficit: Working capital was $155 million and the stockholders' deficit was $376 million at September 30, 2026, against $567 million and $80 million at December 31, 2025. Source

R&D priorities and launch focus

• R&D focused on DTX301 and UX143: R&D investment will focus on primary responder-endpoint data from the DTX301 Phase 3 study, a decision on any UX143 path forward, and measured spending on early-stage programs. Source
• Launch execution is the priority: The company is focused on driving successful launches for GENGLYCOS and FAYUVI and on continuing to grow current product revenues. Source

Coming up, as management said on 3 November 2026

• DTX301 Phase 3 data expected: Data for the second primary endpoint of the DTX301 Phase 3 study are expected in the first half of 2027. Source
• UX701 data to be presented: Data from the completed UX701 study are expected to be presented at an upcoming medical or scientific congress. Source
• Fourth quarter charges and reduction in force: Fourth quarter 2026 results are expected to include about $25 million of severance and restructuring charges, with the reduction in force substantially completed by year-end. Source
• Full-year GAAP profitability targeted for 2027: The company continues to anticipate full-year GAAP profitability in 2027 and going forward. Source

Figures management reported

Risks management discussed

• Operational: UX701 did not produce the biochemical response and standard-of-care elimination needed to advance, so the program is being terminated and sites and patient groups are being supported through the transition. Source
• Regulatory: The path forward for UX143 is not settled. The company will continue engaging regulators on the data needed for a filing and whether one is possible. Source

Earlier plans management reported on

• Not stated: The company reaffirmed its 2026 total revenue guidance in this release. Source
• Not stated: The company continues to anticipate full-year GAAP profitability in 2027 and going forward, as stated in the prior release. Source
• Not stated: The prior release gave the same first-half 2027 timing for DTX301 data, which this release repeats. Source
• Not stated: The prior release expected fourth-quarter 2026 UX701 data. This release terminates the program. Source
• Not stated: The prior release expected Aspire data in September or October. This release reports wind-down of all ongoing GTX-102 studies. Source

Other quarters

• Q2 FY2026 earnings call
• Q1 FY2026 earnings call
• Q4 FY2025 earnings call
• Q3 FY2025 results

Summarised with AI from the documents the company filed with the SEC. Every point is checked against the filing before it is shown: management’s own words word for word, figures against the line or table row they come from. A point that fails the check is left out. Source opens the filing at that line.