Ultragenyx Pharmaceutical (RARE) Company Report

Ultragenyx (RARE) is advancing its development pipeline, with key near-term value drivers including Osteogenesis Imperfecta (OI) and Angelman Syndrome programs, as well as gene therapies for Sanfilippo and GSD1A.
For the Setrusumab (OI) program, Ultragenyx plans to release Orbit and Cosmic phase three data together, expressing high confidence in both studies' success and noting that even lower-end effect sizes (e.g., low to mid 30% for Orbit, 10-15% for Cosmic) are considered commercially viable.
The company recently secured a non-dilutive royalty financing to bolster its balance sheet, following a delay in potential revenue from its Sanfilippo program.
Ultragenyx's Angelman Syndrome programs (Aspire and Aurora) are progressing, with Aspire fully enrolled and Aurora initiating enrollment, and the company anticipates very low patient dropout rates due to the significant developmental gains observed.

1 day ago

Transcript

Ultragenyx Provides Pipeline Update and Commercial Strategy Insights

RARE

New Projects/Investments

Guidance Update

Product Launch

**Ultragenyx's Chief Medical Officer discussed the company's pipeline, highlighting the upcoming Phase 3 data readouts and filings for Osteogenesis Imperfecta (OI) led by Setrusumab, with high confidence in both Orbit and Cosmic studies. **
**The company recently completed a non-dilutive royalty financing to strengthen its balance sheet following a Complete Response Letter for its Sanfilippo program. **
**For Setrusumab, Ultragenyx expects it to be a lifelong treatment, with pricing modeled after Crysvita, and believes a treatment effect size down to 30-40% would be clinically meaningful and commercially viable. **
**In the Angelman syndrome program, the Aspire Phase 3 study is fully enrolled, and Aurora is initiating enrollment, with very low dropout rates observed and expected due to the potential for patients to gain new skills. **
**Upcoming PDUFA dates in mid-2026 are anticipated for the refiled Sanfilippo gene therapy program (six-month review) and the GSD1A gene therapy program (standard review), with the GSD1A submission expected in December 2025. **

1 day ago

Transcript

Ultragenyx Provides Pipeline and Commercial Strategy Update

RARE

New Projects/Investments

Guidance Update

Ultragenyx is advancing its pipeline, with Osteogenesis Imperfecta (OI) and Angelman Syndrome identified as key near-term value drivers.
For OI, Setrusumab's Orbit and Cosmic phase 3 data readouts are planned to be released together, with Orbit powered to detect a 50% treatment effect. The company expects pricing to model the successful Crysvita launch, and treatment is anticipated to be lifelong.
The Angelman Syndrome program includes the fully enrolled Aspire phase 3 study and the enrolling Aurora study, focusing on patients with full deletions (representing 70-80% of cases) for potential true developmental improvement.
Ultragenyx recently completed a non-dilutive royalty financing to strengthen its balance sheet, following a delay in potential revenue from the Sanfilippo program.
Other gene therapy programs, including Sanfilippo (refiling response to CRL) and GSD1A (submission this month), are expected to have new PDUFA dates in mid-2026.

1 day ago

Transcript

Ultragenyx Provides Updates on Key Clinical Programs and Financial Strategy

RARE

Product Launch

New Projects/Investments

Guidance Update

Ultragenyx expects final data readout for its setrusumab studies (Orbit and Cosmic) for Osteogenesis Imperfecta in December or January. The Phase II part of the Orbit study showed a 67% reduction in fracture rate from baseline.
The BLA for Sanfilippo (UX111) is expected to be refiled at the beginning of next year, and the rolling BLA for GSDIa (UX401) will be finished this month (December 2025). Both programs are anticipated to have PDUFA dates comfortably within the September PRV deadline.
Results from Cohort 4 of the seamless Phase I/II/III Cyprus study for Wilson disease (UX701), which includes increased dose and immunomodulation, are expected next year.
The Phase III Aspire study for Angelman Syndrome (UX102) is enrolled, with data expected in the second half of next year. The company aims for a full label for Angelman, covering various genotypes.
Ultragenyx's recent monetization deal with OMERS for Crysvita provides revenue until January 2028, supporting the goal of achieving profitability within that timeframe.

3 days ago

Transcript

Ultragenyx Provides Updates on Key Pipeline Programs and Financial Strategy

RARE

Product Launch

New Projects/Investments

Guidance Update

Ultragenyx expects final data readout for setrusumab in Osteogenesis Imperfecta (OI) in December or January. The ORBIT study is powered to detect at least a 50% treatment effect, and Phase 1/2 data showed a 67% fracture reduction.
The company anticipates high probability of success for its gene therapies, Sanfilippo (UX111) and GSD1A (DTX401), with GSD1A's rolling BLA completing this month (December 2025) and Sanfilippo's refiling early next year, both expected to meet the PRV window.
Results for UX701 in Wilson Disease (cohort 4) are expected next year, and data for UX102 in Angelman Syndrome is anticipated in the second half of next year.
Ultragenyx recently completed a non-dilutive monetization deal for Crysvita with OMERS, providing financial flexibility and supporting the goal of achieving profitability by January 2028.

3 days ago

Transcript

Ultragenyx Provides Updates on Setrusumab, Gene Therapy Programs, and Angelman Syndrome Studies

RARE

Product Launch

New Projects/Investments

Guidance Update

Setrusumab for Osteogenesis Imperfecta (OI) is a near-term value driver with final data readout expected in December 2025 or January 2026. The company is pursuing a full label for OI, targeting at least 60,000 patients in covered geographies.
Ultragenyx anticipates PDUFA dates for its gene therapy programs, Sanfilippo (UX111) and GSD1A (DTX401), to be comfortably within the September 2026 PRV window. The BLA for GSD1A is expected to be finished in December 2025, and the refiling for Sanfilippo is planned for early 2026.
Data from the enrolled Phase 3 Aspire study for Angelman Syndrome (UX102) is anticipated in the second half of 2026, with a strategy to achieve a full label for all genotypes.
Results for Wilson Disease (UX701) cohort four and first ammonia data for the OTC gene therapy program are both expected in 2026.

3 days ago

Transcript

Mereo BioPharma Reports Q3 2025 Financial Results and Setrusumab Study Update

RARE

Earnings

Guidance Update

New Projects/Investments

Mereo BioPharma reported a net loss of $7.0 million for the third quarter ended September 30, 2025, an improvement compared to a $15.0 million net loss in the third quarter of 2024.
The company held $48.7 million in cash and cash equivalents as of September 30, 2025, which is expected to support operations into 2027.
Data from the Phase 3 Orbit and Cosmic studies for setrusumab in osteogenesis imperfecta are on track to be released around the end of 2025.
Research and development expenses increased to $4.3 million in the third quarter of 2025, up from $3.2 million in the third quarter of 2024, primarily due to setrusumab and alvelestat program activities.

Nov 10, 2025, 9:01 PM

Press Release

Ultragenyx Reports Q3 2025 Results, Secures $400 Million Financing, and Provides Clinical Program Updates

RARE

Earnings

Guidance Update

New Projects/Investments

Ultragenyx reported total revenue of $160 million for Q3 2025, marking a 15% growth over Q3 2024, and reaffirmed its full-year 2025 revenue guidance of $640 million to $670 million.
The company secured $400 million in non-dilutive capital from OMERS through the sale of a portion of its Crysvita royalties, with payments to OMERS starting in January 2028. This financing strengthens the balance sheet and supports the company's path to GAAP profitability in 2027.
Key clinical program updates include the expected readout of UX143 Phase III Orbit and Cosmic study data around December or January , and the completion of enrollment for the GTX-102 ASPIRE study, with data anticipated in H2 2026.
The rolling BLA submission for DTX401 is on track for completion in December, and the BLA resubmission for UX111 is expected in early 2026.

Nov 4, 2025, 10:00 PM

Transcript

Ultragenyx Pharmaceutical Inc. announces Q3 2025 results and secures $400 million in financing

RARE

Earnings

Guidance Update

New Projects/Investments

Ultragenyx reported Q3 2025 total revenue of $160 million, a 15% increase over Q3 2024, and a net loss of $180 million or $1.81 per share. The company held $447 million in cash, cash equivalents, and securities as of September 30, 2025.
The company strengthened its financial position by securing $400 million of non-dilutive capital from OMERS through the sale of a portion of Crysvita royalties, with payments deferred until January 2028.
Ultragenyx reaffirmed its 2025 total revenue guidance of $640 million to $670 million and reiterated its path to GAAP profitability in 2027.
Key pipeline milestones include anticipated top-line data for UX143 (osteogenesis imperfecta) Phase III studies in December or January, completion of the DTX401 BLA submission in December, and GTX-102 (Angelman syndrome) ASPIRE study data readout in the second half of 2026.

Nov 4, 2025, 10:00 PM

Transcript

Ultragenyx Reports Q3 2025 Results, Updates 2025 Guidance, and Secures $400 Million Financing

RARE

Earnings

Guidance Update

Ultragenyx Pharmaceutical Inc. reported Q3 2025 total revenue of $160 million, a 15% increase over Q3 2024, and a net loss of $180 million or $1.81 per share.
The company reaffirmed its full-year 2025 total revenue guidance of $640 million to $670 million, projecting 14%-20% growth over 2024, and expects to achieve full-year GAAP profitability in 2027.
Ultragenyx strengthened its financial position by receiving $400 million in non-dilutive capital from OMERS through the sale of additional Crysvita royalties, with payments deferred until January 2028.
Key clinical milestones include anticipated top-line data for UX143 (osteogenesis imperfecta) from Phase III studies in December or January, and continued progress for GTX-102 (Angelman syndrome) with data expected in the second half of 2026.

Nov 4, 2025, 10:00 PM

Transcript

Earnings Transcripts

Quarterly earnings call transcripts for Ultragenyx Pharmaceutical.

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