Earnings summaries and quarterly performance for Ultragenyx Pharmaceutical.
Executive leadership at Ultragenyx Pharmaceutical.
Emil Kakkis
Detailed
President and Chief Executive Officer
CEO
DH
Dennis Huang
Detailed
Chief Technical Operations Officer and Gene Therapy Operations and Executive Vice President
EC
Eric Crombez
Detailed
Chief Medical Officer and Executive Vice President
EH
Erik Harris
Detailed
Chief Commercial Officer and Executive Vice President
HH
Howard Horn
Detailed
Chief Financial Officer, Corporate Strategy and Executive Vice President
JP
John Pinion
Detailed
Chief Quality Officer and Executive Vice President of Translational Sciences
KP
Karah Parschauer
Detailed
Chief Legal Officer & Corporate Affairs and Executive Vice President
TK
Thomas Kassberg
Detailed
Chief Business Officer and Executive Vice President
Board of directors at Ultragenyx Pharmaceutical.
Research analysts who have asked questions during Ultragenyx Pharmaceutical earnings calls.
TA
Tazeen Ahmad
Bank of America
6 questions for RARE
Also covers: ACAD, ALNY, APLS +20 more
YN
Yigal Nochomovitz
Citigroup Inc.
6 questions for RARE
Also covers: ALDX, APLS, ARCT +22 more
Gena Wang
Barclays
5 questions for RARE
Also covers: ALNY, BCRX, BLUE +12 more
Jack Allen
Robert W. Baird & Co.
5 questions for RARE
Also covers: ALLO, BLUE, CLLS +6 more
YW
Yaron Werber
TD Cowen
5 questions for RARE
Also covers: ALEC, AMGN, ARGX +14 more
JS
Joseph Schwartz
Oppenheimer
4 questions for RARE
Also covers: ASND, AUPH, BMRN +14 more
Kristen Kluska
Cantor Fitzgerald
4 questions for RARE
Also covers: ABEO, ADMA, AQST +24 more
MR
Maury Raycroft
Jefferies
4 questions for RARE
Also covers: ABEO, ALNY, ARWR +11 more
Mehdi Goudarzi
Truist Securities
4 questions for RARE
Also covers: ARGX, EDIT, NTLA +1 more
AR
Anupam Rama
JPMorgan Chase & Co.
3 questions for RARE
Also covers: APLS, BBIO, BOLD +16 more
LI
Luca Issi
RBC Capital Markets
3 questions for RARE
Also covers: ADVM, ALLO, ALNY +12 more
MS
Maxwell Skor
H.C. Wainwright & Co.
3 questions for RARE
Also covers: ASND, AVIR, CRNX +4 more
Salveen Richter
Goldman Sachs
3 questions for RARE
Also covers: ACAD, AGIO, ALLO +20 more
SC
Sami Corwin
William Blair
3 questions for RARE
Also covers: ALLO, BEAM, KRYS +5 more
Gavin Clark-Gartner
Evercore ISI
2 questions for RARE
Also covers: ACRS, ARGX, ASND +11 more
JL
Joon Lee
Truist Securities
2 questions for RARE
Also covers: ADVM, AXSM, CNM +14 more
LC
Laura Chico
Wedbush Securities
2 questions for RARE
Also covers: APLS, ARDX, BCRX +11 more
Liisa Bayko
Evercore ISI
2 questions for RARE
Also covers: ALT, BCRX, INSM +7 more
WS
Will Soghikian
Leerink Partners
2 questions for RARE
Also covers: AUPH, KALV, TVTX
AR
Anyan Rama
JPMorgan Chase & Co.
1 question for RARE
CR
Christopher Raymond
Piper Sandler
1 question for RARE
Also covers: ABBV, AGIO, AMGN +7 more
DG
Dae Gon Ha
Stifel
1 question for RARE
Also covers: ABEO, BEAM, CRBP +10 more
DS
Daniel Smith
H.C. Wainwright & Co.
1 question for RARE
Also covers: AQST, HALO, SRPT
EA
Ed Arce
WestPark Capital
1 question for RARE
Also covers: ABUS, AKBA, ARCT +5 more
Farzin Haque
Jefferies
1 question for RARE
Also covers: ABEO, ARWR, AUPH +3 more
HW
Hannah Wei
Guggenheim Partners
1 question for RARE
HW
Huidong Wang
Barclays
1 question for RARE
Also covers: ALNY, BCRX, BEAM +15 more
JH
Jeff Hung
Morgan Stanley
1 question for RARE
Also covers: ACAD, ALEC, BHVN +10 more
Joyce
JPMorgan Chase & Co.
1 question for RARE
Lydia
Goldman Sachs
1 question for RARE
MK
Malcolm Kuno
JPMorgan Chase & Co.
1 question for RARE
MR
Maurice Raycroft
Jefferies Financial Group
1 question for RARE
Also covers: ABEO, AFMD, ALNY +14 more
Michael Riad
Morgan Stanley
1 question for RARE
Also covers: ACAD, ALEC, ITCI +3 more
Mitchell
H.C. Wainwright
1 question for RARE
RS
Raj Selvaraju
H.C. Wainwright & Co., LLC
1 question for RARE
Shelby
RBC Capital Markets
1 question for RARE
Shelby Hill
RBC Capital Markets
1 question for RARE
S
Steven
ABL Investments
1 question for RARE
Also covers: GV
T
Thomas...
Guggenheim Securities, LLC
1 question for RARE
Also covers: MYGN, NEO
Tommie Reerink
Goldman Sachs
1 question for RARE
Also covers: ALNY, ATRA, BMRN +2 more
WY
Wing Yip
Unspecified
1 question for RARE
Also covers: ABUS, ACXP, ARCT +2 more
Recent press releases and 8-K filings for RARE.
Ultragenyx Provides Business Update at J.P. Morgan Healthcare Conference
RARE
Guidance Update
New Projects/Investments
Layoffs
- Ultragenyx provided an update on its Setrusumab program, noting that two randomized Phase 3 studies missed their primary endpoints but demonstrated substantial improvements in bone mineral density, patient-reported pain, and functional outcomes. The company is conducting further analysis to understand the data before engaging with regulatory agencies.
- The company announced expected 2025 revenue of $62-$674 million, representing 20% growth, and reported $735 million in cash.
- Ultragenyx is planning significant expense and headcount reductions following the Setrusumab trial results and a Sanfilippo CRL, with a goal of achieving profitability in 2027.
- Key pipeline catalysts for 2026 include an expected refiling and potential approval for Sanfilippo syndrome gene therapy around mid-year, a BLA filing for DTX401 for glycogen storage disease, and Phase 3 data for GTX-102 for Angelman syndrome in the second half of the year.
Jan 12, 2026, 6:30 PM
Ultragenyx Provides Business Update at J.P. Morgan Healthcare Conference
RARE
Guidance Update
New Projects/Investments
Revenue Acceleration/Inflection
- Ultragenyx's setrusumab program for rare bone disease missed its primary endpoint in two Phase 3 trials but demonstrated substantial improvements in bone mineral density and a 59% reduction in vertebral fractures in the COSMIC study. The company is conducting further analysis to determine the regulatory path forward.
- The company anticipates several key pipeline milestones in 2026, including a refiling for Sanfilippo syndrome gene therapy early in the year with potential approval around mid-year, and Phase 3 data for GTX-102 in Angelman syndrome in the second half of the year.
- Ultragenyx pre-announced expected revenue of $620 million to $674 million, representing 20% growth, and is implementing significant expense and headcount reductions to achieve profitability in 2027. The company holds $735 million in cash and expects two Priority Review Vouchers.
Jan 12, 2026, 6:30 PM
Ultragenyx Provides Business Update and Pipeline Milestones at J.P. Morgan Healthcare Conference
RARE
Guidance Update
New Projects/Investments
Layoffs
- Ultragenyx reported that its Setrusumab trials missed their primary endpoint, but the drug demonstrated substantial improvements in bone mineral density, vertebral fractures, and patient-reported pain and functional outcomes. The company is conducting further analysis before determining next regulatory steps.
- The company provided pipeline updates, including plans to refile for Sanfilippo syndrome gene therapy early this year with expected approval around mid-year, and a BLA filing completed in December for DTX401 for glycogen storage disease.
- For Angelman syndrome (GTX-102), phase 3 trial enrollment was completed in July last year, with phase 3 data expected in the second half of this year. Early results for Wilson disease gene therapy are encouraging, with higher dose data expected later this year.
- Ultragenyx pre-announced expected revenue of $620-$674 million, representing 20% growth, and holds $735 million in cash.
- Due to the Setrusumab trial results and a previous CRL, the company is planning significant expense and headcount reductions to achieve its goal of profitability in 2027.
Jan 12, 2026, 6:30 PM
Ultragenyx Pharmaceutical Inc. Provides Preliminary 2025 Financial Results and 2026 Catalysts
RARE
Guidance Update
New Projects/Investments
Revenue Acceleration/Inflection
- Ultragenyx Pharmaceutical Inc. reported preliminary unaudited total revenue for 2025 between $672 million and $674 million, exceeding the top end of its guidance and representing approximately 20% growth versus 2024.
- Preliminary unaudited cash and investments were approximately $735 million as of December 31, 2025.
- The company anticipates two potential gene therapy approvals and pivotal Phase 3 data from the GTX-102 Aspire study for Angelman syndrome in 2026.
- Key regulatory catalysts for 2026 include an anticipated PDUFA date in the third quarter of 2026 for DTX401 (pariglasgene brecaparvovec) and the resubmission of the BLA for UX111 (rebisufligene etisparvovec) early in 2026.
Jan 12, 2026, 12:45 PM
Ultragenyx Reports Preliminary 2025 Revenue and Cash, Outlines 2026 Catalysts
RARE
Earnings
Guidance Update
New Projects/Investments
- Ultragenyx reported preliminary unaudited total revenue for 2025 of $672 million to $674 million, exceeding the top end of guidance and representing approximately 20% growth versus 2024.
- The company's preliminary cash and investments stood at approximately $735 million as of December 31, 2025.
- Key 2026 catalysts include anticipated PDUFA dates for DTX401 (Q3 2026) and UX111 (early 2026 resubmission), along with pivotal Phase 3 data from the GTX-102 study for Angelman syndrome in the second half of 2026.
Jan 12, 2026, 12:30 PM
Ultragenyx Completes BLA Submission for DTX401
RARE
Product Launch
New Projects/Investments
- Ultragenyx Pharmaceutical Inc. has completed the rolling submission of its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for DTX401 AAV gene therapy.
- DTX401 is being developed as a treatment for Glycogen Storage Disease Type Ia (GSDIa), a rare genetic disease estimated to affect approximately 6,000 people in commercially accessible geographies.
- The BLA is supported by data from a clinical development program, including a Phase 3 study, which demonstrated significant reductions in daily cornstarch intake, improved euglycemia, and improved fasting tolerance, with an acceptable safety profile.
- DTX401 has received Rare Pediatric Disease, orphan drug, Fast Track, and regenerative medicine advanced therapy (RMAT) designations from the U.S. FDA, as well as orphan drug and PRIority MEdicines (PRIME) designations from the European Medicines Agency.
Dec 30, 2025, 9:05 PM
Ultragenyx Announces Phase 3 Setrusumab Study Results
RARE
Layoffs
New Projects/Investments
- Ultragenyx Pharmaceutical announced that its Phase 3 Orbit and Cosmic studies for setrusumab (UX143) in Osteogenesis Imperfecta (OI) did not achieve their primary endpoint of reducing annualized clinical fracture rates.
- Both studies, however, successfully met the secondary endpoint, demonstrating statistically significant improvements in bone mineral density.
- In response to these results, Ultragenyx will implement significant expense reductions.
- The company's CEO noted continued commercial revenue from four approved products and preparations for two near-term gene therapy launches and a pivotal Phase 3 readout in Angelman syndrome.
Dec 29, 2025, 1:30 PM
Ultragenyx Discusses Pipeline Progress and Royalty Financing
RARE
New Projects/Investments
Debt Issuance
Product Launch
- 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.
Dec 3, 2025, 5:55 PM
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. **
Dec 3, 2025, 5:55 PM
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.
Dec 3, 2025, 5:55 PM
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