Highest-materiality recent filing
Ultragenyx Q2 revenue $214M, net loss narrows to $0.90/share; reaffirms 2026 guidance
Q2 total revenue $214M (up 28% YoY); Crysvita $156M, Dojolvi $27M, Evkeeza $21M, Mepsevii $10M.
Ultragenyx shareholders OK 14.5M-share equity plan; re-elect directors
Third Amended and Restated 2023 Incentive Plan approved; up to 14.5M new shares available plus shares from prior plans.
Total revenue $136M (Q1 2025: $139M); Crysvita $93M, Dojolvi $18M, Evkeeza $18M, Mepsevii $7M.
FDA accepts resubmitted BLA for UX111 gene therapy; PDUFA date Sept 19, 2026
FDA accepted for review resubmitted BLA for UX111 (rebisufligene etisparvovec) AAV9 gene therapy.
DTX301-treated patients (n=18) showed 18% reduction in 24-hour plasma ammonia AUC vs placebo (n=19) at Week 36 (p=0.018).
FDA accepted Biologics License Application for DTX401 (pariglasgene brecaparvovec) to treat Glycogen Storage Disease Type Ia.
Ultragenyx Q4 rev $207M (+25%), net loss $129M; restructures 10% headcount; UX111 gets IRL
Full year 2025 total revenue $673M (+20% YoY); Crysvita $481M, Dojolvi $96M, Evkeeza $59M.
Children under 2 treated with UX111 showed +23.2 point improvement in Bayley-III cognitive raw score vs natural history (p<0.0001).
Ultragenyx resubmits UX111 BLA for Sanfilippo type A to FDA; PDUFA expected Q3 2026
Resubmitted BLA for UX111 gene therapy for Sanfilippo type A on Jan 30, 2026.
Preliminary FY2025 total revenue $672-674M, ~20% YoY, exceeding top end of guidance.
Ultragenyx completes rolling BLA submission for DTX401 gene therapy to FDA for GSDIa
BLA submission for DTX401 (pariglasgene brecaparvovec) completed for glycogen storage disease type Ia; based on 52 treated patients with up to 6 years follow-up.
Ultragenyx's setrusumab fails Phase 3 primary endpoints in OI; expense cuts planned
Orbit and Cosmic studies did not meet primary endpoint of reducing annualized clinical fracture rate versus comparators.
Ultragenyx Q3 revenue $160M (+15% YoY); sells 25% Crysvita royalty for $400M
Total revenue $160M (+15% YoY); Crysvita $112M, Dojolvi $24M, Evkeeza $17M.
First patient dosed in global Aurora study (NCT07157254) evaluating GTX-102 (apazunersen) for Angelman syndrome across all genotypes and ages 1 to <65.
Ultragenyx reports positive longer-term Phase 3 data for DTX401 gene therapy in GSDIa
DTX401 met primary endpoint at Week 48: mean cornstarch reduction 41% vs 10% placebo (p<0.0001).
Ultragenyx starts rolling BLA submission for DTX401 gene therapy in GSDIa
Initiated rolling BLA submission to FDA for DTX401 AAV gene therapy for Glycogen Storage Disease Type Ia.
Ultragenyx Q2 revenue $166M (+13% YoY); net loss narrows; UX111 BLA receives CRL
Q2 total revenue $166M, Crysvita $120M, Dojolvi $23M; net loss $115M ($1.17/sh) vs $132M loss prior year.
Ultragenyx fully enrolls Phase 3 Aspire study (129 patients) for GTX-102 in Angelman Syndrome
Phase 3 Aspire study fully enrolled with ~129 patients ages 4-17 with maternal UBE3A deletion.
FDA CRL received July 11, 2025, for UX111 (ABO-102) AAV gene therapy for Sanfilippo syndrome type A.
Ultragenyx's UX143 Phase 3 Orbit passes DMC safety review; final analysis year-end 2025
DMC confirms acceptable safety profile for UX143 in pediatric/young adult OI patients; study continues to final analysis.
Ultragenyx receives FDA Breakthrough Therapy Designation for GTX-102 in Angelman syndrome
FDA granted Breakthrough Therapy Designation for GTX-102 (apazunersen) for Angelman syndrome.
Ultragenyx Q1 revenue $139M, reaffirms FY2025 guidance; UX111 PDUFA Aug 18, 2025
Q1 total revenue $139M (+28% YoY); net loss $151M ($1.57/share) vs $171M ($2.03) in Q1 2024.
Ultragenyx Q4 revenue $165M, FY $560M; guides 2025 revenue $640-670M
Q4 2024 total revenue $165M (+29% YoY); FY 2024 total revenue $560M (+29% YoY).
Ultragenyx preliminary 2024 revenue $555-560M above guidance; 2025 guide $640-670M
Preliminary 2024 total revenue $555-560M, exceeding top end of guidance, ~29% YoY growth.
Ultragenyx submits BLA to FDA for UX111 gene therapy in Sanfilippo syndrome type A
Submitted BLA to FDA seeking accelerated approval for UX111 (AAV gene therapy) in Sanfilippo syndrome type A (MPS IIIA).
First patient dosed in Ultragenyx Phase 3 Aspire study of GTX-102 for Angelman syndrome
Global Phase 3 Aspire study will enroll ~120 children ages 4-17 with full maternal UBE3A deletion.
Week 48 Bayley-4 Cognition GSV mean change +6.7 vs minimally important difference +5 (n=40).
Total revenue $139M, up 42% YoY; Crysvita $98M (+31%), Dojolvi $21M (+29%), Evkeeza $11M.
Ultragenyx gets FDA Breakthrough Therapy Designation for setrusumab in osteogenesis imperfecta
Received FDA Breakthrough Therapy Designation for setrusumab (UX143) for OI Type I, III, IV in patients ≥2 years.
15 patients enrolled across 3 dose cohorts; 6 completely tapered off chelators/zinc therapy; 1 additional began tapering.
Ultragenyx Q2 revenue $147M (+36% YoY), raises 2024 guidance; key FDA agreements advance pipeline
Q2 total revenue $147M (Crysvita $114M, Dojolvi $19M); net loss narrows to $132M from $160M.
Ultragenyx aligns with FDA on Phase 3 design for GTX-102 in Angelman syndrome
FDA alignment on EoP2 meeting for GTX-102; Phase 3 trial to enroll ~120 UBE3A deletion patients.
Ultragenyx raises ~$330.7M in public offering of common stock and pre-funded warrants
Offering of 7,435,898 shares at $39/share plus pre-funded warrants for 1,538,501 shares at $38.999.
Ultragenyx and Mereo report setrusumab cuts fracture rate 67% and boosts BMD 22% in OI Phase 2
Median annualized fracture rate reduced 67% from 0.72 to 0.00 (p=0.0014) over mean 16 months in 24 patients.
FDA agrees CSF heparan sulfate as surrogate endpoint for UX111 accelerated approval BLA in MPS IIIA
FDA agreed cerebral spinal fluid heparan sulfate is a reasonable surrogate endpoint supporting accelerated approval for UX111 gene therapy in Sanfilippo syndrome type A (MPS IIIA).
Ultragenyx Phase 3 DTX401 gene therapy meets primary endpoint in GSDIa
DTX401 reduced daily cornstarch intake by 41.3% vs 10.3% for placebo at Week 48 (p<0.0001).
Ultragenyx Q1 2024 revenue $109M (+8% YoY); net loss $171M; reaffirms guidance; pipeline advances
Q1 total revenue $109M (+8% YoY); Crysvita $83M (+9%), Dojolvi $16M (+14%).
Expansion Cohorts A&B (24 patients) at Day 170 showed clinically meaningful improvements across cognition, behavior, sleep, motor, and communication domains vs natural history.
Ultragenyx FY2023 revenue $434M (+20% YoY); net loss narrows; 2024 guide $500-530M
Total revenue 2023 $434M (+20% YoY); Crysvita $328M (+17%), Dojolvi $71M (+27%).
Ultragenyx reports preliminary 2023 revenue $430-435M; guides 2024 revenue $500-530M
Preliminary 2023 total revenue $430-435M; Crysvita $325-330M, Dojolvi $70-71M.
Ultragenyx Q3 revenue $98.1M (+8% YoY); reaffirms FY2023 guidance $425-450M
Total revenue $98.1M, up 8% YoY; Crysvita $74.9M, Dojolvi $16.6M.
Ultragenyx prices $300M public offering of common stock and pre-funded warrants
8,333,334 shares at $30.00/share plus 1,666,722 pre-funded warrants at $29.9990 each.
Ultragenyx reports prelim Q3 rev $96-100M; UX143 cuts fractures 67% in Phase 2
Prelim Q3 total rev $96-100M; Crysvita $74-76M (impacted by inventory shift to KKC); cash $525M.
Ultragenyx Q2 2023 revenue $108.3M (+21% YoY); reaffirms 2023 guidance
Q2 total revenue $108.3M vs $89.3M YoY; Crysvita $83.0M (+20%), Dojolvi $16.5M, Mepsevii $8.4M.
Mr. Horn and the Company have modified the effective date of Mr. Horn’s appointment as the Company’s CFO and EVP, Corporate Strategy from October 16, 2023 to October 9, 2023 (the “Effective Date”).
On July 12, 2023, Ultragenyx Pharmaceutical Inc. (the “Company”) announced that Howard Horn has been appointed as the Company’s Chief Financial Officer and Executive Vice President, Corporate Strategy, effective as of October 16, 2023
On March 20, 2023, Lars Ekman, M.D., Ph.D. notified Ultragenyx Pharmaceutical Inc. (the “ Company ”) of his decision to retire from the Board of Directors of the Company (the “ Board ”), effective immediately after the Company’s 2023 Annual Meeting of Stockholders, currently scheduled for June 7, 2023.
Eric Crombez, M.D., the Company’s current Chief Medical Officer for Gene Therapy and Inborn Errors of Metabolism, will assume the role of Executive Vice President, Chief Medical Officer, effective as of the Effective Date.
On March 14, 2023, Ultragenyx Pharmaceutical Inc. (the “ Company ”) announced that Camille L. Bedrosian, M.D., the Company’s Executive Vice President and Chief Medical Officer, will be transitioning to a new role as Strategic Development Advisor to the Company, effective May 1, 2023
On November 10, 2022, Ultragenyx Pharmaceutical Inc. (the “ Company ”) announced that Mardi Dier had informed the Company of her decision to voluntarily resign from her position as the Company’s Executive Vice President and Chief Financial Officer, effective as of November 15, 2022, to pursue another opportunity.
William Aliski notified Ultragenyx Pharmaceutical Inc. (the “ Company ”) that he will not stand for re-election as a member of the Board of Directors
the Board appointed Amrit Ray, M.D. as a Class III director of the Board effective April 19, 2022
Max materiality 0.85 · Median 0.75 · Most common event other_material