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Ultragenyx Pharmaceutical (RARE)

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Earnings summaries and quarterly performance for Ultragenyx Pharmaceutical.

Research analysts who have asked questions during Ultragenyx Pharmaceutical earnings calls.

YN

Yigal Nochomovitz

Citigroup Inc.

7 questions for RARE

Also covers: ALDX, APLS, ARCT +22 more
MR

Maury Raycroft

Jefferies

6 questions for RARE

Also covers: ABEO, ALNY, ARWR +14 more
TA

Tazeen Ahmad

Bank of America

6 questions for RARE

Also covers: ACAD, ALNY, APLS +22 more
Gena Wang

Gena Wang

Barclays

5 questions for RARE

Also covers: ALNY, BCRX, BLUE +13 more
Jack Allen

Jack Allen

Robert W. Baird & Co.

5 questions for RARE

Also covers: ACAD, ALLO, BLUE +8 more
LI

Luca Issi

RBC Capital Markets

5 questions for RARE

Also covers: ADVM, ALLO, ALNY +13 more
MS

Maxwell Skor

H.C. Wainwright & Co.

5 questions for RARE

Also covers: ASND, AVIR, CRNX +4 more
SC

Sami Corwin

William Blair

5 questions for RARE

Also covers: ALLO, BEAM, KRYS +5 more
YW

Yaron Werber

TD Cowen

5 questions for RARE

Also covers: ALEC, AMGN, ARGX +15 more
JS

Joseph Schwartz

Oppenheimer

4 questions for RARE

Also covers: ASND, AUPH, BMRN +14 more
Kristen Kluska

Kristen Kluska

Cantor Fitzgerald

4 questions for RARE

Also covers: ABEO, ADMA, AQST +24 more
Mehdi Goudarzi

Mehdi Goudarzi

Truist Securities

4 questions for RARE

Also covers: ARGX, EDIT, NTLA +1 more
WS

Will Soghikian

Leerink Partners

4 questions for RARE

Also covers: AUPH, KALV, TVTX
AR

Anupam Rama

JPMorgan Chase & Co.

3 questions for RARE

Also covers: APLS, BBIO, BOLD +18 more
JL

Joon Lee

Truist Securities

3 questions for RARE

Also covers: ADVM, ALKS, AXSM +15 more
Salveen Richter

Salveen Richter

Goldman Sachs

3 questions for RARE

Also covers: ACAD, AGIO, ALLO +21 more
Allison Bratzel

Allison Bratzel

Piper Sandler Companies

2 questions for RARE

Also covers: ABCL, AKBA, ALXO +7 more
Gavin Clark-Gartner

Gavin Clark-Gartner

Evercore ISI

2 questions for RARE

Also covers: ACRS, ARGX, ASND +12 more
LC

Laura Chico

Wedbush Securities

2 questions for RARE

Also covers: APLS, ARDX, BCRX +11 more
Liisa Bayko

Liisa Bayko

Evercore ISI

2 questions for RARE

Also covers: ALT, BCRX, INSM +7 more
LE

Lydia Edelman

Goldman Sachs

2 questions for RARE

PN

Priyanka Nagpal

JPMorgan

2 questions for RARE

RM

Rick Miller

Cantor Fitzgerald

2 questions for RARE

Also covers: ABEO, ADMA, SELB
Steven Scala

Steven Scala

TD Cowen

2 questions for RARE

Also covers: BMY, NVS
TA

Tara Ahmad

Bank of America

2 questions for RARE

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

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

Joyce

JPMorgan Chase & Co.

1 question for RARE

JL

June Lee

Truist Securities

1 question for RARE

Lydia

Lydia

Goldman Sachs

1 question for RARE

Also covers: AGIO
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

Michael Riad

Morgan Stanley

1 question for RARE

Also covers: ACAD, ALEC, ITCI +3 more
Mitchell

Mitchell

H.C. Wainwright

1 question for RARE

RS

Raj Selvaraju

H.C. Wainwright & Co., LLC

1 question for RARE

Shelby

Shelby

RBC Capital Markets

1 question for RARE

Also covers: AGIO, FULC, NTLA
Shelby Hill

Shelby Hill

RBC Capital Markets

1 question for RARE

Also covers: MRNA
S

Steven

ABL Investments

1 question for RARE

Also covers: GV, XEL
T

Thomas...

Guggenheim Securities, LLC

1 question for RARE

Also covers: ARDX, FBK, MYGN +2 more
Tommie Reerink

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
YN

Ygal Nochomovitz

Citi

1 question for RARE

Also covers: NBIX

Recent press releases and 8-K filings for RARE.

Ultragenyx Faces Securities Class Action Lawsuit
RARE
Legal Proceedings
  • Faruqi & Faruqi, LLP is investigating Ultragenyx Pharmaceutical for alleged federal securities law violations related to its setrusumab Phase III Orbit and Cosmic studies.
  • The lawsuit alleges the company created a false impression of reliable information and minimized risks regarding the studies' outcomes, which ultimately failed to achieve statistical significance.
  • Investors who acquired securities between August 3, 2023, and December 26, 2025, have until April 6, 2026, to seek lead plaintiff status in the class action.
  • Following announcements of the study failures, Ultragenyx stock prices fell more than 25% on July 9, 2025, and more than 42% on December 29, 2025.
14 hours ago
Ultragenyx Faces Securities Class Action Lawsuit
RARE
Legal Proceedings
  • Faruqi & Faruqi, LLP is investigating Ultragenyx Pharmaceutical (RARE) for a federal securities class action, with a lead plaintiff deadline of April 6, 2026.
  • The complaint alleges the company made false and/or misleading statements and failed to disclose risks regarding its setrusumab Phase III Orbit study for Osteogenesis Imperfecta (OI).
  • Ultragenyx's optimism in the Phase III Orbit study's results and interim analysis benchmark was allegedly misplaced because it failed to convey the risk associated with basing such figures on Phase II results that lacked a placebo control group.
  • Ultragenyx stock prices fell more than 25% on July 9, 2025, and more than 42% on December 29, 2025, following announcements that its Phase III Orbit and Cosmic studies failed to achieve statistical significance.
  • The class action covers investors who purchased or acquired securities in Ultragenyx between August 3, 2023, and December 26, 2025.
2 days ago
Ultragenyx Pharmaceutical Faces Class Action Lawsuit Over Phase III Clinical Trial Design
RARE
Legal Proceedings
  • A class action lawsuit has been filed against Ultragenyx Pharmaceutical Inc. on behalf of shareholders who purchased securities between August 3, 2023, and December 26, 2025, alleging misleading statements regarding the Phase III Orbit and Cosmic studies for setrusumab.
  • The lawsuit contends that management's expressions of confidence in the studies' ability to reduce annualized fracture rates were unfounded due to inherent study design limitations, including reliance on uncontrolled Phase II observations and patient population mismatches.
  • A key allegation is the disconnect between setrusumab achieving statistically significant improvements in bone mineral density (BMD) and its failure to achieve the primary endpoint of reduced fracture rates. The deadline for investors to file applications to serve as lead plaintiff is April 6, 2026.
4 days ago
Ultragenyx Faces Class Action Lawsuit Over Setrusumab Study Results
RARE
Legal Proceedings
Profit Warning
  • A class action lawsuit has been initiated against Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE).
  • The lawsuit alleges that the company made misleading statements regarding its Phase III Orbit and Cosmic Studies for setrusumab (UX 143), which failed to meet primary endpoints for reducing annualized clinical fracture rates.
  • Following the announcement of the study failures on December 29, 2025, Ultragenyx's stock price declined by approximately 42.32%.
  • The class period for the lawsuit is August 3, 2023, to December 26, 2025, with a lead plaintiff deadline of April 6, 2026.
Feb 18, 2026, 12:06 AM
Ultragenyx Pharmaceutical Faces Securities Class Action Lawsuit
RARE
Legal Proceedings
Profit Warning
  • Faruqi & Faruqi, LLP is investigating Ultragenyx Pharmaceutical Inc (RARE) for alleged federal securities law violations related to its setrusumab Phase III Orbit and Cosmic studies.
  • The complaint alleges false or misleading statements regarding the studies' prospects, specifically minimizing risks and basing benchmarks on Phase II results without a placebo control.
  • Ultragenyx's stock price experienced significant declines: over 25% on July 9, 2025, and over 42% on December 29, 2025, following announcements that the Phase III studies failed to achieve statistical significance.
  • Investors who incurred losses exceeding $100,000 between August 3, 2023, and December 26, 2025, are encouraged to contact the firm, with a lead plaintiff deadline of April 6, 2026.
Feb 15, 2026, 12:55 PM
Ultragenyx Pharmaceutical Reports Strong 2025 Revenue, Issues 2026 Guidance, and Announces Restructuring
RARE
Earnings
Guidance Update
Layoffs
  • Ultragenyx Pharmaceutical reported total revenue of $673 million for the full year 2025, representing 20% growth over 2024 and exceeding the upper end of their guidance range, with Crysvita contributing $481 million.
  • The company provided 2026 total revenue guidance of $730 million to $760 million, reflecting 8%-13% growth over 2025, and expects combined R&D and SG&A expenses to be flat to down low single digits compared to 2025.
  • As part of a strategic restructuring plan aimed at achieving profitability in 2027, Ultragenyx announced a 10% workforce reduction, impacting approximately 130 full-time employees, and anticipates a decrease of at least 15% in combined R&D and SG&A expenses in 2027 versus 2025.
  • Key pipeline updates include an expected PDUFA action date in Q3 2026 for DTX-401, ongoing efforts to resubmit the BLA for UX111 after receiving an Information Request Letter (IRL), and anticipated ASPIRE phase III data for GTX-102 in H2 2026.
Feb 12, 2026, 10:00 PM
Ultragenyx Pharmaceutical Reports Strong 2025 Revenue, Issues 2026 Guidance, and Announces Strategic Restructuring
RARE
Earnings
Guidance Update
Layoffs
  • Ultragenyx Pharmaceutical reported total revenue of $673 million for full year 2025, representing 20% growth over 2024, and provided 2026 revenue guidance between $730 million and $760 million.
  • The company announced a strategic restructuring plan to reduce operating expenses, including a 10% workforce reduction impacting approximately 130 full-time employees, with the goal of achieving profitability in 2027.
  • Key pipeline updates include receiving an incomplete response letter (IRL) from the FDA for the UX111 BLA for MPS IIIA, an expected PDUFA action date in Q3 2026 for DTX-401 (glycogen storage disease type Ia), and anticipated Phase 3 data for GTX-102 (Angelman syndrome) in the second half of 2026.
Feb 12, 2026, 10:00 PM
Ultragenyx Pharmaceutical Reports Strong 2025 Revenue, Issues 2026 Guidance, and Announces Restructuring for 2027 Profitability
RARE
Earnings
Guidance Update
Layoffs
  • Ultragenyx Pharmaceutical reported total revenue of $673 million for 2025, representing 20% growth over 2024 and exceeding the upper end of its guidance range.
  • For 2026, the company expects total revenue between $730 million and $760 million, reflecting 8%-13% growth over 2025, excluding potential revenue from new product launches.
  • A strategic restructuring plan was implemented, including a 10% workforce reduction impacting approximately 130 full-time employees, with the aim of achieving profitability in 2027.
  • As part of the restructuring, combined R&D and SG&A expenses are expected to be flat to down low single digits in 2026 versus 2025, and decrease by at least 15% in 2027 versus 2025.
  • The BLA resubmission for UX111 (MPS IIIA) received an incomplete response letter (IRL) from the FDA, requiring additional supportive documentation. However, the company expects a PDUFA action date in Q3 2026 for DTX-401 (glycogen storage disease type Ia) and anticipates sharing ASPIRE phase 3 data for GTX-102 (Angelman syndrome) in the second half of 2026.
Feb 12, 2026, 10:00 PM
Ultragenyx Reports Q4 and Full Year 2025 Results, Initiates Restructuring, and Provides 2026 Guidance
RARE
Earnings
Guidance Update
Layoffs
  • Ultragenyx Pharmaceutical Inc. reported total revenues of $207 million for the fourth quarter of 2025 and $673 million for the full year ended December 31, 2025. The company recorded a net loss of $129 million (or $1.29 per share) for Q4 2025 and $575 million (or $5.83 per share) for the full year 2025.
  • For 2026, Ultragenyx expects total revenue from current products to be between $730 million and $760 million, with Crysvita revenue projected at $500 million to $520 million and Dojolvi revenue at $100 million to $110 million.
  • The company has initiated a strategic restructuring plan, including a 10% workforce reduction impacting approximately 130 employees, to reduce operating expenses and aims to achieve profitability in 2027. This restructuring is estimated to incur approximately $50 million in charges during the first half of 2026.
  • Key 2026 catalysts include an anticipated PDUFA action date for DTX401 in Q3 2026 and expected pivotal Phase 3 data for GTX-102 in H2 2026. However, the Biologics License Application (BLA) for UX111 received an Incomplete Response Letter.
Feb 12, 2026, 9:11 PM
Ultragenyx Reports Q4 and Full Year 2025 Financial Results and 2026 Guidance
RARE
Earnings
Guidance Update
Layoffs
  • Ultragenyx reported total revenue of $673 million for the full year 2025, marking a 20% growth compared to the prior year, with Q4 2025 total revenue reaching $207 million, a 25% increase over Q4 2024.
  • The company provided 2026 total revenue guidance for current products in the range of $730 million to $760 million, an anticipated 8% to 13% increase over 2025.
  • A strategic restructuring plan was initiated, involving a 10% workforce reduction (approximately 130 employees), aimed at reducing expenses and reaffirming the path to profitability in 2027.
  • For the full year 2025, Ultragenyx reported a net loss of $575 million, or $5.83 per share, and held $737 million in cash, cash equivalents, and marketable securities as of December 31, 2025.
Feb 12, 2026, 9:01 PM

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In-depth analysis and coverage of Ultragenyx Pharmaceutical.