Ultragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in the United States. Its biologic products include Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia, as well as tumor-induced osteomalacia; Mepsevii, an enzyme replacement therapy for the treatment of children and adults with Mucopolysaccharidosis VII; and Dojolvi for treating long-chain fatty acid oxidation disorders. The company is also developing DTX401, an adeno-associated virus 8 (AAV8) gene therapy clinical candidate for the treatment of patients with glycogen storage disease type Ia; DTX301, an AAV8 gene therapy product candidate for the treatment of patients with ornithine transcarbamylase; UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome; UX701, for the treatment of Wilson disease; and UX053 for the treatment of glycogen storage disease type III. Ultragenyx Pharmaceutical Inc. has collaboration and license agreement with Kyowa Kirin Co., Ltd.; Saint Louis University; Baylor Research Institute; REGENXBIO Inc.; Bayer; GeneTx; Mereo; University of Pennsylvania; Arcturus Therapeutics Holdings Inc., Solid Biosciences Inc.; and Daiichi Sankyo Co., Ltd. Ultragenyx Pharmaceutical Inc. was incorporated in 2010 and is headquartered in Novato, California.
IPO Year: 2014
Exchange: NASDAQ
Website: ultragenyx.com
| Date | Price Target | Rating | Analyst |
|---|---|---|---|
| 6/6/2024 | $56.00 → $67.00 | Neutral → Buy | Goldman |
| 4/22/2024 | $77.00 | Outperform | RBC Capital Mkts |
| 12/8/2023 | $72.00 | Overweight | Wells Fargo |
| 6/14/2023 | $96.00 | Outperform | Credit Suisse |
| 6/6/2023 | $60.00 → $80.00 | In-line → Outperform | Evercore ISI |
| 4/26/2023 | $114.00 | Overweight | Cantor Fitzgerald |
| 1/18/2023 | $90.00 | Buy | Canaccord Genuity |
| 12/30/2022 | $82.00 | Buy | H.C. Wainwright |
| 11/3/2022 | $50.00 | Neutral → Outperform | Robert W. Baird |
| 10/13/2022 | $50.00 | Neutral → Buy | Guggenheim |
NOVATO, Calif., March 19, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare diseases, today reported the grant of 21,806 restricted stock units of the company's common stock to 16 newly hired non-executive officers of the company. The awards were approved by the compensation committee of the company's board of directors and granted under the Ultragenyx Employment Inducement Plan, with a grant date of March 16, 2025, as an inducement material to the new employees entering into employment with Ultragenyx in accordance with Nasdaq Listing Rule 5635(c)(4).
NOVATO, Calif., Feb. 24, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultrarare genetic diseases, today announced participation in three upcoming investor conferences. 45th Annual Cowen Healthcare Conference (Boston, MA) Monday, March 3, 2025, Eric Crombez, M.D., Chief Medical Officer, will participate in a fireside chat and host 1x1 meetings. Barclays 27th Annual Global Healthcare Conference (Miami, FL) Tuesday, March 11, 2025, Eric Crombez will participate in a fireside chat and host 1x1 meetings. Leerink Partners Global Biopharma Conference
NOVATO, Calif., Feb. 19, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare diseases, today reported the grant of 23,760 restricted stock units of the company's common stock to 10 newly hired non-executive officers of the company. The awards were approved by the compensation committee of the company's board of directors and granted under the Ultragenyx Employment Inducement Plan, with a grant date of February 16, 2025, as an inducement material to the new employees entering into employment with Ultragenyx in accordance with Nasdaq Listing Rule 5635(c)(4
NOVATO, Calif., Feb. 18, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) today announced the U.S. Food and Drug Administration (FDA or the Agency) has accepted for review the Biologics License Application (BLA) seeking accelerated approval for UX111 (ABO-102) AAV gene therapy as a treatment for patients with Sanfilippo syndrome type A (MPS IIIA). The FDA granted the BLA Priority Review with a Prescription Drug User Fee Act (PDUFA) action date of August 18, 2025. The FDA also informed the company that they are not currently planning to hold an advisory committee meeting to discuss this application. "Acceptance of the UX111 BLA brings us closer to being able to provi
2024 Total Revenue of $560 million, exceeding guidanceCrysvita® revenue of $410 million and Dojolvi® revenue of $88 million 2025 Financial Guidance: Total Revenue between $640 million to $670 million, Crysvita revenue of $460 million to $480 million, and Dojolvi revenue of $90 million to $100 million NOVATO, Calif., Feb. 13, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultrarare genetic diseases, today reported its financial results for the quarter and full year ended December 31, 2024 and shared financial guidance for 2025. "We have created a ne
NOVATO, Calif., Feb. 06, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultrarare genetic diseases, today announced that it will host a conference call at 5:00 p.m. ET on Thursday, February 13, 2025, to discuss its financial results and corporate update for the quarter and the year ending December 31, 2024. The live and replayed webcast of the call will be available through the company's website at https://ir.ultragenyx.com/events-presentations. The replay of the call will be available for three months. About Ultragenyx Pharmaceutical Inc.Ultragenyx
Modified intent-to-treat group demonstrated +22.7 point (p<0.0001) treatment effect in the mean Bayley-III cognitive raw score compared to natural history data (ages 24-60 months) Older children with more advanced disease demonstrated retention of clinically meaningful functional abilities, including communication, ambulation and self-feeding The latest data from the pivotal Transpher A and long-term follow-up studies will be presented at WORLDSymposium™ 2025 NOVATO, Calif., Feb. 05, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) today announced new data demonstrating treatment with UX111 (ABO-102) AAV gene therapy led to a statistically significant improvement in
NOVATO, Calif., Jan. 17, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare diseases, today reported the grant of 7,750 restricted stock units of the company's common stock to eight newly hired non-executive officers of the company. The awards were approved by the compensation committee of the company's board of directors and granted under the Ultragenyx Employment Inducement Plan, with a grant date of January 16, 2025, as an inducement material to the new employees entering into employment with Ultragenyx in accordance with Nasdaq Listing Rule 5635(c)(
Preliminary 2024 total revenue of $555 million to $560 million, exceeding top end of guidance, including Crysvita® revenue of $405 million to $410 million, and Dojolvi® revenue of $87 million to $89 million 2025 expected total revenue guidance of $640 million to $670 millionUX143 (setrusumab) Phase 3 Orbit study for osteogenesis imperfecta progressing to second interim analysis in mid-2025 GTX-102 Phase 3 Aspire study for Angelman syndrome expected to complete enrollment in second half of 2025 NOVATO, Calif., Jan. 12, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel p
NOVATO, Calif., Jan. 06, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultrarare genetic diseases, today announced that Emil D. Kakkis, M.D., Ph.D., the company's chief executive officer and president, will present at the 43rd Annual J.P. Morgan Healthcare Conference on Monday, January 13, 2025, at 10:30 AM PT. The live and archived webcast of the presentation will be accessible from the company's website at https://ir.ultragenyx.com/events-presentations. The replay of the webcast will be available for 30 days. About Ultragenyx Pharmaceutical Inc.
NOVATO, Calif., July 12, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare diseases, today announced that it has appointed Howard Horn as executive vice president and chief financial officer (CFO) effective October 16, 2023. Mr. Horn will be responsible for leading the finance, accounting, corporate strategy and investor relations functions. "Howard is joining Ultragenyx and its leadership team during a pivotal period as our robust late-stage pipeline is maturing and we are preparing for the next phase of company growth," said Emil D. Kakkis, M.D.,
NOVATO, Calif., March 14, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) announced that Eric Crombez, M.D., has been promoted to chief medical officer and executive vice president effective May 1, 2023, succeeding Camille Bedrosian, M.D., who will remain with the company in a full-time strategic advisory role. Dr. Crombez currently serves as Ultragenyx's chief medical officer for gene therapy and inborn errors of metabolism. "Eric has extensive expertise in the development and execution of clinical development programs for rare genetic disorders and has been a driving force for our entire gene therapy pipeline," said Emil D. Kakkis, M.D., Ph.D., chief executive of
NOVATO, Calif., April 22, 2022 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced the appointment of Amrit Ray, M.D., M.B.A. to the company's Board of Directors. Dr. Ray will serve on the Board's Research and Development Committee. "Dr. Ray brings to our Board of Directors decades of global experience as a physician researcher, biopharmaceutical executive, and champion for patient access to innovative treatments," said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. "His expertise
Accomplished biotechnology executive brings over 40 years of commercial and leadership experience across multinational pharmaceutical and biotechnology companies to ShouTi ShouTi Inc., a clinical-stage biopharmaceutical company replacing biologics with world-class small molecule medicines using advanced computational and structure-based technology, today announced that Daniel Welch has been named Chairman of the company's board of directors. "It is a true privilege to welcome Dan to ShouTi as Chairman of our board," said Raymond Stevens, Ph.D., Chief Executive Officer, ShouTi. "As an industry veteran with an outstanding track record in guiding companies across all stages of development an
Dr. Suliman brings over 25 years of drug development, deal-making and company building expertise as ReCode continues advancing its pipeline of novel genetic medicines using its powerful selective organ targeting (SORT) LNP delivery platform ReCode Therapeutics (the "Company"), a biopharmaceutical company pioneering disease-modifying genetic medicines using its proprietary LNP delivery platform, today announced the appointment of Shehnaaz Suliman, M.D., MBA, M.Phil., as chief executive officer and as a member of the board of directors effective today. Former CEO, David Lockhart, Ph.D., will transition to the role of chief scientific officer and will remain president and a member of the boar
NOVATO, Calif., June 30, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today announced the appointment of Corsee Sanders, Ph.D., to the Board of Directors, effective June 29, 2021. Dr. Sanders, an experienced veteran of the biopharma industry, will serve as an independent director. "Dr. Sanders' established experience in global clinical development and especially with new technologies will be instrumental as we further advance our ongoing clinical trials and continue to expand our rare disease pipeline of novel therapeut
2024 Total Revenue of $560 million, exceeding guidanceCrysvita® revenue of $410 million and Dojolvi® revenue of $88 million 2025 Financial Guidance: Total Revenue between $640 million to $670 million, Crysvita revenue of $460 million to $480 million, and Dojolvi revenue of $90 million to $100 million NOVATO, Calif., Feb. 13, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultrarare genetic diseases, today reported its financial results for the quarter and full year ended December 31, 2024 and shared financial guidance for 2025. "We have created a ne
NOVATO, Calif., Feb. 06, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultrarare genetic diseases, today announced that it will host a conference call at 5:00 p.m. ET on Thursday, February 13, 2025, to discuss its financial results and corporate update for the quarter and the year ending December 31, 2024. The live and replayed webcast of the call will be available through the company's website at https://ir.ultragenyx.com/events-presentations. The replay of the call will be available for three months. About Ultragenyx Pharmaceutical Inc.Ultragenyx
Third quarter 2024 total revenue grew 42% versus prior year to $139 million, including Crysvita® revenue of $98 million and Dojolvi® revenue of $21 million Reaffirmed 2024 expected total revenue guidance of $530 million to $550 million Breakthrough Designation granted for setrusumab (UX143) in osteogenesis imperfecta DTX401 Phase 3 follow-up data demonstrated higher and faster 62% mean reduction in cornstarch in crossover patients with glycogen storage disease type Ia (GSDIa) NOVATO, Calif., Nov. 05, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious r
NOVATO, Calif., Oct. 29, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultrarare genetic diseases, today announced that it will host a conference call at 5:00 p.m. ET on Tuesday, November 5, 2024, to discuss its financial results and corporate update for the quarter ending September 30, 2024. The live and replayed webcast of the call will be available through the company's website at https://ir.ultragenyx.com/events-presentations. The replay of the call will be available for three months. About Ultragenyx Pharmaceutical Inc.Ultragenyx is a biopharma
NOVATO, Calif., July 25, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultrarare genetic diseases, today announced that it will host a conference call at 5:00 p.m. ET on Thursday, August 1, 2024, to discuss its financial results and corporate update for the quarter ending June 30, 2024. The live and replayed webcast of the call will be available through the company's website at https://ir.ultragenyx.com/events-presentations. The replay of the call will be available for three months. About Ultragenyx Pharmaceutical Inc.Ultragenyx is a biopharmace
Treatment with DTX401 resulted in a statistically significant reduction in daily cornstarch intake at Week 48 (p<0.0001) with maintenance of glucose control Company will host investor call today at 5:00 p.m. ET NOVATO, Calif., May 30, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) today announced positive topline results from the Phase 3 GlucoGene study (NCT05139316) evaluating DTX401, an investigational gene therapy for the treatment of patients aged eight years and older with glycogen storage disease type Ia (GSDIa). The study achieved its primary endpoint, demonstrating that treatment with DTX401 resulted in a statistically significant and clinically meaningful
First quarter total revenue of $109 million, Crysvita® revenue of $83 million and Dojolvi® revenue of $16 million Reaffirmed 2024 expected total revenue guidance between $500 million to $530 million, Crysvita revenue of $375 million to $400 million, and Dojolvi revenue of $75 million to $80 million Presented positive interim Phase 1/2 data from GTX-102 Angelman syndrome study demonstrating that Expansion Cohorts showed rapid, clinically meaningful improvement across multiple domains consistent with or exceeding Dose-escalation Cohort data at Day 170 NOVATO, Calif., May 02, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the d
NOVATO, Calif., April 25, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultrarare genetic diseases, today announced that it will host a conference call at 5:00 p.m. ET on Thursday, May 2, 2024, to discuss its financial results and corporate update for the quarter ending March 31, 2024. The live and replayed webcast of the call will be available through the company's website at https://ir.ultragenyx.com/events-presentations. To participate in the live call, please register by clicking on the following link (registration link) and you will be provided
Expansion Cohorts showed rapid, clinically meaningful improvement across multiple domains; improvements consistent or exceeding Dose-escalation Cohorts data at Day 170 Additional long-term data in Dose-escalation Cohorts showed increasing and sustained clinical benefit through Day 758 Company will host investor call at 8:00 a.m. ET NOVATO, Calif., April 15, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) today announced new data from the Phase 1/2 study of GTX-102 for the treatment of Angelman syndrome. Patients in Expansion Cohorts A & B treated with a set dose and regimen of GTX-102 showed rapid and clinically meaningful improvement across multiple domains
Conference call now scheduled for Monday, April 15 at 8:00 a.m. ET Updated registration link below NOVATO, Calif., April 12, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) today announced that the conference call to discuss new clinical efficacy and safety data from the Phase 1/2 study of GTX-102 for the treatment of Angelman syndrome that will be presented at the American Academy of Neurology Meeting (AAN), will now be taking place on Monday, April 15, 2024 at 8 a.m. Eastern Time (ET). The slides accompanying the call will be shared on company's website at https://ir.ultragenyx.com/events-presentations before the call. Conference Call and Webcast Information T
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Goldman upgraded Ultragenyx Pharma from Neutral to Buy and set a new price target of $67.00 from $56.00 previously
RBC Capital Mkts initiated coverage of Ultragenyx Pharma with a rating of Outperform and set a new price target of $77.00
Wells Fargo initiated coverage of Ultragenyx Pharma with a rating of Overweight and set a new price target of $72.00
Credit Suisse resumed coverage of Ultragenyx Pharma with a rating of Outperform and set a new price target of $96.00
Evercore ISI upgraded Ultragenyx Pharma from In-line to Outperform and set a new price target of $80.00 from $60.00 previously
Cantor Fitzgerald initiated coverage of Ultragenyx Pharma with a rating of Overweight and set a new price target of $114.00
Canaccord Genuity resumed coverage of Ultragenyx Pharma with a rating of Buy and set a new price target of $90.00
H.C. Wainwright resumed coverage of Ultragenyx Pharma with a rating of Buy and set a new price target of $82.00
Robert W. Baird upgraded Ultragenyx Pharma from Neutral to Outperform and set a new price target of $50.00
Guggenheim upgraded Ultragenyx Pharma from Neutral to Buy and set a new price target of $50.00
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