SEC Form SC 13G/A filed by Ultragenyx Pharmaceutical Inc. (Amendment)
$RARE
Biotechnology: Pharmaceutical Preparations
Health Care
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| Date | Price Target | Rating | Analyst |
|---|---|---|---|
| 5/28/2025 | Outperform | William Blair | |
| 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 |
Complete Response Letter (CRL) cited specific chemistry, manufacturing and controls (CMC) related observations that are resolvable FDA clinical reviews acknowledged that the clinical data are robust and biomarker data are supportive NOVATO, Calif., July 11, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), today announced that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) for its Biologics License Application (BLA) for UX111 (ABO-102) AAV gene therapy as a treatment for patients with Sanfilippo syndrome type A (MPS IIIA). "Our goal is to get UX111 to patients as quickly as possible knowing how critical this first therapy i
NOVATO, Calif., July 09, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) and Mereo BioPharma Group plc (NASDAQ:MREO), today announced that the randomized, placebo-controlled Phase 3 portion of the Orbit study evaluating UX143 (setrusumab) in pediatric and young adult patients with osteogenesis imperfecta (OI) is progressing toward a final analysis consistent with the original plan, around the end of the year. The Data Monitoring Committee (DMC) met and informed the company that UX143 demonstrates an acceptable safety profile and the company should continue the study to the final analysis. "Based on the feedback we hear from investigators and families who participate
Phase 3 Aspire study enrollment on track to complete in 2025 Aurora study to evaluate GTX-102 in other Angelman syndrome genotypes and ages expected to initiate later this year NOVATO, Calif., June 27, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), today announced that it has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for GTX-102 (apazunersen) as a treatment for Angelman syndrome. "FDA Breakthrough Therapy Designation underscores both the urgent need for an effective treatment for patients and families affected by Angelman syndrome and the clinically meaningful results demonstrated to date with GTX-102," said Eric C
8-K - Ultragenyx Pharmaceutical Inc. (0001515673) (Filer)
8-K - Ultragenyx Pharmaceutical Inc. (0001515673) (Filer)
8-K - Ultragenyx Pharmaceutical Inc. (0001515673) (Filer)
4 - Ultragenyx Pharmaceutical Inc. (0001515673) (Issuer)
4 - Ultragenyx Pharmaceutical Inc. (0001515673) (Issuer)
4 - Ultragenyx Pharmaceutical Inc. (0001515673) (Issuer)
William Blair initiated coverage of Ultragenyx Pharma with a rating of Outperform
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
First quarter total revenue of $139 million, Crysvita® revenue of $103 million and Dojolvi® revenue of $17 million Reaffirm 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 UX111, an investigational treatment for Sanfilippo syndrome, biologics license application (BLA) on track for PDUFA action date of August 18, 2025 NOVATO, Calif., May 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 ultra-rare genetic diseases, today
NOVATO, Calif., April 30, 2025 (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 that it will host a conference call at 5:00 p.m. ET on Tuesday, May 6, 2025, to discuss its financial results and corporate update for the quarter ending March 31, 2025. 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 biopharmaceutica
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., 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
SC 13G/A - Ultragenyx Pharmaceutical Inc. (0001515673) (Subject)
SC 13G/A - Ultragenyx Pharmaceutical Inc. (0001515673) (Subject)
SC 13G/A - Ultragenyx Pharmaceutical Inc. (0001515673) (Subject)