NOVATO, Calif., May 20, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc . (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare diseases, today reported the grant of 44,805 restricted stock units of the company’s common stock to 20 newly hired non-executive officers of the company. The awards were approved by the compensation committee... Read More

NOVATO, Calif., May 14, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) released its 2024 Impact Report today, which covers the company’s approach to corporate responsibility. The report highlights efforts to transform the lives of individuals and families affected by rare diseases, empower its employees, and make a meaningful impact on the communities it operates within and serves. “Since founding... Read More

NOVATO, Calif., May 07, 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 announced that Howard Horn, the company's chief financial officer and executive vice president, will participate in a fireside at Bank of America’s 2025 Healthcare Conference on... Read More

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... Read More

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.... Read More

NOVATO, Calif., April 22, 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 36,831 restricted stock units of the company’s common stock to 21 newly hired non-executive officers of the company. The awards were approved by the compensation committee... Read More

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... Read More

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. 45 th Annual Cowen Healthcare Conference (Boston, MA) Monday, March 3, 2025, Eric Crombez, M.D., Chief Medical... Read More

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... Read More

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... Read More

2024 Total Revenue of $560 million, exceeding guidance Crysvita® 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... Read More

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... Read More

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... Read More

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... Read More

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 million UX143 (setrusumab) Phase 3 Orbit study for osteogenesis imperfecta progressing to second interim analysis in mid-2025 GTX-102 Phase 3 Aspire study for... Read More

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 43 rd Annual J.P. Morgan Healthcare Conference on Monday,... Read More

NOVATO, Calif., Dec. 20, 2024 (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 15,175 restricted stock units of the company’s common stock to 15 newly hired non-executive officers of the company. The awards were approved by the compensation committee of... Read More

NOVATO, Calif., Dec. 19, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), today announced that the first patient has been dosed in the pivotal Phase 3 Aspire study ( NCT06617429 ) evaluating the efficacy and safety of GTX-102, its investigational antisense oligonucleotide (ASO) for Angelman syndrome. "Initiation of patient dosing in our Phase 3 Aspire study represents an important step forward in the... Read More

NOVATO, Calif., Dec. 19, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA or the Agency) seeking accelerated approval for UX111 (ABO-102) AAV gene therapy as a treatment for patients with Sanfilippo syndrome type A (MPS IIIA). “The path to get a treatment to the point of a BLA filing has... Read More

NOVATO, Calif., Nov. 26, 2024 (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. Citi’s 2024 Global Healthcare Conference (Miami, FL) Tuesday, December 3, 2024, Emil Kakkis, M.D., Ph.D., CEO and... Read More

NOVATO, Calif., Nov. 20, 2024 (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 20,820 restricted stock units of the company’s common stock to 13 newly hired non-executive officers of the company. The awards were approved by the compensation committee of... Read More

Phase 1/2 data show improvements across all domains and confirm that Phase 3 Aspire study is amply powered to establish efficacy of GTX-102 Phase 3 program on track to begin enrollment by end-of-year NOVATO, Calif., Nov. 09, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced Phase 1/2 data in support of the Phase 3 Aspire study for GTX-102, its investigational antisense oligonucleotide for... Read More

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... Read More

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... Read More

NOVATO, Calif., Oct. 18, 2024 (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 22,405 restricted stock units of the company’s common stock to six newly hired non-executive officers of the company. The awards were approved by the compensation committee... Read More

NOVATO, Calif., Oct. 07, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that it has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for setrusumab (UX143) as a treatment to reduce the risk of fracture associated with osteogenesis... Read More

NOVATO, Calif., Oct. 03, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today reported that the Phase 1/2/3 Cyprus 2+ study of its UX701 gene therapy has demonstrated meaningful clinical activity as well as improvements in copper metabolism in Stage 1. Multiple responders have completely tapered off of standard-of-care treatment with responses seen in all three dose cohorts. The company plans to... Read More

NOVATO, Calif., Sept. 26, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that it will present seven abstracts related to its ongoing late-stage program evaluating setrusumab (UX143) and osteogenesis imperfecta (OI), including a late-breaker oral presentation of the 14-month data from the Phase 2/3 Orbit study, at the American Society for Bone and Mineral Research (ASBMR) 2024 Annual... Read More

NOVATO, Calif., Sept. 18, 2024 (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 62,555 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... Read More

Morgan Stanley 22 nd Annual Global Healthcare Conference on September 4 Cantor Global Healthcare Conference on September 17 Bank of America Global Healthcare Conference on September 18 NOVATO, Calif., Aug. 29, 2024 (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,... Read More

NOVATO, Calif., Aug. 21, 2024 (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 37,975 restricted stock units of the company’s common stock to 12 newly hired non-executive officers of the company. The awards were approved by the compensation committee of... Read More

Second quarter total revenue of $147 million, Crysvita ® revenue of $114 million and Dojolvi ® revenue of $19 million Increased 2024 expected total revenue guidance to $530 million to $550 million NOVATO, Calif., Aug. 01, 2024 (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... Read More

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,... Read More

NOVATO, Calif., July 24, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that the company will share the latest clinical data, regulatory progress and program next steps for GTX-102, its investigational antisense oligonucleotide for Angelman syndrome, on Wednesday, July 24, at the 2024 Angelman Syndrome Foundation (ASF) Family Conference and Research Symposium in Sandusky, Ohio. “Since... Read More

NOVATO, Calif., July 19, 2024 (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 19,875 restricted stock units of the company’s common stock to 14 newly hired non-executive officers of the company. The awards were approved by the compensation committee of... Read More

Alignment with FDA on Phase 3 study primary endpoint of Bayley-4 cognition and key secondary endpoint of Multi-Domain Responder Index (MDRI) Phase 3 study on track to initiate by the end of this year NOVATO, Calif., July 17, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced the successful completion of an end-of-Phase 2 (EoP2) meeting with the U.S. Food and Drug Administration (FDA),... Read More

NOVATO, Calif., June 20, 2024 (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 45,525 restricted stock units of the company’s common stock to 26 newly hired non-executive officers of the company. The awards were approved by the compensation committee of... Read More

NOVATO, Calif., June 13, 2024 (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 the pricing of its underwritten public offering of 7,435,898 shares of its common stock at a price to the public of $39.00 per share. In addition, in lieu of issuing... Read More

NOVATO, Calif., June 12, 2024 (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 it has commenced an underwritten public offering of up to $350,000,000 of shares of its common stock and, in lieu of issuing common stock to certain investors,... Read More

Agreement reached with FDA that cerebral spinal fluid (CSF) heparan sulfate (HS) can be used as a reasonable surrogate endpoint for accelerated approval Data from existing clinical studies appear sufficient for a biologics license application (BLA) submission NOVATO, Calif., June 12, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that the company held a successful meeting with the U.S.... Read More

14-month data show treatment with setrusumab resulted in a large, sustained 67% reduction in annualized fracture rate and persistent median annualized fracture rate of 0.00 (p=0.0014) Treatment resulted in continued, substantial improvements in bone mineral density (BMD) with a mean increase from baseline of 22% (p<0.0001) and a mean improvement in Z-score of +1.25 (p<0.0001) NOVATO, Calif. and LONDON, June 11, 2024 (GLOBE... Read More

NOVATO, Calif., June 04, 2024 (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 Kakkis, M.D., Ph.D., the company's chief executive officer, will participate in a fireside at the Goldman Sachs 45th Annual Global Healthcare Conference on... Read More

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... Read More

NOVATO, Calif., May 17, 2024 (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 17,180 restricted stock units of the company’s common stock to ten newly hired non-executive officers of the company. The awards were approved by the compensation committee of... Read More

NOVATO, Calif., May 07, 2024 (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 Eric Crombez, M.D., the company's chief medical officer and executive vice president, will participate in a fireside at Bank of America’s 2024 Healthcare Conference... Read More

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... Read More

NOVATO, Calif., April 30, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today released its 2023 Corporate Responsibility Report, which showcases the company’s efforts to transform the lives of individuals and families impacted by rare and ultrarare diseases, empower its employees, and make a meaningful impact on the communities it partners with. “In rare disease, corporate responsibility means... Read More

NOVATO, Calif., April 30, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that all patients have been enrolled across the Phase 3 Orbit and Cosmic studies evaluating setrusumab (UX143) in pediatric and young adult patients with osteogenesis imperfecta (OI). The pivotal Phase 3 portion of the Orbit study has randomized 158 patients ages 5 to 25 years, and the Cosmic study has completed... Read More

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.... Read More

NOVATO, Calif., April 19, 2024 (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 44,965 restricted stock units of the company’s common stock to 14 newly hired non-executive officers of the company. The awards were approved by the compensation committee... Read More

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:... Read More

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... Read More

NOVATO, Calif., April 12, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that preclinical and clinical data, including new clinical efficacy and safety data from the expansion cohorts in the Phase 1/2 study of GTX-102 for the treatment of Angelman syndrome, will be presented at the 76th Annual American Academy of Neurology Meeting (AAN) being held April 13-18 in Denver and virtually.... Read More

NOVATO, Calif., March 22, 2024 (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 13,020 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... Read More

44 th Annual Cowen Healthcare Conference on March 4 Leerink Partners Global Biopharma Conference on March 12 Barclays 26 th Annual Global Healthcare Conference on March 13 NOVATO, Calif., Feb. 27, 2024 (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... Read More

NOVATO, Calif., Feb. 23, 2024 (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 10,995 restricted stock units of the company’s common stock to nine newly hired non-executive officers of the company. The awards were approved by the compensation committee... Read More

2023 Total Revenue of $434 million, Crysvita® revenue of $328 million and Dojolvi® revenue of $71 million 2024 Financial Guidance: Total Revenue between $500 million and $530 million, Crysvita revenue of $375 million to $400 million, and Dojolvi revenue of $75 million to $80 million Year-end 2023 cash balance of $777 million and 2024 guidance for Net Cash Used in Operations expected to be less than $400 million NOVATO,... Read More

NOVATO, Calif., Feb. 08, 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, February 15, 2024, to discuss its financial results and corporate update for the fourth quarter and the... Read More

NOVATO, Calif., Feb. 06, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced data demonstrating treatment with UX111 (ABO-102) AAV gene therapy resulted in rapid and sustained decreased levels of heparan sulfate (HS) in cerebrospinal fluid (CSF) in patients with Sanfilippo syndrome type A (MPS IIIA), and that sustained reduction in CSF HS exposure over time was correlated with improved... Read More

GTX-102 is the first Angelman syndrome therapeutic candidate to receive PRIME designation Phase 1/2 study fully enrolled; expansion data expected in first half of 2024 NOVATO, Calif., Feb. 05, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that the European Medicines Agency (EMA) has granted Priority Medicine (PRIME) designation to GTX-102 for the treatment of Angelman syndrome (AS).... Read More

Safety and initial efficacy data from Stage 1 expected in the first half of 2024 Dose selection and initiation of Stage 2 to follow in the second half of 2024 NOVATO, Calif., Jan. 25, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that all patients have been dosed with UX701 across the three dose-escalation cohorts in Stage 1 of its pivotal Phase 1/2/3 Cyprus 2+ study. The company's... Read More

NOVATO, Calif., Jan. 19, 2024 (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 12,130 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... Read More

Preliminary 2023 Total Revenue of $430 million to $435 million, Crysvita® Revenue of $325 million to $330 million and Dojolvi® revenue of $70 million to $71 million 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 Year-end 2023 cash balance of approximately $776 million and 2024 guidance for net cash... Read More

NOVATO, Calif., Jan. 04, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that the National Institute for Health and Care Excellence (NICE) has issued a final draft guidance recommending Evkeeza® (evinacumab) to NHS England. Evkeeza is recommended within its marketing authorization as an adjunct to diet and other low-density lipoprotein-cholesterol (LDL-C) lowering therapies for the... Read More

NOVATO, Calif., Jan. 03, 2024 (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 the completion of patient enrollment in its Phase 1/2 clinical trial of GTX-102 for the treatment of pediatric patients with Angelman syndrome (AS). The dose-expansion... Read More

NOVATO, Calif., Jan. 02, 2024 (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 42 nd Annual J.P. Morgan Healthcare Conference on Monday,... Read More

NOVATO, Calif., Dec. 21, 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 reported the grant of 30,325 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... Read More

NOVATO, Calif., Dec. 18, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that the European Commission (EC) has extended the approval of Evkeeza® (evinacumab) as an adjunct to diet and other lipid-lowering therapies to treat children aged 5 to 11 years with homozygous familial hypercholesterolemia (HoFH). Evkeeza is the first angiopoietin-like 3 (ANGPTL3) inhibitor treatment indicated... Read More

6 th Annual Evercore ISI HealthCONx on November 28 in Miami Piper Sandler 35th Annual Healthcare Conference on November 29 in New York City NOVATO, Calif., Nov. 20, 2023 (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 management, will... Read More

NOVATO, Calif., Nov. 17, 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 reported the grant of 22,765 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... Read More

NOVATO, Calif., Nov. 07, 2023 (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 participate in a fireside chat at the upcoming Jefferies London Healthcare... Read More

Third quarter 2023 total revenue of $98.1 million, Crysvita ® revenue of $74.9 million and Dojolvi ® revenue of $16.6 million Reaffirmed 2023 expected total revenue guidance between $425 million to $450 million, Crysvita revenue of $325 million to $340 million, and Dojolvi revenue of $65 million to $75 million Presented positive updates on three lead clinical programs at Analyst Day in October NOVATO, Calif., Nov. 02, 2023... Read More

NOVATO, Calif., Oct. 26, 2023 (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, November 2, 2023, to discuss its financial results and corporate update for the quarter ending September... Read More

NOVATO, Calif., Oct. 23, 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 the closing of its previously announced underwritten public offering of 9,833,334 shares of its common stock, including the exercise in full by the underwriters of their option to... Read More

NOVATO, Calif., Oct. 20, 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 reported the grant of 18,865 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... Read More

NOVATO, Calif., Oct. 18, 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 the pricing of its underwritten public offering of 8,333,334 shares of its common stock at a price to the public of $30.00 per share. In addition, in lieu of issuing common stock to... Read More

NOVATO, Calif., Oct. 17, 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 commenced an underwritten public offering of up to $300,000,000 of shares of its common stock. In addition, the company is expected to grant the underwriters of the offering... Read More

Treatment with setrusumab (UX143) for at least 6 months resulted in 67% reduction in annualized fracture rate in patients with OI in Phase 2/3 Orbit study Quantitative data from the Phase 1/2 study of GTX-102 for AS show clinically meaningful improvements in multiple domains as compared to natural history 4 of 5 patients in lowest-dose cohort of Phase 1/2/3 study of UX701 in Wilson disease are tapering off of chelators... Read More

Phase 2 data presented at ASBMR 2023 show treatment with setrusumab resulted in 67% reduction in annualized fracture rate associated with continuous and meaningful improvements in bone mineral density (BMD) Ultragenyx hosting Analyst Day on Monday, October 16 at 8:30 a.m. ET NOVATO, Calif., VANCOUVER, British Columbia and LONDON, Oct. 14, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) and Mereo... Read More

NOVATO, Calif., Oct. 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 reported the grant of 80,366 restricted stock units of the company’s common stock and the grant of non-qualified stock options to purchase an aggregate of 140,469 shares of common stock of the... Read More

NOVATO, Calif., Oct. 09, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that data from its ongoing late-stage program evaluating setrusumab (UX143) in osteogenesis imperfecta (OI) will be presented at the American Society for Bone and Mineral Research 2023 Annual Meeting (ASBMR) October 13-16, 2023, in Vancouver, British Columbia. Ultragenyx and its collaborators will present... Read More

NOVATO, Calif., Oct. 05, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare genetic diseases, will host an Analyst Day at 8:30 a.m. ET on Monday, October 16, 2023. Ultragenyx leaders and external experts will provide an update on the company’s development pipeline. The agenda will... Read More

TORONTO, Sept. 25, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare genetic diseases, today announced the approval of Evkeeza ® (evinacumab) in Canada. Evkeeza ® was approved by Health Canada as an adjunct to diet and other low-density lipoprotein cholesterol (LDL-C) lowering therapies... Read More

NOVATO, Calif., Sept. 20, 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 reported the grant of 33,475 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... Read More

Citi BioPharma Conference on September 6 in Boston Morgan Stanley Healthcare Conference on September 12 in New York City NOVATO, Calif., Aug. 31, 2023 (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... Read More

NOVATO, Calif., Aug. 18, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare diseases, today reported the grant of 42,840 restricted stock units of the company’s common stock to 18 newly hired non-executive officers of the company. The awards were approved by the compensation committee... Read More

Second quarter 2023 total revenue of $108.3 million, Crysvita® revenue of $83.0 million and Dojolvi® revenue of $16.5 million Total revenue grew 21% and total Crysvita revenue grew 20% versus the second quarter 2022 Reaffirmed 2023 expected total revenue guidance between $425 million to $450 million, Crysvita revenue of $325 million to $340 million, and Dojolvi revenue of $65 million to $75 million NOVATO, Calif., Aug. 03,... Read More

NOVATO, Calif., July 31, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced it has begun dosing the second dose-escalation cohort in its pivotal Phase 1/2/3 Cyprus 2 + study following completion of dosing and safety review in the first cohort. The company's investigational AAV9 gene therapy is designed to deliver stable expression of the ATP7B copper transporter following a single... Read More

NOVATO, Calif., July 27, 2023 (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 3, 2023, to discuss its financial results and corporate update for the quarter ending June 30,... Read More

NOVATO, Calif., July 18, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare diseases, today reported the grant of 36,300 restricted stock units of the company’s common stock to 21 newly hired non-executive officers of the company. The awards were approved by the compensation committee... Read More

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,... Read More

Pivotal Phase 3 portion of Orbit study now enrolling approximately 195 pediatric and young adult patients Newly initiated Phase 3 Cosmic study now enrolling approximately 65 younger pediatric patients NOVATO, Calif., July 06, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that the first patients have been dosed in both of its late-stage clinical trials evaluating setrusumab in... Read More

BEDFORD, Mass., June 21, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. , a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare diseases, today officially opened its gene therapy manufacturing facility in Bedford, Massachusetts. The company has a large... Read More

NOVATO, Calif., June 20, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc . (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare diseases, today reported the grant of 20,405 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... Read More

NOVATO, Calif., June 07, 2023 (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 participate in a fireside at the upcoming Goldman Sachs 44 th Annual Global... Read More

Pediatric data show substantial induction of bone production in 1 week and a large increase in bone formation within 3 months of initiating monthly setrusumab treatment Phase 3 sites beginning to screen patients Conference call to discuss data planned for 5 p.m. Eastern Time NOVATO, Calif. and LONDON, June 05, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) and Mereo BioPharma Group plc (NASDAQ: MREO)... Read More

NOVATO, Calif., May 19, 2023 (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 Eric Crombez, M.D., the company's chief medical officer, will present at the 2023 Barclays - Gene Editing & Gene Therapy Summit on Wednesday, May 24, 2023, at 8:50... Read More

NOVATO, Calif., May 18, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare diseases, today reported the grant of non-qualified stock options to purchase an aggregate of 810 shares of common stock of the company and 31,690 restricted stock units of the company’s common stock to 23 newly... Read More

NOVATO, Calif., May 17, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that the U.S. Food and Drug Administration (FDA) has reviewed and agreed to a protocol amendment to the Phase 1/2 study of GTX-102 in pediatric patients with Angelman syndrome that enables the company to harmonize dose ranges in the U.S. with those being used in ex-U.S. cohorts of the study. Outside of the U.S., the... Read More

NOVATO, Calif., May 08, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultrarare diseases, today announced that clinical, preclinical and manufacturing data from its investigational gene therapy programs will be presented at the American Society of Gene & Cell Therapy (ASGCT) 26 th Annual... Read More

NOVATO, Calif., May 05, 2023 (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 Bank of America’s 2023 Health Care Conference on Wednesday, May 10,... Read More

First quarter 2023 total revenue of $100.5 million, Crysvita ® revenue of $76.0 million and Dojolvi ® revenue of $14.3 million Total revenue grew 26% and Crysvita revenue grew 28% versus the first quarter 2022 Reaffirmed 2023 expected total revenue guidance between $425 million to $450 million, Crysvita revenue of $325 million to $340 million, and Dojolvi revenue of $65 million to $75 million NOVATO, Calif., May 04, 2023... Read More

BEDFORD, Mass., April 28, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. , a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare diseases, today announced Dennis Huang, chief technical operations officer and executive vice president, gene therapy, has... Read More

NOVATO, Calif., April 27, 2023 (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 4, 2023, to discuss its financial results and corporate update for the quarter ending March 31, 2023.... Read More

NOVATO, Calif., April 19, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare diseases, today reported the grant of non-qualified stock options to purchase an aggregate of 11,035 shares of common stock of the company and 17,710 restricted stock units of the company’s common stock to 11... Read More

NOVATO, Calif., March 28, 2023 (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 Guggenheim’s Genomic Medicines and Rare Disease Days on Monday,... Read More

NOVATO, Calif., March 21, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare diseases, today reported the grant of non-qualified stock options to purchase an aggregate of 7,130 shares of common stock of the company and 5,885 restricted stock units of the company’s common stock to ten... Read More

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... Read More

NOVATO, Calif., Feb. 28, 2023 (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 participate in an Orphan Neuro Panel at Cowen’s 43rd Annual Health Care... Read More

2022 Total Collaboration and Product Revenue of $355.6 million, Crysvita ® revenue of $279.4 million and Dojolvi ® revenue of $55.6 million 2023 expected Total Revenue guidance between $425 million to $450 million, Crysvita revenue of $325 million to $340 million, and Dojolvi revenue of $65 million to $75 million Year-end 2022 cash balance of $896.7 million and 2023 guidance for Net Cash used in Operations expected to be... Read More

NOVATO, Calif., Feb. 09, 2023 (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 on Thursday, February 16, 2023, at 5:00 p.m. ET to discuss its financial results and corporate update for the fourth quarter and the... Read More

Preliminary 2022 Total Product Revenue of $352 million to $356 million, Crysvita ® Revenue in Ultragenyx Territories 1 of $257 million to $258 million and Dojolvi ® revenue of $55 million to $56 million 2023 expected Total Product Revenue guidance between $425 million to $450 million, Crysvita revenue of $325 million to $340 million, and Dojolvi revenue of $65 million to $75 million Year-end 2022 cash balance of... Read More

NOVATO, Calif., Jan. 04, 2023 (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 that Emil D. Kakkis, M.D., Ph.D., the company's chief executive officer and president, will present at the 41st Annual J.P. Morgan Healthcare Conference on Tuesday,... Read More

NOVATO, Calif., Dec. 21, 2022 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare diseases, today reported the grant of non-qualified stock options to purchase an aggregate of 29,950 shares of common stock of the company and 17,505 restricted stock units of the company’s common stock to three... Read More