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Ultragenyx Pharmaceutical Inc. (0LIF.IL)

45.42

+1.31

+(2.96%)

At close: July 23 at 9:00:00 PM GMT+1

Ultragenyx to Host Conference Call for First Quarter 2025 Financial Results and Corporate Update

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

GlobeNewswire 2 days ago

RARE
Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)

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 of the company’s board of directors and granted under the Ultragenyx Empl

GlobeNewswire 10 days ago

RARE
Ultragenyx price target lowered to $115 from $140 at Piper Sandler

Piper Sandler lowered the firm’s price target on Ultragenyx (RARE) to $115 from $140 and keeps an Overweight rating on the shares after analyst coverage transfer. The firm says that while this name boasts a strong commercial business, Piper notes a collection of high value, relatively low risk assets with appropriate balance across early, mid- and late-stage development. Several key clinical and regulatory catalysts are on tap, most near-term and top of mind being the second interim analysis of

TipRanks last month

RARE
Ultragenyx to Participate at Investor Conferences in March

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 2

GlobeNewswire 2 months ago

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Exploring Three High Growth Tech Stocks in the United States

In the last week, the United States market has been flat, yet it has shown a robust 21% increase over the past year, with earnings expected to grow by 14% annually in the coming years. In this context of overall market growth and stability, identifying high growth tech stocks involves looking for companies with strong innovation potential and solid financial health that can capitalize on these favorable conditions.

Simply Wall St. 2 months ago

CMRX

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MVIS
Ultragenyx Announces FDA Acceptance and Priority Review of the Biologics License Application (BLA) for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA)

FDA decision expected by August 18, 2025NOVATO, 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) ac

GlobeNewswire 2 months ago

RARE
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) Just Reported Yearly Earnings: Have Analysts Changed Their Mind On The Stock?

Last week, you might have seen that Ultragenyx Pharmaceutical Inc. ( NASDAQ:RARE ) released its yearly result to the...

Simply Wall St. 2 months ago

RARE
Ultragenyx Reports Fourth Quarter and Full Year 2024 Financial Results and Corporate Update

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 s

GlobeNewswire 2 months ago

RARE
Ultragenyx to Host Conference Call for Fourth Quarter and Full-Year 2024 Financial Results and Corporate Update

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

GlobeNewswire 2 months ago

RARE
Ultragenyx announces new data on UX111 AV gene therapy

Ultragenyx (RARE) Pharmaceutical announced new data demonstrating treatment with UX111 AAV gene therapy led to a statistically significant improvement in the Bayley-IIIi raw scores for the subdomains of cognition, receptive communication and expressive communication in patients with Sanfilippo syndrome type A compared to natural history data from untreated patients. These clinical endpoints were correlated with substantial and sustained reduction in levels of heparan sulfate in cerebrospinal flu

TipRanks 2 months ago

RARE
Ultragenyx Announces New Data Demonstrating that Treatment with UX111 AAV Gene Therapy Significantly Improved Clinical Function Across Multiple Developmental Domains in Children with Sanfilippo Syndrome Type A (MPS IIIA) Correlated with Sustained Reductions in CSF-HS

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

GlobeNewswire 2 months ago

RARE
Ultragenyx Reports Preliminary 2024 Revenue, Financial Guidance for 2025, Pipeline Updates, and 2025 Milestones

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 N

GlobeNewswire 3 months ago

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Ultragenyx to Present at the 43rd Annual J.P. Morgan Healthcare Conference

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 present

GlobeNewswire 3 months ago

RARE
European Commission (EC) Extends the Approval of Evkeeza® (evinacumab) to Children as Young as 6-months Old with Homozygous Familial Hypercholesterolemia (HoFH)

First and only medicine approved in the EU for paediatric patients aged 6-months to 5 years old with HoFH, an ultrarare, inherited form of high cholesterolBASEL, Switzerland, Jan. 06, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialisation of novel therapies for rare and ultrarare genetic diseases, today announced that the European Commission (EC) has extended the approval of Evkeeza® (evinacumab) as an

GlobeNewswire 3 months ago

RARE
NHS England Rolls Out Evkeeza® ▼(evinacumab) for Eligible Adults and Adolescents Aged 12 Years and Older with Homozygous Familial Hypercholesterolaemia (HoFH)

LONDON, Dec. 20, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialisation of novel therapies for rare and ultrarare genetic diseases, today announced that NHS England has implemented the commissioning of Evkeeza (evinacumab) following the National Institute for Health and Care Excellence (NICE) final guidance in September. The use of Evkeeza in eligible people aged 12 years and older will be routinely com

GlobeNewswire 4 months ago

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Ultragenyx Announces First Patient Dosed in Pivotal Phase 3 Aspire Study Evaluating GTX-102 in Angelman Syndrome

Company on track to initiate the Aurora study to evaluate GTX-102 in other Angelman syndrome genotypes and in other age groups in 2025NOVATO, 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

GlobeNewswire 4 months ago

RARE
Ultragenyx Submits Biologics License Application to the U.S. FDA for UX111 AAV Gene Therapy for the Treatment of Sanfilippo Syndrome Type A (MPS IIIA)

If approved, UX111 would be the first approved therapy in the U.S. for Sanfilippo Syndrome Type ANOVATO, 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

GlobeNewswire 4 months ago

RARE
Ultragenyx to Participate in Investor Conferences in December

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 President and Howard Horn, Chief Financial Officer, will participate in a

GlobeNewswire 5 months ago

RARE
Ultragenyx Presents Positive Update on GTX-102 Angelman Syndrome Program at FAST’s 17th Annual Global Science Summit

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 Angelman syndrome, that will be presented at the 2024 Foundation for An

GlobeNewswire 5 months ago

RARE
Ultragenyx Pharmaceutical Third Quarter 2024 Earnings: Beats Expectations

Ultragenyx Pharmaceutical ( NASDAQ:RARE ) Third Quarter 2024 Results Key Financial Results Revenue: US$139.5m (up 42...

Simply Wall St. 5 months ago

RARE

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Ultragenyx Pharmaceutical Inc. (0LIF.IL)

Ultragenyx to Host Conference Call for First Quarter 2025 Financial Results and Corporate Update

Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)

Ultragenyx price target lowered to $115 from $140 at Piper Sandler

Ultragenyx to Participate at Investor Conferences in March

Exploring Three High Growth Tech Stocks in the United States

Ultragenyx Announces FDA Acceptance and Priority Review of the Biologics License Application (BLA) for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA)

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) Just Reported Yearly Earnings: Have Analysts Changed Their Mind On The Stock?

Ultragenyx Reports Fourth Quarter and Full Year 2024 Financial Results and Corporate Update

Ultragenyx to Host Conference Call for Fourth Quarter and Full-Year 2024 Financial Results and Corporate Update

Ultragenyx announces new data on UX111 AV gene therapy

Ultragenyx Announces New Data Demonstrating that Treatment with UX111 AAV Gene Therapy Significantly Improved Clinical Function Across Multiple Developmental Domains in Children with Sanfilippo Syndrome Type A (MPS IIIA) Correlated with Sustained Reductions in CSF-HS

Ultragenyx Reports Preliminary 2024 Revenue, Financial Guidance for 2025, Pipeline Updates, and 2025 Milestones

Ultragenyx to Present at the 43rd Annual J.P. Morgan Healthcare Conference

European Commission (EC) Extends the Approval of Evkeeza® (evinacumab) to Children as Young as 6-months Old with Homozygous Familial Hypercholesterolemia (HoFH)

NHS England Rolls Out Evkeeza® ▼(evinacumab) for Eligible Adults and Adolescents Aged 12 Years and Older with Homozygous Familial Hypercholesterolaemia (HoFH)

Ultragenyx Announces First Patient Dosed in Pivotal Phase 3 Aspire Study Evaluating GTX-102 in Angelman Syndrome

Ultragenyx Submits Biologics License Application to the U.S. FDA for UX111 AAV Gene Therapy for the Treatment of Sanfilippo Syndrome Type A (MPS IIIA)

Ultragenyx to Participate in Investor Conferences in December

Ultragenyx Presents Positive Update on GTX-102 Angelman Syndrome Program at FAST’s 17th Annual Global Science Summit

Ultragenyx Pharmaceutical Third Quarter 2024 Earnings: Beats Expectations