RARE - Ultragenyx Pharmaceutical Inc.

NasdaqGS - NasdaqGS Real Time Price. Currency in USD
42.57
-0.80 (-1.84%)
At close: 4:00PM EDT
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Previous Close43.37
Open43.20
Bid42.63 x 1300
Ask42.66 x 800
Day's Range42.17 - 44.60
52 Week Range37.44 - 90.98
Volume1,365,747
Avg. Volume506,028
Market Cap2.446B
Beta (3Y Monthly)2.85
PE Ratio (TTM)N/A
EPS (TTM)N/A
Earnings DateN/A
Forward Dividend & YieldN/A (N/A)
Ex-Dividend DateN/A
1y Target EstN/A
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All
News
Press Releases
  • GlobeNewswire

    Ultragenyx and Kyowa Kirin Announce Intent to Submit Supplemental Biologics License Application to U.S. FDA for Crysvita® (burosumab) in Tumor-Induced Osteomalacia (TIO)

    NOVATO, Calif. and TOKYO, Sept. 10, 2019 -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and.

  • GlobeNewswire

    Ultragenyx Announces Positive Data from Phase 1/2 Study of DTX401 Gene Therapy in Glycogen Storage Disease Type Ia

    Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced positive data from the second dose cohort of the ongoing Phase 1/2 study of DTX401, an adeno-associated virus (AAV) based gene therapy for the treatment of glycogen storage disease type Ia (GSDIa). All three patients in Cohort 2 have shown a clinical response with improvements in glucose control and other metabolic parameters compared to baseline.

  • GlobeNewswire

    GeneTx and Ultragenyx Announce Orphan Drug Designation and Rare Pediatric Disease Designation for GTX-102

    GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation and Rare Pediatric Disease Designation to GTX-102 for the treatment of Angelman Syndrome, a serious, debilitating rare neurogenetic disorder that affects approximately 1 in 15,000 people worldwide. “The granting of these designations from the FDA is an enormous milestone for the Angelman community,” stated Paula Evans, Chief Executive Officer of GeneTx.

  • GlobeNewswire

    Ultragenyx to Present at Upcoming Investor Conferences

    The live and archived webcast of the company presentations will be accessible from the company’s website at http://ir.ultragenyx.com/events.cfm. Ultragenyx is a biopharmaceutical company committed to bringing patients novel products for the treatment of serious rare and ultra-rare genetic diseases.

  • GlobeNewswire

    Ultragenyx Announces Partnership with GeneTx to Advance Treatment for Angelman Syndrome

    Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare diseases, and GeneTx Biotherapeutics LLC, today announced a partnership to develop GeneTx’s GTX-102, an antisense oligonucleotide (ASO) for the treatment of Angelman syndrome, a serious, debilitating, rare neurogenetic disorder that affects approximately 1 in 15,000 people worldwide. GTX-102 is currently in late preclinical development with an investigational new drug (IND) application expected to be filed with the U.S. Food and Drug Administration (FDA) in the first half of 2020.

  • GlobeNewswire

    Ultragenyx to Present at Wedbush PacGrow Healthcare Conference

    Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that Tom Kassberg, the company’s Chief Business Officer, will present at the Wedbush PacGrow Healthcare Conference on Wednesday, August 14, 2019 at 1:20 PM ET in New York, NY. The live and archived webcast of the company presentations will be accessible from the company’s website at http://ir.ultragenyx.com/events.cfm. Ultragenyx is a biopharmaceutical company committed to bringing patients novel products for the treatment of serious rare and ultra-rare genetic diseases.

  • GlobeNewswire

    Ultragenyx Reports Second Quarter 2019 Financial Results and Corporate Update

    Strong Crysvita® (burosumab) Launch Continues with Approximately 960 Patients on Reimbursed Commercial Therapy in the United States Ultragenyx Submits New Drug Application to.

  • GlobeNewswire

    Ultragenyx Announces Submission of New Drug Application to FDA for UX007 (triheptanoin) for Treatment of Long-Chain Fatty Acid Oxidation Disorders

    Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare diseases, today announced that it has submitted to the U.S. Food and Drug Administration (FDA) a New Drug Application (NDA) for UX007 (triheptanoin) for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD), a group of genetic disorders in which the body is unable to convert long-chain fatty acids into energy. The FDA previously granted Rare Pediatric Disease Designation and Fast Track designation, which enables eligibility for Priority Review, if relevant criteria are met.

  • GlobeNewswire

    Ultragenyx to Host Conference Call for Second Quarter 2019 Financial Results and Corporate Update

    Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that it will host a conference call on Thursday, August 1, 2019 at 5pm ET to discuss second quarter 2019 financial results and provide a corporate update. Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

  • GlobeNewswire

    Evolus Expands Board of Directors with Appointment of Independent Directors Peter Farrell, Ph.D. and Karah Parschauer, J.D.

    NEWPORT BEACH, Calif., July 15, 2019 -- Evolus, Inc. (NASDAQ: EOLS), a performance beauty company with a customer-centric approach focused on delivering breakthrough products,.

  • GlobeNewswire

    Ultragenyx and Arcturus Therapeutics Expand Existing Research Collaboration and License Agreement to Develop Additional Nucleic Acid Therapies for Rare Diseases

    Scope of the collaboration expands to include up to 12 rare disease targets and includes Arcturus nucleic acid technologies to enable mRNA, DNA, and siRNA therapeutics$30.

  • GlobeNewswire

    Ultragenyx Expands Leadership Team and Promotes Erik Harris to Chief Commercial Officer

    Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced it has promoted Erik Harris to Executive Vice President and Chief Commercial Officer, effective June 11, 2019. Mr. Harris joined Ultragenyx in 2017 as Senior Vice President, Head of North American Commercial Operations, leading the launches of Crysvita® (burosumab) and Mepsevii® (vestronidase alfa).

  • GlobeNewswire

    Ultragenyx Announces Emil D. Kakkis, M.D., Ph.D. as Recipient of BIO’s 2019 Henri A. Termeer Biotechnology Visionary Award

    Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that the Biotechnology Innovation Organization (BIO) has awarded this year’s Henri A. Termeer Biotechnology Visionary Award to Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer, President and founder of Ultragenyx. The award, which will be presented to Dr. Kakkis on June 5 during the BIO 2019 International Convention keynote session, recognizes Dr. Kakkis’ transformative work to accelerate research and development of novel treatments for rare diseases.

  • GlobeNewswire

    Ultragenyx to Present at Bank of America Merrill Lynch Healthcare Conference

    Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that Shalini Sharp, the company’s Chief Financial Officer, will present at the Bank of America Merrill Lynch Healthcare Conference on Tuesday, May 14, 2019 at 3:40 p.m. PT in Las Vegas, NV. The live and archived webcast of the company presentations will be accessible from the company’s website at http://ir.ultragenyx.com/events.cfm. Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of serious rare and ultra-rare genetic diseases.

  • GlobeNewswire

    Ultragenyx Reports First Quarter 2019 Financial Results and Corporate Update

    Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today reported its financial results and corporate update for the quarter ended March 31, 2019. “We are seeing strong, sustained momentum from the Crysvita launch in the United States, driven by increased breadth and depth of prescribers treating both adults and children with XLH,” said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. “We are also on track to submit UX007 for LC-FAOD for regulatory review in the coming months, and are advancing our gene therapy platform with key data readouts expected from two clinical programs.

  • GlobeNewswire

    Ultragenyx to Host Conference Call for First Quarter 2019 Financial Results and Corporate Update

    Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that it will host a conference call on Monday, May 6, 2019 at 5pm ET to discuss first quarter 2019 financial results and provide a corporate update. Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

  • GlobeNewswire

    Ultragenyx Announces UX007 Granted Fast Track Designation and Rare Pediatric Disease Designation by U.S. FDA for Treatment of Long-Chain Fatty Acid Oxidation Disorders

    Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation and Rare Pediatric Disease designation to UX007 for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD), a group of genetic disorders in which the body is unable to convert long-chain fatty acids into energy.

  • GlobeNewswire

    Ultragenyx to Present Corporate Update at Analyst and Investor Day on April 17 in New York

    NOVATO, Calif., April 11, 2019 -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and.

  • GlobeNewswire

    Ultragenyx Announces Approval of Crysvita® (burosumab) in Brazil for the Treatment of X-linked Hypophosphatemia (XLH) in Adults and Children

    Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that Brazil’s National Health Surveillance Agency (ANVISA) has approved Crysvita® (burosumab) for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients one year of age and older. “This approval of Crysvita offers patients in Brazil the first treatment option that targets the underlying cause of XLH, and also marks the first Crysvita approval in Latin America,” said Eduardo Thompson, Senior Vice President and Regional Head, Latin America at Ultragenyx.

  • GlobeNewswire

    New Research Coverage Highlights UnitedHealth Group, Ultragenyx Pharmaceutical, Navistar International, REGENXBIO, Par Pacific, and Athenex — Consolidated Revenues, Company Growth, and Expectations for 2019

    NEW YORK, March 26, 2019 -- In new independent research reports released early this morning, Fundamental Markets released its latest key findings for all current investors,.

  • GlobeNewswire

    Ultragenyx Announces Pricing of Public Offering of Common Stock

    Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced the pricing of its underwritten public offering of 5,072,464 shares of its common stock at a price to the public of $60.00 per share resulting in gross proceeds of $304.3 million, before underwriting discounts. In addition, the company has granted the underwriters of the offering an option for a period of 30 days to purchase up to an additional 760,869 shares of the company's common stock at the public offering price, less the underwriting discount. J.P. Morgan Securities LLC, Goldman Sachs & Co. LLC, BofA Merrill Lynch, and Cowen are acting as joint book-running managers for the offering.

  • GlobeNewswire

    Ultragenyx Announces Proposed Public Offering of Common Stock

    Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that it has commenced an underwritten public offering of up to $250,000,000 of shares of its common stock. In addition, the company is expected to grant the underwriters of the offering an option for a period of 30 days to purchase up to an additional $37,500,000 of shares of common stock at the public offering price, less the underwriting discount. The offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering may be completed.  J.P. Morgan Securities LLC, Goldman Sachs & Co. LLC, BofA Merrill Lynch and Cowen are acting as joint book-running managers for the offering.

  • GlobeNewswire

    Ultragenyx Announces Positive 24-week Data from First Cohort of Phase 1/2 Study of DTX401 Gene Therapy in Glycogen Storage Disease Type Ia

    DTX401 Response in Time to Hypoglycemia and Improved Glucose Control Maintained or Improved in All Three Patients Data from Higher-dose Cohort 2 Expected Mid-2019 NOVATO,.