RARE - Ultragenyx Pharmaceutical Inc.

NasdaqGS - NasdaqGS Real Time Price. Currency in USD
90.04
+4.00 (+4.65%)
As of 2:24PM EDT. Market open.
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Performance Outlook
  • Short Term
    2W - 6W
  • Mid Term
    6W - 9M
  • Long Term
    9M+
Previous Close86.04
Open87.06
Bid90.64 x 800
Ask90.70 x 1300
Day's Range86.66 - 90.98
52 Week Range31.99 - 90.98
Volume527,341
Avg. Volume574,672
Market Cap5.38B
Beta (5Y Monthly)2.27
PE Ratio (TTM)N/A
EPS (TTM)-7.36
Earnings DateJul 30, 2020 - Aug 03, 2020
Forward Dividend & YieldN/A (N/A)
Ex-Dividend DateN/A
1y Target Est82.00
  • MarketWatch

    Ultragenyx gets second FdA approval in June

    Shares of Ultragenyx Pharmaceutical Inc. gained 1% in premarket trading on Tuesday, the day after the Food and Drug Administration approved Dojolvi in the treatment of a rare set of genetic metabolic disorders. People with long-chain fatty acid oxidation disorders can't convert fatty acids into energy. "While we have been able to identify these disorders at birth for many years, treatment options have been limited," Dr. Jerry Vockley, chief of medical genetics at Children's Hospital of Pittsburgh of UPMC, said in a news release. "Dojolvi brings hope as a new tool now available to clinicians taking care of these patients." The net price for the drug is $138,000 for adults, according to Bernstein analyst Vincent Chen, who also said "pricing reasonably in line with but slightly to the higher end of expectations." The company's estimates for the patient population in the U.S. range between 2,000 and 3,500 people. Ultragenyx also received another FDA approval in June, for Crysvita as a treatment for some patients with tumor-induced osteomalacia. The company's stock is up 83.1% year-to-date, while the S&P 500 is down 4.0%.

  • GlobeNewswire

    Ultragenyx Announces U.S. FDA Approval of Dojolvi™ (UX007/triheptanoin), the First FDA-Approved Therapy for the Treatment of Long-chain Fatty Acid Oxidation Disorders

    Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved Dojolvi™ (triheptanoin) as a source of calories and fatty acids for the treatment of pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD). LC-FAOD are a group of rare, lifelong and life-threatening genetic disorders in which the body is unable to convert long-chain fatty acids into energy.

  • Ultragenyx Pharmaceutical Inc (RARE): Hedge Funds In Wait-and-See Mode
    Insider Monkey

    Ultragenyx Pharmaceutical Inc (RARE): Hedge Funds In Wait-and-See Mode

    At the end of February we announced the arrival of the first US recession since 2009 and we predicted that the market will decline by at least 20% in (Recession is Imminent: We Need A Travel Ban NOW). In these volatile markets we scrutinize hedge fund filings to get a reading on which direction each […]

  • MarketWatch

    FDA grants a second approval to Ultragenyx drug for rare disease

    The Food and Drug Administration on Thursday approved Ultragenyx Pharmaceutical Inc.'s Crysvita as a treatment for some patients with tumor-induced osteomalacia (TIO), a rare disease that affects between 500 and 1,000 people in the U.S. Crysvita was first approved by the FDA in 2018 for a rare form of rickets. The drug generated net revenue of $103.7 million in 2019. "While the expansion of Crysvita's label into TIO ...is a smaller indication, which may only incrementally increase sales, it further demonstrates RARE's ability to get programs across the finish line, providing more credibility to the mgmt. team," SVB Leerink's Joseph Schwartz wrote in a note to investors on Thursday. A spokesperson for Ultragenyx said the drug costs $160,000 per year for children and $200,000 per year for adults. Ultragenyx's stock has gained 69.7% year-to-date, while the S&P 500 is down 3.5% since the start of the year.

  • Ultragenyx Gets FDA Nod for Label Expansion of Crysvita
    Zacks

    Ultragenyx Gets FDA Nod for Label Expansion of Crysvita

    Ultragenyx (RARE) gets FDA approval for the label expansion of Crysvita into tumor-induced osteomalacia.

  • GlobeNewswire

    Ultragenyx and Kyowa Kirin Announce U.S. FDA Approval of Crysvita® (burosumab) for the Treatment of Tumor-Induced Osteomalacia (TIO)

    NOVATO, Calif. and TOKYO, June 18, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, and Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE: 4151), a global specialty pharmaceutical company creating innovative medical solutions using the latest biotechnology, today announced that the U.S. Food and Drug Administration (FDA) has approved Crysvita® (burosumab) for the treatment of fibroblast growth factor 23 (FGF23)-related hypophosphatemia in tumor-induced osteomalacia (TIO) associated with phosphaturic mesenchymal tumors that cannot be curatively resected or localized in adults and pediatric patients 2 years of age and older. Crysvita is a human antibody that blocks excess activity of FGF23, a hormone that causes phosphate urinary excretion and suppresses active vitamin D production by the kidney.

  • The Week Ahead In Biotech: FDA Decisions In The Cards For Merck, Epizyme, Evoke And Ultragenyx
    Benzinga

    The Week Ahead In Biotech: FDA Decisions In The Cards For Merck, Epizyme, Evoke And Ultragenyx

    Biotech stocks came under pressure along with the broader market in the week ended June 12, as a worsening pandemic situation and its impact on the economy led to an across-the-board sell-off in the global markets.The week kickstarted with the rumor of a big-ticket acquisition. AstraZeneca plc (NYSE: AZN) reportedly approached Gilead Sciences, Inc. (NASDAQ: GILD) with a bid, although experts and analysts shrugged off the possibility of a deal materializing.The FDA promptly approved Viela Bio Inc's (NASDAQ: VIE) drug for treating neuromyelitis optica spectrum disorder.More importantly, five biopharma companies debuted on Wall Street, raising a combined $983.25 million. The week also witnessed virtual presentations at key conferences such as the European Hematology Association Congress.Here are the key catalysts for the unfolding week.Conferences * The Endocrine Society's ENDO Online 2020: June 8-22 * 25th Edition of the European Hematology Association, or EHA, Annual Congress held in virtual format: June 11-21 * American Academy of Dermatology, or AAD, Virtual Meeting Experience: June 12-14 * The American Diabetes Association, or ADA, 80th Scientific Sessions - Virtual: June 12-16 * The World Federation of Hemophilia, or WFH, Virtual Summit: June 14-19PDUFA Dates The FDA is scheduled to give its verdict on Merck & Co., Inc.'s (NYSE: MRK) Keytruda as a monotherapy option for unresectable or metastatic solid tumors, with tissue tumor mutational burden-high. (Tuesday)Ultragenyx Pharmaceutical Inc (NASDAQ: RARE) and its Japanese partner Kyowa Kirin have a tryst with the FDA, as the agency is set to rule on hypophosphatemia treatment candidate burosumab. (Thursday)View more earnings on MRKThe regulatory agency will also decide on Epizyme Inc's (NASDAQ: EPZM) regulatory application for label expansion for tazemetostat, this time for follicular lymphoma. (Thursday)Nabriva Therapeutics PLC - ADR (NASDAQ: NBRV) is knocking at the FDA altar for the second time for its investigational antibiotic contepo for treating complicated urinary tract infection. Late May, the company hinted at the decision being delayed due to the FDA requiring to inspect facilities of third-party manufacturers in Europe (Friday)Evoke Pharma Inc (NASDAQ: EVOK) has a PDUFA date for its Gimoti to treat women with acute and recurrent diabetic gastroparesis. (Friday)See Also: Moderna On Track To Start Phase 3 Coronavirus Vaccine Trial In July Adcom Meetings FDA's Pediatric Oncology Subcommittee of the Oncologic Drugs Advisory Committee will consider through teleconferencing the SP 2577 presentation by Salarius Pharmaceuticals Inc (NASDAQ: SLRX) and marizomib, presentation by Celgene, a subsidiary of Bristol-Myers Squibb Co (NYSE: BMY). SP 2577 is being evaluated for the treatment of relapsed/refractory patients with Ewing sarcoma and marizomib for multiple myeloma.The FDA said the subcommittee will consider and discuss issues relating to the development of each product for pediatric use and provide guidance to facilitate the formulation of written requests for pediatric studies, if appropriate.Clinical Readouts/Presentations ProQR Therapeutics N.V. (NASDAQ: PRQR) will make a virtual presentation of data from the Phase 1/2 trial of intravitreal sepofarsen, an antisense oligonucleotide, in Leber congenital amaurosis 10 due to p.Cys998X mutation in the CEP290 gene. The data is to be shared via a video presentation through the Association for Research in Vision and Ophthalmology. (available online from Monday)Sanofi SA (NASDAQ: SNY) is scheduled to host a virtual scientific session to present data from the Phase 3 COMET trial of investigational enzyme replacement therapy avalglucosidase alfa in patients with late-onset Pompe disease. (Tuesday)Catalyst Biosciences Inc (NASDAQ: CBIO) is due to present at the WFH virtual summit Phase 2a data for factor IX levels of a daily subcutaneous prophylaxis treatment regimen of dalcinonacog alfa in hemophilia B.Earnings * Centogene NV (NASDAQ: CNTG) (Monday, before the market open) * BioNano Genomics Inc (NASDAQ: BNGO) (Thursday, after the close) * Urovant Sciences Ltd (NASDAQ: UROV) (Thursday, after the close)IPOs Royalty Pharma, which buys royalties for late-stage and commercial drugs, has filed to offer 70 million shares in an initial public offering at an estimated price range of $25-$28. The company has applied for listing its shares on the Nasdaq under the ticker symbol RPRX.IPO Quiet Period Expiry Inari Medical Inc (NASDAQ: NARI)See more from Benzinga * The Daily Biotech Pulse: FDA Nod For Viela, 3 IPOs And Hematology Congress Presentations * The Daily Biotech Pulse: Keytruda Setback For Merck, Denali Pulls The Plug On Neurological Asset * The Daily Biotech Pulse: Soleno Flunks Late-Stage Study, Sanofi Strikes Breast Cancer Partnership, NanoViricides Shortlists Coronavirus Treatment Candidates(C) 2020 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.

  • Utragenyx Shares Soar Above 60% YTD on Pipeline Developments
    Zacks

    Utragenyx Shares Soar Above 60% YTD on Pipeline Developments

    Ultragenyx's (RARE) shares rise more than 60% year to date on various positive pipeline developments.

  • Why Is Ultragenyx (RARE) Up 10.7% Since Last Earnings Report?
    Zacks

    Why Is Ultragenyx (RARE) Up 10.7% Since Last Earnings Report?

    Ultragenyx (RARE) reported earnings 30 days ago. What's next for the stock? We take a look at earnings estimates for some clues.

  • GlobeNewswire

    Ultragenyx to Present at Upcoming Investor Conferences

    Shalini Sharp, the company's Chief Financial Officer, will hold a virtual presentation at the Jefferies Healthcare Conference on Wednesday, June 3, 2020 at 2 PM ET. Emil D. Kakkis, M.D., Ph.D., the company's Chief Executive Officer and President, will hold a virtual presentation at the Goldman Sachs 41st Annual Global Healthcare Conference on Tuesday, June 9, 2020 at 3:50 PM ET.

  • GlobeNewswire

    Ultragenyx Announces Exercise of Option to Purchase Additional Stock of Arcturus Therapeutics

    Ultragenyx Pharmaceutical, Inc. (RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases and Arcturus Therapeutics Holdings Inc. (ARCT), a leading messenger RNA medicines company, today announced that Ultragenyx has exercised its option to purchase 600,000 shares of Arcturus common stock at $16.00 per share. “We are encouraged by the advancement of Arcturus’s broad nucleic acid platform across multiple therapeutic areas, including their self-replicating mRNA-based COVID-19 vaccine candidate,” said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx.

  • GlobeNewswire

    Ultragenyx Announces Positive Data from Confirmatory Cohort of Phase 1/2 Study of DTX401 Gene Therapy for Glycogen Storage Disease Type Ia (GSDIa)

    Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, today announced positive initial data from the confirmatory third cohort and longer-term data from the first two cohorts 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 3 responded to therapy and demonstrated more rapid reductions in cornstarch requirements compared to the first two cohorts.

  • GlobeNewswire

    Ultragenyx Announces Positive Longer-term Results from First Three Cohorts of Phase 1/2 Study of DTX301 Gene Therapy in Ornithine Transcarbamylase (OTC) Deficiency

    Six of nine patients in the study have responded to the gene therapy (three female, three male), including all three patients in Cohort 3 who are now confirmed responders. The three previously disclosed complete responders, who have discontinued all ammonia scavengers and liberalized their diet, remain clinically and metabolically stable after longer-term follow-up.

  • GlobeNewswire

    Ultragenyx to Present at BofA Securities Global Health Care 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 hold a virtual presentation at the BofA Securities Global Health Care Conference on Thursday, May 14, 2020 at 10:20 AM ET. The live and archived webcast of the presentation 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.

  • Analyst Estimates: Here's What Brokers Think Of Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) After Its First-Quarter Report
    Simply Wall St.

    Analyst Estimates: Here's What Brokers Think Of Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) After Its First-Quarter Report

    Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) last week reported its latest quarterly results, which makes it a good...

  • Thomson Reuters StreetEvents

    Edited Transcript of RARE earnings conference call or presentation 6-May-20 9:00pm GMT

    Q1 2020 Ultragenyx Pharmaceutical Inc Earnings Call

  • Ultragenyx Pharmaceutical Inc (RARE) Q1 2020 Earnings Call Transcript
    Motley Fool

    Ultragenyx Pharmaceutical Inc (RARE) Q1 2020 Earnings Call Transcript

    RARE earnings call for the period ending March 31, 2020.

  • Ultragenyx (RARE) Q1 Earnings & Sales Fall Short of Estimates
    Zacks

    Ultragenyx (RARE) Q1 Earnings & Sales Fall Short of Estimates

    Ultragenyx (RARE) reports wider-than-expected first-quarter 2020 loss.

  • Ultragenyx (RARE) Reports Q1 Loss, Lags Revenue Estimates
    Zacks

    Ultragenyx (RARE) Reports Q1 Loss, Lags Revenue Estimates

    Ultragenyx (RARE) delivered earnings and revenue surprises of -27.50% and -3.70%, respectively, for the quarter ended March 2020. Do the numbers hold clues to what lies ahead for the stock?

  • GlobeNewswire

    Ultragenyx Reports First Quarter 2020 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 for the quarter ended March 31, 2020 and maintained its full year 2020 financial guidance. The FDA reviews for Crysvita for TIO and UX007 for LC-FAOD are on track to be completed in the coming months.

  • Will Ultragenyx Pharmaceutical Continue to Surge Higher?
    Zacks

    Will Ultragenyx Pharmaceutical Continue to Surge Higher?

    As of late, it has definitely been a great time to be an investor Ultragenyx Pharmaceutical

  • GlobeNewswire

    GeneTx and Ultragenyx Announce GTX-102 Granted Fast Track Designation by U.S. FDA for Treatment of Angelman Syndrome

    GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to GTX-102 for the treatment of Angelman syndrome (AS). GTX-102 is an investigational antisense oligonucleotide currently being evaluated in a Phase 1/2 study for the treatment of AS. “GeneTx is committed to improving the quality of life of individuals living with Angelman syndrome,” said Paula Evans, Chief Executive Officer, GeneTx.

  • GlobeNewswire

    Ultragenyx to Host Conference Call for First Quarter 2020 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 Wednesday, May 6, 2020 at 5pm ET to discuss first quarter 2020 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.