RARE - Ultragenyx Pharmaceutical Inc.

NasdaqGS - NasdaqGS Real Time Price. Currency in USD
50.71
-0.75 (-1.46%)
At close: 4:00PM EDT
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Performance Outlook
  • Short Term
    2W - 6W
  • Mid Term
    6W - 9M
  • Long Term
    9M+
Previous Close51.46
Open52.77
Bid0.00 x 900
Ask0.00 x 1100
Day's Range50.11 - 53.90
52 Week Range31.99 - 74.11
Volume636,650
Avg. Volume608,637
Market Cap2.999B
Beta (5Y Monthly)2.09
PE Ratio (TTM)N/A
EPS (TTM)-7.12
Earnings DateMay 03, 2020 - May 07, 2020
Forward Dividend & YieldN/A (N/A)
Ex-Dividend DateN/A
1y Target Est72.94
Fair Value is the appropriate price for the shares of a company, based on its earnings and growth rate also interpreted as when P/E Ratio = Growth Rate. Estimated return represents the projected annual return you might expect after purchasing shares in the company and holding them over the default time horizon of 5 years, based on the EPS growth rate that we have projected.
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    • Ultragenyx Inks Deal With Daiichi For Gene Therapy Platform While Also Extending Cash Runway
      SmarterAnalyst

      Ultragenyx Inks Deal With Daiichi For Gene Therapy Platform While Also Extending Cash Runway

      Biopharma Ultragenyx (RARE), which focuses on rare and ultra-rare diseases, has announced a key strategic partnership with Japanese global pharma Daiichi Sankyo.The deal grants Daiichi a non-exclusive license to RARE’s HeLa PCL and HEK293 mammalian cell AAV manufacturing system. Ultragenyx’s HeLa producer cell line (PCL) platform is intended to enable large scale AAV-based gene therapy product manufacturing that is highly reproducible, more consistent, and less expensive than other gene therapy manufacturing platforms.In return, RARE will receive $125M cash and $75M equity (at $60/ share) upfront, $25M upon completion of tech transfer, and single-digit royalties on net sales of future products. The company is currently trading at $44/ share.Crucially, RARE retains an option to co-develop and co-commercialize Daiichi’s rare disease candidates and retains the exclusive right to use its manufacturing system for current and future targets.“We view this last night’s deal with Daiichi Sankyo for non-exclusive rights to RARE’s mammalian AAV manufacturing system as a positive by further validating the company’s gene therapy platform and helping to further bolster the balance sheet” cheered JP Morgan analyst Cory Kasimov on April 1.According to the analyst’s estimates, the $200 million upfront extends the company’s cash runway all the way into 2022, taking any near-term dilutive financing off the table. Plus he is reassured that the non-exclusive nature of the deal does not pose a competitive risk to RARE’s current or future targets.As a result, the analyst reiterated his buy rating on the stock with a $76 price target (71% upside potential). This isn’t the only analyst singing RARE’s praises- overall TipRanks shows that Ultragenyx holds a bullish Strong Buy consensus from the Street with a $72 average analyst price target. (See RARE’s stock analysis on TipRanks)Related News: Battered Cruise Operator Carnival Seeks $6 Billion To Deal With Covid-19 Fallout Verint Systems Misses Profit Estimates, Sees Growing Cloud Demand Momentum Facebook’s User Engagement and Business Interaction Make It Unstoppable, Says Top Analyst More recent articles from Smarter Analyst: * China’s Streaming Platform iQIYI Retaliates Against Short Seller Report * SAGE Announces Massive Restructuring To Focus On Failed Depression Drug * Amazon Halts New Delivery Service To Focus On Demand Surge * GenMark Sees 80% Q1 Revenue Boost on Covid-19 Tests

    • GlobeNewswire

      Ultragenyx Enters into Strategic Partnership with Daiichi Sankyo for Gene Therapy Manufacturing Technology

      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 a strategic partnership and non-exclusive license and technology access agreement with Daiichi Sankyo Company, Limited for Ultragenyx’s proprietary AAV-based gene therapy manufacturing technologies. Ultragenyx’s HeLa producer cell line (PCL) platform enables large commercial-scale AAV-based gene therapy product manufacturing that is intended to be highly reproducible, more consistent, and less expensive than other gene therapy manufacturing platforms.

    • GlobeNewswire

      REGENXBIO and Ultragenyx Announce New License Agreement for Use of NAV® Technology Platform for the Treatment of Rare Metabolic Disorder

      REGENXBIO Inc. (RGNX), a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAV Technology Platform, 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 a new exclusive, worldwide license agreement, extending the companies’ existing gene therapy partnership. Under the terms of the agreement, REGENXBIO has granted Ultragenyx an exclusive, worldwide license, with rights to sublicense, to REGENXBIO’s NAV AAV8 and AAV9 Vectors for the development and commercialization of gene therapy treatments for a rare metabolic disorder.

    • Hedge Funds Were Selling Ultragenyx Pharmaceutical Inc (RARE) Before The Coronavirus
      Insider Monkey

      Hedge Funds Were Selling Ultragenyx Pharmaceutical Inc (RARE) Before The Coronavirus

      Coronavirus is probably the 1 concern in investors' minds right now. It should be. On February 27th we published an article with the title Recession is Imminent: We Need A Travel Ban NOW. We predicted that a US recession is imminent and US stocks will go down by at least 20% in the next 3-6 […]

    • Have Insiders Been Buying Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) Shares This Year?
      Simply Wall St.

      Have Insiders Been Buying Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) Shares This Year?

      It is not uncommon to see companies perform well in the years after insiders buy shares. Unfortunately, there are also...

    • GlobeNewswire

      GeneTx and Ultragenyx Announce First Patient Dosed in Phase 1/2 Clinical Trial of GTX-102 in Patients with 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 it has dosed the first patient in its KIK-AS (Knockdown of UBE3A-antisense in Kids with Angelman Syndrome) study of GTX-102, an experimental antisense oligonucleotide being evaluated for the treatment of Angelman syndrome (AS). The Phase 1/2 open-label, multiple-dose, dose-escalating study will enroll 20 patients to evaluate the safety, tolerability, and potential efficacy of GTX-102 in pediatric patients with Angelman syndrome. This is the first investigational study testing an antisense oligonucleotide as a potential therapy to treat AS.

    • Ultragenyx (RARE) Down 31.1% Since Last Earnings Report: Can It Rebound?
      Zacks

      Ultragenyx (RARE) Down 31.1% Since Last Earnings Report: Can It Rebound?

      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 Barclays Global 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 hold a virtual presentation at the Barclays Global Healthcare Conference on Wednesday, March 11, 2020 at 1:35 PM 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.

    • GlobeNewswire

      Ultragenyx Announces Planned Transition of Chief Financial Officer

      Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare diseases, today announced that Shalini Sharp will step down from her position as Chief Financial Officer and Executive Vice President by September 2, 2020. At such time, Ms. Sharp plans to focus her time on her family and her role as an advisor and board member for various biotechnology companies. Ultragenyx will initiate a search for a successor, and Ms. Sharp will continue to serve as CFO until a replacement is found or until September 2, 2020.

    • GlobeNewswire

      Ultragenyx and Kyowa Kirin Announce FDA Acceptance and Priority Review Designation of Supplemental Biologics License Application for Crysvita® (burosumab) for Tumor-Induced Osteomalacia (TIO)

      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 accepted for review the supplemental Biologics License Application (sBLA) for Crysvita® (burosumab) for the treatment of FGF23-related hypophosphatemia associated with phosphaturic mesenchymal tumors (tumor-induced osteomalacia; TIO) that cannot be curatively resected or localized. The FDA has assigned priority review designation with a Prescription Drug User Fee Act (PDUFA) target date of June 18, 2020.

    • GlobeNewswire

      Ultragenyx to Present at SVB Leerink Global 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 SVB Leerink Global Healthcare Conference on Wednesday, February 26, 2020 at 11:00 AM ET in New York, NY. 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.

    • Thomson Reuters StreetEvents

      Edited Transcript of RARE earnings conference call or presentation 13-Feb-20 10:00pm GMT

      Q4 2019 Ultragenyx Pharmaceutical Inc Earnings Call

    • Ultragenyx (RARE) Reports Wider-Than-Expected Loss in Q4
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      Ultragenyx (RARE) Reports Wider-Than-Expected Loss in Q4

      Ultragenyx (RARE) reports a wider-than-expected loss and beats sales estimates in the fourth quarter of 2019.

    • GlobeNewswire

      Ultragenyx Reports Fourth Quarter and Full Year 2019 Financial Results and Corporate Update

      2019 total revenue is $103.7 million; 2019 Crysvita® (burosumab) revenue to Ultragenyx is $87.3 million 2020 Crysvita revenue in Ultragenyx territories guidance of $125.

    • GlobeNewswire

      Ultragenyx to Host Conference Call for Fourth Quarter and Full-Year 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, February 13, 2020 at 5pm ET to discuss its financial results and corporate update for the fourth quarter and the year ended December 31, 2019. 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.

    • How Should Investors Feel About Ultragenyx Pharmaceutical Inc.'s (NASDAQ:RARE) CEO Pay?
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      How Should Investors Feel About Ultragenyx Pharmaceutical Inc.'s (NASDAQ:RARE) CEO Pay?

      Emil Kakkis has been the CEO of Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) since 2010. First, this article will...

    • Benzinga

      The Daily Biotech Pulse: Osmotica Soars On Insider Buys, Ultragenyx's Partnered Rare Disease Drug Moves Into Clinics

      Here's a roundup of top developments in the biotech space over the last 24 hours. Scaling The Peaks (Biotech Stocks Hitting 52-week highs on Jan. 15) 10X Genomics Inc (NASDAQ: TXG ) Aimmune Therapeutics ...

    • GlobeNewswire

      GeneTx and Ultragenyx Announce Investigational New Drug (IND) Application Active for GTX-102 in Patients with Angelman Syndrome

      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 GeneTx’s Investigational New Drug (IND) Application for GTX-102, an experimental antisense oligonucleotide being evaluated for the treatment of Angelman syndrome (AS), filed with the U.S. Food and Drug Administration (FDA) is now active. "The FDA’s clearance of the IND to evaluate GTX-102 in patients with Angelman syndrome represents a significant milestone for the Angelman community,” said Paula Evans, Chief Executive Officer at GeneTx.

    • Ultragenyx Provides Preliminary Crysvita 2019 Revenues
      Zacks

      Ultragenyx Provides Preliminary Crysvita 2019 Revenues

      Ultragenyx (RARE) provides preliminary 2019 revenue numbers and 2020 outlook for Crysvita, along with other pipeline updates.

    • Ultragenyx's Gene Therapy Positive in Genetic Disorder Study
      Zacks

      Ultragenyx's Gene Therapy Positive in Genetic Disorder Study

      Shares of Ultragenyx (RARE) rise 23.4% after releasing positive top-line data from the phase I/II study of its gene therapy candidate DTX301 for the treatment of OTC deficiency.

    • GlobeNewswire

      Ultragenyx Reports Preliminary 2019 Revenue and Provides 2020 Crysvita Revenue Guidance

      Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today reported preliminary unaudited 2019 revenue and cash and investments at year end 2019, and provided 2020 revenue guidance for Crysvita in Ultragenyx territories.

    • GlobeNewswire

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

      Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, and Kyowa Kirin Co., Ltd., (Kyowa Kirin, TYO: 4151) today announced that they submitted a supplemental Biologics License Application (sBLA) to the U.S. Food and Drug Administration (FDA) on December 18, 2019, for Crysvita® (burosumab) for the treatment of FGF23-related hypophosphatemia associated with phosphaturic mesenchymal tumors (tumor-induced osteomalacia; TIO) that cannot be curatively resected or localized. The companies expect to hear back from FDA on submission acceptance and review designation in February 2020. “Approximately half of patients with TIO have tumors that cannot be surgically removed, leaving them with no other current treatment options,” said Camille L. Bedrosian, M.D., Chief Medical Officer of Ultragenyx.

    • Benzinga

      The Daily Biotech Pulse: Positive Data Readouts To Spur Aclaris, Moderna And Ultragenyx

      Here's a roundup of top developments in the biotech space over the last 24 hours. Scaling The Peaks (Biotech Stocks Hitting 52-week highs on Jan. 9) Acceleron Pharma Inc (NASDAQ: XLRN ) Allergan plc (NYSE: ...

    • GlobeNewswire

      Ultragenyx Announces Positive Topline Cohort 3 Results and Improved Longer-Term Cohort 2 Results from Phase 1/2 Study of DTX301 Gene Therapy in Ornithine Transcarbamylase (OTC) Deficiency

      Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced topline positive safety and efficacy data from Cohort 3 and longer-term data from Cohort 2 of the ongoing Phase 1/2 study of DTX301, an investigational adeno-associated virus (AAV) gene therapy for the treatment of ornithine transcarbamylase (OTC) deficiency. In Cohort 3 (n=3), there were two confirmed female responders as well a third potential male responder who requires longer-term follow-up to confirm response status.