|Bid||90.64 x 800|
|Ask||90.70 x 1300|
|Day's Range||86.66 - 90.98|
|52 Week Range||31.99 - 90.98|
|Beta (5Y Monthly)||2.27|
|PE Ratio (TTM)||N/A|
|Earnings Date||Jul 30, 2020 - Aug 03, 2020|
|Forward Dividend & Yield||N/A (N/A)|
|1y Target Est||82.00|
Ultragenyx (RARE) saw a big move last session, as its shares jumped more than 8% on the day, amid huge volumes.
Ultragenyx (RARE) gets FDA approval forDojolvi for the treatment of pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders.
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%.
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.
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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.
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.
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.
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.
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.
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.
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.
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.
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) last week reported its latest quarterly results, which makes it a good...
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?
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.
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.
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.