Company Overview and News
Ultragenyx Pharmaceutical Inc. (RARE - Free Report) announced positive top-line safety and efficacy data from the second dose cohort and positive longer-term data from the first dose cohort of the ongoing OTC phase I/II study of its adeno-associated virus (“AAV”) gene therapy candidate — DTX301.DTX301 is being evaluated forthe treatment of ornithine transcarbamylase (OTC) deficiency.
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NOVATO, Calif., Sept. 26, 2018 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that Emil D. Kakkis, M.D., Ph.D., the company's Chief Executive Officer, will present at the Jefferies Gene Therapy Summit on Thursday, September 27, 2018 at 8:00am ET in New York.
2018-09-25 globenewswire - 3
SAN DIEGO, Sept. 25, 2018 (GLOBE NEWSWIRE) -- Arcturus Therapeutics Ltd. (NASDAQ: ARCT), a leading RNA medicines company, today announced that Joseph Payne will present at four investor conferences in October: Microcap Conference, Ladenburg Thalmann 4th Annual Healthcare Conference, LEERINK Partners Roundtable Series – Rare Disease & Oncology, and Chardan 2nd Annual Genetic Medicine Conference. In addition, Arcturus will be present at the invitation-only JLABS CEO Summit where Melinda Richter, Global Head, Johnson & Johnson Innovation, JLABS, will host a fireside chat with Arcturus CEO Joseph Payne.
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GAINESVILLE, Fla. and CAMBRIDGE, Mass., Sept. 24, 2018 (GLOBE NEWSWIRE) -- Applied Genetic Technologies Corporation (NASDAQ: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, today announced it has appointed William Aliski to the company’s Board of Directors.
SAN DIEGO, Sept. 21, 2018 (GLOBE NEWSWIRE) -- Arcturus Therapeutics Ltd. (NASDAQ: ARCT), a leading RNA medicines company, today announced the appointment of Kevin T. Skol as Senior Vice President of Business Development & Alliance Management, and Suezanne Parker, Ph.D., as Vice President of Translational Biology. In parallel with these appointments, KC Kummerfeld CPA is promoted to Vice President of Finance and Corporate Controller reporting to Interim CFO Andy Sassine.
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rare-8k_20180917.htm UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 _______________
NOVATO, Calif., Sept. 17, 2018 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that it has expanded its leadership team with the appointment of Wladimir (Vlad) Hogenhuis, M.D., as Chief Operating Officer, effective September 28, 2018. In this newly created role, Dr.
Coda Biotherapeutics launched in South San Francisco with a $19 million Series A financing. Lead investors were MPM Capital and Versant Ventures. They were joined by Astellas Venture Management and Novartis.
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2018-08-31 seekingalpha - 7
Karyopharm Therapeutics (KPTI) reported that they plan to present, at the Society of Hematologic Oncology (SOHO) Annual Meeting in Houston, data from their Phase 2b STORM study evaluating selinexor in heavily pretreated patients with refractory multiple myeloma (NYSE:MM). The presentation is scheduled on September 12-15.
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NOVATO, Calif., Aug. 30, 2018 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that Shalini Sharp, the Company's Chief Financial Officer, will present at the following upcoming investor conferences:
2018-08-29 sec.gov - 1
rare-8k_20180827.htm UNITED STATES
Positive top-line results from the pivotal Phase 3 trial of Esperion’s (ESPR) bempedoic acid/ezetimibe combination pill study (1002-053) has been reported by the company. This trial was conducted in a 380 high-risk patient group with maximally tolerated statins. The trial evaluated the efficacy and safety of the bempedoic acid 180 mg/ezetimibe 10 mg combination pill compared to bempedoic acid, ezetimibe or placebo.
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NOVATO, Calif., Aug. 29, 2018 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted Ultragenyx’s most recent proposal to submit a New Drug Application (NDA) for UX007 for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD) based on existing data.
2018-10-01 - Asif
Overview The following discussion and analysis should be read in conjunction with its unaudited interim condensed consolidated financial statements and the related notes that appear elsewhere in this quarterly report on Form 10-Q. This discussion contains forward-looking statements reflecting its current expectations that involve risks and uncertainties. Actual results may differ materially from those discussed in these forward-looking statements due to a number of factors, including those set forth in the section entitled “Risk Factors” in its most recent annual report on Form 10-K. For further information regarding forward-looking statements, please refer to the “Special Note Regarding Forward-Looking Statements and Projections” immediately after the index to this quarterly report on Form 10-Q. Alimera Sciences, Inc., and its subsidiaries (we or Alimera), is a pharmaceutical company that specializes in the commercialization and development of prescription ophthalmic pharma...
2018-09-25 - Asif
Background DelMar Pharmaceuticals, Inc. is a clinical stage drug development company with a focus on the treatment of cancer. The company's mission is to benefit patients and create shareholder value by developing and commercializing anti-cancer therapies for patients whose tumors exhibit features that make them resistant to, or unlikely to respond to, currently available therapies, particularly for orphan cancer indications where patients have failed, or are unlikely to respond to, currently available therapy. DelMar Pharmaceuticals is developing VAL-083, a novel, DNA-targeting agent, for the treatment of glioblastoma multiforme (“GBM”) and potentially other solid tumors, including ovarian cancer. VAL-083 is a first-in-class, DNA-targeting chemotherapeutic that demonstrated activity against a range of tumor types in prior Phase 1 and Phase 2 clinical studies sponsored by the US National Cancer Institute (“NCI”). The company's recent research has highlighted the opportunit...
2018-09-18 - Asif
Overview Rigel Pharmaceuticals is a biotechnology company dedicated to discovering, developing and providing novel small molecule drugs that significantly improve the lives of patients with immune and hematologic disorders, cancer and rare diseases. The company's pioneering research focuses on signaling pathways that are critical to disease mechanisms. The company's first FDA-approved product is TAVALISSE™ (fostamatinib disodium hexahydrate), an oral spleen tyrosine kinase (SYK) inhibitor, for the treatment of adult patients with chronic immune thrombocytopenia who have had an insufficient response to a previous treatment. The company's current clinical programs include Phase 2 studies of fostamatinib in autoimmune hemolytic anemia and IgA nephropathy, and a Phase 1 study for its IRAK program. In addition, Rigel Pharmaceuticals has product candidates in development with partners BerGenBio AS, Daiichi Sankyo, and Aclaris Therapeutics. Since inception, Rigel Pharmaceuticals h...
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