Specialty Pharma Journal Specialty, Precision & Technology
CAMBRIDGE, Mass. and CARLSBAD, Calif., Dec. 06, 2018 (GLOBE NEWSWIRE) — Biogen Inc (Nasdaq: BIIB) and Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that Biogen exercised its option to obtain from Ionis a worldwide, exclusive, royalty-bearing license to develop and commercialize BIIB067 (IONIS-SOD1RX), an investigational treatment for amyotrophic lateral sclerosis (ALS) with …
Tag Archives: ALS
December, 2018
November, 2018
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26 November
FDA Accepts Biohaven’s NDA for BHV-0223, a Sublingual Delivery Form of Riluzole for Treatment of Amyotrophic Lateral Sclerosis
NEW HAVEN, Conn., Nov. 26, 2018 /PRNewswire/ — Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN) today announced that its 505(b)(2) new drug application (NDA) for BHV-0223, an investigational sublingual form of riluzole for the treatment of people living with amyotrophic lateral sclerosis (ALS), has been accepted for review by the U.S. Food and …
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1 November
Sanofi and Denali Therapeutics Partner to Develop Treatments for Neurological and Inflammatory Diseases
PARIS, France – November 1, 2018 – Sanofi plans to collaborate with Denali Therapeutics Inc. on the development of multiple molecules with the potential to treat a range of neurological and systemic inflammatory diseases. The two lead molecules (DNL747 and DNL758) target a critical signaling protein known as the receptor-interacting serine/threonine-protein …
January, 2018
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25 January
Biogen Acquires Karyopharm’s Neurological Drug in $217 Million Deal
NEWTON, Mass., Jan. 25, 2018 (GLOBE NEWSWIRE) — Karyopharm Therapeutics Inc. (NASDAQ:KPTI) today announced its entry into an agreement for Biogen to acquire Karyopharm’s investigational oral SINE compound KPT-350 and other assets for the treatment of certain neurological and neurodegenerative conditions. KPT-350 is a novel therapeutic candidate that works by …
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11 January
Biohaven Announces Positive Results from Bioequivalence Study with its Amyotrophic Lateral Sclerosis Drug
NEW HAVEN, Conn., Jan. 9, 2018 /PRNewswire/ — Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN) (“Biohaven” or the “Company”) announced positive results today from its bioequivalence study with BHV-0223, an innovative sublingual formulation of riluzole. The study was designed to demonstrate pharmacokinetic equivalence of sublingual BHV-0223 compared to the reference …
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3 January
Sangamo and Pfizer Collaborate for Development of Potential Gene Therapy for Treatment of ALS
RICHMOND, Calif. and NEW YORK, Jan. 3, 2018 /PRNewswire/ — Sangamo Therapeutics, Inc. (Nasdaq: SGMO) and Pfizer Inc. (NYSE: PFE) today announced a collaboration for the development of a potential gene therapy using zinc finger protein transcription factors (ZFP-TFs) to treat amyotrophic lateral sclerosis (ALS) and frontotemporal lobar degeneration (FTLD) …
July, 2017
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28 July
Flex Pharma’s FLX-787 Granted FDA Fast Track Status for Treatment of Severe Muscle Cramps Associated with ALS
Flex Pharma, Inc. (NASDAQ: FLKS), a clinical-stage biotechnology company that is developing innovative and proprietary treatments for cramps and spasticity associated with serious neurological diseases including amyotrophic lateral sclerosis (ALS), multiple sclerosis (MS), and Charcot-Marie-Tooth (CMT), today announced that the US Food and Drug Administration (FDA) has granted Fast Track designation for …
May, 2017
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8 May
FDA Approves MT Pharma America’s Radicava for Treatment of Amyotrophic Lateral Sclerosis
JERSEY CITY, N.J., May 5, 2017 – MT Pharma America, Inc. today announced the U.S. Food and Drug Administration (FDA) has granted approval of RADICAVA™ (edaravone), as an intravenous infusion treatment for amyotrophic lateral sclerosis (ALS), a rapidly progressive neurodegenerative disease in which the majority of patients die within two …
December, 2016
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16 December
Celgene and Evotec Partner for Development of Drugs for Neurodegenerative Diseases
HAMBURG, Germany–(BUSINESS WIRE)–* Exclusive broad R&D collaboration based on Evotec’s unique induced pluripotent stem cell (“iPSC”) platform which enables systematic drug screening in patient-derived disease models Evotec AG (Frankfurt Stock Exchange: EVT, TecDAX, ISIN: DE0005664809) announced today that Evotec and Celgene Corporation have entered into a strategic drug discovery and …
August, 2016
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15 August
New Protein Target Emerges for C9orf72 the Most Common Genetic Form of ALS
WASHINGTON, Aug. 12, 2016 /PRNewswire-USNewswire/ — Targeting a single protein, called SUPT4H1, reduces the levels of the three toxic entities created by the C9orf72 gene expansion, the most common genetic cause of ALS, according to a study published in the journal Science. The finding suggests that SUPT4H1 may be a …