GW Pharmaceuticals Announces Positive Phase 3 Pivotal Study Results for Epidiolex® (cannabidiol)

- Study in the treatment of Dravet syndrome – a rare and severe form of epilepsy in children with no FDA-approved treatments -
 
- Primary endpoint achieved with high statistical significance (p=0.01) showing that Epidiolex treatment reduces convulsive seizures in children compared to placebo -
 
- Company to hold investor conference call today at 8:00 a.m. EDT/12:00 Noon GMT -
 
LONDON, March 14, 2016  -- GW Pharmaceuticals plc (GWPH) (GWP.L) (“GW,” “the Company” or “the Group”), a biopharmaceutical company focused on discovering, developing and commercializing novel therapeutics from its proprietary cannabinoid product platform, announces the positive results of the first pivotal Phase 3 study of its investigational medicine Epidiolex® (cannabidiol or CBD) for the treatment of Dravet syndrome. In this study, Epidiolex achieved the primary endpoint of a significant reduction in convulsive seizures assessed over the entire treatment period compared with placebo (p=0.01). Epidiolex has both Orphan Drug Designation and Fast Track Designation from the U.S. Food and Drug Administration (FDA) in the treatment of Dravet syndrome, a rare and debilitating type of epilepsy for which there are currently no treatments approved in the U.S.
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Vericel Announces Positive Top-Line Results From Phase 2b ixCELL-DCM Clinical Trial of Ixmyelocel-T in Patients With Heart Failure Due to Ischemic Dilated Cardiomyopathy

Study Meets Primary Endpoint of Reduction in Clinical Cardiac Events
 
CAMBRIDGE, Mass., March 10, 2016 -- Vericel Corporation (VCEL), a leading developer of patient-specific expanded cellular therapies for the treatment of severe diseases and conditions, today announced top-line results from the company’s Phase 2b ixCELL-DCM clinical trial of ixmyelocel-T in patients with advanced heart failure due to ischemic dilated cardiomyopathy (DCM).  The trial met its primary endpoint of demonstrating a reduction in the total number of deaths, cardiovascular hospitalizations or unplanned outpatient and emergency department visits to treat acute decompensated heart failure during the 12 months following treatment with ixmyelocel-T compared to placebo.  All clinical events in the primary and secondary endpoints were adjudicated in a blinded fashion by an independent adjudication committee.  The incidence of adverse events, including serious adverse events, in patients treated with ixmyelocel-T was comparable to patients in the placebo group.  Ixmyelocel-T has been granted orphan product designation by the U.S. Food and Drug Administration for use in the treatment of DCM.
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Benitec's Hepatitis B Therapy Reduces HBV DNA by 98.5% In Vivo

SYDNEY, March 8, 2016 - Benitec Biopharma Limited (ASX: BLT; NASDAQ: BNTC; NASDAQ: BNTCW) is pleased to announce that BB-HB-331, a DNA-directed RNA interference (ddRNAi) therapy targeting the hepatitis B virus (HBV), demonstrates robust and durable suppression of HBV in vivo following a single administration.

Benitec's ddRNAi technology is a unique combination of gene silencing using RNA interference coupled with the long term therapeutic potential of gene therapy.  BB-HB-331 is comprised of an AAV8 capsid and recombinant DNA engineered to express three short hairpin RNA (shRNA) that target and inhibit viral RNA expressed from three well conserved regions across multiple HBV genotypes.

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Verastem Reports Year-End 2015 Financial Results

BOSTON-- Verastem, Inc. (VSTM), focused on discovering and developing drugs to treat cancer, today reported financial results for the year ended December 31, 2015, and also provided an overview of certain corporate developments.

“We are developing treatments that reduce cancer stem cells and modulate the local tumor microenvironment to allow both cancer treatments and the immune system to do their job more efficiently,” said Robert Forrester, President and Chief Executive Officer of Verastem. “Our recently announced collaborations with Pfizer and Merck KGaA, and with Merck & Co. and Washington University in St. Louis, to evaluate the combination of our FAK inhibitors with immune-oncology agents speak to the understanding among the clinical community that innovative combination therapies have the potential to complement and enhance existing therapies. We begin 2016 with a strong balance sheet and expect significant progress from our ongoing programs targeting high unmet need cancers including non-small cell lung, ovarian, lymphoma, mesothelioma and pancreatic cancer. We are also planning for several trial initiations this year and we look forward to keeping you updated on our progress.”

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Aurina Receives FDA Fast Track Designation for Voclosporin for the Treatment of Lupus Nephritis

VICTORIA, British Columbia-- Aurinia Pharmaceuticals Inc. (NASDAQ:AUPH / TSX:AUP) (“Aurinia” or the “Company”) announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for voclosporin, the Company’s next generation calcineurin inhibitor, for the treatment of Lupus Nephritis (LN).

The Fast Track program was created by the FDA to facilitate the development and expedite the review of new drugs that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address significant unmet medical needs. Compounds that receive this FDA designation benefit from more frequent meetings and communications with the FDA to review the drug’s development plan including the design of clinical trials and the use of biomarkers to support approval. Additionally, Fast Track designation allows the Company to submit parts of the New Drug Application (NDA) on a rolling basis for review as data becomes available.

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