Leading Biotech Companies Seek Biologic Treatments for Rare Diseases Utilizing Viable Therapeutic Approach - Company Receives FDA Orphan Drug Designation for Bryostatin

CORAL SPRINGS, Florida, April 9, 2015 /PRNewswire/ --

According to a recent article published on the Wall Street Journal, the debate over prescription drug pricing has grown increasingly contentious, particularly as drug makers issue more new medicines with high price tags. But despite the tumult, the cost for orphan drugs - medicines that are designated by the FDA to treat a rare disease and may cost as much as $400,000 annually - is apparently not fazing some insurers.  Billions of dollars a year are being earmarked for new clinical studies and trials for the treatment of neurodevelopmental disorders as leading biotech companies race to identify treatments for genetic, cognitive, and neural levels for autism disorders.  Biotech Companies in focus today are Neurotrope, Inc. (OTC: NTRP), Alkermes plc (NASDAQ: ALKS), Regulus Therapeutics Inc. (NASDAQ: RGLS), Inovio Pharmaceuticals, Inc. (NASDAQ: INO), Gilead Sciences Inc. (NASDAQ: GILD) and Abbot Laboratories (NYSE: ABT).

Neurotrope, Inc. (OTCQB: NTRP) today announced that Neurotrope BioScience, Inc., its wholly-owned operating subsidiary, (the "Company") has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for its lead proprietary drug candidate, bryostatin, in the treatment of Fragile X Syndrome (FXS).  Bryostatin is a potent activator of Protein Kinase C (PKC), which the Company believes is a viable therapeutic approach for the treatment of Fragile X Syndrome.  Neurotrope is developing bryostatin under a licensing agreement with the Blanchette Rockefeller Neuroscience Institute (BRNI).

Read the full NTRP Press Release at http://www.financialnewsmedia.com/profiles/ntrp.html

"We are pleased to have received orphan drug designation for bryostatin for the treatment of FXS which represents an area of significant unmet need for patients and families who live with the consequences of this genetic disorder," said Charles S. Ramat, President and Chief Executive Officer of Neurotrope, Inc. "One of the key strategies for our Company's future is the licensing of novel therapeutics to develop treatments for orphan diseases such as FXS.   In keeping with this strategic imperative, we have also initiated preclinical work with bryostatin for the treatment of Niemann-Pick type C, a rare devastating genetic disorder in children.  As we progress our clinical development programs for Fragile X and NPC we continue to work towards building a robust portfolio of drug development candidates in orphan disease indications."  Orphan drug designation is granted by the FDA Office of Orphan Products Development (OOPD) to novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. The designation provides the drug developer with a seven-year period of U.S. marketing exclusivity, as well as tax credits for clinical research costs, the ability to apply for annual grant funding, clinical research trial design assistance and waiver of Prescription Drug User Fee Act (PDUFA) filing fees. The OOPD also works on rare disease issues with the medical and research communities, professional organizations, academia, governmental agencies, industry, and rare disease patient groups.

About Fragile X Syndrome - FXS is the most common cause of inherited intellectual disability and the most common known genetic cause of autism or autism spectrum disorders. There is currently no FDA approved treatment for FXS available on the market today.  Symptoms of FXS include moderate to severe learning disabilities, behavioral disorders, seizures and cognitive impairment.  FXS is caused by a partial or a full mutation of the FMR1 gene.

In other Biotech News & Happenings in the sector: Alkermes plc (NASDAQ: ALKS) recently announced positive topline results from the complete, six-month, randomized, dose-ranging phase 2 study of ALKS 3831, an investigational, novel, oral atypical antipsychotic drug candidate designed to be a broad-spectrum treatment for schizophrenia. The study was designed in two stages: for the initial three months, patients were randomized to receive olanzapine or one of three doses of ALKS 3831, and antipsychotic efficacy and weight gain were assessed. Positive topline data from this stage were announced in January 2015, showing that ALKS 3831 met the study's primary endpoint, demonstrating antipsychotic efficacy equivalent to olanzapine, as well as key secondary endpoints showing ALKS 3831's favorable effects on weight gain compared to olanzapine. For the second three months, all patients who received ALKS 3831 during the initial three months continued to receive ALKS 3831, and patients who had received olanzapine were switched to ALKS 3831. Data from the completed study support and extend the initial positive results showing ALKS 3831's favorable efficacy and mean weight gain profile and show for the first time that switching patients from olanzapine to ALKS 3831 resulted in a cessation of mean weight gain.

Regulus Therapeutics Inc. (NASDAQ: RGLS), a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, recently announced that the European Commission has granted orphan medicinal product designation for RG-012, a single stranded, chemically modified oligonucleotide that binds to and inhibits the function of microRNA-21 ("miR-21") for the treatment of Alport syndrome, a life-threatening genetic kidney disease with no approved therapy. In July 2014, the U.S. Food & Drug Administration granted orphan drug designation to RG-012 for the treatment of Alport syndrome. "We are pleased to have received orphan medicinal product designation in the European Union for RG-012, a key microRNA therapeutic program under our 'Clinical Map Initiative'," said Paul Grint, M.D., Chief Medical Officer of Regulus.

Inovio Pharmaceuticals, Inc. (NASDAQ: INO) recently announced that the company has been selected to receive a grant from the Defense Advanced Research Projects Agency (DARPA) to lead a collaborative team to develop multiple treatment and prevention approaches against Ebola. Inovio is the prime contractor on the DARPA program. Other collaborators are: MedImmune, the global biologics research and development arm of AstraZeneca; GeneOne Life Sciences and its manufacturing subsidiary, VGXI, Inc.; and Professor David B. Weiner, PhD, professor of Pathology and Laboratory Medicine at The Perelman School of Medicine at the University of Pennsylvania, Emory University and Vanderbilt University.

Gilead Sciences Inc. (NASDAQ: GILD) recently announced that it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for two doses of an investigational fixed-dose combination of emtricitabine and tenofovir alafenamide (200/10 mg and 200/25 mg) (F/TAF) for the treatment of HIV-1 infection in adults and pediatric patients age 12 years and older, in combination with other HIV antiretroviral agents. TAF is a novel nucleotide reverse transcriptase inhibitor (NRTI) that has demonstrated high antiviral efficacy at a dose less than one-tenth that of Gilead's Viread^® (tenofovir disoproxil fumarate, TDF), as well as improved renal and bone laboratory parameters as compared to TDF in clinical trials.

Abbot Laboratories (NYSE: ABT) manufactures and sells health care products worldwide. Its Established Pharmaceutical Products segment offers branded generic pharmaceuticals for the treatment of pancreatic exocrine insufficiency; irritable bowel syndrome; intrahepatic cholestasis or depressive symptoms; gynecological disorders; dyslipidemia; hypertension; hypothyroidism; pain, fever, and inflammation; hormone replacement therapy; anti-infective and influenza vaccines; and product that regulates physiological rhythm of the colon. On Wednesday, ABT closed up .91% on over 6 million shares traded.

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