Preclinical Studies Data Used to Pinpoint Potential Disease-Modifying Therapies & Drug Delivery Candidates for Patients with Rare Diseases

May 19, 2015, 08:40 ET from FN Media Group, LLC

CORAL SPRINGS, Florida, May 19, 2015 /PRNewswire/ --

Clinical-stage biotech companies continue to seek treatments & therapeutic diagnostics using proprietary gene therapy platforms to develop products designed to transform the lives of patients with severe diseases and other rare orphan conditions.  Biotech & Pharma Companies in focus today are:  Amarantus BioScience Holdings, Inc. (OTC:AMBS), Achillion Pharmaceuticals, Inc. (NASDAQ: ACHN), MannKind Corp. (NASDAQ: MNKD), Idera Pharmaceuticals, Inc. (NASDAQ: IDRA) and Synergy Pharmaceuticals Inc. (NASDAQ: SGYP)

Amarantus BioScience Holdings, Inc. (OTCQB:AMBS), a biotechnology company focused on developing therapeutic and diagnostic products for neurological disorders and orphan indications, announced late last week that it has completed a study of mesencephalic-astrocyte-derived neurotrophic factor (MANF) administration to the putamen and the substantia nigra of pigs by convection-enhanced delivery. This study generated MANF brain delivery and distribution data, further supporting the rationale for MANF's development in the treatment of Parkinson's disease (PD). The study was conducted in the United Kingdom in collaboration with Renishaw plc's (LON:RSW) Neurological Applications Department and its leading academic partner, Functional Neurosurgery Research Group at the University of Bristol.

In previously reported studies, MANF has been demonstrated by multiple laboratories to be neuroprotective and neurorestorative in rat models of Parkinson's disease. The current study demonstrated that: (i) MANF can be delivered to the porcine putamen and substantia nigra, brain areas centrally involved in PD, and (ii) that pharmacologically efficacious levels are achieved using Renishaw's convection-enhanced delivery device currently in human clinical development. The porcine brain is considered a good model to evaluate the feasibility of delivering therapeutic agents to the human brain, as it has a significantly larger brain volume than that of a rat or non human primate (NHP). These data demonstrating accurate surgical targeting and distribution of MANF mark an important step in the development of MANF for the treatment of PD.

Read the full AMBS Press Releases at

Amarantus announced today that it will be presenting at two upcoming investor conferences.

Event:                  2015 Marcum Microcap Conference
Date:                   Thursday, May 28, 2015
Time:                   2:30 p.m. EDT / 11:30 a.m. PDT
Location:               Grand Hyatt, New York, NY
Event:                  LD Micro Invitational
Date:                   Tuesday, June 2, 2015
Time:                   1:30 p.m. EDT / 10:30 a.m. PDT
Location:               Luxe Sunset Boulevard, Los Angeles, CA

A webcast of the LD Micro presentation may be accessed via the News and Events page of the Investor Relations section of the Amarantus corporate web site under the IR Calendar at A webcast replay will be available approximately two hours after the presentation ends and will be archived for 30 days.

In other biotech and pharmaceutical company news and developments:  Biotech issue Achillion Pharmaceuticals, Inc. (NASDAQ: ACHN) zoomed higher around midday on Monday, amid reports the company canceled a presentation at today's UBS Global Healthcare Conference.  At last look, the stock was up 11% at $10.62. Against this backdrop, ACHN options are in demand, trading at 12 times the average intraday pace. What's more, the stock's 30-day at-the-money implied volatility rocketed 49.1% higher to 85%.  Read more at

Analysts at Jefferies issued a note last Friday morning saying they see "good interest" for use of MannKind's (NASDAQ: MNKD) Afrezza drug.  Afrezza, the company's signature drug, is an inhalable product to treat diabetes.  The firm reiterated its "buy" rating with a price target of $9 following its recent Afrezza survey.  Read the full article at

Idera Pharmaceuticals, Inc. (NASDAQ: IDRA), a clinical-stage biopharmaceutical company developing toll-like receptor and RNA therapeutics for patients with cancer and rare diseases, announced the achievement of key development milestones for its product candidate IMO-9200, an antagonist of Toll-like receptors (TLRs) 7, 8 and 9. Specifically, the company today reported top-line data from a Phase 1 clinical trial of IMO-9200 in healthy subjects and announced the presentation of new preclinical data for IMO-9200 in models of inflammatory bowel disease (IBD) at the 2015 Digestive Disease Week Conference (DDW) in Washington, DC.

Synergy Pharmaceuticals Inc. (NASDAQ: SGYP), a biopharmaceutical company focused on the development of novel treatments for gastrointestinal diseases and disorders, last Monday reported its financial results and business update for the first quarter ended March 31, 2015.  "We have made significant progress during the first quarter, highlighted by key advancements in our plecanatide phase 3 CIC and IBS-C clinical trials," said Gary S. Jacob, Ph.D., Chairman and CEO of Synergy Pharmaceuticals Inc. "Both phase 3 programs are progressing as expected, and we remain on track to report important milestones in the coming months, the most imminent being top-line data results from our two phase 3 CIC clinical trials."  "2015 is a pivotal year for Synergy and I am confident we are well-positioned to achieve our clinical objectives, and will remain focused on advancing these programs, as well as filing our first NDA with plecanatide for CIC by year-end," added Dr. Jacob.  Read the full report at is leading provider of third party publishing & news dissemination services.  If you would like more information regarding our news coverage solutions, please visit for more details.  Get an edge on the market with our Premium News Alerts that are FREE for a limited time at  Follow us on Facebook: and Twitter: 

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