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Monmouth Junction, NJ, August 2, 2006 - Transave, Inc., a biopharmaceutical company focused on the development of inhaled, liposomal formulation of drugs for treating life threatening lung diseases, today announced that the European Agency for the Evaluation of Medicinal Products (EMEA) has granted SLIT™ Amikacin orphan drug status for the treatment of Pseudomonas aeruginosa (Pa) lung infections in cystic fibrosis (CF).
“We are pleased to receive the EMEA’s Orphan Drug designation for SLIT™ Amikacin” said Timothy G. Whitten, President and Chief Executive Officer of Transave, Inc. “This complements the recent granting of orphan drug status for SLIT™ Amikacin by the U.S. FDA (March 2006). We look forward to working in partnership with the regulatory agencies and continuing our efforts with the CF community for the timely development of this novel, inhaled antibiotic.”
Cystic fibrosis is a life-threatening, genetic disease affecting approximately 40,000 people in Europe. Approximately 80% of CF patients are chronically infected with Pa and the infection is associated with a more rapid decline of lung function. More information regarding CF is available through the European Cystic Fibrosis Society (www.ecfsoc.org) and the North America Cystic Fibrosis Foundation (www.cff.org) websites.
About SLIT™ Amikacin
Transave’s product development candidates are based on its proprietary Sustained release Lipid Inhaled Targeting (SLIT™) technology. SLIT™ Amikacin utilizes lipid constituents endogenous to the lung, i.e. the pulmonary surfactant, resulting in a safe and biocompatible inhalable formulation. In addition, SLIT™ Amikacin is designed to provide for sustained release of the antibiotic, effective penetration of SLIT™ particle through the bacterial biofilm and minimized local and systemic side effects. The product profile of SLIT™ Amikacin should allow for once a day or less frequent dosing with an optimized pharmacokinetic profile which provides both high peak lung concentrations and a prolonged therapeutic effect. Transave has begun a 14-day multi-dose Phase IIa trial to confirm the safety and efficacy of nebulized SLIT™ Amikacin in CF patients with chronic Pa infections. An earlier single dose Phase I trial in CF patients demonstrated that the drug is well tolerated at all doses with no significant adverse events.
About the Orphan Drug Act
The European Orphan Drug Act is intended to encourage companies to develop therapies for the treatment of diseases with a prevalence of less than 5:10,000 individuals. Further criteria include the ability of the product to address an unmet medical need where no approved treatment option exists or to provide significant benefit over available treatments. Orphan drug designation, granted by the EMEA’s Office of Medicinal Products, provides the company with a number of potential benefits for SLIT™ Amikacin. Approval by the EMEA of a drug that has been granted orphan drug designation typically results in ten years of market exclusivity in Europe, provided that the sponsor company continues to meet certain conditions established by the EMEA. In addition, during the period of market exclusivity, the EMEA will not approve other applications to market the same drug for the same indication unless the sponsor of the approved orphan drug fails to satisfy the conditions of approval. Other incentives provided by orphan designation include tax credits, protocol assistance and eligibility for research and development support. Protocol assistance includes regulatory assistance and a waiver of certain filing fees, as well as advice on the conduct of clinical trials (EMEA Committee on Orphan Medicines publication; www.emea.eu.int).
About Transave, Inc.
Transave is a privately-held biopharmaceutical company focused on the development of innovative, site-specific treatments for lung diseases. The company’s approach is based on its proprietary Sustained-release Lipid Inhaled Targeting (“SLIT™”) technology designed to sustain release of active drugs in the lung’s microenvironment potentially enhancing the therapeutic benefits. The Company has two products in clinical development focused on pulmonary infections and lung cancer: SLIT™ Amikacin, for Pseudomonas aeruginosa infections for cystic fibrosis and SLIT™ Cisplatin for lung and ovarian cancer.
Inhaled SLIT™ drug formulations utilize lipid constituents endogenous to the lung to make biocompatible, inhalable lipid/drug complexes that are well tolerated. This approach may help to reduce or eliminate toxicities or dosing problems associated with systemic drugs through localized drug administration and controlled release in the lung and create new or improved product profiles for already well-characterized drugs. For more information about Transave’s technology and development programs, visit www.transaveinc.com.
Contact Information:
Andrew J. Gorman, Ph.D.
Vice President, Business Development
Transave, Inc.
732-438-9434
agorman@transaveinc.com
Cambridge, MA 02142
Tel: (617) 231-2400
Fax: (617) 231-2425
82 Devonshire Street, EPC13A
Boston, MA 02109-3614
E-Mail: FidelityBiosciences@fmr.com
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