MONTREAL, May 19, 2009 (GLOBE NEWSWIRE) -- Enobia Pharma, an emerging biotech company focused on developing novel therapeutics for serious bone disorders, announced that the U.S. Food and Drug Administration (FDA) granted Fast Track status to ENB-0040 for the treatment of hypophosphatasia (HPP). ENB-0040 is Enobia's enzyme replacement therapy (ERT) for HPP, a rare, potentially life-threatening genetic bone disease for which there is no currently approved therapy.
"We're pleased that the FDA has recognized that ENB-0040 may provide a much needed treatment for patients with this devastating, often fatal inherited bone disease," said Hal Landy, MD, Chief Medical Officer and Vice-President of Medical Affairs at Enobia.
Enobia will now have access to ongoing discussions and communications with the FDA to facilitate the ENB-0040 development and review process.
In its letter to the company, the FDA stated that they granted Fast Track designation in part because HPP is an untreated, serious and potentially life-threatening disorder. The FDA went on to note that Enobia's proposal to evaluate the efficacy and safety of ENB-0040 in restoring bone mineralization would target "a serious aspect of the disorder". FDA also stated that "preliminary clinical data have suggested that ENB-0040 has potential as a specific treatment for hypophosphatasia."
Results from the company's Phase I trial in HPP adults and its ongoing trial in severely affected HPP infants will be presented by Michael Whyte, M.D. of the Shriners Hospitals (St. Louis, MO) at The Endocrine Society's 91st Annual Meeting ENDO 09 Conference on Thursday, June 11, 2009 at 9:30 a.m. ET in Washington, DC.
Additional Phase II clinical trials are planned for children and adults with HPP during 2009 (www.clinicaltrials.gov).
About Hypophosphatasia
Hypophosphatasia is a rare, inherited, and sometimes fatal metabolic bone disease. Affected patients have low levels of the tissue non-specific form of alkaline phosphatase, an essential regulator of bone mineralization, leading to rickets in infants and children and osteomalacia ("soft bones" resulting from poor mineralization) in adults. Disease severity is inversely proportional to the age at symptom onset, but morbidity is cumulative and worsens with age. Clinical severity ranges from the severe perinatal or infantile forms, with profound skeletal hypomineralization and respiratory compromise often causing death, to a more slowly progressive and debilitating osteomalacia in adults.
In the infantile form, infants may appear normal at birth but develop serious symptoms in the first six months of life. These can include failure to thrive, respiratory failure, fractures, and seizures. Radiographic findings include generalized hypomineralization and rickets. Mortality in these patients may be as high as 50%. In the childhood form, patients have varying degrees of hypomineralization, frank rickets, short stature, bone pain, muscle weakness, delayed motor milestones, early loss of deciduous teeth, and may experience frequent, poorly-healing fractures. In the adult form, the underlying osteomalacia causes pathological fractures that in some cases stops ambulation.
About ENB-0040
ENB-0040 is a subcutaneous enzyme replacement therapy of tissue non-specific alkaline phosphatase (TNSALP) fused to a patented bone targeting peptide. Preclinical studies in the "knockout" mouse model of severe hypophosphatasia showed that subcutaneous administration of ENB-0040 significantly improved survival, prevented the skeletal and dental manifestations of the disease and corrected skeletal defects in mice with established disease. ENB-0040, awarded orphan designation in the US and EU in 2008, is currently in phase II testing.
About Enobia Pharma Inc.
Enobia Pharma Inc., is a private, Montreal-based company focused on the development of therapeutics to treat serious bone disorders for which there is no currently approved drug therapy. Enzyme Replacement Therapy for the treatment of hypophosphatasia is the Company's lead program.