MONTREAL, June 11, 2009 (GLOBE NEWSWIRE) -- Enobia Pharma, an emerging biotech company focused on developing novel therapeutics for serious bone disorders, announced that results from two early-stage studies of the company's lead product, ENB-0040, were presented today during a Symposium Session at the Endocrine Society's 91st Annual Meeting (ENDO 09) in Washington, DC. ENB-0040, a bone targeted enzyme replacement therapy, is under investigation as a treatment for hypophosphatasia (HPP), a severe, sometimes deadly genetic bone disorder.
Michael P. Whyte, M.D., Medical/Scientific Director of the Center for Metabolic Bone Disease and Molecular Research at Shriners Hospitals for Children in St. Louis presented the results of Enobia's Phase I safety trial of ENB-0040 in adults and early safety and efficacy findings from an ongoing study of infants with the severe form of HPP.
There were no drug-related serious adverse events reported in either study, nor did any of the patients develop anti-ENB-0040 antibodies. After 12 weeks of treatment, three of five treated infants with profound rickets have shown marked radiographic, respiratory and functional improvement, one has shown respiratory and functional improvement and one has shown functional gains.
"We're encouraged by these early findings and look forward to seeing the full results of the infantile study, which will be presented at the American Society of Bone Mineral Research (ASMBR) meeting in September," said Hal Landy, MD, Chief Medical Officer & VP, Medical Affairs of Enobia. "Additional Phase II clinical trials in children and adults with HPP are planned to commence this summer."
Shriners issued a statement regarding Dr. Whyte's presentation. To download the press release, please visit www.enobia.com.
About Hypophosphatasia
Hypophosphatasia is a rare, inherited, and sometimes fatal metabolic bone disease. Affected individuals 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 can worsen with age. Clinical severity ranges from the severe perinatal or infantile forms, with profound skeletal hypomineralization and respiratory compromise often causing death, to 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 percent. 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 manifestations of the disease and corrected skeletal defects in mice with established disease. ENB-0040, awarded orphan designation in the U.S. and EU in 2008 and Fast Track status in 2009, 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.