MONTREAL, June 11 /CNW Telbec/ - 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
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, 1 has shown
respiratory and functional improvement and 1 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.
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%. 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
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 US 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.
For further information:
For further information: Julie Anne Smith, Vice-President, Chief
Commercial Officer, Enobia Pharma, (514) 596-2901, extension 214,
JulieAnne.Smith@enobia.com; Media Contact: Hollister Hovey, Lazar Partners
Ltd., (646) 871-8482, firstname.lastname@example.org