MONTREAL, Sept. 19 /CNW Telbec/ - Enobia Pharma, an emerging biotech
company focused on developing novel therapeutics for serious bone disorders,
announced that it has received Orphan Drug designation from the U.S. Food and
Drug Administration (FDA) for ENB-0040, its enzyme replacement therapy (ERT)
for hypophosphatasia, a rare, life-threatening genetic bone disease. In August
2008, Enobia dosed the first patient in a Phase I clinical trial of ENB-0040.
"ENB-0040 represents a potential drug therapy for the patients with
hypophosphatasia, an under-recognized disease that can be fatal in infants and
cause serious disability in older patients that has no currently approved FDA
treatment," said Robert Heft PhD, Chief Executive Officer of Enobia. "The
recent dosing of the first adult hypophosphatasia patient in our Phase I
clinical trial and receipt of orphan drug designation are important milestones
for this program."
The FDA grants Orphan Drug Designation to encourage biotechnology and
pharmaceutical companies to develop products that demonstrate promise for the
treatment of rare diseases affecting fewer than 200,000 people in the United
States. This designation will entitle Enobia to a seven-year period of
marketing exclusivity for the drug upon FDA approval, as well as the
opportunity to apply for funding from the U.S. government to defray costs of
clinical trial expenses, tax credits for clinical research expenses and
potential waiver of the FDA's application user fee.
Hypophosphatasia is a rare, inherited, and sometimes fatal metabolic bone
disease. Patients have low levels of the tissue non-specific form of alkaline
phosphatase, an important 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 can be cumulative and worsen with age.
Clinical severity ranges from the severe perinatal or infantile form, 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 bone pain due to overt or poorly-healing stress
fractures that in some cases stops ambulation.
ENB-0040 is a fusion protein that includes the catalytic domain of human
tissue non-specific alkaline phosphatase (TNSALP), an immunoglobulin Fc domain
and a patented anionic peptide used to target the enzyme to bone. Preclinical
studies of ENB-0040 in the "knockout" mouse model of severe hypophosphatasia
were recently published in the Journal of Bone and Mineral Research
(June 2008:23:777-787) and showed that subcutaneous administration of ENB-0040
significantly improved survival and prevented the skeletal and dental
manifestations of the disease.
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. In 2007 Enobia
completed a $40M Series B financing lead by OrbiMed Advisors and CTI Life
For further information:
For further information: Julie Anne Smith, (514) 596-2901, extension
214; Source: Enobia Pharma