MONTREAL, May 19 /CNW Telbec/ - 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
"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 ENB0-0040 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).
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
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.
For further information:
For further information: Company Contact: Julie Anne Smith, (514)
596-2901, extension 214; Media Contact: Hollister Hovey, Lazar Partners Ltd.,
(646) 871-8482, email@example.com