MONTREAL, Sept. 15 /CNW Telbec/ - Enobia Pharma, an emerging biotech
company focused on developing novel therapeutics for serious bone disorders,
presented pre-clinical data demonstrating that its enzyme replacement therapy
(ERT) for hypophosphatasia appears to heal bones that have already been
severely weakened by the disease. Earlier pre-clinical data showed that ERT
with Enobia's product ENB-0040, significantly increases survival and prevents
bone hypomineralization associated with hypophosphatasia, a rare genetic bone
Dr. Isabelle Lemire presented the positive results from several
pre-clinical studies of ENB-0040 at the American Society of Bone and Mineral
Research's 30th Annual Meeting in Montreal, Quebec on September 13th. Results
of efficacy, safety and toxicology pre-clinical studies supported the
initiation of clinical trials, announced last month.
"We are extremely pleased to see evidence that enzyme replacement therapy
with ENB-0040 may help repair skeletal damage caused by hypophosphatasia, as
well as prevent the onset of dangerous bone hypomineralization," said
Dr. Lemire, Associate Director of Non Clinical Studies, Enobia Pharma. "We
look forward to advancing our clinical studies and confirming these results in
patients with this deadly disease who currently have no approved treatment
The treatment data were not available at the time of the original
publication of Enobia's pre-clinical studies in the Journal of Bone and
Mineral Research (June 2008:23:777-787). In these studies, subcutaneous
administration of ENB-0040 was shown to significantly improve survival and
prevent skeletal and dental manifestations of the disease.
Doctors at the University of Manitoba, Winnipeg dosed the first patient
in Enobia's clinical program investigating ERT with ENB-0040 as a treatment of
this rare and often crippling genetic bone disorder in August.
Dr. Thomas Loisel, Associate Director of Process Development from Enobia
Pharma will discuss the challenges in engineering the fusion molecule ENB-0040
consisting of TSNALP, an immunoglobulin Fc domain, and an anionic peptide used
to target the enzyme to bone at the upcoming IBC 2008 Bioprocessing
International Conference, Sept. 23-26, 2008, in Anaheim, CA.
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