Advertisement
Dr. Isabelle Lemire presented the positive results from severalpre-clinical studies of ENB-0040 at the American Society of Bone and MineralResearch's 30th Annual Meeting in Montreal, Quebec on September 13th. Resultsof efficacy, safety and toxicology pre-clinical studies supported theinitiation of clinical trials, announced last month.
Advertisement
"We are extremely pleased to see evidence that enzyme replacement therapywith ENB-0040 may help repair skeletal damage caused by hypophosphatasia, aswell as prevent the onset of dangerous bone hypomineralization," said Dr.Lemire, Associate Director of Non Clinical Studies, Enobia Pharma. "We lookforward to advancing our clinical studies and confirming these results inpatients with this deadly disease who currently have no approved treatmentoptions."
The treatment data were not available at the time of the originalpublication of Enobia's pre-clinical studies in the Journal of Bone andMineral Research [June 2008:23:777-787]. In these studies, subcutaneousadministration of ENB-0040 was shown to significantly improve survival andprevent skeletal and dental manifestations of the disease.
Doctors at the University of Manitoba, Winnipeg dosed the first patient inEnobia's clinical program investigating ERT with ENB-0040 as a treatment ofthis rare and often crippling genetic bone disorder in August.
Dr. Thomas Loisel, Associate Director of Process Development from EnobiaPharma will discuss the challenges in engineering the fusion molecule ENB-0040consisting of TSNALP, an immunoglobulin Fc domain, and an anionic peptide usedto target the enzyme to bone at the upcoming IBC 2008 BioprocessingInternational Conference, Sept. 23-26, 2008, in Anaheim, CA.
About Hypophosphatasia
Hypophosphatasia is a rare, inherited, and sometimes fatal metabolic bonedisease. Patients have low levels of the tissue non-specific form of alkalinephosphatase, an important regulator of bone mineralization, leading to ricketsin infants and children and osteomalacia ("soft bones" resulting from poormineralization) in adults. Disease severity is inversely proportional to theage at symptom onset, but morbidity can be cumulative and worsen with age.Clinical severity ranges from the severe perinatal or infantile form, withprofound skeletal hypomineralization and respiratory compromise often causingdeath, to a more slowly progressive and debilitating osteomalacia in adults.
In the infantile form, infants may appear normal at birth but developserious symptoms in the first six months of life. These can include failureto thrive, respiratory failure, fractures, and seizures. Radiographicfindings include generalized hypomineralization and rickets. Mortality inthese patients may be as high as 50%. In the childhood form, patients havevarying degrees of hypomineralization, frank rickets, short stature, bonepain, muscle weakness, delayed motor milestones, early loss of deciduousteeth, and may experience frequent, poorly-healing fractures. In the adultform, the underlying osteomalacia causes bone pain due to overt orpoorly-healing stress fractures that in some cases stops ambulation.
About ENB-0040
ENB-0040 is a fusion protein that includes the catalytic domain of humantissue non-specific alkaline phosphatase (TNSALP), an immunoglobulin Fc domainand a patented anionic peptide used to target
