Enobia Pharma Presents PreClinical Data Showing ENB-0040 Significantly Improved Survival and Healed Skeletal Manifestations of Severe Hypophosphatasia in Mice
MONTREAL, Sept. 15 /PRNewswire/ — 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 disease.
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 options.”
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 Sciences.
Company Contact: Julie Anne Smith, (514) 596-2901, extension 214
CONTACT: Julie Anne Smith, +1-514-596-2901, extension 214, for EnobiaPharma