Enobia Provides Update on Progress With ENB-0040, a Bone-Targeted Enzyme Replacement Therapy for Hypophosphatasia

MONTREAL, Sept. 8 /PRNewswire/ -- Enobia Pharma today provided an update on key developments in its comprehensive effort to develop ENB-0040 (asfotase alfa), an experimental bone-targeted enzyme replacement therapy, for the treatment of hypophosphatasia (HPP). Hypophosphatasia is a rare, inherited, and sometimes fatal metabolic bone disease that affects individuals of all ages.

Enobia announced today that it has been issued United States Patent No. 7,763,712 B2, providing robust intellectual property coverage for targeting alkaline phosphatase to bone. Coverage also extends to methods used to treat hypophosphatasia. The patent is projected to be in force until 2026.

Enobia was also awarded the Henning Anderson Prize for best clinical abstract from the European Society for Paediatric Endocrinology (ESPE).  The company will present new data from ongoing Phase 2 clinical studies of ENB-0040 in juveniles with HPP on September 24 at ESPE's Annual Meeting in Prague, Czech Republic.  Data will also be presented at the American Society for Bone and Mineral Research (ASBMR) Annual Meeting in Toronto (October 16). These data build on positive preliminary findings presented in June at the 92nd Annual Meeting of The Endocrine Society (ENDO).

In addition, researchers will present new data from Enobia's Phase 2 trials of ENB-0040 in infants at the American Society of Human Genetics (ASHG) in Washington, DC (November 3). These data build on positive results presented at the ASBMR meeting in 2009, which demonstrated that in the majority of patients, six months of treatment with ENB-0040 led to marked improvements in bone mineralization, correction of skeletal defects, better respiratory function, and improvements in motor development.

Enobia also announced that the first patient has been dosed in its adolescent and adult HPP study. This 6-month study in 18 patients will compare the safety and efficacy of two different doses of ENB-0040 to an observational control group.

"We continue to advance all aspects of our clinical development program, and remain intensely focused on strong execution as we work to bring ENB-0040 to HPP patients of all ages as rapidly as possible," Robert Heft, PhD, President and CEO of Enobia. "We are very encouraged by the results we have seen to date in our ongoing Phase 2 clinical trials, and look forward to sharing these with the clinical community in the coming months."

About Hypophosphatasia

Hypophosphatasia is a rare, inherited, and sometimes fatal metabolic bone disease. Affected individuals 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. Clinical severity ranges from the severe perinatal or infantile forms, with marked skeletal hypomineralization and respiratory compromise often causing death, to a persistent 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. First year mortality in these patients is estimated at 50 percent. In the childhood form, patients have varying degrees of skeletal hypomineralization and may have 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 impair ambulation and often debilitating bone pain.

About ENB-0040

There are currently no therapies approved for HPP, a rare genetic disease characterized primarily by defective bone mineralization caused by a deficiency in the enzyme tissue non-specific alkaline phosphatase (TNSALP). ENB-0040 (asfosfotase alfa), an investigational treatment for hypophosphatasia, is a subcutaneous enzyme replacement therapy of tissue non-specific alkaline phosphatase (TNSALP) fused to a patented bone targeting peptide. ENB-0040 is designed to directly target TNSALP to the bone in order to correct the enzyme deficiency, which could lead to restoration of normal bone mineralization. ENB-0040, awarded orphan designation in the U.S. and EU in 2008 and Fast Track status in 2009, is currently in Phase 2 clinical development.

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 are no drug therapies currently approved. ENB-0040, an investigational drug for the treatment of hypophosphatasia, is the Company's lead program. For more information, please visit www.enobia.com.

SOURCE Enobia Pharma Inc.