Enobia Pharma Inc., today announced that it has successfully completed a Phase II study of ENB-0040, a bone targeted enzyme replacement therapy being investigated in juveniles with hypophosphatasia (HPP), a serious, rare metabolic bone disorder. The results were presented by Robert Heft, Ph.D., President and Chief Executive Officer of Enobia, at the 29th Annual J.P. Morgan Healthcare Conference in San Francisco.
The company announced today that the juvenile study has met its primary endpoint, demonstrating a statistically significant improvement in rickets when compared with historical matched cohort controls.
The positive data follow an interim analysis announced in September 2010 demonstrating that all 12 juvenile patients with repeated assessments in the study achieved radiographic and functional improvement in objective measures of walking ability, running speed, agility and strength. There was one non-treatment related discontinuation. The results build on the successful completion of an earlier trial in infants, in which participants continue to demonstrate functional and respiratory improvements in longer-term follow-up.
"We are extremely pleased with these results, which contribute to a mounting body of clinical evidence in support of ENB-0040 as a potential treatment for hypophosphatasia in multiple patient populations," said Dr. Heft. "We are continuing to see progress across all key areas as we prepare for the introduction of this vitally needed therapy."
Full results of the Phase II juvenile study will be presented at the upcoming American College of Medical Genetics Annual Meeting, March 16 – 20 in Vancouver, British Columbia.
Advancing Toward Commercial Readiness
In addition to the successful completion of the juvenile and infantile clinical studies, Dr. Heft highlighted other key achievements by Enobia in 2010, including the issuance of United States patent protection until 2026 for methods of targeting alkaline phosphatase to bone, which also extends to methods used to treat HPP. In addition, the company formalized plans for scale-up to commercial supply, completing a manufacturing agreement with Lonza, a global leader in the production and support of active pharmaceutical ingredients for the life sciences industry.
In the year ahead, Enobia will advance its ongoing discussions with the U.S. Food and Drug Administration and the European Medicines Agency regarding requirements for registration; will work to complete its ongoing Phase II study in adults/adolescents with HPP; and will complete preparations to transition to commercial scale manufacturing. The company expects commercial scale material to be used in clinical trials during 2011.