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Enobia Pharma Announces Successful Completion of Phase II Juvenile Study of ENB-0040, a Bone Targeted Enzyme Replacement Therapy

1/11/2011 7:03:23 AM

SAN FRANCISCO, Jan. 10, 2011 /PRNewswire/ -- 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 (p=0.002). Nine of 13 children enrolled (69%) and nine of 12 children completing the study (75%) achieved a substantial improvement in rickets as assessed by skeletal radiographs of the wrists and knees, compared with two of 17 (12%) historical controls. The radiographs were assessed by blinded radiologists. Rickets, a softening and weakening of the bones, is often seen in children with HPP and can lead to fractures, pain and deformity.

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.

About the ENB-0040 Phase II Juvenile Trial Design

The six-month, Phase II, multinational, open-label investigation of the safety, tolerability, pharmacokinetics (PK), and efficacy of ENB-0040 treatment enrolled 13 children (ages five12 years, Tanner Stage less than or equal to 2) with rickets and gross motor deficits from HPP. One patient discontinued the trial due to elective scoliosis surgery. Inclusion criteria included low serum ALP activity for age, plasma PLP greater than or equal to 2x normal range, normal serum vitamin D, and evidence of HPP associated rickets on radiographs. Exclusion criteria included significant co-morbid disease, nutritional rickets, bisphosphonate exposure, hypocalcemia or hypophosphatemia, or experimental treatment for HPP (e.g., bone marrow transplantation). All patients who have completed the six-month study have been enrolled in an extension study.

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 (asfotase alfa), an investigational treatment for HPP, is a subcutaneous enzyme replacement therapy of TNSALP fused to a 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 2b 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

Company Contact:

Julie Anne Smith, Enobia Pharma Inc.

(514) 596-2901, extension 214

Media Contact:

Feinstein Kean Healthcare

Liz Falcone

(617) 256-6622

SOURCE Enobia Pharma Inc.

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