Company Highlights Clinical Progress, Advances toward Commercial
Preparedness at J.P. Morgan Healthcare Conference
SAN FRANCISCO, CA, Jan. 10 /CNW Telbec/ - 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 9 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
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
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 5—12 years, Tanner Stage ≤ 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 ≥ 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 6 month study have been enrolled in an
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 www.enobia.com.
SOURCE ENOBIA PHARMA
For further information:
Julie Anne Smith, Enobia Pharma Inc.
(514) 596-2901, extension 214
Feinstein Kean Healthcare