MONTREAL, Nov. 3 Enobia Pharma announced today the initiation of a Phase 2 clinical study of ENB-0040, a bone-targeted enzyme replacement therapy, under investigation for the treatment of hypophosphatasia (HPP). The six-month study will assess the safety, efficacy and pharmacokinetics of ENB-0040 in up to 12 children with HPP. This study follows the recent presentation of positive Phase 2 data in infants, which showed 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 cognitive and motor development.
Enobia also announced today the launch of the Hypophosphatasia Impact Patient Survey (HIPS), an online questionnaire intended to better understand the burden of HPP on patients and their families. The survey is designed to document the disease history, medical history, functional disability, and quality of life in patients with HPP.
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). This enzyme plays a key role in regulating skeletal mineralization. As an enzyme replacement therapy designed to specifically target TNSALP to the bones, ENB-0040 may help correct the enzyme deficiency and restore bone mineralization.
"We continue to make strong progress with ENB-0040, and we are pleased to expand our clinical program with the start of the Phase 2 study in children and the launch of the HPP Impact Patient Survey," said Robert Heft, Ph.D., President and Chief Executive Officer of Enobia. "The expansion of our clinical program and insights gained from our online questionnaire will add to the collective body of knowledge about HPP and support our efforts to bring this promising therapy to patients as quickly as possible."
Study Design and Patient Survey
The Phase 2 study is designed to assess the safety, efficacy, pharmacokinetics and pharmacodynamics of two different doses of ENB-0040 in up to 12 children with HPP aged five to 12. Patients will be randomized to receive subcutaneous injections of either 2 mg/kg or 3 mg/kg ENB-0040 three times weekly for 24 weeks. The efficacy of ENB-0040 will be evaluated primarily based on improvements in rickets (a softening of bones leading to fractures, pain, and deformity), as measured by X-rays.
The clinical study is taking place at centers in the U.S. and Canada and is being led by Dr. Michael Whyte at the Shriners Hospitals for Children in St. Louis, Missouri and Dr. Cheryl Greenberg at the Children's Hospital Health Sciences Centre in Winnipeg, Canada. Enobia expects the results of the study to be available in the second half of 2010.
Enobia's online patient questionnaire, HIPS, can be accessed from the Company's homepage, www.enobia.com. The information gathered from the survey will be kept anonymous and confidential. Enobia is collaborating on HIPS with U.S. Soft Bones, HPP ev, Hypophosphatasie Europe, and Canadian Organization for Rare Disorders (CORD). Patient members who complete HIPS will be able to direct a small donation from Enobia to the participating patient group of their choice.
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.
ENB-0040, 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.