Enobia Pharma: ENB-0040, a Bone Targeted Enzyme Replacement Therapy, Shows Promise in Children with Hypophosphatasia
There are currently no therapies approved for HPP, a rare, inherited, and sometimes fatal metabolic bone disease that affects individuals of all ages. HPP is caused by a deficiency in 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.
"As we have already seen in infants, we are encouraged to see that children with hypophosphatasia begin to show improvements within a few weeks after first receiving ENB-0040. We hope to confirm these early data once the trial is completed. We expect to present the full results from the study in children during the second half of 2010," stated Hal Landy, MD, Chief Medical Officer and Vice President, Medical Affairs at Enobia. "We continue to make strong progress with the ENB-0040 clinical program and are committed to bringing ENB-0040 to patients as quickly as possible."
Study Design
The Phase 2, open-label, historical cohort controlled study is designed to assess the safety, efficacy, pharmacokinetics and pharmacodynamics of two different doses of ENB-0040 in 13 children with HPP between the ages of 5 and 12. Patients were randomized to receive subcutaneous injections of either 2 mg/kg or 3 mg/kg ENB-0040 three times weekly for 24 weeks.
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.
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 typically 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.
About ENB-0040
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.