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Shriners Hospitals for Children's Dr. Michael Whyte Presents First
Efficacy and Safety Results of Targeted Enzyme Replacement Therapy
in Patients With Rare Genetic Bone Disease
Data Presented at The Endocrine Society 2009 Meeting
in Washington, D.C.
ST. LOUIS, June 11, 2009 (GLOBE NEWSWIRE) -- A new experimental targeted enzyme replacement therapy strengthens the bones of infants with a severe, sometimes deadly, genetic bone disorder known as hypophosphatasia (HPP), according to early clinical data presented by Michael P. Whyte, M.D., medical/scientific director of the center for metabolic bone disease and molecular research at Shriners Hospitals for Children - St. Louis.
"There is an urgent need for an effective treatment for these infants whose bones are so brittle that their rib cages often break just from breathing; about half of the babies with the severe infantile form of hypophosphatasia will die before their first birthday," Dr. Whyte said. "Without any approved treatments, we currently can only try to manage symptoms of hypophosphatasia without addressing the underlying problem - a profound lack of bone mineralization. This is the first time we've seen a drug therapy positively impact bone formation in these severely sick infant patients."
Dr. Whyte presented the results of a Phase I safety trial of the experimental treatment - currently known as ENB-0040 - in adults, and early safety and efficacy findings from an ongoing study in severely affected infants at The Endocrine Society's 91st Annual Meeting (the ENDO 09 Conference) today in Washington, D.C. There were no drug-related serious adverse events reported in either study, nor did any of the patients develop anti-ENB-0040 antibodies.
Patients with HPP lack an enzyme called tissue non-specific alkaline phosphatase (TNSALP) that plays a key role in bone formation. ENB-0040 is an enzyme replacement therapy designed to specifically target TNSALP to the bones, with the goal of "normalizing" bone mineralization.
In the infant trial, all patients had undermineralized "soft" and deformed bones (rickets) when they entered the study. After an initial single intravenous dose, the patients received thrice-weekly subcutaneous doses of ENB-0040 for up to six months.
So far, X-rays at three months showed substantial new mineralization in the ribs, wrists, knees and long bones in the first three of five patients. The patients' overall clinical status improved as well, with most showing improved growth and requiring less respiratory support over the course of treatment.
Enobia Pharma, the Montreal, Canada-based company developing ENB-0040, sponsored both studies.
Additional Phase II clinical trials are planned for children and adults with HPP during 2009 (www.clinicaltrials.gov).