Enobia Pharma Inc., today announced that it has successfully completed enrollment in its Phase II study of ENB-0040, a bone targeted enzyme replacement therapy being investigated in adolescents and adults with hypophosphatasia (HPP), a serious, rare metabolic bone disorder. Upon anticipated completion of the study later this year, Enobia will have collected safety and efficacy data for ENB-0040 in HPP patients of all ages. If approved, ENB-0040 would represent the first available therapy for patients with HPP.
In advance of next week's Rare Disease Day, Michael Whyte, MD, Professor of Medicine, Pediatrics and Genetics, and Medical-Scientific Director, Center for Metabolic Bone Disease and Molecular Research, Shriners Hospitals for Children, St. Louis, and principal investigator for the ENB-0040 studies, will provide an update on this potential advance for patients with HPP at the Sanford-Burnham Medical Research Institute's second annual Rare Disease Symposium being held in San Diego, CA this Friday, February 25. The event will highlight ENB-0040 and several other new approaches to treating rare diseases, and will bring together scientists, patients, parents and disease advocates to discuss how they can work together to find cures for rare diseases.
Deborah Sittig, who founded Soft Bones: The U.S. Hypophosphatasia Foundation after her son was diagnosed with HPP, will participate in the event, facilitating a question & answer session with attendees and presenters.
HPP patients and their families are invited to pre-register for the event at: http://www.sanfordburnham.org/rarediseasevideo.aspx. Additional details for accessing the webcast and submitting questions for Dr. Whyte will be provided upon completing registration.
Dr. Whyte's presentation will be webcast from 11:55 am – 12:25 pm PST; the question and answer session will immediately follow, at approximately 12:25 – 1:00 pm PST.
"We are delighted with the steady progress of our development program for ENB-0040, and remain committed to our ultimate goal of delivering a safe and effective therapy to address the significant unmet clinical needs of patients suffering with HPP," said Robert Heft, Ph.D., President and Chief Executive Officer of Enobia. "All of Enobia is privileged to contribute to the development of treatments for rare metabolic bone disorders. We take this opportunity on Rare Disease Day to highlight the need for sustained research to ensure that rare diseases, and the patients and their families impacted by them, receive the attention and therapies they deserve."