Healthcare Industry News: Pompe disease
News Release - September 22, 2008
Enobia Pharma Appoints Industry Veterans with Commercial and Manufacturing Expertise to Expand Senior Management TeamMONTREAL, Sept. 22 (HSMN NewsFeed) -- Enobia Pharma, a biotech company focused on developing novel therapeutics for serious bone disorders, today announced key appointments to add commercial and manufacturing expertise to the Company's senior management team in conjunction with the recent initiation of two clinical trials of ENB-0040 for the treatment of hypophosphatasia.
Julie Anne Smith has been appointed as Enobia's Vice President, Chief Commercial Officer, and Jayant Aphale, PhD., MBA has been appointed as Vice President, Manufacturing and Process Sciences. Prior to joining Enobia, Ms. Smith was Vice President, Commercial at Jazz Pharmaceuticals Inc. where she led commercial strategy and operations. Previously, Ms. Smith was Vice President, Global Marketing for Genzyme and led the Global Marketing department for Genzyme's rare disease therapeutics business division. Her responsibilities included leading the worldwide commercialization and planning for Myozyme®, an enzyme replacement therapy for ultra-orphan Pompe disease.
Dr. Aphale was the former Vice President Operations and Project/Portfolio Management at Acambis plc. At Acambis, Dr. Aphale was responsible for R&D project management as well overseeing multiple vaccine manufacturing plants, which included cGMP upstream and downstream manufacturing, and a lyophilization facility. Dr. Aphale previously held Director level positions at Wyeth Vaccines and Diosynth Biotechnology, a biologics contract manufacturing organization. Prior to that, Dr. Aphale was a senior scientist, cGMP manufacturing at Roche Molecular Systems where he was responsible for technology transfer and manufacturing and release of heat stable enzymes.
"Julie and Jayant are seasoned executives who bring to Enobia extensive industry experience that will be extremely valuable as we continue to progress our clinical program for hypophosphatasia and to prepare our commercial efforts," stated Robert Heft, PhD., Chief Executive Officer.
Hypophosphatasia is a rare, inherited, and sometimes fatal metabolic bone disease. Patients have low levels of the tissue non-specific form of alkaline phosphatase, an important 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, but morbidity can be cumulative and worsen with age. Clinical severity ranges from the severe perinatal or infantile form, with profound skeletal hypomineralization and respiratory compromise often causing death, to a more slowly progressive 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. Mortality in these patients may be as high as 50%. In the childhood form, patients have varying degrees of hypomineralization, 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 bone pain due to overt or poorly-healing stress fractures that in some cases stops ambulation.
ENB-0040 is a fusion protein that includes the catalytic domain of human tissue non-specific alkaline phosphatase (TNSALP), and a patented peptide used to target the enzyme to bone. Preclinical studies of ENB-0040 in the "knockout" mouse model of severe hypophosphatasia were recently published in the Journal of Bone and Mineral Research [June 2008:23:777-787] and showed that subcutaneous administration of ENB-0040 significantly improved survival and prevented the skeletal and dental manifestations of the disease.
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 is no currently approved drug therapy. Enzyme Replacement Therapy for the treatment of hypophosphatasia is the Company's lead program. In 2007 Enobia completed a $40M Series B financing lead by OrbiMed Advisors and CTI Life Sciences.
Source: Enobia Pharma
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