Ceregene, Inc., a biopharmaceutical company, announced today that enrollment is proceeding in a new Phase 1/2 clinical study evaluating CERE-120, a gene therapy product which delivers the neurotrophic factor neurturin, to dying neurons in Parkinson's disease patients. This new clinical study follows a completed Phase 2 trial and builds on experience gained in that trial, by enhancing the dosing regimen and optimizing the duration of patient follow up.
The new study was initiated in the fourth quarter of 2009 and is proceeding as planned. The ongoing Phase 1/2 trial will enroll six patients with advanced Parkinson's disease. The first four patients have been treated safely and two additional patients will be treated over the next two months. This Phase 1 open label portion of the trial is being conducted by Drs. Mark Stacy and Dennis Turner at Duke University School of Medicine, Drs. Michele Tagliati and Ron Alterman at Mount Sinai Medical Center, New York, and Drs. Stewart Factor and Nicholas Boulis at Emory University Hospital. Ceregene expects to initiate the next phase of this trial (a sham-surgery-controlled, double-blind Phase 2 portion) beginning during the third quarter of this year. The Phase 2 portion will include 10 leading US neurological medical centers.
The first Phase 2 clinical trial of CERE-120 initially reported in November 2008, did not meet its pre-designated primary endpoint (UPDRS or Unified Parkinson's Disease Rating Scale- Motor Off, at 12 months) although modest benefit was seen in improving motor performance in Parkinson's disease patients, based on several secondary endpoints. Even greater improvement was seen following an analysis of all patients who were assessed under blinded conditions at 15 to 18 months post-treatment, including on the primary measure (UPDRS motor offs disease gained from two CERE-120-treated patients who died of unrelated causes, combined with the clinical results, led to important changes in CERE-120 dosing that may significantly enhance its bioactivity and response to treatment. This revised dosing paradigm now targets both the terminals (or nerve endings) of the degenerating neurons in a site in the brain called the putamen, as well as the cell bodies of these neurons, located in another region called the substantia nigra.
"We are pleased to report on progress being made in the development of CERE-120," stated Jeffrey M. Ostrove, Ph.D., president and chief executive officer of Ceregene. "We continue to strongly believe that CERE-120 has the potential to improve the symptoms of Parkinson's disease while also delaying further disease progression, and may therefore represent a significant advancement in the treatment of patients for whom existing treatments inevitably fail as their disease progresses."
"We are optimistic that the improved dosing method used in the current trial will assure that adequate neurturin protein is expressed throughout the degenerating nigrostriatal system and significantly enhance the biological effects and clinical benefit of CERE-120," stated Raymond T. Bartus, Ph.D., Ceregene's executive vice president and chief scientific officer.
"We are pleased that we have observed no safety issues with the revised CERE-120 dosing procedure, to date, and that our investigators have been able to implement the dosing protocol with no apparent complications or difficulty," added Joao Siffert, M.D., vice president and chief medical officer at Ceregene. "We look forward to completing the Phase 1 safety portion and proceeding to the Phase 2 effectiveness portion of developing CERE-120."
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