March 5 /PRNewswire/ -- Repligen Corporation (Nasdaq: RGEN - news) announced today plans for a Phase 2 trial of uridine therapy in a subset of mitochondrial disease patients based on Phase 1 data obtained by investigators at the University of California, San Diego (UCSD). The Phase 1 clinical trial results were presented today at ``Mitochondria 2001'', a joint meeting of the Mitochondrial Medicine Society and the Mitochondrial Research Society held in San Diego.
The Phase 1 results indicate that therapy with uridine, or a prodrug of uridine (triacetyl uridine, ``TAU'') was well tolerated in children with severe to moderate disease. In addition, several patients were observed to have improvements in one or more of their major symptoms, including reduction in the number of neurologic setbacks with infection, reduced frequency of seizures and improvements in growth, development, coordination or strength. Data were also presented on four patients with a form of kidney disease called renal tubular acidosis, a condition which can lead to severe acidosis (acidification of the blood), susceptibility to infections, malnutrition and failure to thrive. All four patients showed a rapid improvement or correction of kidney function. Changes observed included improved control of blood pH (neutrality) and reduced loss of protein, glucose and essential electrolytes into the urine. Based on the safety and efficacy profile observed in the Phase 1 study, six patients from the Phase 1 trial have continued to receive TAU on a daily basis for one to three years.
The Phase 2 trial will be a double-blind, placebo-controlled, randomized clinical trial evaluating the potential of TAU to correct kidney disease in patients with mitochondrial disease and renal tubular acidosis. Secondary endpoints will include, as appropriate, seizure reduction, growth, development, neuromuscular function and behavior. The lead site will be the UCSD Metabolic and Mitochondrial Research Center and will include several other sites in North America. Pending discussions with the FDA, the trial is designed to recruit 40-60 patients who will receive TAU or a placebo for approximately 6 months.
``We are pleased to announce plans for the Phase 2 clinical trial,'' stated Walter C. Herlihy, President and CEO of Repligen. ``Through our clinical collaborators we will seek to prospectively identify patients who may participate in the trial to insure rapid accrual of patients, pending approval of the clinical study by FDA.''... |
