The world's largest trial in Parkinson's disease got under way, testing a creatine-based neuroprotective agent from Avicena Group Inc.

The Phase III trial in more than 1,720 patients will evaluate the ability of PD-02 to slow progression in Parkinson's disease. The double-blind, placebo-controlled study, expected to take up to five years, is being run and funded by the National Institute of Neurological Disorders and Stroke of the National Institutes of Health.

The study will include patients whose disease was diagnosed no more than five years earlier and who already are on dopaminergic therapy, said Avicena CEO Belinda Tsao-Nivaggioli. Disease status will be measured by results on the Unified Parkinson's Disease Rating Scale.

PD-02 is an ultra-pure form of creatine designed to boost production of ATP, or adenosine triphosphate. The product, from Avicena's cellular energy modulation platform, targets the creatine kinase enzyme for increasing production or delivery of cellular energy. The company also is testing creatine formulations in other neurodegenerative diseases.

"We are very encouraged by the collaboration with the NIH, and very happy they are funding the Phase III trial," Tsao-Nivaggioli told BioWorld Today. "It is also nice to see such a good network of neurologists working on the trial," which will include more than 50 sites in the U.S. and Canada.

She said interim data are expected to be released along the way, but the timing is dependent on the rate of enrollment. The study design in earlier-stage patients was approved in April 2006 by the FDA, which first wanted the company to conduct a dose-escalating chronic toxicology trial. That study was completed in February.

Avicena is providing drugs for the Phase III trial, and will have rights to the study results, and retains complete ownership of the product, Tsao-Nivaggioli said.

Avicena was founded in the late 1980s as Amira Inc. and was acquired by Repligen Corp. in 1991. Amira's initial focus was on inhibition of the creatine kinase enzyme, or taking energy away from cells, a process that might have cell death-related oncology applications.

Avicena, spun back out of Repligen in the late 1990s, realized it might be easier to use the technology to boost cellular energy for neurodegenerative diseases, rather than inhibit it, since energy deficit was one of the common underlying themes in such diseases.

A higher-dose formulation of creatine, HD-02, is undergoing toxicology studies in preparation for a Phase III trial in Huntington's disease, which Tsao-Nivaggioli said is expected to begin late this year or early in 2008.

The company also has cellular energy regulation programs in amyotrophic lateral sclerosis. Testing of ALS-08 recently began in a Phase II trial evaluating its efficacy, safety and tolerability in separate combinations with minocycline and celecoxib. The combination with the better result will be taken into a more traditional Phase II trial, she said. A Phase III trial of another ALS compound, ALS-02, that was completed in May 2006 showed improved mortality outcomes but did not meet primary or secondary endpoints.

Tsao-Nivaggioli said Avicena is starting a dose-escalation study of ALS-02 before moving into studies to evaluate survival.

Avicena separately has a line of skin care products, a business to supply dermaceutical ingredient blends, and a creatine-based nutritional supplement program, Neotine, designed to promote neuronal cell health.

Avicena reported less than $100,000 in cash and equivalents as of Sept. 30, with nine-month revenues of about $313,000 and a nine-month loss from operations of $3.8 million. Since then it has raised at least $2.5 million in a private financing. The company is in the process of seeking additional funds, Tsao-Nivaggioli said.

Avicena had 51 million shares outstanding as of Sept. 20. Its stock (OTC BB:AVGO) closed at $5.85 Thursday, unchanged.