By Frances Bishopp

CoCensys Inc., which develops drugs for epilepsy, migraine, stroke and other neurodegenerative disorders, reported that Phase II clinical trial results of its lead product, ganaxolene, demonstrated reduced epileptic spasms in children with uncontrolled epilepsy.

Ganaxolene is a synthetic version of naturally occurring neuroactive steroids, called epalons, that bind to specific receptors in the brain. The GABAA receptor complex is the major inhibitory, or calming, complex in the brain.

CoCensys scientists were among the first to discover that epalons help modulate the activity of the GABAA receptor complex. CoCensys' compounds bind to a different receptor site on the GABAA receptor complex than do existing therapies for epilepsy, anxiety and insomnia.

The open label, Phase II trial enrolled 20 patients, ages six months to seven years, who had persistent seizures despite treatment with currently available antiepileptic drugs, Christi Foster, director of communications at CoCensys, told BioWorld Today.

Sixteen of 20 patients completed the three-month study. Of those, 15 had a primary diagnosis of infantile spasms, a seizure type for which prognosis is poor.

After treatment with ganaxolene, five of the 15 infantile spasm patients showed a clinically relevant reduction in spasm frequency of greater than 50 percent and five others showed a 25 to 50 percent decrease during the two-month maintenance period when ganaxolene was added to their treatment regimen.

Ganaxolene was well tolerated with one side effect of sedation, which, Foster said, is to be expected and can be controlled through dosage regulation.

"This was a pilot study to show U.S. investigators the drug was safe and to prove the concept," Foster said. The next study will be a Phase II double-blind, placebo-controlled study, she said, which will be conducted in the U.S.

The study results mirror findings in a similar study conducted in France last year, Foster said.

Ganaxolene has orphan drug status for infantile spasms, or West syndrome. CoCensys, of Irvine, Calif., is continuing the Phase II study in France for dose-ranging purposes and is also conducting double-blind, placebo-controlled U.S. Phase II trials with ganaxolene in adult epilepsy patients and in migraine patients.

CoCensys' cash on hand, as of June 30, 1997, is $17.2 million. CoCensys' stock (NASDAQ:COCN) closed Tuesday at $3.375, up $0.250. *