January 25, 2008

FDA Puts Hold
On Arimoclomol Trial
In ALS

Further clinical testing of the drug arimoclomol, an experimental compound developed by CytRx Corp. of Los Angeles to treat amyotrophic lateral sclerosis (ALS), has been placed on hold by the Food and Drug Administration (FDA).

In a Jan. 22 press release and Webcast, CytRx announced that the FDA wanted to see more toxicity data from previously completed animal studies before allowing a phase 2b trial to test arimoclomol’s safety and efficacy in 390 people with ALS to proceed.

In the Webcast of a phone conference open to the public, CytRx CEO Steven Kriegsman said the company was informed of the FDA’s decision in a recent telephone conversation and that it expects written clarification “in the coming weeks.” Meanwhile, he said, CytRx has begun preparations to respond to the FDA.

Arimoclomol is designed to stimulate a normal protein repair pathway through the activation of so-called molecular chaperones. A previous trial has shown it to be safe and well tolerated at 600 milligrams three times a day for seven days. The phase 2b trial plans to compare 400 milligrams of arimoclomol three times a day with a placebo, after an initial lead-in phase to assess the safety of this dose in 24 patients.

Kriegsman said the company intends “to keep the public informed of future developments” and that it is committed to developing an effective treatment for ALS.

So far, only one trial participant has received arimoclomol in the phase 2b trial. The drug will be discontinued until the FDA releases the hold on the study.