CYTRX TO LAUNCH MAXIMUM-DOSE STUDY OF ALS DRUG

CytRx has announced its plans for completing the next step towards initiating its Phase IIb clinical trial for the treatment of amyotrophic lateral sclerosis (ALS), planned to begin in the third quarter of this year. As an important step toward this goal, CytRx plans to determine the maximum safe and well-tolerated dose of orally administered arimoclomol. The company then intends to proceed with this maximum dose in the Phase IIb efficacy trial for ALS, subject to FDA approval.

"The favorable safety and tolerability profile observed for arimoclomol in our recent Phase IIa ALS trial, combined with previous and recent animal toxicology results, suggests that we may be able to safely increase the dose without causing significant side effects," CytRx Chief Scientific Officer Jack Barber said.

This Phase IIa European single-site, double-blind, placebo-controlled multiple-ascending-dose study is designed to test the safety, tolerability and pharmacokinetics of increasing dosages of arimoclomol in healthy volunteers over a seven-day period. The plan includes enrolling a total of approximately 40 healthy volunteers, with 10 subjects in each of four groups. Within each group, seven subjects will receive arimoclomol and three will receive placebo.

The first group will be dosed with 100 mg three times daily, the highest dose in the trial. Safety results from this group will provide a baseline that will be compared with subsequent higher dose groups. Dosing of the remaining three groups will proceed sequentially, with progressively higher doses to be administered to the next group only if the dosage in the prior lower dose group is determined to be safe and well-tolerated. If significant side effects occur at any dose level, the study will end and the previous dose level will be considered to be the maximum tolerated dose. If toxicity is not observed at lower doses, this cautious increase in dose is planned to reach a maximum dose of 600 mg three times a day. After the maximum tolerated dose is determined, CytRx plans to evaluate the longer-term safety of this dose in a subsequent study.

Arimoclomol is one of CytRx's three orally administered, small-molecule compounds. The drug is believed to function by stimulating a normal cellular protein repair pathway through the activation of "molecular chaperones." Since damaged proteins called aggregates are thought to play a role in many diseases, CytRx believes that activation of molecular chaperones could have therapeutic efficacy for a broad range of diseases.