Associated Press
Update 1: CytRx Seeks to Speed ALS Drug Approval
05.23.2005, 02:32 PM

CytRx Corp. said Monday that it hopes to seek approval for its experimental drug to treat amyotrophic lateral sclerosis, the nervous system disorder commonly called Lou Gehrig's disease, on data from two clinical trials, rather than the three usually required.

The company's shares rose 6 cents, or 7.3 percent, to 88 cents in afternoon trading on the Nasdaq.

Earlier this month, CytRx's arimoclomol therapy received "orphan drug" status from the Food and Drug Administration. The label often helps speed a drug's approval and provides other incentives such as trial funding and tax breaks.

CytRx expects to begin its Phase II study in June and for the trial to last about 18 months.

If the trial shows that arimoclomol is effective against ALS, President and Chief Executive Steven A. Kriegsman said the company plans to ask the FDA to approve the drug without the usual Phase III study.

"This is a disease with no cure and no effective treatment," Kriegsman said, explaining the decision, in an interview. He added that the upcoming study has been designed to be very similar to a Phase III trial, but declined to elaborate on the details of the study pending FDA authorization of the plan.

If the FDA approves the drug on the more limited trial data, the company would then skip straight to a Phase IV, or post-approval, study. CytRX said Monday that it has already submitted a preliminary "investigational new drug" application to the FDA, a required step in the clinical trial process. Kriegsman added that the company also plans to apply for a "fast-track" designation which could speed up FDA deliberation on the drug.

CytRx said arimoclomol appears to work by amplifying a natural "stress response" protein repair mechanism to combat the progressive degeneration of the brain and spinal column nerve cells associated with ALS.

About 20,000 Americans have ALS and about 5,000 are diagnosed each year in the United States, according to the National Institutes of Health. The disease is characterized by the progressive loss of motor nerves in the spinal cord and brain, leading to muscle weakness and eventually, paralysis.