CytRx granted "Fast Track" designation for ALS drug

Sep 26 2005

CytRx Corporation today announced that the U.S. Food and Drug Administration (FDA) has granted "Fast Track" designation for the company's leading drug candidate arimoclomol for the treatment of amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease).

Last week, CytRx announced the initiation of a Phase II clinical trial with orally-administered arimoclomol for ALS, and patient identification for this trial is underway at several clinical trial sites. Arimoclomol was granted orphan drug status designation by the FDA for the treatment of ALS in May 2005.

"We are delighted that the FDA has permitted us to begin this important clinical trial, and the fast track designation constitutes yet another significant milestone in our effort to help those suffering from this deadly neurodegenerative disease for which there is no effective treatment," said Steven A. Kriegsman, President and CEO of CytRx.

Under the FDA Modernization Act of 1997, the Fast Track process is designed to facilitate the development and expedite the review of a new drug that demonstrates the potential to address a significant unmet medical need for the treatment of a serious or a life-threatening condition. The benefits of Fast Track include scheduled meetings seeking FDA input into development plans, the option of submitting a new drug application (NDA) in sections rather than submitting all components simultaneously, and the option of requesting evaluation of studies using surrogate endpoints.

ALS is a progressive degeneration of the brain and spinal column nerve cells that control the muscles that allow movement. According to the ALS Survival Guide, 50% of ALS patients die within 18 months of diagnosis and 80% die within five years. There are more than 120,000 people living with ALS worldwide.

Arimoclomol is believed to function by a mechanism that stimulates 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 with its drug candidates could demonstrate therapeutic efficacy over ALS as well as a broad range of additional diseases.