Arimoclomol

CytRx Corporation, expande o desenvolvimento do Arimoclomol para a fase II

CytRx Corporation, a biopharmaceutical company engaged in the development and commercialization of human therapeutics, today announced that it has submitted a protocol to the FDA reflecting its plans to treat patients in an open-label extension of its ongoing Phase II clinical trial with its orally administered, small molecule product candidate arimoclomol for the treatment for amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease).
CytRx expects to provide all patients who complete the ongoing Phase II trial with the opportunity for treatment with arimoclomol at the highest dose level three times daily for an additional six months. In the currently-underway 80-patient, 10-center, double-blind Phase II study, patients are receiving either placebo (a capsule without drug), or one of three dose levels of arimoclomol capsules three times daily for a period of 12 weeks. The open-label extension trial is designed to provide additional safety and tolerability data in combination with the current Phase II trial.
"It is not surprising that patients enrolled in our trial have expressed high interest in an open-label extension option with arimoclomol for the treatment of ALS. Currently, there are no effective therapeutic treatments
available for those suffering from this devastating neurodegenerative disease that carries an average life expectancy of 18 months following diagnosis," said Steven A. Kriegsman, CytRx's President and CEO. "We are exceptionally pleased to offer this opportunity to patients who completed our trial, and particularly to those who received placebo or arimoclomol at lower dose levels." CytRx expects to report final data from its ongoing Phase II trial with arimoclomol for ALS in the third quarter of this year, followed by initiation of a pivotal Phase IIb clinical trial subject to FDA review and acceptance.
The Company believes that successfully demonstrating safety and efficacy in this latter Phase II clinical trial could be sufficient to support product registration with the FDA.
In 2005 CytRx announced that arimoclomol was granted orphan drug status and Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of ALS. Benefits of orphan designation include opportunities for grant funding toward clinical trial costs, tax advantages, FDA user-fee benefits, seven years of U.S. market exclusivity should the FDA grant marketing approval for the drug and an added mechanism for more frequent communication with the FDA. 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.