LOS ANGELES, Jan. 23
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.
