CAMBRIDGE, Mass., Nov. 10
/PRNewswire/ -- Vertex
Pharmaceuticals Incorporated
(NASDAQ:VRTX) announced today that it
has begun the
first-ever clinical trial investigating
the potential of a neurophilin
compound to slow or reverse neural
dysfunction in humans. The
Company has started a Phase II clinical
trial with timcodar
dimesylate (VX-853), an orally delivered
neurophilin ligand, in
patients with diabetic neuropathy.
Approximately 70 patients will
be enrolled in the trial, which will be
conducted in 8 centers in the
United States.
Neurophilin ligands are small molecules
that have the potential to
promote neurite outgrowth and accelerate
recovery following
nerve injury. They may play a future
role in the treatment of a
variety of acute and chronic
neurological conditions. Neurophilin
compounds designed by Vertex are orally
deliverable, and have
been shown to accelerate functional
recovery and promote nerve
growth in several different animal
models of central and peripheral
nervous system injury.
"Peripheral neuropathies represent an
important starting point for
clinical development, because of both
the lack of existing
therapies for these patients and the
possibility of affirming the
broader potential of neurophilin ligands
in a variety of neurological
indications," said Dr. Vicki Sato,
Senior Vice President of
Research and Development and Chief
Scientific Officer of Vertex.
"The safety data we have collected to
date gives us confidence
that we can move timcodar forward into
Phase II testing and
begin to understand its potential for
producing clinical efficacy.
Results of several laboratory and
preclinical studies, which we
expect to present publicly in the coming
months, have
demonstrated timcodar to have potent
activity in promoting nerve
growth and restoring nerve function."
Clinical safety and pharmacokinetic data
supporting timcodar's
potential as a therapeutic compound are
available from a
single-dose study of four different
doses in healthy volunteers,
which was completed earlier in 1998. In
vivo laboratory
experiments indicate that timcodar can
prevent neural dysfunction
in a model of diabetic polyneuropathy
and in vitro results have
shown timcodar's ability to promote
neurite outgrowth. Results
presented at the Society for
Neuroscience 27th Annual Meeting in
October 1997 showed that an oral
neurophilin compound
designed by Vertex that is related to
timcodar could enhance
functional recovery through neuronal
regeneration in animal
models of peripheral nerve injury,
Parkinson's disease, and spinal
cord injury.
The double-blind, placebo controlled
Phase II clinical trial
announced today will enroll
approximately 70 patients with
diabetic neuropathy in 8 clinical
centers in the United States,
under an investigational new drug
application (IND) that has been
reviewed by the U.S. Food and Drug
Administration (FDA).
Primary objectives will be to evaluate
the safety and tolerability of
six different dose regimens of timcodar
administered orally over a
28-day period. Nerve function will also
be monitored.
"Neuronal regeneration represents a
major market opportunity,
and we are positioned at the forefront
of clinical research in this
area," said Dr. Joshua Boger, Chairman,
President, and CEO of
Vertex. "Vertex's information- intensive
approach to drug
discovery and development has enabled us
to make rapid
progress with timcodar, and allows us to
continue building our
innovative clinical pipeline."
In August 1998, Schering AG, Germany and
Vertex formed a
collaboration to research, develop and
commercialize novel, orally
active neurophilin compounds to promote
nerve growth and
repair. Under terms of the agreement,
Schering AG, Germany
agreed to provide $28 million in upfront
and research support
payments over five years. An additional
$60 million in milestone
payments is possible through the
development of specific
compounds arising from the
collaboration. Under the agreement,
Schering AG has an option to co-develop
timcodar with Vertex.
Neuropathy is a frequent long-term
complication of diabetes that
can involve persistent painful sensation
or loss of sensation, most
commonly in the hands, feet and legs.
Diabetic neuropathy affects
an estimated 1.3 million patients in the
United States, and if
severe, can lead to amputation of an
affected limb. According to
the American Diabetes Association,
approximately 56,000
amputations in the United States each
year are due to diabetic
neuropathy.
Timcodar dimesylate began development at
Vertex under the
name VX-853, as an oral agent for the
reversal of cancer
multidrug resistance (MDR). A Phase I/II
trial examining high
doses of VX-853 in patients with
advanced cancer is ongoing.
The dose range under investigation for
neurological diseases is
substantially lower than that believed
to be optimal for MDR.
Vertex holds an issued United States
patent directed to
compound classes that include timcodar.
Vertex also holds a
United States patent application under a
Notice of Allowance
claiming the use of these compound
classes for stimulating nerve
growth and for the treatment of various
neurological diseases.
Vertex Pharmaceuticals Incorporated is
engaged in the discovery,
development and commercialization of
novel, small molecule
pharmaceuticals for the treatment of
diseases for which there are
currently limited or no effective
treatments. The Company is a
leader in the use of structure-based
drug design, an approach to
drug discovery that integrates advanced
biology, biophysics and
chemistry. The Company is concentrating
on the discovery and
development of drugs for the treatment
of viral diseases, multidrug
resistance in cancer, autoimmune and
inflammatory diseases, and
neurodegenerative diseases.
There can be no assurance that clinical
trials will continue, that
initial results will be predictive of
any future results, that drugs
under development by Vertex or its
partners will receive
marketing approval from the U.S. Food
and Drug Administration
or other regulatory authorities, or that
drugs, if any, which receive
such approval will be marketed
successfully. There can be no
assurance that Schering will exercise
its option to participate in the
development and commercialization of
timcodar dimesylate.
Investors are also directed to consider
other risks and
uncertainties discussed in Vertex
documents filed with the
Securities and Exchange Commission.