Valentis
Reports Pre-Clinical Results of Plasmid-Based Gene medicine for the Treatment of Hemophilia B and
Anemia
June
2, 2000 Burlingame, CA — Valentis, Inc.
(NASDAQ:
VLTS)
today announced that its proprietary
plasmid‑based Factor IX gene medicine
demonstrated correction of bleeding
in a large animal model of hemophilia
following a single administration of the
therapy. Valentis researchers presented
the data at the American Society for Gene
Therapy's Third Annual Meeting in Denver,
Colorado. The Factor IX gene medicine,
consisting of a DNA plasmid containing the
Factor IX gene formulated with one of
Valentis' proprietary PINCô polymers, is
a non-viral product administered by intrañmuscular
injection. Expression of the therapeutic protein
is enhanced by electroporation, the
application of a small amount of electricity
at the site of injection for a few
milliseconds. In addition, regulated expression,
after a single administration of the Company's
erythropoietin gene medicine controlled by
the Company's GeneSwitchô, was shown for
up to one year.
"Our
Factor IX gene medicine is the first product
delivered without using a virus that has
demonstrated the ability to correct an
inherited disorder in a large animal model,"
stated Ben McGraw, Valentis' President and
CEO. "These
results illustrate the potential of our
technology, without the safety concerns that
have been raised by the use of viral
delivery systems. Our non-viral technologies allow the
sustained production of a protein at
therapeutic levels using the muscle as a
bioreactor and enable multiple product
opportunities."
Valentis
researchers reported that four hemophiliac
dogs were administered the Factor IX gene
medicine using in vivo electroporation
into the muscle. The dogs tolerated the
electroporation procedure very well and did
not require administration of the factor IX
protein during the procedure. Whole blood clotting times began to
decline within 24 hours of administration.
In
small animal studies using the Company's
EPO gene medicine under the control of the
Company's proprietary GeneSwitch® technology,
Valentis researchers administered plasmids
encoding erythropoietin and the GeneSwitch®
to mice. Upon oral administration of a small
molecule inducer, a dose-dependent increase
in erythropoietin protein was detected in
the blood. Several cycles of increased
hematocrit levels were achieved throughout
the one-year study.
"For
widespread use of gene therapy it is
essential to control both the level and
duration of the therapeutic protein being
expressed," stated Alain Rolland, Vice
President of Research and Development and
Head of Valentis' Woodlands Center in
Texas. "Valentis' GeneSwitch technology
has been designed to provide this sort of
control and has successfully been shown in
animal models to enable tight regulation of
gene expression in response to oral
administration of specific inducers
for up to one year."
>Valentis,
Inc. is a leader in the field of biologics
delivery. The Company develops proprietary
technologies and applies its preclinical and
early clinical development expertise to
create novel therapeutics. The
Company's core technologies include
multiple gene delivery and gene expression
systems and PEGylation technologies designed
to improve the safety, efficacy and dosing
characteristics of genes,
proteins, peptides, peptidomimetics,
antibodies and replicating and
non-replicating viruses.
These
technologies are covered by a broad patent
portfolio that includes issued U.S. and
European claims. Valentis' commercial strategy is to
enter into corporate collaborations for
full-scale clinical development and
marketing and sales of products. Valentis
currently has corporate collaborations with
Roche Diagnostics, Eli Lilly, Glaxo Wellcome,
Boehringer Ingelheim, Heska Corporation,
Transkaryotic Therapies, Onyx
Pharmaceuticals and Bayer International, and
a manufacturing partnership (the pAlliance )
with DSM Biologics and Qiagen N.V. Additional information about Valentis
can be found at www.valentis.com.
Statements
in this press release that are not strictly
historical are "forward looking"
statements as defined in the Private
Securities Litigation Reform Act of 1995. The words "believes," "expects,"
"intends," "anticipates," variations
of such words, and similar expressions
identify forward-looking statements, but
their absence does not mean that the
statement is not forward-looking. These statements are not guarantees
of future performance and are subject to
certain risks, uncertainties and assumptions
that are difficult to predict. Factors that could affect the
Company's actual results include the need
for additional capital, the early stage of
product development, uncertainties related
to clinical trials, and uncertainties
related to patent position. There can be no assurance that
Valentis will be able to develop
commercially viable gene-based therapeutics
or PEGylated products, that any of its
programs will be partnered with a
pharmaceutical partner, that necessary
regulatory approvals will be obtained, that
any clinical trial will be successful, or
that successful preclinical results in
animal tests will translate into positive
human clinical data. The actual results may differ from
those projected in the forward-looking
statement due to risks and uncertainties
that exist in the Company's operations and
business environment. These are described more fully in the
Valentis Annual Report on Form 10-K for the
period ended June 30, 1999 and
Quarterly Reports on Form 10-Q for the
periods ended December 31, 1999 and March
31, 2000, each as filed with the Securities
and Exchange Commission.
