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Archemix Announces Initiation of Phase 2 Trial
for Aptamer Product Candidate ARC1779 in Patients with TTP, a
Rare, Life-Threatening Blood Disorder
CAMBRIDGE, Mass.
March 17, 2008
Archemix Corp., a
biotechnology company focused on discovering, developing and commercializing
aptamer
therapeutics for rare hematological diseases, today announced
that it started a Phase 2 clinical trial of ARC1779, which is designed
to evaluate the safety and efficacy of ARC1779 as a first-in-class
anti-platelet agent in patients suffering from a rare, life-threatening
blood disorder known as thrombotic thrombocytopenic purpura, or
TTP. There is no drug approved for the treatment of TTP and acute
episodes of TTP are associated with a significant mortality rate,
estimated to be as high as 20 percent.
"The initiation of this Phase 2 trial represents an important
strategic milestone for Archemix because it demonstrates the continued
advancement of our product portfolio of novel aptamer therapeutics
for rare hematological diseases," said Errol DeSouza, Ph.D,
President and Chief Executive Officer, Archemix. "Our emerging
pipeline of aptamers for rare hematological conditions includes
preclinical programs in sickle cell disease and hemophilia, as well
as our clinical programs in TTP and other related platelet-mediated
disorders. Focusing on rare diseases represent a strategic opportunity
because there are often few or no approved therapies, a high unmet
medical need, and a rapid regulatory path to commercialization,
all in addition to requiring less commercialization infrastructure."
TTP
is caused by failure of regulation of the activity of the protein
known as von Willebrand Factor (vWF), which under normal conditions
plays a key role in protecting the body from bleeding. However,
if vWF is not properly regulated, it can lead to the formation
of blood clots. These blood clots consume platelets causing a severe
drop in a patient's normal platelet level. Patients suffering
an
acute episode of TTP experience widespread clotting and the resulting
restriction of blood flow to critical organs such as the brain,
kidneys and heart, which may cause strokes, seizures, kidney failure
or heart attack. ARC1779 is an novel aptamer therapeutic designed
to bind to and inhibit vWF, thus potentially increasing or restoring
platelet counts to normal levels and reducing or eliminating the
formation of blood clots that cause the morbidity and mortality
associated with acute episodes of TTP.
This Phase 2 trial of ARC1779
in TTP is being conducted at the Medical University of Vienna,
Austria and will assess the safety,
pharmacokinetics and pharmacodynamic effects of ARC1779 in approximately
28 patients with vWF-mediated platelet function disorders, including
acute TTP.
"This Phase 2 trial is a unique opportunity to characterize
the pharmacodynamic effects of ARC1779 with respect to its fundamental
mechanism of action in patients with a spectrum of vWF-related platelet
function disorders," commented Dr. Bernd Jilma of the University
of Vienna. "This trial will also assess the safety of ARC1779
in critically ill patients with acute TTP, and it will teach us
how to optimize the regimen for administration of ARC1779 in such
patients who are undergoing plasma exchange therapy, the current
mainstay of treatment for TTP."
About TTP
Thrombotic thrombocytopenic purpura,
or TTP, is a rare blood disorder that arises from uncontrolled activation
of von Willebrand Factor
(vWF), a protein which under normal conditions plays a key role
in maintaining the normal balance between bleeding and clotting.
In patients suffering from TTP, vWF binds excessively to platelets,
causing excessive blood clots. These clots form throughout the circulation
and can lead to serious medical consequences such as strokes, seizures,
kidney failure and heart attack. Each year in the United States,
between four and 11 new cases of TTP per million of the total population
are diagnosed. There is no approved drug treatment for patients
with TTP.
About ARC1779
ARC1779, a pegylated aptamer consisting
of 40 nucleotides, is Archemix' lead product candidate for the treatment
of TTP. ARC1779 is designed
to inhibit the function of a protein called von Willebrand Factor,
or vWF, which is, when activated, responsible for the adhesion,
activation and aggregation of platelets.
In a Phase 1 trial conducted
in 47 healthy volunteers, intravenous administration of ARC1779
demonstrated dose- and concentration-dependent
inhibition of plasma vWF activity and platelet function. Furthermore,
no serious adverse events were reported in the trial and no subject
was withdrawn from the trial due to an adverse event. The key
results of this trial were presented at the 2007 annual meeting
of the American
Heart Association, and were published in full in the journal Circulation
in December, 2007.
About Aptamers
Aptamers are synthetically-derived oligonucleotides, or short
nucleic acid sequences, that bind to protein targets with high affinity
and specificity and can be designed to have a specified duration
of action. Aptamers represent an emerging class of potential therapeutic
agents that Archemix believes may have broad application to treat
a variety of human diseases.
About Archemix
Archemix is a biotechnology company
focused on discovering, developing and commercializing aptamer therapeutics.
Using Archemix's proprietary
processes for discovering aptamers, which are protected by its broad
patent portfolio, Archemix is developing aptamer product candidates
for rare hematological diseases. In addition, Archemix has licensed
its intellectual property to third parties to develop their own
aptamer product candidates. Currently, Archemix and its licensees
are evaluating five different aptamer product candidates in human
clinical trials; three in Phase 2 and two in Phase 1. To date, Archemix
has licensed its intellectual property to discover and develop aptamer
product candidates to nine biotechnology and pharmaceutical companies,
including Pfizer, Merck Serono, Takeda Pharmaceuticals and Elan
Pharma.
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