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Archemix' ARC1779 Granted Orphan Designation
For The Treatment Of TTP, A Life-Threatening Blood Disorder
CAMBRIDGE, Mass.
April 22, 2008
Archemix Corp., a biotechnology company focused
on discovering, developing and commercializing aptamer therapeutics
for rare hematological diseases, announced that ARC1779 has
received
orphan drug designation from the U.S. Food and Drug Administration.
ARC1779, a novel aptamer therapeutic, is a first-in-class anti-platelet
agent for the treatment of 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.
ARC1779 is currently in Phase 2 clinical
development with a clinical trial designed to assess the safety,
pharmacokinetics and pharmacodynamic
effects of ARC1779 in approximately 28 patients with vWF-mediated
platelet function disorders, including acute TTP.
"The designation of ARC1779 as an orphan drug underscores
the importance of developing effective treatments for patients with
TTP," said Errol DeSouza, President and Chief Executive Officer,
Archemix. "To date we have been encouraged by ARC1779's early-stage
clinical results, and we continue to believe it has the potential
to offer a new and effective treatment in a disease for which there
is currently no drug therapy available."
ARC1779 is designed
to bind to and inhibit a protein known as von Willebrand Factor
(vWF) that protects the body from bleeding and
blood clots and, therefore, the administration of ARC1779 potentially
increases or restores platelet counts to normal levels reducing
or eliminating the formation of blood clots that cause the morbidity
and mortality associated with acute episodes of TTP. 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.
About The Orphan Drug Act
The Orphan Drug Act of
1983 allows the FDA to grant orphan drug status to novel drugs or
biologics that treat a rare disease or
condition affecting fewer than 200,000 patients in the U.S. The
Orphan Drug Act provides the drug developer with several financial
benefits and incentives related to the orphan drug, including tax
credits for clinical research costs, ability to apply for annual
grant funding, clinical research trial design assistance, waiver
of Prescription Drug User Fee Act (PDUFA) filing fees, and a seven-year
period of U.S. marketing exclusivity if the drug is the first of
its type approved for the specified indication.
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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