Knopp Biosciences LLC today announced a collaboration agreement with the
National Institute of Allergy and Infectious Diseases (NIAID), part of
the National Institutes of Health (NIH), to conduct a clinical trial of
dexpramipexole in hypereosinophilic syndrome (HES).
Under the Cooperative Research and Development Agreement (CRADA), NIAID
will sponsor the Phase 2 proof-of-concept trial, while Knopp will
provide the dexpramipexole study drug and will collaborate on trial
design and study oversight.
The 24-week, open-label clinical trial in HES subjects receiving
corticosteroid therapy is expected to initiate enrollment in the first
quarter of this year at the NIH Clinical Center in Bethesda, MD. The
principal investigator is Amy Klion, M.D., immediate past president of
the International Eosinophil Society and chief of the Eosinophil
Pathology Unit of NIAID's Laboratory of Parasitic Diseases.
The principal objective of the trial is to evaluate the potential of
dexpramipexole to reduce long-term and acute treatment with
corticosteroids in people with HES while maintaining or reducing
eosinophil count. Data presented at the July 2013 meeting of the
International Eosinophilia Society in Oxford, England, demonstrated that
dexpramipexole safely and persistently reduced blood eosinophil counts
by approximately 70% (p < 0.0001) in people with amyotrophic lateral
sclerosis (ALS) enrolled in a large Phase 3 trial (n=943).
Evidence from multiple preclinical and ALS clinical studies showing that
dexpramipexole reduces blood eosinophils is the basis for the hypothesis
that the drug may be a potential treatment for HES, for which off-label
corticosteroid administration remains first-line treatment. HES is a
colection of rare disorders characterized by excess levels of
circulating eosinophils, a type of white blood cell, eventually leading
to tissue damage in end organs. Dr. Klion will evaluate the primary
endpoint of minimally effective corticosteroid dose reduction after
treatment with dexpramipexole.
"Knopp is very pleased to collaborate with a researcher of Dr. Klion's
experience and leadership in evaluating investigational treatments for
HES," said Michael Bozik, M.D., president and CEO of Knopp. "HES is a
very serious orphan disease with limited treatment options, and we
expect this collaboration to provide an important assessment of the
drug's potential for benefiting patients with this aggressive
In addition to assessing the potential of dexpramipexole as a
corticosteroid-sparing treatment for HES, the planned Phase 2 study will
examine drug effects on bone marrow eosinophils and myeloid precursors
prior to steroid taper; changes in tissue eosinophils; and measures of
eosinophil activation, cytokine/chemokine profile, and other immunologic
Study objectives also include evaluating the safety of 150 mg of
dexpramipexole administered twice daily in HES patients currently
treated with corticosteroids. The selection of dose and regimen is based
on the safety and tolerability of dexpramipexole in earlier clinical
studies of healthy volunteers and ALS subjects receiving 50 mg, 150 mg,
and 300 mg total daily doses and in a healthy volunteer study of 600 mg
total daily dose.
About Knopp Biosciences
Knopp Biosciences, based in Pittsburgh, PA, USA, is a drug discovery and
development company focused on delivering breakthrough treatments for
unmet medical needs through innovation, experience, and partnership.
Knopp's lead product candidate is dexpramipexole, an orally bioavailable
small molecule in research and development for the treatment of
eosinophil-associated disorders and amyotrophic lateral sclerosis (ALS).
Knopp's discovery platform is directed to next-generation mitochondrial
modulators for the treatment of neurodegenerative disorders,
leukocyte-lowering agents for immunological disorders, and ion channel
modulators for neuropathic pain and epilepsy.
This announcement contains "forward-looking statements," including
statements relating to Knopp's plans regarding regulatory filings and
clinical development programs for KNS-760704 (dexpramipexole). All
forward-looking statements are based on management's current assumptions
and expectations and involve risks, uncertainties, and other important
factors, specifically including the uncertainties inherent in clinical
trials and product development programs, the availability of funding to
support continued research and studies, the availability or potential
availability of potential alternative therapies or treatments, the
availability of patent protection for the discoveries and strategic
alliances, as well as additional factors that may cause Knopp's actual
results to differ from our expectations. There can be no assurance that
KNS-760704 will be successfully developed or manufactured or that final
results of clinical studies will be supportive of regulatory approvals
required to market the products. Knopp undertakes no obligation to
update or revise any such forward-looking statements, whether as a
result of new information, future events, or otherwise.
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