Knopp Neurosciences Inc. ("Knopp")
announced that the U.S. Food and Drug Administration's Office of Orphan
Products Development has granted orphan drug designation to KNS-760704 for
the treatment of amyotrophic lateral sclerosis (ALS).
KNS-760704 is now completing Phase 1 studies to evaluate the safety,
tolerability and pharmacokinetics of the compound in healthy human
subjects. Knopp expects to initiate Phase 2 studies in ALS patients in the
first quarter of 2008.
"Congress passed the Orphan Drug Act because it recognized that
adequate drugs for many rare diseases have not been developed," said
Michael Bozik, M.D., president and CEO of Knopp. "The designation of
KNS-760704 as an orphan drug underscores the importance of developing
effective treatments for patients with ALS."
About KNS-760704
KNS-760704 is an orally administered small molecule in clinical
development by Knopp for the treatment of amyotrophic lateral sclerosis
(ALS). The drug is an optical enantiomer of a selective, high affinity
dopamine agonist marketed in other neurological indications. Both
KNS-760704 and the marketed agonist demonstrate neuroprotective properties
independent of dopamine receptor activity, but KNS-760704 exhibits greatly
reduced dopamine receptor affinity. This makes it possible to clinically
evaluate the potential neuroprotective activity of KNS-760704 over a broad
dose range.
About ALS
Amyotrophic lateral sclerosis (ALS), often called Lou Gehrig's disease,
is a rapid, universally fatal neurodegenerative disorder characterized by
progressive muscle weakness and wasting. ALS affects adults in the prime of
life and creates a substantial burden for caregivers. U.S. prevalence is
nearly 30,000 and the incidence is 1.2 per 100,000. Only a single drug has
been approved for the treatment of ALS. Life expectancy after symptom onset
is usually three to five years.
About Knopp Neurosciences Inc.
Knopp Neurosciences is a drug discovery and development company focused
on delivering breakthrough treatments for neurological disorders through
innovation, experience, and partnership. The company's lead product
candidate is KNS-760704, an orally bioavailable small molecule in
development for the treatment of ALS. Knopp's leadership includes
experienced neuroscience drug development and discovery executives formerly
associated with major pharmaceutical companies. Knopp's financing has been
led by Saturn Capital Inc. of Boston as placement agent and Saturn Partners
II as lead funder.
This press release contains "forward-looking statements," including
statements relating to Knopp's planned regulatory filings and clinical
development programs for KNS-760704. 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 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.
Knopp Neurosciences Inc.
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