Market Overview

Knopp Biosciences Announces Year 3 Renewal of NIH Blueprint Grant Award for Advancing KCNQ2 Activators in Rare Neonatal Epilepsy


Knopp Biosciences LLC today announced the renewal of its grant award for
Year 3 from the National Institutes of Health Blueprint
Neurotherapeutics Network (BPN) to advance novel treatments for epilepsy.

Based on milestone attainment, Knopp was awarded an additional $693,000
in direct support. The grant has a total potential value of $2.3 million
over four years. Beyond direct support, the NIH BPN may underwrite the
cost of key external studies at BPN-sponsored-contract research

As previously reported, Knopp is directing its Kv7 potassium channel
activator program to the discovery and development of best-in-class
small-molecule drug candidates, consistent with the goals of the NIH
BPN. The BPN, part of the NIH Blueprint for Neuroscience Research, is a
collaboration of NIH Institutes and Centers that supports research on
the nervous system with the goal of developing new neurotherapeutic

These novel molecules have been synthesized to modulate an anti-seizure
pharmaceutical target encoded by the KCNQ2 gene, which contributes
significantly to early brain development. Knopp intends to first advance
its novel KCNQ2 activators in neonatal epileptic encephalopathy, a rare
disorder caused by sporadic mutations in the KCNQ2 gene. Neonatal
epileptic encephalopathy is characterized by persistent seizures from
the beginning of life and profound developmental disability in children
for whom current anti-seizure medications are insufficient or

Knopp President and CEO Michael Bozik, M.D., a neurologist with clinical
development expertise in potassium channel modulators, serves as
Principal Investigator on the NIH BPN grant. He said, "We're very
pleased to receive this latest Blueprint award, which provides ongoing
support that will help us advance promising clinical drug candidates to
treat a rare and devastating disease without approved treatment
options." He said Knopp expects its first KCNQ2 candidate to enter Phase
1 testing in 2019.

Knopp is collaborating with key clinical investigators in the
development of its first candidate, as well as with the KCNQ2 Cure
Alliance, whose 2018 Family and Professional Summit will be held Sept.
14-16, 2018, in Anaheim, CA.


Knopp Biosciences, based in Pittsburgh, PA, USA, is a privately held
drug discovery and development company focused on delivering
breakthrough treatments for inflammatory and neurological diseases of
high unmet need. Knopp's clinical-stage small molecule, dexpramipexole,
is entering Phase 3 clinical studies in hypereosinophilic syndrome and
Phase 2 clinical studies in eosinophilic asthma. Knopp's preclinical Kv7
platform is directed to small molecule treatments for neonatal epileptic
encephalopathy, other rare epilepsies, tinnitus, and neuropathic pain.
Please visit


The Blueprint Neurotherapeutics Network provides non-dilutive support
for small molecule drug discovery and development, from hit-to-lead
chemistry through phase I clinical testing. Please visit BPN.


KCNQ2 Cure Alliance is a non-profit organization dedicated to raising
research funds for KCNQ2 epileptic encephalopathy, a rare and
catastrophic form of epilepsy beginning in the first days of life.
Please visit

Knopp's Kv7 research is supported under Award Number U44NS093160 of
the National Institute of Neurological Disorders and Stroke of the
National Institutes of Health (NIH). The content of this announcement is
solely the responsibility of Knopp and does not necessarily represent
the views of the NIH.

This press release contains "forward-looking statements," including
statements relating to planned regulatory filings and clinical
development programs. 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 any investigational drug product will be
successfully developed or manufactured or that final results of clinical
studies will be supportive of regulatory approvals required to market a
product. 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's pipeline consists of investigational drug products that have
not been approved by the U.S. Food and Drug Administration. These
investigational drug products are still undergoing clinical study to
verify their safety and effectiveness.

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