Prize4Life and Knopp Neurosciences Announce Donation of EMPOWER Phase 3 Data to PRO-ACT Database

  Prize4Life and Knopp Neurosciences Announce Donation of EMPOWER Phase 3 Data
  to PRO-ACT Database

Business Wire

CAMBRIDGE, Mass. & PITTSBURGH -- June 13, 2013

Prize4Life and Knopp Neurosciences Inc. today announced an agreement under
which Knopp will donate data from one of the largest clinical trials ever
conducted in amyotrophic lateral sclerosis (ALS) to the Prize4Life PRO-ACT

PRO-ACT was created as a freely accessible resource to the global research
community by Prize4Life, a nonprofit organization whose mission is to
accelerate the discovery of treatments and a cure for ALS, also known as Lou
Gehrig's disease. Under the agreement with Knopp, de-identified clinical data
from 468 placebo subjects in the Phase 3 EMPOWER study of dexpramipexole in
ALS will be provided to the PRO-ACT database. Knopp also plans to contribute
the treatment-arm data from EMPOWER to PRO-ACT following the completion of
future dexpramipexole studies in ALS.

“This agreement is a first step in further expanding the PRO-ACT database,
which is already leading to new insights in the search for a cure for ALS,”
said Melanie Leitner, Ph.D., Prize4Life's Chief Scientific Officer. “We are
thankful for Knopp’s generous data donation and excited to provide even
greater access to ALS clinical trials data to the researchers and clinicians
making progress in the fight against ALS every day.”

“Knopp is very pleased to contribute these data to PRO-ACT, which is rapidly
emerging as a key resource in the global effort to unravel the pathophysiology
of ALS and to pursue meaningful treatments,” said Michael Bozik, M.D.,
president and CEO of Knopp. “We applaud Prize4Life’s leadership in promoting
the collaboration of researchers, clinicians and statisticians from industry
and academia.”

The PRO-ACT database contains more than 8,600 fully de-identified unique
clinical patient records - both treatment and placebo data from 17 late-stage
industry and academic trials - and over 8 million data points, including 1.7
million records of lab test results from ALS patients.

Prize4Life and the Neurological Clinical Research Institute (NCRI) at
Massachusetts General Hospital created the PRO-ACT database with funding from
the ALS Therapy Alliance and in partnership with the Northeast ALS Consortium
(NEALS). Academic institutions and pharmaceutical companies - including
Sanofi, Novartis, Teva Pharmaceutical Industries, and Regeneron
Pharmaceuticals - contributed data to this initiative, and other potential
data donations are currently being pursued.

Utilizing the datasets should further encourage collaboration between academic
and industry researchers, statisticians, and clinicians throughout the world.
The PRO-ACT database is freely accessible to the global research community for
download and analysis at

About Prize4Life
Prize4Life is a 501(c)(3) nonprofit organization whose mission is to
accelerate the discovery of treatments and a cure for ALS by using powerful
incentives to attract new ideas, foster new research, and drive innovation.
Prize4Life believes that solutions to some of the biggest challenges in ALS
research will require out-of-the-box thinking, and that some of the most
critical discoveries may come from unlikely places. Founded in 2006 by Avi
Kremer, who was diagnosed with ALS at age 29, Prize4Life offers financial
prizes and research programs aimed at yielding real results for ALS patients.
For more information, visit

About Neurological Clinical Research Institute
The Neurological Clinical Research Institute (NCRI) at Massachusetts General
Hospital accelerates translational research in neurological disorders through
initiating and testing novel therapies. The NCRI has an extensive history in
leading clinical research to find new treatments for neurological diseases
including ALS, myasthenia gravis, diabetic neuropathy, stroke, multiple
sclerosis, Parkinson's disease, and Huntington's disease. For more
information, visit

About Knopp Neurosciences
Knopp Neurosciences, based in Pittsburgh, PA, is an affiliate of Knopp
Biosciences, a drug discovery and development company focused on delivering
breakthrough treatments for unmet needs through innovation, experience, and
partnership. Knopp's lead product candidate is dexpramipexole, an orally
bioavailable small molecule in development for the treatment of ALS and
eosinophil-associated disorders. Knopp's discovery platform is directed to
next generation mitochondrial modulators for the treatment of
neurodegenerative and immunological disorders and novel ion channel mediators
for the treatment of epilepsy and neuropathic pain.


Version 2.0 Communications for Prize4Life
Meghan Gardner, 617-426-2222
Knopp Neurosciences
Tom Petzinger, 412-488-1776
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