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Prize4Life and Knopp Neurosciences Announce Donation of Empower Phase 3 Data to Pro-Act Database

June 13, 2013

CAMBRIDGE, MA, AND PITTSBURGH, PA, 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 database.

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- 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 www.ALSDatabase.org.

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 www.prize4life.org.

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 http://www.ncrinstitute.org/.

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.

Media Contact:
Thomas Petzinger Jr.
412-488-1776
tom@knoppbio.com