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ALS Association of Western Pennsylvania to Honor Knopp Biosciences with Anne Lewis Humanitarian Award

March 31, 2011

--Promising New ALS Drug Treatment Enters Phase III of Development--

PITTSBURGH, PA (March 31, 2011) - The ALS Association of Western Pennsylvania announced today that Knopp Biosciences will be honored with the Anne Lewis Humanitarian Award for its ground-breaking development of a potential treatment for people with familial or sporadic ALS. ALS, also known as Lou Gehrig's disease, is a rapidly progressing disease with limited treatment options.

In related news, Knopp and its partner, Biogen Idec, announced the companies will begin Phase III testing stage of the drug by administering the first dose to the first patient enrolled in the trial today. The drug KNS-760704 (dexpramipexole) has shown neuroprotective properties in multiple in vitro and in vivo studies and may work by increasing the efficiency of mitochondria in the motor neurons, which undergo significant stress in ALS patients. In a Phase II study, dexpramipexole showed a trend toward dose-related slowing of functional decline and a trend toward improved survival at the highest dose. Dexpramipexole has been granted Fast Track status by the FDA, which may result in an expedited review, and has received orphan drug designation for the treatment of ALS from both the FDA and the European Medicines Agency.

"The identification of this drug represents the most promising news the ALS community has had in decades," said Michael Bernarding, executive director of the ALS Association Western Pennsylvania Chapter. "It is both exciting and humbling to be able to recognize Knopp, a South Side-based company, for this pharmaceutical development that in the not-too-distant future could have the potential to improve the lives of ALS patients around the world."

The Anne Lewis Humanitarian Award will be presented to Knopp at a ceremony on Thursday, June 9, at 6 p.m. at the Fairmont Hotel (Grand Ballroom) on Market Street in downtown Pittsburgh.

Now in its second year, the Anne Lewis Humanitarian Award is given to individuals or organizations that by virtue of their exemplary work have a lasting impact on the ALS community.

"We are honored to receive this recognition from the ALS Association of Western Pennsylvania on behalf of all of the people who have contributed to the development of this drug, including scientists, clinical researchers, and, most importantly, the patients who have participated in our Phase II trials," said Michael Bozik, M.D., President and CEO of Knopp. "This recognition is especially significant to us because it calls attention to the important work underway by people in laboratories and clinical centers around the world who are dedicated to combating life-threatening diseases."

During Phase III - estimated to span 12 months from the completion of enrollment-approximately 804 patients will be randomly assigned to receive the drug or a placebo control, which is essential for measuring the safety and efficacy of the compound in the intended population. Phase III testing will occur around the world, including in the U.S., Canada, Australia, and several European countries. Phase III testing of dexpramipexole will be conducted in Pittsburgh under the auspices of David Lacomis, M.D., through University of Pittsburgh School of Medicine Center for ALS Research; enrollment is expected to occur in April 2011.

About The ALS Association

The ALS Association is the only national not-for-profit organization dedicated solely to the fight against ALS. The Western Pennsylvania Chapter relies on the support of its volunteers and donors to provide a multitude of free services within 31 counties of Western Pennsylvania to persons living with ALS (PALS), caregivers and families.

Pam Golden
office 412-281-0995
mobile 412-302-2354

Media Contact:
Thomas Petzinger Jr.