Knopp Biosciences Announces Key Executive Hires and Completion of Lab Expansion

Knopp Biosciences

Knopp Biosciences LLC today announced the recruiting of key scientific executives and the completion of a significant laboratory build-out as part of its expanded activity in pharmaceutical discovery.

In addition to doubling its R&D facilities to 20,000 square feet, Knopp has recently doubled its head count to 33 with the addition of mostly Ph.D.-level biologists and medicinal chemists. Hired to lead Knopp’s newly launched chemistry effort is Steven Boyd, Ph.D., formerly of Array BioPharma and Abbott Laboratories. Leading the expanded biological research effort is Ian Reynolds, Ph.D., formerly of Merck & Co. and the University of Pittsburgh.

“Dr. Boyd brings to Knopp veteran industry leadership and a track record of discovering new molecules successfully advanced into clinical development,” said Steven Dworetzky, Ph.D., Knopp’s Vice President of Discovery Research. “Working alongside Dr. Boyd, Ian Reynolds will lead our expanded biology team after decades of pharmacology experience, with a focus on Parkinson’s disease and the role of mitochondrial dysfunction in neurodegeneration. Their collective leadership strengthens our position to discover and advance novel modulators in our target areas of interest, namely mitochondrial function and ion channel activity.”

The expansion of Knopp’s pharmaceutical R&D effort follows an August 2010 agreement under which the company exclusively licensed the development and commercialization of dexpramipexole, a candidate for the treatment of amyotrophic lateral sclerosis (ALS), to Biogen Idec of Weston, Mass. As previously reported, dexpramipexole is now in a global Phase 3 clinical trial, called EMPOWER, with top-line data expected to read out in the fourth quarter of 2012. Knopp retains milestone and royalty interests in dexpramipexole as well as development support and certain U.S. commercialization roles.

“While dexpramipexole in ALS remains our most advanced asset, the pharmacological mechanism of the drug represents the foundation of our discovery research,” said Michael Bozik, M.D., president and CEO of Knopp. “We’ve greatly strengthened this foundation by recruiting Steve Boyd to head medicinal chemistry and Ian Reynolds to lead our biology team.”

Dr. Boyd joins Knopp as Senior Director of Chemistry after 11 years at Array BioPharma in Boulder, Colo., where he was most recently responsible for metabolic disease drug discovery programs and where he led discovery collaborations with Eli Lilly, Amgen, ICOS, Procter & Gamble, and Genentech. Previously he spent 13 years as a research investigator and group leader in chemistry at Abbott Laboratories. He completed his graduate studies in chemistry at the University of California at Los Angeles and postdoctoral research funded by the National Cancer Institute at the University of Wisconsin.

Dr. Reynolds joins Knopp as Senior Director of Biology after seven years at Merck Research Laboratories in West Point, Pa., where he led drug discovery groups in Parkinson’s disease, stroke, Alzheimer’s disease, and ophthalmology. A recognized authority in mitochondrial biology, he served earlier as Professor and Vice Chair in the Department of Pharmacology at the University of Pittsburgh and as Associate Dean of Graduate Studies at the University of Pittsburgh School of Medicine. He completed his graduate studies in pharmacology at Johns Hopkins University and his postdoctoral research at the University of Chicago.

Data from Phase 2 Study of Dexpramipexole in ALS published in Nature Medicine

Knopp Biosciences LLC today announced the publication in Nature Medicine of comprehensive results from the Phase 2 study of dexpramipexole, a small-molecule modulator of mitochondrial bioenergetics, in people with amyotrophic lateral sclerosis (ALS). Dexpramipexole is currently in Phase 3 development under an exclusive worldwide license with Biogen Idec.

“Results from this Phase 2 study demonstrate why we’re so enthusiastic about the rapid advance of dexpramipexole to a Phase 3 trial,” said Merit Cudkowicz, M.D., primary author of the Nature Medicine manuscript and director of the MDA ALS Clinic at Massachusetts General Hospital and the Northeast ALS Consortium. Dr. Cudkowicz was the Principal Investigator for the Phase 2 study of dexpramipexole in ALS. “In this well-controlled Phase 2 study, we detected an apparent clinically significant, dose-dependent effect on both function and mortality – an exciting milestone. This is the first time we’ve seen data suggesting benefits on both function and mortality from a drug candidate in ALS.”

The Phase 2 clinical trial, designed by Knopp and its principal investigators and conducted at 20 U.S. sites, involved a novel design for an ALS trial in which the same subjects were randomized twice, essentially creating two separate, double-blind studies over which treatment effects could be observed on safety, functional decline, and mortality. In Part 1 of the study, 102 subjects received daily divided doses of 50 mg, 150 mg, or 300 mg of dexpramipexole or placebo for 12 weeks. In Part 2, 92 continuing subjects received placebo during a 30-day, single-blind washout period, followed by re-randomization to divided daily doses of either 50 mg or 300 mg for 24 weeks.

The Nature Medicine article details treatment effects across both parts of the study, including mortality effects. During the 12 weeks of Part 1, there were no deaths. During the 30-day placebo washout period, there were three deaths, limited to patients who had received placebo or low-dose treatment (50 mg) during Part 1. During the 24-week double-blind active treatment period in Part 2, there were 12 deaths, with nine deaths involving patients receiving the low dose (50 mg) and three deaths involving patients receiving the high dose (300 mg). This represented a 68% reduction in the hazard of mortality for the 300 mg group over the 24 weeks of Part 2 (p=0.07).

The article also describes treatment effects on measures of disease progression in both parts of the study, as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R), a widely used and validated measure of function. In Part 1, while the overall effect on function across all dose groups was not significant (p=0.11), the mean slope of decline for patients receiving 300 mg was reduced by 39% compared to the placebo group (0.88 units per month versus 1.28 units per month, respectively). A dose-dependent trend was seen in the attenuation of median decline in ALSFRS-R scores from baseline to 12 2

weeks (4.0 points for the placebo group, 3.0 points for the 50 mg group, 2.5 points for the 150 mg group, and 2.0 points for the 300 mg group, representing a 50% attenuation of median decline in the 300 mg group compared to the placebo group).

The trend in function favoring the 300 mg group was sustained in Part 2 of the study, with a 20.5 percent attenuation in the rate of decline compared to the active control group receiving 50 mg (p=0.177).

A significant effect was seen in a Combined Assessment of Function and Survival (CAFS), a joint-rank test that analyzes functional outcomes while accounting for in-study mortality. In this pre-specified test in Part 2 of the Phase 2 study, the mean CAFS score for the 300 mg group was 27 percent higher (more favorable outcomes) than for the 50 mg group (p=0.046).

Dexpramipexole demonstrated favorable safety and tolerability profiles in this Phase 2 trial, supporting further investigation in a Phase 3 trial. Adverse events noted in the trial included falls, muscle weakness, post-lumbar puncture syndrome, and headache, with no trend in differences in frequency among dose groups. Infrequent, reversible neutropenia was observed in both parts of the study among subjects in the 300 mg group.

About dexpramipexole 

Dexpramipexole (also known as KNS-760704) is a low molecular weight benzothiazole which in pre-clinical models has been shown to improve mitochondrial function and to confer significant protection to neurons under stress. Dexpramipexole is highly orally bioavailable, water soluble, renally excreted, and only moderately protein bound. The compound was originally identified as a candidate therapy for ALS by James Bennett, M.D., Ph.D., then of the University of Virginia. Dexpramipexole has received orphan drug designation from the U.S. Food and Drug Administration and the European Commission for the treatment of patients with ALS, as well as Fast Track designation from the FDA.

About ALS 

Amyotrophic lateral sclerosis, also known as Lou Gehrig’s disease and motor neuron disease, is a universally and rapidly 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 and the health care system. U.S. prevalence is approximately 20,000 and the global incidence is approximately two per 100,000. Beyond symptomatic care, only one drug has been approved for the treatment of ALS. Life expectancy after symptom onset is usually three to five years.

About Knopp Biosciences LLC 

Knopp Biosciences LLC, based in Pittsburgh, PA, USA, is a drug discovery and development company focused on delivering breakthrough treatments for unmet needs in neurology through innovation, experience, and partnership. The company’s lead product candidate is dexpramipexole, an orally bioavailable small molecule in Phase 3 development for the treatment of ALS. Biogen Idec holds an exclusive worldwide license from Knopp to develop and commercialize dexpramipexole, with Knopp providing development support and conducting certain U.S. commercialization activities. Knopp’s discovery platform is directed to identifying next generation mitochondrial bioenergetic modulators for the treatment of Parkinson’s disease and other neurodegenerative disorders.

This press release contains “forward-looking statements,” including statements relating to planned regulatory filings and clinical development programs for dexpramipexole. 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, 3

as well as additional factors that may cause Knopp’s actual results to differ from our expectations. There can be no assurance that dexpramipexole 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. 

BioBlast Event Hits Knopp Biosciences Sept. 6

Kick off the fall chatting it up with your colleagues at BioBlast hosted by Knopp Biosciences.  Knopp is a South Side based discovery and development company with a lead molecule in global Phase 3 development for Amyotrophic Lateral Sclerosis (ALS), or Lou Gehrig’s disease.

Nearly a third of the proceeds from the registration fee will go to support the ALS Association for their Annual Walk to Defeat ALS on September 10.

BioBlast at Knopp Biosciences
Tuesday, September 6, 2011 | 4:00 PM – 6:00 PM
Location: Knopp Biosciences LLC, 2100 Wharton Street, Suite 615, Pittsburgh, PA 15203
Cost: $35 Member* | $185 Non-Member*


Leaders of nine Pittsburgh-area companies named regional Ernst & Young Entrepreneur of the Year®; will now compete for national recognition

Ron Morris awarded Lifetime Achievement Award for his personal entrepreneurship and work mentoring local entrepreneurs.

Ernst & Young LLP is pleased to announce this year’s winners of the regional Entrepreneur Of The Year® program.  The awards recognize outstanding entrepreneurs who demonstrate excellence and extraordinary success in such areas as innovation, financial performance and personal commitment to their businesses and communities.  Winners were selected by an independent panel of judges, and the awards were presented at a gala event today at the Westin Convention Center Hotel in Pittsburgh.

 The Ernst & Young Entrepreneur Of The Year 2011 regional winners are:

Industrial Solutions Category:

Bossa Nova Robotics  – Pittsburgh

Martin Hitch – Chief Executive Officer

Sarjoun Skaff, PhD – Chief Technology Officer

David Palmer – Chief Strategy Officer

Technology Category:

Confluence – Pittsburgh

Mark Evans – Chairman and Chief Executive Officer

Legacy Business Category:

Giant Eagle, Inc. – Pittsburgh

Laura Shapira Karet – Senior Executive Vice President and Chief Strategy Officer

Manufacturing & Construction Category:

Glenn O. Hawbaker, Inc. – State College

Daniel R. Hawbaker – President

Life Sciences Category

Knopp Biosciences, LLC – Pittsburgh

Michael E. Bozik, MD – President and Chief Executive Officer

Business Services Category:

Paris Companies – Du Bois

David Stern – President & CEO

Not-for-Profit Category:

St. Barnabas Health System – Gibsonia

William V. Day – President

Health Services Category:

The Tomayko Group – Pittsburgh

John R. Tomayko, PhD – President

eCommerce Category:

Wolfe, LLC – Pittsburgh

Jason Wolfe – President and Chief Executive Officer

“These individuals and organizations truly embody innovation by creating novel solutions to relevant problems faced by either businesses or consumers,” said Kevin Pickels, Program Director for the Entrepreneur Of The Year awards in Western Pennsylvania and West Virginia.

In addition to the honorees, local entrepreneur and radio host Ron Morris was honored for his work with entrepreneur mentorship, as well as his own personal start-up successes.

“Ernst & Young is especially proud to honor Ron Morris,” said Pickels.  “He has added countless new jobs to our economy and has truly enriched our local community as a result.”

 For over thirty years Morris has followed his passion for entrepreneurship, and to date has successfully started several businesses in the Pittsburgh area.  Currently Morris actively sits on the Board of eight companies.  In addition to starting businesses, Morris has decided to share his passion for entrepreneurship with others.  In 2003, Duquesne University named Morris the Director of the Entrepreneurial Studies program, through which he has been able to bestow his wisdom upon hundreds of business students whom also share his passion for entrepreneurship.

In addition to his work at Duquesne University, Ron Morris is also known for his business-based talk radio show, The American Entrepreneur.  Since starting Entrepreneur, Morris has been able to evolve the talk show into a nationally syndicated radio broadcast; and has used the show’s success to create an all-business local radio station, WMMY 1360 AM.  Through these broadcasts, Morris has been able to advise and inspire thousands of individuals who are interested in starting their own businesses.

He has also provided insight for the Pittsburgh Technology Council’s TEQ magazine; and in addition to his work as a mentor and advisor for countless entrepreneurs, has provided startup capital for many local businesses as an active angel investor.

The Entrepreneur Of The Year Program honors entrepreneurs regionally in June, leading up to the national awards in November.  Additionally, venture-backed companies that win an Entrepreneur Of The Year Award regionally are eligible for the Entrepreneur Of The Year Venture Capital Award Of Excellence. The overall U.S. winner then moves on to compete for the Ernst & Young World Entrepreneur Of The Year Award in June.

This year, the Entrepreneur Of The Year Program celebrates its 25th anniversary.  The program has expanded to recognize business leaders in more than 140 cities and more than 50 countries throughout the world.  All regional honorees are invited to the Entrepreneur Of The Year National Awards, hosted by Jay Leno on November 12, 2011, in Palm Springs, California. The gala is the culminating event of the Ernst & Young Strategic Growth Forum®, the nation’s most prestigious gathering of high-growth, market-leading companies.

*For more information on the Ernst & Young Strategic Growth Forum, please visit

About the Entrepreneur of the Year Lifetime Achievement Award

For the last 25 years, the Ernst & Young Entrepreneur Of The Year Program has recognized outstanding business leaders who inspire others with their innovative vision and established track record of achievement. The program identifies, honors and supports those outstanding business leaders who excel in the following areas: financial performance, risk and commitment, innovation, relationships and community contributions.

However, in the world of business there are some who have achieved such great success that their efforts demand special recognition. The winners of this award have, over a sustained period of time, achieved entrepreneurial success through one outstanding venture or, in many cases, a series of entrepreneurial endeavors. They have built a significant presence in their business community and are admired not only for their business activities but also for the way they have inspired others through their mentorship and active engagement in community and philanthropic efforts.


Founded and produced by Ernst & Young LLP, the Entrepreneur of the Year Awards are pleased to have the Ewing Marion Kauffman Foundation as a national sponsor.

In Western Pennsylvania and West Virginia, sponsors include PNC Bank, ParenteBeard, Buchanan Ingersoll & Rooney, PC, Smart Business Network and Bravo Group.

For more information about the program, please visit

 About Ernst & Young Entrepreneur of the Year

Ernst & Young Entrepreneur of the Year is the world’s most prestigious business award for entrepreneurs. The unique award makes a difference through the way it encourages entrepreneurial activity among those with potential, and recognizes the contribution of people who inspire others with their vision, leadership and achievement. As the first and only truly global award of its kind, Entrepreneur Of The Year celebrates those who are building and leading successful, growing and dynamic businesses, recognizing them through regional, national and global awards programs in more than 140 cities in more than 50 countries.

 About Ernst & Young

Ernst & Young is a global leader in assurance, tax, transaction and advisory services. Worldwide, our 141,000 people are united by our shared values and an unwavering commitment to quality. We make a difference by helping our people, our clients and our wider communities achieve their potential.

Ernst & Young refers to the global organization of member firms of Ernst & Young Global Limited, each of which is a separate legal entity. Ernst & Young LLP is a client-serving member firm of Ernst & Young Global Limited operating in the US. For more information about our organization, please visit

This news release has been issued by Ernst & Young LLP, a US client-serving member firm of Ernst & Young Global Limited.

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

The ALS Association of Western Pennsylvania recently announced 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.