Knopp Neurosciences Inc. (“Knopp”) announced the presentation today of encouraging clinical results in a Phase 2 safety and tolerability study of KNS-760704 in amyotrophic lateral sclerosis (“ALS”). The results were presented at the 20th International Symposium on ALS/MND in Berlin, Germany, by Merit Cudkowicz, M.D., Associate Professor of Neurology at the Massachusetts General Hospital of Harvard Medical School.
The two-part Phase 2 study found that KNS-760704 was safe and well-tolerated in ALS patients for up to nine months. The study results also showed trends suggesting the potential for improved outcomes in function and survival. Knopp emphasized that the compound remains early in its development and that further testing in large, long-term, well-controlled Phase 3 studies is required to establish the necessary evidence that the drug is both safe and effective for patients with ALS.
“Knopp is very encouraged by the results presented today in Berlin and, at the same time, we’re acutely aware of the work that remains to be done,” said Michael Bozik, M.D., the President and CEO of Knopp. “We look forward to confirming and extending these results in Phase 3 studies, which we hope to initiate in 2010.” Knopp has formally engaged regulators in the U.S. and European Union to obtain scientific input on its planned Phase 3 program, including a scheduled meeting with the U.S. FDA in January 2010.
Added Dr. Cudkowicz: “The safety results and the trends in improved functional and survival outcomes observed in this study provide preliminary evidence supporting the ongoing evaluation of KNS-760704 in Phase 3 clinical trials.”
The primary objective of the Phase 2 study was to assess the safety and tolerability of KNS-760704 in ALS subjects for up to nine months. Secondary objectives included measuring the clinical effects of KNS-760704 on functional decline and mortality. The two-part design of the study provided the opportunity to assess the effects of KNS-760704 in the same sample of ALS subjects in two randomized, double-blind treatment periods separated by a one-month placebo washout.
In Part 1 of the study, 102 subjects received daily doses of 50 mg, 150 mg, or 300 mg of KNS-760704 or placebo for 12 weeks. KNS-760704 showed a dose-dependent trend in slowing the rate of disease progression as measured by the difference in slopes of ALS Functional Rating Scale-Revised (ALSFRS-R) across treatment groups, with the greatest benefit observed in the 300 mg dose group.
In Part 2 of the study, 92 subjects were re-randomized to receive daily doses of 50 mg or 300 mg of KNS-760704 for 24 weeks. In addition to results again suggesting a dose-dependent trend in slowing the rate of disease progression as measured by the ALSFRS-R, there was also a trend toward a survival benefit in the 300 mg group compared with the 50 mg group. In an exploratory test combining mortality and functional outcomes, subjects in the 300 mg group had a significantly improved outcome compared with the 50 mg group.
KNS-760704 is a low molecular weight benzothiazole shown to improve mitochondrial function and to confer significant cellular protection in neurons under stress. The chirally pure form of the synthetic benzothiazole (6R)-2-amino-4,5,6,7-tetrahydro-6-(propylamino) benzothiazole, KNS-760704 is highly orally bioavailable, water soluble, renally excreted, and only moderately protein bound. The compound 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.
Amyotrophic lateral sclerosis, also known as Lou Gehrig’s disease and Charcot’s sclerosis, is a rapid, universally 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. U.S. prevalence is approximately 20,000 and the global incidence is approximately two per 100,000. Only one drug has been approved for the treatment of ALS. Life expectancy after symptom onset is usually three to five years.
About Knopp Neurosciences Inc.
Knopp Neurosciences, based in Pittsburgh, PA, USA, is a drug discovery and development company focused on delivering breakthrough treatments for neurological disorders through innovation, experience, and partnership. The company’s lead product candidate is KNS-760704, an orally bioavailable small molecule in development for the treatment of ALS. Knopp’s leadership includes experienced neuroscience drug development and discovery executives formerly associated with major pharmaceutical companies. Knopp’s financing has been led by Saturn Capital Inc. of Boston as placement agent and Saturn Partners II as lead funder.
The Pittsburgh Life Sciences Greenhouse (PLSG), the organization providing capital investments and customized company formation and business growth programs to the region’s life sciences enterprises, announced that three life sciences industry leaders have joined the PLSG’s Executive Program.
Joining the PLSG as Executives in Residence (EIRs) are Maureen Peszko and Michael Lang. Pierre E. Queiroz de Oliveira, Ph.D. has joined the Executive Program as an Executive Associate.
Maureen brings a strong background and senior management experience in the healthcare information technology segment. Most recently, Ms. Peszko was Senior Vice President, Strategy, Product Development, Government Affairs & Business Development at Sage Health Care. Prior to Sage Health Care, Maureen was Senior Vice President, Strategic Services and US Operations for Thomson Healthcare, a division of Thomson Reuters. From 1999 to 2007, Maureen served as Vice President & General Manager of Cerner Corporation, the leading US supplier of healthcare information technology solutions that optimize clinical and financial outcomes.
Maureen’s career also includes positions with Deloitte Consulting and McKesson. Maureen holds a Bachelor of Arts Degree in Economics from Vanderbilt University and attended the Heinz Graduate School for Public Policy at Carnegie Mellon University.
Michael Lang joins the PLSG with 27 years of executive experience in the medical devices industry. He has an extensive background in general management, strategic business development, fundraising, marketing, product development and biomedical engineering. Most recently he served as the President of Galt Medical; a Dallas, TX based cardiovascular products company. His prior experience includes Vice President of Business Development at BioEnterprise in Cleveland, OH, Division General Manager at Avery Dennison Medical, Director of Market Development at wound healing company, Gliatech, Director of Product Development at Focal Interventional Therapeutics, Product Manager at Johnson & Johnson, Product Development Engineering Manager at CR Bard and Product Development Engineer at Gore and Associates, Medical Division. Michael holds a Bachelor of Science Degree in Biomedical Engineering from Northern Arizona University and a Master of Business Administration from Arizona State University.
Pierre E. Queiroz de Oliveira, Ph.D. has joined the PLSG as an Executive Associate focusing on the therapeutics segment. His experience is in Pharmacology and Drug Discovery. Pierre was selected as a Fellow in Molecular Pharmacology at the University of Pittsburgh and has received recognition for his outstanding research presentations at the University of Pittsburgh and the University of Bath, United Kingdom. Pierre holds a Master of Science Degree from the University of Bath and a Ph.D. in Molecular Pharmacology from the University of Pittsburgh.
“The availability and attraction of domain-specific talent to our regional life sciences community has been paramount to the PLSG’s success,” said John W. Manzetti, PLSG’s President and Chief Executive Officer. “The world-class caliber of our Executive Program and the extensive background of our EIRs continue to build on our national reputation for attracting world-class talent to the region and building great life sciences companies.” Manzetti continues, “By helping them build sustainable business models and secure capital investments, we make it possible for companies to deliver biomedical innovations to the marketplace more quickly and efficiently than they ever could alone.”
Techburgher came across a very telling story at the San Francisco Chronicle’s SFGate.com. Looks like the high cost of living and the related stresses are catching up to folks in big cities like San Fran and New York. Rob Baedeker wrote an interesting article detailing his experience trying to make ends meet in the Bay Area. Check it out right here. Techburgher thinks Pittsburgh is a great place for folks like Rob. We’re going to try to line him up as guest on a future TechVibe Radio Radio Show. We’ll keep ya posted.
On the field, Pittsburgh Steelers quarterback Charlie Batch is known for his reliable and accurate throwing arm. Off the field, he’s known and recognized for his dedication to mentoring children through his Best of the Batch Foundation.
For the past two years, Batch has held the “In the Pocket” fundraiser to benefit Best of the Batch’s reading and computer literacy program. The event features Batch, his teammates and other local celebrities playing friendly games of ping-pong, pool and poker. However, everything from registration to game management and scoring was handled on paper — causing logistical difficulties from game queue management to real-time scoring.
To ensure that this year’s event ran more smoothly and showcased real-time results, Batch teamed up with the information systems (IS) application course — a senior-level course within Carnegie Mellon University’s College of Humanities & Social Sciences’ Program. Students Thomas Flavin, Meredith Huffsmith, Phil Pantalone and Amritha Prasad worked under faculty advisors Jeria Quesenberry and Larry Heimann to design and build an event scheduling system for “In the Pocket.” The project team attended the third annual event in November and unveiled their work — a software application that registered and checked-in players, updated schedule changes, informed players of game times and displayed live results on a large, projected scoreboard.
“The project was a huge success,” said Quesenberry, an IS assistant teaching professor. “In 12 weeks, the students met with the foundation’s leadership and delivered a customized and reusable application. A similar commercial software solution would have cost the foundation several thousand dollars, so by working with us, they were able to improve their event without spending money that could otherwise impact the lives of children. And, our students were able to take their classroom knowledge and apply it in a real world setting.”
A total of 15 IS teams created projects for clients, which were shown during a presentation in early December. During Fall Semester, 2009, a total of 15 IS teams created projects for community clients, which were shown during a presentation in early December. “These projects give local non-profit clients made-to-order software solutions that they otherwise couldn’t afford. It’s a win-win situation,” said Randy Weinberg, teaching professor and head of the Information Systems program.