Inspired to help
Knopp Biosciences LLC (Knopp) was founded in 2004 as Knopp Neurosciences Inc. by Greg Hebrank and Launchcyte LLC. The company was named for Walter Knopp, a Pittsburgh area entrepreneur and philanthropist afflicted with amyotrophic lateral sclerosis (ALS), a progressive neurological disorder often referred to as “Lou Gehrig’s disease”. With intellectual property licensed from both the University of Pittsburgh and the University of Virginia, the company built its initial drug pipeline around a molecule named dexpramipexole (Dex), shown in preclinical studies to protect nerve cells under stress, and thought to be a potential treatment for ALS.
Under the leadership of company CEO Mike Bozik, Knopp advanced Dex to a Phase I clinical trial in September 2007, and a follow-on Phase II study in April 2008. As published in Nature Medicine, the Phase II clinical data showed efficacy at slowing the progression of ALS. The favorable data led to a licensing transaction with Biogen Inc. in 2010. In 2011, Biogen launched a 900+ patient Phase III clinical trial for Dex in ALS. In 2013, the Phase III study failed to replicate the results in the Phase II study. The Biogen license was terminated in 2014, and the development rights and clinical data for Dex were returned to Knopp.
Clinical breakthroughs
The clinical data from the Phase II and Phase III ALS studies produced an unexpected result. Routine blood analysis of patients participating in the clinical trial showed a marked decrease of eosinophils, a type of white blood cell. Eosinophils protect humans from certain pathogens, including many parasites. Elevated levels of eosinophils in the blood are often associated with asthma and other inflammatory disorders. Armed with these data, Knopp launched Phase II clinical trials for Dex in eosinophilic asthma and hyper eosinophilic syndrome in 2015 and 2016.
Simultaneous with the clinical work on Dex, Knopp expanded its pipeline by developing a set of small molecule compounds to treat dysfunction of the Kv7 ion channels in nerve-cell membranes. Dysfunction of Kv7 ion channels is often associated with epilepsy and other neurological disorders. The Kv7 development effort was led by CSO Steve Dworetzky.
Growth & Expansion
Following a significant capital raising transaction in 2019, the company commenced a concerted effort to find a partner to fund and manage a large multi-center Phase III clinical trial for Dex in eosinophilic asthma. Those efforts resulted in a partnership with Population Health Partners (PHP) and syndicate of private equity investors led by Bain Capital. In June 2022, Knopp contributed its Dex pipeline to Areteia Therapeutics, Inc., a new entity with $350 million of committed capital.
In parallel with the process of identifying a partner for Dex, Biohaven Pharmaceuticals Limited developed an interest the company’s Kv7 preclinical program. Biohaven purchased the company’s Kv7 platform in April 2022.
Knopp Today
Today, Knopp is no longer an operating company. Its former employees are now employed by Biohaven and Areteia. Instead of test tubes, microscopes, and intellectual property, the company’s assets consist of equity securities of Areteia and Biohaven, as well as contract rights to potential future payments from Biohaven related to the Kv7 platform.
Areteia is currently conducting three Phase III clinical studies of dexpramipexole for the treatment of eosinophilic asthma (EXHALE 2, EXHALE 3 and EXHALE 4). Biohaven is currently conducting a Phase II/III clinical study of BHV-7000 for the treatment of focal epilepsy. If either program (or both) is successful, the company expects to realize significant cash from these assets, which, subject to the discretion of the Board of Managers, will eventually be distributed to the company’s owners.
The People
Behind the Medicine
The People
Behind the Medicine
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C David Adair
Member — Board of Directors
Steve Butts
Member — Board of Directors
Michael E. Bozik
Member — Board of Directors
Benjamin Gomez
Member — Board of Directors
Gregory T. Hebrank
Member — Board of Directors
Jeffrey McCormick
Member — Board of Directors
Thomas Petzinger, Jr.
Member — Board of Directors
F.J. Lucchino
President