Knopp Biosciences Presents Data Further Characterizing Its Lead Kv7.2 Activator Drug Candidate for the Treatment of KCNQ2 Epileptic Encephalopathy at the American Epilepsy Society’s 2019 Annual Meeting
Knopp Biosciences LLC, a privately held drug discovery and development company focused on delivering breakthrough treatments for immunological and neurological diseases with high unmet need, today presented data characterizing KB-3061, the lead molecule in the Company’s Kv7 ion channel platform, at the American Epilepsy Society’s (AES) 2019 Annual Meeting in Baltimore, Maryland.
Knopp is advancing the development of KB-3061 as a potential precision medicine treatment for the rare, neonatal disease KCNQ2 epileptic encephalopathy (KCNQ2-EE), a genetically defined disease associated with seizures beginning in the first days of life and profound neurodevelopmental delay. The disease is caused by dominant-negative mutations in the KCNQ2 gene, which produces a potassium channel, Kv7.2, critical to early brain development.
Data presented at AES by Knopp scientists and its academic collaborators support the continued development of KB-3061 as a potential treatment for children with KCNQ2-EE. Key highlights of these and previously reported data include:
- KB-3061 fully restored the function of Kv7 potassium channels in cells engineered to express gene variants from three “hot spot” domains – the voltage sensor, the pore, and the C-terminus – that cause KCNQ2-EE.
- Nanomolar brain concentrations of KB-3061 conferred significant seizure protection in the maximal electroshock model (MES) of epilepsy with minimal impact on neurobehavior.
- KB-3061 is differentiated from other molecules in its class, including ezogabine, having demonstrated improved stability to photo-oxidation, reduced GABAA receptor activity, and greater potency with increased tolerability in preclinical epilepsy models.
- KB-3061 has no significant activity against a broad range of off-targets, including GPCRs, ion channels, transporters, cardiac ion channels, and kinases.
- KB-3061 IND-enabling toxicology and drug manufacturing process development studies are ongoing with support from the National Institutes of Health Blueprint Neurotherapeutics Network (BPN).
“Our conversations with members of the pediatric epilepsy community have underscored the urgent need for novel medicines that not only treat seizures but address the underlying cause of KCNQ2-EE,” said Michael Bozik, M.D., Chief Executive Officer of Knopp Biosciences. “While the potential of KB-3061 must be assessed clinically, we are vigorously pursuing its development as a targeted treatment for children affected by this disease.”
Knopp has initiated studies to support the filing of an Investigational New Drug (IND) application with the goal to assess the clinical effects of KB-3061.
Follow Knopp Biosciences on Twitter via @KnoppBio and keep up to date with AES Annual Meeting news and updates using the hashtag #AES2019.
ABOUT KNOPP BIOSCIENCES LLC
Knopp Biosciences is a privately held drug discovery and development company focused on delivering breakthrough treatments for immunological and neurological diseases of high unmet need. Knopp’s clinical-stage small molecule, dexpramipexole, is in Phase 2 clinical trials in moderate-to-severe eosinophilic asthma. Knopp’s preclinical Kv7 platform is directed to small molecule treatments for neonatal epileptic encephalopathy, other rare epilepsies, and neuropathic pain. Please visit www.knoppbio.com.
Knopp's Kv7 research is supported under Award Number U44NS093160 and Award Number U44NS115732 of the National Institute of Neurological Disorders and Stroke of the National Institutes of Health (NIH). The content of this announcement is solely the responsibility of Knopp and does not necessarily represent the views of the NIH.
This press release contains "forward-looking statements," including statements relating to planned regulatory filings and clinical development programs. 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, as well as additional factors that may cause Knopp's actual results to differ from our expectations. There can be no assurance that any investigational drug product will be successfully developed or manufactured or that final results of clinical studies will be supportive of regulatory approvals required to market a product. Knopp undertakes no obligation to update or revise any such forward-looking statements, whether as a result of new information, future events or otherwise.
Knopp's pipeline consists of investigational drug products that have not been approved by the U.S. Food and Drug Administration. These investigational drug products are still undergoing clinical study to verify their safety and effectiveness.
About Business Wire
For more than 50 years, Business Wire has been the global leader in press release distribution and regulatory disclosure.
Subscribe to releases from Business Wire
Subscribe to all the latest releases from Business Wire by registering your e-mail address below. You can unsubscribe at any time.
Latest releases from Business Wire
HighRes Biosolutions and Novo Nordisk partner to transform drug discovery in automated Lab of the Future24.1.2020 17:00:00 EET | Press release
HighRes Biosolutions and Novo Nordisk A/S today announced that they will collaborate to design a state-of-the-art robotic platform for high-throughput biologics engineering and characterisation. The integration of a multi-system robotics platform with sophisticated software control architecture will enable a digital transformation and accelerated discovery for Novo Nordisk’s peptide and large molecule therapeutics research. “In order to maintain our leadership within biologics engineering we are investing in a fully automated and digital design platform. This investment is linked to our build-up in AI and advanced analytics and demonstrates Novo Nordisk’s commitment as a world leading biologics research company,” said Lars Fogh Iversen, senior vice president, Global Research Technologies, Novo Nordisk. The platform will comprise several highly automated and integrated cells for production, characterisation and functional analysis of thousands of biologic compounds every month. Its scop
Conquest Invests in 5 Greenfield Onshore Wind Parks Totalizing 43MW in the Flanders Region24.1.2020 15:37:00 EET | Press release
Conquest, acting through the Conquest Sustainable Infrastructure fund, announced the transaction was closed after an exclusive agreement had been reached with ENGIE Electrabel. Conquest acquires 51% of a portfolio expected to provide 111 GWh per annum of renewable power representing the annual electricity consumption of about 32,000 Belgian homes from early 2021 onwards. The projects benefit from 15 to 20-year green certificates local remuneration schemes, and thus generate yearly secured revenues. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200124005221/en/ Credit: Electrabel While the energy and digital transitions continue to drive the infrastructure sector, Conquest keeps building its diversified sustainable real asset portfolio in Western European markets. The success lies in years of collaboration and long-lasting partnerships with fellow industry experts who can notably facilitate access to new growth opportunities
Fluence Expands EMEA Footprint with Horticulture Partners in Papenburg, Germany24.1.2020 14:00:00 EET | Press release
Fluence by OSRAM (Fluence), a leading global provider of energy-efficient LED lighting solutions for commercial cannabis and agriculture production, announced today it is expanding its presence in the Emsland region of Germany through partnerships with growers and other horticulture specialists operating out of Papenburg. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200124005109/en/ Friedrich Schulz’s 5.5 hectare farm—which grows herbs year-round in addition to lettuce, peppers and chives—utilizing VYPR 2p fixtures. (Photo: Business Wire) Given Papenburg’s decades-long history of attracting horticulture experts, Fluence’s innovative lighting solutions for horticulture are a natural fit for the region’s tomato, cucumber, leafy greens and herb growers. Forging connections with Papenburg’s horticultural leaders Elektro Evers, an engineering firm specializing in greenhouse technology, is Fluence’s exclusive systems integrator
HCL Technologies Honors Global Goodwill Champions at the 2020 World Economic Forum in Davos24.1.2020 12:47:00 EET | Press release
HCL Technologies (HCL), a leading global technology company, today presented its Wave Maker Awards 2020, commemorating global goodwill champions from around the world. The award ceremony, which was held at the HCL Pavilion in Davos, celebrated organizations and individuals passionately committed to such noble causes as diversity & inclusion, education & community upliftment, environment & sustainability, and technology for change. Special guest at the awards was Australian cricket legend Glenn McGrath, who is also founder of the McGrath Foundation, which provides support for Australian women diagnosed with breast cancer and their families. The award recipients were; Lois Auta, Founder and Executive Director of Cedar Seed Foundation, which supports persons with disabilities to participate in all aspects of life; Chido Govera, Founder and Director of The Future of Hope Foundation (TFoHF), which supports marginal and vulnerable members of society through entrepreneurial and self-developme
Ipsen’s palovarotene clinical program in fibrodysplasia ossificans progressiva reaches prespecified interim analysis futility criteria24.1.2020 09:00:00 EET | Press release
Regulatory News: Ipsen (Euronext: IPN; ADR: IPSEY) today announced the decision to pause dosing patients in the global Phase III (PVO-1A-301) study designed to evaluate the efficacy and safety of palovarotene in patients with fibrodysplasia ossificans progressiva (FOP), as well as the ongoing Phase II (PVO-1A-202/204) extension studies. In both the Phase III and Phase II extension studies, palovarotene is dosed both chronically (daily) and episodically (during flare-ups). The decision to pause dosing patients in the trial is based on results of a futility analysis reviewed by the Independent Data Monitoring Committee (IDMC) as part of the prespecified interim analysis. The results of a futility analysis indicated that the Phase III FOP trial was unlikely to meet its primary efficacy endpoint (annualized change in new HO volume as compared with Natural History Studyi) upon completion. Despite the results of the prespecified interim analysis, signals of encouraging therapeutic activity w
Amazentis Announces the Launch of Timeline™ Cellular Nutrition at the World Economic Forum in Davos23.1.2020 23:56:00 EET | Press release
Amazentis announces the debut of Timeline, a next generation nutrition product designed to promote healthy aging, as part of the Swiss Food & Nutrition Valley in the House of Switzerland at the World Economic Forum in Davos, Switzerland. Given the increase in the aging population globally -- according to a 2019 Deutsche Bank study, there are more people on Earth older than sixty five than younger than five for the first time -- it is imperative for consumers to take proactive measures to maintain a high quality of life, for as long as they live. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200123005820/en/ (Photo: Business Wire) Timeline contains the proprietary nutrient Mitopure™, a highly pure form of Urolithin A. After a decade of rigorous research by leading scientists around the world, Mitopure has been shown to help counter age-associated cellular and muscular decline by revitalizing the mitochondria, the powerplants
In our pressroom you can read all our latest releases, find our press contacts, images, documents and other relevant information about us.Visit our pressroom