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Knopp Biosciences Receives Rare Pediatric Disease Designation for Kv7 Activator KB-3061 for Treatment of KCNQ2 Epileptic Encephalopathy

Retrieved on: 
Wednesday, July 15, 2020

Food and Drug Administration(FDA) forits therapeutic candidate KB-3061, an activator of voltage-gated Kv7.2/7.3 potassium channels, for the treatment of KCNQ2 epileptic encephalopathy (KCNQ2-EE).

Key Points: 
  • Food and Drug Administration(FDA) forits therapeutic candidate KB-3061, an activator of voltage-gated Kv7.2/7.3 potassium channels, for the treatment of KCNQ2 epileptic encephalopathy (KCNQ2-EE).
  • The granting of this designation from the FDA represents a major milestone for Knopp Biosciences, said Michael Bozik, M.D., Chief Executive Officer.
  • The Rare Pediatric Disease Designation by FDA for KB-3061 advances our mission of promoting research and supporting families of children with KCNQ2 developmental and epileptic encephalopathy, added Jim Johnson, president of the KCNQ2 Cure Alliance .
  • Knopps preclinical Kv7 platform is directed to small molecule treatments for neonatal epileptic encephalopathy, other rare epilepsies, tinnitus, and neuropathic pain.

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

Retrieved on: 
Monday, December 9, 2019

The disease is caused by dominant-negative mutations in the KCNQ2 gene, which produces a potassium channel, Kv7.2, critical to early brain development.

Key Points: 
  • 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.
  • Nanomolar brain concentrations of KB-3061 conferred significant seizure protection in the maximal electroshock model (MES) of epilepsy with minimal impact on neurobehavior.
  • Follow Knopp Biosciences on Twitter via @KnoppBio and keep up to date with AES Annual Meeting news and updates using the hashtag #AES2019.

Knopp Biosciences to Present Data Characterizing Its Potassium Channel Activator Candidate for the Treatment of KCNQ2 Epileptic Encephalopathy at the American Epilepsy Society’s 2019 Annual Meeting

Retrieved on: 
Wednesday, November 27, 2019

Among ion channel targets, potassium channels play a key role in the pathophysiology of epilepsy and other unmet needs in diseases of the nervous system.

Key Points: 
  • Among ion channel targets, potassium channels play a key role in the pathophysiology of epilepsy and other unmet needs in diseases of the nervous system.
  • Details related to Knopp Biosciences AES abstracts and poster presentations can be found in the table below.
  • Follow Knopp Biosciences on Twitter via @KnoppBio and keep up to date with AES Annual Meeting news and updates using the hashtag #AES2019.
  • Knopps preclinical Kv7 platform is directed to small molecule treatments for neonatal epileptic encephalopathy, other rare epilepsies, and neuropathic pain.

Knopp Biosciences Receives Year 4 Renewal of NIH Blueprint Grant Award for Advancing the Kv7 Activator KB-3061 in a Rare Neonatal Epilepsy

Retrieved on: 
Wednesday, September 25, 2019

Knopp Biosciences LLC today announced the renewal of its grant award for Year 4 from the National Institutes of Health Blueprint Neurotherapeutics Network (BPN) to advance its lead Kv7 potassium channel activator, KB-3061, in rare neonatal epilepsy.

Key Points: 
  • Knopp Biosciences LLC today announced the renewal of its grant award for Year 4 from the National Institutes of Health Blueprint Neurotherapeutics Network (BPN) to advance its lead Kv7 potassium channel activator, KB-3061, in rare neonatal epilepsy.
  • The grant has a total potential direct award value of $2.4 million over four years.
  • The disease is caused by dominant-negative mutations in the KCNQ2 gene, which produces a potassium channel, Kv7.2, critical to early brain development.
  • The content of this announcement is solely the responsibility of Knopp and does not necessarily represent the views of the NIH.

Knopp Biosciences Reports Data Demonstrating Restoration of Function by KB-3061 in Cellular Model of Gene Variants Causing KCNQ2 Epileptic Encephalopathy

Retrieved on: 
Tuesday, September 17, 2019

When treated with KB-3061, the function of the mutated channels was fully restored, producing normal Kv7.2 channel current density.

Key Points: 
  • When treated with KB-3061, the function of the mutated channels was fully restored, producing normal Kv7.2 channel current density.
  • The disease is caused by dominant-negative mutations in the KCNQ2 gene, which normally produces a potassium channel, Kv7.2, critical to early brain development.
  • The results suggest that restoring suppressed Kv7.2 channel activity may ameliorate the dysfunction caused by KCNQ2 gene mutations.
  • As previously announced by Knopp, KB-3061 has also demonstrated potent seizure control in an in vivo model of epilepsy.

 Knopp Biosciences to Present Data Characterizing Its Kv7 Technology Platform’s Potential for Delivering Precision Medicines to Treat Diverse Neurological and Smooth Muscle Diseases

Retrieved on: 
Friday, September 13, 2019

Knopp Biosciences LLC today reports data demonstrating that compound subgroups from its proprietary library of potassium-channel modulators selectively target Kv7 channel subtypes associated with a wide range of neurological and smooth muscle diseases.

Key Points: 
  • Knopp Biosciences LLC today reports data demonstrating that compound subgroups from its proprietary library of potassium-channel modulators selectively target Kv7 channel subtypes associated with a wide range of neurological and smooth muscle diseases.
  • By characterizing a custom library of Kv7 modulators for activity and specificity, we have shown evidence of the potential to develop targeted treatments for diverse diseases of the central nervous system and smooth muscle, said Dr. Bozik.
  • Accordingly, we believe our Kv7 library represents a precision tool-set for investigating Kv7 pharmacology across a wide range of therapeutic areas with high unmet need.
  • Knopp Biosciences is a privately held drug discovery and development company focused on delivering breakthrough treatments for neurological and immunological diseases of high unmet need.