KCNQ2 Preclinical Program
Ion Channel Biology
Pursuing Novel Drug Treatments
Ion channels, proteins that regulate the excitability of cells, represent an important class of drug targets. Drugs that modulate ion channels play an important role in treating a variety of neurological and cardiovascular conditions. Among ion channel targets, potassium channels play a key role in the pathophysiology of neuropathic pain, epilepsy, and other unmet needs in diseases of the nervous system.
Knopp has reached the lead optimization stage of identifying novel selective modulators of a voltage-gated potassium channel family known as KCNQ2, building on the significant experience of its scientific leadership in characterizing and exploiting the pharmacology of this target. The KCNQ2 program provides the opportunity to secure composition-of-matter patent positions for development candidates. Knopp chemists have synthesized a library of novel compounds that modulate ion channel activity relevant in indications of persistent unmet need related to neuronal excitability.
- In neonatal encephalopathy we are targeting KCNQ2 gene mutations associated with seizures and profound developmental effects.
- In neuropathic pain, we are using translational studies to better understand potassium channel profiles in specific pain indications and to explore the potential for topical skin administration as well as oral administration.
- In epilepsy, we have synthesized channel-subtype selective agents with improved potency vs. ezogabine, the only approved KCNQ2 channel modulator, with the goal of an improved safety profile and one-daily oral dosing.
- In tinnitus, we are developing potent channel-subtype activators for proof-of-concept studies in animal models.