Additional Pipeline Programs
Friedreich’s ataxia (FA) is a rare, severe, inherited neurological disease caused by mutations in the frataxin (FXN) gene leading to decreased expression of FXN, which results in severe sensory impairment, progressive loss of the ability to walk, generalized weakness and loss of sensation, as well as severe and potentially fatal cardiomyopathy.
In partnership with Voyager Therapeutics as part of a strategic collaboration announced in January 2019, Neurocrine Biosciences is developing VY-FXN01, a gene therapy for the treatment of Friedreich’s ataxia. Additional preclinical studies are underway at Voyager including steps to identify a lead clinical candidate for the treatment of FA during 2019.
NEW VMAT2 INHIBITOR
Neurocrine Biosciences is developing a novel, internally discovered VMAT2 inhibitor that has the potential to be used in the treatment of several neurology and/or psychiatry disorders. The Company has completed dosing in the single ascending dose portion of a Phase I study designed to assess initial safety, tolerability and pharmacokinetics. The multiple dosing portion of this Phase I study is ongoing and is expected to be completed during the first half of 2019.
Neurocrine Biosciences entered into a license and collaboration agreement with Xenon Pharmaceuticals to exclusively license NBI-921352, a clinical stage, potent, highly selective Nav1.6 sodium channel inhibitor being developed to treat pediatric patients with SCN8A developmental and epileptic encephalopathy (SCN8A-DEE) and other potential indications, including adult focal epilepsy. The safety, tolerability and pharmacokinetics of NBI-921352 have been evaluated in a randomized, double-blind, placebo-controlled Phase 1 study using a powder-in-capsule formulation of NBI-921352 in healthy adult subjects. Xenon has developed a pediatric-specific, granule formulation of NBI-921352, and completed juvenile toxicology studies to support pediatric development activities. We plan to file an investigational new drug application, or IND, in mid-2020 to initiate a Phase II clinical study for NBI-921352 in pediatric patients with SCN8A-DEE.
The Company has a rich and diversified pipeline with compounds in all phases of clinical development.
Neurocrine Biosciences’ research and development focus is on addressing diseases and disorders of the central nervous and endocrine systems, which include therapeutic categories ranging from diabetes to stress-related disorders and neurodegenerative diseases.