AUT00201 / Autifony Therap - LARVOL DELTA

Kv3 channel agonist ameliorates the phenotype of a mouse model of amyotrophic lateral sclerosis. (PubMed, Acta Neuropathol Commun) - "The expression of the Kv3 channel is lost upon acute motor unit damage, in mouse models of amyotrophic lateral sclerosis (ALS) and spinal and bulbar muscular atrophy (SBMA), and the skeletal muscle of patients with sporadic ALS. Early treatment of ALS and SBMA mice with AUT00201, a positive allosteric modulator of Kv3 channels, improved the phenotype of ALS mice specifically, suggesting that positive modulation of Kv3 channels is a novel therapeutic option for patients with ALS."

Journal • Preclinical • Amyotrophic Lateral Sclerosis • CNS Disorders

AUT022201: Safety, Blood Levels and Effects of AUT00201 in Patients With MEAK (clinicaltrials.gov) - P1 | N=6 | Completed | Sponsor: Autifony Therapeutics Limited | Recruiting ➔ Completed | N=10 ➔ 6

Enrollment change • Trial completion • Ataxia • CNS Disorders • Epilepsy • Movement Disorders

AUT022201: Safety, Blood Levels and Effects of AUT00201 in Patients With MEAK (clinicaltrials.gov) - P1 | N=10 | Recruiting | Sponsor: Autifony Therapeutics Limited | Trial completion date: Dec 2023 ➔ Mar 2024 | Trial primary completion date: Dec 2023 ➔ Mar 2024

Trial completion date • Trial primary completion date • Ataxia • CNS Disorders • Epilepsy • Movement Disorders

A Potent and Novel Potassium Channel Positive Modulator AUT00201 Attenuates Cellular Dysfunction, Ataxia, and Seizure Susceptibility in a Mouse Model of Progressive Myoclonus Epilepsy Type 7 (AES 2023) - "Previously, we demonstrated that the complex features of EPM7 correlate with the dysfunction of Kv3.1-expressing neurons in distributed brain regions in a novel mouse model of EPM7, Kcnc1-p.Arg320His/+ (H/+) mice, and investigated the therapeutic potential of a Kv3 channel positive modulator, AUT00206. Kcnc1-p.Arg320H/+ mice exhibit progressive ataxia and enhanced seizure susceptibility, and hence reproduce key disease features of, and represent a model for, the study of EPM7. Impaired excitability of Kv3.1-expressing neurons in vitro and indices of cerebellar dysfunction and seizure susceptibility in vivo could be ameliorated with the potent Kv3-channel modulator AUT00201, which may demonstrate therapeutic potential for the treatment of human patients with EPM7."

Preclinical • Ataxia • CNS Disorders • Epilepsy • Movement Disorders

AUT022201: Safety, Blood Levels and Effects of AUT00201 in Patients With MEAK (clinicaltrials.gov) - P1 | N=10 | Recruiting | Sponsor: Autifony Therapeutics Limited

New P1 trial • Ataxia • CNS Disorders • Epilepsy • Movement Disorders

Safety, Blood Levels and Effects of AUT00201 (clinicaltrials.gov) - P1; N=80; Completed; Sponsor: Autifony Therapeutics Limited; Recruiting ➔ Completed; Trial completion date: Aug 2020 ➔ Dec 2020; Trial primary completion date: Aug 2020 ➔ Dec 2020

Clinical • Trial completion • Trial completion date • Trial primary completion date