- US FDA designates AUT00201 as a drug for rare pediatric disease and
as an orphan drug
Stevenage, UK - 13 June 2023 - Autifony Therapeutics Limited (“Autifony”), which is pioneering the development of novel pharmaceutical treatments for rare CNS disorders and other serious brain diseases, today announced that a Phase Ib trial of AUT00201, a potent and selective modulator of Kv3 ion channels, is enrolling patients with Myoclonus Epilepsy and Ataxia due to Potassium (K+) Channel Mutation or MEAK (also known as EPM7) at the Hospital of the University of Pennsylvania.
Epilepsy is a brain disorder that causes recurring, unprovoked seizures. It is the fourth most common neurological disorder worldwide. Patients with EPM7 have a very rare form of epilepsy, caused by reduced function of Kv3.1 ion channels, which gives rise to myoclonic seizures (brief shock-like jerks of a muscle or a group of muscles), action-induced myoclonus and a progressive ataxia (impairing co-ordination, balance and speech).
EPM7 symptoms typically emerge between the ages of 3 and 15, with progressively severe myoclonus, ataxia, and occasional tonic-clonic seizures. Patients are typically wheel-chair bound by the time they are in their late teens. Current anti-seizure drugs can help to reduce the incidence of tonic-clonic seizures, but these drugs do not improve the myoclonus or ataxia.
AUT00201 is a potent positive modulator of Kv3.1 ion channels. In a genetic mouse model of EPM7 conducted by Dr Ethan Goldberg at the Children’s Hospital of Philadelphia, AUT00201 completely reversed the seizure and ataxia phenotype. The Phase Ib study in patients with EPM7 is a randomized, double-blind, placebo-controlled, crossover study in 8-10 patients aged 18 or over in which effects both on clinical symptoms and biomarkers related to dysfunction of Kv3.1 channels are evaluated.
Separately, the US FDA has designated AUT00201 for treatment of EPM7 as a drug for a ‘rare pediatric disease’ as defined in section 529(a)(3) of the Federal Food, Drug, and Cosmetic Act. The Office of Orphan Products Development has also granted orphan-drug designation for AUT00201 for the treatment of EPM7.
Dr Charles Large, Chief Executive Officer of Autifony Therapeutics, commented: “This is a unique opportunity for precision medicine, in which we aim to reverse the impact of a genetic mutation that causes a highly debilitating disorder. We are hopeful that this will offer benefit to patients with EPM7 where there are currently no effective treatments, and might one day also bring benefits to patients suffering from other forms of myoclonic epilepsy, which we will be looking to investigate in future.”
Notes to Editors
About Autifony Therapeutics Ltd
Autifony Therapeutics is an independent UK based biotechnology company formed in 2011 as a spin-out from GSK, which retains equity in the company. The Company is focused on the development of high value, novel medicines to treat serious diseases of the central nervous system. It is funded by SV Health Investors, IP Group, Pfizer Ventures, International Biotechnology Trust PLC, and UCL Business plc. For more information, please visit www.autifony.com
AUT00201 is a novel, orally active small molecule designed to selectively modulate Kv3 potassium channels. Safety and toxicology studies support the clinical development of AUT00201.
For more information, please contact:
Autifony Therapeutics Limited
Dr Charles Large, CEO
Giulia Lasagni / Batoul Ali
T: +44 (0) 20 7457 2020