Oryzon secures AEMPS approval to initiate ETHERAL trial

5th April 2018 (Last Updated April 5th, 2018 00:00)

Oryzon Genomics has received approval from the Spanish Agency of Medicines and Medical Devices (AEMPS) to begin the Epigenetic THERapy in Alzheimer’s Disease (ETHERAL) trial.

Oryzon Genomics has received approval from the Spanish Agency of Medicines and Medical Devices (AEMPS) to begin the Epigenetic THERapy in Alzheimer’s Disease (ETHERAL) trial.

The approval is based on a clinical trial application (CTA), which is the European equivalent of the US Food and Drug Administration’s investigational new drug (IND) programme.

The Phase IIa, 26 week parallel-group ETHERAL study aims to evaluate the safety and tolerability of ORY2001 in patients with mild and moderate Alzheimer’s disease (AD).

"Preclinical studies validate the potential of ORY-2001 to treat cognitive defects and neuroinflammation by increasing the plasticity and functionality of neurons."

It will be conducted in different hospitals across Spain, as well as in the UK and France once the corresponding approvals from the UK and French regulatory authorities are obtained.

A total of 90 patients will be enrolled in the trial, which features measurements in different domains of the disease, including memory and behaviour alterations as secondary endpoints.

The trial, which seeks to begin enrolment this quarter, will further observe the variations of diverse yet significant CSF biomarkers.

Oryzon Genomics chief medical officer Roger Bullock said: “The approval of ETHERAL, the first Phase IIa clinical trial for an epigenetic agent in AD, represents an important milestone for the company and the scientific community.

“Preclinical studies validate the potential of ORY-2001 to treat cognitive defects and neuroinflammation by increasing the plasticity and functionality of neurons.

“This is the first step in exploring this novel approach and we have chosen to study this in mild to moderate AD patients where we believe there is still physiological room to make a significant therapeutic intervention, as this is a patient population that is underserved with the conventional approaches.”

Oryzon further plans to conduct a twin study of ETHERAL in the US with a number of additional patients.

The company’s ORY-2001 is an oral and brain penetrant drug that selectively inhibits lysine-specific histone demethylase 1 (LSD1) and monoamine oxidase B (MAOB).