Aevi Genomic initiates Phase I/II trial of AEVI-001 to treat 22q DS

1st February 2017 (Last Updated February 1st, 2017 18:30)

US-based Aevi Genomic Medicine has initiated its Phase I/II clinical trial with the enrolment and dosing of its first patient with AEVI-001 (NFC-1) to treat 22q11.2 deletion syndrome (22q DS) in children.

US-based Aevi Genomic Medicine has initiated its Phase I/II clinical trial with the enrolment and dosing of its first patient with AEVI-001 (NFC-1) to treat 22q11.2 deletion syndrome (22q DS) in children.

AEVI-001 is an investigational glutamate modulator designed to relieve psychiatric symptoms associated with 22q DS patients caused due to mutations in RANBP1 gene that disrupts glutamatergic signalling.

The Phase I/II trial will evaluate the changes in symptoms observed in three neuropsychiatric disorders, anxiety, attention deficit hyperactivity disorder (ADHD) and autism spectrum disorders (ASD), which are often found in patients affected by 22q DS.

Aevi Genomic Medicine chief scientific officer Garry Neil said: "We are pleased to have the first patient enrolled into this important study.

"We are pleased to have the first patient enrolled into this important study."

"Patients with 22q Deletion Syndrome often suffer from a range of debilitating psychiatric conditions, including anxiety disorders, ASD, and ADHD, with many patients progressing to psychosis and schizophrenia."

22qDS is a condition caused by the deletion of a small piece of chromosome 22 and is characterised by heart and facial abnormalities, as well as cleft palate.

The trial is to be conducted at the Children's Hospital of Philadelphia (CHOP), US, and will enrol 22q DS patients aged between 12 and 17 years with a diagnosis of anxiety, ASD, or ADHD.

It will be ensured that the patients have a confirmed deletion of the RANBP1 gene and will receive five weeks of open-label treatment with AEVI-001.

The primary endpoints will be the changes from baseline on established metrics related to the three neuropsychiatric disorders.