Aevi Genomic Medicine Announces Enrollment of First Patient in Phase 1/2 Clinical Trial of AEVI-001 in 22q Deletion Syndrome

Aevi Genomic Medicine, Inc. (NASDAQ: GNMX) (the Company) announced today that the first patient has been dosed in a Phase 1/2 clinical trial to assess the efficacy of its lead investigational drug, AEVI-001 (NFC-1), in children with 22q11.2 Deletion Syndrome (22q DS), a rare disorder caused by an abnormality found on the 22^nd chromosome. The trial will evaluate the changes in symptoms associated with three neuropsychiatric disorders — including anxiety, attention deficit hyperactivity disorder (ADHD) and autism spectrum disorders (ASD) — that are often found in patients affected by 22q DS.
“We are pleased to have the first patient enrolled into this important study,” said Garry Neil, Chief Scientific Officer of Aevi Genomic Medicine. “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. The patients enrolled into this study will have a confirmed deletion of the RANBP1 gene, a common component of 22q DS. Mutations in this gene are thought to disrupt glutamatergic signaling, and may be causally associated with the psychiatric symptoms exhibited by these patients. AEVI-001 is a glutamate modulator that may ameliorate these symptoms.”
About the Trial (MDGN-NFC1-22Q-101)
The study is being conducted at The Children’s Hospital of Philadelphia (CHOP) and will enroll subjects ages 12-17 years with 22q DS who have a diagnosis of anxiety, ASD, or ADHD. The primary endpoints in the trial will be the changes from baseline on established metrics associated with the three neuropsychiatric disorders. Enrolled subjects will receive 5 weeks of open-label treatment with AEVI-001. More information on the trial is available at www.clinicaltrials.gov (Identifier: NCT02895906).
About 22q11.2 Deletion Syndrome
22q11.2 deletion syndrome is a disorder caused by the deletion of a small piece of chromosome 22. Individuals with 22q DS can have a wide range of signs and symptoms including heart abnormalities, cleft palate, and facial abnormalities. Children with 22q DS can also suffer from developmental delays, including delayed growth and speech development, learning disabilities, and are at an increased risk of developing mental illnesses such as schizophrenia, depression, anxiety, and bipolar disorder. Additionally, affected children are more likely than children without 22q DS to have ADHD and developmental conditions, such as ASD, that affect communication and social interaction.
About Aevi Genomic Medicine, Inc.
Aevi Genomics Medicine, Inc. is dedicated to unlocking the potential of genomic medicine to translate genetic discoveries into novel therapies. Driven by a commitment to patients with pediatric onset life-altering diseases, the company’s research and development efforts leverage an internal genomics platform and an ongoing collaboration with the Center for Applied Genomics (CAG) at The Children’s Hospital of Philadelphia (CHOP).
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