Aevi Genomic Medicine, Inc. (NASDAQ: GNMX) (the Company) today announced that it has reached full enrollment of Part B of its Phase 2 ASCEND clinical trial. The ASCEND trial is a genomically-guided study in pediatric and adolescent patients with Attention Deficit Hyperactivity Disorder (ADHD) assessing the safety and efficacy of AEVI-001, a novel, non-stimulant therapy.

• Part B of the ASCEND trial is studying pediatric and adolescent patients with ADHD without mGluR mutations.
• Part A of the ASCEND trial is studying a mGluR mutation positive genetic subset of pediatric and adolescent patients. Part A completed enrollment in August 2018.
• The accelerated enrollment in Part B will allow the Company to provide complete top line data for both Parts A and B of the ASCEND trial in January 2019.
• Individual and pooled analysis of Parts A and B will be conducted simultaneously, and will inform the design of the planned Phase 3 program.

"We are pleased that we were able to complete enrollment of Part B so quickly and look forward to presenting the results," said Garry A. Neil, M.D., Chief Scientific Officer of the Company. "Attention Deficit Hyperactivity Disorder affects millions of pediatric and adolescent patients and despite a number of drugs on the market today, we believe treatment is suboptimal for many patients. AEVI-001 is a unique, oral, non-stimulant activator/modulator of mGluRs, with the potential to help many patients and their families."

Development of AEVI-004, a new chemical entity (NCE), co-crystal of AEVI-001 continues in order to maximize the value of Part B of the ASCEND trial. AEVI-004 potentially expands the composition of matter patent protection through 2039 and broadens market potential beyond ADHD into other neuropsychiatric diseases.

About the ASCEND Clinical Trial

ASCEND is an adaptive, 6-week, double-blind parallel-group study in children and adolescents (ages 6-17 years) with ADHD with and without copy number variants (CNVs) in specific genes implicated in glutamatergic signaling and neuronal connectivity. Part A includes subjects determined to have one of eight specific gene mutation(s) implicated in glutamatergic signaling and neuronal connectivity. Part B will assess subjects who do not have CNVs in any of the specific gene mutation(s) implicated in glutamatergic signaling and neuronal connectivity. Once subjects are confirmed as eligible for each part of the study, they are randomized to one of two treatment groups (AEVI-001 or placebo) in a 1:1 ratio.

About AEVI-001

AEVI-001 is an oral non-stimulant pan selective activator/modulator of mGluRs. The molecule has excellent pharmacokinetic and metabolic profiles and crosses the blood brain barrier.

The Company is developing AEVI-001 as a potential treatment for a sub-population of Attention Deficit Hyperactivity Disorder (ADHD) patients with and without genetic mutations that disrupt the mGluR network. In the United States, the Center for Disease Control and Prevention estimates that 6.4 million children 4-17 years of age (11%) have been diagnosed with ADHD. Many ADHD patients remain unsatisfied with existing therapies, particularly with respect to safety, tolerability and treatment of comorbidities.

AEVI-001 is an investigational agent that has not been approved by the U.S. Food and Drug Administration or any other regulatory agencies.

About AEVI-004

AEVI-004 is an oral non-stimulant pan selective activator/modulator of mGluRs. The molecule has excellent pharmacokinetic and metabolic profiles and crosses the blood brain barrier. The molecule is a co-crystal of AEVI-001, crystallized with a pharmacologically inert conformer with a favorable toxicological profile. The molecule's pharmacological properties have been designed, and are expected, to be very similar to those of AEVI-001. As such, the Company believes that the molecule may progress directly to phase 3 studies with only minimal bridging preclinical and clinical pharmacological studies.

AEVI-004 has several distinct advantages over AEVI-001, including better stability and better manufacturability owing to a significantly higher melting point.

AEVI-004 is an investigational agent that has not been approved by the U.S. Food and Drug Administration or any other regulatory agencies.

About Aevi Genomic Medicine, Inc.

Aevi Genomic 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).