Aevi Genomic Medicine, Inc. (NASDAQ: GNMX) (the Company) today announced the sale of 5.1 million shares of its common stock for aggregate proceeds of $4.9 million, before expenses. The sales were through the Company's existing ATM program, for which JMP Securities LLC acts as the Company's sales agent.

• The Company recently announced the completion of enrollment in Part A of the ASCEND trial and intends to use the net proceeds raised from the ATM to accelerate Part B of the ASCEND trial.
• Enrollment in Part B of the ASCEND trial has been initiated and will study patients without mGluR mutations.
• The Company has approximately 1,500 mGluR mutation negative patients in the ASCEND database from which it can enroll patients for Part B of the study.
• Pooled analysis of Part A and Part B of the ASCEND trial will also be conducted, if appropriate. A pooled analysis may provide greater power and enhance the ability to detect a smaller treatment effect.
• The Company currently anticipates top-line data for Parts A and B of the ASCEND trial during the fourth quarter of 2018.

"We are pleased to be in a position to accelerate Part B of the ASCEND trial. AEVI-004 increases the importance and potential value of Part B of the ASCEND trial as it allows for long patent protection for treating all ADHD patients. mGluR mutation negative patients make up approximately 75-80% of the approximately $11 billion ADHD market," said Mike Cola, CEO of the Company.

The shares of common stock sold through the Company's ATM program have been issued and sold pursuant to the Company's shelf registration statement on Form S-3 (File No. 333-209737), previously filed with the Securities and Exchange Commission ("SEC") on February 26, 2016 and declared effective by the SEC on April 1, 2016. A prospectus supplement related to the offering was filed with the SEC on May 15, 2018. This press release shall not constitute an offer to sell or the solicitation of an offer to buy shares of the common stock nor shall there be any sale of the common stock in any state in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state.

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 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).