PHILADELPHIA, PA--(Marketwired - Jan 31, 2017) - 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 22nd 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 (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).

Forward-looking Statements

This release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, Section 21E of the Securities Exchange Act of 1934 and as that term is defined in the Private Securities Litigation Reform Act of 1995, which include all statements other than statements of historical fact, including (without limitation) those regarding the Company's financial position, its development and business strategy, its product candidates and the plans and objectives of management for future operations. The Company intends that such forward-looking statements be subject to the safe harbors created by such laws. Forward-looking statements are sometimes identified by their use of the terms and phrases such as "estimate," "project," "intend," "forecast," "anticipate," "plan," "planning, "expect," "believe," "will," "will likely," "should," "could," "would," "may" or the negative of such terms and other comparable terminology. All such forward-looking statements are based on current expectations and are subject to risks and uncertainties. Should any of these risks or uncertainties materialize, or should any of the Company's assumptions prove incorrect, actual results may differ materially from those included within these forward-looking statements. Accordingly, no undue reliance should be placed on these forward-looking statements, which speak only as of the date made. The Company expressly disclaims any obligation or undertaking to disseminate any updates or revisions to any forward-looking statements contained herein to reflect any change in the Company's expectations with regard thereto or any change in events, conditions or circumstances on which any such statements are based. As a result of these factors, the events described in the forward-looking statements contained in this release may not occur.

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