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Aevi Genomic Medicine Presents Updated Results from SAGA Trial of AEVI-001 at the 6th World Congress on ADHD

PHILADELPHIA, PA -- (Marketwired) -- 04/20/17 --

-- Novel genetic mutations predictive of response to AEVI-001 found in approximately 10% of pediatric ADHD patients --

-- New results clarify a path forward for the continued development of AEVI-001 in ADHD patients and potential future development in other neurodevelopmental disorders --

Aevi Genomic Medicine, Inc. (NASDAQ: GNMX) (the Company) today announced updated responder data from a subset of genomically identified patients in the SAGA trial of AEVI-001 in adolescent Attention Deficit Hyperactivity Disorder (ADHD). The new data will be presented at the 6th World Congress on ADHD in Vancouver, Canada on April 21, 2017. Key results of the trial include:

  • Identification of nine genes that are predictive of clinically meaningful and statistically significant response [ADHD-Rating Scale (ADHD-RS) reduction of 17.6, p < .005] in the SAGA trial. These genes include certain glutamate metabotropic receptors (GRM) and neurodevelopmental genes and are found in approximately 10% of pediatric ADHD patients.(1)
  • One of the neurodevelopmental genes, contactin-4 (CNTN4) previously identified as important in Autism Spectrum Disorder (ASD), was highly enriched in the responder population (43%) and represents approximately 5% of pediatric ADHD patients.(1)
  • CNTN4 mutation phenotype is more severe with a higher prevalence of emotional dysregulation, including: anger control, risk taking, inappropriate movements and sounds.
  • All CNTN4 mutation positive patients on treatment (n=6) had clinically meaningful and statistically significant response to AEVI-001 (ADHD-RS reduction of 20.8, p=.03).

"The SAGA trial post hoc responder analysis represents a novel genomic approach to psychiatric drug development. Genetic stratification of patient subpopulations appears to identify patients with specific gene mutations who are more likely to respond to AEVI-001," said Robert L. Findling, M.D., M.B.A., Principal Investigator of the SAGA trial, Vice President of Psychiatry Services and Research at Kennedy Krieger Institute and Professor of Psychiatry and Behavioral Sciences at The Johns Hopkins University School of Medicine.

"This is an exciting and novel finding in ADHD, that CNTN4 mutations have a meaningful prevalence in ADHD and are highly associated with a response to AEVI-001. CNTN4 mutations and deletions have been previously associated with neurodevelopmental delay, ASD, and other neuropsychiatric conditions. ADHD and ASD are frequently co-morbid with each other, and these new data suggest that CNTN4 mutations may be a common genetic link for these diseases. I look forward to working with the Company to study this further in children with mutation positive ADHD as well as the potential to study AEVI-001 in patients with ASD."

The recent analysis demonstrates that of the 42 patients with a mutation in one of nine genes (n=18 patients on treatment, n=24 patients on placebo), 89% of patients on treatment (n=16) had a clinically meaningful and statistically significant response to AEVI-001 in the SAGA trial (defined as a 30% or greater reduction in ADHD-RS score from baseline), vs 21% on placebo (p < .0001). Patients on treatment had a reduction in ADHD-RS of 17.6 versus 5.9 on placebo (p < .005). This subset of patients had mutations in the CNTN4 gene as well as certain GRM and neurodevelopmental genes.

More specifically, 18 patients with copy-number variation (CNV) mutations in CNTN4 were enrolled in the SAGA trial (six patients on treatment and 12 patients on placebo). 100% of CNTN4 positive patients (n=6) treated with AEVI-001 had a reduction in ADHD-RS of > 30% vs. 25% of placebo patients (p=.0027). The magnitude of response of CNTN4 positive patients treated with AEVI-001 was a 20.8 point reduction in ADHD-RS vs. an 8.9 point reduction in ADHD-RS in patients on placebo (p=.03).

"The robust response of CNTN4 mutation-positive ADHD adolescents to AEVI-001 in the SAGA trial suggests that CNTN4 associated ASD may be successfully treated with AEVI-001," said Garry A. Neil, M.D., Chief Scientific Officer, Aevi Genomic Medicine. "Based on these new findings, we are validating the prevalence of CNTN4 mutations in ASD and will further characterize the phenotypes associated with this mutation. Importantly, the Company intends to continue the development of AEVI-001 in ADHD and ASD, as well as other potential neuropsychiatric indications in genomically defined populations based on the responder analysis of the SAGA trial."

Based on the Company's study "Glutamatergic Network Gene Mutations in Children and Adolescents with ADHD (Phenotype/Genotype study)," approximately 10% of ADHD patients bear a CNV mutation in one of the nine genes. As found in the SAGA trial, the most prevalent gene in this phenotype-genotype study was CNTN4, with an approximate prevalence in ADHD of nearly 5%.

Patients bearing mutations in CNTN4 exhibited a unique and more severe ADHD phenotype. Compared to other ADHD patients, they had significantly higher rates of parent-reported behaviors suggestive of emotional dysregulation, including: disruptive behavior, anger control, risk-taking, and inappropriate movements and sounds.

The Company will present two posters at the World Congress on ADHD:

Title: Double-blind placebo-controlled study of the novel therapeutic AEVI-001 in adolescents with ADHD and glutamatergic network gene mutations in children and adolescents with ADHD (SAGA Trial)
Presenter: Garry A. Neil, M.D., Chief Scientific Officer, Aevi Genomic Medicine, Inc.
Date: Friday, April 21, 2017, 14:30-16:00 PDT
Session: Pharmacological treatment of children and adolescents II
Abstract: P-17-001

Title: Glutamatergic network gene mutations in children and adolescents with ADHD (Phenotype/Genotype Study)
Liza A. Squires, M.D., Vice President, Head of Neuroscience, Aevi Genomic Medicine, Inc.
Date: Friday, April 21, 2017, 14:30-16:00 PDT
Session: Aetiology II
Abstract: P-02-006

The posters are available on the Company website at www.aevigenomics.com.

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.

Aevi Genomic Medicine remains committed to the development of 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. The Company intends to develop AEVI-001 for the treatment of autism spectrum disorder (ASD) patients with genomically identifiable mutations in the mGluR network. In the US, the CDC estimates that 6.4 million children 4-17 years of age (11%) have ever been diagnosed with ADHD(2). Many ADHD patients remain unsatisfied with existing therapies, particularly with respect to safety, tolerability and treatment of comorbidities. In the US in 2012, 1 in 68 children were diagnosed with ASD (2012) increasing from 1 in 150 (2000)(3). There is a high unmet need for pharmaceutical treatments for ASD as currently approved medications are indicated for symptoms of irritability in ASD patients.

AEVI-001 is an investigational agent that has not been approved by the US FDA or any other regulatory agencies.

About the SAGA Trial
SAGA was a multicenter, dose-optimized trial in adolescents with ADHD. The trial was designed as a randomized, double-blind, placebo-controlled, parallel-group study of AEVI-001 versus placebo in adolescent patients with ADHD who have genetic disorders impacting the mGluR network. The trial enrolled 101 patients (96 evaluable) between the ages of 12-17 years old. The primary and secondary endpoints in the trial were the change from baseline in the ADHD-Rating Scale Version 5 (ADHD-RS-5) Total Score and the percentage of subjects who responded as determined by the Clinical Global Impression of Improvement (CGI-I), respectively. Patients were randomized 1:1 to receive either a six-week course of AEVI-001 or placebo, with a one-week follow-up. Patients were enrolled from sites that participated in the recent phenotype/genotype study. More information on the SAGA trial is available at www.ClinicalTrials.gov (Identifier: NCT02777931).

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 at The Children's Hospital of Philadelphia.

The Company is also progressing its second program, AEVI-002, into clinical development for Severe Pediatric Onset Crohn's Disease.

More information on the Company and pipeline is located on its website www.aevigenomics.com

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.

(1)Data on file and Elia, J. et al, Glutamatergic network gene mutations in children and adolescents with ADHD (Phenotype/Genotype Study). Poster presented at: 6th World Congress on ADHD; April 21, 2017; Vancouver, Canada


Aevi Genomic Medicine, Inc.
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Westwicke Partners
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FTI Consulting
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