- Top-line data for Parts A and B of ASCEND expected in January 2019
PHILADELPHIA/PRNewswire/ -- 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.
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.
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.
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, status and timing of clinical trials, its development and business strategy, its product candidates and the plans and objectives of management for future operations and future financings. 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.
Aevi Genomic Medicine, Inc.