PHILADELPHIA, Jan. 2, 2019 /PRNewswire/ -- Aevi Genomic
Medicine, Inc. (NASDAQ: GNMX) ("the Company") today announced that
the ASCEND trial, a genomically-guided Phase 2 double-blind,
placebo-controlled clinical trial of orally-administered AEVI-001
(100 – 400 mg BID) in children aged 6 – 17 with Attention Deficit
Hyperactivity Disorder (ADHD) with an mGluR copy number variant
(Part A) or without an mGluR copy number variant (Part B), did not
achieve statistical significance on the primary endpoint of
reduction of ADHD-RS in either Part A or Part B after 6 weeks of
treatment with AEVI-001. AEVI-001 was safe and well
tolerated. Reported adverse events were minimal and similar across
both Part A and Part B treatment groups.
"We are very disappointed that the ASCEND trial did not achieve
its primary endpoint," said Michael F.
Cola, CEO of Aevi Genomic Medicine. "We plan to
conduct a full review of the data with our scientific advisers in
the coming days and consider our options. We want to thank
the investigators, study coordinators and the entire Aevi team for
their hard work on this innovative study. We also especially
want to thank the patients and their families for their
participation. We remain committed to our mission of bringing
novel targeted therapies to children with serious rare and orphan
disease. We will continue to focus on our pipeline of
additional molecules, including AEVI-002 for severe pediatric-onset
Crohn's Disease and AEVI-005 for an undisclosed autoimmune orphan
disease, both partnered with Kyowa Hakko Kirin. In the
near-term, we anticipate reducing the scope of our operations to
preserve our net working capital, which was approximately
$5 million (unaudited) as of
December 31, 2018."
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).
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. In the US, the CDC
estimates that 6.4 million children 4-17 years of age (11%) have
ever 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 US FDA or any other regulatory agencies.
About the ASCEND Trial
The AEVI-001-ADHD-202 study (ASCEND) was a 2-part, 6-week,
double-blind, dose-optimization, parallel-group study in children
and adolescents (ages 6-17 years) with attention deficit
hyperactivity disorder (ADHD) with and without copy number variants
(CNVs) in specific genes implicated in glutamatergic signaling and
neuronal connectivity. Part A included subjects determined to
have one of 8 specific gene mutation(s) implicated in glutamatergic
signaling and neuronal connectivity. Part B included subjects
who did not have CNVs in any of the specific gene mutation(s)
implicated in glutamatergic signaling and neuronal
connectivity. Once subjects were confirmed as eligible for
each part of the study, they were randomized to one of two
treatment groups (AEVI-001 or placebo) in a 1:1 ratio.
In Part A of the study, 69 unique subjects were enrolled. In
Part B of the study, 109 unique subjects were enrolled. After
screening and washout of prior ADHD therapy, subjects entered a
dose optimization phase wherein doses were titrated to a dose up to
400 mg BID. Subjects then entered a maintenance phase with
weekly follow-up for up to 6 weeks.
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, status and timing of clinical trials, 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.
CONTACT:
Aevi Genomic Medicine, Inc.
Brian Piper
Brian.Piper@aevigenomics.com
Westwicke Partners
Chris Brinzey
339-970-2843
Chris.brinzey@westwicke.com
View original
content:http://www.prnewswire.com/news-releases/aevi-genomic-medicine-announces-top-line-results-from-placebo-controlled-ascend-trial-parts-a--b-of-aevi-001-in-children-with-adhd-300772052.html
SOURCE Aevi Genomic Medicine, Inc.