PHILADELPHIA, Aug. 2, 2018 /PRNewswire/ -- Aevi Genomic
Medicine, Inc. (NASDAQ: GNMX) (the "Company") announced today
financial and operational results for the three months ended
June 30, 2018 and provided an
overview of the Company's recent corporate progress.
Second Quarter Financial Results and Recent Corporate
Highlights
- AEVI-001 ASCEND clinical trial update: In
June 2018 the Company announced that
the sample size for Part A of the trial, which is enrolling an
8-gene subset of pediatric and adolescent mGLuR mutation positive
ADHD patients, will be increased from 42 to 64 patients. The
decision to increase the sample size was made according to a
prespecified sample size re-estimation design, which allows an
adjustment in the sample size after an interim analysis of the
placebo arm to ensure the trial is appropriately powered. The
Company anticipates data from Part A of the ASCEND trial in the
fourth quarter of 2018.
- AEVI-004 Co-crystal version of AEVI-001: During the
second quarter of 2018 the Company announced that it received
positive feedback from the U.S. Food and Drug Administration (FDA)
on an improved version of AEVI-001 (AEVI-004). AEVI-004 is a
co-crystal of fasoracetam with a higher melting point and greater
stability characteristics which has been engineered to maintain
solubility, dissolution and pharmacokinetics substantially similar
to AEVI-001, and composition of matter patents are expected through
2039. The Company has received feedback from the FDA provisionally
indicating that AEVI-004 is a co-crystal of AEVI-001 and a novel
drug substance. The FDA also provisionally indicated that existing
toxicology and pathology studies can support clinical development
with AEVI-004. Assuming positive results from the ongoing Phase 2
ASCEND clinical trial, and following minimal bridging preclinical
and clinical pharmacological studies requested by FDA, the Company
anticipates progressing the molecule directly into Phase 3 clinical
studies.
- AEVI-002: Patient screening at multiple clinical
trial sites in the United States
continues for the Company's Phase 1b
open-label, signal-finding trial which will evaluate the safety,
tolerability, pharmacokinetics and short-term efficacy of the
Anti-Light Monoclonal Antibody (AEVI-002) in severe pediatric onset
Crohn's disease patients that have previously failed anti-tumor
necrosis factor alpha (anti-TNFα) treatment. The Company has
not yet enrolled any patients into this trial. To address the
continued enrollment challenges for this program, the Company is
evaluating the addition of clinical trial sites outside the United
States. Assuming a small number of patients can be enrolled
by the end of the third quarter 2018, initial data from a small
number of patients would be expected by year-end 2018.
- AEVI-005: The Company initiated a preclinical research
program for AEVI-005 during the second quarter. AEVI-005 is
the second monoclonal antibody in development as part of the
company's ongoing collaboration with Kyowa Hakko Kirin.
AEVI-005 is being studied in an undisclosed ultra-orphan
auto-immune pediatric disease.
"We remain focused on advancing our programs in ADHD and severe
pediatric onset Crohn's disease," said Mike
Cola, Chief Executive Officer of the Company. "We look
forward to sharing data from the ASCEND trial in the fourth quarter
of this year, while expanding our efforts to enroll patients into
the Phase 1b trial of severe
pediatric onset Crohn's disease and working towards preclinical
proof-of-concept data for AEVI-005."
Second Quarter 2018 Financial Results
The Company had cash and cash equivalents of $19.2 million at June 30,
2018, compared to $33.7
million as of December 31,
2017. The decrease in cash was primarily related to the
advancement of the Company's AEVI-001 and AEVI-002 programs. Based
upon current management projections, the Company expects the
current cash balance to fund operations into early in the first
quarter of 2019.
Research and development expenses for the three months ended
June 30, 2018 were $5.8 million, approximately equivalent to
$5.7 million for the same period in
2017.
General and administrative expenses for the three months ended
June 30, 2018 were $2.5 million, approximately equivalent to
$2.4 million for the same period in
2017.
For the three months ended June 30,
2018, the Company reported a net loss of $8.2 million or $0.14 per share, compared with a net loss of
$8.0 million or $0.22 per share for the same period in 2017.
CONDENSED
CONSOLIDATED BALANCE SHEETS
|
(In thousands,
except share data)
|
|
|
|
June
30,
2018
|
|
|
December
31,
2017
|
|
|
|
Unaudited
|
|
|
Audited
|
|
ASSETS
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
CURRENT
ASSETS:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Cash and cash
equivalents
|
|
$
|
19,151
|
|
|
$
|
33,729
|
|
Prepaid expenses and
other current assets
|
|
|
855
|
|
|
|
893
|
|
|
|
|
|
|
|
|
|
|
Total current
assets
|
|
|
20,006
|
|
|
|
34,622
|
|
|
|
|
|
|
|
|
|
|
LONG-TERM
ASSETS:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Lease
deposits
|
|
|
11
|
|
|
|
11
|
|
Property and
equipment, net
|
|
|
53
|
|
|
|
85
|
|
Other long-term
assets
|
|
|
163
|
|
|
|
43
|
|
|
|
|
|
|
|
|
|
|
Total long-term
assets
|
|
|
227
|
|
|
|
139
|
|
|
|
|
|
|
|
|
|
|
Total
assets
|
|
$
|
20,233
|
|
|
$
|
34,761
|
|
|
|
|
|
|
|
|
|
|
LIABILITIES AND
STOCKHOLDERS' EQUITY
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
CURRENT
LIABILITIES:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Trade
payables
|
|
$
|
1,463
|
|
|
$
|
943
|
|
Other accounts
payable and accrued expenses
|
|
|
3,480
|
|
|
|
3,197
|
|
|
|
|
|
|
|
|
|
|
Total current
liabilities
|
|
|
4,943
|
|
|
|
4,140
|
|
|
|
|
|
|
|
|
|
|
Total
liabilities
|
|
|
4,943
|
|
|
|
4,140
|
|
|
|
|
|
|
|
|
|
|
STOCKHOLDERS'
EQUITY:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Common stock -
$0.0001 par value; 200,000,000 shares authorized; 59,340,731
shares issued and outstanding at June 30, 2018; 59,332,265 shares
issued and
outstanding at December 31, 2017
|
|
$
|
6
|
|
|
$
|
6
|
|
Additional paid-in
capital
|
|
|
247,162
|
|
|
|
245,593
|
|
Accumulated
deficit
|
|
|
(231,878)
|
|
|
|
(214,978)
|
|
|
|
|
|
|
|
|
|
|
Total stockholders'
equity
|
|
|
15,290
|
|
|
|
30,621
|
|
|
|
|
|
|
|
|
|
|
Total liabilities and
stockholders' equity
|
|
$
|
20,233
|
|
|
$
|
34,761
|
|
CONDENSED
CONSOLIDATED STATEMENTS OF OPERATIONS
|
(In thousands,
except share and per share data)
|
|
|
|
Six months
ended
June
30,
|
|
Three months
ended
June
30,
|
|
|
|
2018
|
|
|
|
2017
|
|
2018
|
|
|
2017
|
|
|
|
Unaudited
|
|
Unaudited
|
|
Research and
development expenses
|
$
|
12,308
|
|
|
$
|
13,614
|
|
$
|
5,747
|
|
|
$
|
5,667
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
General and
administrative expenses
|
|
4,678
|
|
|
|
5,357
|
|
|
2,504
|
|
|
|
2,369
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Operating
loss
|
|
(16,986)
|
|
|
|
(18,971)
|
|
|
(8,251)
|
|
|
|
(8,036)
|
|
Financial
income
|
|
86
|
|
|
|
21
|
|
|
60
|
|
|
|
3
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss
|
$
|
(16,900)
|
|
|
$
|
(18,950)
|
|
$
|
(8,191)
|
|
|
$
|
(8,033)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Basic and diluted
loss per share
|
$
|
(0.28)
|
|
|
$
|
(0.51)
|
|
$
|
(0.14)
|
|
|
$
|
(0.22)
|
|
Weighted average
number of common
stock used in
computing basic and
diluted loss per share
|
|
59,336,547
|
|
|
|
37,109,157
|
|
|
59,338,255
|
|
|
|
37,110,043
|
|
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.
Aevi Genomic Medicine 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 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 AEVI-004
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.
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).
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
MEDIA INQUIRIES:
FTI Consulting
Irma Gomez-Dib
+1212-850-5761
+1-415-706-9155
irma.gomez-dib@fticonsulting.com
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SOURCE Aevi Genomic Medicine, Inc.