Clinically meaningful and statistically
significant improvement on CAPS-5 Arousal and Reactivity Score
observed with NYX-783
Clinically meaningful improvement from baseline
on CAPS-5 Total Score observed in 50 mg dose arm
Statistically significant separation of 50 mg
from placebo achieved on multiple measures of responder rate on
CAPS-5 Total Score
Observed safety and tolerability similar to
placebo
Data support discussion with FDA and
advancement into pivotal study
Conference call and webcast presentation
tomorrow at 8:30 a.m. ET
Aptinyx Inc. (Nasdaq: APTX), a clinical-stage biopharmaceutical
company developing transformative therapies for the treatment of
brain and nervous system disorders, today announced positive
results from the first Phase 2 study of its novel NMDA receptor
modulator, NYX-783, in 153 patients with post-traumatic stress
disorder (PTSD). In the Phase 2 study, NYX-783 demonstrated
statistically significant and clinically meaningful efficacy
results and a favorable adverse event and tolerability profile.
Based on these results, the company expects to initiate a pivotal
study in 2021.
“Post-traumatic stress disorder is one of the most complex and
difficult-to-treat psychiatric conditions due to a host of
debilitating symptoms,” said Murray Stein, MD, MPH, FRCPC,
Distinguished Professor of Psychiatry and Public Health and Vice
Chair for Clinical Research in Psychiatry at the University of
California San Diego and a consultant to Aptinyx. “It is
encouraging to see such positive effects with NYX-783 in this
study, especially given the relatively short duration of treatment.
Although few drugs have shown efficacy in PTSD, this study of
NYX-783 has demonstrated preliminary evidence of clinically
meaningful effect along with excellent tolerability. These data
position NYX-783 as a promising therapeutic candidate moving into
further clinical development, which is welcomed news for the
underserved patients currently living with PTSD.”
The primary objective of the first-in-patient study was achieved
as both dose levels studied (10 mg and 50 mg once daily)
demonstrated clinically meaningful and statistically significant
improvement on the Clinician-Administered PTSD Scale for DSM-5
(CAPS-5) Arousal and Reactivity Score (p=0.040 and p=0.049 on 50 mg
and 10 mg vs. placebo, respectively). This scale evaluates symptoms
of PTSD such as hypervigilance, exaggerated startle response,
irritability and aggression, reckless or self-destructive
behaviors, and concentration and sleep disturbances. The resolution
of such symptoms is highly relevant to the mechanism of action of
NYX-783, which has been shown to enhance extinction learning.
Clinically meaningful improvement was also observed in the
CAPS-5 Total Score within just four weeks in the 50 mg dose arm,
which trended toward significance. In the intention-to-treat (ITT)
population that completed Stage 1 at week four, 78% of subjects
taking NYX-783 50 mg achieved a 30% improvement from baseline in
the CAPS-5 Total Score, compared to 44% of subjects taking placebo
(p=0.008). In the same population and time period, 50% of subjects
taking NYX-783 50 mg achieved a 50% CAPS-5 Total Score improvement
from baseline, compared to 26% of subjects taking placebo
(p=0.044).
The improvements on the primary, clinician-administered CAPS-5
endpoints were concordant with improvements observed on multiple
secondary and exploratory endpoints.
Across endpoints in the study, a clear dose response was evident
with the 50 mg dose demonstrating more consistent effects than the
10 mg dose.
In the study, NYX-783 was well tolerated and exhibited a
favorable adverse event profile.
“We are very pleased with these impressive results, which
surpassed our expectations for this initial exploratory study and
clearly indicate the strong potential of NYX-783 to rapidly and
reliably address some of the most challenging symptoms of PTSD,”
said Norbert Riedel, Ph.D., president and chief executive officer
of Aptinyx. “People suffering from PTSD have immense unmet medical
needs and the few existing therapeutic options offer limited
benefit. We believe these results indicate that the mechanism of
NYX-783, which modulates NMDA receptors to enhance extinction
learning, addresses the putative underlying pathology of PTSD. We
expect the data from this study will support discussion with the
FDA and the initiation of a first pivotal study. In addition, these
results provide further clinical validation of our NMDA receptor
modulation platform and reinforce our confidence in its therapeutic
utility across our other clinical-stage programs in chronic pain
and cognition.”
Aptinyx plans to discuss these results on a conference call
scheduled for 8:30 a.m. ET tomorrow. Additionally, the company
plans to submit the detailed results from this study for
publication and presentation at future scientific and medical
meetings.
About the Phase 2 Exploratory Study of
NYX-783 in PTSD
The Phase 2 exploratory study was a multi-center,
placebo-controlled, double-blind, randomized, Sequential Parallel
Comparison Design (SPCD) study of 153 patients with PTSD, as
characterized by criteria set forth in the Diagnostic and
Statistical Manual of Mental Disorders, 5th Edition (DSM-5).
In accordance with the SPCD design, the study was conducted in
two, four-week sequential stages. No patient in the study received
more than 4 weeks of NYX-783 treatment. Patients were randomly
assigned to receive placebo, NYX-783 10 mg, or NYX-783 50 mg in
Stage 1. Patients on placebo in Stage 1 were re-randomized in Stage
2 to again receive placebo, or start NYX-783 10 mg, or NYX-783 50
mg. All patients from Stage 1 as well as the patients in the
placebo non-responder group at week 4 (defined as CAPS-5 total
score >26 and a percent-reduction from baseline in CAPS-5 total
score of <35% at week 4), in Stage 2 were combined for primary
efficacy analysis. The primary endpoint of the study was the change
in CAPS-5 Total Score and symptom domain sub-scores (Arousal and
Reactivity, Negative Cognitions and Mood, Intrusions, and
Avoidance) for Stage 1 and Stage 2, combined as weighted effects.
In total, the Phase 2 study duration was ten to thirteen weeks
overall, consisting of a screening period, two four-week treatment
periods (Stage 1 and Stage 2), and a safety follow-up period.
Conference Call
Information
To access the live conference call and webcast presentation,
please dial (866) 939-3921 (U.S. Toll Free) or (678) 302-3550 (U.S.
Toll) and refer to conference ID 49992933. A live audio and webcast
presentation will be available on the Investors & Media section
of Aptinyx’s website at https://ir.aptinyx.com. A replay of the
webcast will be archived on Aptinyx’s website for 30 days following
the event.
About Post-Traumatic Stress Disorder
Approximately eight and a half million people in the United
States suffer from PTSD, which is characterized by intrusive
symptoms, avoidance, negative alteration in cognition and mood,
hyperarousal, and/or arousal alterations following the experience
of trauma. PTSD can result from various forms of trauma, including
combat exposure, car accidents, sexual or other physical assault,
abuse, natural disasters, and others. The lifetime prevalence of
PTSD is approximately seven percent in the general population but
is much higher in populations at risk for exposure to trauma, such
as military service members and first responders. In addition to
the challenges associated with the direct symptoms, PTSD sufferers
have a higher rate of suicide and often struggle with simultaneous
addiction, leading to an even greater social and economic burden of
the disorder. Available therapeutic options are limited, including
only two approved conventional SSRI antidepressants, which have
limited efficacy, undesirable side effects, and target only the
symptoms of PTSD, not the underlying disorder itself.
About NYX-783
NYX-783 is a novel, oral NMDA receptor modulator currently in
Phase 2 development for the treatment of post-traumatic stress
disorder (PTSD). In preclinical studies of NYX-783, particularly
strong results were observed in psychiatric models, models of fear
extinction, and models of substance abuse. In a Phase 1 clinical
study of NYX-783, ample central nervous system exposure was
observed and the product candidate demonstrated a favorable adverse
event and tolerability profile, with no serious adverse effects,
across a wide dose range. The U.S. Food and Drug Administration has
granted Fast Track designation to the development of NYX-783 for
the treatment of PTSD.
About Aptinyx
Aptinyx Inc. is a clinical-stage biopharmaceutical company
focused on the discovery, development, and commercialization of
proprietary synthetic small molecules for the treatment of brain
and nervous system disorders. Aptinyx has a platform for discovery
of novel compounds that work through a unique mechanism to
modulate—rather than block or over-activate—NMDA receptors and
enhance synaptic plasticity, the foundation of neural cell
communication. The company has three product candidates in clinical
development in central nervous system indications, including
chronic pain, post-traumatic stress disorder, and cognitive
impairment associated with Parkinson’s disease. Aptinyx is also
advancing additional compounds from its proprietary discovery
platform, which continues to generate a rich and diverse pipeline
of small-molecule NMDA receptor modulators with the potential to
treat an array of neurologic disorders. For more information, visit
www.aptinyx.com.
Forward-Looking Statements
Statements contained in this press release regarding matters
that are not historical facts are “forward-looking statements”
within the meaning of the Private Securities Litigation Reform Act
of 1995. Because such statements are subject to risks and
uncertainties, actual results may differ materially from those
expressed or implied by such forward-looking statements. Such
statements include, but are not limited to, statements regarding
the company’s business plans and objectives, including future plans
or expectations for NYX-783 and potential therapeutic effects of
NYX-783, the timing and outcome of discussions with FDA and other
regulatory agencies, expectations regarding the design,
implementation, timing, and success of its future clinical studies
of NYX-783, including whether they are pivotal or would support
registration, and expectations regarding its other NMDA receptor
modulation platform development activities. Risks that contribute
to the uncertain nature of the forward-looking statements include:
the effect of COVID-19 on our business and financial results,
including with respect to disruptions to our clinical trials,
business operations, and ability to raise additional capital; the
success, cost, and timing of the company’s product candidate
development activities and planned clinical studies; the company’s
ability to execute on its strategy; that positive results from a
clinical study may not necessarily be predictive of the results of
future or ongoing clinical studies; future clinical studies may
fail to demonstrate adequate safety and efficacy of our product
candidates, which would prevent, delay, or limit the scope of
regulatory approval and commercialization; regulatory approval
processes of the FDA and comparable foreign regulatory authorities
are lengthy, time‑consuming, and inherently unpredictable;
regulatory developments in the United States and foreign countries;
the company’s estimates regarding expenses, future revenue, and
capital requirements; as well as those risks and uncertainties set
forth in the company’s most recent annual report on Form 10-K and
subsequent filings with the Securities and Exchange Commission. All
forward-looking statements contained in this press release speak
only as of the date on which they were made. Aptinyx undertakes no
obligation to update such statements to reflect events that occur
or circumstances that exist after the date on which they were
made.
Source: Aptinyx Inc.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20201019005852/en/
Investor & Media Contact: Nick Smith Aptinyx Inc.
ir@aptinyx.com or corporate@aptinyx.com 847-871-0377
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