Aptinyx Inc. (Nasdaq: APTX), a clinical-stage biopharmaceutical
company developing transformative therapies for the treatment of
brain and nervous system disorders, today announced positive
top-line results from a 23-patient, single-blind, sequential design
Phase 2 study of its novel NMDA receptor modulator, NYX-2925, in
patients with fibromyalgia. Administration of NYX-2925 resulted in
statistically significant effects on the primary endpoint, brain
activity biomarkers associated with central pain processing,
measured using advanced imaging techniques. Statistically
significant and clinically meaningful improvements were also
observed on secondary patient-reported endpoints, including pain
scores, the Revised Fibromyalgia Impact Questionnaire (FIQR), and
other fibromyalgia symptom scales. The brain activity biomarkers
and improvements in patient-reported pain were correlated. Based on
these results, the company will begin enrollment in a larger,
12-week, randomized, placebo-controlled study in patients with
fibromyalgia in the second half of 2019, which will evaluate
patient-reported outcomes as the primary endpoint.
“The statistically significant effects on both pain-related
brain activity and patient-reported clinical measures elegantly
demonstrates that NYX-2925 is acting in the brain to alter pain
processing, leading to pain alleviation,” said Norbert Riedel,
Ph.D., president and chief executive officer of Aptinyx. “The
results of this study reinforce what we observed in patients with
advanced painful diabetic peripheral neuropathy in our recent Phase
2 DPN study, in which NYX-2925 greatly alleviated the centralized
pain that is predominant in these patients. The consistency of
these data confirms our confidence in advancing NYX-2925 as a
treatment for chronic pain. We look forward to initiating follow-up
studies in both painful DPN and fibromyalgia later this year.”
Summary of Top-line Study Results
Primary Endpoint Findings – Neuroimaging BiomarkersFibromyalgia
is associated with increased overall levels of glutamate/glutamine
(Glx) in certain brain regions and studies have shown a correlation
between pain severity and these higher Glx levels. Placebo
administration did not result in significant differences from
baseline in any of the neuroimaging biomarkers. Compared to
placebo, administration of NYX-2925 resulted in statistically
significant reductions of Glx levels in these key pain-regulating
brain regions, including the dorsal anterior cingulate cortex at
rest (p<0.05) and the posterior insular cortex following an
evoked pain stimulus (p<0.05). This Glx reduction in the
posterior insular cortex correlated with reductions in clinical
pain (p<0.05). NYX-2925 administration also resulted in reduced
connectivity between brain regions that are known to be associated
with the processing of centralized chronic pain.
Secondary Endpoint Findings – Patient-Reported
OutcomesSignificant clinical improvements on key symptoms of
fibromyalgia were observed following treatment with NYX-2925 (week
6) compared to baseline (week 0) and placebo (week 2). NYX-2925
resulted in statistically significant improvements across multiple
patient-reported clinical measures, including:
- Average daily pain score: 1.09-point reduction from baseline
(p=0.0027) and 0.66-point reduction vs. placebo (p=0.0072) on a
scale ranging from 0 to 10
- Worst daily pain score: 0.98-point reduction from baseline
(p=0.0169) and 0.61-point reduction vs. placebo (p=0.0360) on a
scale ranging from 0 to 10
- Total FIQR score: 9.6-point reduction from baseline (p=0.0085)
and 6.3-point reduction vs. placebo (p=0.0135) on a scale ranging
from 0 to 100
- PROMISFM Fatigue Profile total score: 5.4-point reduction from
baseline (p=0.0081) and 5.6-point reduction vs. placebo (p=0.0049)
on a scale ranging from 16 to 80
In addition, trends of improvement were observed on the other
clinical measures evaluated in the study. Together, the effects on
the clinical measures demonstrate improvements across a broad range
of fibromyalgia symptoms with NYX-2925.
Across all patients in the study, NYX-2925 was well tolerated
with no serious adverse events reported.
“The results with NYX-2925 are compelling and compare very
favorably with the effects of approved fibromyalgia drug treatments
we previously evaluated in separate and similar studies,” said
Daniel Clauw, M.D., professor of anesthesiology, medicine
(rheumatology), and psychiatry at the University of Michigan and an
investigator in the study. “The activity demonstrated by NYX-2925
on these pre-specified imaging markers indicates that it is acting
in a manner that is relevant in addressing the type of centralized
pain evident in patients with fibromyalgia, as well as other
chronic pain disorders. It is notable that, despite the small
number of patients in the study, the effects on these clinical
assessments of pain and other symptoms were also statistically
significant.”
Lesley Arnold, M.D., professor of psychiatry and behavioral
neuroscience at the University of Cincinnati and an investigator in
the study, commented, “Knowing the challenges these patients face
and the limited therapeutic options, it is very encouraging to see
these results on both imaging and clinical measures. The
therapeutic potential indicated by these findings highlights the
importance of advancing NYX-2925 in development as a therapy for
patients suffering from fibromyalgia. These data meaningfully
inform further clinical development, and I look forward to working
with Aptinyx as the team moves into its next Phase 2 study.”
The company plans to submit the detailed results from this study
for publication and presentation at future scientific and medical
meetings.
Conference Call InformationAptinyx will host a
conference call and webcast today at 8:30 a.m. ET to discuss the
top-line results from the study. The conference call can be
accessed by dialing 866-930-5579 (domestic) or +1-409-216-0606
(international) and using conference ID 3093727. A live webcast of
the call will be available on the Investors & Media section of
Aptinyx’s website at https://ir.aptinyx.com. The archived webcast
will be available approximately two hours after the conference call
and for 30 days thereafter.
About the Phase 2 Fibromyalgia StudyThe study
was a single-blind, placebo-controlled study to assess the efficacy
and safety of daily oral administration of NYX-2925 in 23 female
patients with a confirmed diagnosis of fibromyalgia (NCT03249103).
In a sequential manner, but blinded to the patient, all patients
received daily doses of placebo, 20 mg NYX-2925, and 200 mg
NYX-2925 for two weeks each. At baseline and during each two-week
treatment period, patients underwent a series of functional
magnetic resonance imaging (fMRI) scans, combined with proton
magnetic resonance spectroscopy (1H-MRS), to measure key brain
activity and neurochemistry biomarkers known to be associated with
perception and processing of centralized chronic pain. The study’s
primary endpoint was the evaluation of changes in these specific
biomarkers. Imaging data analysis was performed by analysts who
were blinded to treatment sequence. Secondary endpoints included
several patient-reported assessments to evaluate the effects of
NYX-2925 on fibromyalgia symptoms. These patient-reported outcomes
included average daily pain and worst daily pain measured using the
10-point Numeric Rating Scale (NRS), the impact of patients’
fibromyalgia on daily living measured by the Revised Fibromyalgia
Impact Questionnaire (FIQR), scores on the Patient Reported
Outcomes Measurement Information System Fibromyalgia (PROMISFM)
scale, pain severity and impact on functioning measured by the
Brief Pain Inventory (BPI), mood and anxiety measured by the
Hospital Anxiety and Depression Scale (HADS), and cognitive
impairment measured using the Multidimensional Inventory of
Subjective Cognitive Impairment (MISCI).
At baseline, patients in the study had a mean average daily pain
score of 5.3 on the NRS, which has a range from 0 to 10 (where 0 =
no pain and 10 = worst pain imaginable) and had a mean baseline
total FIQR score of 54.4 (this scale has a maximum score of 100,
with higher scores indicating worse fibromyalgia). Based on these
scores, the patients in the study were considered to have
moderate-to-severe fibromyalgia.
About FibromyalgiaFibromyalgia is a chronic
condition associated with widespread pain and tenderness, as well
as general fatigue. Fibromyalgia is considered by many to be a
condition that is largely mediated in the central nervous system,
given that fibromyalgia sufferers often present without a direct
peripheral insult or injury. People suffering from fibromyalgia
also often experience sleep disruption, depressed mood, and
cognitive impairment. It is estimated that, in the United States,
fibromyalgia affects more than 5 million people. Currently, there
are only three FDA-approved pharmacologic treatments for
fibromyalgia, but they have limited efficacy and burdensome side
effects in many patients.
About NYX-2925NYX-2925 is a novel oral NMDA
receptor modulator currently in Phase 2 clinical development for
the treatment of chronic pain. In clinical studies, NYX-2925 has
been shown to have activity that affects central pain processing,
resulting in alleviation of pain and other symptoms associated with
chronic pain conditions. In preclinical models of numerous
neuropathic pain conditions, NYX-2925 has shown robust activity
with a favorable tolerability profile. In Phase 1 and Phase 2
clinical studies, NYX-2925 has exhibited a favorable safety and
tolerability profile across a wide dose range. The U.S. Food and
Drug Administration has granted Fast Track designation to Aptinyx’s
development of NYX-2925 for the treatment of neuropathic pain
associated with DPN.
About AptinyxAptinyx 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 StatementsStatements 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-2925,
therapeutic effects of the company’s product candidates,
expectations regarding the design, implementation, timing, and
success of its current and planned clinical studies, and
expectations regarding its uses and sufficiency of capital. Risks
that contribute to the uncertain nature of the forward-looking
statements include: 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; positive
results from a clinical study may not necessarily be predictive of
the results of future or ongoing clinical studies; regulatory
developments in the United States and foreign countries; 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.
Investor & Media Contact:Nick SmithAptinyx
Inc.ir@aptinyx.com or corporate@aptinyx.com847-871-0377
Source: Aptinyx Inc.
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