Phase 2 studies in fibromyalgia and painful DPN
enrolling as planned – data readouts expected in 1H 2022
Phase 2 study in cognitive impairment expected
to recommence imminently – data readout expected in 2H 2022
Reported data from Phase 2 exploratory study of
NYX-783 in PTSD, supporting further clinical development – meeting
with FDA to discuss future development path April 29, 2021
Strong cash position expected to support
operations through multiple clinical data readouts and into
2023
Conference call today at 5:00 p.m. EST
Aptinyx Inc. (Nasdaq: APTX), a clinical-stage biopharmaceutical
company developing transformative therapies for the treatment of
brain and nervous system disorders, today reported financial
results for the fourth quarter and full year 2020 and provided
updates across the company’s pipeline of novel NMDA receptor
modulators.
“Despite the COVID-19 pandemic, we are excited to be advancing
clinical development across all four of our pipeline programs,”
said Norbert Riedel, Ph.D., chief executive officer of Aptinyx.
“The recent and upcoming recommencements of key clinical studies
are a testament to the dedication of our team at Aptinyx, and I am
grateful to all of my colleagues who have been instrumental in
these efforts.”
Dr. Riedel continued, “2020 was a very important year in further
validating the novel mechanism through which our NMDA receptor
modulators act. The results from our completed PTSD study enhance
our understanding of the neurobiology of PTSD and the potential for
NYX-783 to be a significant advancement in the field. We have
observed the therapeutically relevant activity of our compounds
across numerous studies, both in patients and in healthy
volunteers. We believe the foundational data we have collected
across our pipeline thus far have informed and de-risked the
multiple Phase 2 studies that we expect to readout in the next
12-24 months. All of this progress sets us up nicely to bring much
needed therapeutic innovation to multiple challenging CNS disorders
in the years to come. And, with these important clinical milestones
ahead, we are pleased to be in a strong financial position, with a
cash balance that we anticipate will support our planned operations
into 2023.”
Pipeline Updates
Chronic Pain Pipeline – NYX-2925
Aptinyx is developing NYX-2925 for the treatment of chronic
centralized pain conditions. To date, NYX-2925 has been studied in
more than 400 human subjects, including in three Phase 1 studies in
healthy volunteers and two completed Phase 2 studies in patients
with chronic pain. Across all studies, NYX-2925 has been well
tolerated with no drug-related serious adverse events reported.
- Development in fibromyalgia: Following the completion of
a successful Phase 2a neuroimaging biomarker study in 2019, Aptinyx
initiated a Phase 2b study to evaluate NYX-2925 in patients with
fibromyalgia. In March 2020, Aptinyx temporarily suspended
enrollment of new patients in the Phase 2b study due to the
COVID-19 pandemic.
- In September 2020, Aptinyx recommenced enrollment in the Phase
2b study with targeted protocol amendments designed to streamline
enrollment and ensure safety of patients and study personnel amidst
the ongoing COVID-19 pandemic.
- Study design:
- The study is a randomized, double-blind, parallel-design,
placebo-controlled study to evaluate once-daily oral dosing of two
dose levels of NYX-2925 (50 mg and 100 mg) compared to placebo in
approximately 300 patients with fibromyalgia.
- The primary endpoint is the change in average daily pain score
over a 12-week treatment period as reported on the 10-point numeric
rating scale (NRS).
- Multiple secondary endpoints will be evaluated to characterize
the effects of NYX-2925 across the broad array of symptoms
experienced by patients with fibromyalgia.
- Enrollment to date has been consistent with the company’s plan
and Aptinyx anticipates reporting top-line data from the study in
the first half of 2022.
- Development in painful diabetic peripheral neuropathy
(DPN): Following the completion of a Phase 2 study in 2019, in
which NYX-2925 exhibited the strongest activity in patients with
advanced DPN, Aptinyx initiated a Phase 2b study to support
findings from the prior study. In March 2020, Aptinyx temporarily
suspended enrollment of new patients in the Phase 2b study due to
the COVID-19 pandemic.
- In December 2020, Aptinyx recommenced the Phase 2b study with
targeted protocol amendments designed to streamline enrollment and
ensure safety of patients and study personnel amidst the ongoing
COVID-19 pandemic.
- Study design:
- The study is a randomized, double-blind, parallel-design,
placebo-controlled study to evaluate once-daily oral dosing of 50
mg of NYX-2925 compared to placebo in approximately 200 patients
with advanced DPN.
- The primary endpoint is the change in average daily pain score
over a 12-week treatment period as reported on the 10-point
NRS.
- Multiple secondary endpoints will be evaluated to characterize
the effects of NYX-2925 across symptoms experienced by patients
with painful DPN.
- Enrollment to date has been consistent with the company’s plan
and Aptinyx anticipates reporting top-line data from the study in
the first half of 2022.
- Publication of review article in Medicine in Drug Discovery
highlighting preclinical data supporting the potential of NYX-2925
to treat chronic, centralized pain conditions: In January 2021,
Aptinyx announced the publication of a recent review article in
Medicine in Drug Discovery featuring data on its investigational
novel NMDA receptor modulator, NYX-2925. The data span across
numerous preclinical models and highlight the potential therapeutic
benefits of NYX-2925 in treating chronic, centralized pain
conditions.
Psychiatry Pipeline – NYX-783
Aptinyx is developing NYX-783 for the treatment of PTSD. Across
the completed Phase 1 and Phase 2 studies, including more than 200
human subjects combined, NYX-783 was well tolerated with no
drug-related serious adverse events reported.
- Development in PTSD: In October 2020, Aptinyx reported
results from a Phase 2a exploratory study of NYX-783 in patients
with PTSD.
- The study was a double-blind, placebo-controlled, sequential
parallel comparison design (SPCD) study in approximately 160
patients with PTSD. The study consisted of two four-week treatment
stages, in which patients were randomly assigned to receive
once-daily oral doses of either placebo, 10 mg NYX-783, or 50 mg
NYX-783.
- This was an initial exploratory study and thus was powered
based on clinical, and not statistical, considerations to detect
signals of efficacy. Nonetheless, statistically significant
separation from placebo was observed on some measures.
- Results:
- Clinically meaningful improvements from baseline were observed
on the CAPS-5 Total score within four weeks in the 50 mg dose arm.
- Compared to placebo, a significantly greater proportion of
patients on NYX-783 50 mg achieved a clinically meaningful
improvement in CAPS-5 Total score in Stage 1 of treatment.
- Statistically significant improvement was observed on CAPS-5
Arousal and Reactivity score.
- Consistent improvements were observed across three of the four
CAPS-5 symptom cluster scores, with statistically significant
improvements compared to placebo observed on two of the four
symptom clusters in Stage 1.
- Next steps:
- Aptinyx is scheduled to hold a Type C meeting with the U.S.
Food and Drug Administration (FDA) on April 29, 2021 to discuss the
future development path for NYX-783 in PTSD.
- Aptinyx expects to initiate a Phase 2b study in the second half
of 2021, with a design consistent with that of a
registration-supportive study and focused on evaluating effects on
the CAPS-5 Total score.
- Data from the Phase 2a exploratory study will be presented at
the 2021 Society of Biological Psychiatry (SOBP) Annual Meeting to
be held virtually on April 29 – May 1, 2021.
Neurology Pipeline – NYX-458
Aptinyx is developing NYX-458 for the treatment of cognitive
impairment. In a Phase 1 study conducted in 62 healthy volunteers,
NYX-458 exhibited a favorable pharmacokinetic profile and was well
tolerated with no drug-related serious adverse events reported.
- Development in cognitive impairment associated with
Parkinson’s disease and dementia with Lewy bodies: In December
2019, based on positive preclinical data, including those from a
highly translatable model in non-human primates, Aptinyx initiated
a Phase 2a exploratory study of NYX-458 in patients with mild
cognitive impairment associated with Parkinson’s disease. In March
2020, Aptinyx temporarily suspended enrollment of new patients in
the Phase 2 study due to the COVID-19 pandemic.
- Following the suspension of enrollment, Aptinyx incorporated
certain study design changes to improve study feasibility amidst
the COVID-19 pandemic, simplify study execution, and optimize the
potential for the study to detect signals of efficacy.
- Phase 2a exploratory study design:
- The Phase 2 study is a randomized, double-blind,
parallel-design, placebo-controlled study to evaluate the safety
and potential cognitive benefits of NYX-458 in patients with
cognitive impairment associated with Parkinson’s disease or
dementia with Lewy bodies.
- Patients with dementia with Lewy bodies have been included in
consideration of the pathophysiology in common with Parkinson’s
cognitive impairment – believed to be caused by increased levels of
alpha synuclein, which, in turn, decreases NMDA receptor expression
and activity.
- The study will evaluate daily oral dosing of NYX-458 30 mg
compared to placebo over a 12-week period.
- The study is expected to enroll approximately 100
patients.
- The primary endpoint in the study is safety and
tolerability.
- Secondary endpoints include multiple computerized
neurocognitive assessments to evaluate the effects of NYX-458 on
attention, memory, and executive functions.
- Exploratory endpoints include various clinical assessments to
broadly evaluate the signals of efficacy of NYX-458 in patients
with cognitive impairment.
- Next steps:
- Aptinyx expects to re-initiate patient screening and enrollment
in the coming weeks and anticipates reporting top-line data from
this study in the second half of 2022.
Corporate Updates
- Harald Murck, M.D, Ph.D., joined Aptinyx as vice president,
medical and pharmacovigilance: In January 2021, Harald Murck,
an experienced drug development executive trained at the Max Planck
Institute of Psychiatry, Germany, and a leader in NMDA receptor
research since the late 1990s, began working with Aptinyx in the
role of vice president, medical and pharmacovigilance.
- Andy Kidd, M.D., appointed president and COO: In
December, Aptinyx announced the appointment of Andy Kidd, the
company’s then-incumbent chief operating officer, to the role of
president and chief operating officer.
- Strengthened the company’s financial position through a $48
million common stock offering: In early October 2020, Aptinyx
announced the closing of a public offering of common stock with
gross proceeds totaling $48 million, inclusive of the full exercise
of the underwriters’ option to purchase additional shares and
before deducting underwriting discounts and commissions and
offering expenses. The offering included participation from new and
existing investors. The company’s current cash balance, inclusive
of the proceeds from the offering, is expected to provide financial
support into 2023.
Upcoming Milestones
- Recommencement of patient screening in Phase 2a study of
NYX-458 in patients with cognitive impairment associated with
Parkinson’s disease or dementia with Lewy bodies – First week of
April 2021
- Meeting with FDA to discuss development path for NYX-783 in
PTSD – April 29, 2021
- Presentation of detailed data from Phase 2a exploratory study
of NYX-783 in PTSD at SOBP – April 29 – May 01, 2021
- Initiation of Phase 2b study, with registration-supportive
design, of NYX-783 in patients with PTSD – 2H 2021
- Data readout from Phase 2b study of NYX-2925 in patients with
fibromyalgia – 1H 2022
- Data readout from Phase 2b study of NYX-2925 in patients with
painful DPN – 1H 2022
- Data readout from Phase 2a study of NYX-458 in cognitive
impairment – 2H 2022
Fourth Quarter and Full Year 2020
Financial Results
Cash Position: Cash and cash equivalents were $141.0
million at December 31, 2020, compared to $98.8 million at December
31, 2019.
Collaboration Revenue: Revenue was $0.0 million and $1.6
million for the fourth quarter and full year 2020, respectively, as
compared to $1.0 million and $3.7 million for same periods in 2019.
Aptinyx’s revenue was derived from its research collaboration
agreement with Allergan, now a wholly owned subsidiary of AbbVie,
which came to its contractual conclusion during the third quarter
of 2020.
Research and Development (R&D) Expenses: R&D
expenses were $6.8 million and $32.8 million for the fourth quarter
and full year 2020, respectively, as compared to $10.6 million and
$44.3 million for same periods in 2019. The decrease in R&D
expenses during 2020 was primarily driven by reduced costs
associated with the temporary suspension of enrollment across three
Phase 2 clinical studies.
General and Administrative (G&A) Expenses: G&A
expenses were $4.8 million and $19.5 million for the fourth quarter
and full year 2020, respectively, as compared to $4.5 million and
$19.0 million for the same periods in 2019.
Net Loss: Net loss was $11.5 million for the fourth
quarter of 2020 compared to a net loss of $13.8 million for the
same period in 2019. For the year ended December 31, 2020, net loss
was $50.1 million, or basic and diluted net loss per share
attributable to common stockholders of $1.02, compared to a net
loss of $57.4 million, or basic and diluted net loss per share
attributable to common stockholders of $1.71, for the year ended
December 31, 2019.
Conference Call
The Aptinyx management team will host a conference call and
webcast today at 5:00 p.m. EDT to review its financial results and
highlights for the full year 2020 and to provide other business
updates. To access the live conference call, please dial (833)
772-0394 (domestic) or (236) 738-2205 (international) and refer to
conference ID 4846816. A live audio webcast of the event 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 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 are believed to 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. 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-2925, NYX-783, and NYX-458, therapeutic
effects of the company’s product candidates and discovery platform,
expectations regarding the design, implementation, timing, and
success of its current and planned clinical studies, including
providing updated guidance with respect thereto, the timing for the
company’s receipt and announcement of data from its clinical
studies, the timing and outcome of discussions with FDA and other
regulatory agencies, expectations regarding its preclinical
development activities, expectations regarding its uses and
sufficiency of capital, the company’s growth and the anticipated
contribution of its executive officers and management to its
operations and progress, and its expectations regarding its uses of
capital, expenses, future accumulated deficit and other fourth
quarter and year end 2020 financial results, and the effect of the
COVID-19 pandemic on the foregoing. Risks that contribute to the
uncertain nature of the forward-looking statements include: the
effect of the COVID-19 pandemic 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; positive results from
a clinical study may not necessarily be predictive of the results
of future or ongoing clinical studies; 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; the company’s ability to fund operations into
2023; as well as those risks and uncertainties set forth in the
company’s most recent quarterly report on Form 10-Q and subsequent
filings with the Securities and Exchange Commission, including our
upcoming Annual Report on Form 10-K for the year ended December 31,
2020. 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.
APTINYX INC.
CONDENSED BALANCE
SHEETS
(in thousands)
(Unaudited)
Assets
December 31, 2020
December 31, 2019
Current Assets:
Cash and cash equivalents
$
141,028
$
98,849
Restricted cash
179
179
Accounts receivable
257
444
Prepaid expenses and other current
assets
8,140
5,637
Total current assets
149,604
105,109
Property and equipment and other long-term
assets
1,002
1,370
Total assets
$
150,606
$
106,479
Liabilities and stockholders’
equity
Current Liabilities:
Accounts payable
$
1,209
$
1,555
Accrued expenses and other current
liabilities
3,374
3,341
Total current liabilities
4,583
4,896
Other long-term liabilities
114
272
Total liabilities
4,697
5,168
Stockholders’ equity
145,909
101,311
Total liabilities and stockholders’
equity
$
150,606
$
106,479
APTINYX INC.
CONDENSED STATEMENTS OF
OPERATIONS
(in thousands, except per
share data)
(Unaudited)
Three Months Ended December
31,
Year Ended December
31,
2020
2019
2020
2019
Revenues
Collaboration revenue
$
—
$
918
$
1,564
$
3,669
Operating expenses
Research and development
6,786
10,598
32,835
44,330
General and administrative
4,775
4,533
19,494
18,952
Total operating expenses
11,561
15,131
52,329
63,282
Loss from operations
(11,561
)
(14,213
)
(50,765
)
(59,613
)
Other income
73
435
712
2,203
Net loss and comprehensive loss
$
(11,488
)
$
(13,778
)
$
(50,053
)
$
(57,410
)
Net loss per share - basic and diluted
$
(0.20
)
$
(0.41
)
$
(1.02
)
$
(1.71
)
Weighted average shares outstanding -
basic and diluted
58,882
33,692
48,866
33,556
Source: Aptinyx Inc.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20210324005911/en/
Investor and Media Contact: Nick Smith Aptinyx Inc.
ir@aptinyx.com or corporate@aptinyx.com 847-871-0377
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