Investigational Device Exemption (IDE)
approval by the U.S. Food and Drug Administration (FDA) allows
AcelRx to begin pivotal study of Niyad™
Single registration study with pre-agreed upon
endpoints planned to initiate in Q4 2023 with topline data expected
mid-2024; clinical site readiness is currently underway
Niyad would be the first and only approved
regional anticoagulant in the U.S. for the extracorporeal circuit,
if approved by the FDA
SAN
MATEO, Calif., Oct. 3, 2023
/PRNewswire/ -- AcelRx Pharmaceuticals, Inc. (Nasdaq:
ACRX), (AcelRx), a specialty pharmaceutical company focused on the
development and commercialization of innovative therapies for use
in medically supervised settings, today announced that it is
advancing Niyad™ (a lyophilized formulation of nafamostat) into a
registrational study following the recent approval of an
Investigational Device Exemption (IDE) submission to the United
States Food and Drug Administration (FDA). This clinical
trial will evaluate the safety and efficacy of Niyad to support a
premarket approval application (PMA) expected to be submitted in
the second half of 2024. The single study will consist of 166 adult
patients undergoing renal replacement therapy (RRT) who cannot
tolerate heparin or are at risk for bleeding. If approved,
Niyad would be the first-ever FDA-approved regional anticoagulant
for the extracorporeal circuit in the U.S.
"We are thankful that the FDA has reviewed and approved our IDE
application so we can now proceed with our registrational study to
evaluate our lead product candidate, Niyad, which we believe to be
a paradigm-shifting product candidate for the medically supervised
setting," said Vince Angotti, Chief Executive Officer of
AcelRx. "We are on track to start this study later this year,
with top-line data expected by mid-2024 and submission of a PMA
planned in the second half of 2024."
The registration study is designed as a prospective,
double-blinded trial to be conducted at approximately 10 U.S.
hospital intensive care units that will enroll and evaluate 166
patients that will be randomized into either active or placebo
arm. The primary endpoint of the study is mean post-filter
activated clotting time (ACT) over the first 24 hours versus
placebo. Key secondary endpoints include filter lifespan,
number of filter changes over 72 hours, number of transfusions over
72 hours and dialysis efficacy (based on urea concentration) over
the first 24 hours.
Pamela P. Palmer, M.D., Ph.D.,
Chief Medical Officer and Co-Founder of AcelRx, commented,
"Importantly, we are encouraged by our interactions with the FDA -
not only by being granted Breakthrough Device Designation status
for Niyad, but also in terms of the Agency's acceptance of a
straightforward registrational trial design to evaluate
Niyad. Based on the feedback from the Emergency Use
Authorization request, the FDA stated that AcelRx should proceed
with the clinical study. We're looking forward to our upcoming
Investigator's Meeting just prior to starting enrollment. We
remain steadfast in our commitment to advance Niyad for use as a
regional anticoagulant during renal replacement therapy, as current
options are limited and patients often receive care below the
recommended international standards. We also look forward to
publishing our independent qualitative market research related to
these findings later this year."
About AcelRx Pharmaceuticals, Inc.
AcelRx Pharmaceuticals, Inc. is a specialty pharmaceutical
company focused on the development and commercialization of
innovative therapies for use in medically supervised settings.
AcelRx's lead product candidate, Niyad™ is a lyophilized
formulation of nafamostat and is currently being studied under an
investigational device exemption, or IDE, as an anticoagulant for
the extracorporeal circuit, and has received Breakthrough Device
Designation status from the FDA. AcelRx is also developing two
pre-filled syringes in-licensed from its partner Aguettant:
Fedsyra™, a pre-filled ephedrine syringe, with an expected NDA
filing in 2023, and PFS-02, a pre-filled phenylephrine syringe with
an expected NDA filing in 2024. This release is intended for
investors only. For additional information about AcelRx, please
visit www.acelrx.com.
About Nafamostat
Nafamostat is a broad spectrum, synthetic serine protease
inhibitor with anticoagulant, anti-inflammatory and potential
anti-viral activities. Niyad™ is a lyophilized formulation of
nafamostat and is currently being studied under an investigational
device exemption, or IDE, as an anticoagulant for the
extracorporeal circuit, and has received Breakthrough Device
Designation Status from the FDA. LTX-608 is a proprietary
nafamostat formulation for direct IV infusion that will be
investigated and developed as a potential anti-viral for the
treatment of COVID, acute respiratory distress syndrome (ARDS),
disseminated intravascular coagulation (DIC) and acute
pancreatitis.
Forward-looking statements
This press release contains forward-looking statements based
upon AcelRx's current expectations. These and any other
forward-looking statements are made pursuant to the safe harbor
provisions of the Private Securities Litigation Reform Act of 1995.
These statements may be identified by the use of forward-looking
terminology such as "potential," "believe," "expect," "expects,"
"expected," "anticipate," "may," "will," "enable," "should,"
"seek," "approximately," "intends," "intended," "plans," "planned,"
"planning," "estimates," "benefits," or the negative of these words
or other comparable terminology. The discussion of strategy, plans
or intentions may also include forward-looking statements, which
are predictions, projections and other statements about future
events that are based on current expectations and assumptions.
These forward-looking statements involve risks and uncertainties
that could cause actual results to differ materially from those
projected, anticipated or implied by such statements, including:
(i) risks relating to AcelRx's product development activities and
ongoing commercial business operations; (ii) risks related to the
ability of AcelRx and its business partners to implement
development plans, launch plans, forecasts and other business
expectations; (iii) risks related to unexpected variations in
market growth and demand for AcelRx's commercial and developmental
products and technologies; (iv) risks related to AcelRx's liquidity
and its ability to maintain capital resources sufficient to conduct
the required clinical studies; (v) AcelRx's ability to retain its
listing on the Nasdaq exchange; and (vi) risks relating to AcelRx's
ability to obtain regulatory approvals for its developmental
product candidates. Although it is not possible to predict or
identify all such risks and uncertainties, they may include, but
are not limited to, those described under the caption "Risk
Factors" and elsewhere in AcelRx's annual, quarterly and current
reports (i.e., Form 10-K, Form 10-Q and Form 8-K) as filed or
furnished with the Securities and Exchange Commission (SEC) and any
subsequent public filings. You are cautioned not to place undue
reliance on any such forward-looking statements, which speak only
as of the date such statements were first made. To the degree
financial information is included in this press release, it is in
summary form only and must be considered in the context of the full
details provided in AcelRx's most recent annual, quarterly or
current report as filed or furnished with the SEC. AcelRx's SEC
reports are available at www.acelrx.com under the "Investors" tab.
Except to the extent required by law, AcelRx undertakes no
obligation to publicly release the result of any revisions to these
forward-looking statements to reflect new information, events or
circumstances after the date hereof, or to reflect the occurrence
of unanticipated events.
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SOURCE AcelRx Pharmaceuticals, Inc.
