Submission of a Prior Approval Supplement for
a reduction in the number of patients in the NEPHRO CRRT study is
expected within the coming week
The FDA agreed to two additional protocol
changes expected to accelerate enrollment in the NEPHRO CRRT
study
SAN
MATEO, Calif., Jan. 14,
2025 /PRNewswire/ -- Talphera, Inc. (Nasdaq:
TLPH), ("Talphera"), a specialty pharmaceutical company focused on
the development and commercialization of innovative therapies for
use in medically supervised settings, today announced that
following a meeting with the U.S. Food and Drug Administration
(FDA), the agency has agreed to review a Prior Approval Supplement
(PAS) requesting a reduction in the number of patients in the
NEPHRO CRRT clinical study. A PAS is reviewed by the FDA
within 30 days, and its approval by the agency is required before
formally amending the study protocol.
During this same meeting, the agency agreed to two other changes
to broaden the clinical study inclusion criteria which would allow
the Company to enroll patients already on continuous renal
replacement therapy (CRRT) beyond 48 hours as well as
heparin-tolerant patients at certain institutions. These changes
are being made through a five-day protocol amendment notice to the
FDA with no additional FDA review required.
"We continue to evaluate and focus on opportunities to support
completion of the NEPHRO CRRT study by the end of the year. In
addition to new clinical sites, our continuous improvement efforts
in study execution were strengthened following our positive
face-to-face meeting with the FDA where the agency agreed we should
submit a PAS to reduce the number of patients in the NEPHRO CRRT
study. We plan to submit the PAS in the coming week which requires
a review period of up to 30 days," stated Shakil Aslam, MD, Talphera's Chief Medical
Officer. "Our breakthrough designation status and close
collaboration with the FDA continue to support our confidence in
the importance of nafamostat as an alternative anticoagulant for
patients on CRRT," continued Dr. Aslam.
About Talphera, Inc.
Talphera, Inc. is a specialty pharmaceutical company focused on
the development and commercialization of innovative therapies for
use in medically supervised settings. Talphera's lead product
candidate, Niyad™ is a lyophilized formulation of nafamostat and is
currently being studied under an investigational device exemption
(IDE) as an anticoagulant for the extracorporeal circuit, and has
received Breakthrough Device Designation status from the U.S. Food
and Drug Administration (FDA).
This release is intended for investors only. For additional
information about Talphera, please visit www.talphera.com.
About Niyad and 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 IDE, as an
anticoagulant for the extracorporeal circuit, and has received
Breakthrough Device Designation Status from the FDA. Talphera's
registrational study of Niyad™ is named the NEPHRO CRRT
(Nafamostat Efficacy in Phase 3
Registrational Continuous Renal
Replacement Therapy) study. An ICD-10 procedural
code, XY0YX37, has been issued for the extracorporeal
introduction of nafamostat. The ICD-10 code is a
specific/billable code that can be used to indicate a procedure.
LTX-608 is a proprietary nafamostat formulation for direct IV
infusion that may be investigated and developed for the treatment
of acute respiratory distress syndrome (ARDS), disseminated
intravascular coagulation (DIC), acute pancreatitis or as an
anti-viral treatment, amongst other potential targets.
About the NEPHRO CRRT Study
The NEPHRO Study, which has received central IRB approval, is
designed as a prospective, double-blinded trial to be conducted at
up to 14 U.S. hospital intensive care units. The study will enroll
and evaluate 166 adult patients undergoing renal replacement
therapy, who cannot tolerate heparin or are at risk for bleeding.
The primary endpoint of the study is mean post-filter activated
clotting time using Niyad versus placebo over the first 24 hours.
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.
Forward-looking statements
This press release contains forward-looking statements based
upon Talphera's current expectations and assumptions. 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 "believe," "expect,"
"anticipate," "may," "if," "intends," "plans," "potential,"
"projected," "will," or the negative of these words or other
comparable terminology, and include: Talphera's expectation that
the NEPHRO study enrollment rates will accelerate due to the
protocol changes, and will support the study completion by the end
of 2025, and that the PAS will be submitted in the coming week.
Talphera's discussion of its strategy, plans and intentions 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
Talphera's product development activities, including that clinical
studies may not be fully enrolled or completed and/or confirm any
safety, efficacy or other potential developmental product
characteristics described or assumed in this press release; (ii)
Talphera's developmental product candidates may not be beneficial
to patients or healthcare providers or be successfully
commercialized; (iii) risks relating to Talphera's ability to
obtain regulatory approvals for its developmental product
candidates; (iv) risks related to the ability of Talphera and its
business partners to implement development plans, commercial launch
plans, forecasts and other business expectations; and (v) risks
related to Talphera's liquidity and its ability to maintain capital
resources sufficient to conduct its clinical studies. 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
Talphera's annual, quarterly and current reports (i.e., Form 10-K,
Form 10-Q and Form 8-K) as filed or furnished with the 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 Talphera's most recent annual, quarterly or
current report as filed or furnished with the SEC. Talphera's SEC
reports are available at www.talphera.com under the "Investors"
tab. Except to the extent required by law, Talphera 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 Talphera, Inc.