Pacira BioSciences, Inc. (Nasdaq: PCRX), the industry leader in its
commitment to non-opioid pain management and regenerative health
solutions, today announced that the U.S. Food and Drug
Administration (FDA) has granted Regenerative Medicine Advanced
Therapy (RMAT) designation to PCRX-201 (enekinragene inzadenovec),
the company’s novel, intra-articular helper-dependent adenovirus
(HDAd) gene therapy product candidate that codes for interleukin-1
receptor antagonist (IL-1Ra), for the treatment of osteoarthritis
of the knee.
“We are honored to receive FDA’s first-ever RMAT designation for
a gene therapy product candidate in osteoarthritis," said Frank D.
Lee, chief executive officer of Pacira BioSciences, Inc. “We
continue to be encouraged by the preliminary clinical findings
supporting PCRX-201 as a potential disease-modifying therapy for
osteoarthritis, and we look forward to presenting additional
follow-up data later this year.”
The company’s RMAT application was supported by the preliminary
safety and efficacy findings from a Phase 1 open-label,
proof-of-concept, single ascending dose trial that enrolled 72
patients in two three-dose cohorts: a co-administered
intra-articular steroid cohort and a cohort that did not receive a
steroid. PCRX-201 was well tolerated, with efficacy observed
through at least 52 weeks at all doses and cohorts. The highest
level of efficacy was achieved in the co-administered steroid
group, which showed a greater percentage of patients with at least
a 50% improvement in Western Ontario and McMaster Universities
Osteoarthritis Index (WOMAC) pain and stiffness scores, as well as
a meaningful improvement in (Knee Injury and Osteoarthritis
Outcomes Score) KOOS functional assessment. Preliminary 36-week
data were presented at the Osteoarthritis Research Society
International (OARSI) 2023 World Congress, the premier annual
international forum in osteoarthritis research and treatment. The
52-week data have been accepted for presentation at OARSI 2024
taking place in Vienna, Austria in April 2024 and the company
expects to present 104-week efficacy and safety data later this
year.
Established under the 21st Century Cures Act, RMAT designation
is a dedicated program designed to expedite the development and
review processes for promising therapies, including genetic
therapies, that are intended to treat, modify, reverse, or cure a
serious or life-threatening disease or condition, and for which
preliminary clinical evidence indicates that the drug or therapy
has the potential to address an unmet medical need.
RMAT designation provides the benefits of intensive FDA guidance
on efficient drug development, including the ability for early
interactions with FDA to discuss surrogate or intermediate
endpoints, potential ways to support accelerated approval and
satisfy post-approval requirements, potential priority review of
the Biologics License Application (BLA), and other opportunities to
expedite development and review. PCRX-201 was also granted Advanced
Therapy Medicinal Products (ATMP) designation by the European
Medicines Agency in May 2023.
About PCRX-201
PCRX-201 was acquired from GQ Bio Therapeutics GmbH, a privately
held biopharmaceutical company headquartered in Hamburg, Germany.
GQ Bio’s product candidates are next-generation gene transfer
vehicles. These gene therapy vectors are highly efficient in
entering joint cells to confer multi-year clinical benefit. In
PCRX-201, the high-capacity adenoviral gene therapy vector codes
for the expression of IL-1Ra, a cytokine inhibitor that plays a
central role in blocking inflammation and catabolic processes that
are associated with pain and disease progression in osteoarthritis.
Its unique design includes an inducible promoter so that, only in
the presence of inflammation signaling, the vector turns joint
cells into factories to produce sustained therapeutic levels of
IL-1Ra to manage pain and mitigate osteoarthritis-related joint
damage while remaining localized to the joint space.
About Pacira
Pacira BioSciences, Inc. (Nasdaq: PCRX) is committed to
providing a non-opioid option to as many patients as possible to
redefine the role of opioids as rescue therapy only. Pacira has
three commercial-stage non-opioid treatments: EXPAREL® (bupivacaine
liposome injectable suspension), a long-acting local analgesic
currently approved for infiltration, fascial plane block, and as an
interscalene brachial plexus nerve block for postsurgical pain
management; ZILRETTA® (triamcinolone acetonide extended-release
injectable suspension), an extended-release, intra-articular
injection indicated for the management of osteoarthritis knee pain;
and ioveraº®, a novel, handheld device for delivering immediate,
long-acting, drug-free pain control using precise, controlled doses
of cold temperature to a targeted nerve. To learn more about
Pacira, including the corporate mission to reduce overreliance on
opioids, visit www.pacira.com.
Forward-Looking Statements
Any statements in this press release about Pacira’s future
expectations, plans, trends, outlook, projections and prospects,
and other statements containing the words “believes,”
“anticipates,” “plans,” “estimates,” “expects,” “intends,” “may,”
“will,” “would,” “could,” “can” and similar expressions, constitute
forward-looking statements within the meaning of Section 21E of the
Securities Exchange Act of 1934, as amended (the “Exchange Act”),
and the Private Securities Litigation Reform Act of 1995,
including, without limitation, statements related to our growth and
future operating results and trends, our strategy, plans,
objectives, expectations (financial or otherwise) and intentions,
future financial results and growth potential, including our plans
with respect to the repayment of our indebtedness, anticipated
product portfolio, development programs, patent terms, development
of products, strategic alliances and intellectual property and
other statements that are not historical facts. For this purpose,
any statement that is not a statement of historical fact should be
considered a forward-looking statement. We cannot assure you that
our estimates, assumptions and expectations will prove to have been
correct. Actual results may differ materially from those indicated
by such forward-looking statements as a result of various important
factors, including risks relating to, among others: the integration
of our new chief executive officer; risks associated with
acquisitions, such as the risk that the acquired businesses will
not be integrated successfully, that such integration may be more
difficult, time-consuming or costly than expected or that the
expected benefits of the transaction will not occur; our
manufacturing and supply chain, global and U.S. economic conditions
(including inflation and rising interest rates), and our business,
including our revenues, financial condition, cash flow and results
of operations; the success of our sales and manufacturing efforts
in support of the commercialization of EXPAREL, ZILRETTA and
iovera°; the rate and degree of market acceptance of EXPAREL,
ZILRETTA and iovera°; the size and growth of the potential markets
for EXPAREL, ZILRETTA and iovera° and our ability to serve those
markets; our plans to expand the use of EXPAREL, ZILRETTA and
iovera° to additional indications and opportunities, and the timing
and success of any related clinical trials for EXPAREL, ZILRETTA
and iovera°; the commercial success of EXPAREL, ZILRETTA and
iovera°; the related timing and success of U.S. Food and Drug
Administration supplemental New Drug Applications and premarket
notification 510(k)s; the related timing and success of European
Medicines Agency Marketing Authorization Applications; our plans to
evaluate, develop and pursue additional product candidates
utilizing our proprietary multivesicular liposome (“pMVL”) drug
delivery technology; the approval of the commercialization of our
products in other jurisdictions; clinical trials in support of an
existing or potential pMVL-based product; our commercialization and
marketing capabilities; our ability to successfully complete
capital projects; the outcome of any litigation; the ability to
successfully integrate any future acquisitions into our existing
business; the recoverability of our deferred tax assets;
assumptions associated with contingent consideration payments; and
factors discussed in the “Risk Factors” of our most recent Annual
Report on Form 10-K and in other filings that we periodically make
with the Securities and Exchange Commission (the “SEC”). In
addition, the forward-looking statements included in this press
release represent our views as of the date of this press release.
Important factors could cause actual results to differ materially
from those indicated or implied by forward-looking statements, and
as such we anticipate that subsequent events and developments will
cause our views to change. Except as required by applicable law, we
undertake no intention or obligation to update or revise any
forward-looking statements, whether as a result of new information,
future events or otherwise, and readers should not rely on these
forward-looking statements as representing our views as of any date
subsequent to the date of this press release.
These forward-looking statements involve known and unknown
risks, uncertainties and other factors that may cause our actual
results, levels of activity, performance or achievements to differ
materially from those expressed or implied by these statements.
These factors include the matters discussed and referenced in the
“Risk Factors” of our most recent Annual Report on Form 10-K and in
other filings that we periodically make with the SEC.
Investor Contact:
Susan Mesco, (973) 451-4030
susan.mesco@pacira.com
Media Contact:
Sara Marino, (973) 370-5430
sara.marino@pacira.com
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