– PRGN-2012 has the potential to
be the first FDA-approved therapeutic for the treatment of adults
with RRP, a rare and devastating chronic disease for which the
current standard-of-care is repeated surgeries –
– PRGN-2012 received Breakthrough Therapy
Designation from the FDA and Orphan Drug Designation from the FDA
and the European Commission –
– The BLA,
under an accelerated approval pathway, is supported by data from
the Phase 1/2 pivotal study in which more than 50% of patients
achieved Complete Response and more than 85% of patients had a
decrease in surgical interventions in the year after PRGN-2012
treatment compared to the year prior to treatment
–
– PRGN-2012 was well-tolerated with
no dose-limiting toxicities and no treatment-related adverse events
greater than Grade 2 –
GERMANTOWN, Md., Dec. 30,
2024 /PRNewswire/ -- Precigen,
Inc. (Nasdaq: PGEN), a biopharmaceutical company specializing
in the development of innovative gene and cell therapies to improve
the lives of patients, today announced the completion of the
rolling submission for a biologics license application (BLA) to the
US Food and Drug Administration (FDA) for PRGN-2012 (INN:
zopapogene imadenovec†) for the treatment of
adult patients with recurrent respiratory papillomatosis (RRP). The
submission is in the initial 60 day review period, during which
time the FDA will decide whether to accept the BLA for further
review and set the Prescription Drug User Fee Act (PDUFA) action
date. The BLA included a request for priority review, which, if
granted, would reduce the review timeline from the standard
10-month to a priority 6-month review from the date the submission
is accepted by the FDA.
PRGN-2012 is an investigational AdenoVerse®
gene therapy designed to elicit immune responses directed against
cells infected with human papillomavirus (HPV) 6 or HPV 11.
PRGN-2012 received Breakthrough Therapy Designation, Orphan Drug
Designation, and an accelerated approval pathway from the US Food
and Drug Administration (FDA), and Orphan Drug Designation from the
European Commission.
PRGN-2012 has the potential to be the first FDA-approved
therapeutic for the treatment of adults with RRP. RRP is a rare,
difficult-to-treat, lifelong neoplastic disease of the upper and
lower respiratory tracts caused by infection with HPV 6 or HPV 11
that can be fatal. Currently, there is no cure for RRP and the
current standard-of-care is repeated surgeries, which do not
address the underlying cause of disease and are associated with
significant morbidity. As a result, the cycle of recurrences and
surgeries continues and patients can require hundreds of lifetime
surgeries.1-7
The BLA is supported by data from the pivotal Phase 1/2 clinical
study of PRGN-2012 for the treatment of RRP (NCT04724980),
which evaluated the safety and efficacy of PRGN-2012 in adult RRP
patients. Of the 38 total patients enrolled in the study, three
patients received four administrations of PRGN-2012 at 1x
1011 particle units (PU)/dose and 35 patients
received four administrations of PRGN-2012 at the recommended phase
2 dose (RP2D) of 5 x 1011 PU/dose over a 12 week
treatment period via subcutaneous injection. Primary endpoints
included safety and Complete Response rate defined as the
percentage of patients who require no RRP surgeries in the 12-month
period after PRGN-2012 treatment completion. Key secondary
endpoints included HPV-specific immune responses, extent of
papilloma growth as measured by Derkay scoring, and quality of life
as measured by Vocal Handicap Index-10 (VHI-10). As reported in the
groundbreaking results from the pivotal study presented at the 2024
American Society of Clinical Oncology (ASCO) annual meeting, the
primary safety and efficacy endpoints were met.
"The impact of this debilitating disease on patients, families,
and their caregivers has been overlooked for more than half a
century. There is currently no approved therapy for RRP patients
and the submission of our BLA is an extremely important step in
bringing the first therapy to fight this devastating
disease," said Helen Sabzevari,
PhD, President and CEO of Precigen. "We look forward to working
closely with the FDA on next steps now that we have completed the
BLA submission and we are excited by the potential to bring
PRGN-2012 to RRP patients as quickly as possible. With our most
recent financial transactions announced last week to enhance our
balance sheet, we have extended our cash runway into 2026, well
beyond potential commercial launch in the second half of
2025."
AdenoVerse®
Precigen's AdenoVerse platform
utilizes a library of proprietary adenovectors for the efficient
gene delivery of therapeutic effectors, immunomodulators, and
vaccine antigens designed to modulate the immune system. Precigen's
gorilla adenovectors, part of the AdenoVerse library, have
potentially superior performance characteristics as compared to
current competition. AdenoVerse gene therapies have been shown to
generate high-level and durable antigen-specific T-cell immune
responses as well as an ability to boost these responses via repeat
administration. Superior performance characteristics and high yield
manufacturing of AdenoVerse vectors leveraging
UltraVector® technology allows Precigen to engineer
cutting-edge investigational gene therapies to treat complex
diseases.
Precigen: Advancing Medicine with
Precision™
Precigen (Nasdaq: PGEN) is a dedicated
discovery and clinical stage biopharmaceutical company advancing
the next generation of gene and cell therapies using precision
technology to target the most urgent and intractable diseases in
our core therapeutic areas of immuno-oncology, autoimmune
disorders, and infectious diseases. Our technologies enable us to
find innovative solutions for affordable biotherapeutics in a
controlled manner. Precigen operates as an innovation engine
progressing a preclinical and clinical pipeline of
well-differentiated therapies toward clinical proof-of-concept and
commercialization. For more information about Precigen, visit
www.precigen.com or follow us on LinkedIn or YouTube.
Trademarks
Precigen, AdenoVerse, UltraVector and
Advancing Medicine with Precision are trademarks
of Precigen and/or its affiliates. Other names may be
trademarks of their respective owners.
Cautionary Statement Regarding Forward-Looking
Statements
Some of the statements made in this press release
are forward-looking statements. These forward-looking statements
are based upon the Company's current expectations and projections
about future events and generally relate to plans, objectives, and
expectations for the development of the Company's business,
including the timing and progress of preclinical studies, clinical
trials, regulatory approvals, commercial launches and related
milestones, the promise of the Company's portfolio of therapies,
and in particular its CAR-T and AdenoVerse therapies. Although
management believes that the plans and objectives reflected in or
suggested by these forward-looking statements are reasonable, all
forward-looking statements involve risks and uncertainties and
actual future results may be materially different from the plans,
objectives and expectations expressed in this press release. The
Company has no obligation to provide any updates to these
forward-looking statements even if its expectations change. All
forward-looking statements are expressly qualified in their
entirety by this cautionary statement. For further information on
potential risks and uncertainties, and other important factors, any
of which could cause the Company's actual results to differ from
those contained in the forward-looking statements, see the section
entitled "Risk Factors" in the Company's most recent Annual Report
on Form 10-K and subsequent reports filed with the Securities and
Exchange Commission.
Investor Contact:
Steven M.
Harasym
Vice President, Investor Relations
Tel: +1 (301) 556-9850
investors@precigen.com
Media Contacts:
Donelle M.
Gregory
press@precigen.com
Glenn Silver
Lazar-FINN Partners
glenn.silver@finnpartners.com
|
†zopapogene
imadenovec is the international nonproprietary name (INN) for the
investigational therapeutic known as PRGN-2012. Zopapogene
imadenovec has not been approved by any health authority in any
country for any indication.
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"Human papillomavirus infection in papillomas and nondiseased
respiratory sites of patients with recurrent respiratory
papillomatosis using the polymerase chain reaction." Arch
Otolaryngol Head Neck Surg 119(5): 554-557.
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Derkay, CS et al.
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SOURCE Precigen, Inc.
