– PRGN-2012 has potential to be
first FDA-approved therapeutic for treatment of RRP, a rare and
devastating chronic disease –
– Company completed BLA submission
for PRGN-2012 for treatment of adults with
RRP –
– Commercial readiness activities
underway in anticipation of potential launch of PRGN-2012; Company
started 2025 with approximately $100
million cash on-hand* with cash runway well into
2026, beyond the anticipated launch in the second half of
2025 –
– According to recently updated
internal analysis derived from review of claims data, the market
opportunity for PRGN-2012 in RRP is estimated to be approximately
27,000 adult patients in the US –
– Immense market
potential for AdenoVerse platform in other HPV6/11-driven
indications, such as genital warts, which has significant unmet
need with annual global incidence of more than 4 million and
prevalence of more than 25 million, and HPV16/18-driven
indications, such as cervical cancer and head and neck
cancers –
– Company presentation on
Thursday, January 16, 2025 at
9:00 AM PT in San
Francisco –
GERMANTOWN, Md., Jan. 13,
2025 /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 highlights for the upcoming
company presentation at the 43rd Annual J.P. Morgan Healthcare
Conference on Thursday, January 16,
2025 at 9:00 AM
PT/12:00 PM ET. Participants
may view details for Precigen's company presentation at Precigen's
website in the Events & Presentations section at
investors.precigen.com/events-presentations.
"Our recent BLA submission for PRGN-2012 in RRP puts our
commercial readiness activities in high gear and we are well
underway to ensure full readiness in anticipation of a potential
launch in the second half of this year. We have made tremendous
progress in the past six months on payer and prescriber analysis,
clarifying the market opportunity, building out our commercial and
distribution infrastructure, and other activities to ensure we
fully coordinate the payor, provider, and patient journeys for
launch," said Phil Tennant, Chief
Commercial Officer of Precigen. "Our updated analysis of the market
using claims data and electronic health records underscores the
significant potential for PRGN-2012 in RRP with approximately
27,000 adult patients now identified in the US and we expect more
than 125,000 patients ex-US. We are confident that we will be ready
to commercialize in the US as soon as we receive approval so that
patients can gain access to this game-changing treatment."
"With 100% manufacturing success rate demonstrated to date, we
anticipate our in-house commercial drug substance cGMP
manufacturing facility will be ready to meet the projected demand
to support potential commercial launch in the second half of this
year," said Rutul Shah, Chief
Operating Officer of Precigen.
"We continue to de-risk our PRGN-2012 asset and now that we have
submitted the BLA in RRP, we are a step closer to completing
our transition to a commercial-stage company. As we start 2025, we
are laser focused on working with the FDA and advancing commercial
readiness efforts in anticipation of a potential launch in the
second half of this year. In addition, we are preparing for
submissions to other health authorities in our prioritized global
markets. Beyond RRP, we see immense potential for the AdenoVerse
platform in other HPV6/11-driven indications, such as genital
warts, and HPV16/18-driven indications, such as cervical cancer and
head and neck cancers," said Helen
Sabzevari, PhD, President and CEO of Precigen. "As
communicated previously, we plan to pursue strategic partnerships
to advance our UltraCAR-T programs, which deliver autologous,
antigen-specific CAR-T cells overnight to a patient at the
patient's medical center. In conjunction, we are preparing for an
end of Phase 1b meeting with the FDA
for PRGN-3006 to share results for this highly promising program,
including new clinical biomarker data that may further enable
patient stratification and positively impact efficacy."
Realizing Precigen's Commercial Vision for PRGN-2012
AdenoVerse® Gene Therapy in RRP
- PRGN-2012 (INN: zopapogene imadenovec†) is an
investigational off-the-shelf AdenoVerse gene therapy designed to
elicit immune responses directed against cells infected with human
papillomavirus (HPV) 6 or HPV 11 for the treatment of recurrent
respiratory papillomatosis (RRP), a rare and devastating chronic
disease for which the current standard-of-care is repeated
surgeries. If approved, PRGN-2012 has the potential to be the first
US Food and Drug Administration (FDA)-approved therapeutic for the
treatment of RRP.
- PRGN-2012 received Breakthrough Therapy Designation from the
FDA. PRGN-2012 also received Orphan Drug Designation from the
FDA and Orphan Drug Designation from the European
Commission.
- In December 2024, the Company
announced that it had completed the submission of a biologics
license application (BLA) with a request for priority review for
PRGN-2012 for the treatment of adults with RRP to the FDA. 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.
- 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.
- The confirmatory clinical trial of PRGN-2012 was initiated and
is enrolling patients in accordance with the guidance from the FDA
to initiate the study prior to submission of the BLA.
- The Company continues to rapidly advance its commercial and
manufacturing readiness campaign in anticipation of a potential
2025 launch.
Maximizing the Potential of PRGN-2009
AdenoVerse® Gene Therapy in HPV-associated
Cancers
- PRGN-2009 Phase 2 clinical trials under a cost-effective
cooperative research and development agreement (CRADA) with the
National Cancer Institute (NCI) in recurrent/metastatic cervical
cancer and in newly diagnosed HPV-associated oropharyngeal cancer
are ongoing.
- Enrollment was paused in the cervical cancer Phase 2 clinical
trial at non-NCI sites as part of strategic
reprioritization activities in 2024.
Maximizing the Value of the UltraCAR-T®
Platform through Strategic Partnerships
- Enrollment was completed for the Phase 1b trial for PRGN-3006, which received Fast Track
designation from the FDA for the treatment of relapsed or
refractory (r/r) acute myeloid leukemia (AML).
- Based on the results of correlative studies of the patient
samples from the Phase 1/1b study,
the Company has identified clinical biomarkers that correlate to
objective responses after PRGN-3006 treatment in r/r AML patients.
This advancement may further enable patient stratification and
positively impact efficacy.
- The Company is preparing for an end of Phase 1b meeting with the FDA to discuss results and
next steps.
- The Company plans to focus on strategic partnership
opportunities to advance this promising UltraCAR-T program in
AML.
*Cash on-hand is preliminary and unaudited and reflects
preliminary financial information as of December 31, 2024. In preparing this information,
the Company's actual financial position as of December 31, 2024 has not yet been finalized by
management or reviewed or audited by the Company's independent
registered public accounting firm. This information is also not a
comprehensive statement of financial position or results of
operations as of or for the year-ended December 31, 2024. Subsequent information or
events may lead to material differences between the foregoing
preliminary financial information and those reported in the
Company's subsequent SEC filings. Accordingly, investors should not
place undue reliance on this preliminary financial
information.
†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.
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, UltraCAR-T,
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
View original content to download
multimedia:https://www.prnewswire.com/news-releases/precigen-to-present-plans-for-realizing-commercial-vision-for-prgn-2012-at-the-43rd-annual-jp-morgan-healthcare-conference-302348934.html
SOURCE Precigen, Inc.