Full Press Release Details
Precigen Achieves Significant Clinical Progress
for UltraCAR-T and AdenoVerse Therapies
50% (3 out of 6) objective response rate (ORR) in relapsed or refractory (r/r) acute myeloid leukemia (AML) patients treated with PRGN-3006
at the two lowest dose levels (a single administration of 4 to 28 million UltraCAR-T cells) in the lymphodepletion cohort of the ongoing
40% ORR (2 out of 5) and 60% disease control rate (DCR) (3 out of 5) in recurrent or metastatic human papilloma virus (HPV)-associated
cancer patients treated in the PRGN-2009 combination arm of the ongoing Phase 1 trial -
Preliminary PRGN-2012 Phase 1 data show encouraging clinical activity, including ongoing reduction/elimination of the need for surgical
intervention at the most recent follow-up, up to 12 weeks after PRGN-2012 treatment -
PRGN-3005 and PRGN-3006 UltraCAR-T demonstrate favorable safety profiles with no dose-limiting toxicities or neurotoxicity, dose-dependent
in vivo expansion and durable persistence -
PRGN-2009 and PRGN-2012 AdenoVerse immunotherapies demonstrate favorable safety profiles with no dose-limiting toxicities, strong antigen-specific
immune response and lack of significant neutralizing antibody response upon repeat administrations -
PRGN-3006 clinical abstract (Abstract# 825) selected for oral presentation at the 63rd American Society of Hematology (ASH) Annual Meeting
Precigen to host 2021 R&D Day virtual event today at 11:00 AM ET -
GERMANTOWN, MD, November 4, 2021 -
Precigen, Inc. (Nasdaq: PGEN), a biopharmaceutical company specializing in the development of innovative
gene and cell therapies to improve the lives of patients, announced a topline summary of the presentations planned for today's
2021 R&D Day virtual event, which begins at 11:00 AM ET. Participants may register and access the live webcast through Precigen's
investor relations website in the Events & Presentations
event will showcase clinical progress for Precigen's UltraCAR-T platform, including PRGN-3005 UltraCAR-T, PRGN-3006 UltraCAR-T,
PRGN-3007 UltraCAR-T and the AdenoVerse Immunotherapy platform, including PRGN-2009 off-the-shelf (OTS) AdenoVerse immunotherapy and
PRGN-2012 OTS AdenoVerse Immunotherapy. Presentations will be made by Precigen executives and clinical trial investigators,
R&D Day highlights the most significant clinical data presented for the UltraCAR-T and AdenoVerse platforms to date," said
Helen Sabzevari, PhD, President and CEO of Precigen, "and we are highly encouraged by the initial results we are seeing across
assets in both platforms. With UltraCAR-T, initial data for PRGN-3005 and PRGN-3006 continue to demonstrate favorable safety
profiles, dose-dependent expansion, and durable persistence. The very encouraging clinical responses in relapsed or refractory AML
patients treated with PRGN-3006 at the two lowest
the lymphodepletion cohort, which are administered at significantly lower doses than competing approaches, highlight the potential of
the UltraCAR-T platform. Our AdenoVerse immunotherapy platform is equally impressive with initial data for PRGN-2009 and PRGN-2012 showing
antigen-specific immune responses, low neutralizing antibody responses, and favorable safety profiles highlighting the potential for repeat
administrations. Preliminary data for PRGN-2009 show encouraging objective responses and suggest an attractive opportunity for potential
combination of PRGN-2009 with checkpoint inhibitors in multiple HPV-associated cancers. Finally, preliminary data for PRGN-2012 show encouraging
clinical responses in RRP patients, including a reduction in surgical interventions following PRGN-2012 treatment. We are on track to
pursue potentially registrational trials for therapeutic candidates in both the UltraCAR-T and AdenoVerse platforms upon dose confirmation
PRGN-3006 UltraCAR-T
Non-lymphodepletion Cohort (Cohort 1)
| Dose Level (DL) | Subjects | Dose Range (UltraCAR-T Cells/kg) | Total UltraCAR-T Dose Administered |
| DL1 | N=3 | >3x10 4 to 1x10 5 | 1.8 to 7.1 x10 6 cells |
| DL2 | N=3 | >1x10 5 to 3x10 5 | 24 to 29 x10 6 cells |
| DL3 | N=3 | >3x10 5 to 1x10 6 | 34 to 50 x10 6 cells |
Lymphodepletion Cohort (Cohort 2)
| Dose Level (DL) | Subjects | Dose Range (UltraCAR-T Cells/kg) | Total UltraCAR-T Dose Administered |
| DL1 | N=3 | >3x10 4 to 1x10 5 | 4.4 to 10 x10 6 cells |
| DL2 | N=3 | >1x10 5 to 3x10 5 | 18 to 28 x10 6 cells |
PRGN-3005 UltraCAR-T
| Dose Level (DL) | Subjects | Dose Range (UltraCAR-T Cells/kg) | Total UltraCAR-T Dose Administered |
| DL1 | N=3 | >3x10 4 to 1x10 5 | 6 to 7.6 x10 6 cells |
| DL2 | N=3 | >1x10 5 to 3x10 5 | 12 to 21 x10 6 cells |
| DL3 | N=4 | >3x10 5 to 5x10 6 | 33 to 321 x10 6 cells |
Generation UltraCAR-T with Intrinsic PD-1 Inhibition
PRGN-2012 OTS AdenoVerse Immunotherapy
PRGN-2009 OTS AdenoVerse Immunotherapy
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 unique therapies toward clinical proof-of-concept
and commercialization. For more information about Precigen, visit www.precigen.com or follow us
on Twitter @Precigen and LinkedIn.
UltraCAR-T is a multigenic autologous CAR-T platform
that utilizes Precigen's advanced non-viral Sleeping Beauty system to simultaneously express an antigen-specific CAR to specifically
target tumor cells, mbIL15 for enhanced in vivo expansion and persistence, and a kill switch to conditionally eliminate CAR-T cells
for a potentially improved safety profile. Precigen has advanced the UltraCAR-T platform to address the inhibitory tumor microenvironment
by incorporating a novel mechanism for intrinsic checkpoint blockade without the need for complex and expensive gene editing techniques.
UltraCAR-T investigational therapies are manufactured via Precigen's overnight manufacturing process using the proprietary UltraPorator
electroporation system at the medical center and administered to patients only one day following gene transfer. The overnight UltraCAR-T
manufacturing process does not use viral vectors and does not require ex vivo activation and expansion of T cells, potentially
addressing major limitations of current T cell therapies.
AdenoVerse Immunotherapy
Precigen's AdenoVerse immunotherapy 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 immunotherapies have been shown to generate high-level and durable antigen-specific
neutralizing antibodies and effector T cell immune responses as well as an ability to boost these antibody and T cell responses via repeat administration.
Superior performance characteristics and high yield manufacturing of AdenoVerse vectors combined with UltraVector technology
allows Precigen to engineer cutting-edge investigational gene therapies to treat complex diseases.
The UltraPorator system is an exclusive
device and proprietary software solution for the scale-up of rapid and cost-effective manufacturing of UltraCAR-T therapies
and potentially represents a major advancement over current electroporation devices by significantly reducing the processing time and
contamination risk. The UltraPorator device is a high-throughput, semi-closed electroporation system for modifying T cells using Precigen's
proprietary non-viral gene transfer technology. UltraPorator is being utilized for clinical manufacturing of Precigen's investigational
UltraCAR-T therapies in compliance with current good manufacturing practices.
Precigen, UltraCAR-T, AdenoVerse, UltraVector,
UltraPorator 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
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, discovery programs 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, including
the possibility that the timeline for the Company's clinical trials might be impacted by the COVID-19 pandemic, 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.