Full Press Release Details
Precigen, Inc. Helen Sabzevari, PhD
President & CEO 14 January 2020 38th Annual J.P. Morgan Healthcare Conference Exhibit 99.1
Forward-looking statements Precigen,
Inc. is a subsidiary of Intrexon Corporation (Nasdaq: XON). Some of the statements made in this presentation are forward-looking statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These
forward-looking statements are based upon Intrexon's and Precigen's current expectations and projections about future events and generally relate to plans, objectives and expectations for the development of Precigen's business and can be identified
by forward-looking words such as "may," "will," "potential," "expect," "believe," "anticipate," "intend," "continue," "opportunity,"
"groundwork," "poised," "future," "update" and similar expressions. Examples of forward-looking statements in his presentation, include statements about the timing, pace and progress of preclinical and
clinical trials and discovery programs, potential benefits of platforms and product candidates including in comparison to competitive platforms and products, and the expected closing date of transactions with Third Security, the renaming of Intrexon
Corporation to Precigen, Inc., and future plans for the company's remaining non-healthcare assets. Although management believes that the plans, objectives and results 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 presentation. These risks and uncertainties include, but
are not limited to, (i) the fulfillment of closing conditions, (ii) the distraction of management from business operations, (iii) the risks associated with separating businesses out from ongoing operations, (iv) Intrexon's strategy and overall
approach to its business model, its efforts to realign its business, and its ability to exercise more control and ownership over the development process and commercialization path; (v) the ability to successfully enter new markets or develop
additional products, including the expected timing and results of investigational studies and preclinical and clinical trials, whether with its collaborators or independently; (vi) the ability to successfully enter into optimal strategic
relationships with its subsidiaries and operating companies that it may form in the future; (vii) the ability to hold or generate significant operating capital, including through partnering, asset sales and operating cost reductions; (viii) actual
or anticipated variations in operating results; (ix) actual or anticipated fluctuations in competitors' or collaborators' operating results or changes in their respective growth rates; (x) cash position; (xi) market conditions in the
company's industry; (xii) the volatility of Intrexon's stock price; (xiii) the ability, and the ability of collaborators, to protect Intrexon's intellectual property and other proprietary rights and technologies; (xiv) the ability,
and the ability of collaborators, to adapt to changes in laws or regulations and policies; (xv) outcomes of pending and future litigation; (xvi) the rate and degree of market acceptance of any products developed by Intrexon, its subsidiaries,
collaborations or joint ventures; (xvii) the ability to retain and recruit key personnel; (xviii) expectations related to the use of proceeds from public offerings and other financing efforts; (xix) estimates regarding expenses, future revenue,
capital requirements and needs for additional financing; (xx) the successful completion of certain anticipated transactions, and (xxi) the challenges inherent in leadership transitions. For a discussion of other risks and uncertainties, and other
important factors, any of which could cause actual results to differ from those contained in the forward-looking statements, see the section entitled "Risk Factors" in Intrexon's Annual Report on Form 10-K, as well as discussions of potential risks,
uncertainties, and other important factors in Intrexon's subsequent filings with the Securities and Exchange Commission. All information in this presentation is as of the date its cover page, and Intrexon undertakes no duty to update this
information unless required by law. All of the pharmaceutical products described in this presentation are investigational new drugs, which are currently undergoing pre-clinical and/or human clinical trial testing. As a result, none of them have had
Intrexon: Becoming dedicated healthcare
company operating as Precigen Announced January 2, 2020 Name to be Precigen with expected stock symbol of PGEN' Assets to encompass wholly-owned subsidiaries Precigen, ActoBio Therapeutics, Exemplar Genetics and majority ownership
interest in Triple-Gene Increased Focus on Healthcare Dr. Helen Sabzevari appointed President and CEO of new Precigen Randal J. Kirk appointed Executive Chairman Leadership Certain non-healthcare assets* to be sold to Third Security (expected to
close late Jan 2020) Interest in EnviroFlight, LLC sold to Darling Ingredients, Inc. Divestment of Non-Healthcare Assets Previous cash position and expected proceeds from divestments and stock purchase approximate $175M at year end Significant
runway with increased focus and reduced cash burn for efficient use of capital Financial Strength *Ag Biotech Division (AgBio), Intrexon Laboratories Hungary (ILH), Intrexon Produce Holdings, Inc. (owner of Okanagan Specialty Fruits), Intrexon UK
Holdings, Inc. (owner of Oxitec, Ltd.), Intrexon's nominal equity interests in Oragenics and Parallel (formerly Surterra), and the internet domain name DNA.com.
Precigen: Maximizing platform
technology utilization Shared focus on immuno-oncology, infectious disease, cardiovascular disease, and autoimmune disorders Microbe-based agents that deliver disease-modifying therapeutics Multiple clinical and preclinical candidates Key interim
data in 2020 Next generation gene and cellular therapies using precision technology Multiple clinical and preclinical candidates Initial Phase 1 data readout in 2H20 Multigenic gene therapies focused on cardiovascular disease Key asset in Phase 1
Additional Phase 1 data in 2020 Market leader in genetically engineered MiniSwine models of human disease Potential for regenerative medicine applications Wholly owned subsidiary Precigen, Inc. will be renamed in connection with parent
company name change.
FISCAL STRENGTH Significant cash runway
to deliver value inflection RAPID EXECUTION Focus on rapid execution of priority programs with the highest probability of success Precigen's strategic objectives allow us to deliver on our vision for patients ACTIVE PORTFOLIO MANAGEMENT
Continuous evaluation of portfolio based on data to make rapid go/no go decisions STRATEGIC PARTNERSHIPS Seek strategic partnerships to maximize value generation PRECIGEN'S VISION FOR PATIENTS Develop life-saving and cost-conscious therapies
utilizing our cutting-edge platform technologies for patients with unmet need
CONTROL gene expression and regulation
to drive safety CONSTRUCT powerful gene programs to drive efficacy DELIVER gene programs via viral, non-viral, and microbe- based approaches to drive lower costs Precigen's technology platforms provide a strong foundation to realize core
promise of precision medicine UltraVector mbIL15 Sleeping Beauty system AttSiteTM recombinases AdenoVerseTM RheoSwitch Kill switches Tissue specific promoters Lactococcus lactis
PRODUCT PLATFORM INDICATION DISCOVERY
PRECLINICAL PHASE 1 PHASE 2 PHASE 3 STATUS / MILESTONES PARTNER PRECIGEN AG019 ActoBiotics Type 1 Diabetes Interim data in 3Q20 PRGN-3005 UltraCAR-T Ovarian Cancer Initial data in 2H20 PRGN-3006 UltraCAR-T AML, MDS Initial data in 2H20 INXN-4001
Non-viral UltraVector Heart Failure Phase 1 data in 2020 PARTNERED FCX-007 Fibroblast Cell Therapy RDEB Pivotal Phase 3 initiated AG013 ActoBiotics Oral Mucositis Phase 2 interim data in 1H20 CGF166 Gene Therapy Hearing Loss Phase 1/2 ongoing
FCX-013 Fibroblast Cell Therapy Localized Scleroderma Phase 1/2 is enrolling Precigen has robust clinical pipeline of internal and partnered programs with important data readouts in 2020
TA PRODUCT PLATFORM INDICATION
DISCOVERY PRECLINICAL PHASE 1 MILESTONES Immuno-oncology PRGN-2009 OTS AdenoVerse Immunotherapy HPV+ Cancers Phase 1 initiation 2020 PRGN-2011 AdenoVerse Cytokine Therapy Solid Tumors PRGN-5001 Multifunctional Therapeutic Solid Tumors IND-enabling
studies 2020 PRGN-3007 UltraCAR-T Undisclosed IND-enabling studies 2020 PRGN-3008 UltraCAR-T Undisclosed PRGN-5002 Multifunctional Therapeutic Solid Tumors PRGN-2010 OTS AdenoVerse Immunotherapy Solid Tumors Autoimmune Disorders AG017 ActoBiotics
Celiac Disease IND approved PRGN-3009 Undisclosed Undisclosed PRGN-3010 Undisclosed Undisclosed Infectious Disease PRGN-2012 OTS AdenoVerse Immunotherapy Undisclosed IND-enabling studies 2020 PRGN-2013 OTS AdenoVerse Immunotherapy Undisclosed
Precigen has robust preclinical pipeline to drive long-term value creation Agile portfolio management with continuous evaluation of preclinical portfolio based on data to make rapid go/no go decisions OTS: Off-the-shelf
Disrupting the market: Precigen's
UltraCAR-T treatment is delivered to patients one day after non-viral gene transfer Reliance on viral vectors Complexity of manufacturing viral vectors Long and complex CAR-T cell manufacturing process Long delays for patients High cost of
manufacturing Exhausted T cell phenotype Major challenges in solid tumor treatment Non-viral gene delivery Simplified manufacturing of Plasmid DNA Overnight UltraCAR-T manufacturing process No ex vivo expansion necessary Reduced manufacturing cost
Stem-like memory T cell phenotype Enhanced potential for expansion and persistence Conventional CAR-T Viral vectors and ex vivo expansion result in long delays for patient treatment and high cost UltraCAR-T Overnight non-viral gene transfer
eliminates long delays for patient treatment and lower manufacturing cost 1 2 5 6 7 8 3 4 Modified from Iyer et al., Front. Med.,2018
PRGN-3005 UltraCAR-T , a
first-in-class therapy in ovarian cancer Target & Patient Population Preferentially targets MUC16+ cancer cells MUC16 overexpressed on >80% of ovarian tumors Limited expression found in healthy tissues Initial target is advanced stage
platinum resistant ovarian cancer 300k diagnosed annually1/22k in US2 Phase 1 Clinical Trial Ongoing Second cohort for IP arm enrolling patients 100% manufacturing success to date Initial data readout from IP arm expected in 2H20 Encouraging
preliminary findings of UltraCAR-T kinetics 1World Health Organization, International Agency for Research on Cancer, Global Cancer Observatory. Cancer Today, Estimated number of new cases in 2018, worldwide, both sexes, all ages.
Accessed December 2018 via WHO IARC GCO website. 2American Cancer Society Ovarian Cancer Special Section. Access December 2018 via ACS website. MUC16 CAR Arm A: Intraperitoneal (IP) infusion Arm B:
Intravenous (IV) infusion Clinical Trial Schema Advanced non-viral Sleeping Beauty system to co-express MUC16 CAR, mbIL15 and kill switch
PRGN-3006 UltraCAR-T , a
first-in-class therapy in AML Target & Patient Population CD33 is overexpressed on myeloid leukemia cells and leukemic stem cells 85-90% of AML patients show expression of CD33 on blasts 20k diagnosed in US in 20181 with relapsed or refractory
AML Higher risk myelodysplastic syndrome (MDS) has US incidence >10k per year2 Phase 1/1b Clinical Trial Ongoing Second cohort of no lymphodepletion arm and first cohort of lymphodepletion arm are enrolling patients 100% manufacturing success to
date Initial data readout expected in 2H20 Orphan Drug Designation recently granted by FDA Encouraging preliminary findings of UltraCAR-T kinetics 1American Cancer Society. Key Statistics for Acute Myeloid Leukemia (AML). Accessed December
2018 via ACS website. 2American Cancer Society. Key Statistics for Myelodysplastic Syndromes. Accessed December 2018 via ACS website. Clinical Trial Schema Arm 1: No lymphodepletion prior to UltraCAR-T infusion Arm 2:
Lymphodepletion prior to UltraCAR-T infusion CD33 CAR Advanced non-viral Sleeping Beauty system to co-express CD33 CAR, mbIL15 and kill switch
PRGN-2009 off-the-shelf
AdenoVerse immunotherapy for HPV+ cancers Target & Patient Population Designed to activate immune system to recognize and target HPV+ solid tumors HPV+ cancers represent significant health burden in head and neck, cervical, vaginal and
anal cancer Gorilla adenoviral vector with large payload capacity and ability for repeat injections Optimized HPV antigen design for improved immune response differentiates from competition Phase 1 Clinical Trial Initiation Upcoming Currently under
development through CRADA with Dr. Jeffrey Schlom at NCI Phase 1 clinical trial initiation expected in 2020 PRGN-2009 immunotherapy effectively controls tumor in murine model of HPV+ head & neck cancer PRGN-2009 Control Vehicle Gorilla
adenovector with novel HPV antigen design PRGN-2009 Control CD8+ T cells / mg tumor Anti-tumor response CD8+ T cell infiltration in to tumor
Multifunctional therapeutics
overcome tumor microenvironment immunosuppression and improve T cell function compared to anti-PD1 in preclinical mouse models Multifunctional Therapeutic Platform Simultaneously targets multiple pathways to address senescence and trafficking of T
lymphocytes in tumor microenvironment Exhibited superior anti-tumor effects compared to anti-PD1 mAbs Data supports potential for expansion to multiple targets Initiate IND enabling studies for PRGN-5001 in 2020 Evaluating the optimal path forward
for Multifunctional Therapeutic Platform including ongoing partnership discussions with multiple companies PRGN-5001 exhibits superior anti-tumor effect compared to anti-PD1 in humanized mouse model of lung cancer PRGN-5002 exhibits superior
anti-tumor effect compared to anti-PD1 in humanized mouse model of cervical cancer
AG013 for oral mucositis (OM) AG013
is oral solution of ActoBiotics to deliver human Trefoil Factor 1 (hTFF1) to mucosal tissues Ease of administration Target & Patient Population OM is a side effect of chemo/radiation therapy in patients treated for head & neck cancer
and other solid tumors No drug is approved to prevent OM in the broad cancer population 2019 addressable population: approximately 850k Phase 2 Clinical Trial Ongoing in Head & Neck Cancer Patients Enrollment completed in 4Q19 Interim
data from Phase 2 expected in 1H20 Orphan Drug status in European Union FDA Fast Track designation Development under partnership with Oragenics AG013 delivers hTFF1 via genetically modified L. lactis The bacteria is freeze dried into vials Patient
mixes powder with a raspberry-flavored solution Patient swishes for 30 seconds after every meal The activity delivers a protein called trefoil factor, which regrows the oral lining 1 2 3 4 5 Sources:
AG013 Phase 1b data: Demonstrated
efficacy vs placebo * Ulcerative Oral Mucositis (UOM) : WHO score 2 Limaye et al., 2013, Cancer Clinical Trial Design Phase 1b single blind, placebo-controlled study; 6 centers in the US Study measured safety and tolerability of
locally-applied AG013 in head and neck cancer patients receiving induction chemotherapy Clinical Trial Data Summary Safe and well tolerated Consistent effect of active versus placebo across all dose frequencies without a dose frequency-dependent
effect 29% of responders reported fewer than 2 days of UOM while all placebo-treated patients experienced more than two days of UOM 40% reduction in unscheduled office and emergency room visits compared to placebo 35% reduction in percentage of days
with UOM compared to placebo AG013 reduces cumulative days with UOM* vs placebo (PBO) by 35% AG013 treatment reduced unplanned office & ER visits 29% of responders had 1 day of UOM*
AG019, a first-in-class therapy for
type 1 diabetes (T1D) Target & Patient Population First-in-class disease modifying antigen-specific immunotherapy to prevent, delay or reverse T1D Recent-onset T1D patients (children and adults) with residual functional Beta-cell mass Phase
1b/2a Clinical Trial Ongoing Phase 1b/2a study to assess the safety and tolerability of different doses of AG019 administered alone (Phase 1b) or in combination with teplizumab (anti-CD3 mAb) (Phase 2a) Enrollment and treatment completed in Phase 1b
(monotherapy); No discontinuation in treatment to date Enrollment ongoing in Phase 2a (combination) cohorts; No safety issues to date Interim data readout expected in 3Q20 AG019 is a capsule formulation of ActoBiotics to express human
Proinsulin (hPINS) and human Interleukin-10 (hIL-10) AG019 + anti-CD3 treatment is highly effective in diabetic mice Co-expression of KI67 and FoxP3 reveal local proliferation Takiishi et al., 2012, JCI NOD Recent Diabetic 89% of new-onset diabetic
mice cured by AG019 + low dose anti-CD3 PINS-specific FoxP3+ Treg cells accumulate and proliferate in the pancreas & peripheral lymph nodes hPINS & hIL-10 genes inserted
INXN-4001, novel gene therapy
product for heart failure (HF) Target & Patient Population Heart failure is a complex, multi-modal and progressive disease that requires targeting multiple pathways for successful outcome Therapeutic options for end-stage HF are limited Three
effector genes in INXN-4001 designed to address multiple malfunctions of cardiomyocytes in patients with heart failure Approximately 6M adults in US have heart failure Phase 1 Clinical Trial Ongoing Phase 1 enrollment complete Initial data shows
improvement in cardiac function and no product related adverse events Phase 1 data completion in 2020 Retrograde Coronary Sinus Infusion (RCSI) Avoids wall thickness concerns, potential coronary dissection, potential ventricular perforation,
electro-mapping and thrombus formation Is on the low pressure, venous side Allows for much larger dose delivery to entire ventricle Non-viral triple effector plasmid based on UltraVector platform to simultaneously express human S100A1,