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May 19, 2021 Next Generation Personalized Medicines

Next Generation Personalized Medicines · 2021. 5. 21. · Allison O’Neill –Dana Farber Cancer Institute Gregory Wolf, ... Results supported further clinical development in multiple

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Page 1: Next Generation Personalized Medicines · 2021. 5. 21. · Allison O’Neill –Dana Farber Cancer Institute Gregory Wolf, ... Results supported further clinical development in multiple

May 19, 2021

Next Generation

Personalized

Medicines

Page 2: Next Generation Personalized Medicines · 2021. 5. 21. · Allison O’Neill –Dana Farber Cancer Institute Gregory Wolf, ... Results supported further clinical development in multiple

Disclaimer

This presentation is intended to provide summary information about the business of Brooklyn ImmunoTherapeutics LLC (“BTX” or the “Company”). The information in this presentation is in no

respects complete, comprehensive, or exhaustive, and reference is made to the proxy statement/prospectus/consent solicitation statement of NTN Buzztime, Inc.dated February 8, 2021, which has

been filed by NTN with the U.S. Securities and Exchange Commission.

This presentation is not an offer to sell securities nor should it be deemed to imply an offer of securities.

This presentation, among other matters, contains forward-looking statements based on estimates and assumptions. Forward-looking statements include information concerning possible or assumed

future results of operations of the Company, and possible future financing, partnering, strategic, sale or other transaction(s) and other information relating to the Company. Forward-looking

statements include statements containing the words "believes," "plans," "hopes," "expects," "anticipates," "intends," "estimates" or other similar words or expressions. Except for the historical

information contained herein, the matters discussed are forward-looking statements. Forward-looking statements involve significant known and unknown risks and uncertainties and other factors

which may cause the actual results, performance or achievements of the Company to differ materially from any actual future results, performance or achievements expressed or implied by those

projected in the forward-looking statements for any reason. These statements involve risks and uncertainties as to which the Company can provide no assurances. No assurance can be given that

the Company will succeed in consummating any commercial relationship or transaction or the terms and conditions upon which any such relationship or transaction may be consummated. Further,

a number of factors including (i) the impact of the ongoing COVID-19 pandemic on the timeline for the Company’s clinical trials and on the Company’s business activities generally, (ii) the Company’s

ability to complete its clinical trials on a timely basis and within the budgets for such trials, (iii) whether the clinical trials undertaken or will undertake in the future will be successful, (iv) whether the

Company’s API manufacturing operations can maintain compliance with current Good Manufacturing Practices, (v) the scope of the Company’s intellectual property protections and the outcome of

any future challenges or opposition to the Company’s intellectual property, (vi) whether the Company’s future efforts to acquire or in-license complementary programs will prove successful, (vii)

whether the Company’s efforts to pursue partnerships to advance and accelerate clinical programs will prove successful, (viii) whether the Company’s merger with NTN will be completed, (ix)

whether the Company will be able to successfully list the common stock of the combined company on the NYSE American following the merger, and (x) the other factors described in the “Risk

Factors” section of the Registration Statement, could adversely affect the Company. Further, market and industry statistics contained in this presentation are based on information available to us.

While we believe that information to be accurate, it was not prepared for purposes of a securities offering or economic analysis.

All forward looking statements speak only as of the date hereof and except as required by law, the Company assumes no obligation to update these forward-looking statements even if new

information becomes available.

2

Page 3: Next Generation Personalized Medicines · 2021. 5. 21. · Allison O’Neill –Dana Farber Cancer Institute Gregory Wolf, ... Results supported further clinical development in multiple

Brooklyn ImmunoTherapeutics

3

We are a clinical-stage biopharmaceutical company developing treatments for patients suffering from cancer and rare diseases

IRX-2, our lead program, is a human cell-derived cytokine therapy in Phase 2b development in head and neck cancer and is also being studied in other solid tumors both as a monotherapy and in combination with other anti-cancer therapies.

We have an exclusive license for an mRNA Gene Editing/Cell Therapy platform allowing for the development of a set of next products.

*Updated on 19 May 2021

Page 4: Next Generation Personalized Medicines · 2021. 5. 21. · Allison O’Neill –Dana Farber Cancer Institute Gregory Wolf, ... Results supported further clinical development in multiple

Brooklyn ImmunoTherapeutics (BTX) - Overview

4

• Strong and efficient leadership team with

60+ years of drug development expertise

• GMP Manufacturing Facility with

capabilities on both sides of the company

Cytokine TherapiesLicense with Factor: mRNA Gene Editing/Cell Therapies

IRX-2: • Ongoing Phase 2b study in

Neoadjuvant Head and Neck Cancer

• Additional Investigator Sponsored Studies in various tumor types (including combinations with a variety of Checkpoint Inhibitors)*

• 3 Independent human studies showing increases in numbers and types of immune cells after treatment with IRX-2

Exclusive license for a broad mRNA technology platform:• mRNA cell

reprogramming (cell therapy)

• mRNA based gene editing

• Proprietary gene editing protein

• Proprietary lipid delivery system

Broad Portfolio including a Phase 2b cytokine asset, potential for additional cytokine compounds, and an exclusive license for a multi product/indication gene editing/cell therapy platform

*Updated on 19 May 2021

Page 5: Next Generation Personalized Medicines · 2021. 5. 21. · Allison O’Neill –Dana Farber Cancer Institute Gregory Wolf, ... Results supported further clinical development in multiple

IRX-2 - Overview

• Phase 2 Company Sponsored Study in 1 IST

Indication targeted to begin in 2022

• Phase 3 Study in Neoadjuvant Head and Neck

Cancer targeted to begin in 2023

5

Renal Cell Cancer

Liver Cancer

Head and Neck Cancer

Gastrointestinal Cancer

Currently in Phase 2b for Neoadjuvant Head and Neck Cancer Final data readout expected in 1H2022

Additional Investigator Sponsored Trials (ISTs) in:

Future Planned Studies:

Strong IP and Patent Position

Cervical/Vulvar Interstitial Neoplasia

Triple Negative Breast Cancer

Early Stage Breast Cancer

*Updated on 19 May 2021

Page 6: Next Generation Personalized Medicines · 2021. 5. 21. · Allison O’Neill –Dana Farber Cancer Institute Gregory Wolf, ... Results supported further clinical development in multiple

mRNA Gene Editing - Overview

Factor/Novellus has an extensively patented process to

develop gene editing compounds using mRNA which are

more efficient, non immunogenic, and non mutagenic –

making this a first in class gene editing technology. License

includes opportunity for us to use mRNA gene editing

technology to develop treatments for:

6

License Overview

• Solid tumor indications

• Liquid tumor indications (refractory AML under

consideration as lead candidate)

• Sickle cell anemia

• Other inherited disorders

Projected Timeline

Initiate Pre-Clinical Development Program 1H 2021

Plan to be at IND by 2024

First patient dosed in Phase 1 on 1st indication(s) in 2025

Refractory AML and sickle cell are front runners. Development plans in place by 2Q 2021.Other indications identified

Page 7: Next Generation Personalized Medicines · 2021. 5. 21. · Allison O’Neill –Dana Farber Cancer Institute Gregory Wolf, ... Results supported further clinical development in multiple

BTX is Led by a Strong, Experienced Management Team

Established clinical operations professional with extensive experience in the immuno-oncology space

• Bristol Myers Squibb, Director Global Clinical Operations and Strategy

• Bristol Myers Squibb, Clinical Operations Regional Manager for Romania, Czech Republic, Hungary, and Turkey

• Bristol Myers Squibb Clinical Operations Lead for Elotuzumab

• Bristol Myers Squibb Clinical Operations Integration Manager for Medarex acquisition

LynnSadowski MasonExecutive Vice-President, Clinical Operations

7

Extensive experience in domestic and international product development across diverse biotech and pharmaceutical companies

• Veloxis Pharmaceuticals, SVP Global Regulatory Affairs and Chief Compliance Officer

• Retrophin, VP Regulatory Affairs

• Pfizer, Senior Director & Therapeutic Area Head, CV• Precision Pharma Services, Sr. VP Regulatory Affairs and

Quality Systems

Ron GuidoChief Development Officer

Howard Federoff, MD, PhDChief Executive Officer and President

Proven leader in small biotech with extensive experience in drug development and gene editing/cell therapy.

• CEO of UCI Health, Vice Chancellor for health affairs and Dean of the UCI School of Medicine

• Executive Vice President of Health Sciences and executive dean at Georgetown University.

• Cofounder MedGenesis Therapeutix and Brain Neurotherapy Bio

• CEO of Aspen Neuroscience

• Chair of the NIH Recombinant DNA Advisory Committee, the NHILBI Gene Therapy Resource and the Board of the Association of the Academic Health Centers.

*Updated on 19 May 2021

Page 8: Next Generation Personalized Medicines · 2021. 5. 21. · Allison O’Neill –Dana Farber Cancer Institute Gregory Wolf, ... Results supported further clinical development in multiple

• CEO of Brooklyn ImmunoTherapeutics

• Former CEO of UCI Health, vice chancellor for health affairs and dean of the UCI School of Medicine

• Former Executive Vice President of Health Sciences and Executive Dean at Georgetown University.

• Cofounder MedGenesisTherapeutix and Brain Neurotherapy Bio

• Former CEO of Aspen Neuroscience

• Chair of the NIH Recombinant DNA Advisory Committee, the NHILBI Gene Therapy Resource and the Board of the Association of the Academic Health Centers.

BTX’s Experienced Board of Directors

• Senior advisor to the CEO of Dana-Farber Cancer Institute

• Strategic Business Development & Corporate Ventures, Verily (Google Life Sciences)

• Formerly VP, Global Mergers & Acquisitions and Business Development, Roche

• Formerly Venture Partner, Colt Ventures

• Led over $5bn in deals and investments across multiple therapeutic areas & life science sectors, co-founded biotech companies in immunotherapy & microbiome space

• Board Member, MassBio(trade association)

Luba Greenwood

• Founder & Co-Head Managing Partner of ARA Partners, a PE firm managing $2.5bn in assets

• Had led over 50 investments and sits on the board of ten companies

• Prior to ARA Partners, founded and led Intervale Capital, a PE firm

• Generated gross realized returns of 3.1x capital & 45% IRR

• Director of the Conservation Fund

Charles CheringtonChairperson

Howard Federoff, M.D., Ph.D

• Chairman of Oak Bay Biosciences

• Chairman and Chief Executive Officer of MedGenesis Therapeutics Inc.

• Previous Chief Scientific Officer of PRA International and Chief Executive Officer of CroMedica International.

• Graduate from the University of the Pacific (Bachelors degree in Chemistry/Biology) and Ph.D. in Neuropsychology from the University of Victoria

Erich Mohr, Ph.D.

8

Dennis Langer, M.D., J.D.

• Director of the Whitehead Institute for Biomedical Research, Myriad Genetics, Inc, and several private companies

• Former CEO of Neose Technologies, Inc

• Former President of Dr Reddy’s North American business

• Former SVP of Research and Development at GlaxoSmithKine plc

• Graduate of Columbia University and earned his M.D. at Georgetown University School of Medicine and his J.D. at Harvard Law School

*Updated on 19 May 2021

Page 9: Next Generation Personalized Medicines · 2021. 5. 21. · Allison O’Neill –Dana Farber Cancer Institute Gregory Wolf, ... Results supported further clinical development in multiple

BTX’s Cutting Edge Scientific Advisory Board

9

IRX-2 SAB

Steven Schnittman – Independent consultant, former Vice President, Global Clinical Research Bristol Myers Squibb

Allison O’Neill – Dana Farber Cancer Institute

Gregory Wolf, MD – University of Michigan

mRNA Gene Editing/Cell Therapy SAB

Michael Andreef, MD, PhD – MD Anderson

Matthew During, MD, PhD – MeiraGTX

Christopher Rohde, PhD – Factor Bioscience

*Updated on 19 May 2021

Page 10: Next Generation Personalized Medicines · 2021. 5. 21. · Allison O’Neill –Dana Farber Cancer Institute Gregory Wolf, ... Results supported further clinical development in multiple

IRX-2 Human Derived IL-2 Product Stimulates a Broad

Immune Response IRX-2 initiates a broad set of effects across multiple immune cells

NK

Natural Killer (NK Cells) are highly cytotoxic immune effectors that can kill cancer cells without prior sensitization

Cancer-specific T cells kill cancer cells by recognizing

expressed neoantigen targets

Dendritic cells present antigens to T cells leading to T cell division and

enhancement of cell killing activity

Enhances generation andT cell stimulatory capacity of

dendritic cells

Promotes cancer-specific T cell expansion and killing

capacity

Augments capacity to kill tumors formerly insensitive to NK

mediated destruction

Function

IRX-2 Impact

10

Results supported further clinical development in multiple indications

T T Cell NK CellDendritic Cell

Page 11: Next Generation Personalized Medicines · 2021. 5. 21. · Allison O’Neill –Dana Farber Cancer Institute Gregory Wolf, ... Results supported further clinical development in multiple

IRX-2 is Differentiated from other IL-2 FormulationsUnlike recombinant IL-2 drugs and drug candidates, IRX-2 is derived from human blood cells (huIL-2 ).

This difference may confer several distinct advantages

11

IRX-2 huIL-2 Recombinant IL-2 Pegylated IL-2 Engineered IL-2

Well-tolerated ToxicityToxicity reduced as compared

to native IL-2Toxicity data limited –

preclinical animal studies

Multiple cytokines Single cytokine Single cytokine Engineered cytokine

Natural conformation leading to greater

functionality

Abnormal folding impacting functionality

Pegylation hides active site, impairing functionality

Preclinical data only: impact of modification and selective

pegylation unknown

Physiologic dosing High dosesDoses exceeding physiologic levels

Dosing not established

Page 12: Next Generation Personalized Medicines · 2021. 5. 21. · Allison O’Neill –Dana Farber Cancer Institute Gregory Wolf, ... Results supported further clinical development in multiple

Comparative Binding Affinity rIL-2 (recombinant) v huIL-2 (human)

• huIL-2 is functionally distinct from rIL-2, as

related to its ability to affect the proliferation of

resting lymphocytes

• Data has shown that huIL-2 has significantly

higher bioactivity than rIL-2 suggesting

effectiveness at lower doses and less frequent

administration than rIL-2

• This may potentially mitigate some of the

known off-target, and toxicity issuesHigh binding affinity of moIL-2* (huIL-2) , rIL-2 for the intermediate and high affinity cell types.

M.C. Denis, B.T. Huber / Molecular Immunology 40 (2003) 279–286

60

50

40

30

20

YT-1

100 1000 10,000

rIL-2

huIL-2

% I

nh

ibit

ion

IL-2 (IU/ml)

60

50

40

30

20

Kit-225

100 1000 10,000

rIL-2

huIL-2

% I

nh

ibit

ion

IL-2 (IU/ml)

12

Data demonstrate that natural huIL-2 exhibits distinct functional properties compared to recombinant rIL-2

Page 13: Next Generation Personalized Medicines · 2021. 5. 21. · Allison O’Neill –Dana Farber Cancer Institute Gregory Wolf, ... Results supported further clinical development in multiple

Biologic Activity of IRX-2 in Clinical Trials

1: Page et al. A phase Ib study of pre-operative, locoregional IRX-2 cytokine immunotherapy to prime immune responses in patients with early stage breast cancer. Clin Cancer Res December 2019 2: Berinstein et al., OncoImmunology, February 2018; Barnes and Amir, British Journal of Cancer, July 2017; Nguyen et al. Head and Neck, July 2016.3: Wolf et al., Oral Oncology, July 2020 Tumor Infiltrating Lymphocytes after neoadjuvant IRX-2 immunotherapy in oral squamous cell carcinoma: interim findings from the INSPIRE trial

Results from early-stage breast cancer clinical trial 1 Results from Phase 2a head and neck cancer clinical trial 2

13

Demonstrated Biologic Activity in 3 Independent Studies

• Studies show immune marker activation in patients treated with IRX-2 in all studies

• Correlation between marker activation and improved disease survival in Phase 2a head and neck cancer trial

Results from Phase 2b head and neck cancer clinical trial 3

Control IRX-2

Page 14: Next Generation Personalized Medicines · 2021. 5. 21. · Allison O’Neill –Dana Farber Cancer Institute Gregory Wolf, ... Results supported further clinical development in multiple

D a y s p o s t T u m o r C e l l I n o c u la t io n

Tu

mo

r v

olu

me

( m

m3

)

1 0 1 5 2 0 2 5 3 0 3 5 4 0 4 5

0

2 0 0

4 0 0

6 0 0

8 0 0

1 0 0 0

1 2 0 0

1 4 0 0

1 6 0 0

In je c t S C C 7 s .c .

1 x 1 06/m o u s e

a n t i - m P D L 1 0 .0 3 m g /m o u s e

P B S

I R X -2 1 0 0 I U /m l

I R X -2 1 0 0 I U /m l+ a n t i- m P D L 1 0 .0 3 m g /m o u s e

p = 0 .0 2 5 5

p < 0 .0 1

Robust Additive Preclinical Efficacy Demonstrated with

IRX-2 in Combination with Checkpoint Inhibitor

141. IRX-2 therapy with PD-L1 blockade in immunocompetent animal model. Lu Wen, Gregory T. Wolf, Monil Shah, David B. Page, Lynn Sadowski-Mason, Mark Prince, Jeffrey Moyer, Alfred E. Chang, and Qiao LiJournal of Clinical Oncology 2019 37:15_suppl, e14149-e14149

Page 15: Next Generation Personalized Medicines · 2021. 5. 21. · Allison O’Neill –Dana Farber Cancer Institute Gregory Wolf, ... Results supported further clinical development in multiple

Global Phase 2B Study: INSPIRE Study Design + Safety Data

Surgery(Post treatment

sample)

Randomization

Investigational Arm (Regimen 1)

Control Arm (Regimen 2)

SOC: Chemo+/- RTAdjuvant Therapy

SurgeryRegimen minus IRX-2 biologic

SOC: Chemo+/- RT Adjuvant Therapy

Booster IRX-2 Regimen (Every 3 months for 1

year)

Biopsy(Pre-Treatment

Sample)

Approximately D35

Follow-up

Follow-up

Booster IRX-2 regimen minus IRX-2 biologic

(Every 3 months for 1 year)

Single Low Dose of Cyclophosphamide

Subcutaneous IRX-2 (10 days)

141 4

Fully enrolled

15

• Completed randomization of 105 patients, 2:1 in favor of the investigational arm (March 2016 –February 2018)

• All receive standard of care surgery and postoperative adjuvant therapy, as appropriate

• Primary endpoint: Event Free Survival (EFS)

Topline readout anticipated - 1H 2022

IRX-2 Related Adverse Events >5% have included: • Nausea (10.3%)• Fatigue (5.9%)• Injection Site Pain (5.9%)• and Neck Pain (5.9%)

Page 16: Next Generation Personalized Medicines · 2021. 5. 21. · Allison O’Neill –Dana Farber Cancer Institute Gregory Wolf, ... Results supported further clinical development in multiple

BTX Cytokine Platform Clinical Pipeline

2019 2020 2021 2022

Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4

INSPIRE Phase 2BNeoadjuvant SCCHN

105 Pts

Neoadjuvant Breast Cancer (BR-101) *16 pts

Cervical Neoplasm/Vulvar Neoplasm(CIN/VIN) (CIN-201) *

60 pts

Basket Study - Metastatic Bladder, Renal,NSCLC, Melanoma, H+N (BAS-104) *

11 pts

Metastatic Hepatocellular Carcinoma (HCC-107) * 28 pts

Metastatic Gastric and Esophageal (GI-106) * 26 pts

Metastatic Head and Neck (MHN-102) * 15 pts

Neoadjuvant Triple Negative Breast Cancer (NeoTNBC) (BR-202) *

30 pts

Completed, biomarker endpoint

16

Monotherapy studies

Combination studies

PD1 + ChemoPartnered with large pharma

* Investigator Sponsored Trial (IST)

Page 17: Next Generation Personalized Medicines · 2021. 5. 21. · Allison O’Neill –Dana Farber Cancer Institute Gregory Wolf, ... Results supported further clinical development in multiple

IRX-2 is Protected by a Strong IP Portfolio

• Manufacturing process covered by patents worldwide

• New IP related to combination of IRX-2 with checkpoint inhibitors

• Own or license 90+ issued patents and 21 pending patent applications covering a variety of subject matter

• Patent protection until 2037

17

• IRX-2 is a complex biologic, manufactured in a particular way to meet consistent specifications

• Trade secrets relating to manufacturing process: IRX-2 will be very difficult to duplicate

Patent Protection

Data Exclusivity

• IRX-2 is a biologic – 12 years data exclusivity in US and 10 years data exclusivity in Europe

• Orphan drug status for Head and Neck cancer granted in US

• Investigating potential opportunities to add exclusivity

Trade Secret Protection

Future Exploration of Orphan/Rare Disease

• Potential orphan exclusivity

• Potential for new patents, term extension

Page 18: Next Generation Personalized Medicines · 2021. 5. 21. · Allison O’Neill –Dana Farber Cancer Institute Gregory Wolf, ... Results supported further clinical development in multiple

18

Over 20 production runs of IRX-2 have been conducted, yielding

consistent and releasable clinical supply material

In-house manufacturing and process development capabilities provide

opportunity to develop additional drugs with a variety of cytokine mixtures

to expand product offerings

Little or no outsourcing of cell manufacturing, increases speed and

reduces cost

Established Biologics Manufacturing Capability

Page 19: Next Generation Personalized Medicines · 2021. 5. 21. · Allison O’Neill –Dana Farber Cancer Institute Gregory Wolf, ... Results supported further clinical development in multiple

mRNA Gene Editing/Cell Therapy - Exclusive License Option

19

Exclusive Option signed December 2020 with Factor Bio and Novellus (Cambridge, MA) to enter into a definitive agreement by 4/30/21. $500k break up fee/$4M upfront due 2Q 2021.

The license includes intellectual property held by Factor Bio and Novellus for novel Gene Editing and Cell Therapy – 92 Issued Patents, over 75 pending.

License is for exclusive use to develop gene editing and cellular therapies for Liquid Tumor Types, Solid Tumor Types, Sickle Cell Anemia and other indications.

We believe this is a potentially disruptive technology given high efficiency and reduced cost of manufacturing

On completion of the license, BTX immediately becomes a player in Gene Editing for Cell Therapies for use in Oncology and other indications: augments BTX’s existing CMC.

Transforms BTX to a PLATFORM company with numerous products in its pipeline of next generation engineered cellular medicines.

*Updated on 19 May 2021

Page 20: Next Generation Personalized Medicines · 2021. 5. 21. · Allison O’Neill –Dana Farber Cancer Institute Gregory Wolf, ... Results supported further clinical development in multiple

Factor Bioscience Patented Technologies

20 © Factor Bioscience Limited

mRNA Cell Reprogramming

ToRNAdo™ DeliverySystem

• Protects from RNase• Not inhibited by serum• Delivers to primary & iPS cells• Delivers ex vivo and in vivo• Multiple granted U.S. patents

mRNA Gene Editing

• Highest efficiency• No risk of vector insertion• Delete, repair & insert• Multiple granted patents cover mRNA

encoding CRISPRs, TALENs, ZFNs, etc.

Context-Specific Gene-Editing Protein• High specificity (40-base)• Blocked by histone modifications

(prevents unwanted cutting)• Multiple granted patents, not

limited by expression vector

The foundational mRNA Reprogramming patent portfolio

• 27 granted patents (11 U.S.)

Engineered Cellular Medicines

The desired mechanism of action is engineeredin

• Allogeneic – off-the-shelf• Clonal – superior batch-to-batch consistency• Restored telomeres – enormous expansion potential

Page 21: Next Generation Personalized Medicines · 2021. 5. 21. · Allison O’Neill –Dana Farber Cancer Institute Gregory Wolf, ... Results supported further clinical development in multiple

mRNA Cell Reprogramming Platform

21 © Factor Bioscience Limited

Teratoma

(proof of pluripotency)

• Regenerative Therapeutic Platform

– Highest efficiency & footprint-free

– Applications in autologous & allogeneic cell

therapies

– Combine with gene editing to eliminate off-target

effects

“The main advantages of the RNA method are speed of colony

emergence, high efficiency, a complete absence of integration,a very low aneuploidy rate and a low donor cell requirement”

– Thorsten M Schlaeger, PhD, Boston Children’s HospitalSchlaeger, et al. A comparison of non-integrating reprogramming methods. Nature Biotechnology. 2015.

The foundational mRNA Cell Reprogramming patent portfolioU.S. Patent Nos: 8,497,124; 9,127,248; 9,399,761; 9,562,218; 9,695,401; 9,879,228; 9,969,983; 10,131,882; 10,201,599; 10,443,045;

Granted patents in: CH, DE, FR, GB, IE, CN, JP, MX, HK, AU, RU;Multiple patents pending in the US and other major market countries.

Mesenchymal Stem Cells (MSCs)

AdipocytesOil Red O stain

OsteoblastsAlizarin Red S stain

ChondrocytesAlcian Blue stain

Typical Results

Neuronsβ-tubulin

Retinal Pigment Epithelial Cells

Cardiomyocytes

Page 22: Next Generation Personalized Medicines · 2021. 5. 21. · Allison O’Neill –Dana Farber Cancer Institute Gregory Wolf, ... Results supported further clinical development in multiple

Multimodal Gene-Editing Platform

22 © Factor Bioscience Limited

mRNA Vectorization of Gene-Editing Proteins

(Multiple U.S. Patents)

Kopacz, M., et al. Mol Ther, Vol 28 No 4S1,2020.

Chromatin Context-Sensitive Gene-Editing Endonuclease

(Multiple U.S. Patents)

Figure 1. High-efficiency gene editing of TRAC and PD1 in human epidermal keratinocytes and human iPS cells.

Figure 2. High-efficiency gene editing of the AAVS1 genomic safe harbor locus in human iPS cells.

Page 23: Next Generation Personalized Medicines · 2021. 5. 21. · Allison O’Neill –Dana Farber Cancer Institute Gregory Wolf, ... Results supported further clinical development in multiple

ToRNAdo™ Delivery System

23 © Factor Bioscience Limited

• Efficient delivery of mRNA ex vivo and in vivo to skin, brain, eye, and lung

Subretinal injection illustration adapted from Ochakovski, et al., Lipofectamine® is a registered trademark of Thermo Fisher Corporation

Rat Brain (in vivo) Lateral Ventricle(brown cells = GFP+)

Rat Retina (in vivo)(brown cells = GFP+)

Human Skin (in vivo)Ventral Forearm

(red cells = RFP+)

Very low expression, inhibited by serum

Human Epidermal Keratinocytes (ex vivo)

(green cells = GFP+)

ToRNAdo™ outperforms Lipofectamine®2000 and 3000 (“LF3000” shown above),which are inhibited by serum.

ToRNAdo™ safely and effectively delivers mRNA to human skin in vivo when administered by intradermal injection.

Kostas, F., et al. Mol Ther, Vol 28 No 4S1, 2020.

Page 24: Next Generation Personalized Medicines · 2021. 5. 21. · Allison O’Neill –Dana Farber Cancer Institute Gregory Wolf, ... Results supported further clinical development in multiple

100 Patents in 14 Countries (>40 in the United States)

24 © Factor Bioscience Limited

• Global IP portfolio with patent terms extending into 2039

– Deep portfolio in 7 families, including CRISPR and TALEN coverage

2018 2019

CN JP

CN

AU

EUR

U

AU JPR

epro

gram

min

g

U.S

.

2017 2019Ther

apeu

tic

Ind

icat

ion

s

U.S

.

20172012 2013 2014 2015 2016

Priority

Filing

Gen

eEd

itin

g

2018 2019

U.S

.

Del

iver

ySy

stem

2019 2020

U.S

.

2011 2012 2013 2014 2015 2016 2017

HK

HK

MX

RU

CN

MX

AU JP RU

EB Cancer Cancer Cancer Cancer AATA

U

AU

EU RU

EB EBEB HIV

HIV

2018

HIV HIV HIV EB

CH,DE,FR, GB,IE

Priority

Granted= Patent

2020

Filing

Page 25: Next Generation Personalized Medicines · 2021. 5. 21. · Allison O’Neill –Dana Farber Cancer Institute Gregory Wolf, ... Results supported further clinical development in multiple

BTX Clinical Pipeline

25

Technology

Program 2021 2022 2023 2024 2025

Cytokine Therapy

IRX2: Neoadj

H&N

IRX2: IST Lead

Indication

New Cytokine Formul.

mRNA Gene

Editing/ Cell

Therapy

Indication #1

Indication #2

Phase 2b Phase 3

Research Lead Optimization IND Enabling

Research IND Enabling IND Filed First in Human Study

Research IND Enabling IND Filed First in Human Study

Phase 1 IST

Mtg with FDA

Phase 2 Company Sponsored Study

Mtg with FDA

*Updated on 19 May 2021

Page 26: Next Generation Personalized Medicines · 2021. 5. 21. · Allison O’Neill –Dana Farber Cancer Institute Gregory Wolf, ... Results supported further clinical development in multiple

May 19, 2021

Next Generation

Personalized

Medicines