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© 2018 MaxCyte, Inc. All Rights Reserved. | Company Confidential
Driving The Next Generation of Cell-Based MedicinesDoug Doerfler | Founder, President & CEOSeptember 2018 LSE: MXCT / MXCR
NOT FOR PUBLICATION, DISTRIBUTION OR RELEASE, DIRECTLY OR INDIRECTLY, IN OR INTO THE UNITED STATES OR ANY OTHER JURISDICTION IN WHICH THE DISTRIBUTION OR RELEASE WOULD BE UNLAWFUL. ANY FAILURE TO COMPLY WITH THESE RESTRICTIONS MAY CONSTITUTE A VIOLATION OF APPLICABLE SECURITIES LAWS.
The information contained in this document and to be communicated during the presentation of these slides, including the talks given by presenters, any question and answer session and any documents or other materials distributed at or in connection with the presentation (together, this “presentation”) is strictly confidential, and is supplied on the understanding that it will be held in confidence, and not copied, reproduced, distributed, published or disclosed to third parties. This presentation has been prepared by MaxCyte, Inc. (the “Company”) in connection with a proposed placing of the common stock of the Company. This presentation does not constitute a prospectus or admission document or form part of any offer or invitation to purchase, sell or subscribe for, or any solicitation of any such offer to purchase, sell or subscribe for, any securities in the Company nor shall this presentation or any part of it, or the fact of its distribution, form the basis of, or be relied on in connection with, any contract therefor.
This presentation is not for distribution, directly or indirectly, in whole or in part, in or into the United States, Australia, Canada, Japan, the Republic of South Africa or any jurisdiction where it would be unlawful to do so. The distribution of this presentation or any information contained in it may be restricted by law in certain jurisdictions, and any person into whose possession any document containing this presentation or any part of it comes should inform themselves about, and observe, any such restrictions.
No reliance may be placed, for any purposes whatsoever, on the information contained in this presentation or on its completeness and this presentation should not be considered a recommendation by the Company, Panmure Gordon (UK) Limited (“Panmure”), any of their respective directors, partners, officers, employees, advisers or any other party in relation to any purchase of or subscription for securities of the Company. No representation or warranty, express or implied, is given by or on behalf of the Company or Panmure or any of their respective directors, partners, officers, employees, advisers or any other persons as to the accuracy, fairness or sufficiency of the information or opinions contained in this presentation and none of the information contained in this presentation has been independently verified by any person. Save in the case of fraud, no liability is accepted for any errors, omissions or inaccuracies in such information or opinions.
The information contained in this presentation is intended to be made available only to persons who qualify to receive it as (A) persons in member states of the European Economic Area who are “qualified investors” within the meaning of article 2(1)(e) of the Prospectus Directive; and (b) in the United Kingdom, persons who (i) have professional experience in matters relating to investments and fall within article 19(5) of the Financial Services and Markets Act 2000 (Financial Promotion) Order 2005 (the "Order"); or (ii) are persons falling within article 49(2)(a) to (d) ("high net worth companies, unincorporated associations, etc.") of the Order; or (iii) are persons to whom it may otherwise be lawfully communicated. It is a condition of your receiving this presentation that you have confirmed to the Company and produced evidence satisfactory to Company that you are within one of the categories described above.
The common stock of the Company will not be registered under the US Securities Act of 1933, as amended (“Securities Act”) or under any securities laws of any state or other jurisdiction of the United States in connection with the proposed placing to be undertaken by the Company. The common stock of the Company are being offered only to non-US Persons outside the United States in transactions exempt from the registration requirements of the Securities Act in reliance on Category 3 of Regulation S. The common stock of the Company being offered to non-US Persons are subject to the conditions listed under section 903(b)(3), or Category 3, of Regulation S. Under Category 3, Offering Restrictions (as defined under Regulation S) must be in place in connection with the Placing and additional restrictions are imposed on resales of the common stock. The Common Stock are “restricted securities” as defined in Rule 144 under the Securities Act.
Such common stock may not be offered or sold in the United States or to a US person (within the meaning of regulations made under the Securities Act) unless the common stock are registered under the Securities Act or an exemption from the registration requirements of the Securities Act is available, including a transaction specified by Regulation S.
The information contained in this presentation is confidential and must not be copied, reproduced, published, distributed, disclosed or passed to any other person at any time without the prior written consent of Panmure, as agent for the Company. This presentation may include inside information under Regulation (EU) No 596/2014 (Market Abuse Regulation) and accordingly attendees at this presentation should not deal, for its account or the account of any third party, directly or indirectly, in any securities of the Company before the information is made public. In agreeing to attend this presentation, you have consented to the receipt of the inside information and to be treated as an "insider" in relation to the information to be disclosed at this presentation.
This presentation may include opinions, forward-looking statements and estimates, many of which are based upon various assumptions including, without limitation, management's intentions going forward, projects or product development that is underway or may be undertaken or management's examination of historical operating trends, data contained in the Company’s records and other data available from third parties. Forward-looking statements speak only as at the date of the 2016 full-year results announcement and the Company and its advisers expressly disclaim any obligations or undertaking to release any update of, or revisions to, any forward-looking statements in this presentation. Although the Company believes that these assumptions were reasonable when made, these assumptions are inherently subject to significant uncertainties and contingencies which are difficult or impossible to predict and are beyond its control, and the Company may not achieve or accomplish these expectations, beliefs or projections. Neither the Company, nor any of its members, directors, officers, agents, employees or advisers intend or have any duty or obligation to supplement, amend, update or revise any of the opinions, forward-looking statements or estimates contained in this presentation. No statement in the presentation is intended to be, or intended to be construed as, a profit forecast or profit estimate or to be interpreted to mean that earnings per Company share for the current or future financial years will necessarily match or exceed the historical earnings per Company share. As a result, no undue reliance should be placed on such statements.
Panmure is regulated in the United Kingdom by the Financial Conduct Authority and is acting only for the Company in connection with the proposed placing and is not acting for or advising any other person, or treating any other person as its client, in relation thereto and will not be responsible for providing the regulatory protection afforded to clients of Panmure or advice to any other person in relation to the proposed placing.
September 2018 CONFIDENTIAL 2
Disclaimer
MaxCyte: Global cell-based medicines and life sciences company with patented cell engineering technology
Revenues of approx. $14M, stable gross margins of near 90% and 10+ years of consecutive growth
*By revenue
Focus on cell-based medicines, including: • Discovery • Development • Manufacturing• Commercialisation
Unique platform based around proprietary and FDA-cleared Flow Electroporation® technology
US-based; business model in three parts:• Drug discovery, clients include all top ten pharma companies worldwide• Cell Therapy, 55+ programmes with leading cell therapy developers• CARMA™, MaxCyte’s own next-generation immunotherapy entering clinical
trials second half 2018
September 2018 CONFIDENTIAL 3
September 2018 4
Proprietary Flow Electroporation®Patented, proprietary large-scale and regulatory-compliant platform for cell engineering
Any Molecule. Any Cell.
Any Scale.®
DNAmRNA, siRNA protein
Produce
biologics for
development
Redirect
cells to kill
cancer
Edit and
correct
genes
Cells to
discover
drugs
MaxCyte:Three-part business model
September 2018 CONFIDENTIAL 5
CARMACELL THERAPYDRUG DISCOVERY
Blue-chip client base
including all top 10 and 20
of 25 top global pharma
companies
2017 - $14m
2016 - $12m
2015 - $9m
Stable ~90% Gross Margin
$Multi-million
Licencing
Opportunities
Cells to
discover drugsCells as drugs
Direct cells to
kill cancer
Licenses granted to 55+
cell therapy programmes,
25+ for clinical use
CARMA: Next
generation mRNA
CAR-based product
Drug Discovery
CARMA
Cell Therapy
Flow Electroporation®
Drug discovery
September 2018 CONFIDENTIAL 6
CARMA Platform
Cell Therapy
Flow Electroporation®
Technology
Enabling the development of novel
cell therapies with leading players
✓ 55+ partnered programmes
- 25+ Phase I/II
- Immuno-oncology
- Gene editing
- Regenerative
medicine
✓ Annual licensing fees and
PA sales provide recurring
revenue stream
✓ Validated upside potential
of $10m per commercial
deal
Wholly owned next generation mRNA CAR-based product
✓ IND submitted to FDA in 2017 with first-in-human trial
expected to start in 2018
✓ Leverages MaxCyte’s extensive experience at the cutting edge of CAR-T
✓ $150m to $300m per product partnering opportunities
✓ Significant potential patient benefits
✓ Investment in cutting edge science
Drug Discovery
& Development
Instruments and processing assemblies (PAs) sold to pharma and biotech companies worldwide
✓ Customers include all top 10 pharma companies
✓ Sale of PAs provide recurring revenue stream
✓ Global footprint of field force
Flow Electroporation®
• MaxCyte solves problems for world’s largest pharma and biotech companies
• Instruments, processing assemblies and technology sold worldwide
• 200+ systems in the field
• Drug discovery, development and biomanufacturing
• Significant untapped market
• Growing recurring revenue element
• Consistent high margins
CONFIDENTIAL 7
Drug discovery
1 Source: MarketsandMarkets, as of January 13, 2018
MaxCyte VLX for high volume biomanufacture
MaxCyte STX for drug discovery
Global transfection market1
(reagents and equipment only)
$958m(2020)
7.5% CAGR
$677m(2015)
September 2018
Sampling of Customers
September 2018 CONFIDENTIAL 8
Cell therapy
CARMA
Flow Electroporation®
Technology
Drug Discovery
Instruments and processing assemblies (PAs) sold to pharma and biotech companies worldwide
✓ Customers include all top 10 pharma companies
✓ Sale of PAs provide recurring revenue stream
✓ Global footprint of field force
Wholly owned next generation mRNA CAR-based product
✓ IND submitted to FDA in 2017 with first-in-human trial
expected to start in 2018
✓ Leverages MaxCyte’s extensive experience at the cutting edge of CAR-T
✓ $150m to $300m per product partnering opportunities
✓ Significant potential patient benefits
✓ Investment in cutting edge science
Cell Therapy
Enabling the development of novel
cell therapies with leading players
✓ 55+ partnered programmes
- 25+ Phase I/II
- Immuno-oncology
- Gene editing
- Regenerative
medicine
✓ Annual licensing fees and
PA sales provide recurring
revenue stream
✓ Validated upside potential
of $10m per commercial
deal
Flow Electroporation®
• Rapidly growing opportunity: over 800 companies developing cell and gene based therapies
• More than 55 non-exclusive, non-CARMA programs currently licensed, 25+ for clinical use- GT instruments licensed for partnered programs ISO 9001, GMP
compliant, FDA cleared- Annual licensing fee + sale of processing assemblies
• Broad exposure to the leading developments in cell therapy in immuno-oncology, gene editing and regenerative medicine- Indications include: HIV; paediatric leukaemia; Hodgkin’s lymphoma;
triple negative breast cancer; pancreatic cancer; neuroblastoma; AML; blood cancers; CDG; pulmonary arterial hypertension; SCD
• Validated multi-million $ commercial license/milestone opportunities- First MaxCyte commercial license in gene editing with CRISPR/Bayer
March 2017
CONFIDENTIAL 9
Partnered cell therapy programmes
1 Source: Informa, Alliance for Regenerative Medicine. Total financing include M&A, PE
and VC, PIPEs, Corporate partnerships, IPOs and follow-ons; not to scale
Upfront payments from
M&A and corporate
partnerships
Total financing
$7.5bn(2017)
c.$2.2bn(2017)
Strong Financing Momentum in Cell Therapy1
Partnered Cell-Based Programmes
2011: 6
2013: 12
2015: 30+
2017: 50+
2018: 55+
September 2018
Oncology
Renal cell carcinoma **
Pancreatic cancer
Neuroblastoma
Hodgkin's lymphoma
Triple negative breast cancer
Leukemia
Ovarian cancer
Mesothelioma
Inherited Genetic Diseases
Beta thalassemia
**Sickle cell disease
Severe combined immunodeficiency disease
Cardiovascular Pulmonary arterial hypertension
Infectious disease HIV
Cell therapy programmes and partners
September 2018 CONFIDENTIAL 10
RESEARCH & CLINICAL PARTNERS* PRE-CLINICAL RESEARCH CLINICAL* NON-EXCLUSIVE COMMERCIAL RIGHTS
Onkimmune
* Confidential partners/programmes not listed** Rights granted are non-exclusive
• Programmes managed and funded by the relevant partner (not MaxCyte)
• Regulatory agencies are accelerating approvals for novel therapies that treat serious disease
• 55+ pre-clinical research licences, of which 25+ are clinical programme licences, can result in MaxCyte capturing significant commercial value
Sickle Cell Disease
Chronic Granulomatous Disease
11
Revenue potential across multiple applications/indications
Annual License Fees and Disposables + Share in Value of Therapeutic including Milestones
IMMUNO-ONCOLOGY
Renal Cell CarcinomaPancreatic MesotheliomaTriple Negative Breast NeuroblastomaHodgkin’s LymphomaAMLPaediatric Leukaemia
GENE EDITING
CAR THIVBeta-thalassemiaSickle Cell DiseaseCGDPAH
REGENERATIVE MEDICINERESEARCH CLINICAL COMMERCIAL
55+ Licensed
Partnered
Programmes
POTENTIAL
September 2018 CONFIDENTIAL
September 2018 CONFIDENTIAL 12
Potential large cell therapy commercial opportunitiesExample of typical licence deal in typical market
Instruments and
Processing Assemblies
Milestones
Sales Based Payments
Cell Therapy Partner
Programme Value Schematic
Drug Discovery
Instruments and processing assemblies (PAs) sold to pharma and biotech companies worldwide
✓ Customers include all top 10 pharma companies
✓ Sale of PAs provide recurring revenue stream
✓ Global footprint of field force
CARMA™
September 2018 CONFIDENTIAL 13
Cell Therapy
Flow Electroporation®
Technology
Enabling the development of novel
cell therapies with leading players
✓ 55+ partnered programmes
- 25+ Phase I/II
- Immuno-oncology
- Gene editing
- Regenerative
medicine
✓ Annual licensing fees and
PA sales provide recurring
revenue stream
✓ Validated upside potential
of $10m per commercial
deal
CARMA
Wholly owned next generation mRNA CAR-based product
✓ IND submitted to FDA in 2017 with first-in-human trial
expected to start in 2018
✓ Leverages MaxCyte’s extensive experience at the cutting edge of CAR-T
✓ $150m to $300m per product partnering opportunities
✓ Significant potential patient benefits
✓ Investment in cutting edge science
Flow Electroporation®
CARMA: Patented transfection of mRNA into fresh (i.e., unexpanded, unselected) cells provides a simple, rapid to manufacture, dose controllable product
• Permits the treatment of a broad range of cancers including solid tumours
Foundation work: transfection of mRNA into expanded cells at leading institutions
• 9 independent clinical trials using MaxCyte transfected mRNA involving more than 20 patients; showing evidence of anti-tumour activity, including on solid tumours
• No change in activity in expanded vs. fresh cells
CARMA: Reduced complexity, low cost, highly scalable; potential for increased safety
• Pre-clinical CARMA in vivo studies progressing in solid tumour and leukaemia models
CAR-T: recent news
• Novartis and Kite/Gilead receive approvals for Kymriah and Yescarta• Gilead acquires Kite for $11.9bn• Celgene acquires Juno for $9bn
September 2018 CONFIDENTIAL 14
CARMA™ platform The next generation of autologous CAR therapies in oncology
15
Phase 1 proof-of-concept for engineered mRNA expanded cells in pancreatic cancerUniversity of Pennsylvania clinical study showed anti-tumor activity in humans
September 2018
Complete metabolic reduction in liver FDG uptake at 1 month in all liver lesions- Three additional patients with metabolic stable disease- Two patients with stable disease by RECIST criteria
MaxCyte engineered mRNA CART meso cells
demonstrate clinical activity in chemotherapy
refractory metastatic pancreatic cancer
Meaningful progression-free survival in 2 of 6
patients
Beatty et Al. Activity of Mesothelin-specific Chimeric Antigen Receptor T cells Against Pancreatic Carcinoma Metastases in a Phase 1 Trial
• Unique platform has potential to target any protein in any indication- CAR therapies as initial application
• mRNA approach offers significant additional target opportunities in both solid and blood cancers
• High value product and platform licensing opportunities 16
CARMA™Potentially Safe, non-viral, commercial approach to cancer therapies
Blood Collection
Patient
Apheresis
Product
Infusions
mRNA CAR
MaxCyte
Aliquot and
Cryopreservation
Multiple
Doses
< 1 Day
September 2018
CARMA POTENTIAL
Low off-target toxicity due to shortened persistence
Rapid turnaround of cell therapy to patient (reduced CMC complexity)
Virus free
Simple, rapid manufacture
No pre-treatment required prior to patient dosing
Multi-dose allows greater potentialcontrol of safety
Solid and liquid tumours
17
CARMA™ versus other autologous CAR therapies
OTHER CARs
Uncontrolled toxicity
Much longer turnaround time to patient
Often employ viral components increasing risk of toxicities
Potential delays due to manufacturing capacity and reliance on viruses
Pre-treatment required
Single dose
Liquid tumours
September 2018
Mesothelin (MSLN):First solid tumour target for CARMA™
• GPI anchored membrane protein (~40 kDa)• Shown to bind to CA-125 suggestive of role in
cell adhesion, tumour invasion and metastasis• Very low expression on normal tissues, mainly
restricted to “non critical” tissues• Over-expressed on multiple adenocarcinomas
with high unmet medical need- Mesothelioma, Pancreatic & Ovarian (~80%+)- TN Breast, Lung, Gastro-esophageal, colorectal (~40%+)
• Currently available clinical experience:- Established ability to specifically target mesothelin and localize
to mesothelin positive tumors- Established preliminary safety- Demonstrated early signals of clinical benefit- Observed evidence of immune activation and induction of
mesothelin-specific T cell responses
18
Cancer Discov. 2016 Feb; 6(2):133-46, Pastan, PNAS (1996)
Frequency and distribution pattern of the MSLN protein in solid malignancies
September 2018
IP Delivery Mesothelin Targeted Therapies
Ovarian Cancer, Peritoneal Mesothelioma
IV Delivery Mesothelin Targeted Therapies
Multiple solid tumor Indications
AML (CD123 Targeted Therapy)
Undisclosed Targets
CARMA™ pipeline – Wholly owned by MaxCyteStrong therapeutic potential in solid tumours and other diseases
September 2018 19
PRE-CLINICAL
RESEARCH
POTENTIAL CLINICAL
INDICATIONDISCOVERY OPTIMIZATION IND-ENABLING PHASE 1
Current Status Expected Status: End of 2019
Dose first patient in 2019
Dose first patient in 2H18
Financials and summary
September 2018 CONFIDENTIAL 20
CARMA
Flow Electroporation®
Technology
Drug Discovery
Cell Therapy
Flow Electroporation®
• Revenues of $6.9 million for the six months ended 30 June 2018, a 11.6% increase over $6.2 million for same period of 2017
• Gross margins remained consistent at 89% for the six months ended 30 June 2018, compared to 90% for same period of 2017
• Investment in CARMA™ was $2.6 million (first half 2017: $2.1 million) as the Company completed submissions for its IND application to FDA
• Operating expenses (including CARMA investment) increased to $10.7 million for the six months ended 30 June 2018 (first half 2017: $9.5 million)
• Cash and cash equivalents, including short term investments totalled $18.8 million at 30 June 2018 (31 December 2017: $25.3 million)
CONFIDENTIAL 21
First-half 2018 financial highlights
23% two-year
CAGROrganic revenue growth
2015-2017
200+ instruments
placed
September 2018
CONFIDENTIAL 22
Operating results: H1 2017-2018
Millions June 30, 2017 June 30, 2018
Revenue $6.2 $6.9
Costs of goods sold .6 .8
Gross Margin 90% 89%
Operating expenses
Research and development 2.1 2.3
CARMA research and development 2.1 2.6
Sales and marketing 2.9 3.3
General and administrative 2.4 2.5
Operating loss before CARMA (1.9) (1.9)
Operating loss (4.0) (4.5)
Interest expense/income 0.3 0.3
Net loss (4.3) (4.8)
Note: financial information presented under US GAAP
September 2018
Operating Expenses include $0.1M and $0.4M in non-cash stock option compensation expense in H1 2017 and 2018, respectively.
CONFIDENTIAL 23
Summary financials: balance sheet
Note: financial information presented under US GAAP
Assets December 31, 2017 June 30, 2018
Current assets:
Cash and cash equivalents, including short-term investments $ 25.3 $ 18.8
Accounts receivable 3.2 3.8
Inventory 1.3 2.0
Other current assets 0.7 1.1
Total current assets 30.6 25.7
Property and equipment, net 0.8 1.1
Total assets $ 31.4 $ 26.8
Accounts payable, accrued and other 4.3 3.0
Deferred revenue 2.1 2.8
Note payable, short term 0.8 0.0
Total current liabilities 7.2 5.8
Other Non-current Liabilities 0.4 0.5
Note payable, long term 4.2 5.0
Total liabilities $ 11.8 $ 11.4
Total stockholders' equity (deficit) $ 19.6 $ 15.4
September 2018
Key progress since 2016 IPO
MaxCyte- Consistent revenue growth and high gross
margins (~90%)
Drug Discovery - c.100+ to c.200+ in the field- Clients now include all top ten (and 20 of
25 top) pharma companies worldwide
Cell Therapy- 30+ cell therapy programmes grown to 55+- CRISPR/Casebia commercial deal
CARMA™- Pre-clinical programme to IND-clearance
for first trial
CONFIDENTIAL 24September 2018
Drug Discovery
CARMA
Cell Therapy
Flow Electroporation®
Expected 12-month news flow
Drug Discovery - Expansion of core customer base- Continued growth of single use products
Cell Therapy- Additional cell therapy programmes
- Further commercial deals
CARMA™- CARMA first in human Ph I trial to commence in Q4
2018- Trial data developing for potential safety and
efficacy
CONFIDENTIAL 25September 2018
Drug Discovery - Strong revenue growth year-on-year 2014-2017 and anticipated for 2018- Focus remains on driving top-line growth; continued development and
adoption of technology through instrument and PA sales; planned expansion of the overall business
Cell Therapy- Focus on driving adoption, expanding recurring revenue from licences
and PA sales- Potential to secure additional commercialization license deals from cell
therapy programmes- Plan to invest in product development to enable partners to
commercialize novel cell based medicines- Advance proprietary gene correction process as potential long term
treatment for inherited diseases
CARMA™- Progress MCY-M11 to dosing of first patient now that IND has been
cleared by US FDA
CONFIDENTIAL 26
Strong outlook: trading in line with expectations for FY 2018
September 2018
CONFIDENTIAL 28
Summary financials: cash flow
Note: financial information presented under US GAAP
30 June 2017 30 June 2018
Cash flows from operating activities:
Net loss $ (4.3) $ (4.8)
Balance sheet changes (1.0) (1.7)
Net cash used in operating activities (5.3) (6.5)
Cash flows from investing activities: (0.1) (3.0)
Cash flows from financing activities: 23.9 .2
Net increase (decrease) in cash and cash equivalents 18.4 (9.3)
Cash and cash equivalents, beginning of period 11.7 25.3
Cash and cash equivalents, end of period $ 30.2 $ 16.0
September 2018
• OvCA• OvCA incidence US and EU = ~47K• Advanced Stage = 60%• Platinum Refractory = 100%• Patient Growth rate = 1%
September 2018 29
Peritoneal Mesothelioma
• PM incidence US and EU = ~700• Advanced Stage = 100%• Platinum Refractory = 100%• Patient Growth rate = 0.5%
M11 Revenue Potential
• Global Total Peak projected revenues = ~$1B
Sources - https://seer.cancer.gov/statfacts/html/ovary.html; https://academic.oup.com/annonc/article/27/11/2017/2467050
OvCA & peritoneal meso global market opps:Based on current protocol and initial approval
September 2018
In Vitro meso expression of healthy donor cellsDetection and functional activity - over 7 days
MCY-M11 is transient and potentiated by subsequent administrations
Despite short duration of expression, ability of
repeated dosing may
provide:
1. Overcome tolerance
2. Re-activation of immune system
3. Generation of immune cascade
100
80
60
40
20
0
+EP +EP +EP
donor 1 donor 2 donor 3
CA
RM
A-h
Me
so
Ex
pre
ss
ion
(%)
Kinetics of MCY-M11 Expression
Donors
1D 4D 7D
30
DOSE #2
Reactivates immune system
DOSE #3
Generates an immune cascadeOvercome immune tolerance
DOSE #1
MaxCyte CAR triggers immune cascade Reshaping the endogenous immune system to a more effective environment
September 2018
Tumour LysisRelease TAA, Epitope Spreading
CytokinesInterleukin
Recruit Inflammatory Cascade
Tonsils
Lymph Nodes
Thymus
Spleen
Lymph Nodes
31
MCY-M11Inhibits growth of human mesothelin-expressing tumour (ID8) cells in nude mouse in dose-dependent manner
September 2018 32
MC
Y-M
11
CO
NT
RO
L
-80 000
-60 000
-20 000
-100 000
▲ Min= 10,000 | Max = 1e+05
-60 000
-40 000
-20 000
-100 000
-0.8
-0.6
-0.4
-0.2
-1.0
▲ Min= 1e+05 | Max = 1e+06
DAY 1 DAY 5 DAY 8 DAY 13
1x108
Phosphate-buffered saline
1x107
1x107 non-specific CAR
1x108 non-specific CAR
DOSING ▼ Min= 10,000 | Max = 1e+05
Results given demonstrate only direct impact on ID8 cells
September 2018 33
MCY-M11Multiple (weekly) administrations prolong OS
Hung et al, Human Gene Therapy, 2018
Enhanced Survival of Solid Tumour Bearing Nude MiceTumour
Injections
M11 injections
M11 M11 Control
September 2018 34
MCY-M11Phase 1 clinical trial design
IND cleared in July 2018
Dose Escalation 3+3 Design Intraperitoneal
DL1: 1 x 107
cells/doseWeekly dosing x3
DL2: 5 x 107
cells/doseWeekly dosing x3
DL3: 1 x 108
cells/doseWeekly dosing x3
DL4: 5 x 108
cells/doseWeekly dosing x3
• Dosed via intraperitoneal infusion into abdominal cavity• Patients with advanced and relapsed ovarian cancer and peritoneal
mesothelioma to be enrolled• Primary endpoint: Safety• Secondary endpoints: Efficacy and immune correlates
September 2018 35
MCY-M11Targeting significant unmet needs
Ovarian Cancer
• OvCA incidence US and EU = 47K• Diagnosed @ Advanced Stage = 60%• Platinum Refractory = 100%• Patient Growth rate = 1%• Standard of Care for patients diagnosed at
Advanced Stage is surgery, radiation and platinum therapy
• 5-year survival rate at advanced stage is 20-30%
Peritoneal Mesothelioma
• PM incidence US and EU = 700• Advanced Stage = 100%• Platinum Refractory = 100%• Patient Growth rate = 0.5%• Standard of Care is surgery and platinum
therapy• Survival rate is ~ 1 year
Sources - https://seer.cancer.gov/statfacts/html/ovary.html; https://academic.oup.com/annonc/article/27/11/2017/2467050
MYC-M11 targeted treatment population
• Advanced stage OvCA which have failed platinum therapy• Advanced stage PM which have failed platinum therapy