Driving the next generation of cell-based medicines · • Global cell therapy company driving the...
Transcript of Driving the next generation of cell-based medicines · • Global cell therapy company driving the...
© 2019 MaxCyte, Inc. All Rights Reserved. | Company Confidential
Driving the next generation of cell-based medicinesDoug Doerfler | Founder, President & CEOAnnual results to 31 December 2018April 2019
LSE: MXCT / MXCS
Disclaimer
April 2019 CONFIDENTIAL 2
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Single technology platformFlow 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 correctgenes
Cells to discover
drugs
CONFIDENTIALApril 2019
Underpins MaxCyte three-part business model
4April 2019 CONFIDENTIAL
Industry-Leading Cell Engineering Platform
5
Operational highlights
CONFIDENTIALApril 2019
• Global cell therapy company driving the next generation of cell-based medicines based on proprietary flow electroporation technology
• High efficiency, reproducible, scalable cell engineering, overcomes need for viral vectors• Proprietary platform unlocks the potential of mRNA engineered stem cells, NK cells, and T-cells
• CARMA: Unique, novel mRNA-based CAR platform• First patient treated in Phase I dose-escalation clinical trial with MCY-M11, wholly-owned therapeutic candidate;
second trial planned to commence in 2019• Presentations at AACR 2019 Meeting and ASGCT 22nd Annual Meeting• Significant reductions in cell processing and manufacturing time
• Significant commercial momentum: Licenses granted to 70+ cell therapy programs, 35+ for clinical use excluding CARMA• New commercial agreements with CRISPR Therapeutics and Precision BioSciences• Research agreement with Kite, a Gilead Company, entered into in November 2018 expanded into multi-drug clinical
and commercial agreement in March 2019
• Strong and consistent revenue growth (24% CAGR) with ~90% margins provides validation and upside• Partnerships reduce need for capital• April 2019: Launched next generation of commercially-oriented instruments and consumables under ExPERT™ brand
MaxCyte: Three-part business model
CONFIDENTIAL 6
CARMACELL THERAPYDRUG DISCOVERY & DEVELOPMENT
Blue-chip client base including all top 10 and 20 of 25 top global pharma companies*
2018 – $17m2017 - $14m2016 - $12m2015 - $9m
Stable ~90% Gross Margin
$Multi-million Licencing
Opportunities
Cells to discover drugs Cells as drugs Direct cells to
kill cancer
Licenses granted to 70+ cell therapy programmes, 35+ for clinical use
CARMA: Next generation mRNA CAR-based product
* By revenue
April 2019
0
2,000,000
4,000,000
6,000,000
8,000,000
10,000,000
12,000,000
14,000,000
16,000,000
18,000,000
2014 2015 2016 2017 2018
Revenue in USD
Consistent Rapid Revenue Growth
Revenues from Life Sciencesbusiness
• Four-year revenue CAGR now 24%
Flow Electroporation®
Technology
April 2019 7
Diverse business model drives value
PartneredCell Therapy Programs
Enabling the development
of novel cell therapies
with leading players
CARMA Platform
Wholly owned next
generation mRNA
CAR-based product
Drug Discovery & Development
Instruments and consumables
sold to pharma and biotech
companies worldwide
CONFIDENTIAL
• MaxCyte solves problems for world’s largest pharma and biotech companies
• Instruments, processing assemblies and technology sold worldwide
• Drug discovery & development market- Higher productivity- Shortens timelines/eliminates bottlenecks- Commercial biomanufacturing market- Rapid response vaccines - Viral vectors
• Significant untapped market• Growing recurring revenue element• Consistent high margins
CONFIDENTIAL 8
Patient-focused drug discovery and biomanufacturing
1 Source: MarketsandMarkets, as of January 13, 2018
MaxCyte VLX for high volume biomanufacture
ExPERT STx for drug discovery
Global transfection market1
(reagents and equipment only)
$958m(2020)
7.5% CAGR
$677m(2015)
April 2018
Flow Electroporation®
Technology
April 2019 9
Diverse business model drives value
CARMA Platform
Wholly owned next
generation mRNA
CAR-based product
Drug Discovery & Development
Instruments and consumables
sold to pharma and biotech
companies worldwide
PartneredCell Therapy Programs
Enabling the development
of novel cell therapies
with leading players
CONFIDENTIAL
• Rapidly growing opportunity: 800+ companies developing cell and gene based therapies
• 70+ non-exclusive, non-CARMA programs currently licensed, 35+ for clinical use- GT instruments licensed for partnered programs ISO 9001, GMP compliant,
FDA cleared- Annual licensing fee + sale of processing assemblies
• Diversified exposure to leading developments in cell therapy in immuno-oncology, gene editing and regenerative medicine- Indications: solid and haem cancers, autoimmune disease, viral infections,
congenital disorders
• Validated multi-million $ commercial license/milestone opportunities• MaxCyte commercial license in gene editing with CRISPR/Casebia,
CRISPR (oncology), Precision Biosciences, Kite (A Gilead Company)- Announced commercial licenses could bring $250M+ in milestone payments
prior to product launches
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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
$7.6bn(2018)
c.$10.45bn(2018)
Strong Financing Momentum in Cell Therapy1
Partnered Cell Therapy Programmes
April 2019 CONFIDENTIAL
2011: 62013: 12
2015: 30+
2017: 50+
2018: 70+
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Revenue potential across multiple applications/indications
Annual License Fees and Disposables + Share in Value of Therapeutic including Milestones
IMMUNO-ONCOLOGYRenal Cell CarcinomaPancreatic MesotheliomaTriple Negative Breast NeuroblastomaHodgkin’s LymphomaAMLPaediatric Leukaemia
GENE EDITING CAR THIVBeta-thalassemiaSickle Cell DiseaseCGDPAH
REGENERATIVE MEDICINERESEARCH CLINICAL COMMERCIAL
70+ Licensed
Partnered
Programmes
POTENTIAL
April 2019 CONFIDENTIAL
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
MaxCyte’s ‘enabling’ technology at the center of 70+ non-exclusive, non-CARMA drug development partnerships
CONFIDENTIAL 12
RESEARCH & CLINICAL PARTNERS* PRE-CLINICAL RESEARCH CLINICAL* NON-EXCLUSIVE COMMERCIAL RIGHTS
Onkimmune
* Confidential partners/programmes not listed** Includes limited exclusivity+ Potential for combined clinical and approval milestones >350M USD
• Programmes managed and funded by the relevant partner (not MaxCyte) under non-exclusive licences
• Regulatory agencies are accelerating approvals for novel therapies that treat serious disease
• 70+ pre-clinical research licences, of which 35+ are clinical programme licences, can result in MaxCyte capturing significant commercial value
Sickle Cell Disease
Chronic Granulomatous Disease
April 2019
+
+
+
+
Deal value
Research stage:Instruments and
disposables
April 2019 CONFIDENTIAL 13
Clinical stage:Development milestones,
technology access fees and disposables
Commercial stage: Transition to commercial
delivers a significant step-up in value
Growing upside (milestones and revenue share) as partner’s programs progress
Intellectual property: Strong issued patents and pending applicationsSignificant trade secret protection: Instrument, consumables, applications, CARMA™
8 / Rep
✓ Flow electroporation (2029)
✓ Method applications (2036)
✓ CARMA* platform
✓ *Methods of treatment
Intellectual Property High Efficiency
✓ Controlled / Consistent
✓ Scalable
✓ Non-toxic
✓ Skew therapeutic index
Clinically Validated✓ Technical support
✓ Regulatory support
✓ Provide FTO
✓ Enabling Technology
✓ GMP
✓ Regulatory master file
✓ In clinical trials✓ 70+ licensed programs,
35+ for clinical use
De-risked Product Profile
April 2019 CONFIDENTIAL 14
PartneredCell Therapy Programs
Enabling the development
of novel cell therapies
with leading players
Flow Electroporation®
Technology
April 2019 15
Diverse business model drives value
Drug Discovery & Development
Instruments and consumables
sold to pharma and biotech
companies worldwide
CONFIDENTIAL
CARMA Platform
Wholly owned next
generation mRNA
CAR-based product
• 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
PatientApheresis Product
Infusions
mRNA CARMaxCyte
Aliquot andCryopreservation
Multiple Doses
< 1 Day
CONFIDENTIALApril 2019
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 tumors
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-tumor activity, including on solid tumors• 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 tumor and leukemia models
April 2019 CONFIDENTIAL 17
CARMA™ platform The next generation of autologous CAR therapies in oncology
Activity of Mesothelin-specific Chimeric Antigen Receptor T cells
Against Pancreatic Carcinoma Metastases in a Phase 1 Trial
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Phase 1 proof-of-concept for engineered mRNA expanded T cells in pancreatic cancerUniversity of Pennsylvania clinical study showed anti-tumor activity in humans
April 19
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 Flow
Electroporation®
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
CONFIDENTIAL
CARMA
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 tumors
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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 tumors
April 2019
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
April 19
Multiple dosing potentially leads to:Tumor Lysis
Release TAA, Epitope SpreadingCytokines
ChemokinesRecruit Inflammatory Cascade
Tonsils
Lymph Nodes
Thymus
Spleen
Lymph Nodes
22CONFIDENTIAL
April 2019 21
MCY-M11Phase 1 clinical trial
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
CONFIDENTIAL
Dose Escalation 3+3 Design Intraperitoneal
First and second patients dosed 4Q 2018, no adverse events observed
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
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Cancer Discov. 2016 Feb; 6(2):133-46, Pastan, PNAS (1996)
Frequency and distribution pattern of the MSLN protein in solid malignancies
CONFIDENTIALApril 2019
IV
IV
IVIP
IP
IV
IV
PRE-CLINICAL RESEARCH
POTENTIAL CLINICAL INDICATION DISCOVERY OPTIMIZATION IND-ENABLING PHASE 1 MARKET (PATIENTS)
IP Delivery Mesothelin Targeted Therapies
Ovarian Cancer, Peritoneal Mesothelioma
30K
IV Delivery Mesothelin Targeted Therapies
Further solid tumor Indications 110K
AML (CD123 Targeted Therapy) 5K
Undisclosed Targets
Dose first patient in 2019
CARMA™ pipeline – Wholly owned by MaxCyteStrong therapeutic potential in solid tumors and other diseases
April 2019 23
Dosed first patient in 2H18
Current Status Expected Status: End of 2019
CONFIDENTIAL
Sources - https://seer.cancer.gov/statfacts/html/ovary.html; https://academic.oup.com/annonc/article/27/11/2017/2467050 https://seer.cancer.gov/statfacts ; https://academic.oup.com/annonc/article/27/11/2017/2467050 ; https://www.medscape.com/viewarticle/727195_2 ; https://www.cancercenter.com/lung-cancer/types/tab/non-small-cell-lung-cancer/Primary market research analysis of published literature on meso expression in all tumors, data on file, 2017
April 2019 CONFIDENTIAL 24
2018 financial highlights
Accelerating revenue growth in 2018• 2018 revenues of $16.7 million
o 19% growth vs 2017o Revenue accelerated in H2 2018 ($9.7 million), increasing approximately 25% over 2H2017
• Gross margins remained stable at ~90% • EBITDA for 2018 ($.8M before CARMA) improved over expectations and 2017 ($1.2M before
CARMA) • High proportion of recurring revenues• Aggregate potential milestone payments from the commercial agreements are currently in excess
of $250 million (excluding any royalty payments)• CARMA investment $6.5 million
Cash and short-term investments of approximately $14.4 million at 31 December 2018• Partnerships reduce need for capital
April 2019 CONFIDENTIAL 25
Summary: Full-Year 2018 Financials
Revenue Analysis• Recurring revenues from
disposables sales and Cell Therapy instrument licenses
• Four-year revenue CAGR now 24%
• Potential for accelerating revenue growth and larger deals from:
• Commercial rights deals for partnered programmes in Cell Therapy
• Development deals based on CARMA progress and human proof-of-concept data
7.2
8.4 9.3
10.6
12.3 13.0
14.0 14.7
16.7
-
2.0
4.0
6.0
8.0
10.0
12.0
14.0
16.0
18.0
2014 2015 2016 2017 2018
MaxCyte Total Revenue TTM in (M’s) as of 12/31/2018
TotalNote: financial information presented under US GAAP
MaxCyte- Strong revenue growth year-on-year 2014-2018 (24% CAGR) and consistently strong
gross margins
CARMA™- MCY-M11: Clinical trial progressing as planned with no adverse events observed to date- Phase one completion expected in 1H2020 - Initiation of second program in 2019
Cell Therapy- Additional commercialization license deals signed in 2018/2019 with leading industry
partners provides a significant expansion of pipeline and milestones - Accelerating adoption leads to expanding recurring revenue from licenses and
disposables sales- Continue 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
Drug Discovery - Industry leading performance driving growth in global biotech/pharma market- Driving top-line growth with investment in sales and marketing
April 2019 CONFIDENTIAL 26
Strong outlook for 2019 and beyond
Thank You! www.MaxCyte.com
MaxCyte at forefront of the cell therapy revolution:Solving challenges critical to industry success
April 2019 CONFIDENTIAL 28
A validated and differentiated approach to cell engineering
Simplified manufacturing Regulatory complianceRegulatory Compliant, Non-viral, Scalable
Efficiency & Potency
Cellular Engineering
Reproducible Therapeutic advancement
Safe
CONFIDENTIAL 29
Operating Results: FY 2017-2018
Millions December 31, 2017 December 31, 2018
Revenue $14.0 $16.7
Costs of goods sold 1.4 1.8
Gross Margin 90% 89%
Operating expenses
Research and development 3.8 6.7
CARMA research and development 7.5 6.5
Sales and marketing 6.0 6.0
General and administrative 4.5 5.0
Operating loss before CARMA (1.8) (1.9)
Operating loss (9.3) (8.4)
Interest expense/income 0.6 0.4
Net loss (9.9) (8.9)
Note: financial information presented under US GAAP
April 2019
Operating Expenses include $0.5M and $1.3M in non-cash stock option compensation expense in 2017 and 2018, respectively.
CONFIDENTIAL 30
Summary Financials: Balance Sheet
Note: financial information presented under US GAAP
Assets December 31, 2017 December 31, 2018
Current assets:
Cash and cash equivalents, including ST investments $ 25.3 $ 14.4
Accounts receivable 3.2 4.9
Inventory 1.3 2.2
Other current assets 0.7 0.9
Total current assets 30.6 22.5
Property and equipment, net 0.8 1.8
Total assets $ 31.4 $ 24.3
Accounts payable, accrued and other 4.3 4.1
Deferred revenue (ST and LT) 2.2 2.8
Total current liabilities 6.5 6.9
Other Non-current Liabilities 0.2 0.0
Note payable, long term 5.0 5.1
Total liabilities $ 11.8 $ 12.0
Total stockholders' equity (deficit) $ 19.6 $ 12.3
April 2019
CONFIDENTIAL 31
Summary Financials: Cash Flow
Note: financial information presented under US GAAP
31 December 2017 31 December 2018
Cash flows from operating activities:
Net loss $ (9.9) $ (8.9)
Balance sheet changes 0.2 (1.6)
Net cash used in operating activities (9.7) (10.5)
Cash flows from investing activities: (0.6) (3.9)
Cash flows from financing activities: 23.9 .2
Net increase (decrease) in cash and cash equivalents 13.6 (14.1)
Cash and cash equivalents, beginning of period 11.7 25.3
Cash and cash equivalents, end of period $ 25.3 $ 11.2
April 2019
Expected 12-month news flow
CONFIDENTIAL 32
Program Milestones / News Flow Timing
CARMA™
• IND cleared for CARMA MCY-M11• CARMA First in Human • Initial CMC Validation and Safety Data• Expansion of CARMA programs
July 2018 √ 1Q2018 √ 1H 2019Ongoing
Cell Therapy • Development of additional cell therapy programs Ongoing √
Corporate/Drug Discovery & Bio-manufacturing
• Deal announcements as they occur Ongoing √
April 2019