One Target: infinite Hope - Q3, 2017 Corporate Presentation
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one Target: infinite Hope™ Corporate Presentation Q3, 2017
Forward Looking Statements
Certain statements in this presentation are “forward-looking statements. Any statements that express or involve discussions with
respect to predictions, expectations, beliefs, plans, projections, objectives, assumptions or future events or performance (often,
but not always using words or phrases such as “expect”, “seek”, “endeavour”, “anticipate”, “plan”, “estimate”, “believe”, “intend”,
or stating that certain actions, events or results may, could, would, might or will occur or be taken, or achieved) are not
statements of historical fact and may be “forward-looking statements”. Forward-looking statements are based on expectations,
estimates and projections at the time the statements are made that involve a number of risks and uncertainties which would
cause actual results or events to differ materially from those presently anticipated. Forward-looking statements are based on
expectations, estimates and projections at the time the statements are made and involve significant known and unknown risks,
uncertainties and assumptions. A number of factors could cause actual results, performance or achievements to be materially
different from any future results, performance or achievements that may be expressed or implied by such forward-looking
statements. These include, but are not limited to, the risk factors discussed in the public filings made by Medicenna with the
applicable securities commissions in Canada, including the Annual Information Form dated June 15, 2017. Should one or more
of these risks or uncertainties materialize, or should assumptions underlying the forward-looking statements prove incorrect,
actual results, performance or achievements could vary materially from those expressed or implied by the forward-looking
statements contained in this document. These factors should be considered carefully and prospective investors should not place
undue reliance on these forward-looking statements. Although the forward-looking statements contained in this document are
based upon what Medicenna currently believes to be reasonable assumptions, Medicenna cannot assure prospective investors
that actual results, performance or achievements will be consistent with these forward-looking statements. Except as required by
law, Medicenna does not have any obligation to advise any person if it becomes aware of any inaccuracy in or omission from
any forward-looking statement, nor does it intend, or assume any obligation, to update or revise these forward-looking
statements to reflect new events or circumstances.
2
Medicenna: Corporate Highlights
➢ Publicly listed (TSXV: MDNA), clinical-stage, immuno-oncology company
developing a novel therapy targeting the Interleukin-4 Receptor (IL4R) biomarker
➢ Every year >1 million cancer patients afflicted with IL4R tumors1
➢ MDNA55 (lead): highly compelling, Phase II clinical data for recurrent
glioblastoma (rGB), the most common and aggressive form of brain cancer
➢ MDNA55 market opportunity: $650 million in annual sales for rGB; >$2 billion
including other brain cancers1,2
➢ MDNA55 has Orphan Drug (FDA, EMA) & Fast Track Designations (FDA)
➢ Exciting pre-clinical IL-2, IL-4 and IL-13 Superkine platform
➢ Well funded with $14M US non-dilutive grant and $14M CAD Private Placement
➢ Seasoned management with technology platform protected by 12 patent families
1. BioXcel Strategic Analysis Report, 2014.
2. Decision Resources, Inc Glioblastoma Report, Sept 2013
3
Treatment Pathway for Glioblastoma (GB)
GB is uniformly fatal; virtually all tumors will recur (rGB)
GB Surgery Radiotherapy
Diagnosis (85-90%) +
Chemotherapy
55% of GB
Chemotherapy Chemo.
Resistant*
25%
MDNA55 Treatment Relapse Surgery
(Direct infusion into tumor - CED)
Add’l Chemo.or
75% of rGB is non-operable Experimental
Therapies
* Expression of the DNA repair protein O6-methylguanine-DNA methyltransferase (MGMT) is
responsible for resistance to alkylating agents used in GB treatment.
4
MDNA55: Targeted Dual-Action
Immunotherapeutic
A Powerful Molecular Trojan Horse
Tumor Targeting Domain Tumor Killing “Cytotoxic” Domain
PE AAs 253-364, 381-608
➢ Potently toxic to tumor cells with a wide therapeutic window
➢ Simultaneously purges the Tumor Microenvironment (TME) and un-blinds the
immune system to cancer cells
➢ Proven payload efficacy– identical to Medimmune’s anti-CD22 immunotoxin,
Moxetumomab Pasudotox, currently in PhIII trial for Hairy Cell Leukemia1
➢ Reliable, cost-efficient fermentation-based manufacture
1 https://www.medimmune.com/our-therapy-areas/oncology.html
5
MDNA55: Brain Cancer Market Opportunity
Tumor Type Annual Incidence Projected Market
Recurrent Glioblastoma
33,3001 $650M2
(rGB)
Metastatic Brain Cancer 91,5003 $1.30B4
Pediatric Glioma 3,8001 $50M4
TOTAL 133,500 $2.0B
1. Decision Resources Glioblastoma Report, Sept 2013
2. Assumes peak sales for rGB monotherapy and combination therapy at $43K per patient – BioXcel Strategic Analysis Report, 2014
3. Breast, Colon and Kidney Cancer Metastasis to Brain – BioXcel Strategic Analysis Report, 2014
4. Assumes 33% treatable with MDNA55 and priced at $43K per patient - BioXcel Strategic Analysis Report, 2014
6
Current Therapies Do Not Address
Key Challenges
Therapeutic Challenges Rationale for MDNA55
➢ 55% of GBs are chemo-resistant 1 ➢ MDNA55 targets resistant tumors3
➢ Immunosuppressive tumor ➢ IL4R over-expressed in GB and its
microenvironment (TME) TME (Myeloid Derived Suppressor
comprises 40% of GB tumor mass 2 Cells) but not in normal brain 4
➢ Blood Brain Barrier (BBB) blocks ➢ Delivery by direct injection (CED) of
transport of therapeutic to tumor MDNA55 by-passes the BBB
➢ High doses are required due to ➢ Precision delivery achieves high
BBB causing systemic toxicities doses without systemic exposure
1. Hegi ME (2005). N Engl J Med;352(10):997-1003.
2. Kennedy B, et al (2013). J Oncol. Vo; 2013: 486912.
3. Shimamura, et al.(2007.Cancer Res;67:9903-9912.
4. Kohanbash et al (2013).Cancer Res.;73(21):6413-23
7
Compelling Efficacy in Non-Resected rGB
(n=25)
Complete
Response
(CR): 5/25
High
Objective Pre-treatment 9 months
Response
Rate
Partial
Response
(PR): 9/25
Pre-treatment Week 26
Kawakami, et al (2003)
Interleukin-4-Pseudomonas exotoxin chimeric fusion protein
for malignant glioma therapy
Journal of Neuro-Oncology Vol 65 p 15-25
8
MDNA55: Clinical Efficacy
Long Term Survival Results Consistent With Immunotherapy
Benefits
Non-Resectable Recurrent GBM: Superior Long Term Survival When Compared to
Survival of Responders vs Non Responders Avastin Despite Poorer Patient Population (N =57)
Responders (CR + PR):
100
MS = 379 days (n=14)
Percent survival
Non-Responders (SD + PD)
MS = 98 days (n=11)
50
0
0 300 600 900 1200 1500
Days
SD – Stable disease
PD – Progressive disease
Investigators Brochure (page 82)
9
2nd Generation Infusion Will Improve Outcomes
1st Generation CED: Past Studies 2nd Generation CED: Future Studies
• Image-guided
• Inaccurate catheter
catheter placement
placement
• New catheters
• Drug leakage due to
prevent backflow
backflow
• Real-time monitoring
• Inadequate tumor
ensures tumor
coverage
coverage
Images courtesy of John Sampson, Duke University
Real-Time
Monitoring
of Drug
Distribution
Saito and Tominaga (2012), Neurol Med Chir (Tokyo) 52, 531
10Phase 2b Study Design Summary
CED Of MDNA55 in IL4R Up-Regulated GB Patients At First Relapse (COUGAR)
Open-Label Single Arm Study in 43 Patients
Primary Objectives:
ORR
1 2 3 4 SECONDARY
OBJECTIVES:
MOS
Safety
PFS-6
DIAGNOSIS PLANNING ONE TREATMENT FOLLOW-UP
TERTIARY
OBJECTIVES:
Correlate IL4R
Expression with Efficacy
11Efficacy Analysis
Statistical Design and Sample Size
Primary Endpoint: Objective Response Rate (ORR) per modified RANO (Response
Assessment for Neuro-Oncology) Criteria relative to pre-treatment baseline in adult subjects
with GB that has recurred or progressed following standard therapy
Test Hypothesis: Null hypothesis that ORR is 6% (kill) versus the alternative hypothesis
(pursue) that ORR is 18% following treatment with MDNA55. Assumptions regarding primary
end point are based on ORR from previous rGB studies1
Primary Efficacy Analysis: Assessed according to a single-arm, single-stage binomial
design at 1-sided alpha = 0.10. A total of 43 Subjects will provide 80% power.
1 Levin VA, Tonge PJ, Gallo JM, et al. CNS Anticancer Drug Discovery and Development Conference White Paper. Neuro-Oncology, v17:1–26, 2015
12US Sites Participating in the Study
OSU (Columbus, OH)
Cleveland Clinic (Cleveland,
OH)
Weill Cornell +
UCSF MSKCC
(San Francisco, CA) (New York, NY)
JWCI
(Santa Monica, CA)
Duke
(Durham, NC)
UT Southwestern (Dallas, TX)
Marcus Neuroscience
UT San Antonio (San Antonio, TX) Institute
(Boca Raton, FL)
13Future Indications: 1 Million IL4R Cancers Annually
>2000 Patient Biopsies Analyzed Consistently Show IL4R Over-Expression 1-14
B-Cell CLL Hodgkins Lymphoma Biliary Tract Bladder
78% 67% 56% 73%
Breast Colorectal Head and Neck NSCLC
82% 89% 75% 79%
Mesothelioma Ovarian Pancreatic Anaplastic Thyroid
96% 60% 60% 91%
1. BioXcel Strategic Analysis Report, 2014 6. Strome SE, et al (2002).Clin Cancer Res.n;8(1):281-6. 11. Burt, et al (2012) Clin Cancer Res;18(6):1568-77
2. Ishige et al (2008); Int J Cancer;123(12):2915-22. 7. Puri, et al (1996). Cell Immunol.10;171(1):80-6. 12. Kioi, et al (2005) Cancer Res;65(18):8388-96
3. Joshi et al (2014 Cancer Med. 3(6):1615-28. 8. Kawakami, et al (2005) Blood; 105(9): 3707–3713. 13. Kawakami et al (2002) Cancer Res.;62(13):3575-80.
4. P. Leland, et al (2000) Mol Med; 6(3): 165–178. 9. Kay, et al (2005) Leuk Res.;29(9):1009-18. 14. Joshi et al (2015) Discov. Med.;20(111):273-84.
5. Koller , et al (2010); Carcinogenesis 31(6), 1010-17 10. Kawakami, at al (2002). Clin Cancer Res.;8(11):3503-11. 14IL-2 Superkines: Tunable Immune Modulators
Secured Exclusive World Wide Rights from Stanford University
15Deep Early Stage Pipeline
Targets Validated by Multiple Big Pharma Transactions
TARGET AND POTENTIAL RECENT TRANSACTIONS DEAL SIZE
CANDIDATE
MECHANISM INDICATION(S)
IL4/13 Dual Solid Tumors $2 Billion
MDNA413 Super- Respiratory, with $60M
Antagonist Fibrotic and Upfront
Atopic
Diseases
UNDISCLOSED
IL-2 Super- Cancer
MDNA109
Agonist Immunotherapy
UNDISCLOSED
$775M with
IL-2 Super- Autoimmune $300M
MDNA209 Upfront
Antagonist Diseases
$400M with
$150M
16
UpfrontMDNA109 Synergizes With Anti-PD-1
17
Immunotherapy
Combination Therapy Produces Robust Responses
➢ MDNA109 and anti-PD-1 produce limited efficacy alone
➢ Combination treatment sufficient to cure most mice without increased toxicities
1718
Multiple Near Term Value Inflection Milestones
Pursue Accelerated Approval for rGB in 2018
Milestone Estimated Timing
Commenced Enrollment in Phase 2b rGB Trial
First Patient In - Phase 2b rGB Trial
Commence Phase 2 Metastatic Brain Cancer Trial Q4/2017
Complete Enrollment in Phase 2b rGB Trial Q4/2017
Report rGB Phase 2b Interim Top-Line Results Q1/2018
End of Phase 2 Meeting with FDA Q2/2018
Commence IND Enabling Studies with MDNA109 Q2/2018
Pursue Accelerated Approval for rGB Q3/2018
Report Interim Top-Line Results from P2 Metastatic Brain Q3/2018
Cancer Trial
Commence IND Enabling Studies with MDNA57 Q4/2018
19USD$14M Non-Dilutive Grant Validates Platform
Recipient of Cancer Prevention & Research
Institute of Texas (CPRIT) Grant
➢ Diligence by top-tier scientific, clinical, regulatory, chemistry
manufacturing and control, intellectual property & venture capital teams
➢ Solid third-party platform validation
➢ Funds MDNA55 Phase 2b rGB clinical development and next generation
pre-clinical IL-4 Empowered Cytokine program
➢ The USD$14.1M grant effectively provides 2:1 leverage on USD$7M
investment1
➢ Favorable grant repayment terms begin post-launch (low single digit
royalties to a maximum payment amount of 4 times the original grant)
1. http://www.cprit.state.tx.us/images/uploads/rfa-172-txco.pdf
20Use of Cash Resources
Lead Programs Funded Through 2018
Estimated Cost
Cost Center Status by end of 2018
(in Cdn)
MDNA55 Clinical Program $ 14,300,000
- Recurrent Glioblastoma (rGB) Pursue Accelerated Approval
Top-line Phase 2 data
- Metastatic Brain Cancer
MDNA57 and MDNA109 Pre-Clinical Complete PoC Studies: Ready for 2,100,000
Research IND Enabling Studies
General and Administrative On-going 6,000,000
Total Projected Spend $22,400,000
Balance of Grant Revenue from CPRIT US$6.5million to be advanced (8,700,000)
Net Cash required $13,700,000
Cash available (as at 3/31/17)* $14,000,000
*As reported June 15, 2017
21Capitalization
Publicly Listed as of March 3, 2017
➢ Listed on the Toronto Stock Exchange Venture on March 3, 2017 at
$2.00 per share following a successful Reverse Takeover
➢ Trading under the Ticker “MDNA”
➢ Funded for two years with cash on hand and funds remaining to be
advanced under the CPRIT grant
Number
Issued and Outstanding 24,313,334
Fully Diluted* 28,899,096
* Fully diluted includes 3,294,105 warrants with a $2.00 exercise price
and 1,291,657 stock options with a weighted average exercise price of $1.97
22Medicenna Public Company Comparables
All amounts in USD, unless noted otherwise
Price Market Enterprise
Company (Listing/Symbol) Lead Indication (Stage)
(03-Jul-2017) Cap (MM) Value (MM)
ZIOPHARM Oncology, Inc. Breast Cancer (PhII), Recurrent or Progressive
(NASDAQ:ZIOP)
US$6.15 $873.9 $936.8 Glioblastoma (Ph I)(w/ CED*)
Agenus Inc.
(NASDAQ:AGEN)
US$4.00 $396.5 $384.3 Newly Diagnosed Glioblastoma (Ph II)
Tocagen Inc.
(NASDAQ:TOCA)
US$12.12 $240.1 $141.8 Recurrent High Grade Glioma (Ph II)
Stemline Therapeutics, Inc.
(NASDAQ:STML)
US$9.25 $232.3 $142.7 Recurrent Glioblastoma (Ph II)
Newlink Genetics Corporation
(NASDAQ:NLNK)
US$7.50 $219.2 $107.4 Malignant Brain Tumors (Ph II)
Kadmon Holdings, Inc.
(NYSE:KDMN)
US$3.98 $206.4 $234.2 Recurrent Glioblastoma (Ph II)
Vascular Biogenics Ltd.
(NASDAQ:VBLT)
US$4.50 $120.8 $81.2 Recurrent Glioblastoma (Ph III)
Diffusion Pharmaceuticals Inc.
(NASDAQ:DFFN)
US$2.40 $24.8 $15.0 Newly Diagnosed Glioblastoma (Ph II)
Average $289.2 $255.4
Median $225.8 $142.2
Medicenna Therapeutics1 $36.5
C$1.95 $25.7 Recurrent Glioblastoma (Ph II)
(TSXV:MDNA) (C$47.4M)
(1) Medicenna market cap estimate based on current basic shares O/S and current share price. Enterprise value estimate based on net debt as of Mar. 31, 2017
Source: FactSet & Company filings
23Seasoned Management and Experienced Board
Management Team Board of Directors
Fahar Merchant, PhD: Chairman, President & CEO Fahar Merchant, PhD
Former CEO Sophiris Bio (TSX); Former Director, President & CTO at Chairman, President & CEO
KS Biomedix (LSE); Founder, President & CEO of Avicenna Medica
and IntelliGene Expressions Albert Beraldo, CPA, CA
Independent Director
Jay Stoudemire, PhD: Chief Scientific Officer
Founder, President and CEO of Alveda Pharmaceuticals until its
Former VP Preclinical Development, Regulatory, and QA at Mirna
Therapeutics, previously at Genentech, Ascenta, Chugai-Roche, Cytel,
acquisition by Teligent, Inc. (NASDAQ: TLGT); Former President
Genetics Institute, and Xoma and CEO of Bioniche (TSX) and Director of Telesta (TSX); Currently
Independent Director of Helix Biopharma (TSX).
Elizabeth Williams, CPA,CA: Chief Financial Officer
Former VP Finance & Admin and interim CFO at Aptose (TSX and Chandra Panchal, PhD
Nasdaq); Previously with Ernst & Young Independent Director
Founder, Chairman and CEO of Axcelon; Former Co-Founder,
Martin Bexon, MD: Head of Clinical Development President, and CEO of Procyon Biopharma Inc (TSX); Former
Former Medical Director at CSL Behring; Medical Director at Hoffman Senior Executive VP of Business Development at Ambrilia
La Roche (UK and Switzerland)
Biopharma Inc. (TSX)
Nina Merchant, MESc.: Chief Development Officer
Andrew Strong, JD
Former SVP Development at Sophiris Bio; Formerly VP Development
Independent Director
at KS Biomedix (LSE); Previously at Avicenna Medica, IntelliGene,
Partner at Pillsbury Winthrop Shaw Pittman - leading the Life
Pharmacia and Sanofi Pasteur
Sciences Team in Houston, TX. Formerly General Counsel and
Compliance Officer for the Texas A&M University System. Led
Patrick Ward, MBA: Chief Operating Officer formation of bio-manufacturing company, Kalon Biotherapeutics;
Former COO of Aviara Pharma; President/COO at Ocusoft, Executive CEO of Kalon until its sale to FujiFilm Diosynth Biotech. Director of
Director at Encysive Pharma Ashford Hospitality Prime (NYSE)
Shafique Fidai, PhD: Head of Corp Development Nina Merchant, M.E.Sc
Former VP of Business Development at Sophiris Bio; Formerly with Director, Chief Development Officer
Xenon Pharma, Chromos
16World Class Advisors and Collaborators
Clinical & Scientific Advisors Collaborators & Inventors
John Sampson, MD, PhD, MBA Michael Rosenblum, PhD
Duke University: MD Anderson Cancer Center
Principal Investigator and Expert in Drug Delivery to the Brain Head, Immunopharmacology and Targeted Therapy
Collaborator: MDNA57
Sam Denmeade, MD Raj Puri, MD
Johns Hopkins University:
USFDA
Professor of Oncology: Targeted therapies for cancer
Director at CBER
Inventor of MDNA55
Nicholas Butowski, MD
UCSF: Aaron Ring, MD, PhD
Principal Investigator; Novel therapies for brain cancer Yale University
Asst. Prof Immunobiology & Cancer Biology
Guido Kroemer, MD, PhD Co-Inventor of IL-2 Superkines
University of Paris:
Chair: SAB and Expert in Cancer Immunotherapy Chris Garcia, PhD
Stanford University
Ralph Smalling, MSc Co-Inventor of IL-2, IL-4 and IL-13 Superkines
Regulatory Advisor:
Former VP Regulatory Affairs at Amgen; Filed 40 INDs; 5 NDAs Haya Loberboum Galski. PhD
Hebrew University of Jerusalem
Inventor of Fully Human Payloads
25Medicenna: Corporate Highlights
26Thank You one Target: infinite Hope™ www.medicenna.com
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