Professor of Neurosurgery at Stanford University;
Inventor of MDNA55
Sam Denmeade, MD
Johns Hopkins University:
Chief Scientific Officer; Expert in Prostate Cancer Therapeutics
David M. Nanus, MD
Memorial Sloan Kettering Cancer Center:
Expert in Immunotherapy for Solid Tumors
David Spetzler, MD, PhD
CSO, Xencor:
Expert in Protein Engineering and Antibody Therapeutics
John Sampson, MD, PhD
Duke University:
Principal Investigator and Expert in Drug Delivery to the Brain
17
2. Forward Looking Statements
Certain statements in this presentation are âforward-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 âexpectâ, âseekâ, âendeavourâ, âanticipateâ, âplanâ, âestimateâ,
âbelieveâ, âintendâ, 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 âforward-looking statementsâ. 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 filing statement dated
February 27, 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
3. Medicenna: Corporate Highlights
Ă Publicly listed (TSXV: MDNA), clinical-stage, immuno-oncology company
developing a novel therapy targeting the Interleukin-4 Receptor (IL4R) biomarker
Ă Every year >1 million cancer patients afflicted with IL4R tumors1
Ă MDNA55 (lead): highly compelling, Phase II clinical data for recurrent
glioblastoma (rGB), the most common and aggressive form of brain cancer
Ă MDNA55 market opportunity: $650 million in annual sales for rGB; >$2 billion
including other brain cancers1,2
Ă MDNA55 has Orphan Drug (FDA, EMA) & Fast Track Designations (FDA)
Ă Exciting pre-clinical IL-2, IL-4 and IL-13 Superkine platform
Ă Well funded with $14M US non-dilutive grant and $14M CAD Private Placement
Ă 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
4. Treatment Pathway for Glioblastoma (GB)
Surgery
(85-90%)
GB
Diagnosis
Radiotherapy
+
Chemotherapy
Relapse
Chemotherapy
SurgeryMDNA55 Treatment
(Direct infusion into tumor - CED)
Addâl Chemo.or
Experimental
Therapies
GB is uniformly fatal; virtually all tumors will recur (rGB)
55% of GB
Chemo.
Resistant*
* Expression of the DNA repair protein O6-methylguanine-DNA methyltransferase (MGMT) is
responsible for resistance to alkylating agents used in GB treatment.
25%
75% of rGB is non-operable
4
5. MDNA55: Targeted Dual-Action Immunotherapeutic
A Powerful Molecular Trojan Horse
Ă Potently toxic to tumor cells with a wide therapeutic window
Ă Simultaneously purges the Tumor Microenvironment (TME) and un-blinds the
immune system to cancer cells
Ă Proven payload efficacyâ identical to Medimmuneâs anti-CD22 immunotoxin,
Moxetumomab Pasudotox, currently in PhIII trial for Hairy Cell Leukemia1
Ă Reliable, cost-efficient fermentation-based manufacture
1 https://www.medimmune.com/our-therapy-areas/oncology.html
PE AAs 253-364, 381-608Circularly
Permuted
Interleukin-4
(cpIL-4)
Catalytic domain of
Pseudomonas
Exotoxin A (PE)
Tumor Targeting Domain Tumor Killing âCytotoxicâ Domain
5
6. MDNA55: Brain Cancer Market Opportunity
Tumor Type Annual Incidence Projected Market
Recurrent Glioblastoma
(rGB)
33,3001 $650M2
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 â BioXcel Strategic Analysis Report, 2014
3. Breast, Colon and Kidney Cancer Metastasis to Brain â BioXcel Strategic Analysis Report, 2014
4. Assumes 33% treatable with MDNA55 and priced at $43K per patient - BioXcel Strategic Analysis Report, 2014
6
7. Current Therapies Do Not Address
Key Challenges
Therapeutic Challenges Rationale for MDNA55
Ă 55% of GBs are chemo-resistant 1
Ă Immunosuppressive tumor
microenvironment (TME) comprises
40% of GB tumor mass 2
Ă Blood Brain Barrier (BBB) blocks
transport of therapeutic to tumor
Ă High doses are required due to
BBB causing systemic toxicities
Ă MDNA55 targets resistant tumors3
Ă IL4R over-expressed in GB and its
TME (Myeloid Derived Suppressor
Cells) but not in normal brain 4
Ă Delivery by direct injection (CED) of
MDNA55 by-passes the BBB
Ă Precision delivery achieves high
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
8. Compelling Efficacy in Non-Resected rGB (n=25)
Pre-treatment 9 months
Pre-treatment Week 26
Complete
Response
(CR): 5/25
Partial
Response
(PR): 9/25
High
Objective
Response
Rate
8
Kawakami, et al (2003)
Interleukin-4-Pseudomonas exotoxin chimeric fusion protein
for malignant glioma therapy
Journal of Neuro-Oncology Vol 65 p 15-25
9. MDNA55: Clinical Efficacy
Long Term Survival Results Consistent With Immunotherapy Benefits
Superior Long Term Survival When Compared to Avastin
Despite Poorer Patient Population (N =57)
0 300 600 900 1200 1500
0
50
100
Days
Percentsurvival
Non-Resectable Recurrent GBM:
Survival of Responders vs Non Responders
Responders (CR + PR):
MS = 379 days (n=14)
Non-Responders (SD + PD)
MS = 98 days (n=11)
9
SD â Stable disease
PD â Progressive disease
Investigators Brochure (page 82)
10. 2nd Generation Infusion Will Improve Outcomes
Images courtesy of John Sampson, Duke University
âą Inaccurate catheter
placement
âą Drug leakage due to
backflow
âą Inadequate tumor
coverage
âą Image-guided
catheter placement
âą New catheters
prevent backflow
âą Real-time monitoring
ensures tumor
coverage
Real-Time
Monitoring of
Drug
Distribution
1st Generation CED: Past Studies 2nd Generation CED: Future Studies
Saito and Tominaga (2012), Neurol Med Chir (Tokyo) 52, 531
10
11. NeuroExactâą: Personalized Molecular Neurosurgery
Combining Personalized Therapy with Precision Delivery
DRUG: MDNA55 Selectively Targets Tumor
Over-Expressing the IL4R
DELIVER: Precise Image Guided Convection
Enhanced Delivery of MDNA55
DISTRIBUTE: Ensure Complete Tumor
Coverage with MDNA55 Using Real Time
Monitoring
11
12. 12
US Sites Participating in the Study
OSU (Columbus, OH)
Cleveland Clinic (Cleveland,
OH)
Weill Cornell +
MSKCC
(New York, NY)
Duke
(Durham, NC)
UT Southwestern (Dallas, TX)
UT San Antonio (San Antonio,
TX)
UCSF
(San Francisco, CA)
JWCI
(Santa Monica, CA)
Marcus
Neuroscience
Institute
(Boca Raton, FL)
13. Future Indications: 1 Million IL4R Cancers Annually
>2000PatientBiopsiesAnalyzedConsistentlyShowIL4ROver-Expression1-14
78%
B-Cell CLL
67%
Hodgkins Lymphoma
56%
Biliary Tract
73%
Bladder
82%
Breast
89%
Colorectal
75%
Head and Neck
79%
NSCLC
96%
Mesothelioma
60%
Ovarian
60%
Pancreatic
91%
Anaplastic Thyroid
1. BioXcel Strategic Analysis Report, 2014
2. Ishige et al (2008); Int J Cancer;123(12):2915-22.
3. Joshi et al (2014 Cancer Med. 3(6):1615-28.
4. P. Leland, et al (2000) Mol Med; 6(3): 165â178.
5. Koller , et al (2010); Carcinogenesis 31(6), 1010-17
6. Strome SE, et al (2002).Clin Cancer Res.n;8(1):281-6.
7. Puri, et al (1996). Cell Immunol.10;171(1):80-6.
8. Kawakami, et al (2005) Blood; 105(9): 3707â3713.
9. Kay, et al (2005) Leuk Res.;29(9):1009-18.
10. Kawakami, at al (2002). Clin Cancer Res.;8(11):3503-11.
11. Burt, et al (2012) Clin Cancer Res;18(6):1568-77
12. Kioi, et al (2005) Cancer Res;65(18):8388-96
13. Kawakami et al (2002) Cancer Res.;62(13):3575-80.
14. Joshi et al (2015) Discov. Med.;20(111):273-84.
13
14. IL-2 Superkines: Tunable Immune Modulators
RECENT IL-2 DEAL ACTIVITY
IL-2 Agonist: MDNA109
Clinical collaboration for NKTR-214 in
combination with Opdivo
RECENT IL-2 DEAL ACTIVITY
IL-2 Antagonist: MDNA209
Acquired by Celgene for $300M upfront and
$475M in contingent value rights
14
Secured Exclusive World Wide Rights from Stanford University
15. MDNA109 Synergizes With Anti-PD-1 Immunotherapy
Combination Therapy Produces Robust Responses
Ă MDNA109 and anti-PD-1 produce limited efficacy alone
Ă Combination treatment sufficient to cure most mice without increased toxicities
15
15
17. USD$14M Non-Dilutive Grant Validates Platform
Ă Diligence by top-tier scientific, clinical, regulatory, chemistry
manufacturing and control, intellectual property & venture capital teams
Ă Solid third-party platform validation
Ă Funds MDNA55 Phase 2b rGB clinical development and next generation
pre-clinical IL-4 Empowered Cytokine program
Ă The USD$14.1M grant effectively provides 2:1 leverage on USD$7M
investment1
Ă 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
Recipient of Cancer Prevention & Research
Institute of Texas (CPRIT) Grant
15
18. 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 Q3/2017
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
Cancer Trial
Q3/2018
Commence IND Enabling Studies with MDNA57 Q4/2018
18
19. Seasoned Management and Experienced Board
Management Team
Fahar Merchant, PhD: Chairman, President & CEO
Former CEO Sophiris Bio (TSX); Former Director, President & CTO at
KS Biomedix (LSE); Founder, President & CEO of Avicenna Medica
and IntelliGene Expressions
Sam Denmeade, MD: Chief Scientific Officer
Prof Oncology at JHU; Former CSO at Sophiris Bio; Co-founder and
Chief Clinical Advisor of Inspyr Therapeutics
Elizabeth Williams, CPA,CA: Chief Financial Officer
Former VP Finance & Admin and interim CFO at Aptose (TSX and
Nasdaq); Previously with Ernst & Young
Martin Bexon, MD: Head of Clinical Development
Former Medical Director at CSL Behring; Medical Director at Hoffman
La Roche (UK and Switzerland)
Nina Merchant, MESc.: Chief Development Officer
Former SVP Development at Sophiris Bio; Formerly VP Development
at KS Biomedix (LSE); Previously at Avicenna Medica, IntelliGene,
Pharmacia and Sanofi Pasteur
Patrick Ward, MBA: Chief Operating Officer
Former COO of Aviara Pharma; President/COO at Ocusoft, Executive
Director at Encysive Pharma
Shafique Fidai, PhD: Head of Corp Development
Former VP of Business Development at Sophiris Bio; Formerly with
Xenon Pharma, Chromos
Board of Directors
Fahar Merchant, PhD
Chairman, President & CEO
Albert Beraldo, CPA, CA
Independent Director
Founder, President and CEO of Alveda Pharmaceuticals until its
acquisition by Teligent, Inc. (NASDAQ: TLGT); Former President
and CEO of Bioniche (TSX) and Director of Telesta (TSX); Currently
Independent Director of Helix Biopharma (TSX).
Chandra Panchal, PhD
Independent Director
Founder, Chairman and CEO of Axcelon; Former Co-Founder,
President, and CEO of Procyon Biopharma Inc (TSX); Former
Senior Executive VP of Business Development at Ambrilia
Biopharma Inc. (TSX)
Andrew Strong, JD
Independent Director
Partner at Pillsbury Winthrop Shaw Pittman - leading the Life
Sciences Team in Houston, TX. Formerly General Counsel and
Compliance Officer for the Texas A&M University System. Led
formation of bio-manufacturing company, Kalon Biotherapeutics;
CEO of Kalon until its sale to FujiFilm Diosynth Biotech. Director of
Ashford Hospitality Prime (NYSE)
Nina Merchant, M.E.Sc
Director, Chief Development Officer
16
20. World Class Advisors and Collaborators
Collaborators & InventorsClinical & Scientific Advisors
John Sampson, MD, PhD, MBA
Duke University:
Principal Investigator and Expert in Drug Delivery to the Brain
Stewart Grossman, MD
Johns Hopkins University:
Novel therapies for primary & metastatic brain tumors
Nicholas Butowski, MD
UCSF:
Principal Investigator; Novel therapies for brain cancer
Guido Kroemer, MD, PhD
University of Paris:
Chair: SAB and Expert in Cancer Immunotherapy
Ralph Smalling, MSc
Regulatory Advisor:
Former VP Regulatory Affairs at Amgen; Filed 40 INDs; 5 NDAs
Michael Rosenblum, PhD
MD Anderson Cancer Center
Head, Immunopharmacology and Targeted Therapy
Collaborator: MDNA57
Raj Puri, MD
USFDA
Director at CBER
Inventor of MDNA55
Aaron Ring, MD, PhD
Yale University
Asst. Prof Immunobiology & Cancer Biology
Co-Inventor of IL-2 Superkines
Chris Garcia, PhD
Stanford University
Co-Inventor of IL-2, IL-4 and IL-13 Superkines
Haya Loberboum Galski. PhD
Hebrew University of Jerusalem
Inventor of Fully Human Payloads
19
21. Capitalization
20
Publicly Listed as of March 3, 2017
Ă Listed on the TSX Venture on March 3, 2017 at $2.00 per share
following a successful Reverse Takeover
Ă Trading under the Ticker âMDNAâ
Ă Fully funded for two years with cash on hand and funds remaining to
be advanced under the CPRIT grant
Number
Issued and Outstanding 24,307,343
Fully Diluted* 28,852,583
* Fully diluted includes 4,402,383 options and warrants with a $2.00 exercise price
and 142,857 options and warrants with a $1.40 exercise price
22. Medicenna Public Company Comparables
Company (Listing/Symbol) Price
(10-Mar-17)
Market
Cap (MM)
Enterprise
Value (MM)
Lead Indication (Stage)
ZIOPHARM Oncology, Inc.
(NASDAQ:ZIOP)
US$6.13 $811.5 $855.7
Breast Cancer (PhII), Recurrent Glioblastoma
(Ph I)(w/ CED*)
Newlink Genetics Corporation
(NASDAQ:NLNK)
US$17.54 $512.5 $387.5 Malignant Brain Tumor (Ph II)
Agenus Inc.
(NASDAQ:AGEN)
US$3.35 $329.4 $364.9 Glioblastoma and Recurrent Glioblastoma (Ph II)
Stemline Therapeutics, Inc.
(NASDAQ:STML)
US$8.25 $206.7 $159.8 Recurrent Glioblastoma (Ph I/II)
Kadmon Holdings, Inc.
(NYSE:KDMN)
US$3.21 $166.4 $199.2 Glioblastoma (Ph II)
Vascular Biogenics Ltd.
(NASDAQ:VBLT)
US$5.35 $143.6 $98.4 Recurrent Glioblastoma (Ph III)
Diffusion Pharmaceuticals Inc.
(NASDAQ:DFFN)
US$3.70 $38.3 $37.7 Glioblastoma (Ph II)
Average $315.5 $300.5
Median $206.7 $199.2
Medicenna Therapeutics1
(TSXV:MDNA)
C$2.80 $53.0
(C$70.5M)
$46.0 Recurrent Glioblastoma (Ph II)
(1)Enterprise value based on net debt as of Dec 31, 2016
21
All amounts in USD, unless noted otherwise