- ne Target: infinite Hope™
Corporate Presentation Q3, 2017
one Target: infinite Hope Corporate Presentation Q3, 2017 Forward - - PowerPoint PPT Presentation
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,
Corporate Presentation Q3, 2017
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.
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Ø Publicly listed (TSXV: MDNA), clinical-stage, immuno-oncology company developing a novel therapy targeting the Interleukin-4 Receptor (IL4R) biomarker Ø Every year >1 million cancer patients afflicted with IL4R tumors1 Ø MDNA55 (lead): highly compelling, Phase II clinical data for recurrent glioblastoma (rGB), the most common and aggressive form of brain cancer Ø MDNA55 market opportunity: $650 million in annual sales for rGB; >$2 billion including other brain cancers1,2 Ø MDNA55 has Orphan Drug (FDA, EMA) & Fast Track Designations (FDA) Ø Exciting pre-clinical IL-2, IL-4 and IL-13 Superkine platform Ø Well funded with $14M US non-dilutive grant and $14M CAD Private Placement Ø Seasoned management with technology platform protected by 12 patent families
1. BioXcel Strategic Analysis Report, 2014. 2. Decision Resources, Inc Glioblastoma Report, Sept 2013
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Surgery (85-90%) GB
Diagnosis
Radiotherapy + Chemotherapy Relapse Chemotherapy Surgery
(Direct infusion into tumor - CED)
Add’l Chemo.or Experimental Therapies
* Expression of the DNA repair protein O6-methylguanine-DNA methyltransferase (MGMT) is responsible for resistance to alkylating agents used in GB treatment.
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Ø Potently toxic to tumor cells with a wide therapeutic window Ø Simultaneously purges the Tumor Microenvironment (TME) and un-blinds the immune system to cancer cells Ø Proven payload efficacy– identical to Medimmune’s anti-CD22 immunotoxin, Moxetumomab Pasudotox, currently in PhIII trial for Hairy Cell Leukemia1 Ø Reliable, cost-efficient fermentation-based manufacture
1 https://www.medimmune.com/our-therapy-areas/oncology.html
PE AAs 253-364, 381-608
Circularly Permuted Interleukin-4 (cpIL-4) Catalytic domain of Pseudomonas Exotoxin A (PE)
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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
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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
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Pre-treatment 9 months Pre-treatment Week 26
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Kawakami, et al (2003) Interleukin-4-Pseudomonas exotoxin chimeric fusion protein for malignant glioma therapy Journal of Neuro-Oncology Vol 65 p 15-25
Superior Long Term Survival When Compared to Avastin Despite Poorer Patient Population (N =57)
300 600 900 1200 1500 50 100
Days Percent survival
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 – Stable disease PD – Progressive disease Investigators Brochure (page 82)
Images courtesy of John Sampson, Duke University
placement
backflow
coverage
catheter placement
prevent backflow
ensures tumor coverage
Saito and Tominaga (2012), Neurol Med Chir (Tokyo) 52, 531 10
Open-Label Single Arm Study in 43 Patients
Primary Objectives:
ORR
SECONDARY OBJECTIVES:
MOS Safety PFS-6
TERTIARY OBJECTIVES:
Correlate IL4R Expression with Efficacy
DIAGNOSIS ONE TREATMENT FOLLOW-UP
PLANNING
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CED Of MDNA55 in IL4R Up-Regulated GB Patients At First Relapse (COUGAR)
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.
Type of Treatment Number
Trials Number
Patients Case number weighted mean ORR (Range) Cytotoxic Agents 21 1,745 6% (0-17%) Non-Cytotoxic / Non-anti- angiogenic 18 1,239 4% (0-9%)
1 Levin VA, Tonge PJ, Gallo JM, et al. CNS Anticancer Drug Discovery and Development Conference White Paper. Neuro-Oncology, v17:1–26, 2015
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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)
78% 78%
B-Cell CLL
67%
Hodgkins Lymphoma
56%
Biliary Tract
73% 73%
Bladder
82% 82%
Breast
89% 89%
Colorectal
75% 75%
Head and Neck Head and Neck
79% 79%
NSCLC NSCLC
96% 96%
Mesothelioma Mesothelioma
60% 60%
Ovarian Ovarian
60% 60%
Pancreatic ancreatic
91% 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.
5. Koller , et al (2010); Carcinogenesis 31(6), 1010-17
7. Puri, et al (1996). Cell Immunol.10;171(1):80-6. 8. Kawakami, et al (2005) Blood; 105(9): 3707–3713. 9. Kay, et al (2005) Leuk Res.;29(9):1009-18.
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TARGET AND MECHANISM CANDIDATE POTENTIAL INDICATION(S)
MDNA109 IL-2 Super- Agonist Cancer Immunotherapy Autoimmune Diseases IL-2 Super- Antagonist MDNA209 Solid Tumors Respiratory, Fibrotic and Atopic Diseases IL4/13 Dual Super- Antagonist MDNA413
RECENT TRANSACTIONS DEAL SIZE
$2 Billion with $60M Upfront
UNDISCLOSED
$775M with $300M Upfront
UNDISCLOSED
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Ø MDNA109 and anti-PD-1 produce limited efficacy alone Ø Combination treatment sufficient to cure most mice without increased toxicities
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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 Cancer Trial Q3/2018 Commence IND Enabling Studies with MDNA57 Q4/2018
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Cost Center Status by end of 2018 Estimated Cost (in Cdn)
MDNA55 Clinical Program
Pursue Accelerated Approval Top-line Phase 2 data
$ 14,300,000 MDNA57 and MDNA109 Pre-Clinical Research
Complete PoC Studies: Ready for IND Enabling Studies
2,100,000 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
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* 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
Company (Listing/Symbol) Price
(03-Jul-2017)
Market Cap (MM) Enterprise Value (MM) Lead Indication (Stage) ZIOPHARM Oncology, Inc.
(NASDAQ:ZIOP)
US$6.15 $873.9 $936.8
Breast Cancer (PhII), Recurrent or Progressive 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
(TSXV:MDNA)
C$1.95 $36.5
(C$47.4M)
$25.7
Recurrent Glioblastoma (Ph II)
(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
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All amounts in USD, unless noted otherwise
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
Jay Stoudemire, PhD: Chief Scientific Officer
Former VP Preclinical Development, Regulatory, and QA at Mirna Therapeutics; Previously at Genentech, Ascenta, Chugai-Roche, Cytel, Genetics Institute, and Xoma
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
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John Sampson, MD, PhD, MBA Duke University:
Principal Investigator and Expert in Drug Delivery to the Brain
Sam Denmeade, MD Johns Hopkins University:
Professor of Oncology: Targeted therapies for cancer
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
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
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