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 Ø developing a novel therapy targeting the Interleukin-4 Receptor (IL4R) biomarker Every year >1 million cancer patients afflicted with IL4R tumors 1 Ø MDNA55 (lead): highly compelling, Phase II clinical data for recurrent Ø glioblastoma (rGB), the most common and aggressive form of brain cancer MDNA55 market opportunity: $650 million in annual sales for rGB; >$2 billion Ø including other brain cancers 1,2 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 Ø 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) Radiotherapy Surgery GB + (85-90%) Diagnosis Chemotherapy 55% of GB Chemo. Chemotherapy Resistant* 25% MDNA55 Treatment Relapse Surgery (Direct infusion into tumor - CED) Add’l Chemo.or Experimental 75% of rGB is non-operable 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 Circularly PE AAs 253-364, 381-608 Catalytic domain of Permuted Pseudomonas Interleukin-4 Exotoxin A (PE) (cpIL-4) 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 Leukemia 1 Ø Reliable, cost-efficient fermentation-based manufacture 1 https://www.medimmune.com/our-therapy-areas/oncology.html 5
MDNA55: Brain Cancer Market Opportunity Tumor T ype Annual Incidence Projected Market Recurrent Glioblastoma 33,300 1 $650M 2 (rGB) Metastatic Brain Cancer 91,500 3 $1.30B 4 Pediatric Glioma 3,800 1 $50M 4 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 Ø 55% of GBs are chemo-resistant 1 Ø MDNA55 targets resistant tumors 3 Ø Immunosuppressive tumor Ø IL4R over-expressed in GB and its microenvironment (TME) comprises TME (Myeloid Derived Suppressor 40% of GB tumor mass 2 Cells) but not in normal brain 4 Ø Blood Brain Barrier (BBB) blocks Ø Delivery by direct injection (CED) of transport of therapeutic to tumor MDNA55 by-passes the BBB Ø High doses are required due to Ø 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 Superior Long Term Survival When Compared to Avastin Non-Resectable Recurrent GBM: Despite Poorer Patient Population (N =57) Survival of Responders vs Non Responders 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
2 nd Generation Infusion Will Improve Outcomes 1 st Generation CED: Past Studies 2 nd 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 10
Phase 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 2 3 4 1 SECONDARY OBJECTIVES: MOS Safety PFS-6 PLANNING DIAGNOSIS ONE TREATMENT FOLLOW-UP TERTIARY OBJECTIVES: Correlate IL4R Expression with Efficacy 11
Efficacy 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 studies 1 Type of Number Number Case number Treatment of Clinical of weighted mean Trials Patients ORR (Range) Cytotoxic 21 1,745 6% (0-17%) Agents Non-Cytotoxic 18 1,239 4% (0-9%) / Non-anti- angiogenic 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 12
US 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 UT San Antonio (San Antonio, Neuroscience TX) Institute ( Boca Raton, FL) 13
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