A Phase 2 company treating Secondary Progressive Multiple Sclerosis. March 2015

Transcription

1 A Phase 2 company treating Secondary Progressive Multiple Sclerosis March 2015

2 Forward Looking Statements This Presentation (and any financial information that may be provided by Innate Immunotherapeutics Limited - the Company ) may contain forward looking statements that involve risks and uncertainties. Such statements include statements regarding the Company s belief or current expectation and are necessarily based on the Company s current understanding of the markets and industries in which it operates. That understanding could change or could prove to be inconsistent with actual developments. The Company s actual results could differ materially from the results discussed in this Presentation, including those anticipated in or implied by any forward looking statements. 2

3 What is multiple sclerosis? Auto-reactive proinflammatory adaptive immune cells cross the blood brain barrier and attack the myelin sheath Triggers inflammatory processes that cause further damage In early disease, myelin is fully or partially repaired Over time, scar-like plaque builds up around damaged axons inhibiting repair (Source: MedicineNet, Inc) 3

4 Living with multiple sclerosis MS is a chronic, disabling disease typically diagnosed between ages 20 and 40 and in three times more women than men MS symptoms include upper and lower extremity disabilities, visual disturbances, balance and coordination problems, spasticity, altered sensation, swallowing disorders, fatigue, bladder and bowel problems, sexual dysfunction, and cognitive and emotional disturbances MS significantly impacts an individual s quality of life and is associated with high costs for patients, their families, and society as a whole MS is associated with reduced life expectancy of between 5 and 15 years MS is the second most costly chronic condition after congestive heart failure 4

5 Unmet medical need & significant market opportunity Relapsing Remitting MS Sufferers worldwide 60% Secondary Progressive MS Sufferers worldwide 30% Number of approved disease modifying drugs 9 Number of approved disease modifying drugs 0 Estimated annual market revenues (2012) US$14B Potential annual market revenues US$3B 5

6 Our lead candidate MIS416 MIS416 is a new generation immuno-modulator MIS416 targets the innate wing of the immune system, specifically myeloid cells MIS416 increases regulatory and anti-inflammatory activity within the immune system Successful modulation of the immune system can result in: Reduced inflammation inside the brain Clearance of myelin debris Myelin repair MIS x 0.5 micron rod shaped microparticles (SEM image of MIS416) Simon Wilkinson, CEO , simon@innateimmuno.com 6

7 Patients / Doctors report significant improvement Compassionate Use programme - 18 SPMS patients treated with MIS416 for 6 months or longer, receiving average of 76 doses over median 30 mths Long term treatment with MIS416 has shown no significant dose intolerance or cumulative toxicity treated patients 83% patients reported significant and sustained improvement in their MS related disabilities and health-related quality of life. 8 new patients provided with MIS416 on compassionate grounds in late 2014 Baseline and quarterly evaluations using most of the Phase 2B tests MIS416 certainly appears beneficial to MS patients, although it remains to be seen if it is curative. However, I would certainly recommend MIS416 as I have seen significant improvement in the motor skills and general wellbeing of my current MS patients. - Christchurch GP Practitioner currently treating ten SPMS patients 7

8 Phase 1B/2A trials (completed) Phase 1B open-label dose escalation study (n=19) completed Oct Weekly dosing from 125 µg to 600 µg for 4 weeks was safe & well tolerated Phase 2A open-label dose confirmation study (n=15) completed July Once weekly dosing at 500 µg for 12 weeks was safe & well tolerated Most common side effects transient self limiting headache, fever, chills, muscle pain and weakness On completion of 3 month Phase 2A study, 80% of subjects showed at least 30% improvement in at least 1 measure of MS clinical status Both studies were conducted with financial support from the US National MS Society (US$550k) and the NZ Government (NZ$600k) 8

9 Phase 2B trial in SPMS (now recruiting) Double blinded, placebo controlled 12 month trial of MIS416 in 90 patients Age 18-70, EDSS , 2 year relapse free, and evidence of progression 2:1 randomisation, once weekly 500 mcg i.v. injection of MIS416 or saline Baseline, quarterly, and end of dosing (EoD) neuromuscular assessments, EDSS, PROs Baseline, month 3 & EoD MRI WBA & MTR The trial has been designed to enable an estimation of the effects seen so that an appropriately powered Phase 3 approval trial can be planned and initiated. No adjustments have been made for multiplicity of outcomes, but success will be judged on consistency of the outcomes rather than purely statistical testing. 9

10 Scientific advisors and collaborators include: Dr Larry Steinman, George A. Zimmerman Professor of Neurology and Neurological Sciences at Stanford University [CyTOF (mass cytometry) platform for analysis of myeloid cell activity] Oleg Butovsky PhD, Center for Neurologic Diseases, Brigham and Women s Hospital (Harvard Medical School, Boston) [major NIH grant recipient for neuro inflammation research - microglia biology] Dr Amit Bar-Or, Professor Neurology and Neurosurgery & Director Experimental Therapeutics Program, Montreal Neurological Institute and Hospital. [MS clinician and MS drug mechanism researcher] Anne La Flamme, A.Prof. Immunology & Cell Biology, Victoria University (Wellington) [MS animal models] Dr David Brown, St Vincent s Centre of Applied Medical Research (Sydney) [neuro inflammation animal models and mechanisms] David Booth, PhD, A.Prof. & MS Australia Principal Research Fellow, Westmead Millennium Institute (Sydney) [MS Genome-Wide Association Study, clinical biomarkers of MS related drug responses] 10

11 Our commercial strategy 30% of all MS patients have SPMS a potential unmet annual market of >US$3B Patents protecting use of MIS416 to treat multiple sclerosis issued in USA and Europe (expiry 2029) Pharma with existing RRMS therapies are actively seeking assets in SPMS We have engaged early & regularly with target acquirers to share Phase 1B/2A data and discuss Phase 2B efficacy trial plans and design Upon completion of Phase 2B (2016) - strategy is to license / sell to Pharma Target Partners or Acquirers 11

12 Intellectual Property (MIS416) Use in Multiple Sclerosis (not limited to SPMS) - NZ & AU (granted) expiry June US 12,636,733 (granted) expiry 2029 (US=75% of market by revenue) - EU (granted) expiry PCT NZ/2010/ Use in Cancer (inc standalone, co-therapy, cancer vaccine adjuvant) - NZ & AU (granted) expiry April PCT/NZ2009/ (national phases in major markets) priority April 2008 Use in Infection (inc prophylactic, therapeutic, vaccine adjuvant) - NZ & AU (granted) expiry April PCT/NZ2009/ (US granted, pending in other major markets) priority April 08 Use in Radiation Exposure (inc pre and post acute or therapeutic exposure) - NZ (granted) - expiry Sept PCT/NZ2009/ (EU Intention to grant; US & Japan pending) 12

13 Active pre-clinical programmes / collaborations include: Ovarian cancer therapeutic vaccine Dr Kunle Odunsi, Professor of Gynaecology & Obstetrics, Roswell Park Cancer Institute (New York) Immune priming for stem cell therapies Dr Kyung-Sung Kang, Kangstem Biotech (Seoul) Nasal route delivery for a non-neurological inflammatory disorder (collaborator and specific indication not disclosed pending IP filing) Treatment for a refractory neurological disorder (collaborator and specific indication not disclosed pending IP filing) 13

14 Key Statistics as at February 2015 ASX Code IIL Share price A$0.20 Shares on issue 172,479,822 Market Cap A$34.5 million Options outstanding 19,698,758 (strike price $0.30 or higher) Loyalty rights 33,031,926 (subject to Phase 2B success) Register Significant Shareholders 18.7% Top % Australian Ethical 9.5% Top % Probe International Inc 3.3% Total shareholders Picton Cove Pty Ltd 2.9% Total small parcel holders <Watkins Family A/C> 2.2% ASX restricted securities National MS Society (USA) 2.0% Voluntary escrow securities Collins Family 2, ,369,703 19,632,865 14

15 Board & Management Michael A Quinn, BSc BEc MBA, Chairman Andrew Sneddon, BEcon, CA Independent Director Elizabeth Hopkins, B.Sc. (Hons) Pharmacology, Independent Director Christopher Collins, B.S, M.B.A. Director Simon Wilkinson Director & Chief Executive Officer Gill Webster PhD Chief Scientific Officer CEO of Sydney based Innovation Capital. Currently or past director of ResMed (ASX, NYSE), QRxPhrama (ASX), & CAP-XX Limited (LSE). Former partner of PWC where he led the life sciences practice. Currently chairman of Elastagen Pty Ltd, MIRteq Pty Ltd, CustomWare Pty Ltd, InterAcct Solutions Pte and TGR BioSciences Pty Ltd. 20 years of experience in successfully commercializing science outcomes. Ten years with Pfizer s European headquarters, including the last two years as Global Project Manager. Currently deputy chair, Christchurch Polytechnic Institute of Technology (CPIT). 30 years of experience in business management. Collins has helped acquire, manage and make profitable 17 companies representing various industries. Collins was elected to the US House of Representatives in Collins holds 19% of IIL. CEO since years experience in finance, banking and business management. He began his civilian career in retail banking after serving as an officer in the Royal New Zealand Navy. CSO since PhD in Immunology from the University of London. Expert in the field of Flow Cytometry systems applied biology. Research publications are in the fields of transplantation and cancer molecular and cellular immunobiology 15

16 Thank you Two clinical trial patients talk about their ongoing experience with MIS416: Simon Wilkinson, CEO