Presentation at DDF Company Forum May 26, CFO / IR Rein Piir Development Disa Böttiger

Transcription

1 Presentation at DDF Company Forum May 26, 2005 CFO / IR Rein Piir Development Disa Böttiger

2 Introduction

3 Skilled and experienced management Anders Vedin M.D., Professor Chairman of the Board Previous positions include President of Astra Hässle Lars Adlersson B.Sc. (Business Adm & Econ) CEO Previous positions include Vice President and General Manager GlaxoSmithKline Austria, Managing Director GlaxoWellcome Sweden. Johan Harmenberg M.D. Associate Professor VP Drug Development Previous positions include Clinical Research Director, Medical Advisor and Medical Director at Roche, Astra and Pharmacia &Upjohn. Bertil Samuelsson Ph.D. Professor VP Discovery Research Previous positions include Head of Medical Chemistry at AstraZeneca. Rein Piir B.Sc. (Business Adm & Finance) CFO / IR Previous positions include Head of Research and Health Care at Carnegie Investment Banking Health Care and Strategy at Alecta Asset Management Paul Wallace - Ph.D. VP Business Development Previous positions include Business Development Manager at Peptide Therapeutics and Direct Research at Edagen.

4 Medivir in brief Develops pharmaceuticals targeting many different therapeutic areas with high market potential. Approaching a leading position in protease research 12 projects in pipeline and a broad range of early activities in pre-clinical discovery Well risk balanced pipeline 9 partnerships with 8 companies and a broad network with academia in Europe and US Nordic marketing rights for all projects Located in Stockholm (Sweden) and Cambridge (UK) with approximately 110 employees in R&D

5 ocused research addressing multiple therapeutic indication Proteases gaining momentum in terms of portfolio value over time Herpes Simplex HIV Rheumatoid arthritis Multiple sclerosis Diabetes ngles Wide range of therapeutic options based on polymerase and protease platforms Asthm COPD ymerase tease Pain Osteoporosis Bacterial infections Hepatitis Hepatitis

6 Pipeline May 2005 ativ Lead Optimization Pre-clinical IND Phase Phase Phase NDA e Identification phase Development I II III aciclovir (Shingles) 9 (Labial Herpes) dine (HIV) 10 (HIV) 50 (HIV) Population Council 60 (MV026048) (HIV) 70 (HIV) t A (Cath S) (Osteoporosis) t B (Cath S) rot (Hepatitis C) ol (Hepatitis C) tes Polymerase inhibitor Polymerase inhibitor Protease inhibitor No internal development imer se

7 The strategic journey

8 Medivir: The strategic journey Research areas Projects herapeutic areas Resources Polymerase inhibitors 8 Herpes virus HIV ~ 40 employees Protease inhibitors Polymerase inhibitors 12 Autoimmune disorders Osteoporosis Hepatitis C Herpes virus HIV ~ 130 employees

9 The result from five years protease research ase: Lead Lead Pre-clinical identification optimization development Diabetes 1) Autoimmune disorders (Project B) Autoimmune disorders (Project A) 3) Alzheimer Hepatitis C 3) Osteoporosis COPD 2) Proof of Cash 4) CD selection recently done 1) Collaboration with Biovitrum 2) Collaboration with Hengrui 3) Collaboration with Peptimmune 4) Collaboration with Johnson&Johnson / Tibotec

10 HCV deal with Tibotec (J&J) giving guidance concerning the value of the proteases HCV (protease) deal with Tibotec based on 4 important building blocks Total deal value: EUR 68.5m of which EUR 6.5m in upfront Substantial financial research support Medivir has retained the marketing rights to the Nordic countries and will receive royalty from global sales Possible quid for the Nordic market from J&J portfolio with a predefined profile

11 A broader clinical portfolio will create higher value e New therapeutic area Osteoporosis Autoimmune disorders Hepatitis C Herpes & HIV Herpes & HIV T

12 Operational focus

13 Operational focus Invest, develop and bring the protease based projects closer t clinical development. Out-license these as franchise deals, lik in the Tibotec deal for HCV-Protease. Continue to seek for quids in future partnerships and deals. Secure partnership for ME-609 and start phase III trials. Secure medium term funding by FTE support or fixed fees as the Tibotec deal. Explore partnership structures in the HIV field (MIV-210, MIV- 310 and the NNRTI programs).

14 Four ways of creating revenues and stability Possible quids and/or Co promotion RP-606 ME 609 Cath S Cath K HIV-portfolio Milestones Royalties Medivir In-house product sales RP 606 HIV-portfolio HCV Prot HCV Pol J & J Quid MIV 160 MV Acquisitions Pure Acquisitions

15 Financial status 31 March 2005 (31 March 2004) Result after financial items: MSEK (MSEK -45.4) Cash: MSEK (MSEK 207.5) 2005 net R&D expenditure will amount to MSEK 165. Lower than previous years Market value MSEK 750 Shareholders as % of capital Founders 10% Private individuals Swedish institutions 8% 35% European institutions Retail 15% 32%

16 Pre-clinical research and Clinical development at Medivir The majority of the pre-clinical resources are invested in protease based projects

17 Pipeline May 2005 ativ Lead Optimization Pre-clinical IND Phase Phase Phase NDA e Identification phase Development I II III aciclovir (Shingles) 9 (Labial Herpes) dine (HIV) 10 (HIV) 50 (HIV) Population Council 60 (MV026048) (HIV) 70 (HIV) t A (Cath S) (Osteoporosis) t B (Cath S) rot (Hepatitis C) ol (Hepatitis C) tes Polymerase inhibitor Polymerase inhibitor Protease inhibitor No internal development imer se

18 Cathepsin S inhibitor

19 The Cathepsin S project (JV with Peptimmune) Mode of action: Inhibitors of Cathepsin S are expected to prevent or reduce activation o certain T cells. Potential indications: Autoimmune disorders (Rheumatoid arthritis (RA), Multiple sclerosis (MS) Allergic reactions (Asthma) Transplantation (GVHD) Chronic pain Competitive advantages: New class of drugs for treating autoimmune and inflammatory diseases. Competitors: Pre-clinical research: Novartis (chronic pain), Celera, GSK, BI No competing projects identified in clinical development.

20 Antigen presenting cells activate T-cells CD4 T cell Activated CD4 T cell B cell Cytokines Disease Pathology e.g. RA & MS MHC Class II APC Macrophage CD8 T cell CD4 T cells control immune response to antigen

21 The Cathepsin S project (JV with Peptimmune) Project status: First Candidate Drug (CD) selected during 2004 Inhibitor class demonstrate efficacy in diseases models in vivo Pre-clinical safety and efficacy studies ongoing Strong progress for new Cathepsin S program B, based on novel SAR developed and extensive DMPK profiling Extensive IP on Program A and B NCEs have been filed

22 Cathepsin K inhibitor

23 Osteoporosis A common disease 1 out of 2 women and 1 out of 8 men will have an osteoporosis related fracture In the US, national expenditure amounts to USD 13.8 billion Major cause of death and disability

24 Cathepsin K inhibitor drug changing the treatment paradigms in osteoporosis Cathepsin K is selectively expressed in the osteoclasts and plays a pivotal role in the degradation of bone matrix Attractive new mechanism of action Potential for bone forming activity and reduced fracture incidence Once-daily oral treatment No gastrointestinal upset and a good tolerability profile Suitable for broad patient populations and both genders Primary indication: Osteoporosis Osteoporosis: The amount of treated patients is expected to grow from 7.8m (2002) to 18.3m (2015)* Market size 2001: USD 5.5bn (2001), expected to grow to USD 11bn (2008)** *Datamonitor, Epidatabase (2003)

25 Bone a living organ! Human osteoclast cells -breaking down the bone (as pictured below) Human osteoblast cells- remodelling and building new bone

26 Project status in Medivir s Cathepsin K program Highly drugable Cathepsin K inhibitors have been developed by Medivir Excellent potency and cell based efficacy in human bone resorption model Highly favourable DMPK properties Candidate Drug selected recently Strong IP on NCE s have been filed A unique human osteoclast resorption assay gives competitive advantage Proof-of-concept for Cathespin K inhibitor from clinical phase I an IIa study by Novartis New clinical surrogate endpoints will allow clinical efficacy assessments after only days of treatment Medivir has a leading position in the Cathepsin K inhibitor area

27 Potential Secondary Indications Rheumatoid Arthritis Bisposphonates are not active Prevention of joint destruction Accumulation of collagen in synovial fibroblasts in humans with cath K deficiency Osteoarthritis - Bisphosphonates are not active Larger market than RA Bone metastasis Cath K expressed by mamary tumour cells & contributes to invasive potential Big market with little competition Hypercalcemia Paget s Disease Patients on steroid treatment suffer bone loss

28 Medivir HCV programs

29 HCV- The basics and disease facts 75-85% of people infected with HCV will become chronically infected Leading cause of liver transplantation (1,000/year in USA) Some estimates suggest that yearly cost of HCV could rise to much as $9 billion in US Three classical enzyme targets Polymerase inhibitors in phase I Protease inhibitors in phase I Helicase - research Early success is being reported for polymerase and protease. latest news concern the Vertex protease based compound VXin clinical phase Ib *Enligt Data Monitor

30 The Hepatitis C (HCV) market and Medivir HCV Polymerase inhibitor Medivir - Roche collaboration announced November 2003 Discovery program for the development of nucleoside pol inhibitors Highly promising results delivered in the program R&D support, milestone and royalties Medivir has kept the Nordic marketing rights HCV Protease inhibitor Clear SAR established Three advanced series being pursued Protease inhibitors exhibit Ki < 1 nm Key inhibitors exhibit high potency in the cell based subgenomic replication assay with IC 50 < 10 nm Extensive non-limiting IP filed during 2004 Rapid progress to Candidate Drug expected

31 Hepatitis Market Forecast to 2010* Sales Forecast ($m) 9,000 8,000 7,000 6,000 5,000 4,000 3,000 2,000 1, Source: Datamonitor Year Interferons Antivirals Immunomodulatory A major and growing market with high unmet medical need

32 Herpes

33 Valomaciclovir (RP 606) Indication: Shingles Competitive advantage: Expected to reduce chronic pain IP protection: Patents to 2017 Partner: Reliant (1.000 sales reps in US) Status: Ongoing discussions with FDA for further clinical development xpected to reduce chronic pain

34 The chronic pain (PHN), is the major problem for shingles patients re of patients Acute phase Chronic pain time expected to reduce chronic pain

35 ME 609 Indication: Competitive advantages: IP protection: Partner: Status: Labial and genital herpes First drug to prevent labial lesions Patents to 2016 in US and EU No Positive phase II data Preparation prior to phase III ongoing in parallel with partnership discussions id your next cold sore

36 You can avoid your next cold sore No treatment 0% xisting drugs 0% ME % ided outbreaks 0% 5% 10% 15% 20% 25% 30% oid your next cold sore

37 HIV

38 Alovudine (MIV 310) Indication: Competitive advantages: IP protection: Status: HIV No resistance development observed in phase IIa, or in vitro Unique multi-resistance profile. Once daily oral dosing Exceptionally low COGS Patents to 2019 (USA) Phase II studies showed unique activity against multi-drug resistant HIV Project parked

39 MIV 210 Indication: Competitive advantages: HIV, Hepatitis B Unique multi-resistance profile, different from MIV-310 Excellent oral bioavailability in humans Low COGS IP protection: Patents to 2018 Status: Phase II POC study to start Q3

40 The Late Stage Preclinical Pipeline iscovery Lead Optimisation Late preclinical IND Phase I Phase II Phase III NDA M identification phase development IV-160 (MV026048) ath S (Project A) ath K IV-170 ath S (Project B) CV CV OPD iabetes lzheimer Strong presence in hepatitis C A new treatment paradigm for RA, MS and asthma. Possible improvements over TNF-alpha blockers. New treatment paradigm for osteoporosis and osteoarthritis. Present therapies like, bisphosphonates and HRT all have limitations Polymerase inhibitor Protease inhibitor

41 Strong pipeline Summary Many projects, some with potential for first-in-class treatment principles Well balanced risk profile Project portfolio Strong balance sheet Cost efficient organisation R&D activities are well functioning and efficient Medivir works disciplined in accordance with a well thought-through strateg High business activity to be expected in order to execute the ambition to establish Medivir Pharma