MolMed S.p.A. Investor presentation

Transcription

1 an integrated strategy to cure cancer MolMed S.p.A. Investor presentation BIO CEO & Investor Conference, New York (USA) 9 February 2010 Claudio Bordignon Chairman & CEO

2 Forward-looking statements This presentation may contain certain forward-looking statements. Although the Company believes its expectations are based on reasonable assumptions, these forward-looking statements are subject to numerous risks and uncertainties, including scientific, business, economic and financial factors, which could cause actual results to differ materially from those anticipated in the forward-looking statements. The company assumes no responsibility to update forward-looking statements or adapt them to future events or developments. This presentation does not constitute an offer or invitation to subscribe or purchase any securities of MolMed S.p.A. Declaration by the official Corporate Financial Reporting Manager: The undersigned herewith attests, pursuant to Article 154-bis, paragraph 2 of the Italian Consolidated Law on Finance (Legislative Decree 58/1998), that the accounting disclosure contained in this presentation matches documentary evidence, corporate books, and accounting records. Enrico Cappelli, Chief Financial Officer, official Corporate Financial Reporting Manager MolMed S.p.A. Company presentation, Bio CEO & Investor Conference, 9 February

3 MolMed highlights Unique value proposition in cancer therapy NGR-hTNF: first-in-class vascular targeting agent (VTA) TK: a breakthrough cell therapy for bone marrow transplant Anticancer therapeutics in advanced clinical development in 7 indications Focus on unmet needs in oncology Two technologies: a diversified, risk mitigating business model Continued access to new technologies and R&D projects Option right to IP of biomedical research leader San Raffaele Institute Access to Asian markets through alliance with Takara Bio Inc. (Japan) Experienced management team combining scientific, industrial and business expertise Support of highly committed institutional investors MolMed S.p.A. Company presentation, Bio CEO & Investor Conference, 9 February

4 Long-term strategy Focus on oncology indications needing new options Business model: innovation & risk mitigation GMP manufacturing of cell/gene therapy products Diversified pipeline to create value for shareholders Industrial project built on a strong science basis MolMed S.p.A. Company presentation, Bio CEO & Investor Conference, 9 February

5 Product pipeline: focus on unmet needs in oncology Indications targeted by MolMed s investigational therapies Incidence (North America, Europe, Japan and Australia) Colon-rectum NGR-hTNF Ovary Leukaemia Sarcomas 1 Mesothelioma 1 TK 0,00 0,33 0,67 1,00 Mortality / incidence ratio Unmet medical need Lung (NSCLC) Lung (SCLC) Liver No or few treatment options approved or in develoment in: First/second line Third/fourth line Source: Globocan 2002; 1 Company estimate MolMed S.p.A. Company presentation, Bio CEO & Investor Conference, 9 February

6 Business model: innovation & risk mitigation Two technologies and two business models for a diversified pipeline MM core assets & competencies: cell & gene therapies Personalised Medicine Licensee for Asia TK Direct production & marketing Feeding pipeline Diversifying assets: targeted biological drugs R&D Preclinical Phase I Phase II Phase III Market NGRhTNF Out-licensing Many indications, broad markets Drug industrialisation MolMed S.p.A. Company presentation, Bio CEO & Investor Conference, 9 February

7 In-house GMP manufacturing facility Status of pharmaceutical company granted by the Italian Drug Agency (AIFA) Authorised in-house GMP manufacturing facility since 2003 Meets EMEA and FDA requirements for production of clinical-grade bulk drug substances Manufacturing of genetically modified patient-specific cells Production of own cell-based therapeutics Clinical-grade cell manipulation services Acknowledged international reputation in GMP activities In cell and gene therapy MolMed S.p.A. Company presentation, Bio CEO & Investor Conference, 9 February

8 MolMed oncology pipeline Product TK NGR-hTNF single agent + doxorubicin + Xelox + cisplatin cis/gem + cis/pem NGR-IFNγ NGR-IL12 Indication (trial code) High-risk leukaemia (TK007, TK008) Leukaemia/Japan [by Takara Bio] Solid tumours [ MTD] (EORTC 16041) Solid tumours [low dose] (NGR002) Colorectal cancer (NGR006) Hepatocarcinoma (NGR008) Mesothelioma (NGR010) Solid tumours [high dose] (NGR013) Solid tumours (NGR003) Small cell lung cancer (NGR007) Ovarian cancer (NGR012) Sarcomas Colorectal cancer (NGR005) Solid tumours (NGR004) Lung cancer/nsclc (NGR014) Solid tumours Solid tumours Res Precl Phase I Phase II Phase III Legenda for clinical trials: planned ongoing completed MolMed S.p.A. Company presentation, Bio CEO & Investor Conference, 9 February

9 MolMed oncology pipeline Product TK NGR-hTNF single agent + doxorubicin + Xelox + cisplatin cis/gem + cis/pem NGR-IFNγ NGR-IL12 Indication (trial code) High-risk leukaemia (TK007, TK008) Leukaemia/Japan [by Takara Bio] Solid tumours [ MTD] (EORTC) Solid tumours [low dose] (NGR002) Colorectal cancer (NGR006) Hepatocarcinoma (NGR008) Mesothelioma (NGR010) Solid tumours [high dose] (NGR013) Solid tumours (NGR003) Small cell lung cancer (NGR007) Ovarian cancer (NGR012) Sarcomas Colorectal cancer (NGR005) Solid tumours (NGR004) Lung cancer/nsclc (NGR014) Solid tumours Solid tumours Res Precl Phase I Phase II Phase III NGR002 publ. Eur J Canc NGR003 publ. Br J Canc TK007 publ. Lancet Onc Legenda for clinical trials: planned ongoing completed MolMed S.p.A. Company presentation, Bio CEO & Investor Conference, 9 February

10 TK opens the door of bone marrow transplant to all patients Cell therapy based on genetically engineered donor T cells Indication: high-risk leukaemia (~ patients/yr in Europe and North America) Orphan Drug designation in EU and US Current therapeutic approach: pharmacotherapy followed by haematopoietic stem cell transplantation (HSCT) is the only potentially curative option Unmet need: ~50% of patients candidate to HSCT miss a fully matched donor Limitations of HSCT from partially compatible donors (haplo-hsct): Graft must be depleted of T cells to prevent Graft versus Host Disease (GvHD) T-cell depletion dramatically increases transplant related mortality, because of delayed immune-reconstitution TK i.e. genetically engineered donor T cells - overcomes limitations of haplo-hsct by providing a fully functional immune system to the patient while preventing GvHD MolMed S.p.A. Company presentation, Bio CEO & Investor Conference, 9 February

11 Key results of Phase I/II trial TK007 Results published by The Lancet Oncology TK007 trial data ITT population: 50 patients receiving haplo-hsct 22 no TK cells infusion 28 infused with TK cells 6 not immune-reconstituted 22 immune-reconstituted (IR) (>100 CD3 + cells/ml) haplo-hsct data (EBMT survey) TK007 IR patients Transplant-related mortality (at 50 months from HSCT) 50% 14% Disease relapse 20-30% 10% 4-year disease-free survival 20-30% 45% GvHD: occurrence control n.a. 50% 100% Source: Ciceri, Bonini et al., Lancet Oncol May 1;10: MolMed S.p.A. Company presentation, Bio CEO & Investor Conference, 9 February

12 Phase III trial TK008 Coordinator center: San Raffaele Hospital, Milan (Italy) Patient population: high-risk adult leukaemia patiens undergoing haplo-hsct Randomisation: TK add-back vs no add-back Ratio 3:1 Study size: 152 patients Primary endpoints: 1. Overall survival (OS) 2. Transplant-related mortality (TRM) Planned to become an international multicentre clinical trial Expansion to European centres by 1H 2010 Expansion to US centres by end 2010 MolMed S.p.A. Company presentation, Bio CEO & Investor Conference, 9 February

13 Automation of TK manufacturing process Current manugfacturing process conducted in semi-automatic closed system MolMed is investing in process optimisation Feasibility study for fully-automated closed system ( manufacturing machine) completed TK-manufacturing machine: feasibility study Investment in process automation scaled to confidence in clinical development MolMed S.p.A. Company presentation, Bio CEO & Investor Conference, 9 February

14 TK: a breakthrough therapy Advantages assessed in Phase II trial (TK007): Early and sustained immune-reconstitution Reduction of transplant-related mortality Protection from relapse No adverse events related to gene transfer procedure Prompt donor availability for all patients (~50%) lacking a fully compatible one Source: Ciceri, Bonini et al., Lancet Oncol May 1;10: MolMed S.p.A. Company presentation, Bio CEO & Investor Conference, 9 February

15 NGR-hTNF has broad therapeutic potential in many solid tumours First-in-class vascular targeting agent (VTA) - vascular targeting peptide (NGR) fused to human cytokine TNF Unique mechanism of action, not tumour-specific Very favourable safety profile, as single agent and in combination therapy Enhances the activity of chemotherapy in combination Orphan Drug designation in EU and US for mesothelioma and for liver cancer The NGR-hTNF molecule ( ) ( ) 1 subunit Treatment option for more than 1.6 million patients worldwide in the six indications investigated in Phase II trials NGR peptide Human TNF MolMed S.p.A. Company presentation, Bio CEO & Investor Conference, 9 February

16 Summary of Phase II clinical trials of NGR-hTNF Trial Dose (μg/m 2 ) Indication Status Results Single agent NGR Colorectal cancer Completed Top line in 2008 Long term in 2009 NGR Primary liver cancer Completed Top line in 2009 NGR Mesothelioma Completed Top line in 2009 Combination therapy NGR005 (+ Xelox) Colorectal cancer Completed First data in 2009 NGR007 (+ Doxorubicin) 0.8 Lung cancer (SCLC) Ongoing First data in 2010 NGR012 (+ Doxorubicin) 0.8 Ovarian cancer Ongoing First data in 2010 NGR014 (+ Cisplatinbased regimens) Randomised 0.8 Lung cancer (NSCLC) Ongoing First data in 2010 MolMed S.p.A. Company presentation, Bio CEO & Investor Conference, 9 February

17 Results in colorectal cancer (Phase II trial NGR006) Antitumour activity (46 heavily pre-treated patients): Schedule: 33 pts q3w 13 pts q1w DCR (SD or PR): 39% Median PFS = 2.5 m (BSC: 1.8 m) Median OS = 13.1 m (BSC: 6 m) Long-term follow-up (q3w cohort): 18-m survival rate = 33% 24-m survival rate = 25% Percent survival Overall survival Best supportive care Median OS: 6 months NGR-hTNF Median OS: 13.1 months Median follow-up: 22.1 months 16 patients (35%) alive Overall survival more than doubled with respect to data reported for best supportive care Time (months) Sources: ASCO 2009, Abstract 4088 (Poster); ECCO-ESMO 2009, Abstract P-6062 MolMed S.p.A. Company presentation, Bio CEO & Investor Conference, 9 February

18 Results in liver cancer (Phase II trial NGR008) Antitumour activity (40 pre-treated patients): Schedule: 27 pts q3w 13 pts q1w DCR: 30% - 1 ongoing CR & 1 PR achieved Median PFS = 2.3 m (no BSC data available) Median OS = 8.9 m (no BSC data available) Survival rates: 12-m: 34% 18-m: 22% Complete tumour necrosis ongoing since May 2008 in a patient refractory to sorafenib Complete response (contrast-enhanced CT-scan) February 2008: baseline May 2008: after 4 cycles of NGR-hTNF Sources: ASCO 2009, Abstract 15500; ECCO-ESMO 2009, Abstract P-6617 MolMed S.p.A. Company presentation, Bio CEO & Investor Conference, 9 February

19 Results in mesothelioma (Phase II trial NGR010) Antitumour activity (57 pre-treated patients): Schedule: 43 pts q3w 14 pts q1w DCR (SD or PR): 46% Median PFS = 2.8 m (BSC: 1.5 m) Dose intensification leads to a nearly threefold PFS at 6 m Median OS = 12.1 m (BSC: 9.7 m) Dose intensification may significantly prolonge disease control duration Phase III planned to start in 2010 PFS comparison: q1w vs q3w schedule Percent survival % 11% 19% 5% Time (months) Sources: ASCO 2009, Abstract 7582 (Poster); ECCO-ESMO 2009, Abstract O-9011 MolMed S.p.A. Company presentation, Bio CEO & Investor Conference, 9 February

20 NGR-hTNF has unique market potential First-in-class VTA with unique mechanism of action, offering new option for: Orphan indications Pre-treated, refractory patient populations in broad indications No tumour-type specificity: clinical activity in very different tumour types observed in Phase II trials Better safety profile than competitors: no GI perforations, bleedling, thromboembolisms (vs anti-angiogenics on the market) no cardiovascular toxicity (vs VTAs in development) Proven clinical activity in Phase II trials as single agent endorsing the vast applicability of NGR-hTNF MolMed S.p.A. Company presentation, Bio CEO & Investor Conference, 9 February

21 MolMed assets for cancer therapy innovation TK Prototype of patient-specific medicine Only approach enabling HSCT for all patients lacking a fully matched donor Project of own industrial production capability and commercialisation Market potential: million expected peak sales 1 NGR-hTNF VTA with unique and broad potential in very different types of solid tumours Partner development and commercialisation with pharma/biotech companies to maximise potential Market potential: 2.6 billion expected peak sales 2 Diversified technologies to fight cancer Note: Peak sales in Europe, U.S. and Japan. Indications: for TK acute myeloid leukaemia (AML); for NGR-hTNF colorectal (CRC), liver (HCC) and smallcell lung cancer (SCLC), and mesothelioma (MPM) Sources: 1 Analyst consensus (Nomura Code, Société Générale); 2 Company assessment MolMed S.p.A. Company presentation, Bio CEO & Investor Conference, 9 February

22 Milestones In 2010 In 2011 TK Expand Ph III (TK008): in Europe In US First results Ph III Updated results Ph III (TK008) NGR-hTNF Start Ph III mesothelioma (NGR015) Top line results Ph II: SC-lung cancer + doxo (NGR007) Ovarian cancer + doxo (NGR012) First results: Ph I high-dose (NGR013) Ph II random. in NSC-lung cancer + cisplatin-based regimens (NGR014) Start random. Ph II + doxo: Soft tissue sarcomas (NGR011) Indication to be selected Top line results Phase I high-dose (NGR013) First results Ph III mesothelioma (NGR015) First/updated results randomised Ph II: NSC-lung cancer + cisplatin-based regimens (NGR014) Soft tissue sarcomas + doxo (NGR011) Start Ph III in indication to be selected MolMed S.p.A. Company presentation, Bio CEO & Investor Conference, 9 February

23 Shareholders MolMed is a public company with strong investors Listed on Milan Stock Exchange (MTA, standard segment class 1) Ticker: MLM - Ticker Reuters: MLMD.MI - Ticker Bloomberg: MLM IM Private capital raised ( ): 60 million MolMed shareholders at December 31, 2009 Capital raised at IPO (2008): 56 million Issued shares: 104,667,808 Fininvest S.p.A % Science Park Raf S.p.A % Airain Lda % Market cap (Jan 27, 2010): 183 million Free float % Arner Bank S.A % H-Equity S.àr.l. SICAR 8.170% Delfin S.àr.l % MolMed S.p.A. Company presentation, Bio CEO & Investor Conference, 9 February

24 Share performance: 1 year to date MLM share performance from 28 January 2009 to 27 January MLM NBI (Nasdaq Biotech Index) FTSE-MIB BTK (Amex Biotech Index) Share price MLM ( ) 2009 Mar Apr May Jun Jul Aug Sep Oct Nov Dec M 1.0 M 0.6 M 0.2 M Volumes Source: finance.yahoo.com MolMed S.p.A. Company presentation, Bio CEO & Investor Conference, 9 February

25 Financials MolMed financials ( thousand) first nine months of year 2009 FY 2006 FY 2007 FY Q Q 2009 Revenues & other income 2,725 3,814 3,963 3,223 3,880 Operating costs (13,563) (16,763) (23,073) (16,865) (17,055) Loss for the period (10,697) (12,696) (19,110) (12,434) (12,673) Net financial position 8,426 5,666 35,281 37,653 23,123 Positive net financial position due to IPO proceeds and careful cash management Increase in revenues: growth of third party services + tax credit on R&D expenses Tight control on cash burn, kept in line with FY 2008 MolMed has sufficient cash at least until end 2010 MolMed S.p.A. Company presentation, Bio CEO & Investor Conference, 9 February

26 Strong position to create value Unique investigational therapies that generate major value 23 million cash (3Q 2009) Strong shareholder committment Diversified, risk mitigating business model Progress in clinical development providing near-term value inflection points Option right from San Raffaele Foundation (opportunity to feed pipeline) Late-stage pipeline Experienced management Proven innovation Leader in oncology clinical development Public company with strong investors base MolMed S.p.A. Company presentation, Bio CEO & Investor Conference, 9 February

27 Contact: Claudio Bordignon, Chairman & CEO MolMed S.p.A. Via Olgettina, Milan, Italy phone: fax: an integrated strategy to cure cancer Thank you very much for your attention This presentation will be available on our website MolMed S.p.A. Company presentation, Bio CEO & Investor Conference, 9 February 2010