Company presentation. Claudio Bordignon, Chairman and CEO. Jefferies Global Healthcare Conference New York, 7 June 2012

Transcription

1 Company presentation Claudio Bordignon, Chairman and CEO Jefferies Global Healthcare Conference New York, 7 June 2012

2 Forward-looking statements The presentation contains 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 is not an offer of securities for sale in any country or jurisdiction, including the United States. Securities may not be sold to the public in the United States, in Australia, in Canada, in Japan, or in other relevant jurisdictions without complying with local registration requirements and other legal restrictions. 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. Jefferies Global Healthcare Conference, 7 June

3 MolMed, an oncology focused company Listed on the Milan Stock Exchange (MLM) Net financial position: 33 M (Mar 31st 2012) 106 employees, 2/3 staff scientists Company core competencies: Recombinant proteins: Recombinant proteins NGR-hTNF: tumour vascular targeting agent expected first filing in 2013 Cell and gene therapy: Cell & gene therapy TK: cell therapy product expected filing in 2013 CMO activities for third parties: growing revenues MolMed S.p.A. Jefferies Global Healthcare Conference, 7 June

4 MolMed business model: innovation and risk mitigation Two innovative technologies with different business strategies Recombinant proteins Cell & gene therapy MolMed S.p.A. Jefferies Global Healthcare Conference, 7 June

5 MolMed business model: innovation and risk mitigation Two innovative technologies with different business strategies Recombinant proteins Cell & gene therapy NGR-hTNF Tumour vascular targeting Potential blockbuster Partnering for larger indications MolMed S.p.A. Jefferies Global Healthcare Conference, 7 June

6 MolMed business model: innovation and risk mitigation Two innovative technologies with different business strategies Recombinant proteins Cell & gene therapy NGR-hTNF Tumour vascular targeting Potential blockbuster TK Patient-specific product for high-risk leukemia Partnering for larger indications Own commercialisation (in EU & US) MolMed S.p.A. Jefferies Global Healthcare Conference, 7 June

7 MolMed business model: innovation and risk mitigation Two innovative technologies with different business strategies Recombinant proteins Cell & gene therapy NGR-hTNF TK CMO activities Tumour vascular targeting Potential blockbuster Patient-specific product for high-risk leukemia Patient-specific cell & gene therapies Partnering for larger indications Own commercialisation (in EU & US) R&D & production for third parties MolMed S.p.A. Jefferies Global Healthcare Conference, 7 June

8 ASCO 2012: overview of extensive clinical development efforts MolMed S.p.A. Jefferies Global Healthcare Conference, 7 June

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13 NGR-hTNF: a selective vascular targeting agent Recombinant fusion trimeric protein: Structure of 1 monomer NGR binding to tumour blood vessels NGR peptide CD13 on tumour neovasculature htnf TNF-Receptor Endothelium marker (green) + NGR (red) Doses of 0.8 µg/sqm systematically show antitumour activity MolMed S.p.A. Jefferies Global Healthcare Conference, 7 June

14 Selective binding to angiogenic tumour vessels Human colon carcinoma Normal human colon Endothelium marker (green) + NGR (red) Endothelium marker (green) + NGR (red) NGR binds to tumour vessels of CRC and not to those of normal intestine MolMed S.p.A. Jefferies Global Healthcare Conference, 7 June

15 Product manufacturing on track for future global market requirements Cost-effectively obtained by fermentation in E.coli (one single gene construct) Development of commercial-scale manufacturing ongoing for liquid and lyophilised formulations MolMed S.p.A. Jefferies Global Healthcare Conference, 7 June

16 NGR-hTNF clinical development: 1 pivotal Ph III and 4 randomized Ph II trials ongoing Indication Trial code Phase I Phase II Phase III Solid tumours MTD EORTC Monotherapy Solid tumours low dose Solid tumours high dose Colorectal cancer Liver cancer Mesothelioma Mesothelioma/maintenance NGR002 NGR013 NGR006 NGR008 NGR010, NGR015 rand. NGR019 random. + doxorubicin Solid Tumours NGR003 Lung cancer/sclc Ovarian cancer Ovarian cancer Soft tissue sarcomas NGR007 NGR012 NGR018 random. NGR016 random. + Xelox Colorectal cancer NGR005 + cisplatin Solid tumours NGR004 ongoing completed Lung cancer /NSCLC NGR014 random. MolMed S.p.A. Jefferies Global Healthcare Conference, 7 June

17 SCLC: efficacy independent of tumour chemo-sensitivity Phase II Single arm + Doxorubicin (75mg/sqm) 2nd line Waterfall plot Platinum-resistant Platinum-sensitive Progressive disease Partial response Complete response Important responses independent of prior tumour chemo-sensitivity MolMed S.p.A. Jefferies Global Healthcare Conference, 7 June

18 Ovarian cancer: response-related long-term efficacy Phase II Single arm + Doxorubicin (60mg/sqm) 2nd line Spider plot Multiple strong and durable responses MolMed S.p.A. Jefferies Global Healthcare Conference, 7 June

19 Mesothelioma: effect of treatment intensification Phase II Single arm Monotherapy 2nd line OS by schedule in pts with disease control3 3-year follow-up data confirm the benefit of dose intensification and validate the design of the ongoing pivotal Phase III study MolMed S.p.A. Jefferies Global Healthcare Conference, 7 June

20 NSCLC: first data from a randomised study suggest efficacy in the squamous subset Phase II Randomized + Cisplatin and Gemcitabine 1st line Decrease in tumor size over treatment Overall survival (n=34) 30% of relative reduction in the risk of tumour progression and 50% relative reduction in the risk of death MolMed S.p.A. Jefferies Global Healthcare Conference, 7 June

21 Very low toxicity profile More than 500 patients treated so far: No grade 3-4 drug-related toxicity No cumulative toxicity No worsening of chemo-associated toxicities No pulmonary hemorrhage or bleeding events No dose delays, dose reductions or treatment discontinuations due to toxicity Suitable for long-term maintenance treatment MolMed S.p.A. Jefferies Global Healthcare Conference, 7 June

22 Potential predictor of efficacy investigated: treatment-induced chills Progression free survival by chills in mesothelioma About two thirds of patients experience chills during first infusions The onset of chills predicts greater treatment benefit MolMed S.p.A. Jefferies Global Healthcare Conference, 7 June

23 Data from five randomised studies expected over the next 18 months Non small-cell lung cancer (1st line) In combination with cisplatin-based chemotherapy Survival data expected in 2H 2012 Ovarian cancer (2nd line) In combination with doxorubicin Results expected in 1H 2013 Sarcomas (1st and 2nd line) In combination with doxorubicin or monotherapy Results expected in 2H 2013 MolMed S.p.A. Jefferies Global Healthcare Conference, 7 June

24 TK: a new technology for haematopoietic stem cell transplant Indication: haematopoietic stem cell transplants (HSCT) for high-risk leukaemias Unmet need: ~50% of patients candidate to HSCT miss a fully matched donor Without a transplant, high-risk leukemia patients have extremely low survival rate

25 TK therapy: provides a fully functional immune system Protection from infections Protection from leukaemia relapse No immunesuppression needed (prompt abrogation of GvHD by administration of ganciclovir) An option to high-risk leukemia patients in need of a transplant MolMed S.p.A. Jefferies Global Healthcare Conference, 7 June

26 Phase II data indicate that TK allows a transplant from a partially compatible donor Haplo-HSCT data (EBMT survey, 266 patients)1 Phase II TK: immune-reconstituted patients (22 patients)2 Median age 35 years 56 years Transplant-related mortality (at 50 months from HSCT) 50% 14% Leukaemia relapse 20-30% 10% 4-year disease-free survival 20-30% 45% GvHD - occurrence - control n.d. 50% 100%.reducing leukaemia relapse and transplant-related mortality resulting in longer disease-free survival MolMed S.p.A. Jefferies Global Healthcare Conference, 7 June

27 Ongoing pivotal Phase III trial (TK008) Enrolment planned: 170 patients, randomisation 3:1 in favour of TK Primary endpoint: disease-free survival Ongoing in Europe in 7 centers, further expansion in additional 10 in Europe and the US (following IND clearance in 2011) Planned application for Conditional Approval in early 2013 MolMed S.p.A. Jefferies Global Healthcare Conference, 7 June

28 CMO activities: on the market in growing cell and gene therapy field Core competencies since inception of the company in 1996 Manufacturing of TK for Phase III and commercialisation Growing interest from pharma companies in potentially revolutionary cell and gene therapies Agreement signed in 2011: up to 13.8 M over 4 years, 104% increase in revenues (1q 2012 vs 1q 2011) Opportunity for further industrial partnerships MolMed S.p.A. Jefferies Global Healthcare Conference, 7 June

29 TK: getting ready for the market Phase II long-term data available and pivotal Phase III trial under way Planned application for Conditional Approval in 2013 based on: Proof of efficacy Established long-term safety data High unmet medical need for patients lacking HLA-matched donor Orphan Drug designation + patent protection (with SPC) up to 2030 Small dedicated sales force required MolMed S.p.A. Jefferies Global Healthcare Conference, 7 June

30 NGR-hTNF: gearing up for regulatory approval Pivotal Phase III on track with enrolment, results expected by 2H 2013 Registration planned in mesothelioma as first indication: Low competition: no products in second-line treatment on the market or in Phase III development Orphan Drug designation + patent protection up to 2029 Potential predictor of efficacy identified MolMed S.p.A. Jefferies Global Healthcare Conference, 7 June

31 MolMed business model: innovation and risk mitigation Two innovative technologies with different business strategies Recombinant proteins Cell & gene therapy NGR-hTNF TK CMO activities Tumour vascular targeting Potential blockbuster Patient-specific product for high-risk leukemia Patient-specific cell & gene therapies Partnering for larger indications Own commercialisation (in EU & US) R&D & production for third parties MolMed S.p.A. Jefferies Global Healthcare Conference, 7 June

32 MolMed business model: innovation and risk mitigation Two innovative technologies with different business strategies Recombinant proteins Cell & gene therapy NGR-hTNF TK CMO activities Tumour vascular targeting Potential blockbuster Patient-specific product for high-risk leukemia Patient-specific cell & gene therapies Partnering for larger indications + Dedicated sales force for mesothelioma Own commercialisation (in EU & US) R&D & production for third parties Opportunities for partnerships MolMed S.p.A. Jefferies Global Healthcare Conference, 7 June

33 Thank you very much for your attention MolMed S.p.A. Via Olgettina Milan (Italy)