MolMed S.p.A. Company presentation

Transcription

1 an integrated strategy to cure cancer MolMed S.p.A. Company presentation 2 nd Italian Stock Market Opportunities Conference Milan, September 17, 2009

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. Italian Stock Market Opportunities Conference, Milan, September 17,

3 Agenda Company overview Long-term strategy MolMed s oncology pipeline A unique value proposition in cancer therapy Milestones Financial outlook Summary MolMed S.p.A. Italian Stock Market Opportunities Conference, Milan, September 17,

4 Clinical-stage company focused on oncology Public company listed on the Milan Stock Exchange (MLM.MI) 2 anticancer therapeutics in advanced clinical development 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 91 full time employees MolMed S.p.A. Italian Stock Market Opportunities Conference, Milan, September 17,

5 Team combining scientific and industrial expertise (I/II) Claudio Bordignon, MD, President and CEO Former Scientific Director of the San Raffaele Scientific Institute, and pioneer in the clinical validation of several successful cell-based therapies, for both genetic and acquired disorders. Among the 22 eminent founding members of the Scientific Council of the European Research Council (ERC) Marina Del Bue, BSC, MBA, General Manager, Member of the Board Over 20 years experience in managing product development in the pharma industry, previously at Menarini Group. She led MolMed s evolution from service to product company. Member of the Boards of EuropaBio and VP of Assobiotec Enrico Cappelli, BEC, Chief Financial Officer 14 years experience in positions of increasing responsibility in finance and administration management, previously at a Group listed at Borsa Italiana. Chartered Accountant and member of the Official Register of Public Auditors Holger Neecke, PhD, MBA, Director Business Development At MolMed since 2001, he is in charge inter alia of building MolMed s strategic alliances. He closed license agreements with pharma and biotech companies in EU, US and Japan MolMed S.p.A. Italian Stock Market Opportunities Conference, Milan, September 17,

6 Team combining scientific and industrial expertise (II/II) Cynthia Giuliani, BA Hons, Director Human Resources 20 years experience in human resources and compensation related matters, gained in multinational and biotech companies such as Roche and Liebert Hiross, and most recently in Bioxell Daniele Pieraccioli, PhD, European Patent Attorney, Director IP 30 years experience in pharma and biotech companies, including GlaxoSmithKline and Serono. Member of the Board of the European Patent Institute and Board Director of the European Association of Patent Attorneys in Industry Gian Paolo Rizzardi, MD, Director Research 15 years experience in research on immunotherapeutic strategies. Working at MolMed, he also developed a broad and in-depth experience in tumour homing pepdies and vascular targeting agents. Previously at the University of Lausanne Raul Ziliotto, BSC, Director Drug Development 20 years experience in drug development focused on biological and biotech-based drugs. Formerly Director of Biological Products Development and responsible for plant validation at Pharmacia, Pharmacia-Upjohn, Pfizer and Nerviano Medical Sciences Marco Dieci, BSC, Qualified Person, Director Operations 10 years experience in QC, QA and GMP production of medicinal products, previously at Chiesi Farmaceutici SpA e Lameplast SpA Antonio Lambiase, MD, Director Clinical Development Over 15 years experience at Roche in the areas of oncology and haematology, as medical responsible for the clinical development of several novel biotech-based compounds MolMed S.p.A. Italian Stock Market Opportunities Conference, Milan, September 17,

7 Long-term strategy Focus on oncology indications needing new options Efficient clinical & pharmaceutical development independently or with partners 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. Italian Stock Market Opportunities Conference, Milan, September 17,

8 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. Italian Stock Market Opportunities Conference, Milan, September 17,

9 Efficient development independently or with partners Molmed has adopted a flexible business strategy: Specialised indications (TK): in-house clinical development, production and marketing (except in Asia) Indications with large market potential (NGR-hTNF): open to partnerships with major pharma and biotech companies for full clinical development, production and marketing Research Pharmaceutical development and manufacturing Clinical-grade Preclinical development Clinical development HSR - San Raffaele Scientific Institute Host institution: strategic context for healthcare R&D Option right on research in cancer Industrial Marketing and Sales Hospital (patients) Avecia Biologics Industrialisation of NGR-hTNF production process Supplier of marketcompliant product for Phase III Takara Bio Inc Licensee and co-developer of TK for Asia Conducting Phase I of TK in Japan MolMed S.p.A. Italian Stock Market Opportunities Conference, Milan, September 17,

10 In-house GMP manufacturing facility Status of pharmaceutical company granted by the Italian Drug Agency (AIFA) Track record of investigational gene therapies for rare diseases Manufacturing of genetically modified patient-specific cells Production of own cell-based therapeutics Clinical-grade cell manipulation services Clinical-grade lots released by Qualified Person Customised project design from preclinical development to Phase III Regulatory support Acknowledged international reputation in GMP activities In cell and gene therapy MolMed S.p.A. Italian Stock Market Opportunities Conference, Milan, September 17,

11 MolMed oncology pipeline Product Indication (trial code) TK High-risk leukaemia (TK007, TK008) Leukaemia/Japan [by Takara Bio] NGR-hTNF Solid tumours [ MTD] (EORTC) Solid tumours [low dose] (NGR002) Colorectal cancer (NGR006) single agent Hepatocarcinoma (NGR008) Mesothelioma (NGR010) Solid tumours [high dose] (NGR013) Solid tumours (NGR003) Small cell lung cancer (NGR007) + doxorubicin Ovarian cancer (NGR012) Sarcomas + Xelox Colorectal cancer (NGR005) + cisplatin Solid tumours (NGR004) cis/gem + Lung cancer/nsclc (NGR014) cis/pem NGR-IFNγ Solid tumours NGR-IL12 Solid tumours Res Precl Phase I Phase II Phase III Legenda for clinical trials: completed ongoing planned MolMed S.p.A. Italian Stock Market Opportunities Conference, Milan, September 17,

12 TK: a cell-based therapy enabling safe and effective haplo-hsct Cell therapy based on genetically engineered donor T cells Enables Haematopoietic Stem Cell Transplant (HSCT) from partially compatible donors (haplo-hsct) Allows to keep the benefits of add-backs of donor T cells in haplo-hsct In Phase III for high-risk leukaemia adult patients Orphan Drug Status in the EU (2003) and in the US (2005) Overview of the TK therapy procedure Donor haemat. stem cells Hospital TK cells Graft versus Infection (GvI) and support to stem cells engraftment Haemat. stem cells HSC transplant (day 0) TK cell infusion (day 21) TK cells Graft versus Leukaemia (GvL) Haploidentical donor (from bone marrow or peripheral blood) MolMed GMP facility Graft versus host disease (GvHD) T cells donor T cells transduction, selection and expansion Abrogation by administration of Ganciclovir donor T cells LTR TK SV LNGFR LTR MolMed S.p.A. Italian Stock Market Opportunities Conference, Milan, September 17,

13 Key results of Phase I/II trial TK007 TK007 trial data 4 drop-out (no HSCT) 54 enrolled 22 no TK cells infusion 28 infused with TK cells 6 not immune-reconstituted 22 immune-reconstituted (IR) (>100 CD3 + cells/ml) TK007 IR patients Historical data Transplant-related mortality (at 50 months from HSCT) 10% 50% Disease relapse 10% 20-30% 4-year disease-free survival 45% 20-30% GvHD occurrence control 50% 100% n.a. Source: Ciceri, Bonini et al., Lancet Oncol May 1;10: MolMed S.p.A. Italian Stock Market Opportunities Conference, Milan, September 17,

14 TK: summary of clinical trials Academic studies: 91 patients treated, proving the safety of gene transfer and cell manipulation processes Phase I/II trial (TK007) completed Results published by The Lancet Oncology (2009 May;10(5): Phase III trial (TK008) started in Italy in 2008 First Phase III trial authorised in Italy for a cell/gene therapy Phase I trials in Japan started in October 2008 and April trials in leukaemia patients: receiving allo-hsct or haplo-hsct Conducted by Takara Bio Inc. at the National Cancer Centre in Tokyo TK clinical validation now in Phase III randomised trial MolMed S.p.A. Italian Stock Market Opportunities Conference, Milan, September 17,

15 TK opens the door of bone marrow transplant to all patients Advantages assessed in Phase II trial (TK007): Early and sustained immune-reconstitution Reduction of transplant-related mortality Protection from relapse Prompt control of Graft versus Host Disease Safety of the gene transfer and cell manipulation procedures Prompt donor availability for all adult patients (~ 50%) lacking a fully compatible one MolMed S.p.A. Italian Stock Market Opportunities Conference, Milan, September 17,

16 NGR-hTNF: a novel vascular targeting agent with broad therapeutic potential in solid tumours First-in-class vascular targeting agent (VTA) Recombinant homotrimeric fusion protein Structure of NGR-hTNF (1 subunit) Cyclic CNGRCG peptide: targets tvcd13, a receptor present on tumour neo-vasculature Resolution: 2.8 Å Human TNF: Binds to TNF receptors (TNF- RI and TNF-RII) on vascular endothelium Strong antitumour effect Destroys blood vessel function Approved as anticancer drug, but toxicity limits application to isolated limb perfusion MolMed S.p.A. Italian Stock Market Opportunities Conference, Milan, September 17,

17 NGR-hTNF Phase II trials as single agent Optimal safety profile: No cumulative or long-term toxicity observed Side effects essentially consitutional symptoms (chills) limited to infusion time 3 single arm trials (enrolment completed) in pre-treated patients with advanced disease, with 2 administration schedules tested (q3w and q1w): Colorectal carcinoma (NGR006): overall survival more than doubled with respect to data reported for best supportive care Hepatocellular carcinoma (NGR008): ongoing complete tumour necrosis in a patient refractory to sorafenib Malignant pleural mesothelioma (NGR010): dose intensification may significantly prolong disease control duration Results presented at ASCO 2009 MolMed S.p.A. Italian Stock Market Opportunities Conference, Milan, September 17,

18 Results in colorectal cancer (Phase II trial NGR006) Antitumour activity (46 heavily pre-treated pts): Schedule: 33 pts q3w, 13 pts q1w DCR (SD or PR): 39% Median PFS = 2.5 months (1.8 months reported for BSC) Median OS = 13.1 months (6 months reported for BSC) Long-term follow-up (Median 22.1 months): 35% of patients alive 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 Source: ASCO 2009, Abstract 4088 (Poster) Time (months) MolMed S.p.A. Italian Stock Market Opportunities Conference, Milan, September 17,

19 Results in liver cancer (Phase II trial NGR008) Antitumour activity (39 pts): Schedule: 27 pts q3w, 12 pts q1w DCR: 28% - 1 ongoing CR & 1 PR achieved Median PFS = 2.3 months (no BSC data available) Median OS not yet reached (60% at 6 months) Top-line results will be presented at ESMO 2009 Contrast-enhanced CT-scan (arterial phase) February 2008: baseline Ongoing, complete tumour necrosis in a patient refractory to sorafenib May 2008: after 4 cycles of NGR-hTNF Source: ASCO 2009, Abstract MolMed S.p.A. Italian Stock Market Opportunities Conference, Milan, September 17,

20 Results in mesothelioma (Phase II trial NGR010) Antitumour activity (57 pts): Schedule: 43 pts q3w, 14 pts q1w DCR (SD or PR): 46% Median PFS = 2.8 months (vs 1.5 months reported for BSC) Median Overall Survival = 12.1 months (vs 9.7 months reported for BSC) Dose intensification leads to a nearly three-fold PFS at 6 months The dose intensification may significantly prolonge disease control duration Percent survival PFS comparison: q1w vs q3w schedule q1w schedule: 6-month PFS rate: 36% Median PFS in pts with DC: 9.1 months q3w schedule: 6-month PFS rate: 13% Median PFS in pts with DC: 4.4 months 36% 13% Time (months) Source: ASCO 2009, Abstract 7582 (Poster) MolMed S.p.A. Italian Stock Market Opportunities Conference, Milan, September 17,

21 NGR-hTNF Phase II trials in combination therapy Favourable safety profile with all combination drugs tested, with no overlapping toxicity 3 single arm trials in pre-treated patients with advanced disease: + Xelox in colorectal carcinoma (NGR005): enrolment completed, started December Results will be presented at ESMO doxorubicin in lung cancer-sclc (NGR007) ongoing, started February doxorubicin in ovarian carcinoma (NGR012) ongoing, started November randomised trial as first-line treatment: + cisplatin-based regimens (cis-gem or cis-pem) in lung cancer-nsclc (NGR014) ongoing, started June 2009 MolMed S.p.A. Italian Stock Market Opportunities Conference, Milan, September 17,

22 Multiple potential applications in cancer therapy Novel mechanism of action offers new treatment options: For refractory patients, pre-treated with anti-angiogenic and chemotherapy regimens For indications with high unmet need and few therapeutic options (Orphan Drug designation in both EU and US for mesothelioma) Very favourable toxicity profile as single agent and in combination Enhances activity of chemotherapy Activity in a variety of solid tumours Proven anticancer activity in single agent Phase II trials, endorsing the vast applicability of NGR-hTNF MolMed S.p.A. Italian Stock Market Opportunities Conference, Milan, September 17,

23 Unique assets for cancer therapy innovation MolMed value proposition TK NGR-hTNF Only approach enabling HSCT for all patients lacking a fully matched donor Phase III in high-risk leukaemia started in Italy Project of own industrial production capability and commercialisation to specialised clinical centres Market potential: million expected peak sales 1 VTA with unique and broad potential in very different types of solid tumours Efficacy in indications with no treatment options and/or in heavily pre-treated patients Commercialise in Europe, partner with pharma/biotech companies in US & ROW Market potential: 2.6 billion expected peak sales 2 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 Updated company assessment after ESMO 2008 MolMed S.p.A. Italian Stock Market Opportunities Conference, Milan, September 17,

24 Milestones 2H 2009 and 2010 TK Anticipated 2009 Expand Ph III trial (TK008) in Europe Anticipated 2010 Expand Ph III trial (TK008) in US Preliminary Ph III data Start 2 randomised Phase II trials: in NSCLC + cisplatin in STS + doxorubicin V Start of Ph III trial in MPM Top line results Ph I trial high dose (NGR013) ASCO 2010 NGR-hTNF Start Ph I trial high doses (NGR013) Top line results of Ph II trials: CRC (NGR006) ASCO 2009 HCC (NGR008) ESMO 2009 MPM (NGR010) ASCO 2009 First results of Ph II trial in CRC + Xelox (NGR005) ESMO 2009 V V V Top line results of Ph II trials: SCLC + doxo (NGR007) ESMO 2010 OC + doxo (NGR012) ESMO 2010 First results Ph II randomised trial NSCLC + cis-gem/cis-pem (NGR014) - ESMO 2010 Randomisation of Ph II trials + doxorubicin (indication tbd) Legenda for NGR-hTNF indications: SCLC = small-cell lung cancer; CRC = colorectal cancer; OC = ovarian cancer; STS = soft tissue sarcomas; HR-PC = hormone-refractory prostate cancer; HCC = liver cancer; MPM = mesothelioma MolMed S.p.A. Italian Stock Market Opportunities Conference, Milan, September 17,

25 Shareholders structure Public company with strong investors Listed on Milan Stock Exchange (MTA, standard segment) since March 5, 2008: Ticker: MLM Ticker Reuters: MLMD.MI Ticker Bloomberg: MLM IM Shareholders (April 14, 2009) Science Park Raf S.p.A % Airain Lda 21.12% Financials: Capital raised at IPO: 56 million Issued shares: 104,467,808 Market cap (September 10, 2009): 173 million Free float 21.55% Arner Bank S.A. 2.01% H-Equity S.àr.l. SICAR 8.19% Delfin S.àr.l. 8.66% Fininvest S.p.A % ( ) Source: MolMed Shareholders Meeting 2009 MolMed S.p.A. Italian Stock Market Opportunities Conference, Milan, September 17,

26 Share performance since IPO Share performance (price & volumes) from March 5, 2008 to September 10, Volumes Share price ( ) MLM FTSE-MIB BTK (Amex Biotech Index) NBI (Nasdaq Biotech Index) 2008 May Jun Jul Aug Sep Oct Nov Dec 2009 Feb Mar Apr May Jun Jul Aug Sep M 1.0M 0.5M Issued shares: 104,467,808 Market cap (September 15, 2009): 172 million MolMed S.p.A. Italian Stock Market Opportunities Conference, Milan, September 17,

27 Financials MolMed financials ( thousand) FY 2006 FY 2007 FY H H 2009 Revenues & other income 2,725 3,814 3, ,879 Operating costs (13,563) (16,763) (23,073) (11,508) (11,054) Loss for the period (10,697) (12,696) (19,110) (9,873) (7,788) Net financial position 8,426 5,666 35,281 44,061 26,935 Strong net financial position due to IPO proceeds and careful cash management Increase in revenues in 1H09 thanks to growth of services provided to third parties and to receipt of tax credit on R&D expenses In 2009 MolMed planned a tight control on cash burn, to be kept in line with 2008 According to current estimate, MolMed has sufficient cash at least until end 2010 MolMed S.p.A. Italian Stock Market Opportunities Conference, Milan, September 17,

28 Forthcoming events 2009 Financial calendar ( ): November 9 Financial report for Q Upcoming presentations September November 2-4 ESMO annual meeting, Berlin: NGR-hTNF clinical development update Bio Europe, Wien: updated company presentation ( ): Reports, presentations and press releases related to the past events of the financial calendar can be downloaded from MolMed s website MolMed S.p.A. Italian Stock Market Opportunities Conference, Milan, September 17,

29 Strong position to create value Unique investigational therapies that generate major value 27 million cash Flexible business strategy: independently and/or with partners Progress in clinical development providing near term value inflection points Option right from San Raffaele Foundation (opportunity to feed pipeline) Late-stage pipeline Team-driven culture Proven innovation Leader in oncology clinical development Financial strength due to IPO MolMed S.p.A. Italian Stock Market Opportunities Conference, Milan, September 17,

30 Contact: Holger Neecke Director Business Development & Investor Relations MolMed S.p.A. Via Olgettina, Milan, Italy phone: fax: an integrated strategy to cure cancer THANK YOU VERY MUCH FOR YOUR ATTENTION MolMed S.p.A. Italian Stock Market Opportunities Conference, Milan, September 17, 2009