Investor analyst presentation by jolinmilioncherie

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									                     Investor & analyst
                     presentation




Claudio Bordignon,
                          Jefferies Global Healthcare
Chairman and CEO          C f          L d
                          Conference, London
                          28 September 2011
Forward-looking statements


                                   forward looking
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
                                         forward looking statements.
materially from those anticipated in the forward-looking statements

The Company assumes no responsibility to update forward-looking statements or adapt
                               p
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
States, Australia, Canada, Japan
complying with local registration requirements and other legal restrictions.

  ecla at o     the o c al Co po ate     a c al epo t g Manager:
Declaration by t e official Corporate Financial Reporting Ma age :
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,                 records.
books and accounting records

Enrico Cappelli, Chief Financial Officer, official Corporate Financial Reporting Manager


 MolMed S.p.A. - Investor & analyst presentation, September 2011                           2
Late-stage company focused on oncology


    Two highly innovative anticancer therapeutics in Phase III
      • TK: cell therapy product for a novel approach to bone marrow transplant
                                        g    g g
      • NGR-hTNF: selective vascular targeting agent for solid tumours

    Two technology platforms – a diversified business model

    Continued access to new technologies and R&D projects
      • Option right to IP of European biomedical research leader San Raffaele

    Solid investor base - listed on the Milan Stock Exchange (MLM)




 MolMed S.p.A. - Investor & analyst presentation, September 2011                  3
Significant progress achieved in 2011


    NGR hTNF:
    NGR-hTNF: moved forward with promising evidence
      • First results of randomised Phase II trial in NSCLC at ASCO and EMCC
                                                            (           )
      • Start of 2 randomised Phase II trials in OC and MPM (maintenance)

    TK: pursued international pivotal Phase III trial
      • IND clearance of pivotal Phase III trial by the US FDA
      • New evidence of TK direct role in fighting infections and disease relapse

    Corporate: new revenue-generating agreements in gene therapy
      • Telethon Foundation: € 8,3 million over 4 years
      • GSK: € 5,5 million over 2 years




 MolMed S.p.A. - Investor & analyst presentation, September 2011                    4
MolMed clinical development pipeline

Product         Indication                        Trial code               Phase I       Phase II    Phase III
                High-risk
                High risk leukaemia               TK007, TK008 random.
                                                  TK007        random
TK
                Leukaemia (Japan/Takara Bio)
                Solid tumours → MTD               EORTC 16041
NGR-hTNF
NGR hTNF        Solid tumours – low dose          NGR002
                Solid tumours – high dose         NGR013
                Colorectal cancer                 NGR006
Monotherapy Liver cancer                          NGR008
                Mesothelioma                      NGR010, NGR015 random.
                Mesothelioma/maintenance NGR019 random.
+ doxorubicin Solid tumours                       NGR003
                Lung cancer/SCLC                  NGR007
                Ovarian cancer                    NGR012
                Ovarian cancer                    NGR018 random.
                Soft tissue sarcomas              NGR016 random.
 Xelox
+X l            C l    t l
                Colorectal cancer                 NGR005
                Solid tumours                     NGR004
+ cisplatin
                Lung cancer/NSCLC                 NGR014 random.

                                                                               ongoing              completed
  MolMed S.p.A. - Investor & analyst presentation, September 2011                                                5
TK opens the door of bone marrow transplant
to more patients

    Indication (Phase III): high-risk leukaemias

    Only established cure: haematopoietic stem cell transplant (HSCT)
    from a fully matched donor

    Unmet need:
    • ~50% of patients candidate to HSCT miss a fully matched donor
    • HSCT from partially compatible donors ( p
                  p       y     p                          )         y
                                               (haplo-HSCT) limited by either
      high risk of graft-versus-host disease (GvHD) or delayed immune-
      reconstitution

    TK enables haplo-HSCT and provides a fully
    functional immune system




 MolMed S.p.A. - Investor & analyst presentation, September 2011                6
   TK: genetically engineered donor T cells
   overcoming the limits of haplo-HSCT
                            haplo HSCT

          Abrogates the need for post-transplant immune-suppression
          Preserves full protection against infections and anti-leukaemia activity
                                                           Hospital
                        Donor                                                                                    Protection from
                       haemat.
                      stem cells                                                           TK cells              infections
                            Haemat.
                                         Day 0                              Day 28
                                                                                                                 Protection from
                           stem cells                                                         TK cells



                                                                                                                 leukaemia relapse

                                                                                                                 No immune-
                                                                                                                 N i
              Haplo-
             identical                                                                                           suppression needed
               donor                                                                                             (prompt abrogation of
                                                    MolMed GMP facility
                                                                                                                 GvHD by administration
                                          Genetic engineering of donor T cells                                   of ganciclovir)
                            T cells




                          Donor
                                                            HSV-TK
                          T cells

           d     d from Bonini et al., Science 1997; Bonini et al., Nat. Med. 2003; Recchia et al., PNAS 2006; Ciceri et al, Blood 2007; Ciceri,
Sources: adapted f                 l                            l          d            h       l                         l l d
Bonini et al., The Lancet Oncology 2009

      MolMed S.p.A. - Investor & analyst presentation, September 2011                                                                              7
   Key results of completed Phase II trial TK007


            TK superiority vs historical data: longer disease-free survival,
             reduced leukaemia relapse and transplant-related mortality

         ITT population:                                22 no TK cells infusion
           50 patients                                                                         6 not immune-reconstituted
      receiving haplo-HSCT
                haplo HSCT                              28 infused
                                                        with TK cells                           22 immune-reconstituted (IR)
                                                                                                    (>100 CD3+ cells/ml)

                                                               haplo-HSCT data                     TK007 IR patients
                                                           (EBMT survey, 266 patients) 1                    (22 patients) 2
                         g
                Median age                                                y
                                                                       35 years                                  y
                                                                                                              56 years
                Transplant-related mortality
                                                                          50%                                    14%
                (at 50 months from HSCT)
                Leukaemia relapse                                       20-30%                                   10%
                4-year disease-free survival                            20-30%                                   45%
                GvHD - occurrence                                                                               50%
                                                                          n.a.
                     - control                                                                                  100%
Sources: 1Ciceri et al. Blood, 2008, 2Ciceri, Bonini et al., Lancet Oncol. 2009, ASCO 2011, abstract 6526

      MolMed S.p.A. - Investor & analyst presentation, September 2011                                                          8
Regulatory path for TK in EU and US



                                                                    CAT classified TK
                                                                    as “cell therapy”
        Orphan Drug                    Protocol                                              Planned MAA
        designation                    Assistance                         Follow-up to
                                       for Phase III                           Protocol
                                                                            Assistance


     2003            2005            2007          2008            2009    2010     2011   2012   2013




 MolMed S.p.A. - Investor & analyst presentation, September 2011                                           9
Regulatory path for TK in EU and US



                                                                    CAT classified TK
                                                                    as “cell therapy”
        Orphan Drug                    Protocol                                                 Planned MAA
        designation                    Assistance                         Follow-up to
                                       for Phase III                           Protocol
                                                                            Assistance


     2003            2005            2007          2008            2009    2010     2011   2012      2013



                                                        Positive opinion                        Planned BLA
                     Orphan Drug                               from RAC
                     designation
                                                                                    Follow-up
                                                            IND for Phase III       meeting



 MolMed S.p.A. - Investor & analyst presentation, September 2011                                              10
NGR-hTNF: a selective vascular targeting agent



         Doses of 0.8 µg/m2 systematically show anti-tumour activity

           NGR-hTNF
           NGR hTNF                                                           NGR binding
    recombinant fusion protein                                          to tumour blood vessels




     NGR
    peptide



                                                                    Endothelium marker (green) + NGR (red)
                                hTNF
      CD13
(aminopeptidase N)
 present on tumour       TNF-Receptor
  neo-vasculature


  MolMed S.p.A. - Investor & analyst presentation, September 2011                                            11
NGR-hTNF: a unique treatment opportunity


  Antitumour activity observed in six indications in Phase II trials

  Active as single agent and in combination with chemotherapy

  Favourable toxicity profile, better than competitors ( > 500 pts treated)

  Neutrophil-lymphocyte ratio (NLR) as a potential biomarker in predicting
  outcome following treatment with NGR-hTNF




 MolMed S.p.A. - Investor & analyst presentation, September 2011          12
   Completed Phase II trials:
   tumour shrinkage observed in 5 indications

       Monotherapy
          • Mesothelioma1:
                    q                                     g      y
            More frequent treatment leads to three-fold higher 2-year survival rate
          • Liver cancer2:
            One complete response, patient disease-free since May 2008
          • Colorectal cancer3:
            Overall survival doubled versus historical controls

       Combination with doxorubicin
          • Ovarian cancer4:
            Pi        d i         f       l i f first       i         ll d
            Primary endpoint met after analysis of fi 17 patients enrolled
          • Small-cell lung cancer5:
            Anti-tumour activity regardless of platinum sensitivity/resistance
            Anti tumour



Sources: 1J Clin Oncol 2010, ASCO 2011, poster 7089; 2Br J Cancer 2010; 3Eur J Cancer 2010; 4ASCO 2011, poster 5022; EMCC 2011, poster 8030;
5ASCO 2011, poster 7077; EMCC 2011, poster 9103


     MolMed S.p.A. - Investor & analyst presentation, September 2011                                                                     13
   Phase II results in mesothelioma:
   Long-term efficacy
   Long term


                                                                                l benefit
                                       Weekly schedule induces prolonged clinical b
                                          kl    h d l    d          l   d l           f
                                                by avoiding long treatment gaps

                              Overall Survival (OS) by disease status                        2-year OS rate by treatment frequency
                                                 Pts with disease control                                      weekly
                                                 Pts with disease progression                                  triweekly
                ability (%)




                                                         Median Overall Survival:




                                                                                         Percent patients
                                                         16.7 versus 8.3 months                                                           57%
      vival proba




                                                                                                                      29%
   Surv




                                                                                                             10%                  10%


                                                                                                            All pts (n=57)   Pts with DC (n=26)
                                              Time (months)

                               Median Overall Survival (all patients): 12.1 months

Sources: Gregorc et al, JCO 2010; ASCO 2011, Abstract 7089

                       MolMed S.p.A. - Investor & analyst presentation, September 2011                                                            14
Ongoing phase II randomised clinical trials


    Four indications:
      • Non-small cell lung cancer (1st line) with cispaltin-based chemotherapy
                     (           )
      • Mesothelioma (maintenance)
      • Soft tissue sarcomas (2nd line) with (or without) doxorubicin
      • Ovarian cancer (2nd line) with pegylated liposomal doxorubicin

   Versus standard of care (not applicable for sarcoma trial)

   Progression Free Survival as primary endpoint

   Results expected in 2012




 MolMed S.p.A. - Investor & analyst presentation, September 2011                  15
   Randomised Phase II + cisplatin-based chemo
   in NSCLC (1st line)


                           i i interim d
                       Promising i
                       P            i data presented at ASCO 2011 and EMCC 2011
                                                   d                d   CC

                                 Subset analysis in patients with squamous cell histology
                                Disease control rate                                Progression-free survival
                                                 St bl di
                                                 Stable disease
                                                 Partial response
  % of patients
              s




                                                                                                                         py
                                                                                                    NGR-hTNF + Chemotherapy
                                                                                                    Chermotherapy alone




                  NGR-hTNF + Chemotherapy               Chemotherapy alone           Median PFS duration (months)

Source: EMCC 2011, poster 9014

                  MolMed S.p.A. - Investor & analyst presentation, September 2011                                         16
 Pivotal Phase III trial in mesothelioma
 as 2nd line therapy (NGR015)


      Double-blind, l b     t ll d
      D bl bli d placebo-controlled

      Primary endpoint: Overall survival

      Results expected in 2013

                                                                     n=195
                                                                             NGR-hTNF (0.8 µg/m2 q1w)
  Adult patients pre-treated                                                 + Best Investigator’s Choice∗
                                                     Randomisation
   with pemetrexed-based
                                                          1:1
       regimen (n=390)                                                                 Placebo
                                                                             + Best Investigator’s Choice∗
                                                                     n=195       ∗   Best supportive care ±
                                                                                     single agent chemotherapy




Hypothesis testing: Overall Survival ∼ + 3 months; HR=0.72; α=0.05; β=0.20
   MolMed S.p.A. - Investor & analyst presentation, September 2011                                           17
 Regulatory path for NGR-hTNF in EU and US




                                Protocol Assistance                                  Planned MAA
                                Phase III in mesothelioma
Orphan Drug des.                                                            Follow-up to
 (mesothelioma)                                  Orphan Drug des.           Protocol
                                                 (liver cancer)             Assistance


                        2008               2009               2010   2011     2012         2013




  MolMed S.p.A. - Investor & analyst presentation, September 2011                                  18
 Regulatory path for NGR-hTNF in EU and US




                                Protocol Assistance                                                   Planned MAA
                                Phase III in mesothelioma
Orphan Drug des.                                                                        Follow-up to
 (mesothelioma)                                  Orphan Drug des.                       Protocol
                                                 (liver cancer)                         Assistance


                        2008               2009               2010              2011      2012           2013

Orphan Drug des.                                                                 Follow-up clinical
                                                                                 Follow up
 (mesothelioma)                                                     IND for      development
                                                                    Phase III
                                 des.
                    Orphan Drug des
                       (liver cancer)                                     Follow-up                   Planned BLA
                                                                         meeting CMC


  MolMed S.p.A. - Investor & analyst presentation, September 2011                                                   19
MolMed business model: innovation
and risk mitigation


                         Two original technology platforms and
                    two business strategies for a diversified pipeline

          Cell & gene therapeutics


                           TK
             Cell therapy
Orphan indication with high unmet need
      Patient-specific medicine



                 Business model
     Own industrial production
     O n ind strial prod ction and
     commercialisation capabilities
Partnered with Takara Bio for Japan/Asia


 MolMed S.p.A. - Investor & analyst presentation, September 2011         20
MolMed business model:
Innovation and risk mitigation


                         Two original technology platforms and
                    two business strategies for a diversified pipeline

          Cell & gene therapeutics                                  Vascular targeting biological drugs


                           TK                                                   NGR-hTNF
              Cell therapy                                                  Recombinant protein
 Orphan indication with high unmet need                                Many indications, broad market
       Patient-specific medicine                                           Potential blockbuster



                 Business model                                              Business model
     Own industrial production
     O n ind strial prod ction and                                                    out-sourced
                                                                         Production out sourced
     commercialisation capabilities                                     Partner development and
Partnered with Takara Bio for Japan/Asia                           commercialisation with pharma/biotech


 MolMed S.p.A. - Investor & analyst presentation, September 2011                                           21
Cash to support
product development programmes


                     Strong positive financial position
         More revenues from new agreements starting from 2H 2011


                          MolMed financials 1H 2011 (€ thousand)
                                                    FY 2008        FY 2009    FY 2010    1H 2011
      Revenues & other
      income                                            3,963        4,712      2,676      1,316

      Operating costs                              (23,073)        (22,519)   (20,424)   (11,784)

      Result for the period                        (17,446)        (17,169)   (17,582)    (9,787)

      Net fi    i l    iti
      N t financial position                          35 281
                                                      35,281        19 567
                                                                    19,567     60,040
                                                                               60 040     50,891
                                                                                          50 891



 MolMed S.p.A. - Investor & analyst presentation, September 2011                                    22
Shareholders’ structure


                          High liquidity: average daily traded shares
                             nearly 1% of total outstanding shares

  Listed on the Milan Stock Exchange (Milan:MLM)
  Market cap (Sep 24, 2011): € 105 million
  Issued shares: 210,541,926
  Average traded volume (3 months): 1.7 million shares
                                                                       Fininvest 23.9%
                                                                                           Science Park Raf
                                                                                                10.5%
                               Others (<2%)
                                                                                              Airain 7.1%
                                  43.5%
                                                                                           Delfin 4.3%
                                                                                    H-Invest 4.1%
                                                                   Lombard   H-Equity 4.1%
                                                                     2.5%
                                                                     2 5%

 MolMed S.p.A. - Investor & analyst presentation, September 2011                                              23
Milestones 2011-2013: getting closer
to the turning point


 Year
 Y                                     TK                                   NGR hTNF
                                                                            NGR-hTNF
2011                 FDA IND clearance of Phase III                First results of randomised Phase
                       (TK008)                                     II trial in NSCLC
                     Expand Phase III trial TK008:                 Start 2 random Phase II trials:
                     • In Europe                                   • In ovarian cancer
                     • In US                                       • I MPM ( i t
                                                                     In      (maintenance))

                     First update on TK008                         First data (enrollment) of Phase III
                                                                                         (
                                                                   trial in mesothelioma (MPM) )
                                                                   Start Phase III trial in second
                                                                   indication
2012                                                               Results of randomised Phase II
                                                                   trials in 3 indications: STS, OC and
                                                                   MPM (maintenance)

2013                 Filing for regulatory approval                Filing for regulatory approval for
                     in EU and US                                  MPM in EU and US


 MolMed S.p.A. - Investor & analyst presentation, September 2011                                        24
Strong position for growth


    Two highly innovative anticancer therapeutics in Phase III
     •      Lead indications: orphan drug designations, limited competition
     •      Proof-of-concept achieved: no toxicity, antitumour activity shown mode of
            Proof of concept              toxicity                      shown,
            action well understood
     •      Randomised data ahead (over next 12 months at ASH, ASCO, ESMO)
     •      IND clearance from FDA: expansion of trials in the US expected in 2011

    Well positioned
    Well-positioned for value creation
     •      Diversified, risk-mitigating technology platforms
     •      Cash to support product development programmes
     •      Experienced top management, strong scientific advisors, solid investor base




 MolMed S.p.A. - Investor & analyst presentation, September 2011                          25
MolMed next update events in 2011


Business & investor meetings
    Oct. 31–Nov. 2                   Bio-Europe, Düsseldorf

Clinical & scientific meetings
    December 10-13                   ASH Annual Meeting, San Diego

Financial calendar
    November 14                      Board review of financial report 3Q 2011




 MolMed S.p.A. - Investor & analyst presentation, September 2011                26
                                         Thank you very much
Contact
                                         for      tt ti
                                         f your attention
   g
Holger Neecke
Director Business Development
& Investor Relations

MolMed S.p.A.
Via Olgettina, 58 - 20132 Milan, Italy
phone: +39 0221277.1
fax: +39 0221277.325
e-mail: holger.neecke@molmed.com
e mail: holger neecke@molmed com

								
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