an integrated g strategy to cure cancer

MolMed S.p.A. p Company presentation 9th Annual A l Bi Biotech t h iin E Europe IInvestor t F Forum, Zürich, September 14-15, 2009 Holger Neecke Director Business Development & IR

Forward-looking statements This presentation may contain certain forward-looking statements. Although the Company believes its expectations are based on reasonable assumptions, 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 Th d ig d h herewith ith attests, tt t pursuantt tto A Article ti l 154 154-bis, bi paragraph g h 2 off th the It Italian li Consolidated Law on Finance (Legislative Decree 58/1998), that the accounting disclosure contained in this presentation matches documentary evidence, corporate books, and accounting g records. Enrico Cappelli, Chief Financial Officer, official Corporate Financial Reporting Manager

MolMed S.p.A. – 9th Annual Biotech in Europe Investor Forum, Zürich, September 14-15, 2009

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Agenda MolMed in brief Long-term strategy MolMed’s oncology gy p pipeline p A unique value proposition in cancer therapy Milestones Financial outlook Summary

MolMed S.p.A. – 9th Annual Biotech in Europe Investor Forum, Zürich, September 14-15, 2009

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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. – 9th Annual Biotech in Europe Investor Forum, Zürich, September 14-15, 2009

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Long-term strategy

Focus on oncology indications needing di new options

Efficient clinical & pharmaceutical development independently or with partners

GMP manufacturing of cell/gene therapy products p

Diversified pipeline to create value for shareholders

Industrial project built on a strong science basis MolMed S.p.A. – 9th Annual Biotech in Europe Investor Forum, Zürich, September 14-15, 2009

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Product pipeline: focus on unmet needs in oncology Indications targeted by MolMed’s investigational therapies 700.000

In ncidence (N North Amerrica, Eurrope, Japan and Australia)

Colon-rectum 600.000 Lung (NSCLC)

500 000 500.000 400.000

No or few treatment options approved or in develoment in: First/second line

NGR-hTNF

300.000

Third/fourth line

200.000 Leukaemia eu ae a 100.000

Lung (SCLC)

Liver

Ovary Sarcomas1

0 0,00

0,33

TK 0,67

Mesothelioma1 1,00

Mortality / incidence ratio

Unmet medical need Source: Globocan 2002; 1Company estimate MolMed S.p.A. – 9th Annual Biotech in Europe Investor Forum, Zürich, September 14-15, 2009

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Efficient development independently or with partners MolMed has adopted a flexible business model:

• Specialised indications (TK): in-house clinical development development, production and marketing (except in Asia)

• Indications with large market potential (NGR-hTNF): open to partnerships with major pharma and biotech companies for clinical development development, production and marketing

Current partnerships: HSR - San Raffaele Scientific Institute: option right on results of cancer research co developer of TK therapy for Asia Takara Bio Inc: licensee and co-developer Avecia Biologics Ltd: industrialisation of NGR-hTNF production process

MolMed S.p.A. – 9th Annual Biotech in Europe Investor Forum, Zürich, September 14-15, 2009

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In-house GMP manufacturing facility Status of pharmaceutical company granted by the Italian Drug Agency g y ((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 Clinical grade cell manipulation services

Customised project design from preclinical development to Phase III Clinical-grade lots released by Qualified Person Regulatory support Acknowledged c owledged international te at o al reputation eputat o in GMP GM activities act v t es In cell and gene therapy MolMed S.p.A. – 9th Annual Biotech in Europe Investor Forum, Zürich, September 14-15, 2009

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MolMed oncology pipeline Product

Indication (trial code) Res Precl High-risk g leukaemia ((TK007,, TK008)) TK Leukaemia/Japan [by Takara Bio] Solid tumours [ÆMTD] (EORTC) NGR-hTNF Solid tumours [low dose] (NGR002) Colorectal cancer (NGR006) single agent Hepatocarcinoma (NGR008) Mesothelioma (NGR010) Solid tumours t mo rs [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 NGR-IL12 Solid tumours Legenda for clinical trials:

completed

ongoing

Phase I

Phase II

Phase III

planned

MolMed S.p.A. – 9th Annual Biotech in Europe Investor Forum, Zürich, September 14-15, 2009

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TK opens the door of bone marrow transplant to all patients Cell therapy based on genetically engineered donor T cells Enables Hematopoietic Stem Cell Transplant from partially compatible donors (haploHSCT) Allows to keep the benefits of add-backs of donor T cells in haplo-HSCT Orphan Drug Status in the EU (2003) and in the US (2005) In Phase III for high-risk leukaemia adult patients Advantages assessed in Phase II trial (TK007): Early and sustained immune-reconstitution Reduction of transplant-related mortality Prompt control of Graft versus Host Disease Protection from relapse Safety of the gene transfer and cell manipulation procedures Prompt donor availability for all patients (~ 50%) lacking a fully compatible one MolMed S.p.A. – 9th Annual Biotech in Europe Investor Forum, Zürich, September 14-15, 2009

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Key results of Phase I/II trial TK007 TK007 trial data 4 drop drop-out out (no HSCT) 54 enrolled

22 no TK cells infusion

22 immune-reconstituted ( 100 CD3+ cells/ml) (>100 ll / l)

28 infused with TK cells

6 not immune-reconstituted

Trial TK007

Historical data

79%

n.a.

10%

50%

Disease relapse

10%

20-30%

4-year disease-free survival GvHD - occurrence - control

45% 50% 100%

20-30%

Immune-reconstitution Transplant-related mortality (at 50 months from HSCT)

n.a.

Source: Ciceri, Bonini et al., Lancet Oncol. 2009 May 1;10:489-500 MolMed S.p.A. – 9th Annual Biotech in Europe Investor Forum, Zürich, September 14-15, 2009

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TK: summary of clinical trials Academic studies: 91 patients treated, proving the safety of gene transfer and cell manipulation p p processes Phase II trial (TK007) completed – Results published by The Lancet Oncology (2009 May;10(5):489-500) Phase III trial (TK008) started in Italy in 2008

•

First Phase III trial authorised in Italyy for a cell/gene g therapy py

Phase I trials in Japan started in October 2008 and April 2009

• •

allo-HSCT haplo-HSCT 2 trials in leukaemia patients: receiving allo HSCT or haplo HSCT Conducted by Takara Bio Inc. at the National Cancer Center in Tokyo

TK clinical validation now in Phase III randomised trial MolMed S.p.A. – 9th Annual Biotech in Europe Investor Forum, Zürich, September 14-15, 2009

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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 neo-vasculature vasculature

Human TNF: Binds to TNF receptors (TNFRI and TNF-RII) on vascular endothelium Strong antitumour effect Destroys blood vessel function Approved as anticancer drug, b t toxicity but t i it li limits it application li ti to isolated limb perfusion

Resolution: 2.8 Å

MolMed S.p.A. – 9th Annual Biotech in Europe Investor Forum, Zürich, September 14-15, 2009

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Multiple potential applications in cancer therapy Novel mechanism of action offers new treatment options:

• •

For refractory patients, pre pre-treated treated with anti anti-angiogenic 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 Activityy in a varietyy of solid tumours Proven anticancer activity in single agent Phase II trials, endorsing the vast applicability of NGR-hTNF: Improvement of survival in heavily pre-treated colorectal cancer patients Long-lasting, ongoing liver cancer L l ti i complete l t response iin li Enhancement of disease control duration in mesothelioma following dose intensification

MolMed S.p.A. – 9th Annual Biotech in Europe Investor Forum, Zürich, September 14-15, 2009

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NGR-hTNF Phase II trials as single agent Optimal safety profile:

• No cu cumulative ulat ve or o long-term lo g te to toxicity c ty obse observed ved • Side effects essentially consitutional symptoms (chills) limited to infusion time

3 single arm trials (enrolment completed) in pre-treated patients with advanced disease, 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. – 9th Annual Biotech in Europe Investor Forum, Zürich, September 14-15, 2009

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Results in colorectal cancer (Phase II trial NGR006) Antitumour activity (46 heavily pre-treated pts):

Overall survival

Schedule: 33 pts q3w, 13 pts q1w

Best supportive care Median OS: 6 months NGR-hTNF

DCR (SD or PR): 39%

Median OS: 13.1 months

Median PFS = 2 2.5 5 months (1.8 months reported for BSC) (6 months reported for BSC)

Long-term follow-up (Median 22.1 months): 35% of patients alive

Median follow-up: 22.1 months 16 patients ti t (35%) alive li

Pe ercent survvival

Median OS = 13.1 months

Overall survival more than doubled with respect to data reported for best supportive care

6

13.1 Time (months) h

Source: ASCO 2009, Abstract 4088 (Poster) MolMed S.p.A. – 9th Annual Biotech in Europe Investor Forum, Zürich, September 14-15, 2009

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Results in liver cancer (Phase II trial NGR008) Antitumour activity (39 pts):

Contrast-enhanced CT-scan (arterial phase)

Schedule: 27 pts q3w, 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)

February 2008: baseline

Top-line T li results l will ill b be presented d at ESMO 2009

Ongoing, complete tumour necrosis Ongoing in a patient refractory to sorafenib

May 2008: after 4 cycles of NGR-hTNF

Source: ASCO 2009, Abstract 15500 MolMed S.p.A. – 9th Annual Biotech in Europe Investor Forum, Zürich, September 14-15, 2009

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Results in mesothelioma (Phase II trial NGR010) Antitumour activity (57 pts):

PFS comparison: q1w vs q3w schedule

Schedule: 43 pts q3w, q3w 14 pts q1w DCR (SD or PR): 46%

q1w schedule: 6-month PFS rate: 36% Median PFS in pts with DC: 9 9.1 1 months

Median PFS = 2.8 months (vs 1.5 1 5 months reported for BSC)

Median Overall Survival = 12.1 months

q3w schedule: 6-month PFS rate: 13% Median PFS in pts with DC: 4.4 months

100

Dose iintensification D ifi i lleads d to a nearly l three-fold PFS at 6 months

The dose intensification may significantly f l prolonge l disease control duration

Perce ent survivall

(vs 9.7 months reported for BSC) 80 60 40

36%

20

13%

0

0

3

6

9

12

15

Time (months) Source: ASCO 2009, Abstract 7582 (Poster) MolMed S.p.A. – 9th Annual Biotech in Europe Investor Forum, Zürich, September 14-15, 2009

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NGR-hTNF Phase II trials in combination therapy Favourable safety profile with all combination drugs tested, with no overlapping pp g toxicity y 3 single arm trials in pre-treated patients with advanced disease:

•

+ Xelox in colorectal carcinoma (NGR005): enrolment completed, started December 2007 - results lt will ill b be presented t d att ESMO 2009

• •

+ doxorubicin in lung cancer-SCLC (NGR007) – ongoing, started February 2008 + doxorubicin in ovarian carcinoma (NGR012) – ongoing, started November 2008

1 randomised trial as first-line treatment:

•

+ cisplatin-based regimens (cis-gem or cis-pem) in lung cancer-NSCLC (NGR014) – ongoing started June 2009 ongoing,

MolMed S.p.A. – 9th Annual Biotech in Europe Investor Forum, Zürich, September 14-15, 2009

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Unique assets for cancer therapy innovation MolMed value proposition TK

Only O l approach h enabling bli g HSCT for f all ll patients ti t lacking l ki g a fully f ll matched t h d donor d Phase III in high-risk leukaemia started in Italy Project of own industrial production capability and commercialisation with a focused sales force Market potential: € 390-420 million expected peak sales1

NGR-hTNF

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 (Phase II data in CRC, MPM and HCC) Commercialise in Europe, partner with pharma/biotech companies in US & ROW Market potential: € 2.6 billion expected peak sales2

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: 1Analyst consensus (Nomura Code, Société Générale); 2Updated company assessment after ESMO 2008 MolMed S.p.A. – 9th Annual Biotech in Europe Investor Forum, Zürich, September 14-15, 2009

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Milestones 2H 2009 and 2010 Anticipated 2009 TK

Anticipated 2010

Expand Ph III trial (TK008) in Europe 9

Expand Ph III trial (TK008) in US Preliminary Ph III data

NGR-hTNF

Start 2 randomised Phase II trials: • in NSCLC + cisplatin p • in STS + doxorubicin

V

Start Ph I trial high doses (NGR013)

V

Top line results of Ph II trials: • CRC (NGR006) – ASCO 2009 • HCC (NGR008) – ESMO 2009 • MPM ((NGR010)) – ASCO 2009 Results of Ph II trial in CRC + Xelox (NGR005) – ESMO 2009

V V

Start of Ph III trial in MPM Top p line results Ph I trial high g dose (NGR013) – ASCO 2010 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 R d i ti off Ph II ttrials Randomisation i l + 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. – 9th Annual Biotech in Europe Investor Forum, Zürich, September 14-15, 2009

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Enough cash to support product development programmes MolMed financials (€ thousand) FY 2006

FY 2007

FY 2008

2,725

3,814

3,963

912

2,879

Operating costs

(13,563)

(16,763)

(23,073)

(11,508)

(11,054)

L Loss for f the th period i d

(10 697) (10,697)

(12 696) (12,696)

(19 110) (19,110)

(9 873) (9,873)

(7 788) (7,788)

8 26 8,426

666 5,666

3 28 35,281

06 44,061

26 93 26,935

Revenues & other income

fi i l position ii Net financial

1H 2008

1H 2009

Strong net financial position due to IPO proceeds and careful cash management IIncrease iin revenues in i 1H09 thanks th k to t growth g th off services i provided id d tto thi third d parties ti 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. – 9th Annual Biotech in Europe Investor Forum, Zürich, September 14-15, 2009

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Share performance since IPO Share performance (price & volumes) from March 5, 2008 to September 10, 2009 2 20 2.20 2.00

Share price e (€)

1.80 1.60 1.40 1.20

MLM FTSE MIB FTSE-MIB BTK (Amex Biotech Index) NBI (Nasdaq Biotech Index) May

Jun

Jul

Aug

0.80 Sep

Oct

Nov

Dec

2009

Feb

Mar

Volumes

2008

1.00

Apr

May

Jun

Jul

Aug

Sep 1.5M 1.0M 0.5M

104 467 808 Issued shares: 104,467,808 Market cap (September 10, 2009): € 173 million MolMed S.p.A. – 9th Annual Biotech in Europe Investor Forum, Zürich, September 14-15, 2009

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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 Late stage pipeline Proven innovation Team-driven culture lt

MolMed S.p.A. – 9th Annual Biotech in Europe Investor Forum, Zürich, September 14-15, 2009

Leader in oncology clinical development

Financial strength due to IPO

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Contact: Holger g Neecke Director Business Development & Investor Relations MolMed S.p.A. Via Olgettina, 58 20132 Mil Milan, It Italy l phone: +39 02212771 fax: +39 0221277.325 e-mail: [email protected] @

THANK YOU VERY MUCH FOR YOUR ATTENTION Check out forthcoming events 2009 at www.molmed.com\investors\events MolMed S.p.A. – 9th Annual Biotech in Europe Investor Forum, Zürich, September 14-15, 2009

an integrated g strategy to cure cancer