MolMed IR presentation - June 7 Jefferies FINAL S.p.A. – Jefferies Global Healthcare Conference, 7 June 2012 Forward-looking statements 2 The presentation contains certain forward-looking

Company presentation

Jefferies Global Healthcare ConferenceNew York, 7 June 2012

Claudio Bordignon, Chairman and CEO

MolMed S.p.A. – Jefferies Global Healthcare Conference, 7 June 2012

Forward-looking statements

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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, 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:

• NGR-hTNF: tumour vascular targeting agent – expected first filing in 2013

• Cell and gene therapy:

• TK: cell therapy product – expected filing in 2013

• CMO activities for third parties: growing revenues

Cell & gene therapy

Recombinant proteins

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Cell & gene therapyRecombinant proteins

MolMed business model:innovation and risk mitigation

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Two innovative technologies with different business strategies



NGR-hTNF

Tumour vascular targeting Potential blockbuster


Partnering for larger indications

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NGR-hTNF TK


Patient-specific product for high-risk leukemia



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Own commercialisation (in EU & US)

MolMed S.p.A. – Jefferies Global Healthcare Conference, 7 June 2012 7


NGR-hTNF TK CMO activities



Patient-specific cell & gene therapies



R&D & production for third parties




ASCO 2012: overview of extensiveclinical development efforts

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NGR binding to tumour blood vessels

hTNF

NGRpeptide

CD13on tumour neo-

vasculature

TNF-Receptor

Recombinant fusion trimeric protein:Structure of 1 monomer

Endothelium marker (green) + NGR (red)

NGR-hTNF: a selective vascular targeting agent

Doses of 0.8 µg/sqm systematically show antitumour activity


Selective binding to angiogenic tumour vessels

Human colon carcinoma Normal human colon

Endothelium marker (green) + NGR (red) Endothelium marker (green) + NGR (red)

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NGR binds to tumour vessels of CRC and not to those of normal intestine


Product manufacturing on track forfuture 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

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NGR-hTNF clinical development: 1 pivotal Ph III and 4 randomized Ph II trials ongoing

Indication Trial code Phase I Phase II Phase III

Monotherapy

Solid tumours MTD EORTC 16041

Solid tumours – low dose NGR002

Solid tumours – high dose NGR013

Colorectal cancer NGR006

Liver cancer NGR008

Mesothelioma NGR010, NGR015 rand.

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 Colorectal cancer NGR005

+ cisplatin Solid tumours NGR004

Lung cancer /NSCLC NGR014 random.

ongoing completed


SCLC: efficacy independent oftumour chemo-sensitivity

Waterfall plot

Important responses independent of prior tumour chemo-sensitivity

Phase II Single arm + Doxorubicin (75mg/sqm) ≥ 2nd line

Platinum-resistantPlatinum-sensitive

Progressive disease

Partial response

Complete response


Ovarian cancer: response-related long-term efficacy

Spider plot

Multiple strong and durable responses

Phase II Single arm + Doxorubicin (60mg/sqm) ≥ 2nd line


Mesothelioma: effect of treatment intensification

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

Phase II Single arm Monotherapy ≥ 2nd line


NSCLC: first data from a randomised study suggest efficacy in the squamous subset

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

Phase II Randomized + Cisplatin and Gemcitabine 1st line


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

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Suitable for long-term maintenance treatment


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


Data from five randomised studies expected over the next 18 months

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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

TK: a new technology forhaematopoietic 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


TK therapy: provides a fully functional immune system

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Protection frominfections

Protection from leukaemia relapse

No immune-suppression needed(prompt abrogation of GvHD by administration of ganciclovir)

An option to high-risk leukemia patients in need of a transplant…


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%

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Phase II data indicate that TK allows a transplant from a partially compatible donor

….reducing leukaemia relapse and transplant-related mortality resulting in longer disease-free survival


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)

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Planned application for Conditional Approval in early 2013


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

CMO activities: on the market ingrowing cell and gene therapy field


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

TK: getting ready for the market


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

NGR-hTNF: gearing up for regulatory approval





















Opportunities for partnerships

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Dedicated sales force for mesothelioma

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Thank you very much for your attention

MolMed S.p.A.Via Olgettina 58 - 20132 Milan (Italy)

[email protected]

Documents

MolMed IR presentation - June 7 Jefferies FINAL S.p.A. – Jefferies Global Healthcare Conference, 7 June 2012 Forward-looking statements 2 The presentation contains certain forward-looking