Company presentation. 7 th European Life Science CEO Forum Zurich, 4-5 March Paolo Bassanini Business Development & IR

Transcription

1 Company presentation 7 th European Life Science CEO Forum Zurich, 4-5 March 2014 Paolo Bassanini Business Development & IR

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. Andrea Quaglino, Director administration, finance and control 2

3 MolMed: at a glance Listed on the Milan Stock Exchange: Mk Cap (Feb 28 th ) ~ 155 million (+60% YoY) Company Focus: oncology and genetic orphan diseases 118 employees, 2/3 staff scientists Company core competencies: Recombinant proteins NGR-hTNF: tumor vascular targeting agent proof of efficacy in randomized Phase II trials, long-term safety data established, pivotal Phase III results in 1H 2014 Cell and gene therapies TK: cell therapy product expected filing for Conditional Approval in EU in 1Q2014 Projects with Third Parties: development and production of gene & cell therapies for rare genetic diseases constantly growing revenues (+11,9% 30 Sept. 2013) 3

4 Shares information As of February 20 th, 2014: Number of shares (fully diluted): Free Float: 48.37% Daily traded volume (average 3 months): ~ 2,300,000 shares Shareholder s structure: Fininvest 24.90% Science Park Raf 8,34% Free float Others (<2%) 48.37% Airain 6.65% Delfin 4.06% H-Invest 3.62% H-Equity 4.06% Strategic investors 4

5 Key financials ( thousand) (first 9 months) 2012 (first 9 months) Operating revenues 5,059 3,418 2, Revenues from activities for third parties 4,593 2,767 2, Other income Operating costs (27,441) (26,098) (20,424) (17.894) (20.076) Result for the period (22,001) (21,569) (17,582) (14.654) (16.733) ( thousand) Dec 31 st 2012 Dec 31 st 2011 Dec 31 st 2010 Sep. 30 th 2013 Sep. 30 th 2012 Net financial position 17,526 38,667 60,

6 MolMed business model: innovation and risk mitigation Two innovative platforms with different business strategies Recombinant proteins Cell and gene therapy NGR-hTNF TK Projects with 3 rd Parties Tumour vascular targeting Potential blockbuster Patient-specific product for high-risk leukaemia Patient-specific cell & gene therapies Partnering Own commercialisation vs partnering opportunities R&D & production for third parties / partnerships 6

7 Advanced clinical development TK Phase II Phase III RANDOM. Placebo High-risk Leukemia * Comments Seeking Conditional Approval in EU Competition Low NGR-hTNF RANDOM. Placebo Pleural mesothelioma* 2 nd line Results in 1H14 None RANDOM. Placebo Pleural mesothelioma* 1 st line maintenance Completing enrolment in 1H14 None RANDOM. Ovarian cancer 2 nd line Results in 1H14 Medium RANDOM. Soft-tissue sarcomas 1 st line Results in 1H14 Low RANDOM. Non-small cell lung cancer 1 st line Completed Low (squamous) *Orphan Drug Designation Liver cancer * 2 nd line Small-cell lung cancer 2 nd line Colorectal cancer 2 nd line Completed Completed Completed Low High High Study completed Recruitment completed, follow-up ongoing Recruitment ongoing 7

8 NGR-hTNF: an anti-tumor fusion protein NGR peptide selectively binding to CD13 on tumor neo-vasculature htnf (human Tumor Necrosis Factor) powerful anti-tumor agent binding to the TNF-Receptor Molecule with unique biological properties Boosting activity of chemotherapy administered in combination Direct anti-tumour activity at very low doses (0.8 µg/sqm) with no toxic side effects 8

9 NGR-hTNF: selective vascular targeting agent Human colon carcinoma Normal human colon CD31 (green) + NGR-Qd (red) CD31 (green) + NGR-Qd (red) Whole mount analysis of tissues obtained from the same patient (N=3) NGR binds to tumour vessels of CRC and not to those of normal intestine 9

10 NGR-hTNF: very low toxicity profile More than 1000 patients treated so far: No grade 3-4 drug-related toxicity No cumulative toxicity No treatment discontinuations due to toxicity No worsening of chemo-associated toxicities No pulmonary hemorrhage or bleeding events Suitable for long-term maintenance treatment 10

11 Median change from baseline (%) Survival probability NSCLC: final data show statistically significant survival improvement in squamous subset 1 Phase II Randomized + Cisplatin and Gemcitabine 1 st line Decrease in tumour size over treatment Overall survival (n=35) 0 2 nd 4 th 6 th (cycle) Time (months) Note: 1 Predefined per protocol After a 2.5-year follow-up time, there is a more than 50% relative reduction in the risk of death 11

12 Soft Tissue Sarcomas: data show statistically significant survival benefit Phase II Randomized + Doxorubicin (60mg/sqm) 2 nd line Overall survival (n=69) Low-dose NGR-hTNF plus doxorubicin induces a statistically significant 100% increase in median survival (p=0.03) 12

13 Mesothelioma: treatment intensification leads to longer patient benefit Phase II Single arm Monotherapy 2 nd line Doubled median survival duration versus historical controls OS rates by schedule NGR-hTNF Ph II (n=57) Placebo vs Monochemoth. Ph III (n=660)* Median PFS (months) vs 1.4 Median OS (months) vs 7.1 *Krug LM et al. EMCC year follow-up results strongly confirm the benefit of dose intensification: survival rate increased 4-5 fold 13

14 Mesothelioma: pivotal Phase III trial as 2 nd line therapy (NGR015) Patients pre-treated with pemetrexed-based regimen (n=390) N=195 Randomization 1:1 N=195 NGR-hTNF (0.8 µg/sqm q1w) + Best Investigator s Choice Placebo + Best Investigator s Choice Design: double-blind, placebo-controlled, with a 1:1 randomization ratio Primary endpoint: overall survival (N=390; 80% power; 0.05 alpha level; HR=0.72) Status: enrolment completed (400 pts. in EU, US, Canada and Egypt) Primary efficacy analysis: expected during first half 2014 Competition: no approved drug in second line 14

15 NO THERAPY ALIMTA Mesothelioma: a new therapeutic plan Present therapeutic option 1. Only approved 1 st line treatment is the administration of pemetrexed (Alimta, Eli Lilly) in combination with cisplatin: average treatment duration months because of maximal cumulated toxicity 2. 70% of patients relapse within 1.5 months (median) from the completion of the first line treatment 1. Currently there is no approved drug for second line treatment of pleural mesothelioma MolMed s therapeutic option NGR-hTNF currently in Phase II as maintenance treatment: administered immediately after the pemetrexed to extend the time before relapse 100% of patients exiting 1 st line therapy are eligible NGR-hTNF currently in Phase III as 2 nd line treatment: administered after relapse to extend patients overall survival 15

16 NGR-hTNF incidence of indications: well positioned to become a potential blockbuster Indications Clinical Phase Incidence (EU27, USA, CA) Incidence (CN, JP) Total Primary indications Pleural Mesothelioma Pleural Mesothelioma NSCLC Second line (@70% ) First line Maintenance III 5'800 2' II 8'300 3' ' '000 II Squamous histology 140' ' '000 Soft tissue sarcomas II 29'200 30' Other relevant indications Ovarian cancer Liver cancer 77' '000 II Platinum resistant 36'000 60'000 96' ' '000 II Sorafenib resistant 70' ' '000 SCLC 152' ' Total 500'000 > 500'000 >

17 NGR-hTNF: competitive scenario key indications Malignant Pleural Mesothelioma (2nd line) No Approved Drug NGR-hTNF only candidate in Phase III clinical development Malignant Pleural Mesothelioma (1st line maintenance) No Approved Drug 1 candidate drug in Phase II clinical development (FAK inhibitor, Small Molecule) NGR-hTNF in Phase II clinical development Squamous NSCLC (1st line) No Approved Drug 2 drug candidates in Phase III clinical development (EFGR Antagonist, mab and mab) NGR-hTNF in Phase II clinical development Soft Tissue Sarcomas (2nd line) 1 approved drug (Votrient - PK Inhibitor, Small Molecule), high liver toxicity 1 drug approved only in EU (Yondelis - DNA Intercalator, Small Molecule) 1 candidate in Phase II clinical development (Cox-2 Inhibitor/ G Protein Coupled Receptor Mas Agonist, Peptide NGR-hTNF in Phase II clinical development 17

18 freedom-to-operate Exclusivity NGR-hTNF: Intellectual Property protection worldwide Use for the treatment of mesothelioma is covered by patent until 2029 NGR-TNF conjugate patent: granted in US, EU, Japan, South Korea and China, valid until 2021, extendable with Supplementary Protection Certificate until 2026 Exclusivity and freedom to operate through proprietary and in-licensed patents Patent claims Family Coverage (underlined = granted) Exp. Use in mesothelioma WO AU, CA, US, JP, EP, CN, IL, IN, KR, NZ, RU, ZA 2029 NGR-TNF conjugate = composition of matter Use in combination with IFNg Use at low dosage in combination with chemotherapy NGR peptide WO AU, CA, US, JP, EP (AL, AT, BE, CH, CY, DE, DK, ES, FI, FR, GB, GR, IE, IT, LT, LU, LV, MC, MK, NL, PT, RO, SE, SI, SK, SL, TR) CN, CZ, BG, EA, HK, HU, IL, IN, KR, SG, BR, MX, NO, PL, UA, UZ, ZA 2021 (with SPC 2026) WO AU, CA, CN, US, JP, EP (DE, ES, FR, GB, IT) 2023 WO US , US WO WO AU, CA, US, JP, EP, CN, EA, HK, IL, IN, NO, KR, ZA CA, EP (DE, GB, IT, FR, CH), US CA, EP (DE; GB; IT, FR, CH), US

19 NGR-hTNF: gearing up for marketing authorization in mesothelioma as first indication Enrolment completed in pivotal Phase III (400 patients), results expected during first half 2014 Results of two randomised Phase II trials in key indications (OC and STS) expected during first half 2014 High unmet medical need and low competition scenario: no drugs registered for second-line treatment or in Phase III development Orphan Drug designation + patent protection up to 2029 Development of commercial-scale manufacturing ongoing for liquid and lyophilized formulations: Low COGS: obtained by fermentation in E.coli (one single gene construct) Strong margin, also in case of price pressure or intense competition Business strategy: out-licensing / partnering 19

20 Thanks for your attention