Jefferies Global Healthcare Conference New York 2014

Transcription

1 Jefferies Global Healthcare Conference New York 2014

2 Disclaimer This document includes only summary information and does not intend to be comprehensive. Facts, figures and opinions contained herein, other than historical, are "forward-looking statements" and are based on Management s current expectations. Factors that could cause future results to differ materially from such expectations include, but are not limited to: the success of the Company s research strategy; the applicability of discoveries made therein; the difficulties inherent in the development of pharmaceuticals, including uncertainties as to the timing and results of preclinical studies; delayed achievements of milestones; reliance on collaborators; uncertainty as to whether the Company s potential products will succeed in entering human clinical trials and uncertainty as to the results of such trials; uncertainty as to whether adequate reimbursement for these products will exist from the government, private healthcare insurers and third-party payers; and the uncertainties as to the extent of future government regulation of the pharmaceutical business. Therefore those statements involve risks and uncertainties beyond the Company's control and actual results may differ materially from those stated by such forward-looking statements. The Company expressly disclaims any obligation to review or update any forwardlooking statements, targets or estimates contained in this document to reflect any change in the assumptions, events or circumstances on which such forward-looking statements are based unless so required by applicable law. 2

3 Corporate Overview: Zeltia S.A. Focused on Oncology Leader in developing and commercializing marine-derived oncology drugs Non-core businesses Molecular diagnostics; targeted RNAi agents with no systemic administration; and consumer chemicals Key Facts Ticker: ZEL SM (MEC) Market Cap*: 622 million 2013 Total Income: EBITDA: 23.8 * As of 30th May 2014 Oncology Non-core businesses Air fresheners, Insecticides Paintings, wood treatment 3

4 Corporate Overview: Unique R&D Capabilities The Sea World leader in development of marine-derived oncology compounds Library of 150,000 marine samples Excellent source of biological material for drug discovery 4

5 Fully Integrated: Drug Development Capabilities IP and Trademarks Commercialization Clinical Trials Production Keep last steps of purification or synthesis within PharmaMar Broad patent coverage License US & RoW Keep EU rights Preclinical Testing International clinical development in Europe, US & Canada Screening and Optimization Medicinal Chemistry Marine Expeditions R4O OR 5 OR 6 OR7 Evaluate New Compounds New chemical scaffolds & New MoAs R 3O R 2O Synthesis of identified NCEs Activity improvement R 1 N R 8 O O Build a Library (150,000 samples) 5

6 Pipeline: Targeting Multiple Indications Clinical Program / Indication Partners Phase I Phase II Phase III Yondelis Soft Tissue Sarcoma 2 nd /3 rd line; EU/others R/R ovarian cancer (Yondelis +Doxil); EU/others Soft Tissue Sarcoma 2nd/3rd line; US R/R ovarian cancer (Yondelis +Doxil); US Soft Tissue Sarcoma 2 nd /3 rd line; Japan (pivotal) Aplidin R/R multiple myeloma R/R T-cell lymphoma (Pivotal) R/R multiple myeloma Combo Bortezomib PM1183 Platinum Resistant ovarian cancer BRCA 1/2 associated breast cancer NSCLC Endometrial cancer Acute leukemia PM184 Combo Studies Registration Application Market 6

7 Yondelis : Commercial Success Validates Marine Biotechnology Platform Main Oncology Markets Europe 30% US 45% Japan 9% Source: IMS 2013 Yondelis approved in >75 countries for advanced STS and, in combination with Doxil / Caelyx, for relapsed platinum-sensitive ovarian cancer Yondelis partnered with Johnson & Johnson and TAIHO Pharmaceuticals 7

8 PFS Probability (%) Yondelis: Positive Survival Trend and Superior PFS (Taiho, Japan) Months Takahashi et al. ASCO

9 OS Probability (%) Yondelis: Positive Survival Trend and Superior OS (Taiho, Japan) N Median OS 95% CI Trabectedin BSC HR= 0.38 (95% CI [0.16, 0.91] ) P value = Months Takahashi et al. ASCO

10 Aplidin: Marine Compound Aplidium albicans Plitidepsin Synthetic cyclic depsipeptide isolated from the marine tunicate Aplidium albicans First in class drug with a new and different mechanism of action compared to current drugs used in the clinic The potential target is the proto-oncogene eef1α 10

11 Aplidin : ADMYRE (Phase III) III) Relapsed/Refractory Multiple Multiple Myeloma Myeloma Design: Phase III, randomized (2:1), multicentre, after 3 but no more than 6 lines of chemotherapy, 2-parallel group Objective: Progression-Free Survival (PFS) Primary Endpoint: Increase of 60% in PFS in Arm A Number of patients: 250 Arms: A: Aplidin in combination with Dexamethasone (n=167) B: Dexamethasone alone (n=83) Interim analysis performed after the inclusion of 79 patients Phase III IDMC Positive Recommendation (December 2012) RR 30% Well tolerated Estimated date of final recruitment 2H14 Centres: America, Europe, Asia, Australia, New Zealand 11

12 PM1183: Potent Oncology Compound PM1183, a second generation Yondelis, with activity in new indications Yondelis Protein-binding moiety: Free to interact PM1183 M e O H O M e O O O A c O O O N H S H N H O O H N O C H 3 O N H O A c O O O N H S H N H O O H N O C H 3 DNA-binding moiety: Binds to minor groove and bends DNA 12

13 PM1183: Favourable PK Profile vs. Yondelis PM1183 is administered as a 1h infusion versus 24h infusion with Yondelis PM1183, 4 mg/m 2 Yondelis, 1 mg/m 2 PM1183 is well-tolerated with peripheral administration, while Yondelis is administered via central catheter PM1183 vs. Yondelis AUC HL PM Yondelis x tolerated dose 15x exposure at RD Less cumulative toxicity, better handling 13

14 PM1183: Compelling Activity in Platinum-Resistant Ovarian Cancer Phase IIb Results* PM1183 Topotecan CR 1 (3%) 0 (0%) PR 9 (27%) 0 (0%) SD 17 (52%) 8 (50%) PD 6 (18%) 8 (50%) ORR 10 (30.3%) 0 (0%) DCR 27 (82%) 8 (50%) Total * Poveda et al. ESMO

15 PFS Cumulative probability PM1183: Positive Survival Trend and Superior PFS Platinum-Resistant Stage II 1.7 mo. 5.7 mo. PM01183 (N=17 C=4) Topotecan (N=16 C=2) Censored HR: 0.30 (95%CI ) p=0.005* Time (months) *log-rank test Poveda et al. ASCO

16 OS Cumulative probability PM1183: Positive Survival Trend and Superior OS Platinum-Resistant Stage II Not reached 8.3 mo Time (months) PM01183 (N=17 C=9) Topotecan (N=16 C=4) Censored HR: 0.40 (95%CI ) p=0.039* *log-rank test Poveda et al. ASCO

17 PM1183: Design of Metastatic Breast Cancer Phase IIb Study PM1183 Single Agent Stage I 4 pts with ORR Arm A Mut-BRCA1/2 (n=20) Stage I I Arm B Unknown status (n=30) 3 pts with ORR + (n=33 patients) + (n=34 patients) Primary Endpoint: Overall Response Rate (RECIST v1.1) Centres USA & Spain: Investigators: Massachusetts General Hospital Dr. Steven Jay Isakoff Dana-Farber Cancer Institute Dr. Judy Garber Beth Israel Deaconess Medical Center Dr. Nadine Tung Hospital Vall d Hebrón Dr. Judith Balmaña Hospital Clínico Universitario de Valencia Dr. Ana Lluch 17

18 PM1183: Phase IIb PM1183/Gemcitabine combo in NSCLC PM1183/Gemcitabine combo EGFR-non mutated advanced NSCLC (1 prior line) n=120 Stratified by histology: squamous vs. non-squamous PM1183/ GEM n=40 PM1183 alone n=40 Docetaxel (ctrl. arm) n=40 Primary endpoint: PFS at 4 months Secondary endpoints: Safety Response rate PFS/ Overall Survival Pharmacogenomics 18

19 Evaluable patients (n=40) PM1183: Phase I Gemcitabine Combo (5%) 4(21%) 7(37%) 7% NSCLC 1(13%) 4(50%) 3% Pancreas RD is 800 mg/m2 Gem mg flat dose PM1183 both D1and D8 q3wk 1(17%) 4(67%) 1% Breast 2(40%) 1 (20%) 2% Ovarian 2 (100%) Mesothelioma RECIST v1.1 CR PR SD PD Excellent chronic tolerance (6+ cycles in 32% of patients) A phase II combination trial in NSCLC to start in 3Q

20 Evaluable patients (n=42) PM1183: Phase I Doxorubicin Combo. RECIST v1.1 CR PR SD PD (42%) 1(11%) 7 6 1(8%) 4(44%) 5 4 1(20%) (100%) Ovarian (n=1) 1(33%) 1(33%) 1(33%) Endometrial (n=3) 1(33%) 1(33%) 1(33%) Breast (n=3) 2(50%) 2(50%) NET (n=4) 6(50%) SCLC (n=12) 4(44%) STS (n=9) 2(40%) 2(40%) Bladder (n=5) 4(80%) 1(20%) Gastric/HCC (n=5) CR, complete response; HCC, hepatocellular carcinoma; NET, neuroendocrine tumours; PD, progressive disease; PR, partial response; pts, patients; SCLC, small-cell lung cancer; SD, stable disease; STS, soft-tissue sarcoma Excellent chronic tolerance (6+ cycles in 34 % of patients) Calvo et al. ESMO

21 Zeltia Group Financial Profile: Improving Total Revenues Total Income (Euro million) ,7 51, ,7 Start of Caelyx Shortage in EU: 3Q ,9 79,4 80,6 End of Caelyx shortage in EU: 2Q ,5 22,8 72,4 79,1 71,2 73,2 71,2 64,7 61, Consumer Chemicals Biopharmaceutical Other Income 21

22 Driving Profitability through Revenue Diversification Net EBITDA (Euro million) 40,0 30,0 + 13,2 + 20,4 4,9 + 23,8 3,8 20,0 10,0 11,3 10,0 8,5 13,1 22,8 26,2 0,0-5,9-8,4-7,3-6,3-10,0-20,0-21,0-6,4-8,0-3,9-30, Biotechnology Consumer Chemicals Other 22

23 Key Upcoming Events* PM1183 Start Phase III Pivotal Trial Endometrial C. Aplidin Final Recruitment Phase III trial Multiple Myeloma PM1183 Start Phase III trial Resistant Ovarian C. PM1183 Start Phase II Pivotal trial SCLC Yondelis: Expected New Approvals J&J Milestone Payment: USD 25mn Yondelis: Expected dossier filing STS Japan Yondelis: Expected dossier filing STS US J&J Milestone Payment Taiho Milestone Payment * Estimated dates 23

24 Thank you