Pharma Mar Sa · BME:PHM
Pharma Mar Sa — Investor Presentation (February 2019)
30 slides from Pharma Mar Sa's investor presentation, February 2019
Slide 1: February 2019
February 2019
Slide 2: Disclaimer
February 2019 Disclaimer This presentation contains forward-looking statements that include information about possible or assumed future results of the business, financial condition, liquidity, results of operation, clinical program, plans and objectives of Pharma Mar, S.A. ("PharmaMar" or the "Comp
Slide 3: Investment Highlights
INVESTMENT HIGHLIGHTS Leader in development & commercialization of marine derived oncology drugs Global integrated biotech developing marine-derived and novel MoA oncology drugs 2 approved oncology drugs, Yondelis® and Aplidin ® Established Yondelis® oncology sales force in Europe • Strong partners
Slide 4: Yondelis: Commercial Expansion Worldwide
Y O N D E L I S ® - C O M M E R C I A L E X PA N S I O N W O R L D W I D E Yondelis Global Sales 2017: €132.5MN EU (PHM): €85MN + ROW (Partners): €48MN Royalty €4.8MN to PHM on sales PharmaMar Territories Janssen Territories Taiho Territories PharmaMar Subsidiaries / Distributors (Partnered) (Partne
Slide 5: Unique Fully Integrated Platform
U N I Q U E F U L LY I N T E G R AT E D P L AT F O R M Cell Biology Pharmaceutical Expeditions Chemistry & Development & Clinical & Preclinical regulatory Commercial and Collection Operations • Marine derived leads • Screening of antitumoral • FDA inspected production • Clinical trials • Oncology-fo
Slide 6: Our Oncology Portfolio
OUR ONCOLOGY PORTFOLIO: Milestone Program / Indication Phase I Phase II Phase III Market timing Soft tissue sarcoma 2nd/3rd line Single agent Yondelis Ovarian cancer 2nd/3rd line (1) Yondelis+Doxil(2) Aplidin R/R Multiple Myeloma 3th/4th line (3) Aplidin+Dexameth Small cell lung cancer relapsed Zeps
Slide 7: Zepsyre (Lurbinectedin)
ZEPSYRE ® (Lurbinectedin) Key oncology compound Zepsyre, a chemical analog of Yondelis®, with significant potential pharmacological advantages including improved tolerability, activity and convenience. Natural marine-based tetrahydroisoquinoline family of antitumor agents DIFFERENT PK PROFILE • Zeps
Slide 8: Zepsyre: Mechanism of Action
ZEPSYRE®: MECHANISM OF ACTION Zepsyre is a selective inhibitor of active transcription of protein-coding genes… Chromatin 1. Inhibition of active transcription SMARCC1 2. Binds to DNA (Cytosine-Guanine-rich motifs) SMARCA4 ARID1A 3. Displaces certain transcription factors from its Trabectedin promot
Slide 9: Zepsyre: Mechanism of Action, Tumor Microenvironment
ZEPSYRE®: MECHANISM OF ACTION …and modulates the tumor microenvironment. 1. Inhibition of TAMs 2. Inhibition of trans-activated transcription of cytokines e.g. IL6, IL8, CCL2 and PTX3 3. Reactivation of the immune system 9> Belgiovine C Br J Cancer 2017 Aug 22;117(5):628-638; Céspedes M Dis Model Me
Slide 10: Transcriptional Addiction as a Target in Cancer
T R A N S C R I P T I O N A L A D D I C T I O N A S A TA R G E T I N C A N C E R • Cancer cells aberrantly deregulate specific gene expression programs with critical functions in cell differentiation, proliferation and survival 1 • These altered gene programs in cancer cells have a striking dependen
Slide 11: Pipeline - Zepsyre (Lurbinectedin)
PIPELINE- ZEPSYRE® (Lurbinectedin) Development and commercial strategy ClinicalPROGRAM/ CLINICAL Program /INDICATION Indication PHASE Phase I I Phase II II PHASE PHASE Market Phase IIIIII MARKET Data DATAtiming TIMING ZEPSYRE® SCLC Combo Relapsed Doxorubicin ATLANTIS ~YE 2019E Basket trial SCLC coho
Slide 12: Zepsyre: SCLC Market Overview
Z E P S Y R E ®: S C L C Market overview: Orphan drug designation granted in the United States; Application expected to be submitted for the European Union in 2018 The American Cancer Society expects that in 2018 Decision Resources, Inc. expects that in 2018, there will be approximately 35,000 new c
Slide 13: SCLC Over the Years; Far Less Progress Than in NSCLC
SCLC OVER THE YEARS; FAR LESS PROGRESS THAN IN NSCLC Cisplatin+ Carboplatin+ First line Etoposide Etoposide 1985 1999 S C Topotecan L Second line 1996 C Third line Opdivo 2018 Adapted from ; Sabari et al, Clinical Oncology; September 2017 FDA approval 1985 1990 1995 2000 2005 2010 2015 2018 N Alkyla
Slide 14: SCLC is difficult to treat
"SCLC is difficult to treat in part because you can’t target an absent protein the way you can target a mutant protein—there’s nothing against which a drug can be directed" 14 > Rudin C. Looking Ahead to New Therapies in Small Cell Lung Cancer. Clinical Advances to Hematology & Oncology 2018:16 (4):
Slide 15: Zepsyre: SCLC Current and Emerging Treatment Paradigm
Z E P S Y R E ®: S C L C Current and emerging treatment paradigm 1st LINE MAINTENANCE 2nd LINE 3rd LINE APPROVED APPROVED Phase III trials APPROVED Platinum/Etoposide Topotecan Zepsyre®* Nivolumab Others NCCN NCCN NCCN Atezolizumab* Atezolizumab1* Irinotecan1* Paclitaxel1* Docetaxel1* Temozolomide1*
Slide 16: Zepsyre: Phase I/II Relapsed Small Cell Lung Cancer
Z E P S Y R E ®: P H A S E I / I I R E L A P S E D S M A L L C E L L L U N G C A N C E R Cohort A: ASCO 2015 n=21 Best RECIST v.1.1 overall response Kaplan-Meier global PFS and according to CTFI (n=21) During treatment (n=21) Median PFS: 4.7 months (95%CI: 3.3-8.0 months) ORR: 67% (95%CI: 43-85) Glo
Slide 17: Zepsyre: Phase I/II 2nd Line SCLC
Z E P S Y R E ®: P H A S E I / I I 2 n d L I N E S M A L L C E L L L U N G C A N C E R Combo and Monotherapy latest data Cohort A+B Sensitive Resistant n=61 CR 2 (10%) 1 (4%) - PR 12 (57%) 9 (33%) 24 (39%) ORR 14 (67%) 10 (37%) 24 (39%) SD 3 (14%) 9 (33%) 21 (34%) PD 4 (19%) 8 (30%) 16 (26%) DCR 17
Slide 18: Why Is Monotherapy Data Better Than Cohort B?
W H Y I S O S M O N O T H E R A P Y D ATA B E T T E R T H A N C O H O R T B ? Demographics & other differences between Cohort Spider plot showing tumor variation over time during treatment B and monotherapy re patients and protocol with L/DOX and L alone in cohort B patients (n=27) 1 FAVOURS COMBO1
Slide 19: Non Head-to-Head Comparisons Second Line
N O N H E A D - T O - H E A D C O M PA R I S O N S S e c o n d l i n e Cohort A, FD Cohort B, BSA Cohort B, BSA Monotherapy Topotecan CAV (from combo doxo combo doxo combo doxo label Topo label) n=21 CTFI ≥0 n=27 CTFI≥0 n=21 CTFI>30 n=61 CTFI≥0 n=107 n=104 IASLC 2017 IASLC 2018 IASLC 2018 ASCO 2018
Slide 20: What Does Zepsyre Ovarian Phase III Trial Tell Us About SCLC?
W H AT D O E S Z E P S Y R E ® O VA R I A N P H A S E I I I T R I A L T E L L U S A B O U T SCLC? OVARIAN: SCLC: • Drug is active • Monotherapy also shows activity. • Drug is tolerated • Phase III BSA Dose ~90% Phase II Fixed RD2 • Phase III BSA Dose ~75% of Phase II Fixed Dose1 • Prophylaxis G-CSF
Slide 21: Zepsyre: Phase III Relapsed Small Cell Lung Cancer
Z E P S Y R E ®: P H A S E I I I R E L A P S E D S M A L L C E L L L U N G C A N C E R ATLANTIS Trial Design SCLC (Trial initiated August 2016); Anticipate data ~YE 2019 • Primary endpoint: median OS HR ≤ 0.75 with 90% power at ~510 events. Control arm modelled for ~7.5m • Key secondary endpoints: -
Slide 22: Zepsyre: ATLANTIS Trial, Change Primary Endpoint to OS
Z E P S Y R E ® : AT L A N T I S T R I A L Change primary endpoint from PFS to OS • PharmaMar recently received the OS analysis for both cohort B of the combo Phase I/II (submitted to IASLC, World conference on Lung Cancer) and the monotherapy trial (presented at ASCO 2018), which prompted the chang
Slide 23: Comparing Patient Populations and Outcomes
C O M PA R I N G PAT I E N T P O P U L AT I O N S A N D O U T C O M E S Cohort A Cohort B Monotherapy ATLANTIS N 21 27 61 (target 100) 600 Data ASCO ´15 ESMO´17/ ASCO ´18 ~Y/E 2019 World Lung 2018 Regimen Combo FD Combo BSA Mono BSA =Cohort B Line Relapsed Relapsed 2nd Relapsed CTFId >30 >30 >0 >30
Slide 24: Key IP and Barriers to Entry
KEY IP AND BARRIERS TO ENTRY Yondelis JP Formulation patent EU Data exclusivity, EU Usage patent EU Formulation patent Ovarian Orphan Sarcoma 2019 2021 2022 2023 2025 2028 2030 2030 EU/US/JP Manufacturing JP Data exclusivity, US Data exclusivity, US Formulation patent exclusivity, Sarcoma Orphan S
Slide 25: Organizational Chart
ORGANIZATIONAL CHART CONSUMER PHARMA MAR, S.A. CHEMICALS ONCOLOGY Zelnova Zeltia, S.A.* (Spain) Pharma Mar GmbH Pharma Mar USA Inc (Germany) (United States) Copyr, SpA DIAGNOSTICS (Italy) Pharma Mar S.a.r.l. Pharma Mar AG (Switzerland) (France) Genomica, S.A. (Spain) Pharma Mar G.m.b.H Pharma Mar S.
Slide 26: Group Revenues and R&D Expenses
GROUP REVENUES AND R&D EXPENSES 16.7 Net Operating cash flow 2016: €-8.4mn 2017: €-1.46mn 9M2018: €-20.3mn 69.7 26 >
Slide 27: Key Facts and Principal Shareholders
K E Y FA C T S A N D P R I N C I PA L S H A R E H O L D E R S KEY FACTS JOSE MARIA FERNANDEZ SOUSA-FARO* 11.08% PEDRO FERNANDEZ PUENTES 4.49% • Ticker: PHM SM • Market Cap*: € 300 million ROSP CORUNNA 5% • 2017 Total revenue: € 179 million OTHERS BOARD MEMBERS 0.6% • 2017 EBITDA: € -7.4 million • 9M
Slide 28: Key Events: Catalyst Calendar
KEY EVENTS Catalyst calendar • ASCO Zepsyre® : Monotherapy SCLC, TiP ATLANTIS, Ewing’s sarcoma • Zepsyre® SCLC ATLANTIS Phase III complete enrolment • Zepsyre® SCLC Orphan drug designation decision (US) • Zepsyre® P I/II cohort B OS data presentation at IASLC World Lung (September 24) • Zepsyre® COR
Slide 29: Management; Who's Here
MANAGEMENT; WHO’s HERE José María Fernández, Ph.D. Founder (1986), CEO and Chairman of the Board Luis Mora Managing Director, Oncology Business Unit José Luis Moreno Director, Investor Relations & Capital Markets Pascal M. Besman Chief Operating Officer, PharmaMar USA Jose-Antonio Lopez-Vilarino 29
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