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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 ®
• 1 oncology candidate, Lurbinectedin filed FDA December 16th 2019
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1. As of Feb 28 2020
PharmaMar & Jazz Pharmaceuticals
Sign a license and distribution agreement for lurbinectedin in US, 19th December 2019
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T R A N S F O R M AT I V E T I M E F O R P H A R M A M A R
BD: Transformative
seek synergistic
assets time
Lurbinectedin: Pipeline
within a drug (e.g.
ovarian, sarcoma, Pan- 5>
small cell, etc.)
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-focused
• Global expeditions activity facility • Post marketing sales force in Europe
• Over 200,000 samples • Synthesis & molecule • GMP Production trials (~ 65 people)
optimization • New drug candidates • Geographic licensing
• Patent protection • New ADC Payloads & partnering with
experienced companies
• Preclinical studies
Regulatory inspections passed from FDA, AEMPS, PMDA (US, Spain/EU, Japan)
6>
OUR ONCOLOGY PORTFOLIO:
Program / Indication Phase I Phase II Phase III Market
Basket trial (small cell lung cancer August 16th 2020 FDA
Single agent
expansion cohort)
Lurbinectedin PDUFA date
Single agent
Solid tumors
and combinations
Single agent
PM184 Solid tumors
and combinations
8
Dr. Luis Paz Ares
PIPELINE- LURBINECTEDIN
Development and commercial strategy
ClinicalPROGRAM/
CLINICAL Program /INDICATION
Indication PHASE
Phase I I Phase II II
PHASE PHASE Market
Phase IIIIII MARKET
LURBINECTEDIN
SCLC Combo
Relapsed Doxorubicin ATLANTIS
August 16th 2020 FDA
Basket trial SCLC cohort Single agent
PDUFA date
Single agent /
Solid tumors combinations
Commercialization Plans:
• EU: Utilize/expand existing Yondelis sales force and select regional distributors
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• US: License and distribution agreement with Jazz
• ROW: Regional partnerships
LURBINECTEDIN: SCLC
Market overview: Orphan drug designation granted in the United States and EU
The American Cancer Society expects that in 2019 Decision Resources, Inc. expects that in 2018,
there will be approximately 30,000 new cases of there will be approximately 61,300 new cases
small cell lung cancer in the United States1 of small cell lung cancer in the EU2
• SCLC represents a significant unmet medical need with limited late stage options.
• The 5-year survival rate is about 5%-10%3
• 2nd line Standard of Care: Topotecan; Median TTP ~3m; OS ~6m4
• Last FDA approved NCE for 2nd line, Topotecan (iv) 1996, (only sensitive patients)
• PHM internal market research estimates ~60% of patients get treated in 2 nd line
Sources:
1, American cancer Society and SEER Cancer Stat Facts https://seer.cancer.gov/statfacts/html/lungb.html
2. Data Monitor: Small cell lung cancer (SCLC) Market Spotlight, May 1 2018
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3. http://www.cancer.gov/types/lung/hp/small-cell-lung-treatment-pdq
4. https://www.accessdata.fda.gov/drugsatfda_docs/label/2014/022453s002lbl.pdf
S C L C O V E R T H E Y E A R S ; FA R L E S S P R O G R E S S T H A N I N N S C L C
Atezolizumab+
Cisplatin+ Carboplatin+ Carboplatin+
First line Etoposide Etoposide Etoposide
1985 1999 2019
S
C Topotecan
L Second line
1996
C
Adapted from ; Sabari et al, Clinical Oncology; September 2017 FDA approval
Alkylating
N Antimetabolites
S Antiangiogenesis
C Microtubule
IO
L EGFR 11 >
C TKI
TRK
Drug class failures 2nd line SCLC
• Aurora Kinase
• BCL2
• C-Kit
• DLL-3
• EGFR
• FLT3
• HDAC
• IGF
• mTOR
• PD1
• Proteosome inhibitor
• VEGF
• GD2
"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“ 12 >
Rudin C. Looking Ahead to New Therapies in Small Cell Lung Cancer. Clinical Advances to Hematology & Oncology 2018:16 (4): 269-272
LURBINECTEDIN: SCLC
USA: Current and emerging treatment paradigm #
14 >
MONO(1): COMBO(2):
• Combo with Doxorubicin (in NCCN guidelines within CAV)
• Mono 57% sensitive, 21% resistant
• Phase II n=27 saw OS 10.2m in CTFI 30+ (n=21)
• Phase II basket trial expansion n=105 with CTFI>0
• Phase III ATLANTIS fully accrued 613 pts;
• Primary endpoint ORR (investigator) 35%
OS endpoint expected 2020
• ITT OS 9.3m
• Stable brain mets allowed
• No brain mets
• Dosing Lurbi 2mg/m2 + 40mg Doxo/m2
• Lurbi dose 3.2mg/m2
• Phase III prophylaxis G-CSF
• G-CSF (22%)
Monotherapy NDA PDUFA date 16th Aug 2020 Combo top-line data expected 2H 2020 for
for Accelerated Approval for relapsed SCLC relapsed SCLC
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1. Source: ASCO 2019
2. Source: IASLC 2018
C O M B I N AT I O N : L u r b i n e c t e d i n + D o x o r u b i c i n
16 >
LURBINECTEDIN: PHASE I/II 2nd LINE SMALL CELL LUNG CANCER
Combination Doxorubicin:
17 >
Source: Forster et al IASLC 2018 “Overall Survival with Lurbinectedin Plus Doxorubicin in Relapsed SCLC. Results from an Expansion Cohort of a Phase Ib Trial”; except *DOR data from ESMO 2017
Lurbinectedin: PHASE III RELAPSED SMALL CELL LUNG CANCER
ATLANTIS Trial Design SCLC (Trial initiated August 2016)
Arm B:
Topotocan or CAV
(42%/58%) 18 >
19 >
M O N O T H E R A P Y F I N A L D ATA : A S C O 2 0 1 9
Efficacy
ORR, % 35.2
(95% CI) (confirmed responses) # ^ (26.2-45.2)
ORR, % 22.2
Resistant CTFI< 90 days (n=45) (11.2-37.1)
ORR, % 45.0
Sensitive CTFI = 90 days (n=60) (32.1-58.4)
Duration of response (months), median 5.3
(95% CI) (4.1-6.4)
Disease Control Rate *, % 68.6
(95% CI) (58.8-77.3)
Prior IO
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M O N O T H E R A P Y F I N A L D ATA : A S C O 2 0 1 9
Safety
* Per protocol: dose had to be reduced in case of grade 4 neutropenia * Lab abnormalities associated with a specific treatment, were considered a SAE, or were reasons for dose reduction or treatment delay
Adverse Events Monotherapy Topotecan label Topotecan von Pawel CAV (from Topo label)
Grade 3-4 2014
n=105 n=107 n=167 n=104
CTFI>0 CTFI>60 CTFI>60
Febrile Neutropenia 4.8% 28% 3% 26%
Anemia 6.7% 42% 30.5% 20%
Thrombocytopenia 4.8% 29% (G4) 54.3%2 5% (G4)
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1. G-CSF give as rescue in 71%, 43% and 18% respectively, Phase III using prophylaxis
2. Treatment-emergent abnormalities
M O D E L L I N G B A S I C S : F O R I L L U S T R AT I V E P U R P O S E S O N LY
USA EU
New per year1 ~30000 New per year1 ~60000
% treated 2nd line2 ~60% % treated 2nd line2 ~60%
DOR3 5.3m DOR3 5.3m
Annual Px IO front line4 $177-180k Annual Px IO front line5 $62-63k
Annual Px IO 3rd line4 $150-153k Annual Px IO 3rd line5 $53-54k
Market Share ? Market Share ?
1. See slide 10
2. Internal estimates 24 >
3. From ASCO 2019 data
4. WAC USA
5. Assumed EU average price ~35% USA
L U R B I NE C TE DI N: K E Y I P A N D B A R R IE RS TO E N T RY
US EU
25 >
Y O N D E L IS ®: K E Y I P A N D B A R R I E RS TO E N T RY
US EU Japan
Manufacturing Protection until 2026 Protection until 2021 Protection until 2021
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94.3 79.6 78.2
100,0 90.6
73.7
90,0 80.2 78.8 80 2,4
1,9
4,9 5,3
4,9
80,0 70 53.6
5,1
70,0 60
2
60,0 50 2,9
50,0 40 72,3 71
40,0 63,7
28.6 30
48,7
30,0
16.9 16.7 20
20,0
10
7.0
10,0
0
0,0 2016 2017 2018 2019
2016 2017 2018 2019
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KEY EVENTS
Catalyst calendar
• Lurbinectedin monotherapy SCLC basket trial oral presentation ASCO June 1st 2019
• Lurbinectedin monotherapy FDA grants Accelerated Approval path
• Lurbinectedin monotherapy filed Accelerated Approval USA December 16th 2019
• Partnership agreement for US rights signed with Jazz Pharma
• USA EAP open Q1 2020
• Lurbinectedin monotherapy NDA accepted 2/14/20 for priority review
• Lurbinectedin monotherapy PDUFA 8/16/2020
• Lurbinectedin ATLANTIS top-line data 2H 2020
• Lurbinectedin potential US launch 2H 2020
• File Lurbinectedin with EMA
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