Investor Presentation - Spring 2021 - Diana Living with post-stroke spasticity Sintra, Portugal - Ipsen
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Diana
Living with post-stroke spasticity
Sintra, Portugal
Investor Presentation
Spring 2021
1
Our Vision
To be a leading global mid-size biopharmaceutical company
with a focus on transformative medicines
in oncology, rare disease & neuroscience
2
Focus. Together. For patients & society
Maximize Strengthen
our brands pipeline
Drive Focus on
efficiencies culture
3
Strong & expanding global footprint
North America Western Europe3 Rest of World
33% of Total Sales1 51% of Total Sales1 16% of Total Sales1
From 11% to 33% of Accelerated
Continued market share
Total Sales over the last development in China
gains in all TAs
five years2 Expansion in new
geographies
countries where
countries with an
34 Ipsen presence 115+ Ipsen products
are marketed
TA: Therapeutic area
1. 2020 Specialty Care sales
4
2. From 2015 to 2020
3. Including France, Germany, Italy, Spain, United Kingdom
Growth drivers
Oncology
Continued growth
driven by 1L RCC &
Limited growth until Group net sales1
potential indication
other potential
expansion
CAGR 20-24 between
indications
+2% & 5%
Continued growth
Attractive growth
despite challenging
until generic erosion
Chinese environment • At constant exchange
rates and scope
Rare disease Neuroscience • Assuming potential
additional indications
Sales contribution Solid growth in line
Palovarotene depending on with attractive
potential FOP label market
5
RCC: Renal cell carcinoma; FOP: Fibrodysplasia ossificans progressive; 1L: First line
1. Including Consumer Healthcare
Investing in R&D for growth
Build a strong and best-in-class R&D
organization
• Streamline organization and increase efficiencies
• Build clinical operations excellence Increase R&D as
a % of net sales
Prioritize key internal development
programs
• Accelerate high-value programs driven by external
• Discontinue or partner low-priority programs innovation strategy
Increase R&D investment through
external innovation
• Early to late-stage transactions
• Leverage existing development organization
6
Accelerate external innovation &
strengthen pipeline
Oncology Rare disease Neuroscience
€3bn
cumulative
• Solid & hematological • Disease areas with • Focus on in-house
tumors unmet needs beyond recombinant long-
firepower for
endocrinology & bone acting toxins & TSIs pipeline
• Niche tumors or disease expansion
biomarker segments • Rare neurological
in broad tumors • Established & innovative disorders by 20241
technologies including
• LCM potential gene-based modalities
Focus on assets across all stages of development
with strengthened organization to execute on external innovation
7
LCM: Life cycle management; TSI: Targeted secretion inhibitors; EBITDA: Earning Before Interest, Tax, Depreciation and Amortization
1. Based on expected free cash flow and net debt ratio remaining under 2x EBITDA
Transforming Ipsen R&D
• Defined therapeutic area units
Organizational
• Centralized clinical operations
transformation
• Strengthened R&D operations team
• New governance model for major decisions
Portfolio
• Alignment of decisions with R&D strategy, priorities & resources
governance
• Assessment & prioritization of portfolio
Scientific • New leadership with biotech & industry experience
rigor • Strengthen links to key opinion leaders
External • External innovation further integrated into R&D
innovation • Expand team & broaden the scope & geographical footprint
8
Significant potential in late-stage pipeline
Expected regulatory submission Potential
2021 2022 2023 peak sales1
1L HCC 2L NSCLC
with with
atezolizumab atezolizumab
Pipeline in
>€700M
a product 2L mCRPC
with
atezolizumab
Potential to
establish SoC in
2L SCLC 1L PDAC >€300M
hard-to-treat
cancers
Depending on
Palovarotene Establish Chronic /
Episodic FOP
potential FOP
leadership in FOP
label
SoC: Standard of Care; RCC: Renal cell carcinoma; HCC: Hepatocellular carcinoma; NSCLC: Non-small cell lung cancer; mCRPC: Metastatic castration-resistant prostate cancer; SCLC: Small cell lung cancer; 9
PDAC: Pancreatic ductal adenocarcinoma; FOP: Fibrodysplasia ossificans progressiva; 1L: First line; 2L: Second line
1. Risk-adjusted
Pipeline
Phase I Phase II Phase III Registration
Cabometyx + atezolizumab IPN60130 Cabometyx + atezolizumab Dysport solution
Solid tumors FOP1 1L HCC Glabellar lines
IPN59011 Cabometyx + atezolizumab Dysport
Longer-acting neurotoxin 2L NSCLC NDO2
Ax
IPN10200 Cabometyx + atezolizumab
Longer-acting neurotoxin 2L mCRPC
Ax / Tx
Onivyde
2L SCLC
Onivyde
1L PDAC
Oncology Rare Disease Neuroscience palovarotene
FOP
Data shown as at the end of Q1 2021 1. Phase II ready 2. Regulatory submission expected in 2021. Ax: aesthetics; Tx: therapeutics; FOP: fibrodysplasia ossificans progressiva; 10
NSCLC: non-small cell lung cancer; mCRPC: metastatic castrate-resistant prostate cancer; SCLC: small cell lung cancer; PDAC: pancreatic ductal adenocarcinoma;
NDO: neurogenic detrusor overactivity; 2L: second line.Cabometyx®: shifting landscape in 1&2L aRCC
Today’s landscape Tomorrow’s landscape
CPI CPIs
combos Compelling 1L 30%
CPI
30% combination data CPI
55%
(CheckMate-9ER) combos
70%
Compelling 2L TKIs Compelling 2L
TKIs
monotherapy 70% monotherapy
70% TKIs data (METEOR) TKIs data (METEOR)
45%
30%
EU5 >50% TKI
market share
1L 2L 1L 2L
11
aRCC: advanced RCC; CPI: Checkpoint inhibitor; TKI: Tyrosine kinase inhibitor; EU5: European Union Five (France, Germany, Italy, Spain, United Kingdom)
Sources: Q3 2020 RCC Rx Tracker (KANTAR). 2L RCC Patient share within the TKI Market.Cabometyx® | COSMIC-312: significant expansion
opportunity in HCC
HCC (EU5) 1L HCC
• CPI combinations to become
Patients not receiving any active new SoC
treatment / deceased patients
• Approval expected in 2022
1L
~14K
patients
2L
~5.6K 2L HCC
patients
3L+ • Strong performance in key
~1.4K patients markets
DoT 7 months
DoT 4-5 months DoT 4-5 months
• Geographic expansion to new
(Combos) markets 2021+
HCC: hepatocellular carcinoma; CPI: Checkpoint inhibitors; SoC: Standard of care; DoT: duration of treatment; 3L+: Third and subsequent lines; EU5: European Union Five (France, Germany, Italy, Spain, United Kingdom)
12
Sources: Epidemiology (2020): Local registries or DRG (Robert Koch Institute (Germany); Decisions Support Group (Italy); Public healthcare security system Sniiram (Open Medic (France)), Patient Metrics Kantar Active
Disease, 2020 (UK and Spain); Treatment rates: Q1’2020 HCC Rx Tracker (GENACTIS)Expanding Cabometyx® potential: NSCLC & mCRPC
Non-mutated NSCLC 2L+ (EU5) High risk mCRPC (EU5)
Patients not receiving any Patients not receiving any
active treatment / deceased active treatment / deceased
patients patients
2L 1L
~50K patients ~22K patients
3L 2L
~16K patients ~13K patients
DoT 6 months DoT 5 months DoT 7 months DoT 6 months
13
NSCLC: Non-small-cell lung cancer ; DoSources: Epidemiology: Patient Metrics (KANTAR – Active Disease, 2026); Treatment Rates: Kantar Treatment Architecture report (2019).; % of high risk mCRPC patients: Ipsos
Oncomonitor (Q1’2020)Onivyde® LCM: expansion into new tumor types
2024+
2023
Other possible LCM
indications
2022 1L PDAC2
2L SCLC2 28K drug treated patients
DoT 8 months
12K drug treated patients
DoT 5 months
2019
2L PDAC
35% in post-gemcitabine 2L+1
12K drug treated patients
DoT 3 months Peak sales for Onivyde® to exceed €300m3,
including additional potential indications
LCM: Life cycle management; PDAC: Pancreatic ductal adenocarcinoma; SCLC: Small cell lung cancer; DoT: Duration of treatment
1. IQVIA APLD claims, September 2020
14
2. Expected regulatory submission dates
3. Risk-adjustedDecapeptyl®: ongoing growth story
Key Facts Growth drivers Drivers of growth
• Attractive market dynamics
+3% CAGR
Net sales growth 2015-2020 • Market share gains in EU and RoW
• China performance impacted by competitive
Market Leader in the EU environment
Commercialized in • Focus on long-acting formulations,
70+ countries worldwide especially 6 months
ADTs remain backbone
therapy in PC1
Continued growth despite challenging environment in China
15
ADT: Androgen Deprivation Therapy; PC: Prostate cancer: EU: European union; RoW: Rest of world
1. EAU Treatment guidelinesDysport®: excellence in neurotoxins
Ipsen Dysport® Total Sales 2015-2020
€m Key Facts
CAGR: +6%
388
+6% CAGR Total Sales growth
348 353 2015-2020
328
279 285
Leading market position
Dysport® #2 globally
#1 in several markets
Complexity hurdles
Specialized & highly regulated
manufacturing process
2015 2016 2017 2018 2019 2020
16Efficiency, focus and agility to fuel growth
Generate efficiencies Focused and agile operating model
Smart spending Simpler operations
Manufacturing efficiencies Excellence in execution
Digital enablement Transformed R&D organization
17Focus & optimize resources
Smart • Focus on high priority projects
• Procurement savings Lower SG&A as a %
spending
• Centralization, outsourcing and right-sizing of net sales by 2024
Simpler • Process optimization & simplification
• Organization & footprint adjustment
operations
• Adoption of new ways of working
Improve COGS to
Manufacturing • Relocation of Onivyde® manufacturing limit negative
• Productivity initiatives
efficiencies
• Process improvement program
impact of product
mix
Digital • Manufacturing 4.0
• Leverage implementation of S4/Hana
transformation
• Digitalization of go-to-market
185,700+ employees committed to society
with clear KPIs by 2024
Compensation
of
management
& credit
Employees Communities Environment facility include
• Best place to work certification • 1/3+ of employees supporting • 21% reduction of greenhouse
social
in >75% of countries healthcare and environment gas emissions1,2 responsibility
• Gender balance1 in global communities1 • 24% reduction of water
leadership team • Continue support for IFPMA consumption metrics1
• Fill 65% of leadership roles via Access Accelerated initiative3 • 20% reduction of process
internal promotion waste
KPI: Key performance indicators; IFPMA: International federation of pharmaceutical manufacturers & associations
1. Metrics included in compensation of management & credit facility
19
2. Carbon equivalent emissions for all possible types of greenhouse gases emitted by Ipsen including scope 1 & 2 emissions
3. Through 2021Financial outlook1 2020 to 2024
Group net sales Commitment to invest €3bn cumulative
CAGR 2020-24 in R&D supported by firepower for pipeline
between +2% & +5% SG&A efficiencies expansion
• At constant exchange rates and scope • Lower SG&A as a % of net sales driven • Excluding the sale of any assets
• Assuming potential additional by focus & optimization • Based on net debt below 2.0x EBITDA
indications • Higher R&D as a % of net sales driven
by external innovation strategy
20
EBITDA: Earnings Before Interest, Tax, Depreciation and Amortization
1. Ipsen is on track to deliver its previous 2022 financial targets and is committed now to a new 2024 financial outlookCapital allocation prioritized to
external innovation
PRIORITIES FOR CAPITAL €3bn cumulative
ALLOCATION firepower for
Sales growth and pipeline expansion
higher conversion • Priority to external
of EBITDA innovation and business by 2024
development
INCREASED • Limited evolution of dividend based on net debt
Working capital CASH FLOW
improvement • Share buyback only to cover below 2.0x EBITDA
GENERATION
management incentive plan
• Limited milestone payments
Lower capital except contingent Onivyde®
expenditures payment for new indications
21
EBITDA: Earnings Before Interest, Tax, Depreciation and AmortizationDiana
Living with post-stroke spasticity
Sintra, Portugal
Investor Presentation
Spring 2021
22APPENDIX
23Q1 2021
Encouraging top-line and pipeline progress
Key highlights
Total Sales +5.5% to €659m Regulatory EU approval
• Specialty Care +6.4% to €612m • Cabometyx + nivolumab in 1L aRCC
COVID-19 Near term
• Limiting diagnoses, treatments and • Cabometyx 1L HCC data readout
patient care • Palovarotene regulatory progress
Full-year guidance confirmed
1L: first-line treatment; aRCC: renal cell carcinoma; HCC: hepatoceullar carcinoma. 24Q1 2021 sales highlights
Oncology
COVID-19
+4.9% limiting diagnoses and treatments
€495m: 75% of Total Sales
+2.5%, driven by North America (+5.1%)
+4.9%
€m Some stocking in Europe in Q1 2020
31 26 Continued share growth
72 83
+12.0%, driven by recovery in China
97 106 Gaining market share
Focusing on the 6m formulation
+16.4%
286 277 Strong volumes across most geographies
Approval in combination in 1L aRCC
-6.9%
Q1 2020 Q1 2021
Particular adverse COVID-19 impact in the U.S.
Somatuline Decapeptyl Cabometyx Onivyde Other Oncology
All growth rates in this presentation are at constant exchange rates, unless otherwise stated. Absolute values are shown at actual exchange rates. 25Q1 2021 sales highlights
Consumer Healthcare
+18.9% -5.4%
€102m: 15% of Total Sales €47m: 7% of Total Sales
+18.9% -5.4%
€m
• Aesthetics driving the €m
• Smecta -4.9%, driven by 7
6 Other
performance the slowdown of the 7
diarrhea market 7 Fortrans/
• Therapeutics and Europe overall 10 Eziclen
9
still impacted by the pandemic 102 • Improving conditions in Tanakan
93 10 9
China
Forlax
• Strategic review ongoing 18 16 Smecta
Q1 2020 Q1 2021 Q1 2020 Q1 2021
All growth rates in this presentation are at constant exchange rates, unless otherwise stated. Absolute values are shown at actual exchange rates. 26Q1 2021 sales growth driven by Specialty Care
Specialty Care Consumer Healthcare
Total Sales: €612m Total Sales: €47m
€m +6.4% €m -5.4%
+2.5%
-4.9% Specialty Care
€2,381.1m
+5.9%1
-5.6%
277 -10.0%
+12.0% +18.9% 16 +6.1%
+16.4% -10.6%
Consumer Healthcare
9
106 102
83 -6.9%
9
€210.6m
7 6
-23.9% -9.7%
26 -21.3%1
8 5
Somatuline Decapeptyl Dysport Cabometyx Onivyde NutropinAq Increlex Smecta Forlax Tanakan Fortrans/Eziclen Other
All growth rates in this presentation are at constant exchange rates, unless otherwise stated. 27Cabometyx® | COSMIC-312: 1L HCC study design
Key inclusion criteria (N=740, global
enrollment completed; continued Cabozantinib 40 mg QD +
extended enrollment for China) Atezolizumab 1200 mg Q3W Primary endpoint
• PFS-BIRC/OS
• No prior systemic anticancer therapy (cabozantinib +
• Child-Pugh Class A atezolizumab
• BCLC Stage B or C R vs. sorafenib)
• ECOG PS < 1 Sorafenib 400 mg BID
2:1:1
• Measurable disease per RECIST v1.1 Secondary endpoint
• PFS
Stratification factors (cabozantinib
• Disease etiology (HBV, HCV, other) vs. sorafenib)
• Region (Asia, other) Cabozantinib 60 mg QD
• Extrahepatic spread (yes, no)
Global topline results expected H1 2021; EU filing in 2021, assuming positive results
BIRC: Blinded independent review committee; HBV: Hepatitis B virus; HCV: Hepatitis C virus; HCC: Hepatocellular carcinoma; BCLC: Barcelona clinic liver cancer staging; ECOG: Eastern cooperative oncology group functional
28
status measure; RECIST: Response evaluation criteria in solid tumors; QD: Once daily; Q3W: Once every three weeks; BID: Twice daily; PFS: Progression-free survival; OS: Overall survival
Source: Kelly et al. ASCO 2019, Future Oncology June 2020, NCT04471428Cabometyx® | CONTACT-011 & CONTACT-021: trial designs
Phase III – NSCLC - CONTACT 01 Enrollment: N = 350; Key milestones: expected topline readout in 2022
• Radiographic progression during or following Primary endpoint
Cabozantinib 40mg QD +
platinum-containing and anti-PD-L1 therapy for • OS
Atezolizumab 1200 mg IV Q3W
metastatic NSCLC
R
• Measurable disease per RECIST 1.1 Secondary endpoints
• Known PD-L1 status or availability of tumor
1:1 • PFS per investigator
tissue for central PD-L1 testing Docetaxel 75 mg/m2 IV Q3W • ORR
• ECOG 0-1 • DOR
• QoL
Phase III – mCRPC - CONTACT 02 Enrollment: N = 580; Key milestones: expected topline readout in 2023
• Measurable visceral metastases, OR measurable Cabozantinib 40mg QD + Primary endpoints
extrapelvic lymph node metastases Atezolizumab 1200 mg IV Q3W • OS, PFS by RECIST
• Received 1 NHT for mCSPC, M0 CRPC, or 1L R 1.1 per BICR
mCRPC 1:1
• No prior chemotherapy for mCRPC Abiraterone 1000 mg QD + Secondary endpoint
Prednisone 5 mg BID, OR • ORR per BICR
• ECOG 0-1
Enzalutamide 160 mg QD
NSCLC: Non-small cell lung cancer; RECIST: Response evaluation criteria in solid tumors; PD-L1: Programmed death-ligand 1; ECOG: Eastern cooperative oncology group functional status measure; QD: Once daily; Q3W: Once every
three weeks; BID: Twice daily; OS: Overall Survival; PFS: Progression-free survival; ORR: Objective response rate; DOR: Duration of Response; QoL: Quality of life; mCRPC: Metastatic castrate-resistant prostate cancer;
29
mCSPC: Metastatic castrate-sensitive prostate cancer; NHT: Neoadjuvant hormonal therapy; 1L: First line; M0 CRPC: Non-metastatic castrate-resistant prostate cancer; BICR: Blinded independent review committee
1. Sponsored by Roche, co-funded by Exelixis/Ipsen/TakedaOnivyde®: 1L pancreatic ductal adenocarcinoma (PDAC)
Phase 2 results Phase 3 NAPOLI-3 study status & design
• Phase 3 study ongoing
sum target lesion diameter (%)
Among 29
Best change from baseline in
70
60
50
40
30
evaluable • Received FDA Fast Track designation in June 2020
20
10
0
No No No
change change change
patients who • Expected topline readout: 2023
received selected
-10
-20
-30
Complete response
NALIRIFOX1
-40
-50
-60 Partial response dose, 23 (79%)
-70
-80
-90
-100
Stable disease (tumor shrinkage)
Tumor growth
had tumor Metastatic 1L R
67 41 73 71 17 51 47 70 79 66 11 9 49 61 48 63 62 72 59 15 40 13 75 53 60 12 50 45 76 shrinkage PDAC 1:1
Individual Patients Gemcitabine /
Abraxane
NALIRIFOX1 Phase 1/2 - 50/60 Cohort
N 32 (29 metastatic & 3 locally advanced)
Complete Response 1 (3.1%) 1L mPDAC (N=750)
• Histologically/cytologically Primary endpoint
Partial Response 10 (31.3%)
confirmed PDAC • OS
Stable Disease 15 (46.9%)
11 (34.4%)
• Not previously treated in the Secondary endpoints
ORR; % (95%)
26 (81.3%)
metastatic setting • PFS
DCR; % (95%)
• >1 metastatic tumor measurable • ORR
DOR (median); % (95% CI) 9.4 months (3.52-NE)
per RECIST v1.1 • Safety
PFS (median); % (95% CI) 9.2 months (7.69-11.96) • ECOG performance status of 0 or 1
OS (median); % (95% CI) 12.6 months (8.74-18.69)
PDAC: Pancreatic ductal adenocarcinoma; ORR: Overall response rate; DCR: Disease control rate; DOR: Duration of response; PFS: Progression-free survival; OS: Overall survival;
RECIST: Response evaluation criteria in solid tumors; ECOG: Eastern cooperative oncology group functional status measure; FDA: Food and Drug Administration 30
1. Onivyde, administered in combination with oxaliplatin, fluorouracil (also known as 5-FU) and leucovorin (which is often abbreviated as LV)
Source: ESMO World Congress on Gastrointestinal Cancer 2020 Oral Presentation. Abstract LBA-1Onivyde®: 2L small cell lung cancer (SCLC)
Phase 2 results Phase 3 RESILIENT study status & design
• Phase 3 study ongoing
• Expected topline readout 2022
• Potential for accelerated regulatory review
Onivyde®
monotherapy
R
2L SCLC
1:1
Topotecan IV
monotherapy
Resilient Study Part 1 – 70 mg/m2 Cohort
2L SCLC (N=450)
N 25 • Histologically/cytologically Primary endpoint
Complete Response 1 (4%) confirmed SCLC with evaluable • OS
Partial Response 10 (40%) disease per RECIST v1.1 Secondary endpoints
Stable Disease 7 (28%) • Progression after 1L platinum- • PFS
ORR; % (95%) 11 (44%) based therapy • ORR
• Prior immunotherapy is allowed • Safety
DCR; % (95%) 18 (72%)
• ECOG performance status of 0 or 1
SCLC: small cell lung cancer; ORR: Overall response rate; DCR: Disease control rate; RECIST: Response evaluation criteria in solid tumors; PFS: Progression-free survival; 31
ECOG: Eastern cooperative oncology group functional status measure; 2L: Second line
Source: IASLC World Congress on Lung Cancer 2019 Oral Presentation, OA03.03.Targeting best-in-class approach to MAPK driven tumors
Pan-RAFi: broader & more
complete activity than
current agents
MAPK pathway is one of the most commonly IRICoR
mutated oncogenic driver pathways in cancers
with high unmet medical need ERKi: prevent pathway
reactivation, more durable
Room for improvement as existing approaches pathway inhibition
provide insufficient pathway inhibition against a
subset of the mutations AGV Discovery
A portfolio with both pan-RAFi & ERKi programs enables us to develop best-in-class wholly owned
monotherapy & combination treatments for MAPK-driven cancers
32
MAPK: Mitogen-activated protein kinases; pan-RAFi: Pan-RAF kinase inhibitors; ERKi: ERK inhibitorsFOP is an ultra-rare, Characteristic malformed great
severely disabling genetic disorder toes & hallux valgus4
• FOP characterized by bilateral malformations of the great toes, &
the formation of bone in soft connective tissues known as
heterotopic ossification (HO)1
• HO leading to progressive, cumulative disability
• Sporadic episodes of painful soft tissue swelling called ‘flare-ups’ Illustration of HO progression over time5
can precede new HO1
• Prevalence of FOP being up to 1.36 per million individuals2
• 97% of patients with FOP have classic FOP, associated
with an R206H mutation in the gene ACVR1 (also known as ALK2)3
FOP: Fibrodysplasia Ossificans Progressiva; HO: Heterotopic Ossification; ACVR1: activin A receptor type I; ALK2: activin receptor-like kinase-2
1. Pignolo RJ. et al. Pediatr Endocrinol Rev 2013;10 Suppl 2:437–48
2. Baujat G. et al. Orphanet J Rare Dis 2017;12:123
3. Zhang W. et al. Bone 2013;57(2):386–91 4-year old 10-year old 33
31-year old
4. Image from Pignolo RJ. et al. Orphanet J Rare Dis 2011;6:80, licensed under CC BY 2.0 (creativecommons.org/licences/by/2.0)
5. Image from Pignolo RJ. et al. Orphanet J Rare Dis 2019;14:98, licensed under CC BY 4.0 (http://creativecommons.org/licenses/by/4.0/)Palovarotene: 62% reduction in mean annualized new HO
volume1 in Phase 3 MOVE trial
35,000
Annualized new HO (mm3 +SEM)
• Demographics & baseline characteristics sufficiently 30,000
similar between MOVE & NHS to support comparison 25,000
20,000 23,318
• New HO volume used as a study endpoint to measure
15,000
FOP disease progression
10,000
• Post hoc analyses showed substantial efficacy at 3rd 5,000
8,821
interim analysis, despite pre-specified futility
0
NHS MOVE
• Most common AEs retinoid-associated & managed with N=98 N=97
prophylactic and/or symptomatic therapy • Weighted linear mixed effects (wLME) model
– Identified risk of premature physeal closure in children estimate: -11,611 mm3
• wLME nominal p-value: p=0.0292
FOP: Fibrodysplasia ossificans progressiva; HO: Heterotopic ossification; NHS: Natural history study; AE: Adverse event; SEM: standard error of the mean
34
1. Non-transformed data; b2-sided p-value ±SEM
Source: Pignolo RJ. et al. ASMBR Sep 2020 Oral PresentationLANTs: differentiated therapeutic properties
Therapeutic efficacy benefits: longer duration of action
Safety benefits: higher therapeutic index enabling wider range of possible doses
Less local and contralateral spread vs native toxins in non-clinical model
Increased convenience: fewer injections/year
Strong IP protection
First-patient dosing in the program
35
LANT: Long-acting neurotoxin; IP: Intellectual property; FPFV: First patient first visitOncology
Key ongoing clinical-trial highlights
Trial Population Patients Design Endpoints Status
Sorafenib
Cabometyx Recruiting
or Primary: PFS, OS
COSMIC 312
1L HCC 740 Cabometyx + atezolizumab Secondary: PFS single-agent
Phase III Data anticipated
or Cabometyx arm
NCT03755791 Q2 2021
Cabometyx
Cabometyx Recruiting
Docetaxel Primary: OS
CONTACT-01
2L NSCLC 350 or Secondary: PFS, ORR, duration
Phase III Data anticipated
Cabometyx + atezolizumab of response
NCT04471428 2023
Second novel hormonal Primary: OS, PFS
Cabometyx Recruiting
therapy (abiraterone and Additional endpoints: ORR,
CONTACT-02
2L CRPC 580 prednisone or enzalutamide) prostate-specific antigen
Phase III Data anticipated
or response rate and duration of
NCT04446117 2024
Cabometyx + atezolizumab response
Primary: maximum tolerated
Cabometyx
dose / recommended dose,
Phase Ib Solid tumors 1,732 Cabometyx + atezolizumab Recruiting
ORR
NCT03170960
Secondary: safety
36
CRPC: castration-resistant prostate cancer; ORR: objective response rate; OS: overall survival; PFS: progression-free survival.Oncology
Key ongoing clinical-trial highlights
Trial Population Patients Design Endpoints Status
Onivyde Nab-paclitaxel + gemcitabine Recruiting
NAPOLI 3 or Primary: OS
1L PDAC 750
Phase III Onivyde + 5-FU/LV + Secondary: PFS, ORR, safety Data anticipated
NCT04083235 oxaliplatin 2023
Onivyde Active, not recruiting
Topotecan
RESILIENT Primary: OS
2L SCLC 461 or
Phase III Secondary: PFS, ORR, safety Data anticipated
Onivyde
NCT03088813 2022
37Neuroscience
Key ongoing clinical-trial highlights
Trial Population Patients Design Endpoints Status
IPN59011 Ax
Moderate to
LONG-SET Dose escalation and dose finding Primary: Safety
severe upper 424 Recruiting
Phase I versus Dysport or placebo Secondary: Efficacy
facial lines
NCT04736745
IPN10200 Ax
Moderate to
LANTIC Dose escalation and dose finding Primary: Safety
severe upper 424 Recruiting
Phase I versus Dysport or placebo Secondary: Efficacy
facial lines
NCT04821089
IPN10200 Tx
Adult patients Primary: Safety
LANTIMA Dose escalation and dose finding
with upper limb 209 Secondary: Efficacy Recruiting
Phase I versus Dysport or placebo
spasticity
NCT04752774
38Rare Disease
Key ongoing clinical-trial highlights
Trial Population Patients Design Endpoints Status
Primary: annualized change in
Palovarotene Palovarotene - 5mg QD and new HO volume
MOVE FOP upon flare-up, 20mg QD for Secondary: subjects with new
107 Active, not recruiting
Phase III (chronic) 28 days, followed by 10mg HO, number of body regions
NCT03312634 for 56 days with HO, subjects with flare-
ups, rate of flare-ups, safety
HO: heterotopic ossification. 39Diana
Living with post-stroke spasticity
Sintra, Portugal
Investor Presentation
Spring 2021
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