SlideShare a Scribd company logo
TARIS® Market Research: Market
Positioning, Pricing, Sales Forecast
Preliminary Draft
Jody Kalt, Terry York, Gabi Forman
Investor Deck part III
CONFIDENTIAL 1
 Bladder cancer represents a large and underserved market opportunity that has been devoid
of innovation for many years
 Level of interest/development activity have increased dramatically in the past several years
 Driven by a better understanding of immune mechanisms at play both systemically and in the
tumor micro-environment
 Bladder cancer is complex - represents a number of different patient cohorts with differing
unmet needs
 Since the AGN transaction in 2014, TARIS has spent a majority of our time focused on
understanding the nuances of treating this disease and advancing our lead program into
clinical proof-of-concept studies, which are now just starting to readout
CONFIDENTIAL 2
Executive Summary: 1 of 2
 Our thinking has evolved in several different directions:
 Target: Initially only NMIBC, now both NMIBC and MIBC, with the later being higher priority
 MOA: Initially improved cytotoxic modality due to PK play, now potential multimodal activity
 Our initial proof-of-principle study in MIBC has validated our belief that our approach has the
opportunity to transform the treatment of this disease
 TARIS just completed a significant market research study working with Defined Health to try
and better understand the commercial opportunity for this technology
 We believe, if successful in development, that this represents a $1B+ opportunity (US) with
several opportunities to pursue a rapid registration pathway
CONFIDENTIAL 3
Executive Summary: 2 of 2
Incidence and Prevalence
Bladder Cancer
CONFIDENTIAL4
5
Bladder Cancer Treated Patients, 2016
(US Only)
Target for Most
Current I/O
Therapies
Managed By:
Uro-oncologists
Managed By:
Uro-oncologists
Medical Oncologists
Radiation Oncologists
Managed By:
Medical Oncologists
Radiation Oncologists
All Bladder
Cancer
102,546 pts
NMIBC
81,725
MIBC
16,855
Metastatic
3,966
 TARIS has completed substantial market research in bladder cancer with DefinedHealth
 First project: 2012, focused on profiles in NMIBC
 Recent project: 2016, focused on profiles in both NMIBC and MIBC
 Summary of scope: current project
 24 qualitative interviews:
 8 KOL uro-oncologists
 16 additional interviews: community urologists, medical oncologists, radiation oncologists
 60 quantitative interviews via web-based survey
 5 payer interviews (Medical/Pharmacy Directors)
 Initially tested 4 product profiles in qualitative interviews, after completion of the initial interviews, added a 5th TPP with an
additional 4 qualitative interviews, prioritized 3 to include in quant
 TPPs numbered by severity of disease (low to high), not necessarily by internal development priority
 TARIS did not evaluate TPPs for low risk NMIBC or treatment-naïve high risk NMIBC
 Included in previous 2012 research
 These indications represent additional potential upside
CONFIDENTIAL 6
TARS® Market Research Methodology
 All specialties generally reacted favorably to the TAR-200 product profile as an easily deployed and retracted device in the office setting for
local, sustained delivery of drugs to the bladder
 However, they wanted to be assured that TAR-200 would be tolerable and not interfere with follow-up:
 Foreign body placement location and rubbing could cause inflammation to the bladder lining
 Continuous delivery of gemcitabine could irritate the bladder
 Either could cause pain and/or increase urinary urge and voiding frequency, dysuria
♦ Physicians generally understood advantage of longer duration tx (7 days vs. 1 hr of BCG/chemo), support the selection of gemcitabine as agent of choice
 However, physicians want to see clinical data on how week-long, sustained, low dose gemcitabine is better than short exposures of multiple
high dose instillations in terms of improvements in efficacy/outcomes
 Urologists: having an intravesical product pre-loaded with gemcitabine on the shelf would increase patient access and physician/office
convenience
 Community urologists wanted to know if it is economical for a Urology office – what’s the profit margin?
 Community radiologist considered TAR-200 as a good option for older, frail (> 80+ yrs in age are not good candidates for surgery or Chemo as
kidney function is not good) that are left with radiation only
Physicians Reacted Quite Positively to The TAR-200 Product
Profile
 Source: 1. DH Primary Research
CONFIDENTIAL 8
MIBC: ~$2.1B Total Addressable Peak US
Market
 Significant market research, including payors, complete
 At least 2x potential w/ EU, Japan, ROW
 US represents only 17% of WW cases
 Additional upside indications, not included in forecast
 High risk/metastatic disease (± checkpoint)
 High Risk NMIBC: BCG Naïve, BCG Unresponsive
$2,069
$0
$500
$1,000
$1,500
$2,000
$2,500
MIBC
Millions
Total Addressable Market
US MIBC Markets at Peak Patients Cost/Dose Doses/Patient Cost/Patient Total Addressable Market
RC Ineligible 7,285
$14k
9 (18 months) $126k $972M
NAC Ineligible 5,562 2 (upfront) $28k $165M
cT2 Bladder Sparing 6,246 11 (2 years) $156k $932M*
 EpiLUTS: US population-based survey in adults
 Total cost of OAB: $36.5B
CONFIDENTIAL 9
The Economic Impact of OAB is High
$3.0
$4.4
$8.1
$2.5
$14.7
$3.9
0 2 4 6 8 10 12 14 16
Lost Productivity
Consequence of Disease
Pad Use
Drug Costs
Outpatient Costs
Inpatient Costs
US Adult Population-based Cost of OAB ($B, 2009)
Onukwugha E, et al. Am J Manag Care 2009; 15, S90-97
Presented at ICS October 2015
 A significant unmet medical need exists in both NDO and iOAB
 Poor efficacy with orals, low satisfaction & adoption with Botox and neuromodulation
 TARIS expects to target ~33% of the currently-treated 3M patients
 TARIS system offers a compelling treatment option
 Better efficacy and safety, less invasive, potential to expand prescriber base
 Strong positive response to product profile in 2 formal qualitative surveys
 Ratings: 7/10 and 6/7
 Several development pathways exist to capture the broad OAB market
 Potential for expedited approach
 Potential peak revenue in the US >$500M
CONFIDENTIAL 10
Executive Summary: Key Takeaways
 The OAB market is a not a top drug-spend category for payers but is still a
significant pharmacy budget
 Payers are not actively monitoring pharmacy/medical budget impact or quality
metrics although new CMS metrics exist related to access to treatment for
patients with “urine leakage problems”
 3 and 4 Tier benefit designs are most common with payers forcing price
competition for preferred seat in each category; Branded agents require step
through generics
 TARIS will go through P&T and Technology Assessment but will not be managed
via pharmacy formulary structures; TARIS will require pre-certification and the
procedure will count against patients’ medical deductible… representing a
potential affordability challenge
 Payers believe this to be a “self-regulated” treatment option… only highly
motivated patients will seek this
CONFIDENTIAL 11
Payer Research: TARIS® in Line with Other Urology
Products
Model Input
Item / Indication NDO iOAB Comment
Total Treated Patients 1 million 2 million Pfizer, ICD9 codes
Target population (TP)
Addressable by specialist
SCI (38%), MS (36%), SB
(18%), Park & Alz (20%)
25%
Seeing specialists/ Failing orals
&
seeking 3rd line treatment
Growth pre-launch 1.84% 1.84%
Stewart W, et al. World J Urol
2003; 20 327-336
Additional Growth post-launch 3% 3% Marketing effort
Current price (2015 dollars) $2000 ($500 x4 per year) Total cost parity to Mirabegron
Price increase post launch 6% 6% Industry norm
Launch year 2021 2022 Assume 6 year to file NDA
Peak Market Share
20-25% (SCI/SB/MS)
5% (Parkinson’s/Alzh)
15%
Estimated from qualitative
market research, 5 year ramp
Output
PEAK YEAR SALES $205,000,000 $369,000,000 $574,000,000
# of patients treated at peak 81,000 174,000
12
Commercial Modal Assumptions: US Only
CONFIDENTIAL 13
OAB Revenue Share and Total Revenues By
Indication
MS
15%
Spina Bifida
2%
SCI
11%
Parkinson's
3%
Alzheimer's
4%
iOAB
65%
Total Revenue Split By Indication
MS Spina Bifida SCI Parkinson's Alzheimer's iOAB
$-
$100
$200
$300
$400
$500
$600
2021 2022 2023 2024 2025 2026 2027
Annual Sales (2027)
iOAB MS Spina Bifida SCI Parkinson's Alzheimer's
$22M
$90M
$173M
$276M
$401M
$529M
$574M
Checkpoint Inhibitors: Bladder Cancer
CONFIDENTIAL14
Checkpoint Inhibitors (Comparators): Bladder Cancer
Company Drug Indications MOA DOA
Annual
WAC
ORR Study
BMS
Opdivo
(Nivolumab)
Second line Pd-1 Feb, 2017 $130,931 19.6% Checkmate275
Genentech
Tecentriq
(Atezolizumab)
First Line; platinum
refractory cisplatin
ineligible
Pd-l1 May 2016 $150,000 23.5% IMVigor210
Merck
Keytruda
(Pembrolizumab)
Second line;
cisplatin ineligible
Pd-1 May 2017 $110,064 29% KEYNOTE045
Pfizer
Bavencio
(Avelumab)
Second line Pd-l1 March 2017 $156,000 33% JavlinMerkel200
Astrazeneca
Imfinzi
(Durvalumab)
Second line Pd-l1 May, 2017 $156,000 17% DANUBECONFIDENTIAL 15
Insert Source
Combination Therapy
CONFIDENTIAL
16
*Single dose immunotherapy for 70kg patient
**cost for complete dosage corresponds to median
number of doses. Wholesale Prices +-17%
Journal of Managed Care & Specialty Pharmacy
Vol 23, No 6
AWP per
dosage *
AWP per course
of treatment
(Base Case) **
Annual WAC Price per
QALY***
Nivolumab $5,732 $85,983 $130,931 $415,950
Ipilimumab $33,162 $132,649 $120,000 $129,000
Nivolumab+
Ipilimumab
$35,073 $140,292 $256,000 $454,092
***$100,000
WTP for 1
QOL
CheckMate 067 Trial: significant increase
in PFS Up to a 75% increase in PFS with
Combination
CONFIDENTIAL 17
Yervoy: 2.9
months
Opdivo: 6.9
months
Combination:
11.4 months
CPI’s: ~$16.55 B market value
 Forecast period: 2014- 2020
 Projected CAGR: 24.1%
 Shift from CTLA-4 submarket domination
in market share to PD-1 domination
 Biggest strides in melanoma and NSCLC
(Non Small Cell Lung Cancers)
CONFIDENTIAL 18
*
Checkpoint Inhibitors for Treating
Cancer: Market Report with Forecasts
2016-2026, Revealing Potentials of
CTLA-4 and PD-1 Inhibitor Drugs and
$16.50
$0.00
$2.00
$4.00
$6.00
$8.00
$10.00
$12.00
$14.00
$16.00
$18.00
INBILLIONS
Checkpoint Inhibitor Projected Value
2020 ($)
Checkpoint Inhibitor projected Value 2020
Costs of Check Point Inhibitors High
 Avg cost of Immuno-oncology drugs: $137,000 per year
19
$120,000.0
$110,064.0
$130,931.0
$150,000.0
$156,000.0
$165,000.0
0 20,000 40,000 60,000 80,000 100,000 120,000 140,000 160,000 180,000
Yervoy
Keytruda
Opdivo
Tecentriq
Imfinizi
Bavencio
Annual Wholesale Acquisition Cost for Checkpoint Inhibitors ( $)
AHIP High priced drugs estimated upon annual
patient expenditures
Leading Markets for Checkpoint
Inhibitors
1. US**
2. Japan
3. Germany, France, UK, Italy, and Spain
4. Overall EU market
5. APAC region
CONFIDENTIAL 20
Timeline Of Approvals2011
• Yervoy; malignant melanoma
2014
• Opdivo; malignant melanoma
• Keytruda; malignant melanoma
2015
• Opdivo: NSCLC
• Opdivo; RCC
• Keytruda; NSCLC
2016
• Opdivo: SCCHNN
• Opdivo: Hodgkins Lymphoma
• Opdivo: Malignant Melanoma
• Tecentriq: Bladder Cancer and NSCLC
2017
• Keytruda: Hodgkins Lymphoma
• Opdivo: Bladder Cancer
• Bavencio :Bladder Cancer; metastatic merkel cell
• Imfinzi: Bladder Cancer
• Opdivo and Yervoy: metastatic melanomaCONFIDENTIAL 21
Opdivo & Yervoy combo. yield better
endpoints overall than other CPI’s alone
2
8
25
12
10
7
45
25
22
12
40
85
80
25
0
10
20
30
40
50
60
70
80
90
Response Rate (%) Overall Survival (Months) 1 year survival Rate (%) 2 year survival rate (%)
Melanoma Trial Data
gp100 Yervoy (phase III data) Opdivo (Phase II data) Opdivo + Yervoy (phase 1 data) Keytruda (phase I data)CONFIDENTIAL 22
$83.00 $110.00
$159.00
$214.00
$249.00
$314.00
$356.00
$483.00
$584.00
$40.00
$162.00
$305.00
$475.00
$704.00
$840.00
$920.00
$1,310.00
$1,127.00
$325.00
$296.00
$240.00 $265.00 $263.00 $241.00
$285.00 $264.00
$330.00
$-
$200.00
$400.00
$600.00
$800.00
$1,000.00
$1,200.00
$1,400.00
1Q15 2Q15 3Q15 4Q15 1Q16 2Q16 3Q16 4Q16 1Q17
CPI Sales Quarterly Sales Growth (millions)
Keytruda Opdivo YervoyCONFIDENTIAL 23
CNBC
$83.00 $110.00 $159.00 $214.00 $249.00 $314.00 $356.00
$483.00
$584.00
$40.00
$162.00
$305.00
$475.00
$704.00
$840.00
$920.00
$1,310.00 $1,127.00
$325.00
$296.00
$240.00
$265.00
$263.00
$241.00
$285.00
$264.00
$330.00
$-
$500.00
$1,000.00
$1,500.00
$2,000.00
$2,500.00
1Q15 2Q15 3Q15 4Q15 1Q16 2Q16 3Q16 4Q16 1Q17
CPI Sales Quarterly Sales Growth (millions)
Keytruda Opdivo YervoyCONFIDENTIAL 24
Number of Anti PD-1/PD-L1 combination
studies 2015 vs 2017
70 73
42
26
268
242
123
83
KEYTRUDA OPDIVO IMFINZI TECENTRIQ
2015 2017
CONFIDENTIAL 25
Jacob & Edwin Elmhirst May
2017: Data from EP Vantage
PD-1 combo report 2015
Checkpoint Inhibitors penetrate Market Share for oncology drugs
55%
20%
19%
6%
2014
Cytotoxic Agents
26
9%
10%
4%
8%
68%
1%
2024
Cytoxic agents EGFR inhibitors
Angiogenesis Inhibitors ALK inhibitors
Immune Checkpoint Inhibitors BRAF/MEK inhibitors
Cancer
Immunotherapy:
Building on Initial
Successes to
Improve Clinical
Outcomes ***
Combinations of Opdivo + Yervoy
(BMS:75 Studies in Total)
28, 37%
10, 13%5, 7%
4, 5%
3, 4%
3, 4%
2, 3%
2, 3%
2, 3%
16, 21%
Melanoma NSCLC Renal Cancer Head and Neck Cancer Prostate
Mesothelloma Breast Cancer SCLC Uveal Melanoma Other indications27
Evaluate Ltd May 2017
Annual WAC vs Price per QALY
avg Price per QALY: $250,155 > >WTP QALY:
$100,000
120,000
130,931
110,064
150,000
129,000
415,950
236,492
219,179
Yervoy Opdivo Keytruda Tecentriq
0
50,000
100,000
150,000
200,000
250,000
300,000
350,000
400,000
450,000
Annual WAC Price per QALY WTP per QALY 28
Checkpoint Inhibitors (Comparators)
Company Drug Indications MOA DOA
Annual
WAC
ORR
BMS
Opdivo
(Nivolumab)
Second line
Metastatic
Melanoma
Pd-1 2015 $130,931 32%
Genentech
Tecentriq
(Atezolizumab)
First line
treatment of
Bladder Cancer
Pd-l1 2016 $150,000 14.8%
BMS
Yervoy
(Ipilimumab)
Second line
Metastatic
Melanoma
CTLA-4 2011 $120,0000 10.9%
Merck
Keytruda
(Pembrolizumab)
Second line
NSCLC,HNSCC,
CHL, Bladder
Cancer,
Metastatic
Melanoma, MSI-H
Pd-1 2014 $110,064 19%;69%
Pfizer
Bavencio
(Avelumab)
Second line
Bladder Cancer,
First line
Metastatic Merkel
Cell Carcinoma
Pd-l1 2017 $156,000 16%
Astrazeneca
Imfinzi
Bladder Cancer
(second line Pd-l1 2017 $156,000 26%
CONFIDENTIAL 29

More Related Content

What's hot

Contract research organization services (cr os) market
Contract research organization services (cr os) marketContract research organization services (cr os) market
Contract research organization services (cr os) market
ameliasimon0
 
Is European market access becoming an incubator for rare disease development?...
Is European market access becoming an incubator for rare disease development?...Is European market access becoming an incubator for rare disease development?...
Is European market access becoming an incubator for rare disease development?...
KateBenson18
 
IVD Market Size and Growth Trend
IVD Market Size and Growth TrendIVD Market Size and Growth Trend
IVD Market Size and Growth Trend
Bruce Carlson
 
Strand genomics features in CIO review
Strand genomics features in CIO reviewStrand genomics features in CIO review
Strand genomics features in CIO review
Strand Life Sciences Pvt Ltd
 
applied strategic 10 years vision for biosimilars
applied strategic 10 years vision for biosimilarsapplied strategic 10 years vision for biosimilars
applied strategic 10 years vision for biosimilars
Richard Littlewood
 
Healthcare Contract Research Organization (CRO) Market
Healthcare Contract Research Organization (CRO) MarketHealthcare Contract Research Organization (CRO) Market
Healthcare Contract Research Organization (CRO) Market
Tom Clark
 
Nanopa4
Nanopa4Nanopa4
Nanopa4gana9
 
18th pharmacovigilance 2019
18th pharmacovigilance 201918th pharmacovigilance 2019
18th pharmacovigilance 2019
Dinesh T
 
Top Trends in Orphan Drugs
Top Trends in Orphan DrugsTop Trends in Orphan Drugs
Top Trends in Orphan Drugs
Bill Smith
 
Genentech
GenentechGenentech
Gene Expression Analysis Market PPT: Growth, Outlook, Demand, Keyplayer Anal...
Gene Expression Analysis  Market PPT: Growth, Outlook, Demand, Keyplayer Anal...Gene Expression Analysis  Market PPT: Growth, Outlook, Demand, Keyplayer Anal...
Gene Expression Analysis Market PPT: Growth, Outlook, Demand, Keyplayer Anal...
IMARC Group
 
Movers & shakers interview with vivek trikha head diagnostics onc quest ...
Movers & shakers interview with vivek trikha head  diagnostics onc quest ...Movers & shakers interview with vivek trikha head  diagnostics onc quest ...
Movers & shakers interview with vivek trikha head diagnostics onc quest ...
oncquest
 
Commercial considerations in early drug development
Commercial considerations in early drug developmentCommercial considerations in early drug development
Commercial considerations in early drug development
Sunil Ramkali
 
The U.S. Biopharmaceutical Industry (2014): Perspectives on Future Growth and...
The U.S. Biopharmaceutical Industry (2014): Perspectives on Future Growth and...The U.S. Biopharmaceutical Industry (2014): Perspectives on Future Growth and...
The U.S. Biopharmaceutical Industry (2014): Perspectives on Future Growth and...
PhRMA
 
Orphan drugs: why the hype?
Orphan drugs: why the hype?Orphan drugs: why the hype?
Orphan drugs: why the hype?
Peder S. Andersen
 
Biosimilar Development EPTM 2015
Biosimilar Development EPTM 2015Biosimilar Development EPTM 2015
Biosimilar Development EPTM 2015
Ajaz Hussain
 
Aiden hollis hif presentation berkeley
Aiden hollis   hif presentation berkeleyAiden hollis   hif presentation berkeley
Aiden hollis hif presentation berkeleyOpen Science Summit
 

What's hot (20)

Contract research organization services (cr os) market
Contract research organization services (cr os) marketContract research organization services (cr os) market
Contract research organization services (cr os) market
 
Is European market access becoming an incubator for rare disease development?...
Is European market access becoming an incubator for rare disease development?...Is European market access becoming an incubator for rare disease development?...
Is European market access becoming an incubator for rare disease development?...
 
IVD Market Size and Growth Trend
IVD Market Size and Growth TrendIVD Market Size and Growth Trend
IVD Market Size and Growth Trend
 
Strand genomics features in CIO review
Strand genomics features in CIO reviewStrand genomics features in CIO review
Strand genomics features in CIO review
 
applied strategic 10 years vision for biosimilars
applied strategic 10 years vision for biosimilarsapplied strategic 10 years vision for biosimilars
applied strategic 10 years vision for biosimilars
 
Healthcare Contract Research Organization (CRO) Market
Healthcare Contract Research Organization (CRO) MarketHealthcare Contract Research Organization (CRO) Market
Healthcare Contract Research Organization (CRO) Market
 
Nanopa4
Nanopa4Nanopa4
Nanopa4
 
18th pharmacovigilance 2019
18th pharmacovigilance 201918th pharmacovigilance 2019
18th pharmacovigilance 2019
 
Claire Hulme
Claire HulmeClaire Hulme
Claire Hulme
 
Top Trends in Orphan Drugs
Top Trends in Orphan DrugsTop Trends in Orphan Drugs
Top Trends in Orphan Drugs
 
Genentech
GenentechGenentech
Genentech
 
Gene Expression Analysis Market PPT: Growth, Outlook, Demand, Keyplayer Anal...
Gene Expression Analysis  Market PPT: Growth, Outlook, Demand, Keyplayer Anal...Gene Expression Analysis  Market PPT: Growth, Outlook, Demand, Keyplayer Anal...
Gene Expression Analysis Market PPT: Growth, Outlook, Demand, Keyplayer Anal...
 
Movers & shakers interview with vivek trikha head diagnostics onc quest ...
Movers & shakers interview with vivek trikha head  diagnostics onc quest ...Movers & shakers interview with vivek trikha head  diagnostics onc quest ...
Movers & shakers interview with vivek trikha head diagnostics onc quest ...
 
Commercial considerations in early drug development
Commercial considerations in early drug developmentCommercial considerations in early drug development
Commercial considerations in early drug development
 
Novartis Oncology Strategy
Novartis Oncology StrategyNovartis Oncology Strategy
Novartis Oncology Strategy
 
durgesh final
durgesh finaldurgesh final
durgesh final
 
The U.S. Biopharmaceutical Industry (2014): Perspectives on Future Growth and...
The U.S. Biopharmaceutical Industry (2014): Perspectives on Future Growth and...The U.S. Biopharmaceutical Industry (2014): Perspectives on Future Growth and...
The U.S. Biopharmaceutical Industry (2014): Perspectives on Future Growth and...
 
Orphan drugs: why the hype?
Orphan drugs: why the hype?Orphan drugs: why the hype?
Orphan drugs: why the hype?
 
Biosimilar Development EPTM 2015
Biosimilar Development EPTM 2015Biosimilar Development EPTM 2015
Biosimilar Development EPTM 2015
 
Aiden hollis hif presentation berkeley
Aiden hollis   hif presentation berkeleyAiden hollis   hif presentation berkeley
Aiden hollis hif presentation berkeley
 

Similar to Marketplace positioning and preliminary Market forecast

Outlook 2011 presentation
Outlook 2011 presentationOutlook 2011 presentation
Outlook 2011 presentationmpadvisor
 
2017 01 04 corporate presentation
2017 01 04 corporate presentation2017 01 04 corporate presentation
2017 01 04 corporate presentationKristine Mechem
 
Future Challenges of Clinical Development; a View from the CRO - Hani Zaki
Future Challenges of Clinical Development; a View from the CRO - Hani ZakiFuture Challenges of Clinical Development; a View from the CRO - Hani Zaki
Future Challenges of Clinical Development; a View from the CRO - Hani ZakiTTC, llc
 
Future Challenges of Clinical Development; a View from the CRO - Hani Zaki
Future Challenges of Clinical Development; a View from the CRO - Hani ZakiFuture Challenges of Clinical Development; a View from the CRO - Hani Zaki
Future Challenges of Clinical Development; a View from the CRO - Hani ZakiTTC, llc
 
Liquid biopsy market
Liquid biopsy marketLiquid biopsy market
Liquid biopsy market
danishsmith01
 
Sponsor-CRO Relationships
Sponsor-CRO RelationshipsSponsor-CRO Relationships
Sponsor-CRO RelationshipsDavid Selkirk
 
Interpace Diagnostics Investor Presentation
Interpace Diagnostics Investor PresentationInterpace Diagnostics Investor Presentation
Interpace Diagnostics Investor Presentation
RedChip Companies, Inc.
 
Clinical Recruitment Planning Strategies
Clinical Recruitment Planning StrategiesClinical Recruitment Planning Strategies
Clinical Recruitment Planning Strategies
MFinch3777
 
LTRN Investor Presentation - October 2021
LTRN Investor Presentation - October 2021LTRN Investor Presentation - October 2021
LTRN Investor Presentation - October 2021
RedChip Companies, Inc.
 
F5 Outcomes based contracting 040416 ARSmm
F5 Outcomes based contracting 040416 ARSmmF5 Outcomes based contracting 040416 ARSmm
F5 Outcomes based contracting 040416 ARSmmNathan White, CPC
 
New Product Planning in the Pharmaceutical Industry
New Product Planning in the Pharmaceutical IndustryNew Product Planning in the Pharmaceutical Industry
New Product Planning in the Pharmaceutical Industry
Anthony Russell
 
Pharma industry: present landscape and main trends
Pharma industry: present landscape and main trendsPharma industry: present landscape and main trends
Pharma industry: present landscape and main trends
Ignacio Riesgo
 
Can-Fite BioPharma - Investor Presentation January 2021
Can-Fite BioPharma - Investor Presentation January 2021Can-Fite BioPharma - Investor Presentation January 2021
Can-Fite BioPharma - Investor Presentation January 2021
RedChip Companies, Inc.
 
Peptide Therapeutics Market, 2021-2030 (Free Insights).pptx
Peptide Therapeutics Market, 2021-2030 (Free Insights).pptxPeptide Therapeutics Market, 2021-2030 (Free Insights).pptx
Peptide Therapeutics Market, 2021-2030 (Free Insights).pptx
Jamesmathur
 
Can Fite Investor Presentation Dec 2022
Can Fite Investor Presentation Dec 2022Can Fite Investor Presentation Dec 2022
Can Fite Investor Presentation Dec 2022
RedChip Companies, Inc.
 
D2M Final Presentation Submission for LinkedIn
D2M Final Presentation Submission for LinkedInD2M Final Presentation Submission for LinkedIn
D2M Final Presentation Submission for LinkedInGregory Petrossian
 
Global pharmaceutical & biotechnology outlook 2013 rising stars - Reports Corner
Global pharmaceutical & biotechnology outlook 2013 rising stars - Reports CornerGlobal pharmaceutical & biotechnology outlook 2013 rising stars - Reports Corner
Global pharmaceutical & biotechnology outlook 2013 rising stars - Reports Corner
Reports Corner
 
New Health Report 2012 - Media Briefing Deck
New Health Report 2012 - Media Briefing Deck New Health Report 2012 - Media Briefing Deck
New Health Report 2012 - Media Briefing Deck Quintiles
 

Similar to Marketplace positioning and preliminary Market forecast (20)

Korea entry strategy (biocon)
Korea entry strategy (biocon)Korea entry strategy (biocon)
Korea entry strategy (biocon)
 
Korea entry strategy (biocon) Hypothetical
Korea entry strategy (biocon) HypotheticalKorea entry strategy (biocon) Hypothetical
Korea entry strategy (biocon) Hypothetical
 
Outlook 2011 presentation
Outlook 2011 presentationOutlook 2011 presentation
Outlook 2011 presentation
 
2017 01 04 corporate presentation
2017 01 04 corporate presentation2017 01 04 corporate presentation
2017 01 04 corporate presentation
 
Future Challenges of Clinical Development; a View from the CRO - Hani Zaki
Future Challenges of Clinical Development; a View from the CRO - Hani ZakiFuture Challenges of Clinical Development; a View from the CRO - Hani Zaki
Future Challenges of Clinical Development; a View from the CRO - Hani Zaki
 
Future Challenges of Clinical Development; a View from the CRO - Hani Zaki
Future Challenges of Clinical Development; a View from the CRO - Hani ZakiFuture Challenges of Clinical Development; a View from the CRO - Hani Zaki
Future Challenges of Clinical Development; a View from the CRO - Hani Zaki
 
Liquid biopsy market
Liquid biopsy marketLiquid biopsy market
Liquid biopsy market
 
Sponsor-CRO Relationships
Sponsor-CRO RelationshipsSponsor-CRO Relationships
Sponsor-CRO Relationships
 
Interpace Diagnostics Investor Presentation
Interpace Diagnostics Investor PresentationInterpace Diagnostics Investor Presentation
Interpace Diagnostics Investor Presentation
 
Clinical Recruitment Planning Strategies
Clinical Recruitment Planning StrategiesClinical Recruitment Planning Strategies
Clinical Recruitment Planning Strategies
 
LTRN Investor Presentation - October 2021
LTRN Investor Presentation - October 2021LTRN Investor Presentation - October 2021
LTRN Investor Presentation - October 2021
 
F5 Outcomes based contracting 040416 ARSmm
F5 Outcomes based contracting 040416 ARSmmF5 Outcomes based contracting 040416 ARSmm
F5 Outcomes based contracting 040416 ARSmm
 
New Product Planning in the Pharmaceutical Industry
New Product Planning in the Pharmaceutical IndustryNew Product Planning in the Pharmaceutical Industry
New Product Planning in the Pharmaceutical Industry
 
Pharma industry: present landscape and main trends
Pharma industry: present landscape and main trendsPharma industry: present landscape and main trends
Pharma industry: present landscape and main trends
 
Can-Fite BioPharma - Investor Presentation January 2021
Can-Fite BioPharma - Investor Presentation January 2021Can-Fite BioPharma - Investor Presentation January 2021
Can-Fite BioPharma - Investor Presentation January 2021
 
Peptide Therapeutics Market, 2021-2030 (Free Insights).pptx
Peptide Therapeutics Market, 2021-2030 (Free Insights).pptxPeptide Therapeutics Market, 2021-2030 (Free Insights).pptx
Peptide Therapeutics Market, 2021-2030 (Free Insights).pptx
 
Can Fite Investor Presentation Dec 2022
Can Fite Investor Presentation Dec 2022Can Fite Investor Presentation Dec 2022
Can Fite Investor Presentation Dec 2022
 
D2M Final Presentation Submission for LinkedIn
D2M Final Presentation Submission for LinkedInD2M Final Presentation Submission for LinkedIn
D2M Final Presentation Submission for LinkedIn
 
Global pharmaceutical & biotechnology outlook 2013 rising stars - Reports Corner
Global pharmaceutical & biotechnology outlook 2013 rising stars - Reports CornerGlobal pharmaceutical & biotechnology outlook 2013 rising stars - Reports Corner
Global pharmaceutical & biotechnology outlook 2013 rising stars - Reports Corner
 
New Health Report 2012 - Media Briefing Deck
New Health Report 2012 - Media Briefing Deck New Health Report 2012 - Media Briefing Deck
New Health Report 2012 - Media Briefing Deck
 

Recently uploaded

Unveiling the Secrets How Does Generative AI Work.pdf
Unveiling the Secrets How Does Generative AI Work.pdfUnveiling the Secrets How Does Generative AI Work.pdf
Unveiling the Secrets How Does Generative AI Work.pdf
Sam H
 
Cree_Rey_BrandIdentityKit.PDF_PersonalBd
Cree_Rey_BrandIdentityKit.PDF_PersonalBdCree_Rey_BrandIdentityKit.PDF_PersonalBd
Cree_Rey_BrandIdentityKit.PDF_PersonalBd
creerey
 
Enterprise Excellence is Inclusive Excellence.pdf
Enterprise Excellence is Inclusive Excellence.pdfEnterprise Excellence is Inclusive Excellence.pdf
Enterprise Excellence is Inclusive Excellence.pdf
KaiNexus
 
Affordable Stationery Printing Services in Jaipur | Navpack n Print
Affordable Stationery Printing Services in Jaipur | Navpack n PrintAffordable Stationery Printing Services in Jaipur | Navpack n Print
Affordable Stationery Printing Services in Jaipur | Navpack n Print
Navpack & Print
 
The-McKinsey-7S-Framework. strategic management
The-McKinsey-7S-Framework. strategic managementThe-McKinsey-7S-Framework. strategic management
The-McKinsey-7S-Framework. strategic management
Bojamma2
 
RMD24 | Retail media: hoe zet je dit in als je geen AH of Unilever bent? Heid...
RMD24 | Retail media: hoe zet je dit in als je geen AH of Unilever bent? Heid...RMD24 | Retail media: hoe zet je dit in als je geen AH of Unilever bent? Heid...
RMD24 | Retail media: hoe zet je dit in als je geen AH of Unilever bent? Heid...
BBPMedia1
 
5 Things You Need To Know Before Hiring a Videographer
5 Things You Need To Know Before Hiring a Videographer5 Things You Need To Know Before Hiring a Videographer
5 Things You Need To Know Before Hiring a Videographer
ofm712785
 
What is the TDS Return Filing Due Date for FY 2024-25.pdf
What is the TDS Return Filing Due Date for FY 2024-25.pdfWhat is the TDS Return Filing Due Date for FY 2024-25.pdf
What is the TDS Return Filing Due Date for FY 2024-25.pdf
seoforlegalpillers
 
Premium MEAN Stack Development Solutions for Modern Businesses
Premium MEAN Stack Development Solutions for Modern BusinessesPremium MEAN Stack Development Solutions for Modern Businesses
Premium MEAN Stack Development Solutions for Modern Businesses
SynapseIndia
 
Discover the innovative and creative projects that highlight my journey throu...
Discover the innovative and creative projects that highlight my journey throu...Discover the innovative and creative projects that highlight my journey throu...
Discover the innovative and creative projects that highlight my journey throu...
dylandmeas
 
FINAL PRESENTATION.pptx12143241324134134
FINAL PRESENTATION.pptx12143241324134134FINAL PRESENTATION.pptx12143241324134134
FINAL PRESENTATION.pptx12143241324134134
LR1709MUSIC
 
Skye Residences | Extended Stay Residences Near Toronto Airport
Skye Residences | Extended Stay Residences Near Toronto AirportSkye Residences | Extended Stay Residences Near Toronto Airport
Skye Residences | Extended Stay Residences Near Toronto Airport
marketingjdass
 
ModelingMarketingStrategiesMKS.CollumbiaUniversitypdf
ModelingMarketingStrategiesMKS.CollumbiaUniversitypdfModelingMarketingStrategiesMKS.CollumbiaUniversitypdf
ModelingMarketingStrategiesMKS.CollumbiaUniversitypdf
fisherameliaisabella
 
Memorandum Of Association Constitution of Company.ppt
Memorandum Of Association Constitution of Company.pptMemorandum Of Association Constitution of Company.ppt
Memorandum Of Association Constitution of Company.ppt
seri bangash
 
The Parable of the Pipeline a book every new businessman or business student ...
The Parable of the Pipeline a book every new businessman or business student ...The Parable of the Pipeline a book every new businessman or business student ...
The Parable of the Pipeline a book every new businessman or business student ...
awaisafdar
 
Sustainability: Balancing the Environment, Equity & Economy
Sustainability: Balancing the Environment, Equity & EconomySustainability: Balancing the Environment, Equity & Economy
Sustainability: Balancing the Environment, Equity & Economy
Operational Excellence Consulting
 
Cracking the Workplace Discipline Code Main.pptx
Cracking the Workplace Discipline Code Main.pptxCracking the Workplace Discipline Code Main.pptx
Cracking the Workplace Discipline Code Main.pptx
Workforce Group
 
Introduction to Amazon company 111111111111
Introduction to Amazon company 111111111111Introduction to Amazon company 111111111111
Introduction to Amazon company 111111111111
zoyaansari11365
 
April 2024 Nostalgia Products Newsletter
April 2024 Nostalgia Products NewsletterApril 2024 Nostalgia Products Newsletter
April 2024 Nostalgia Products Newsletter
NathanBaughman3
 
Meas_Dylan_DMBS_PB1_2024-05XX_Revised.pdf
Meas_Dylan_DMBS_PB1_2024-05XX_Revised.pdfMeas_Dylan_DMBS_PB1_2024-05XX_Revised.pdf
Meas_Dylan_DMBS_PB1_2024-05XX_Revised.pdf
dylandmeas
 

Recently uploaded (20)

Unveiling the Secrets How Does Generative AI Work.pdf
Unveiling the Secrets How Does Generative AI Work.pdfUnveiling the Secrets How Does Generative AI Work.pdf
Unveiling the Secrets How Does Generative AI Work.pdf
 
Cree_Rey_BrandIdentityKit.PDF_PersonalBd
Cree_Rey_BrandIdentityKit.PDF_PersonalBdCree_Rey_BrandIdentityKit.PDF_PersonalBd
Cree_Rey_BrandIdentityKit.PDF_PersonalBd
 
Enterprise Excellence is Inclusive Excellence.pdf
Enterprise Excellence is Inclusive Excellence.pdfEnterprise Excellence is Inclusive Excellence.pdf
Enterprise Excellence is Inclusive Excellence.pdf
 
Affordable Stationery Printing Services in Jaipur | Navpack n Print
Affordable Stationery Printing Services in Jaipur | Navpack n PrintAffordable Stationery Printing Services in Jaipur | Navpack n Print
Affordable Stationery Printing Services in Jaipur | Navpack n Print
 
The-McKinsey-7S-Framework. strategic management
The-McKinsey-7S-Framework. strategic managementThe-McKinsey-7S-Framework. strategic management
The-McKinsey-7S-Framework. strategic management
 
RMD24 | Retail media: hoe zet je dit in als je geen AH of Unilever bent? Heid...
RMD24 | Retail media: hoe zet je dit in als je geen AH of Unilever bent? Heid...RMD24 | Retail media: hoe zet je dit in als je geen AH of Unilever bent? Heid...
RMD24 | Retail media: hoe zet je dit in als je geen AH of Unilever bent? Heid...
 
5 Things You Need To Know Before Hiring a Videographer
5 Things You Need To Know Before Hiring a Videographer5 Things You Need To Know Before Hiring a Videographer
5 Things You Need To Know Before Hiring a Videographer
 
What is the TDS Return Filing Due Date for FY 2024-25.pdf
What is the TDS Return Filing Due Date for FY 2024-25.pdfWhat is the TDS Return Filing Due Date for FY 2024-25.pdf
What is the TDS Return Filing Due Date for FY 2024-25.pdf
 
Premium MEAN Stack Development Solutions for Modern Businesses
Premium MEAN Stack Development Solutions for Modern BusinessesPremium MEAN Stack Development Solutions for Modern Businesses
Premium MEAN Stack Development Solutions for Modern Businesses
 
Discover the innovative and creative projects that highlight my journey throu...
Discover the innovative and creative projects that highlight my journey throu...Discover the innovative and creative projects that highlight my journey throu...
Discover the innovative and creative projects that highlight my journey throu...
 
FINAL PRESENTATION.pptx12143241324134134
FINAL PRESENTATION.pptx12143241324134134FINAL PRESENTATION.pptx12143241324134134
FINAL PRESENTATION.pptx12143241324134134
 
Skye Residences | Extended Stay Residences Near Toronto Airport
Skye Residences | Extended Stay Residences Near Toronto AirportSkye Residences | Extended Stay Residences Near Toronto Airport
Skye Residences | Extended Stay Residences Near Toronto Airport
 
ModelingMarketingStrategiesMKS.CollumbiaUniversitypdf
ModelingMarketingStrategiesMKS.CollumbiaUniversitypdfModelingMarketingStrategiesMKS.CollumbiaUniversitypdf
ModelingMarketingStrategiesMKS.CollumbiaUniversitypdf
 
Memorandum Of Association Constitution of Company.ppt
Memorandum Of Association Constitution of Company.pptMemorandum Of Association Constitution of Company.ppt
Memorandum Of Association Constitution of Company.ppt
 
The Parable of the Pipeline a book every new businessman or business student ...
The Parable of the Pipeline a book every new businessman or business student ...The Parable of the Pipeline a book every new businessman or business student ...
The Parable of the Pipeline a book every new businessman or business student ...
 
Sustainability: Balancing the Environment, Equity & Economy
Sustainability: Balancing the Environment, Equity & EconomySustainability: Balancing the Environment, Equity & Economy
Sustainability: Balancing the Environment, Equity & Economy
 
Cracking the Workplace Discipline Code Main.pptx
Cracking the Workplace Discipline Code Main.pptxCracking the Workplace Discipline Code Main.pptx
Cracking the Workplace Discipline Code Main.pptx
 
Introduction to Amazon company 111111111111
Introduction to Amazon company 111111111111Introduction to Amazon company 111111111111
Introduction to Amazon company 111111111111
 
April 2024 Nostalgia Products Newsletter
April 2024 Nostalgia Products NewsletterApril 2024 Nostalgia Products Newsletter
April 2024 Nostalgia Products Newsletter
 
Meas_Dylan_DMBS_PB1_2024-05XX_Revised.pdf
Meas_Dylan_DMBS_PB1_2024-05XX_Revised.pdfMeas_Dylan_DMBS_PB1_2024-05XX_Revised.pdf
Meas_Dylan_DMBS_PB1_2024-05XX_Revised.pdf
 

Marketplace positioning and preliminary Market forecast

  • 1. TARIS® Market Research: Market Positioning, Pricing, Sales Forecast Preliminary Draft Jody Kalt, Terry York, Gabi Forman Investor Deck part III CONFIDENTIAL 1
  • 2.  Bladder cancer represents a large and underserved market opportunity that has been devoid of innovation for many years  Level of interest/development activity have increased dramatically in the past several years  Driven by a better understanding of immune mechanisms at play both systemically and in the tumor micro-environment  Bladder cancer is complex - represents a number of different patient cohorts with differing unmet needs  Since the AGN transaction in 2014, TARIS has spent a majority of our time focused on understanding the nuances of treating this disease and advancing our lead program into clinical proof-of-concept studies, which are now just starting to readout CONFIDENTIAL 2 Executive Summary: 1 of 2
  • 3.  Our thinking has evolved in several different directions:  Target: Initially only NMIBC, now both NMIBC and MIBC, with the later being higher priority  MOA: Initially improved cytotoxic modality due to PK play, now potential multimodal activity  Our initial proof-of-principle study in MIBC has validated our belief that our approach has the opportunity to transform the treatment of this disease  TARIS just completed a significant market research study working with Defined Health to try and better understand the commercial opportunity for this technology  We believe, if successful in development, that this represents a $1B+ opportunity (US) with several opportunities to pursue a rapid registration pathway CONFIDENTIAL 3 Executive Summary: 2 of 2
  • 4. Incidence and Prevalence Bladder Cancer CONFIDENTIAL4
  • 5. 5 Bladder Cancer Treated Patients, 2016 (US Only) Target for Most Current I/O Therapies Managed By: Uro-oncologists Managed By: Uro-oncologists Medical Oncologists Radiation Oncologists Managed By: Medical Oncologists Radiation Oncologists All Bladder Cancer 102,546 pts NMIBC 81,725 MIBC 16,855 Metastatic 3,966
  • 6.  TARIS has completed substantial market research in bladder cancer with DefinedHealth  First project: 2012, focused on profiles in NMIBC  Recent project: 2016, focused on profiles in both NMIBC and MIBC  Summary of scope: current project  24 qualitative interviews:  8 KOL uro-oncologists  16 additional interviews: community urologists, medical oncologists, radiation oncologists  60 quantitative interviews via web-based survey  5 payer interviews (Medical/Pharmacy Directors)  Initially tested 4 product profiles in qualitative interviews, after completion of the initial interviews, added a 5th TPP with an additional 4 qualitative interviews, prioritized 3 to include in quant  TPPs numbered by severity of disease (low to high), not necessarily by internal development priority  TARIS did not evaluate TPPs for low risk NMIBC or treatment-naïve high risk NMIBC  Included in previous 2012 research  These indications represent additional potential upside CONFIDENTIAL 6 TARS® Market Research Methodology
  • 7.  All specialties generally reacted favorably to the TAR-200 product profile as an easily deployed and retracted device in the office setting for local, sustained delivery of drugs to the bladder  However, they wanted to be assured that TAR-200 would be tolerable and not interfere with follow-up:  Foreign body placement location and rubbing could cause inflammation to the bladder lining  Continuous delivery of gemcitabine could irritate the bladder  Either could cause pain and/or increase urinary urge and voiding frequency, dysuria ♦ Physicians generally understood advantage of longer duration tx (7 days vs. 1 hr of BCG/chemo), support the selection of gemcitabine as agent of choice  However, physicians want to see clinical data on how week-long, sustained, low dose gemcitabine is better than short exposures of multiple high dose instillations in terms of improvements in efficacy/outcomes  Urologists: having an intravesical product pre-loaded with gemcitabine on the shelf would increase patient access and physician/office convenience  Community urologists wanted to know if it is economical for a Urology office – what’s the profit margin?  Community radiologist considered TAR-200 as a good option for older, frail (> 80+ yrs in age are not good candidates for surgery or Chemo as kidney function is not good) that are left with radiation only Physicians Reacted Quite Positively to The TAR-200 Product Profile  Source: 1. DH Primary Research
  • 8. CONFIDENTIAL 8 MIBC: ~$2.1B Total Addressable Peak US Market  Significant market research, including payors, complete  At least 2x potential w/ EU, Japan, ROW  US represents only 17% of WW cases  Additional upside indications, not included in forecast  High risk/metastatic disease (± checkpoint)  High Risk NMIBC: BCG Naïve, BCG Unresponsive $2,069 $0 $500 $1,000 $1,500 $2,000 $2,500 MIBC Millions Total Addressable Market US MIBC Markets at Peak Patients Cost/Dose Doses/Patient Cost/Patient Total Addressable Market RC Ineligible 7,285 $14k 9 (18 months) $126k $972M NAC Ineligible 5,562 2 (upfront) $28k $165M cT2 Bladder Sparing 6,246 11 (2 years) $156k $932M*
  • 9.  EpiLUTS: US population-based survey in adults  Total cost of OAB: $36.5B CONFIDENTIAL 9 The Economic Impact of OAB is High $3.0 $4.4 $8.1 $2.5 $14.7 $3.9 0 2 4 6 8 10 12 14 16 Lost Productivity Consequence of Disease Pad Use Drug Costs Outpatient Costs Inpatient Costs US Adult Population-based Cost of OAB ($B, 2009) Onukwugha E, et al. Am J Manag Care 2009; 15, S90-97 Presented at ICS October 2015
  • 10.  A significant unmet medical need exists in both NDO and iOAB  Poor efficacy with orals, low satisfaction & adoption with Botox and neuromodulation  TARIS expects to target ~33% of the currently-treated 3M patients  TARIS system offers a compelling treatment option  Better efficacy and safety, less invasive, potential to expand prescriber base  Strong positive response to product profile in 2 formal qualitative surveys  Ratings: 7/10 and 6/7  Several development pathways exist to capture the broad OAB market  Potential for expedited approach  Potential peak revenue in the US >$500M CONFIDENTIAL 10 Executive Summary: Key Takeaways
  • 11.  The OAB market is a not a top drug-spend category for payers but is still a significant pharmacy budget  Payers are not actively monitoring pharmacy/medical budget impact or quality metrics although new CMS metrics exist related to access to treatment for patients with “urine leakage problems”  3 and 4 Tier benefit designs are most common with payers forcing price competition for preferred seat in each category; Branded agents require step through generics  TARIS will go through P&T and Technology Assessment but will not be managed via pharmacy formulary structures; TARIS will require pre-certification and the procedure will count against patients’ medical deductible… representing a potential affordability challenge  Payers believe this to be a “self-regulated” treatment option… only highly motivated patients will seek this CONFIDENTIAL 11 Payer Research: TARIS® in Line with Other Urology Products
  • 12. Model Input Item / Indication NDO iOAB Comment Total Treated Patients 1 million 2 million Pfizer, ICD9 codes Target population (TP) Addressable by specialist SCI (38%), MS (36%), SB (18%), Park & Alz (20%) 25% Seeing specialists/ Failing orals & seeking 3rd line treatment Growth pre-launch 1.84% 1.84% Stewart W, et al. World J Urol 2003; 20 327-336 Additional Growth post-launch 3% 3% Marketing effort Current price (2015 dollars) $2000 ($500 x4 per year) Total cost parity to Mirabegron Price increase post launch 6% 6% Industry norm Launch year 2021 2022 Assume 6 year to file NDA Peak Market Share 20-25% (SCI/SB/MS) 5% (Parkinson’s/Alzh) 15% Estimated from qualitative market research, 5 year ramp Output PEAK YEAR SALES $205,000,000 $369,000,000 $574,000,000 # of patients treated at peak 81,000 174,000 12 Commercial Modal Assumptions: US Only
  • 13. CONFIDENTIAL 13 OAB Revenue Share and Total Revenues By Indication MS 15% Spina Bifida 2% SCI 11% Parkinson's 3% Alzheimer's 4% iOAB 65% Total Revenue Split By Indication MS Spina Bifida SCI Parkinson's Alzheimer's iOAB $- $100 $200 $300 $400 $500 $600 2021 2022 2023 2024 2025 2026 2027 Annual Sales (2027) iOAB MS Spina Bifida SCI Parkinson's Alzheimer's $22M $90M $173M $276M $401M $529M $574M
  • 14. Checkpoint Inhibitors: Bladder Cancer CONFIDENTIAL14
  • 15. Checkpoint Inhibitors (Comparators): Bladder Cancer Company Drug Indications MOA DOA Annual WAC ORR Study BMS Opdivo (Nivolumab) Second line Pd-1 Feb, 2017 $130,931 19.6% Checkmate275 Genentech Tecentriq (Atezolizumab) First Line; platinum refractory cisplatin ineligible Pd-l1 May 2016 $150,000 23.5% IMVigor210 Merck Keytruda (Pembrolizumab) Second line; cisplatin ineligible Pd-1 May 2017 $110,064 29% KEYNOTE045 Pfizer Bavencio (Avelumab) Second line Pd-l1 March 2017 $156,000 33% JavlinMerkel200 Astrazeneca Imfinzi (Durvalumab) Second line Pd-l1 May, 2017 $156,000 17% DANUBECONFIDENTIAL 15 Insert Source
  • 16. Combination Therapy CONFIDENTIAL 16 *Single dose immunotherapy for 70kg patient **cost for complete dosage corresponds to median number of doses. Wholesale Prices +-17% Journal of Managed Care & Specialty Pharmacy Vol 23, No 6 AWP per dosage * AWP per course of treatment (Base Case) ** Annual WAC Price per QALY*** Nivolumab $5,732 $85,983 $130,931 $415,950 Ipilimumab $33,162 $132,649 $120,000 $129,000 Nivolumab+ Ipilimumab $35,073 $140,292 $256,000 $454,092 ***$100,000 WTP for 1 QOL
  • 17. CheckMate 067 Trial: significant increase in PFS Up to a 75% increase in PFS with Combination CONFIDENTIAL 17 Yervoy: 2.9 months Opdivo: 6.9 months Combination: 11.4 months
  • 18. CPI’s: ~$16.55 B market value  Forecast period: 2014- 2020  Projected CAGR: 24.1%  Shift from CTLA-4 submarket domination in market share to PD-1 domination  Biggest strides in melanoma and NSCLC (Non Small Cell Lung Cancers) CONFIDENTIAL 18 * Checkpoint Inhibitors for Treating Cancer: Market Report with Forecasts 2016-2026, Revealing Potentials of CTLA-4 and PD-1 Inhibitor Drugs and $16.50 $0.00 $2.00 $4.00 $6.00 $8.00 $10.00 $12.00 $14.00 $16.00 $18.00 INBILLIONS Checkpoint Inhibitor Projected Value 2020 ($) Checkpoint Inhibitor projected Value 2020
  • 19. Costs of Check Point Inhibitors High  Avg cost of Immuno-oncology drugs: $137,000 per year 19 $120,000.0 $110,064.0 $130,931.0 $150,000.0 $156,000.0 $165,000.0 0 20,000 40,000 60,000 80,000 100,000 120,000 140,000 160,000 180,000 Yervoy Keytruda Opdivo Tecentriq Imfinizi Bavencio Annual Wholesale Acquisition Cost for Checkpoint Inhibitors ( $) AHIP High priced drugs estimated upon annual patient expenditures
  • 20. Leading Markets for Checkpoint Inhibitors 1. US** 2. Japan 3. Germany, France, UK, Italy, and Spain 4. Overall EU market 5. APAC region CONFIDENTIAL 20
  • 21. Timeline Of Approvals2011 • Yervoy; malignant melanoma 2014 • Opdivo; malignant melanoma • Keytruda; malignant melanoma 2015 • Opdivo: NSCLC • Opdivo; RCC • Keytruda; NSCLC 2016 • Opdivo: SCCHNN • Opdivo: Hodgkins Lymphoma • Opdivo: Malignant Melanoma • Tecentriq: Bladder Cancer and NSCLC 2017 • Keytruda: Hodgkins Lymphoma • Opdivo: Bladder Cancer • Bavencio :Bladder Cancer; metastatic merkel cell • Imfinzi: Bladder Cancer • Opdivo and Yervoy: metastatic melanomaCONFIDENTIAL 21
  • 22. Opdivo & Yervoy combo. yield better endpoints overall than other CPI’s alone 2 8 25 12 10 7 45 25 22 12 40 85 80 25 0 10 20 30 40 50 60 70 80 90 Response Rate (%) Overall Survival (Months) 1 year survival Rate (%) 2 year survival rate (%) Melanoma Trial Data gp100 Yervoy (phase III data) Opdivo (Phase II data) Opdivo + Yervoy (phase 1 data) Keytruda (phase I data)CONFIDENTIAL 22
  • 23. $83.00 $110.00 $159.00 $214.00 $249.00 $314.00 $356.00 $483.00 $584.00 $40.00 $162.00 $305.00 $475.00 $704.00 $840.00 $920.00 $1,310.00 $1,127.00 $325.00 $296.00 $240.00 $265.00 $263.00 $241.00 $285.00 $264.00 $330.00 $- $200.00 $400.00 $600.00 $800.00 $1,000.00 $1,200.00 $1,400.00 1Q15 2Q15 3Q15 4Q15 1Q16 2Q16 3Q16 4Q16 1Q17 CPI Sales Quarterly Sales Growth (millions) Keytruda Opdivo YervoyCONFIDENTIAL 23 CNBC
  • 24. $83.00 $110.00 $159.00 $214.00 $249.00 $314.00 $356.00 $483.00 $584.00 $40.00 $162.00 $305.00 $475.00 $704.00 $840.00 $920.00 $1,310.00 $1,127.00 $325.00 $296.00 $240.00 $265.00 $263.00 $241.00 $285.00 $264.00 $330.00 $- $500.00 $1,000.00 $1,500.00 $2,000.00 $2,500.00 1Q15 2Q15 3Q15 4Q15 1Q16 2Q16 3Q16 4Q16 1Q17 CPI Sales Quarterly Sales Growth (millions) Keytruda Opdivo YervoyCONFIDENTIAL 24
  • 25. Number of Anti PD-1/PD-L1 combination studies 2015 vs 2017 70 73 42 26 268 242 123 83 KEYTRUDA OPDIVO IMFINZI TECENTRIQ 2015 2017 CONFIDENTIAL 25 Jacob & Edwin Elmhirst May 2017: Data from EP Vantage PD-1 combo report 2015
  • 26. Checkpoint Inhibitors penetrate Market Share for oncology drugs 55% 20% 19% 6% 2014 Cytotoxic Agents 26 9% 10% 4% 8% 68% 1% 2024 Cytoxic agents EGFR inhibitors Angiogenesis Inhibitors ALK inhibitors Immune Checkpoint Inhibitors BRAF/MEK inhibitors Cancer Immunotherapy: Building on Initial Successes to Improve Clinical Outcomes ***
  • 27. Combinations of Opdivo + Yervoy (BMS:75 Studies in Total) 28, 37% 10, 13%5, 7% 4, 5% 3, 4% 3, 4% 2, 3% 2, 3% 2, 3% 16, 21% Melanoma NSCLC Renal Cancer Head and Neck Cancer Prostate Mesothelloma Breast Cancer SCLC Uveal Melanoma Other indications27 Evaluate Ltd May 2017
  • 28. Annual WAC vs Price per QALY avg Price per QALY: $250,155 > >WTP QALY: $100,000 120,000 130,931 110,064 150,000 129,000 415,950 236,492 219,179 Yervoy Opdivo Keytruda Tecentriq 0 50,000 100,000 150,000 200,000 250,000 300,000 350,000 400,000 450,000 Annual WAC Price per QALY WTP per QALY 28
  • 29. Checkpoint Inhibitors (Comparators) Company Drug Indications MOA DOA Annual WAC ORR BMS Opdivo (Nivolumab) Second line Metastatic Melanoma Pd-1 2015 $130,931 32% Genentech Tecentriq (Atezolizumab) First line treatment of Bladder Cancer Pd-l1 2016 $150,000 14.8% BMS Yervoy (Ipilimumab) Second line Metastatic Melanoma CTLA-4 2011 $120,0000 10.9% Merck Keytruda (Pembrolizumab) Second line NSCLC,HNSCC, CHL, Bladder Cancer, Metastatic Melanoma, MSI-H Pd-1 2014 $110,064 19%;69% Pfizer Bavencio (Avelumab) Second line Bladder Cancer, First line Metastatic Merkel Cell Carcinoma Pd-l1 2017 $156,000 16% Astrazeneca Imfinzi Bladder Cancer (second line Pd-l1 2017 $156,000 26% CONFIDENTIAL 29

Editor's Notes

  1. *full slide in backup and make one specific to BC – 5 products
  2. 2015, the nivolumab and ipilimumab combination therapy showed a higher response rate compared with ipilimumab alone. The results mean that more patients taking the combination therapy experienced melanoma tumor shrinkage if they received nivolumab and ipilimumab versus ipilimumab by itself.  Assuming a 20% co pay- out of pocket cost would be around $51,000 for a round of treatment
  3. Main efficacy endpoint was an increase in PFS11.4 months PFS vs 6.9 months Nivo alone 2.9 months Ipilimab alone
  4. Forecasts checkpoint inhibitors will achieve revenues of 16.55 billion by 2020. Revenues will expand as new treatments finish development and hit the market
  5. Bavencio has the highest cost, likely has to do with the fact that it is the hottest new cancer drug out on the market .
  6. Currently: Opdivo and Yervoy: Checkmate 142 Study- entering phase 3- for metastatic CRC
  7. Opdivo Sales grew by 835 million from 2015 to 2016 in total.
  8. Opdivo Sales grew by 835 million from 2015 to 2016
  9. Source: Evaluate Ltd May 2017 Most combination Studies in 2017 done with Keytruda In 2015 the most were done with bavencio
  10. Transition from EGFR inhibitors to Checkpoint Market dominance. CPI’s have rapidly infiltrate cancer settings and are substantially projected to dominate oncology market share
  11. There has been an upsurge in pd-l1 assets combined with othe Mab’s: The single most prolific combo grouping invoves Opdivo + Y Yervoy- with combines a PDL1 and a CTLA4 in has been proven successful but there is significant toxicity attached. Scientific rationale of hitting more that one immune checkpoint is that increase is that it increases the chances of overcoming multiple resistance pathways might be using to evade immune system attack.
  12. *full slide in backup and make one specific to BC – 5 products