Investor Presentation August 2020 Science-Based Innovation-Focused ADC Company
Investor PresentationAugust 2020
Science-Based
Innovation-Focused
ADC Company
Forward-Looking Statements
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This presentation and various remarks we make during this presentation, may contain forward-looking statements made pursuant to the Private Securities
Litigation Reform Act of 1995. Such statements, including statements regarding expectations for achieving full FDA approval based on our confirmatory data
for TRODELVY and the Company’s development of TRODELVY for additional indications, clinical trials (including the funding therefor, anticipated patient
enrollment, trial outcomes, timing or associated costs), regulatory applications and related timelines, including the filing and approval timelines for BLAs and
BLA supplements, out-licensing arrangements, forecasts of future operating results, potential collaborations, capital raising activities, and the timing for
bringing any product candidate to market, involve significant risks and uncertainties and actual results could differ materially from those expressed or
implied herein. Factors that could cause such differences include, but are not limited to, the Company’s reliance on third-party relationships and outsourcing
arrangements (for example in connection with manufacturing, logistics and distribution, and sales and marketing) over which it may not always have full
control, including the failure of third parties on which the Company is dependent to meet the Company’s business and operational needs for investigational
or commercial products and, or to comply with the Company’s agreements or laws and regulations that impact the Company’s business; the Company’s
ability to meet post-approval compliance obligations; imposition of significant post-approval regulatory requirements on our products, including a
requirement for a post-approval confirmatory clinical study, or failure to maintain or obtain full regulatory approval for the Company’s products, if received,
due to a failure to satisfy post-approval regulatory requirements, such as the submission of sufficient data from a confirmatory clinical study; the
uncertainties inherent in research and development; safety and efficacy concerns related to the Company’s products and product candidates; uncertainties
in the rate and degree of market acceptance of products and product candidates, if approved; inability to create an effective direct sales and marketing
infrastructure or to partner with third parties that offer such an infrastructure for distribution of the Company’s products and product candidates, if approved;
inaccuracies in the Company’s estimates of the size of the potential markets for the Company’s products and product candidates or limitations by regulators
on the proposed treatment population for the Company’s products and product candidates; decisions by regulatory authorities regarding labeling and other
matters that could affect the availability or commercial potential of the Company’s products and product candidates; the Company’s dependence on
business collaborations or availability of required financing from capital markets, or other sources on acceptable terms, if at all, in order to further develop
our products and finance our operations; new product development (including clinical trials outcome and regulatory requirements/actions); the risk that we
or any of our collaborators may be unable to secure regulatory approval of and market our drug candidates; risks relating to the COVID-19 pandemic in the
U.S. and around the world; risks associated with litigation to which the Company is or may become a party, including the cost and potential reputational
damage resulting from such litigation; loss of key personnel; competitive risks to marketed products; and the Company’s ability to repay its outstanding
indebtedness, if and when required, as well as the risks discussed in the Company’s filings with the Securities and Exchange Commission. The Company is
not under any obligation, and the Company expressly disclaims any obligation, to update or alter any forward-looking statements, whether as a result of
new information, future events or otherwise.
Seasoned Leadership Team to Drive Growth
Brendan DelaneyCHIEF COMMERCIAL OFFICER
Bryan Ball CHIEF QUALITY OFFICER
Usama MalikCHIEF FINANCIAL OFFICER
CHIEF BUSINESS OFFICER
Jared Freedberg GENERAL COUNSEL
SECRETARY
Kurt AndrewsCHIEF HUMAN
RESOURCES OFFICER
Dr. Behzad AghazadehEXECUTIVE CHAIRMAN
Dr. Loretta ItriCHIEF MEDICAL OFFICER
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John StubenrauchSVP, GLOBAL HEAD
OF MANUFACTURING
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TRODELVY™ – First ADC Approved Specifically for mTNBC
• Approved on April 22, 2020 in U.S.
• Strong initial launch execution
– Product in channel within one week of
approval
– NCCN guidelines updated within two
weeks of approval
• Rapid adoption in advanced mTNBC
– $20.1M net sales in first two months of
commercial launch
TRODELVY’s Launch Strategy is Clear
1. Establish TRODELVY as a standard of care for 3rd-line mTNBC• Drive rapid awareness & adoption through
product education
2. Optimize positive early clinical experience• Minimize barriers, set clear expectations,
educate on adverse event management
3. Become a recognized leader in TNBC• Build strong scientific and development
partnerships
Goals
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Strategic
Imperatives
TRODELVY’s Launch Executed with Strong Sense of Urgency
TRODELVY available in distribution channel
within a week of FDA approval
TRODELVY.com with SEO campaign within
hours of approval
First Peer-to-Peer speaker program
executed in week one of launch
Over 500 attendees for TRODELVY National
Broadcasts
First payer coverage policy in place within
two weeks of approval
KOL National Broadcasts
Trained Speaker Bureau
Broad HCP NPP campaign
Product Distribution
Trodelvy.com Website
Patient HUB
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Robust adoption in both the community and
academic settings (~67% community accounts)
U.S. Healthcare Professionals Rapidly Gaining Clinical Experience with TRODELVY
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*IntrinsiQ survey conducted on June 27th, 2020
NCCN Guidelines were updated to include
Sacituzumab-Govitecan on May 8, 2020
>80% of top 150 accounts penetrated in first
two months of commercial launch
>80% HCPs intend to prescribe TRODELVY in
third-line mTNBC*
>500 accounts ordered TRODELVY in first two
months of commercial launch
TRODELVY Exceeding Payer “Time to Review” Benchmarks
• Majority of national and regional payers have
implemented TRODELVY coverage policies
• Existing TRODELVY coverage policies have limited
restrictions
• Speed and quality of payer coverage resulting in
strong patient access to TRODELVY
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*
* Time-to-Review: represents the elapsed time from FDA approval to a
commercial payer implementing a coverage policy for a new product
Important News Flow to Maintain Launch Momentum
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• ASCENT data presentation, publication and regulatory submission
• Expanded clinical experience with TRODELVY in 3rd-line mTNBC
• Branded promotional campaign set to launch in Q3 2020
Robust Sales &
Marketing Execution Speed & Quality of
Payer Coverage
TRODELVY
Launch Excellence+ =
Upcoming
Market
Catalysts
Transforming the Treatment Paradigm for Complex Cancers
Company Transformed in Less Than Three Years
FTE doubled in size
Field Base, 67
Office, 317
384FTE
5 24 27 ~1,000Pivotal Global
Trials
Scientific
publications &
presentations
Approved
Investigator
Initiated Trials
Patients on
TRODELVY
Trials
Global Partnerships
3Commercial
3Manufacturing
3Clinical
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A Powerful Differentiated ADC Platform: Three Key Advantages
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• ADC platform uses SN-38 as
payload of choice
• SN-38 kills cancer cells by
damaging DNA
1. Payload – Validated & Well Tolerated
• Hydrolyzable linker for payload
release
• Allows for intra- and extra-cellular
(bystander effect) killing of tumor
cells
2. Novel Linker
• hRS7 in TRODELVY targets Trop-2 in multiple solid tumor
indications
• Other pipeline assets: labetuzumab govitecan targets
CEACAM5, IMMU-140 targets HLA-DR
3. Antibody – Highly Tumor Specific
Multiple TRODELVY Programs to Address Unmet Needs in Trop-2-Expressing Cancers
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Indication Designation Phase 1 Phase 2 Phase 3 Approval Partner
mTNBC (3L+)
mTNBC (3L) ASCENT
HR+/HER2‒ mBC TROPiCS-02
mTNBC (3L) (China)
mTNBC (1L) / mUC / mNSCLC (+ Tecentriq) MORPHEUS
mTNBC / mUC / Ovarian (+ Rubraca) SEASTAR
Urothelial (3L) TROPHY U-01
Urothelial (3L) (Pending FDA Discussion)
mNSCLC / H&N / Endometrial (Trop-2-enriched) TROPiCS-03
Cohort 1 Enrollment Completed
Met 1o and key 2o endpoints
TRODELVYTM
Diverse Registration-Oriented & Signal-Seeking Investigator-Initiated Trials to Explore Effectiveness of TRODELVY
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Indication Designation Phase 1 Phase 2 Phase 3 Sponsor
HER2‒ BC (post-neoadjuvant) SASCIA German Breast Group
mTNBC, PD-L1‒ (1L) (+ Keytruda)Dana Farber Cancer Institute
HR+/HER2‒ mBC, PD-L1+ (+ Keytruda)Dana Farber Cancer Institute
TNBC (neoadjuvant) NeoSTARMassachusetts General Hospital
mTNBC (2L) (+ Talzenna) Massachusetts General Hospital
Endometrial Cancer (Persistent or Recurrent) Yale University
Metastatic Prostate Cancer (2L) U of Wisconsin & PCCTC
Breast Brain Metastasis and Glioblastoma UT Health at San Antonio
Multi-Line Strategy to Establish TRODELVY as SoC in TNBC
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Neo/Adjuvant (24- 26k Pts)
1st Line (10-11k Pts)
2nd Line (9-10k Pts)
3rd Line+
(8-9k Pts)
Stage 3 locally advanced (unresectable), Stage 4 metastatic
Stage 1, 2 and 3 (resectable)
Phase 3 ASCENT
Phase 1b/2 MORPHEUS
Phase 1/2 SEASTAR
Phase 3 SASCIA
Highly Differentiated Therapy for mTNBC
Treatment Line
• mTNBC patients with at least 2 prior treatments in the metastatic setting
The Unmet Need
• Low response rates, short response duration and significant side effects with currently available therapies
• Patients with pre-existing peripheral neuropathy or cardiac impairment may only have supportive care options
Market Size
• U.S. ~8k patients
• EU5, Japan ~14k patients
Status
• FDA granted accelerated approval in 3L-mTNBC
• Confirmatory ASCENT study met 1o and key 2o endpoints
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Phase 3 ASCENT Study Confirmed Compelling Safety and Efficacy Profile of TRODELVY
National Institutes of Health. https://clinicaltrials.gov/ct2/show/NCT02574455 17
Continue treatment
until progressionN = 529
mTNBC
≥2 prior treatments
OR
1 therapy for advanced disease who also
progressed within 12 months of (neo)adjuvant
therapy
Primary Endpoint
• PFS (brain mets-neg)
Secondary Endpoint
• OS, ORR, DoR
Indication Endpoint
TRODELVY
10 mg/kg IV
day 1 & 8, every 21 days
Traditional chemotherapy treatment of
physicians’ choice*
Twin Arm Study
* Eribulin, gemcitabine, capecitabine & vinorelbine
Study Met Primary and Key Secondary Endpoints
Brian
mets+
capped
at 15%
TRODELVY – First ADC Shown to Improve Clinical Outcomes in Late-Line mTNBC
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TPC2 TRODELVY
1.7 Months
5.6 Months
ASCENT P3 Topline
mPFS HR=0.41, p<0.00011
1: mPFS: median progression free survival; primary endpoint in brain met negative patients
2: Treatment of physician’s choice: eribulin, capecitabine, gemcitabine, and vinorelbine
• ASCENT met key secondary endpoints,
incl. OS and ORR3
• Safety profile consistent with FDA-approved
label and no new safety signals
• Full data will be submitted to ESMO as late-
breaking abstract
• sBLA submission for full approval expected
in Q4’20, EU submission targeted for H1’21
Other Key Takeaways from ASCENT
3: OS: Overall Survival; ORR: Overall Response Rate by RECIST1.1
Drug Phase N PopulationORR
(%)
Median PFS
(months)
Median OS
(months)
Carboplatin1 3 188 1st line 31 3.1 12.4
Docetaxol1 3 188 1st line 36 4.5 12.3
Cisplatin or
Carboplatin2 2 861st line
(80.2%)25.6 2.9 11.0
Atezolizumab
+ nab-paclitaxel33 451
1st line
(untreated)56.0 7.2 21.3
Nab-paclitaxel3 3 4511st line
(untreated)45.9 5.5 17.6
* Includes breast cancer drugs with data from Phase 2/3 studies with minimum mTNBC sample size > 60; ORR and PFS data.
Source of data: 1) Tutt A, SABCS 2014; 2) Isakoff SJ, J Clin Oncol 2015; 3) Schmid P, et al. N Engl J Med. 2018;379:2108-2121
Room for Improvement with Existing First-Line TNBC Agents*
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Metastatic Urothelial Cancer – Targeting our 2nd High Unmet Need Indication
The Unmet Need
• Current therapies for metastatic disease post chemotherapy and immune checkpoint inhibitors offer low response rate, short response duration and high toxicity
Market Size
• 3rd line mUC – U.S. ~8k patients
• 3rd line mUC – EU5, Japan ~10k patients
Status
• May obtain accelerated approval based on results of Ph 2 TROPHY U-01 trial
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21
31(N=45)
14
8.6 8.9
Docetaxel
in 2nd line
Phase 33
Docetaxel
in 2nd line
Phase 22
Vinflunine
in 2nd line1TRODELVY
in ≥3rd line4
Docetaxel
in 2nd line
Phase 33
Docetaxel
in 2nd line
Phase 22
Vinflunine
in 2nd line1
TRODELVY
in ≥3rd line4
ORR(%)
PFS(months)
3.0
7.3(N=45)
2.8 2.8
TRODELVY Achieved Strong ORR and PFS Compared to SoC in Phase 1/2 Single-Arm Basket Study*
* Information is based on comparative results from independent studies
Source of data: 1) Bellmunt J, JCO 2009; 2) Petrylak D, JCO 2016; 3) Petrylak D, Lancet 2017; 4) Tagawa S, ASCO-GU 2019
Pivotal TROPHY U-01 Study of TRODELVY Designed to Support Accelerated Approval
National Institutes of Health. https://clinicaltrials.gov/ct2/show/NCT03547973 22
• First patient dosed in August 2018 in U.S.
• Interim cohort 1 results presented at ESMO 2019
• Full cohort 1 enrollment reached in October 2019
• Cohort 3 added to evaluate TRODELVY + pembrolizumab in CPI-naïve patients
Continue treatment
until progression
mUC
Cohort 1: Post platinum-and CPI-based therapies
(N = 100)
Cohort 2: 2nd line post CPI for cisplatin-ineligible
patients (N = 40)
Cohort 3: 2nd line post pt-based therapy for CPI-naïve
patients (N = ~60)
Primary Endpoint
• ORR (BICR)
Secondary Endpoint
• DoR, PFS & OS
Indication Endpoint
Cohort 1 & 2: TRODELVY
10 mg/kg IV
day 1 & 8, every 21 days
Cohort 3: TRODELVY +
pembrolizumab 200 mg day 1,
every 21 days
Single-Arm Study
Interim Results Confirm Clinical Activity in mUC
Endpoint Cohort 1 (N=35)
Median follow-up, mon 4.1
Patients continuing treatment, n (%) 20 (57)
ORR, n (%) [95% CI] 10 (29) [15, 46]
CR, n (%) 2 (6)
PR, n (%) 6 (17)
uPR pending confirmation,a n (%) 2 (6)
Median time to onset of response,
mon (range) 1.5 (1.2, 2.8)
a Follow-up scan is pending.
CI, confidence interval; CR, complete response; ECOG PS, Eastern Cooperative Oncology Group
Performance Status; ORR, objective response rate; PR, partial response; uPR, unconfirmed partial
response.
Category Subgroup ORR, % (n/N)
Overall N/A 29 (10/35)
Age<75 29 (8/28)
≥75 29 (2/7)
ECOG PS0 33 (5/15)
1 25 (5/20)
No. prior anticancer regimens
2 18 (2/11)
≥3 33 (8/24)
Visceral involvement at study entry
Yes 23 (5/22)
Liver 25 (2/8)
No 39 (5/13)
Bellmunt risk factors0-1 35 (10/29)
2-3 0 (0/6)
ORR in Patient SubgroupsResponse Outcomes
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New Therapeutic Options Needed for HR+/HER2– mBC
The Unmet Need
• The most common form of breast cancer in U.S.
• Initial treatments, endocrine and CDK4/6 therapy, eventually fail and cancer relapses, requiring chemotherapy treatment
• Prognosis for patients with visceral metastases is poor
Market Size
• 3rd line HR+/HER2‒ mBC – U.S. ~25k patients
• 3rd line HR+/HER2‒ mBC – EU5, Japan ~35k patients
Status
• Potential accelerated approval submission from ORR analysis on pre-specified number of patients in registrational Phase 3 TROPiCS-02 study
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25
31(N=54)
1311.0
Vinorelbine
in 2nd line
chemo mBC1
TRODELVY
in ≥3rd line
chemo3
ORR(%)
PFS(months)
3
6.8(N=54)
3.12.5
TRODELVY Achieved Impressive ORR and PFS Compared to SoC in Late-Line HR+/HER2– mBC*
* Information is based on comparative results from independent studies
Source of data: 1) Jones S, JCO 1995; 2) Kaufman PA, JCO 2015; 3) Kazmi S, ESMO 2019 Abstract 366P; 4) Kalinsky K, SABCS 2018
11.5
TRODELVY
in ≥3rd line
chemo4
Eribulin
in 3rd line
chemo mBC2
Eribulin
in 3rd line
chemo mBC3
Capecitabine
in 3rd line
chemo mBC3
Capecitabine
in 3rd line
chemo mBC2
Vinorelbine
in 2nd line
chemo mBC1
Phase 3 TROPiCS-02 Study in Late-Line HR+/HER2– mBC Designed to Support Accelerated Approval
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Continue treatment
until progressionN = 400
HR+/HER2‒ mBC
• Prior hormonal and CDK4/6 treatments
• ≥2 prior chemotherapies
Protocol Allows ORR Analysis for Potential Accelerated Approval Submission Based
on Pre-determined Number of Patients
Primary Endpoint
• PFS, ORR
Secondary Endpoint
• OS, DoR, Safety & QoL
Indication Endpoint
TRODELVY
10 mg/kg IV
day 1 & 8, every 21 days
Traditional chemotherapy treatment of
physicians’ choice
Twin Arm Study
National Institutes of Health. https://clinicaltrials.gov/ct2/show/NCTNCT03901339
* Eribulin, gemcitabine, capecitabine & vinorelbine
Adverse Event
mTNBC (N=108)1 mUC (N=35)2 HR+/HER2‒
mBC (N=50)3
Grade 3 (%) Grade 4 (%) Grade 3 (%) Grade 4 (%) Grade 3 or 4 (%)
Blood and lymphatic system
Neutropenia 26 16 29 26 42
Anemia 11 0 17 0 6
General and administration-site
Fatigue and asthenia 8 0 6 0 2
Gastrointestinal
Diarrhea 8 0 6 3 4
Nausea 6 0 0 0 2
Vomiting 6 0 0 0 4
Manageable and Predictable Safety Profile Allows for Repeated Dosing & Combination Use
27Source of data: 1) Bardia A, et al. N Engl J Med. 2019; 380:741-51; 2) Tagawa, S, et al. ESMO 2019; 3) Bardia, A, et al. ASCO 2018
No >grade 2 neuropathy or rash and no treatment-related deaths or interstitial lung disease, low discontinuation rates due to AEs
Grades 3 and 4 Adverse Events Occurring in >5% of Patients
TRODELVY has Potential to Change Treatment Landscape of Breast and Urothelial Cancers
Cancer Type
ORR (%) PFS (months)
Other Agents TRODELVY Other Agents TRODELVY
mTNBC11 – 15
(single chemo)33
1.7*
(erib, gem, cap or
vin)
5.6*
mUC9 – 14
(single chemo)
31**
29***
~2.8 – 3
(single chemo)
7.3**
TBD***
HR+/HER2‒ mBC11 – 13
(single chemo)31
~2.5 – 3.1
(cap, gem or erib)6.8
* From ASCENT topline; ** From IMMU-132-01 (full mUC Cohort); ** From TROPHY-U-01 interim
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Non-Small Cell Lung Cancer – Large Population with High Unmet Need
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The Unmet Need
• NSCLC accounts for about 85% of all lung cancers
• Following initial treatment with checkpoint inhibitors and chemotherapy, therapeutic 2nd line options for advanced disease are limited
Market Size
• Trop-2-enriched* 3rd line mNSCLC – U.S. ~10k patients
• Trop-2-enriched* 3rd line mNSCLC – EU5, Japan ~15k patients
Status
• Trop-2 biomarker-selected study (TROPiCS-03) launched to evaluate TRODELVY in NSCLC
* Initially targeting highest 25% Trop-2 expressors with potential increase of this percentage allowed under the study protocol
Trop-2-Enriched Multi-Cohort Study (TROPiCS-03) to Unlock Full Potential of TRODELVY
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Continue treatment
until progression
NSCLC & H&N• 3rd line post CPI- and
chemotherapy
Endometrial• 2nd line post platinum-
based chemotherapy
Primary Endpoint
• ORR
Secondary Endpoint
• DoR, PFS & Safety
Indication Endpoint
TRODELVY
10 mg/kg IV
day 1 & 8, every 21 days
Simon Two-Stage Design
Exploratory
• Biomarker, QoL
Stage 1: 40 Patients
per Indication
Stage 2: 60 Additional
Patients per Indication
National Institutes of Health. https://clinicaltrials.gov/ct2/show/NCT03964727
• Study initiated in July 2019 in U.S.
• First NSCLC patient dosed in October 2019
Significant U.S. Markets for TRODELVY – Large Opportunity in Rest of World
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mTNBC (8k-9k)
mUC (8k-15k)
HR+/HER2‒ mBC (25k/28k)
Cancer Indication (# U.S. Patients 3rd/2nd Line)
mNSCLC (40k/60k)
Well Capitalized to Pursue Strategic Priorities
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Balance Sheet June 30, 2020
Cash, cash equivalents, and marketable securities $975.5 million*
Basic shares outstanding (fully diluted) 231million (242 million)
Projected financial resources adequate to support planned operations into 2021, including:
• Commercial launch of TRODELVY in the United States in mTNBC
• Continue to expand clinical development programs for TRODELVY
• Invest in broader clinical development of ADC platform (including IMMU-130 and IMMU-140)
• Ongoing scale-up of global supply-chain to meet significant future commercial/clinical demand
• General working capital requirements
* Includes a $60 million milestone payment from Everest Medicines for TRODELVY approval in the U.S.
A Transformed Company At Inflection Point
Strong Foundation
• Validated ADC science & Trop-2 target
• Long patent life & unencumbered asset
Significant Market Opportunity
• Multiple tumor types & treatment lines
• Large indications
At Inflection Point
• Accelerated approval granted
• Multiple clinical, regulatory & commercial catalysts
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Partner of Choice
• Clinical: Roche, Clovis, MGH, DFCI, GBG, Wisconsin, MSK, Yale, Fred Hutch …
• Manufacturing: Samsung, JMPS, BSP …
• Commercial: Royalty Pharma, Janssen, Everest …