Q3/FY2023 FINANCIAL RESULTS
ENDED DECEMBER 31, 2023
Atsushi Kitamura
Chief Financial Officer (CFO)
Astellas Pharma Inc.
February 5, 2024
CAUTIONARY STATEMENT REGARDING FORWARD-LOOKING
INFORMATION
2
In this material, statements made with respect to current plans, estimates, strategies and beliefs and other statements that
are not historical facts are forward-looking statements about the future performance of Astellas Pharma. These statements
are based on management’s current assumptions and beliefs in light of the information currently available to it and involve
known and unknown risks and uncertainties. A number of factors could cause actual results to differ materially from those
discussed in the forward-looking statements. Such factors include, but are not limited to: (i) changes in general economic
conditions and in laws and regulations, relating to pharmaceutical markets, (ii) currency exchange rate fluctuations, (iii)
delays in new product launches, (iv) the inability of Astellas to market existing and new products effectively, (v) the inability
of Astellas to continue to effectively research and develop products accepted by customers in highly competitive markets,
and (vi) infringements of Astellas’ intellectual property rights by third parties.
Information about pharmaceutical products (including products currently in development) which is included in this material
is not intended to constitute an advertisement or medical advice. Information about investigational compounds in
development does not imply established safety or efficacy of the compounds; there is no guarantee investigational
compounds will receive regulatory approval or become commercially available for the uses being investigated.
AGENDA
3
I
Q3/FY2023 Consolidated Financial Results
FY2023 Revised Forecasts
II
Initiatives for Sustainable Growth
Q3/FY2023 FINANCIAL RESULTS: OVERVIEW
4
Revenue increased YoY, however, behind the full-year forecast
XTANDI & XOSPATA: In line with the full-year forecast revised upward in Q2
PADCEV: In line with the full-year forecast revised significantly upward in Q2
Potential peak sales revised upward incorporating the robust results of EV-302 study
VEOZAH: Overall initiatives are progressing, however, demand trails internal expectations
Full-year forecast revised downward
IZERVAY: Encouraging first full quarter performance since launch, expect further growth
Cost items
SG&A and R&D expenses were on track
Operating profit
Core OP behind the full-year forecast mainly due to the performance of VEOZAH
Full-year forecast for revenue and operating profit revised downward incorporating VEOZAH’s current progress
Full-year forecast revised in Nov 2023, Exchange rate assumption: 140 yen/USD,152 yen/EUR
(billion yen)
Q3/FY22 Q3/FY23 Change
Change
(%)
FY23
FCST
Revenue
1,164.4
1,189.1 +24.7 +2.1% 1,608.0
Cost of sales
226.1
219.3 -6.8 -3.0%
% of revenue
19.4%
18.4% -1.0 ppt
SG&A expenses
471.0
547.0 +76.0 +16.1% 737.0
US XTANDI co-pro fee
138.2
146.2 +8.0 +5.8% 187.0
SG&A excl. the above
332.7
400.7 +68.0 +20.4% 555.0
R&D expenses
206.1
216.3 +10.3 +5.0% 290.0
Amortisation of intangible assets
29.2
66.2 +37.0 +126.8%
Gain on divestiture of intangible assets
0.2
9.7 +9.5 -
Core operating profit
233.7
149.6 -84.0 -36.0% 199.0
Other income
2.5
8.5 +6.0 +236.6%
Other expenses
54.9
84.0 +29.1 +52.9%
Operating profit
181.3
74.1 -107.2 -59.1% 123.0
Profit before tax
180.2
73.6 -106.6 -59.1% 121.0
Profit
144.8
50.3 -94.5 -65.3% 85.0
Q3/FY2023 FINANCIAL RESULTS
5
＜Full basis＞
+58.8 bil. yen
+10.2 bil. yen
FX impact (YoY)
+13.8 bil. yen
+26.1 bil. yen
+6.9 bil. yen
+19.2 bil. yen
Other expenses
• Organizational restructuring
cost on a global scale:
approx. 18.4 bil. yen
Full-year forecast revised in Nov 2023, Exchange rate assumption: 140 yen/USD,152 yen/EUR
+6.9 bil. yen
Note) Amortisation of IZERVAY’s
intangible assets started
from Q2
(billion yen)
Q3/FY2023 YTD
YoY FY2023 FCST
560.0
+48.1
（+9%）
719.8
Global sales are in line with the full-year forecast revised upward in Q2
~5% growth even excluding FX impact, still growing even 10+ years on the market
Expect to achieve the full-year forecast
Sales expanded in all regions
US: Approval of M0 CSPC additional indication based on EMBARK study in Nov 2023
Steady growth in demand excluding PAP (demand YoY +3%)
41.3
+5.0
（+14%）
55.2
Global sales are in line with the full-year forecast revised upward in Q2
Near double-digit growth even excluding FX impact
Expect to achieve the full-year forecast
6
XTANDI & XOSPATA: BUSINESS UPDATE
Full-year forecast revised in Nov 2023, Exchange rate assumption: 140 yen/USD,152 yen/EUR
M0: Non-metastatic, CSPC: Castration-sensitive prostate cancer, PAP: Patient Assistance Program
Performance in line with the full-year forecast upwardly revised in Q2, expect to achieve the full-year forecast
7
Update of potential peak sales
Updated sales forecast incorporating the robust results of EV-302
study which exceeded initial expectations
Upward revision of potential peak sales:
• Peak sales is disclosed as “in-market sales,” not Astellas revenue
• Indications in early clinical phase are not included (NMIBC and other solid tumors)
(Reference) Image of economic conditions with Pfizer
Intended for approx. 50:50 profit split globally
Pfizer Astellas
Americas*
Pfizer books sales
Receive 50% of gross profit
(recognize in product sales as
PADCEV related revenue)
Ex-Americas
Receive 50% of gross profit Astellas books sales
Note) Receipt/payment percentage and schemes vary by region (profit sharing or royalty payment)
(Latest forecast)
400 - 500 billion yen
(Previous forecast)
300 - 400 billion yen
PADCEV: BUSINESS UPDATE
(billion yen)
Q3/FY2023 YTD
YoY FY2023 FCST
55.6
+22.5
（+68%）
85.2
<US>
Performance in line with full-year forecast revised significantly upward
in Q2, driven by the penetration of 1L mUC based on EV-103 study (cis-
ineligible) approved in April 2023
Approval of 1L mUC additional indication based on EV-302 study (both
cis-eligible and ineligible) in Dec 2023 at an incredible speed, only two
weeks after the FDA filing acceptance
• Expect significant sales contribution in FY2024 and beyond, driven by
the robust data and further expansion of eligible patient population
<Europe>
Reimbursement started in 3 new countries including Spain, a total of 13
countries as of now. Expect further sales growth
Full-year forecast revised in Nov 2023, Exchange rate assumption: 140 yen/USD,152 yen/EUR
*Americas includes the US, Canada and Latin America, 1L: First line, mUC: Metastatic urothelial cancer, Cis: Cisplatin, FDA: Food and Drug Administration
Peak sales revised upward to 400 - 500 billion yen incorporating the robust results of EV-302 study
Latest progress & outlook
Aim for the upper end of 500 billion yen
Q3/FY2023 YTD FY2023 Revised FCST Factors for the downward revision
3.6 bil. yen 7.1 bil. yen
DTC activities have been effective, however, it is taking longer to impact the demand increase
Based on market research, HCP’s perception of the current payer coverage progress is
“insufficient to actively prescribe VEOZAH” which is impacting the uptake
As a result, full-year forecast has been revised downward by incorporating the above factors and
reassessing the timing and pace of demand ramp to delay which was expected particularly in Q4
Only US
($ basis)
$25M $50M
8
VEOZAH: BUSINESS UPDATE
<Latest progress>
Total lives covered (payer coverage) expanded to ~35%
Expect over 50% by the end of FY2023
Reach
DTC campaign has reached an estimated ~56M women
Awareness
Consumer:
HCP:
Activation
Consumer:
HCP:
<Future initiatives & outlook>
Sales force to continue driving rapid and widescale awareness of
VEOZAH and educate HCPs on the expanding payer coverage
VEOZAH TV spot during the Super Bowl in the US
In FY2024, expect % of lives covered (payer coverage) to increase and
continued momentum from commercial investments
Mid- to long-term and peak sales outlook will be reviewed based on the
progress of overcoming HCP’s perception that coverage is insufficient
Overall initiatives are progressing, however, demand trails internal expectations
Downward revision of full-year forecast, reassessed the timing and pace of the FY2023 demand ramp to delay
Market
Access
DTC
Impact*
Approval in Dec 2023, launched in 7 countries including Germany and UK
Update on Europe
Note) Approved as “VEOZA” in Europe,
Exchange rate assumption: 140 yen/USD,152 yen/EUR , *Market Research December 2023, DTC: Direct-to-consumer, HCP: Healthcare professional
53% increase (Sep: ~15% vs Dec: ~25%)
40% increase (Sep: ~50% vs Dec: ~70%)
70% of women reported “High Intent” to ask HCP
about VEOZAH (40% increase from Oct)
76% of HCP’s report they are “Extremely Willing”
to prescribe VEOZAH (19% increase from Sep)
9
IZERVAY: BUSINESS UPDATE
Encouraging first full quarter performance since launch in the US, expect significant growth in FY2024
(billion yen)
Q3/FY2023 YTD
FY2023 FCST
5.3 11.0
Note) Screenshot is from the US Disease education campaign and is intended for US audiences only
Exchange rate assumption: 140 yen/USD,152 yen/EUR, *Excluding clinical trial vials. The figure disclosed in Q2/FY2023 earnings (10K units) was inclusive of clinical trial vials
GA: Geographic atrophy, AMD: Age-related macular degeneration, AAO: American Academy of Ophthalmology
Encouraging performance despite being only the first full quarter
since launch, as well as before permanent J-Code and label update
17,000+* vials shipped and available in 920+ Retina accounts since
launch through Q3, representing ~70% of accounts
Accelerated growth in IZERVAY usage following the GATHER2 data
release at AAO 2023 (nonpromoted use)
Estimate market share in the Q3 period to be ~20% based on
reported volume shipments
Safety profile so far has been consistent with clinical trial results
Progress since launch
Expect significant growth in FY2024 driven by upcoming milestones;
• Received confirmation of permanent J-Code effective Apr 1 which will
be a driver of reimbursement confidence and accelerant of demand
• Anticipate approval of label update within FY2024
Future outlook
Branded campaign for IZERVAY:
• Achieved 55% brand awareness among GA patients post-launch
Disease awareness campaign for GA:
• Contributed to 56% awareness of GA among dry AMD patients
DTC activities to increase awareness (as of Dec 2023)
Disease awareness campaign
with two-time Emmy® Award-
winning actor Eric Stonestreet,
who shared his personal
connection with GA in a national
PR effort (askaboutGA.com
)
SG&A expenses increased YoY due to the impact of the acquisition of Iveric Bio and
the investment in VEOZAH, however, progress in line with expectations
Q3/FY2023 FINANCIAL RESULTS: COST ITEMS
10
Core basis: YoY comparison, ratio to revenue, and progress against FCST, for major cost items
Cost Items YoY change
Ratio to
Revenue
Progress
against FCST
Cost of sales -3.0%
18.4%
(-1.0 ppt YoY)
- Cost of sales ratio was as expected
SG&A
expenses
excl. US XTANDI
co-pro fee
+20.4%
(+14.6% excl.
FX impact)
33.7%
(+5.1 ppt YoY)
72.9％
YoY increase excl. FX impact: approx. +49.0 bil. yen
Impact of Iveric Bio acquisition (approx. +20.0 bil. yen. YoY)
Increase in VEOZAH-related costs (approx. +30.0 bil. yen YoY)
Reduction of mature products-related costs (approx. -6.0 bil. yen YoY)
R&D
expenses
+5.0%
(+1.6% excl.
FX impact)
18.2%
(+0.5 ppt YoY)
74.6% Impact of Iveric Bio acquisition: approx. +8.0 bil. yen
Full-year forecast revised in Nov 2023, Exchange rate assumption: 140 yen/USD,152 yen/EUR
（
billion yen
）
FY2023
FCST*
FY2023
Revised FCST
Change Main items of revision
Revenue 1,608.0
1,562,0 -46.0
Downward revision of VEOZAH: 53.3 bil. yen 7.1 bil. yen
(US only: $375M $50M)
SG&A expenses 737.0
731.0 -6.0
Review of VEOZAH investment timing aligned with reassessing
the timing and pace of demand ramp-up
R&D expenses 290.0
286.0 -4.0
Applied accounting treatment recognizing IZERVAY’s
production cost (R&D expenses) as inventory assets
Core operating profit 199.0
164.0 -35.0
Operating profit 123.0
83.0 -40.0
FY2023 REVISED FORECAST
11
＜Full basis＞
Revenue: Downward revision
VEOZAH: Full-year forecast revised downward incorporating current progress
No change has been made on exchange rates and other products’ full-year forecast
Core OP: Downward revision
Profit also revised downward aligned with VEOZAH’s downward revision
Partially mitigated by the review of cost items
*Revised in Nov 2023, Exchange rate assumption: 140 yen/USD,152 yen/EUR
AGENDA
12
I
Q3/FY2023 Consolidated Financial Results
FY2023 Revised Forecasts
II
Initiatives for Sustainable Growth
INITIATIVES FOR SUSTAINABLE GROWTH: OVERVIEW
13
XTANDI and Strategic products
enzalutamide / XTANDI : Approval of additional indication for M0 CSPC* (US)
enfortumab vedotin / PADCEV : Approval (US) and filing (Europe, Japan) of additional indication for 1L mUC
zolbetuximab : Complete response letter issued (US)
fezolinetant / VEOZAH : Approval (Europe), Phase 3 studies to start (Japan)
avacincaptad pegol / IZERVAY: Submission for label update (US)
Focus Area approach
Clinical studies ongoing:
Early data readout in Phase 1 studies expected in FY2023 for ASP1570, ASP2138 and ASP3082
Others
Open innovation initiatives:
Open labs in Tsukuba and Kashiwa-no-ha area, strategic collaboration with Mass General Brigham
VEOZAH: Approved as “VEOZA” in Europe
*with biochemical recurrence at high risk for metastasis
M0: Non-metastatic, CSPC: Castration-sensitive prostate cancer, 1L: First line, mUC: Metastatic urothelial cancer
14
XTANDI AND STRATEGIC PRODUCTS: KEY EVENTS EXPECTED IN FY2023
VEOZAH: Approved as “VEOZA” in Europe
*with biochemical recurrence at high risk for metastasis. M0: Non-metastatic, CSPC: Castration-sensitive prostate cancer, M1: Metastatic, TLR: Topline results, 1L: First line,
mUC: Metastatic urothelial cancer, CHMP: Committee for Medicinal Products for Human Use, HSCT: Hematopoietic stem cell transplant
Q1 (Apr-Jun) Q2 (Jul-Sep) Q3 (Oct-Dec) Q4 (Jan-Mar)
enzalutamide/
XTANDI
enfortumab
vedotin/
PADCEV
zolbetuximab
fezolinetant/
VEOZAH
avacincaptad
pegol/
I Z ERVAY
<Other updates>
fezolinetant / VEOZAH: Phase 3 studies in Japan (STARLIGHT 2 and STARLIGHT 3) to start in Q4
gilteritinib / XOSPATA: Development for post-HSCT maintenance acute myeloid lymphoma based on MORPHO study discontinued
May
Approval (US)
Aug
Acceptance
(M0 CSPC*; US)
As of Feb 2024
Aug
Approval (US)
Acceptance (Europe)
Jul
Acceptance (US, Europe, China)
Jun
Acceptance (Japan)
Sep
Acceptance
(M0 CSPC*; Europe, M1 CSPC; China)
Sep
EV-302 TLR
Sep
GATHER2 TLR
(24 month)
Oct
CHMP positive
opinion (Europe)
Nov
Approval
(M0 CSPC*; US)
Dec
Approval
(US)
Nov
Acceptance
(1L mUC; US)
Dec
Approval (Europe)
Jan
Complete response (US)
Jan
Acceptance
(1L mUC; Europe, Japan)
Jan
Submission
(Label update; US)
PROGRESS IN LATE-STAGE PIPELINE
15
VEOZAH: Approved as “VEOZA” in Europe
M0: Non-metastatic, CSPC: Castration-sensitive prostate cancer, BCR: Biochemical recurrence, GnRH: gonadotropin-releasing hormone, TLR: Topline results,
sBLA: Supplemental Biologics License Application, VMS: Vasomotor symptoms
4 regulatory approvals for new indication or region received during the quarter
Indication Region
M0 CSPC with BCR at high risk
for metastasis
US
First novel hormonal therapy for the indication
Approved for monotherapy as well as combination
with GnRH analog
Locally advanced or metastatic
urothelial cancer
(combination with pembrolizumab)
US
New treatment option to transform the current
standard of care for decades
Approval in a remarkably short period of time
• 3 months after TLR readout in EV-302 study
• 2 weeks after sBLA acceptance
Moderate to severe VMS
associated with menopause
Europe
First-in-class nonhormonal treatment option
Expansion of opportunities to address unmet
medical needs worldwide
Invasive aspergillosis and invasive
mucormycosis in pediatric patients
US
High unmet medical needs in pediatric patients
Extension of market exclusivity period by 6 months
granted
ZOLBETUXIMAB: LATEST STATUS
16
Addressing unresolved
deficiencies by CMO
Jan 4, 2024
A new PDUFA date will be identified upon FDA acceptance
Target: Q1 FY2024 (Apr-Jun)
<Action plan>
<Complete response letter (CRL) by FDA>
Unresolved deficiencies following pre-license inspection of a third-party manufacturing facility
FDA has not raised any concerns related to the clinical data, and is not requesting additional clinical studies
<Note>
Reviews of applications outside of the US are continuing as planned
Regulatory agencies around the world conduct their reviews independently, and the review decisions are based
on the different requirements and expectations of each regulatory agency
No other Astellas products are affected
FDA: Food and Drug Administration, CMO: Contract manufacturing organization, BLA: Biologics License Application, PDUFA: Prescription Drug User Fee Act
CRL issued
BLA
resubmission
Pre-license
inspection
17
PROGRESS IN FOCUS AREA APPROACH:
CURRENT STATUS OF PROJECTS IN CLINICAL TRIAL
*Not exhaustively listed. AAV: Adeno-associated virus, MTM1: Myotubularin 1, FDA: Food and Drug Administration, GAA: Acid alpha-glucosidase, DGK: Diacylglycerol kinase,
TSPAN8: Tetraspanin-8, IL-2: Interleukin-2, RPE: Retinal pigment epithelium, UDC: Universal donor cell, PPAR: Peroxisome proliferator-activated receptor,
PMM: Primary mitochondrial myopathies, DMD: Duchenne muscular dystrophy, KRAS: Kirsten rat sarcoma viral oncogene homologue
Primary Focus Biology/Modality/Technology* Project Mechanism of Action Current status
Genetic
Regulation
Gene replacement (AAV)
AT132 MTM1 gene ASPIRO study put on clinical hold by FDA in Sep 2021
AT845 GAA gene Phase 1 study ongoing
Gene regulation (AAV)
Immuno-
Oncology
Checkpoint ASP1570 DGKζ inhibitor Phase 1 study ongoing toward early data readout in FY2023
Bispecific immune cell engager
ASP2138
Anti-Claudin 18.2 and
anti-CD3
Phase 1 study ongoing toward early data readout in FY2023
ASP2074
Anti-TSPAN8 and
anti-CD3
Phase 1 study ongoing
ASP1002 Undisclosed Phase 1 study ongoing
Oncolytic virus (systemic) ASP1012 Leptin-IL-2 Phase 1 study under preparation to start in Q4/FY2023
Cancer cell therapy
Blindness &
Regeneration
Cell replacement ASP7317 RPE cells Phase 1b study ongoing
Cell replacement (UDC)
Gene regulation (AAV)
Mitochondria
Gene regulation & mitochondrial
biogenesis
ASP0367 PPARδ modulator
PMM: Phase 2/3 study ongoing
DMD: Next step under discussion
Targeted Protein
Degradation
Protein degradation ASP3082 KRAS G12D degrader Phase 1 study ongoing toward early data readout in FY2023
Primary Focus
Candidate
Immune modulating/regulatory cells
Tissue-specific immune regulation
Small molecule
Antibody
Gene
Cell
Modality
OPEN INNOVATION INITIATIVES
18
Advancing open innovation in life science ecosystems globally and accelerating early R&D
Activities at research stage Activities at early development stage
TME: Tumor microenvironment, TME iLab: TME imaging and interactive research for innovation
Open innovation hub for TME research
TME iLab
Focused on incorporating external innovation and co-
creation through collaborations with academia and other
companies, while contributing to life science ecosystems
Leverage open laboratories as part of these efforts:
Started activities of SakuLab
TM
-Tuskuba and TME iLab
in Tsukuba and Kashiwa-no-ha area
Located at Astellas’ Tsukuba Research Center
Available for academia and start-ups
SakuLab
TM
-Tsukuba
TM
Five-year strategic collaboration
with one of the leading biomedical
research organizations in US
Aim to advance translational
medicine and accelerate early
development of novel therapies
Initial focus in key areas of R&D investment for Astellas:
oncology, rare disease, cell and gene therapy
Expected to better understand diseases and modalities
and optimize clinical trials
Further reinforces Astellas' presence in the Greater
Boston innovation ecosystem
PROGRESS IN FY2023 AND FUTURE OUTLOOK
19
Achieved many key milestones including new product launches and additional indications in FY2023
Expecting Revenue and Profit to increase in FY2024 through contribution of VEOZAH, PADCEV and
IZERVAY as growth drivers
• VEOZAH: launch in US and Europe
• PADCEV (1L mUC): positive results
from EV-302 study, approval in US,
filing in Europe and Japan
• I Z E RVAY : launch in US, filing in
Europe, positive additional results
from GATHER2 study
• zolbetuximab: global filing
Major progress in FY2023
contributing to future growth
VEOZAH: Approved as “VEOZA” in Europe
1L: First line, mUC: Metastatic urothelial cancer
Revenue
Core OP
FY2023
FY2024
I Z E RVAY
PADCEV
zolbetuximab
VEOZAH
FY2025
Optimization of
cost structure
APPENDIX
Q3/FY2023: REVENUE BY REGION
21
(billion yen) Q3/FY2022 Q3/FY2023 Change (%)
Japan
204.5
211.0 +3.2%
United States
501.1
481.4 -3.9%
Established Markets
272.2
306.3 +12.5%
Greater China
65.2
67.3 +3.3%
International Markets
104.2
118.8 +14.0%
Established Markets: Europe, Canada, etc., Greater China: China, Hong Kong, Taiwan,
International Markets: Latin America, Middle East, Africa, Southeast Asia, South Asia, Russia, Korea, Australia, Export sales, etc.
22
Currency Q3/FY2022 Q3/FY2023 Change
USD 137 yen 143 yen +7 yen
EUR 141 yen 155 yen +15 yen
Average rate for the period
<Impact of exchange rate on financial results>
58.8 billion yen increase in revenue, 13.8 billion yen increase in core OP
Q3/FY2023 ACTUAL: FX RATE
23
Currency
Average rate
1 yen depreciation from assumption
Revenue Core OP
USD
Approx. +3.2 bil. yen Approx. +0.1 bil. yen
EUR
Approx. +1.4 bil. yen Approx. +0.6 bil. yen
FY2023 FORECAST: FX RATE & FX SENSITIVITY
Exchange rate
Average for the period
FY2023
Initial FCST
FY2023
Revised FCST
*
Change
USD 130 yen 140 yen +10 yen
EUR 140 yen 152 yen +12 yen
Estimated FX sensitivity (Q3 onwards) of FY2023 revised forecasts by 1 yen depreciation
Forecast rates Q3/FY2023 onwards: 140 yen/USD, 150 yen/EUR
*Disclosed in Nov 2023
24
(billion yen)
FY2022 end Dec 31, 2023
Total assets
2,456.5 3,368.7
Cash and cash equivalents
376.8 254.0
Total equity attributable to owners of the parent
Equity ratio (%)
1,508.0
61.4%
1,503.3
44.6%
(billion yen)
Q3/FY2022 Q3/FY2023
Cash flows from operating activities
212.2 100.5
Cash flows from investing activities
-61.8 -823.6
Free cash flows
150.4 -723.1
Cash flows from financing activities
-91.1 583.1
Increase/decrease in short-term borrowings and CP
-15.0 263.2
Proceeds from issuance of bonds and long-term borrowings
50.0 471.6
Acquisition of treasury shares
-10.6 -10.7
Dividends paid
-100.4 -116.7
As of end of December, Balance of bonds (Incl. CP) and borrowings： 871.0 billion yen
BALANCE SHEET & CASH FLOW HIGHLIGHTS
CP: Commercial Paper
MAIN INTANGIBLE ASSETS (AS OF DEC 31, 2023)
25
Bil. yen Foreign currency
*
AT132 15.3 USD 109M
AT845 10.2 USD 73M
Other gene therapy related program
**
92.5 USD 656M
Gene therapy related technology
**
68.1 USD 483M
VEOZAH 90.0 EUR 566M
EVRENZO 21.4 -
zolbetuximab 64.0 EUR 493M
IZERVAY （US）
702.6 USD 4,981M
IZERVAY (Ex-US）
155.2 USD 1,100M
* VEOZAH, zolbetuximab: foreign currency is a reference value based on the currency at the time of acquisition of the intangible asset
** Acquired during the acquisition of Audentes (now Astellas Gene Therapies)
26
14
16
22
24
25 25 25
26
27
30
32
34
36
38
40
42
50
60
70
0
1,000
2,000
3,000
4,000
5,000
6,000
0
20
40
60
80
100
FY05
FY06
FY07
FY08
FY09
FY10
FY11
FY12
FY13
FY14
FY15
FY16
FY17
FY18
FY19
FY20
FY21
FY22
FY23
FY24
FY25
Dividends
Core OP
Aiming for higher level of dividends increase during CSP2021 aligned with the robust profit growth forecast
Raise dividend level aligned
with profit / cashflow plan and
actual performance
throughout CSP2021 period
Flexibly execute share
buyback by excess cash
Top priority is investment
for business growth
2
1 3
*
Trend of Core OP and dividends
Core OP
(billion yen)
600
500
400
300
200
100
Dividends
(yen)
For illustrative purposes only
CAPITAL ALLOCATION
*Prior to FY2012, operating profit is in accordance with J-GAAP
CSP: Corporate Strategic Plan
27
Please refer to R&D pipeline list for details including target disease.
enfortumab vedotin
(MIBC)
gilteritinib
(Earlier-stage AML, pediatric use)
fezolinetant
(VMS due to menopause: China, Japan)
roxadustat
(Anemia associated with CKD, pediatric
use: Europe)
mirabegron
(Neurogenic detrusor overactivity,
pediatric use: Europe)
Phase 1 Phase 2 Phase 3
Others
Projects with Focus Area approach
XTANDI and Strategic products
enfortumab vedotin
(Other solid tumors)
zolbetuximab
(Pancreatic adenocarcinoma)
resamirigene bilparvovec/
AT132
(XLMTM)
avacincaptad pegol
(Stargardt disease)
bocidelpar/ASP0367
(Primary mitochondrial myopathies)
enfortumab vedotin
(NMIBC)
gilteritinib
(Newly diagnosed AML, HIC-ineligible)
ASP1570
ASP2138
ASP2074
ASP1002
ASP1012
ASP7317
bocidelpar/ASP0367
(Duchenne muscular dystrophy)
zocaglusagene nuzaparvovec/
AT845
ASP3082
abiraterone decanoate/
PRL-02/ASP5541
enzalutamide
(M0 CSPC*: Europe, M1 CSPC: China)
enfortumab vedotin
(mUC previously untreated: Europe, Japan;
mUC pretreated: China)
zolbetuximab
(Gastric and GEJ adenocarcinoma:
Japan, US, Europe, China)
avacincaptad pegol
(GA secondary to AMD: Europe)
peficitinib
(Rheumatoid arthritis: China)
Submitted/Filed
*with biochemical recurrence at high risk for metastasis. NMIBC: Non-muscle-invasive bladder cancer, AML: Acute myeloid leukemia, HIC: High-intensity chemotherapy,
XLMTM: X-linked myotubular myopathy, MIBC: Muscle-invasive bladder cancer, VMS: Vasomotor symptoms, CKD: Chronic kidney disease, M0: Non-metastatic, M1: Metastatic,
CSPC: Castration-sensitive prostate cancer, mUC: Metastatic urothelial cancer, GEJ: Gastroesophageal junction, GA: Geographic atrophy, AMD: Age-related macular degeneration
ROBUST PIPELINE OF ASTELLAS
28
Note: Phase 1 entry is defined as confirmation of IND open.
Phase transition is defined by approval of company decision body for entering to next clinical phase.
Filing is defined as submission of application to health authorities.
Discontinuation is defined by the decision of company decision body.
IND: Investigational New Drug
PROGRESS IN OVERALL PIPELINE
Phase 1 Entry to Approval since the Last Financial Results Announcement
Phase 1 Entry Phase 2 Entry Phase 3 Entry Filing Approval
enzalutamide
Nonmetastatic castration-
sensitive prostate cancer with
biochemical recurrence at high
risk for metastasis: US
enfortumab vedotin
Locally advanced or metastatic
urothelial cancer, previously
untreated (first line): US
fezolinetant
Moderate to severe vasomotor
symptoms associated with
menopause: Europe
isavuconazole
Invasive aspergillosis and
invasive mucormycosis in
pediatric patients: US
enfortumab vedotin
Locally advanced or
metastatic urothelial cancer,
previously untreated (first
line): Europe, Japan
fezolinetant
Vasomotor symptoms
associated with
menopause: Japan
29
(Red: Updates since the last financial results announcement)
Project / Product Indication Current status
enzalutamide/
XTANDI
M1 CSPC • NDA accepted in China in Sep 2023
M0 CSPC with BCR at high risk for metastasis • Approved in US in Nov 2023. Type II variation accepted in Europe in Sep 2023
enfortumab
vedotin/
PADCEV
Metastatic urothelial cancer
• Previously untreated (first line): Approved in US in Dec 2023. Type II variation/sNDA accepted
in Europe/Japan in Jan 2024
• Pretreated: BLA accepted in China in Mar 2023
Muscle-invasive bladder cancer • Phase 3 studies ongoing. Enrollment completed in Phase 3 EV-304 study
Non-muscle-invasive bladder cancer • Phase 1 study ongoing
Other solid tumors • Phase 2 study ongoing
gilteritinib/
XOSPATA
Relapsed and refractory AML • China: Phase 3 study stopped due to efficacy
AML, post-HSCT maintenance • Development based on Phase 3 MORPHO study discontinued
AML, newly diagnosed (HIC-eligible) • Phase 3 study ongoing (enrollment completed)
AML, newly diagnosed (HIC-ineligible) • Phase 1 study ongoing
AML, post-chemotherapy • Obtained topline results from Phase 2 GOSSAMER study
zolbetuximab
Gastric & GEJ adenocarcinoma
• NDA accepted in Japan in Jun 2023. BLA/MAA accepted in Europe and China in Jul 2023.
Received complete response letter in US in Jan 2024
Pancreatic adenocarcinoma • Phase 2 study ongoing
fezolinetant/
VEOZAH
VMS due to menopause
• Europe: Approved in Dec 2023
• China: Obtained topline results from Phase 3 MOONLIGHT 1 and MOONLIGHT 3 studies
• Japan: Phase 3 studies under preparation to start in Q4 FY2023
avacincaptad
pegol/
IZERVAY
GA secondary to AMD • MAA accepted in Europe in Aug 2023. sNDA for label update submitted in US in Jan 2024.
Stargardt disease • Phase 2b study ongoing
VEOZAH: Approved as “VEOZA” in Europe. M1: Metastatic, M0: Non-metastatic, CSPC: Castration-sensitive prostate cancer, BCR: Biochemical recurrence,
(s)NDA: (Supplemental) New Drug Application, BLA: Biologics License Application, AML: Acute myeloid leukemia, HSCT: Hematopoietic stem cell transplant, HIC: High-intensity chemotherapy,
GEJ: Gastroesophageal junction, MAA: Marketing Authorization Application, VMS: Vasomotor symptoms, GA: Geographic atrophy, AMD: Age-related macular degeneration
XTANDI AND STRATEGIC PRODUCTS: STATUS UPDATE
30
Initial
Diagnosis
PROSPER
M0 CRPC
AFFIRM
M1 CRPC
(2nd line+)
Localized Prostate Cancer Castration-Resistant Prostate Cancer
PREVAIL
M1 CRPC
(1st line)
M1 CSPC
newly-diagnosed
M1 CSPC
recurrent
Castration-Sensitive
Prostate Cancer
M0 CSPC
with BCR at high
risk for metastasis
ARCHES
EMBARK
Launched Launched
Launched
US/Europe/
Japan
China
• M1 CSPC: NDA accepted in Sep 2023
Launched
BCR: Biochemical recurrence, M1: Metastatic, M0: Non-metastatic, CSPC: Castration-sensitive prostate cancer, CRPC: Castration-resistant prostate cancer,
ENZA: enzalutamide, ADT: Androgen deprivation therapy, mono: Monotherapy
(Red: Updates since the last financial results announcement)
Local Therapy
Surgery, Radiation
or both
PSA
Recurrence
(BCR)
P3: EMBARK
NCT02319837
M0 CSPC
ENZA + ADT vs. placebo + ADT
vs. ENZA mono
n=1,068
Approved in US in Nov 2023
Type II variation accepted in Europe in Sep 2023
ENZALUTAMIDE (1/2): ANDROGEN RECEPTOR INHIBITOR
Launched (US)
31
Continued potential in earlier lines with consistent survival benefit and longer duration of treatment
Disease stage
Castration-sensitive (CSPC) Castration-resistant (CRPC)
M0 M1 M0
M1
(pre-chemo)
M1
(post-chemo)
Phase 3 study EMBARK ARCHES ENZAMET PROSPER PREVAIL AFFIRM
Control Placebo Placebo
Conventional
NSAA
Placebo Placebo Placebo
Primary
endpoint
MFS
HR 0.42
rPFS
HR 0.39
OS
HR 0.67
MFS
HR 0.29
rPFS
HR 0.17
OS
HR 0.71*
OS
HR 0.63
OS (Ongoing)
HR 0.66
HR 0.67
HR 0.73
HR 0.77
HR 0.63
DoT
32.4 months**
40.2 months
29.5 months
33.9 months
17.5 months
8.3 months
: Data obtained, *: Prespecified interim analysis, **: excluding treatment suspension period
Early stage Late stage
M0: Non-metastatic, M1: Metastatic, CSPC: Castration-sensitive prostate cancer, CRPC: Castration-resistant prostate cancer, NSAA: Non-steroidal antiandrogen,
HR: Hazard ratio, MFS: Metastasis-free survival, rPFS: Radiographic progression-free survival, OS: Overall survival, DoT: Duration of treatment
ENZALUTAMIDE (2/2): PHASE 3 STUDY DATA BY DISEASE STAGE
32
Stages 2 and 3
Stage 4
Clinical
studies
for EV
P2: EV-201
(Cohort 2)
Platinum-naïve
and Cis-ineligible
P3: EV-302
Platinum-eligible
vs. Chemo
Phase 1 or 2
Phase 3
P3: EV-301
vs. Chemo
P2: EV-201
(Cohort 1)
Patient
treatment
MIBC
mUC
Early stage - Disease stage of urothelial cancer - Late stage
RC-eligible
P3: KEYNOTE-905
/ EV-303
Cis-ineligible
vs. SoC (RC alone)
P3: KEYNOTE-B15
/ EV-304
Cis-eligible
vs. SoC (NAC + RC)
Platinum and
PD-1/L1 inhibitor
pretreated
PD-1/L1 inhibitor
pretreated
Previously
untreated
(first line)
EV mono
(i.v.)
Target
EV regimen
P2: EV-203
(Bridging study
in China)
EV+Pembro combo
(i.v.; perioperative)
sBLA approved
EV mono
(i.v.)
EV+Pembro
combo (i.v.)
Stages 0a-1
NMIBC
BCG-
unresponsive
P1: EV-104
EV mono
(intravesical)
P1b/2: EV-103
(Dose escalation
cohort & Cohort A)
Cis-ineligible
(Cohort K)
Cis-ineligible
EV + Pembro,
EV mono
(Cohorts H & L)
Cis-ineligible
EV mono (neoadjuvant
/perioperative) + RC
Approved
(AA)
sBLA (to convert
regular approval)
approved in US.
Approved in JP
and Europe
sBLA approved
(AA)
ADC: Antibody-drug conjugate, mUC: Metastatic urothelial cancer, NMIBC: Non-muscle-invasive bladder cancer, MIBC: Muscle-invasive bladder cancer,
BCG: Bacillus Calmette-Guerin, RC: Radical cystectomy, mono: Monotherapy, Pembro: Pembrolizumab, i.v.: Intravenous, Cis: Cisplatin, SoC; Standard of care,
NAC: Neoadjuvant chemotherapy, Chemo: Chemotherapy, sBLA: Supplemental Biologics License Application, AA: Accelerated Approval
BLA accepted
ENFORTUMAB VEDOTIN (EV) (1/4): NECTIN-4 TARGETED ADC
OVERALL UC PROGRAM
sBLA approved in US
(Red: Updates since the last financial results announcement)
33
P3: EV-301
NCT03474107
mUC, Platinum and PD-1/L1 inhibitor pretreated;
EV mono vs. Chemo
n=608
sBLA (to convert regular approval) approved in US in Jul 2021.
Approved in Japan in Sep 2021, in Europe in Apr 2022
P3: EV-302
NCT04223856
mUC, Previously untreated, Platinum-eligible;
EV + Pembro vs. Chemo
n=990
Approved in US in Dec 2023.
Type II variation/sNDA accepted in Europe/Japan in Jan 2024
P3: EV-303
/KEYNOTE-905
NCT03924895
MIBC, Cis-ineligible;
Pembro +/- EV (perioperative) + RC vs. RC alone
n=857 FSFT in Pembro + EV arm: Dec 2020
P3: EV-304
/KEYNOTE-B15
NCT04700124
MIBC, Cis-eligible; EV + Pembro (perioperative) + RC
vs. Chemo (neoadjuvant) + RC
n=784 Enrollment completed
P2: EV-201
NCT03219333
mUC, PD-1/L1 inhibitor pretreated; EV mono
Cohort 1: Platinum pretreated
Cohort 2: Platinum naïve and Cis-ineligible
n=219
Cohort 1: Approved (under the Accelerated Approval program)
Cohort 2: sBLA approved in US in Jul 2021
P1b/2: EV-103
NCT03288545
Cohorts A - G and K (mUC):
A-G: Combo with Pembro and other chemo
K: EV mono, EV + Pembro
Cohorts H, J and L (MIBC, Cis-ineligible, + RC):
H: EV mono (neoadjuvant)
J (optional): EV + Pembro (neoadjuvant)
L: EV mono (perioperative)
n=348
Dose Escalation/Cohort A and Cohort K: sBLA approved (under
the Accelerated Approval program) in US in Apr 2023.
Enrollment completed
P2: EV-203
NCT04995419
<Bridging study in China>
mUC, Platinum and PD-1/L1 inhibitor pretreated; EV mono
n=40 BLA accepted in China in Mar 2023
P1: EV-104
NCT05014139
NMIBC, High-risk BCG-unresponsive; Intravesical EV mono n=58 FSFT: Jan 2022
P2: EV-202
NCT04225117
HR+/HER2- breast cancer, Triple-negative breast cancer,
Squamous NSCLC, Non-
squamous NSCLC, Head and neck cancer,
Gastric adenocarcinoma or esophageal adenocarcinoma or GEJ
adenocarcinoma, Esophageal squamous cell carcinoma; EV mono
Head and neck squamous cell carcinoma; EV + Pembro
n=320
Enrollment completed for EV mono cohorts.
Initial topline results obtained in Jun 2022
For urothelial cancer
For other solid tumors
(Red: Updates since the last financial results announcement)
ENFORTUMAB VEDOTIN (EV) (2/4): CLINICAL STUDIES
mUC: Metastatic urothelial cancer, mono: Monotherapy, Chemo: Chemotherapy, (s)BLA: (Supplemental) Biologics License Application, Pembro: Pembrolizumab, sNDA: Supplemental
New Drug Application, MIBC: Muscle-invasive bladder cancer, Cis: Cisplatin, RC: Radical cystectomy, FSFT: First subject first treatment, NMIBC: Non-muscle-invasive bladder cancer,
BCG: Bacillus Calmette-Guerin, HR+: Hormone receptor positive, HER2-: HER2 negative, NSCLC: Non-small cell lung cancer, GEJ: Gastroesophageal junction
34
Disease
stage
MIBC mUC
Surgery eligible Previously untreated (first line) PD-1/L1 inhibitor pretreated
Cis-
eligible
Cis-
ineligible
Platinum eligible Cis-ineligible
Platinum naïve
& Cis
-ineligible
Platinum pretreated
Study phase Phase 3 Phase 3 Phase 3 Phase 1b/2 Phase 1b/2 Phase 2 Phase 2 Phase 3
Study No.
KN-B15
/ EV-304
KN-905
/ EV-303
EV-302
EV-103
Cohort K
EV-103
Cohort A
& Others
EV-201
Cohort 2
EV-201
Cohort 1
EV-301
No. of subjects 784 (2 arms) 857 (3 arms) 990 (2 arms) 76 73 45 89 125 608 (2 arms)
EV regimen
Combo w/
Pembro
(perioperative)
Combo w/
Pembro
(perioperative)
Combo w/ Pembro
Combo w/
Pembro
Mono
Combo w/
Pembro
Mono Mono Mono
Control
Chemo
(neoadjuvant)
SoC Chemo n/a n/a n/a n/a n/a Chemo
Primary
endpoint
pCR
&
EFS
pCR
&
EFS
PFS: HR 0.45
OS: HR 0.47
ORR
64%
(CR 11%)
ORR
45%
(CR 4%)
ORR
73% **
(CR 16% **)
ORR
51% **
(CR 22% **)
ORR
44%
(CR 12%)
OS
HR 0.70 *
OS (Ongoing) (Ongoing)
HR 0.47
(31.5 mos
vs.16.1 mos)
(Ongoing)
(21.7 mos)
(26.1 mos **)
(14.7 mos)
(12.4 mos **)
HR 0.70 *
(12.9 mos vs.9.0 mos)
PFS (Ongoing) (Ongoing)
HR 0.45
(12.5 mos
vs.6.3 mos)
(Ongoing)
(8.2 mos)
(12.7 mos **)
(5.8 mos)
(5.8 mos)
HR 0.62 *
(5.6 mos vs.3.7 mos)
ORR (Ongoing) (Ongoing)
67.7%
vs. 44.4%
(CR 29.1% vs. 12.5%)
64%
(CR 11%)
45%
(CR 4%)
73% **
(CR 16% **)
52%
(CR 20%)
44%
(CR 12%)
41%
vs.18% *
(CR 4.9%
vs.2.7%)
DoR (Ongoing) (Ongoing) (Ongoing) (Ongoing) 13.2 mos 22.1 mos ** 13.8 mos ** 7.6 mos
7.4 mos
vs. 8.1 mos *
: Data obtained, *: Prespecified interim analysis, **: Updated data
Early stage Late stage
(m)UC: (Metastatic) urothelial cancer, MIBC: Muscle-invasive bladder cancer, cis: Cisplatin, Pembro: Pembrolizumab, mono: Monotherapy, Chemo: Chemotherapy,
pCR: Pathologic complete response, EFS: Event-free survival, ORR: Objective response rate, CR: Complete response, OS: Overall survival, HR: Hazard ratio,
PFS: Progression-free survival, DoR: Duration of response
ENFORTUMAB VEDOTIN (EV) (3/4):
STUDY DATA BY DISEASE STAGE OF UC
35
The most significant growth driver is 1L mUC indication, which is expected to account for more than half of total sales
in the future
Success in NMIBC and other solid tumors will provide further growth potential
Patient segment
Pivotal study
(EV regimen)
Target filing
timing
Number of
eligible patients*
MIBC
Cis-ineligible
EV-303
(combo w/ Pembro)
FY2025 or later 10,000
Cis-eligible
EV-304
(combo w/ Pembro)
FY2025 or later 37,000
1L mUC
EV-302
EV-103 Cohorts
[Phase 1b/2 for AA in US]
(combo w/ Pembro)
Approved
Approved
[AA in US]
76,000
(incl. US,
Cis-ineligible:
8,000-9,000)
2L+
mUC
PD-1/L1
inhibitor
pretreated &
Cis-ineligible
EV-201 Cohort 2
(monotherapy)
Approved
1,600
(US, Cis-ineligible)
Platinum &
PD-1/L1
inhibitor
pretreated
EV-301
EV-201 Cohort 1
[Phase 2 for AA in US]
(monotherapy)
Approved 38,000
Patient segment
Study
(EV regimen)
NMIBC
High-risk
BCG-unresponsive
EV-104 [Phase 1]
(monotherapy,
intravesical)
Other solid tumors
EV-202 [Phase 2]
(monotherapy* /
combo w/ Pembro**)
<Already approved / pivotal phase> (Included in potential peak sales) <Early clinical phase> (Not included in potential peak sales)
*Monotherapy:
HR+/HER2- breast cancer,
Triple-negative breast cancer,
Squamous NSCLC,
Non-squamous NSCLC,
Head and neck cancer,
Gastric adenocarcinoma or esophageal adenocarcinoma or
GEJ adenocarcinoma,
Esophageal squamous cell carcinoma
**Combo w/ Pembro:
Head and neck squamous cell carcinoma
*Global, based on internal estimates
mUC: Metastatic urothelial cancer, MIBC: Muscle-invasive bladder, 1L: First line, 2L+: Second or later line, Cis: Cisplatin, Pembro: Pembrolizumab,
AA: Accelerated Approval, HR+: Hormone receptor positive, HER2-: HER2 negative, NSCLC: Non-small cell lung cancer, GEJ: Gastroesophageal junction
ENFORTUMAB VEDOTIN (EV) (4/4): FUTURE OUTLOOK
(Red: Updates since the last financial results announcement)
36
FLT3 mut+
AML
Low-intensity
chemo
Chemo
consolidation
Salvage therapy
Transplant
ADMIRAL
VICEROY
Maintenance
GOSSAMER
High-
intensity
induction
chemo
Maintenance
MORPHO
PASHA (HOVON)
PrE0905 (PrECOG)
Launched
China
• R/R AML: Conditional approval obtained in Jan 2021, based on ADMIRAL study data (full approval contingent on
COMMODORE study data) and launched in Apr 2021. Phase 3 COMMODORE study (including China and other countries)
stopped due to efficacy based on the planned interim analysis
FLT3 mut+: FLT3 mutation positive, AML: Acute myeloid leukemia, HIC: High-intensity chemotherapy, FSFT: First subject first treatment, HSCT: Hematopoietic stem cell transplant,
HOVON: The Haemato Oncology Foundation for Adults in the Netherlands, BMT-CTN: Blood and Marrow Transplant - Clinical Trial Network, R/R: Relapsed or refractory
GILTERITINIB: FLT3 INHIBITOR
Relapsed or refractory P3: ADMIRAL
NCT02421939
Monotherapy vs. salvage chemo
(2:1)
n=371 Launched in US, JP, and Europe
Newly diagnosed
(HIC-eligible)
P3: PASHA (HOVON)
NCT04027309
Combo with high intensity chemo
gilteritinib vs. midostaurin (1:1)
n=766 Enrollment completed (Sponsor: HOVON)
P2: PrE0905 (PrECOG)
NCT03836209
n=179 Enrollment completed (Sponsor: PrECOG, LLC.)
Post-HSCT maintenance P3: MORPHO
NCT02997202
Monotherapy vs. placebo (1:1) n=356 Development based on MORPHO study discontinued
Post-chemo maintenance P2: GOSSAMER
NCT02927262
Monotherapy vs. placebo (2:1) n=98 Topline results obtained in Aug 2021
Newly diagnosed
(HIC-ineligible)
P1: VICEROY
NCT05520567
Combo with venetoclax and
azacitidine
n=70 FSFT in Jan 2023
(Red: Updates since the last financial results announcement)
37
Gastric and GEJ adenocarcinoma
Target patient population:
HER2-, Claudin 18.2+ locally advanced and metastatic
gastric and GEJ adenocarcinoma
Metastatic gastric cancer is an area of significant unmet
need, especially in advanced stages with ~6% five-year
survival rate at Stage IV and treatment options are
limited
Target: Claudin 18.2
Claudin is a major structural component of tight junctions
and seals intercellular space in epithelial sheets
Broadly expressed in various cancer types
Prevalence of patients with high expression of
Claudin 18.2 is substantial: 38%
~60% of primary pancreatic adenocarcinomas;
~20% of these meet the eligibility criteria for the
ongoing Phase 2 study
Gastric and GEJ
adenocarcinoma
P3: SPOTLIGHT
NCT03504397
First line, Combo with mFOLFOX6, DB, vs. placebo n=566
NDA accepted in Japan in Jun 2023. BLA/MAA
accepted in Europe and China in Jul 2023. Received
complete response letter in US in Jan 2024
P3: GLOW
NCT03653507
First line, Combo with CAPOX, DB, vs. placebo n=507
P2: ILUSTRO
NCT03505320
Cohort 1: Third or later line, zolbetuximab monotherapy
Cohort 2: First line, Combo with mFOLFOX6
Cohort 3: Third or later line, Combo with pembrolizumab
Cohort 4: First line, Combo with mFOLFOX6 and nivolumab
Cohort 5: Perioperative, Combo with FLOT
n=143 FSFT: Sep 2018
Pancreatic
adenocarcinoma
P2
NCT03816163
First line, Combo with nab-paclitaxel and gemcitabine, open n=369 FSFT: May 2019
GEJ: Gastroesophageal junction, HER2-: HER2 negative, Claudin 18.2+: Claudin 18.2 positive, mFOLFOX6: 5-FU, leucovorin and oxaliplatin, DB: Double-blind,
CAPOX: Capecitabine and oxaliplatin, NDA: New Drug Application, BLA: Biologics License Application, MAA: Marketing Authorization Application,
FLOT: Fluorouracil, leucovorin, oxaliplatin and docetaxel, FSFT: First subject first treatment
ZOLBETUXIMAB: ANTI-CLAUDIN 18.2 MONOCLONAL ANTIBODY
(Red: Updates since the last financial results announcement)
38
P3: MOONLIGHT 1
NCT04234204
Moderate to severe VMS associated with menopause;
The first 12 weeks: DB, 30 mg vs. placebo (1:1)
The last 12 weeks: Active extension treatment period, 30 mg
n=302
Primary endpoints not met
(12w DB period topline results)
P3: MOONLIGHT 3
NCT04451226
VMS associated with menopause; open label, 30 mg for 52 weeks n=150 Topline results obtained in Sep 2022
VMS has a significant negative impact on QoL
Physical symptoms include hot flashes and night sweats, which can
impact sleep
.
Physical symptoms may lead to emotional impact including
embarrassment, irritability, anxiety, and sadness
Symptoms have a negative impact on multiple aspects of everyday life
1
Women’s Health Initiative (WHI) Study
2
Initial data analyses showed an association between chronic HRT use and
increased risk of cardiovascular disease and breast cancer
Since WHI’s findings, use of HRT has dropped
Although subsequent analysis of the WHI data have demonstrated that HRT is safe
and effective when initiated in the appropriate patient in the appropriate manner
(i.e. right time, formulation, dose and duration), prescriptions have not rebounded,
leaving some women with minimal options to satisfactorily manage their VMS
US and Europe
China
Japan
P3: STARLIGHT 2
NCT06206408
Mild to severe VMS associated with menopause;
12 weeks: DB, 2 doses vs. placebo (1:1:1)
n=390
Under preparation to start in Q4 FY2023
P3: STARLIGHT 3
NCT06206421
VMS associated with menopause;
52 weeks: DB, vs. placebo (1:1)
n=260
Under preparation to start in Q4 FY2023
P3: SKYLIGHT 1
NCT04003155
Moderate to severe VMS associated with menopause;
The first 12 weeks: DB, 30 mg and 45 mg vs. placebo (1:1:1)
The last 40 weeks: Active extension treatment period, 30 mg or 45 mg
n=527
Approved in US in May 2023.
Approved in Europe in Dec 2023
P3: SKYLIGHT 2
NCT04003142
n=501
P3: SKYLIGHT 4
NCT04003389
VMS associated with menopause;
52 weeks: DB, 30 mg and 45 mg vs. placebo (1:1:1)
n=1,831
P3b: DAYLIGHT
NCT05033886
Moderate to severe VMS associated with menopause, unsuitable for HRT;
24 weeks, DB, 45 mg vs. placebo (1:1)
n=453 Topline results obtained in Jun 2023
(Red: Updates since the last financial results announcement)
1: DelveInsight, Epidemiology Forecast, Jun 2018. 2: Data Source - IMS NPA (2000-2016), IMS NSP (2000-2016). (3 HTs and SSRI) NAMS 2015 Position Statement.
VMS: Vasomotor symptoms, QoL: Quality of life, HRT: Hormone replacement therapy, DB: Double-blind
FEZOLINETANT: NK3 RECEPTOR ANTAGONIST
39
Characteristics of ACP
Pegylated RNA aptamer (Chemically synthesized)
ACP inhibits complement C5, and slows inflammation and cell
death associated with development and progression of GA
GA secondary to AMD
P2/3: GATHER1
NCT02686658
Part 1: 1 mg, 2 mg vs. Sham (n=77)
Part 2: 2 mg, 4 mg vs. Sham (n=209)
n=286
MAA accepted in Europe in Aug 2023.
sNDA for label update submitted in
US in Jan 2024
P3: GATHER2
NCT04435366
2 mg vs. Sham n=448
Stargardt disease P2b
NCT03364153
vs. Sham n=120 FSFT: Jan 2018
Geographic atrophy (GA)
Advanced form of dry age-related macular degeneration
(AMD)
Globally, approximately 5 million people are estimated to
have GA at least in one eye
1
Approximately 75% of people living with GA in the US are
believed to be undiagnosed
2
Without timely treatment, an estimated 66% of people with
GA may become blind or severely visually impaired
3
AVACINCAPTAD PEGOL (ACP):
COMPLEMENT C5 INHIBITOR / PEGYLATED RNA APTAMER
(Red: Updates since the last financial results announcement)
1. Retina 37:819-835 (2017). 2. IQVIA Medical Claims (DX) data Jan ’20-Dec ’21: 24 Months. 3. JAMA Ophthalmol 139:743-750 (2021)
MAA: Marketing Authorization Application, sNDA: Supplemental New Drug Application, FSFT: First subject first treatment
40
FOCUS AREA APPROACH: KEY EVENTS EXPECTED IN FY2023
Primary Focus IND
Phase 1
Early data readout* Dosing resumption
Genetic Regulation
1 project AT845
Immuno-Oncology
2 projects
( ASP1012)
ASP1570
ASP2138
Blindness &
Regeneration
ASP7317
Targeted Protein
Degradation
1 project
(pan-KRAS)
ASP3082
Expecting Phase 1 entry in 4 projects and several progress in Phase 1 studies toward PoC judgment
*Dose escalation/monotherapy
PoC: Proof of concept, IND: Investigational New Drug
: Achieved
ON THE FOREFRONT OF
HEALTHCARE CHANGE
