Outline of Consolidated Financial Results
for the Year Ended March 31, 2024
May 14, 2024
NIPPON SHINYAKU CO., LTD.

FY2023 RESULTS AND
FY2024 FULL-YEAR FORECASTS
Toru Nakai
Representative Director, President

2

FY2023 Summary（Million yen）

FY2022

FY2023

YoY Change

Results

Ratio

Results

Ratio

Amt

%

Revenue

144,175

100.0%

148,255

100.0%+4,080+2.8%(Pharmaceuticals)(121,988)(84.6%)(125,105)(84.4%)(+3,116)(+2.6%)(Functional Food)(22,187)(15.4%)(23,150)(15.6%)(+963)(+4.3%)

Cost of sales

55,98038.8%

50,23433.9%-5,746-10.3%

SG&A expenses

34,812

24.1%

34,959

23.6%

+146

+0.4%

R&D expenses

24,135

16.7%

31,676

21.4%+7,541+31.2%

Other income

1,908

1.3%

3,163

2.1%

+1,254+65.7%(Foreign exchange gain)(1,193)(0.8%)(2,486)(1.7%)(+1,292)(+108.4%)

Other expenses

1,106

0.8%

1,252

0.8%

+146

+13.2%

Operating profit

30,04920.8%

33,29522.5%+3,245+10.8%

Finance income

575

0.4%

650

0.4%

+75

+13.1%

Finance costs

136

0.1%

329

0.2%

+193

+142.4%

Profit before tax

30,48921.1%

33,61622.7%+3,127+10.3%

Income tax expense, etc

7,676

5.3%

7,765

5.2%

+88

+1.2%

22,81215.8%

25,851

17.4%+3,038+13.3%

Profit attributableto owners of parent

3

Segmental Review - Pharmaceuticals Ethical Drugs 76,141 million yen(- 5,612 million yen, - 6.9%, YoY)

（Million yen）

 Sales growth of Uptravi, Viltepso, etc.
 Decrease in sales of Vidaza, Tramal/Onetram, etc.
 Impact of NHI price revision

121,988- 5,612

Revenues from thelicensing of industrial
property rights+9,590

Revenues from the industrial property rights
40,304 million yen(＋9,590 million yen, ＋31.2%, YoY) Royalty revenue growth due to overseas sales ofUptravi- 861

125,105

Profit in co-promotion 8,658 million yen( - 861 million yen, - 9.0%, YoY)
 Sales growth of Opsumit and Erleada End of co-promotion of Zytiga

4

Sales Trends of Viltepso® (viltolarsen)（Million yen）

Viltepso

FY2022

FY2023Results

Results

YoY Change
Amt

%

Japan

4,139

4,407

+268

+6.5%

✓The number of patients currently being administered is more than two-thirds ofthe peak number of 128 patients in the data from Chuikyo (Central Social
Insurance Medical Council) .

U.S.

10,201

13,123+2,922+28.6%✓The number of patients receiving and wishing to receive Viltepso is increasing.
✓The results of P2 trial (Galactic53 trial) were presented as a poster presentation
at 2024 MDA conference.

total

14,341

17,530+3,189+22.2%FY2022

FY2023

Actual rate

Actual rate

135.5yen

144.6yenExchange rate
1USD

Japan

（Million yen）

U.S.

5

Segmental Review - Functional Food Protein preparations 15,600 million yen(+216 million yen, +1.4%, YoY)
 Steady sales growth in protein preparations due to
end of pandemic impact
 Increase in selling prices due to higher raw material
prices（Million yen）

22,187+216

Preservatives 3,105 million yen(+199 million yen, +6.9%, YoY)

+199

+477

+129

- 60

23,150 Focus on new customer acquisition and extendeduse
Supplements 1,905 million yen(+477 million yen, +33.4%, YoY)
 Sales growth of WINZONE Protein and otherproducts due to increased sporting events
nationwide

Health food ingredients 1,248 million yen(+129 million yen, +11.6%, YoY)

6

Operating Profit（Million yen）
30,049+4,080+5,746- 146- 7,541+1,254- 146

33,295

Cost of sales 50,234 million yen(- 5,746 million yen, -10.3%, YoY)
The ratio was improved by 4.9 points YoY.
 Negative impact of NHI price revision Cost of sales ratio improvement due to factors
such as revenues from industrial property rights
and the change in sales segment mix (pharma vs.
food)
SG&A expenses 34,959 million yen(+146 million yen, +0.4%, YoY)
 Increase in labor costs
 Decrease in commission for promotional
activities of Tramal/Onetram
R&D expenses 31,676 million yen(+7,541 million yen, +31.2%, YoY)
 Increase in contract research expenses (nucleic
acid drugs, etc.)
Other income 3,163 million yen(+1,254 million yen, +65.8%, YoY) Foreign exchange gains (+1,292 million yen)7

Business Forecast for FY2024 (consolidated)(Million Yen）

FY2023

FY2024

YoY Change

Results

Ratio

Forecast

Ratio

Amt

%

Revenue

148,255

100.0%

150,000

100.0%+1,745+1.2%(Pharmaceuticals)(125,105)(84.4%)(128,500)(85.7%)(+3,395)(+2.7%)(Functional Food)(23,150)(15.6%)(21,500)(14.3%)(-1,650)(-7.1%)

Cost of sales

50,23433.9%

48,90032.6%-1,334-2.7%

SG&A expenses

34,95923.6%

38,70025.8%+3,741+10.7%

R&D expenses

31,67621.4%

31,500

21.0%

-176

-0.6%

Other income

3,163

2.1%

500

0.3%

-2,663-84.2%

Other expenses

1,252

0.8%

400

0.3%

-852

-68.1%

Operating profit

33,29522.5%

31,00020.7%-2,295-6.9%

Finance income

650

0.4%

600

0.4%

-50

-7.8%

Finance costs

329

0.2%

100

0.1%

-229

-69.7%

Profit before tax

33,61622.7%

31,50021.0%-2,116-6.3%

Income tax expense, etc

7,765

5.2%

7,000

4.7%

-765

-9.9%

25,851

17.4%

24,50016.3%-1,351-5.2%

Profit attributableto owners of parent

Exchange rate

FY2023
Actual rate

FY2024
Forecast rate

1USD

144.6yen

140.0yen8

Segmental Forecast - Pharmaceuticals （Million yen）

FY2023

FY2024

Results

Ratio

Ethical drugs

76,141

60.9%

Revenue from the licensing ofindustrial property rights

40,304

Profit in co-promotion
Revenue

Forecast

YoY Change
Ratio

Amt

%

77,70060.5%+1,559

+2.0%

32.2%

41,70032.4%+1,396

+3.5%

8,658

6.9%

9,100

7.1%

+442

+5.1%

125,105

100.0%

128,500

100.0%+3,395+2.7%

Despite the impacts from NHI price revision and the competitions with generics,
we expect an increase in revenues mainly due to the growth of Viltepso and Uptravi
as well as growth in royalty income associated with overseas sales of Uptravi.

9Segmental Forecast - Functional Food FY2023（Million yen）

FY2024

Results

Ratio

Protein preparations

15,600

67.4%

Preservatives

3,105

Supplements

Forecast

YoY Change
Ratio

Amt

%

13,00060.5%-2,600-16.7%

13.4%

3,200

14.9%

+95

+3.0%

1,905

8.2%

3,100

14.4%

+1,195+62.7%

Health food ingredients

1,248

5.4%

1,100

5.1%

-148

-11.9%

Others

1,291

5.6%

1,100

5.1%

-191

-14.8%

Revenue

23,150100.0%

21,500100.0%-1,650-7.1%

Although we will further focus on development and introduction of
new products and strengthen sales efforts in marketed products,
a decrease in sales is expected
due to the impact from declining prices of some products.

10Dividends Forecast
FY2023

FY2024

Interim¥62¥62

Annual¥124¥124

Basic earnings per share¥383.82¥363.76

Payout ratio (consolidated)

32.3 ％

34.1 ％

Dividends per share(Yen)

AnnualInterim
FY2020

FY2021

FY2022

FY2023

FY2024(Estimated)11

R&D PIPELINE
Kazuchika Takagaki
Director, Research & Development

12R&D Updates（1/2）

For updates since February 9, 2024,
see highlighted text in red.

Recent status/event

Code No.
(Generic name)Product
nameIndications and topics

Schedule

LaunchZX008（fenfluramine）

Fintepla

Lennox-Gastaut syndrome (additional indication)

Approved in March 2024

Approval

NS-87（daunorubicin /cytarabine）

Vyxeoshigh-risk acute myeloid leukemia

Approved in March 2024
To be launched after
NHI drug price listing

Uptravi

pediatric pulmonary arterial hypertension

April 2024

Fintepla

CDKL5 deficiency

July 2023

Gazyvasystemic lupus erythematosus without nephropathy

October 2023

ー

Duchenne muscular dystrophy

February 2024

NS-863ーcardiovascular diseases

August 2023

idiopathic pulmonary fibrosis

In application

Start of P3

Start of P2
Start of P1

NS-304（selexipag）
ZX008（fenfluramine）
GA101（obinutuzumab）
NS-089/NCNP-02（brogidirsen）

In-license(Vicore Pharma, Sweden)
Alliance Agreement(Eli Lilly Japan)

C21ー

LY3527727(piltobrutinib)ーTemporarily suspended

NS-580ー

NS-018(ilginatinib)ーmyelofibrosisーNS-161ーinflammatory diseaseーTerminatedmantle cell lymphoma (MCL) /chronic lymphocytic leukemia (CLL)
endometriosis chronic prostatitis /chronic pelvic pain syndrome

Contract signed
in February 2024
Contract signed
in March 2024
ー13

R&D Updates（2/2）
Recent status/event
Conference Presentations

Manuscript
Rare Pediatric Disease
Designation
Breakthrough Therapy
Designation

Code No.
(Generic name)
NS-065/NCNP-01(viltolarsen)
NS-065/NCNP-01(viltolarsen)
NS-089/NCNP-02（brogidirsen）

Product
nameViltepso
Viltepsoー

For updates since February 9, 2024,
see highlighted text in red.

Indications and topics
Phase 2 trial (Galactic53): 2024 Muscular Dystrophy
Association Clinical & Scientific ConferenceLong-term study analysis data (after 4 years oftreatment):Journal of Neuromuscular Diseases
Non-clinical data: (Molecular Therapy Nucleic Acids)

Schedule
March 2024
May 2023
October 2023
June 2023 (U.S.)

NS-089/NCNP-02（brogidirsen）

July 2023 (U.S.)ー

Duchenne muscular dystrophy
July 2023 (U.S.)

Orphan Drug Designation
December 2023 (EU)Orphan Drug Designation

NS-401(tagraxofusp)ーblastic plasmacytoid dendritic cell neoplasm

August 2023 (Japan)

Orphan Drug Designation

NS-229ーeosinophilic granulomatosis with polyangiitis

January 2024 (EU)ーa joint research agreement with the aim of creating
nucleic acid medicines that are expected to be applied
to an intractable and rare disease in the CNS field

April 2024

Alliance(MiNA Therapeutics)ー14Vyxeos® (NS-87)
Combination for I.V. Injection for the Treatment of High-risk AML

15

Characteristics of Vyxeos® (NS-87)
 Liposomal formulation of cytarabine and daunorubicin in a 5:1 molar ratio It reaches the bone marrow as liposomes and remains for a long time.
 After the drug is taken up into leukemic cells in the bone-marrow,cytarabine and daunorubicin are released to exert their antitumor effects.

Reference: JAZZ Pharmaceuticals website“Mechanism of Delivery”
https://vyxeospro.com/mechanism-of-delivery

16

Time and Duration of Vyxeos® (NS-87)
NS-87 vs 7+3 therapy (overseas P3 study)

JAZZ-HP VYXEOS preparation and administration
https://vyxeospro.com/dosing-ordering/preparation17

Vyxeos® (NS-87) Overseas P3 study
Subjects: Untreated high-risk AML 309 aged 60-75 years
Control group: 7 + 3 therapy (AraC 100 mg/m2 + DNR 60 mg/m2)[Primary: overall survival(OS)]NS-87(n=153)7+3 therapy(n=156)overall survival(median)

9.56 months

5.95 months

1-year survival
rates

41.5 %

27.6 %

Hazard ratio

0.69
Log rank p=0.003

NS-87
7 + 3 therapy

Reference: J Clin Oncol. 2018 Sep 10;36(26):2684-2692

18

REFERENCE MATERIALS

19

Sales By Product in Pharmaceutical Segment（Million yen）

FY2023Results

YoY Change

FY2024
Forecast

14,341

17,530+22.2%

20,100(4,139)(4,407)(+6.5%)(4,600)(10,201)(13,123)(+28.6%)(15,500)

Uptravi

pulmonary arterial hypertension/
chronic thromboembolic pulmonary
hypertension

10,543

12,918+22.5%

15,400Vidazamyelodysplastic syndrome/acute myeloid leukemia

15,951

10,383- 34.9%

4,800

GazyvaCD20-positive follicular lymphoma/
CD20-positive chronic lymphocytic leukemia

4,904

4,695

- 4.3%

5,100

Tramal/Onetram

cancer pain, chronic pain

5,358

3,927

- 26.7%2,700

Cialiserectile dysfunction

2,938

2,499

- 15.0%2,700

Erizasallergic rhinitis

2,640

2,284

- 13.5%2,100

Zalutia

urinary disorder caused by benign prostatic
hyperplasia

2,826

2,256

- 20.2%

1,600

Adcircapulmonary arterial hypertension

2,649

2,255

- 14.9%1,700

Defitelio

sinusoidal obstruction syndrome

2,524

2,221

- 12.0%2,300

Profit in co-promotion

9,520

8,658

- 9.0%

9,100

Revenues from the licensing of industrial property rights

30,71440,304+31.2%

41,700

Revenue

121,988

125,105+2.6%

128,500

Brand nameIndications

Viltepso(Japan)Duchenne muscular dystrophy(U.S.)FY2022
Results

20

Sales by Product Group in Functional Food Segment（Million yen）

FY2022

FY2023

YoY Change

FY2024

Results

Ratio

Results

Ratio

Amt

%

Forecast

Protein preparations

15,383

69.3%

15,600

67.4%

+216

+1.4%

13,000Preservatives

2,905

13.1%

3,105

13.4%

+199

+6.9%

3,200

Supplements

1,428

6.4%

1,905

8.2%

+477

+33.4%

3,100

Health food ingredients

1,118

5.0%

1,248

5.4%

+129

+11.6%

1,100

Others

1,351

6.1%

1,291

5.6%

-60

-4.5%

1,100

Revenue

22,187100.0%

23,150100.0%

+963

+4.3%

21,50021

Consolidated Balance Sheet
End of

End ofChangeEnd of

End ofChangeFY2022

FY2023Amt

FY2022

FY2023Amt

237,451

263,404+25,952

Current assets

157,873

164,285+6,411

Non-current assets

79,578

99,119+19,541（Million yen）

AssetsTotal aseets

237,451

263,404+25,952

Liabilities

41,51842,870+1,352Current liabilities

35,18337,336+2,153

Non-current liabilities

6,334

5,533

-801

Equity195,933

220,534+24,600

Total liabilities
and equity

237,451

263,404+25,952

Liabilities and Shareholders' Equity

AssetsInventories+ 2,752

Trade and other payables

Trade and other receivables+ 2,551

Liabilities for retirement benefits

Other financial assets (non-current)+ 10,553+4,627- 805

22

Consolidated Statements of Cash Flows（Million yen）

FY2022

FY2023

YoY Change

Results

Results

Amt

Operating activities

26,17016,289-9,880Investing activities-17,631-9,921

7,710

Financing activities-9,605-9,719-113

Cash and cash equivalents
at end of period

60,04758,094-1,953+26,170+16,289- 17,631- 9,92160,566- 9,605
60,047- 9,719

58,094（Million yen）
Beginning of FY2022

End of FY2022 /Beginning of FY2023

End of FY2023

23

Pipeline（1/2）
Stage

Code No.
(Generic name)

OriginApplication
type

Indications

Schedule

Country

LaunchP3

NS-065/NCNP-01

In-house

NME

Duchenne muscular dystrophy

Around the spring of FY2024
P3 data presentation

Japan/U.S.

Preparingfor launch

NS-87（daunorubicin /cytarabine）

In-houseNew
combination

high-risk acute myeloid
leukemia

Approval ：March in 2024

Japan

NDA filing

NS-304(selexipag)In-house

New dosepediatric pulmonary arterial
hypertension

Study Completion：FY 2024
Application：April in 2024

Japan

NDA filing
P3

LY3527727(pirtobrutinib)

Alliance
agreement

NMEmantle cell lymphoma

￣

Japan

ZX008
(fenfluraminehydrochloride)

Distribution
partnershipNewindication

CDKL5 deficiency disorderStudy Completion：FY2026

Japan

lupus nephritis

Projected submission：2026

Japan

pediatric nephrotic syndrome

Projected submission：2026

Japan

extra renal lupus

Projected submission：
2027 and beyond

Japan

P3

GA101
(obinutuzumab)

In-licenseNew
indication

CAP-1002

Partnership

NME

Duchenne muscular dystrophy

Topline data：end of 2024

U.S.

LY3527727(pirtobrutinib)

Alliance
agreement

NMEchronic lymphocytic leukemia

￣

Japan

24

Pipeline（2/2）
Stage

P2

Code No.
(Generic name)

OriginApplication
type

Indications

Schedule

Country

NS-304(selexipag)In-houseNew
indicationarteriosclerosis obliterans

Study Completion：FY2024

Japan

endometriosis

Temporarily suspendedJapan

chronic prostatitis/
chronic pelvic pain syndrome

Temporarily suspendedJapan

NS-580In-house

NME

NS-089/NCNP-02（brogidirsen）

In-house

NME

Duchenne muscular dystrophy

Study Completion：FY2025
FPI：February in 2024

Japan/U.S.

Preparingfor P2

NS-229In-house

NME

eosinophilic granulomatosis
with polyangiitis

Study Completion：FY2025

Japan/U.S.

P1/2

NS-401(tagraxofusp)

In-license

NME

blastic plasmacytoid dendritic
cell neoplasm

Study Completion：FY2026

Japan

Preparingfor P1/2

NS-050/NCNP-03

In-house

NME

Duchenne muscular dystrophy

Study Completion：FY2027
FPI：1H of FY2024（estimated）

Japan/U.S.

NS-917(radgocitabine)

In-license

NME

relapsed/refractory acute
myeloid leukemia

Study Completion：FY2024

Japan

NS-025In-house

NME

urological diseases

Study Completion：FY2024

Japan

NS-863In-house

NME

cardiovascular diseases

Study Completion：FY2024

Japan

P1

25

NS-065/NCNP-01 (viltolarsen)- Treatment for Duchenne muscular dystrophy Development Phase

・Japan : Launch・U.S.
: Launch・Global : P3 in progress

OriginCo-development :National Center of Neurology and Psychiatry

Development

Nippon Shinyaku

Mechanism of action

Exon 53 Skipping

Indication

Duchenne muscular dystrophy

Dosage form

Injection

Feature・Improvement in symptoms and prevention of the diseaseprogression by recovery of dystrophin protein expression・Morpholino based oligonucleotide with possible highsafety profile and maximized activity

26

NS-87 (daunorubicin / cytarabine)- Treatment for high-risk acute myeloid leukemia Development Phase

Japan : Approval

Origin[Mar. 2017]
Licensed-in from: Jazz Pharmaceuticals plc

Development

Nippon Shinyaku

Mechanism of action

Liposomal combination of daunorubicin and cytarabine

Indication

high-risk acute myeloid leukemia (high-risk AML)

Dosage form

Injection

Feature・NS-87 is the first therapy for the treatment ofhigh-risk AML in Japan.
・Accumulation of NS-87 in the bone marrow enhance
antitumor activity and reduces adverse events.

27

ZX008 (fenfluramine hydrochloride)- Treatment for rare intractable epilepsy Development Phase

Japan : Launch (Dravet syndrome)
Japan : Launch（Lennox-Gastaut syndrome)
Japan：P3 (CDKL5 deficiency disorder)

Origin[Mar. 2019] Distribution partnership in Japan : UCB S.A. (former
Zogenix, Inc.)

Development

UCB S.A. (former Zogenix, Inc.)

Mechanism of action

5-HT (serotonin) releaser with agonist activity at several 5-HT
receptors

Indication

Dravet syndrome
Lennox-Gastaut syndrome
CDKL5 deficiency disorder

Dosage form

Oral liquid agent

Feature・Effective for Dravet syndrome, Lennox-Gastaut syndrome and
CDKL5 deficiency disorder patients refractory to existingtreatment options
・ZX008 can be used in combination with other drugs, asstandard of care for intractable epilepsy based on combination
therapy.

28

LY3527727(pirtobrutinib)- Treatment for Mantle cell lymphoma, Chronic lymphocytic leukemia Development Phase

NDA filing, P3

Origin[Mar. 2024] Alliance agreement in Japan : Eli Lilly Japan

Development

Eli Lilly Japan

Mechanism of action

A reversible non-covalent BTK inhibitor

Indication

mantle cell lymphoma (MCL)
chronic lymphocytic leukemia (CLL)

Dosage form

Oral agent

Feature・A highly selective, non-covalent (reversible) inhibitor of the
enzyme Bruton’s tyrosine kinase (BTK), with having a novel binding
mechanism.

29

CAP-1002- Treatment for Duchenne muscular dystrophy Development Phase

U.S. : P3

Origin[Jan. 2022] Partnership for commercialization in U.S.
[Feb. 2023] Partnership for commercialization in Japan: Capricor Therapeutics, Inc.

Development

Capricor Therapeutics, Inc.

Mechanism of action

Exosomes released from cardiosphere-derived cells

Indication

Duchenne muscular dystrophy

Dosage form

Injection

Feature・Exosomes released from this drug are expected to reduce oxidative
stress, inflammation, fibrosis, and increase cell energy and myocyte
generation, resulting in improvement of motor and cardiac
functions.
• Its broad applicability makes it suitable for patients regardless ofthe type of genetic mutation.

30

GA101 (obinutuzumab)- Treatment for lupus nephritis, pediatric nephrotic syndrome, extra renal lupus -Development Phase

Japan：P3（LN）
Global : P3（PNS）
Japan：P3（ERL）

Origin[Nov. 2012]
Licensed-in from : Chugai Pharmaceutical Co., Ltd.

Development

Co-development : Chugai Pharmaceutical Co., Ltd.

Mechanism of action

Anti-CD20 monoclonal antibody

Indication

lupus nephritis (LN)
pediatric nephrotic syndrome (PNS)
extra renal lupus (ERL)

Dosage form

Injection

FeatureAnti-CD20 monoclonal antibody, increased antibody-dependent
cellular cytotoxicity (ADCC) activity and direct cytotoxicity

31

NS-304 (selexipag)- Treatment for Luhritis -- Treatment for pulmonary hypertension, arteriosclerosis obliterans -Development Phase

Japan : P2b（ASO）
Japan : P2, NDA filing (pediatric PAH）

OriginNippon Shinyaku

Development

Nippon Shinyaku（ASO）
Co-development : Janssen Pharmaceutical K.K. (pediatric PAH)

Mechanism of action

Selective IP receptor agonist

Indicationarteriosclerosis obliterans (ASO)
pediatric pulmonary arterial hypertension (pediatric PAH)

Dosage form

TabletFeatureLong-acting oral drug32

NS-580- Treatment for Luhritis -- Treatment for endometriosis, Chronic prostatitis/Chronic pelvic pain syndrome -Development Phase

Japan : P2b (endometriosis)
Japan : P2a (CP/CPPS)

OriginNippon Shinyaku

Development

Nippon Shinyaku

Mechanism of action

Inhibition of membrane-associated prostaglandin E synthase-1

Indication

endometriosis
chronic prostatitis/chronic pelvic pain syndrome (CP/CPPS)

Dosage form

Oral agent

Feature・Treatment for endometriosis without hormonal effectand with possible analgesic potency
・Treatment for CP/CPPS with high safety and long-term
pain control33

NS-089/NCNP-02（brogidirsen）- Treatment for Duchenne muscular dystrophy -Development Phase

Global：P2

OriginCo-development :National Center of Neurology and Psychiatry

Development

Nippon Shinyaku

Mechanism of action

Exon 44 Skipping

Indication

Duchenne muscular dystrophy

Dosage form

Injection

Feature・Improvement in symptoms and prevention of the diseaseprogression by recovery of dystrophin protein expression・Morpholino based oligonucleotide with possible highsafety profile and maximized activity

34

NS-229- Treatment for Eosinophilic granulomatosis with polyangiitis -Development Phase

Global：Preparation for P2

OriginNippon Shinyaku

Development

Nippon Shinyaku

Mechanism of action

JAK1 inhibitor

Indication

eosinophilic granulomatosis with polyangiitis (EGPA)

Dosage form

Oral agent

Feature・Potent and highly selective JAK1 inhibitor
・High efficacy and good safety profiles are expected in
the treatment for EGPA35

NS-401 (tagraxofusp)- Treatment for blastic plasmacytoid dendritic cell neoplasm Development Phase

Japan : P1/2

Origin[Mar. 2021]
Licensed-in from: The Menarini Group

Development

Nippon Shinyaku

Mechanism of action

Induction apoptosis of cells by inhibiting protein synthesis byspecifically targeting cancer cells expressing CD123

Indication

blastic plasmacytoid dendritic cell neoplasm (BPDCN)

Dosage form

Injection

Feature・Composed of diphtheria toxin (DT) fusion protein andrecombinant human IL-3
・Novel targeted therapy directed to CD123 on tumor cells
・IL-3 binds to CD123-expressing tumor cells and delivers
the cytotoxic diphtheria toxin to the cells, resulting in
the blockage of protein synthesis in the cell and causing
cell death in CD123-expressing cells

36NS-050/NCNP-03- Treatment for Duchenne muscular dystrophy Development Phase

Global：Preparation for P1/2

OriginCo-development :National Center of Neurology and Psychiatry

Development

Nippon Shinyaku

Mechanism of action

Exon 50 Skipping

Indication

Duchenne muscular dystrophy

Dosage form

Injection

Feature・Improvement in symptoms and prevention of the diseaseprogression by recovery of dystrophin protein expression・Morpholino based oligonucleotide with possible highsafety profile and maximized activity

37

NS-917 (radgocitabine)- Treatment for relapsed or refractory acute myeloid leukemia Development Phase

Japan : P1

Origin[Mar. 2017]
Licensed-in from : Delta-Fly Pharma, Inc.

Development

Nippon Shinyaku

Mechanism of action

DNA strand-break by incorporating itself into DNA

Indication

relapsed or refractory (r/r) acute myeloid leukemia (AML)

Dosage form

Injection

Feature・Significant anti-leukemic activity with uniquemechanism of action from other nucleoside analogs at
low dose continuous infusion
・Tolerable safety profile available to elderly patients with
r/r AML38

NS-025- Treatment for urological diseases -Development Phase

Japan : P1

OriginNippon Shinyaku

Development

Nippon Shinyaku

Mechanism of action－

Indication

Urological diseases (to be determined)

Dosage form

Oral agent

Feature－39

NS-863- Treatment for cardiovascular diseases Development Phase

Japan : P1

OriginNippon Shinyaku

Development

Nippon Shinyaku

Mechanism of action－

Indication

Cardiovascular diseases (to be determined)

Dosage form

Oral agent

Feature－40

Safe Harbor Statement• Materials and information provided during this presentation may contain so-called “forward-looking statements.” Thesestatements are based on current expectations, forecasts and assumptions that are subject to risks and uncertainties
which could cause actual outcomes and results to differ materially from these statements.
• Risks and uncertainties include general industry and market conditions, and general domestic and international
economic conditions such as interest rate and currency exchange fluctuations. Risks and uncertainties particularly apply
with respect to product-related forward-looking statements. Product risks and uncertainties include, but are not limited
to, technological advances and patents attained by competitors; challenges inherent in new product development,
including completion or failure of clinical trials; claims and concerns about product safety and efficacy; regulatory
agency’s examination, obtaining regulatory approvals; domestic and foreign social security reforms; trends toward
healthcare cost containment; and governmental laws and regulations affecting domestic and foreign operations.

• Also, for products that are approved, there are manufacturing and marketing risks and uncertainties, which include, but
are not limited to, inability to build production capacity to meet demand, unavailability of raw materials, and competition
with others.
• The Company disclaims any intention or obligation to update or revise any forward-looking statements whether as aresult of new information, future events or otherwise.
• This English presentation was translated from the original Japanese version. In the event of any inconsistency between
the Japanese original and the English translation, the former shall prevail.

Nippon Shinyaku Co., Ltd.
IR Meeting (Q4/FY2023)
May 14, 2024

Presentation
Nakai: I am Toru Nakai, President of Nippon Shinyaku, Co., Ltd.
Thank you very much for taking time out of your busy schedule to participate in our FY2023 financial results
briefing today. I appreciate it very much.
Today, I would like to report on our business performance for FY2023 and our full-year business forecast for
FY2024, and Mr. Takagaki will explain the progress of our R&D pipeline.

Please see page three of the slides.
As an overview of our performance in FY2023, we reported consolidated revenue of JPY148,255 million,
operating profit of JPY33,295 million, profit before income taxes of JPY33,616 million, and net profit
attributable to owners of the parent of JPY25,851 million.

Please see page four of the slides.
In the pharmaceuticals business, although sales of products such as Vidaza decreased due to the impact ofdrug price revisions and generic products, sales of Uptravi, a treatment for pulmonary arterial hypertension
and chronic thromboembolic pulmonary hypertension, and Viltepso, a treatment for Duchenne muscular
dystrophy, increased, as well as royalty income from overseas sales of Uptravi.
As a result, consolidated revenue was JPY125,105 million, an increase of 2.6% YoY.

Please see page five of the slides.
Here we show sales of Viltepso, a treatment for Duchenne muscular dystrophy, which is marketed in Japanand the United States.
As for sales results for FY2023, sales in both Japan and the US increased YoY, totaling JPY4,407 million in Japanand JPY13,123 million in the US.

Please see page six of the slides.
In the functional food business, sales of supplements and other products, centering on WINZONE Protein,increased due to changes in the market, including an increase in the number of sporting events held in various
regions as the impact of the new coronavirus infection eased, resulting in consolidated net sales of JPY23,150
million, up 4.3% YoY.

Please see page seven of the slides.
As for operating expenses, the cost of sales ratio improved by 4.9 percentage points YoY to 33.9%, due tofactors such as industrial property revenues and sales mix, despite the impact of the NHI price revision.
SG&A expenses increased 0.4% YoY to JPY34,959 million, mainly due to an increase in personnel expenses,
despite a decrease in commission fees for Tramal's promotional activities.
R&D expenses totaled JPY31,676 million, up 31.2% YoY, mainly due to an increase in contract researchexpenses. As a result, operating profit was JPY33,295 million, up 10.8% YoY.

Please see page eight of the slides. Next, I will explain our earnings forecast for FY2024.
Consolidated sales revenue is expected to be JPY150,000 million. Regarding operating expenses, the cost of
sales ratio is expected to be 32.6%, an improvement of 1.3 percentage points YoY. SG&A expenses are
expected to be JPY38,700 million, and R&D expenses are expected to be JPY31,500 million.
As a result, we expect operating profit of JPY31,000 million, profit before income taxes of JPY31,500 million,
and net profit attributable to owners of the parent of JPY24,500 million, a decrease from the previous year.

Please see page nine of the slides.
In the pharmaceuticals business, we forecast revenue of JPY128,500 million, an increase of 2.7% YoY. Despite
the effect of NHI price revision and generic products, the Company expects an increase in revenues due togrowth in sales of Viltepso, Uptravi, and other products, as well as growth in royalty income from overseas
sales of Uptravi.

Please see page 10 of the slides.
In the functional food business, we expect sales revenue to decrease 7.1% YoY to JPY21,500 million, due inpart to the impact from declining prices of some products, although we will strengthen our efforts in marketed
products.

Please see page 11 of the slides.
We plan to pay an annual dividend of JPY124 for the current fiscal year, with an interim dividend of JPY62 pershare and a year-end dividend of JPY62 per share.
This concludes my presentation for the financial results for FY2023 and the forecast for FY2024.

Takagaki: I am Kazuchika Takagaki, in charge of research and development. I would like to continue with an
explanation of the progress of R&D pipeline updated since the financial results for Q3 FY2023.
Please see page 13 of the slides.
In March 2024, we added the indication of Lennox-Gastaut syndrome to Fintepla, and the drug is alreadybeing utilized in clinical settings.
NS-87 Vyxeos, a treatment for high-risk acute myeloid leukemia, received manufacturing and marketing
approval in March 2024, and preparations are underway for its launch.
In April 2024, we filed an application for NS-304, Uptravi, for an additional indication in Japan for pediatricpulmonary arterial hypertension.
A global Phase II study has been initiated for NS-089/NCNP-02, a treatment for Duchenne muscular dystrophythat skips exon 44.
In February 2024, we entered into an exclusive license agreement with Vicore Pharma for the development
and marketing of C21 for the treatment of idiopathic pulmonary fibrosis in Japan.
The approval of LY3527727, a reversible, non-covalent BTK inhibitor, for the treatment of mantle cell
lymphoma, for which an alliance agreement was concluded with Eli Lilly Japan in March 2024, recently passed
the Drug Subcommittee II on May 9, 2024.
In addition to mantle cell lymphoma, we also have an alliance agreement for the indication of chronic
lymphocytic leukemia, and global Phase II studies are ongoing for both indications.
Under the terms of the agreement, Eli Lilly Japan will be responsible for the supply of the product in Japanafter obtaining the manufacturing and marketing approval for the product, and we will be responsible for
distribution and sales, as well as field activities to provide product information.

The development of NS-580 for both endometriosis and chronic prostatitis/chronic pelvic pain syndrome has
been temporarily suspended.
The development of NS-018 and NS-161 has been discontinued.

Please see page 14 of the slides.
The results of the Galactic53 trial, a Phase II study of viltolarsen, were presented in a poster at the MDA
conference in Florida in March 2024.
In April 2024, we entered into a research collaboration agreement with MiNA Therapeutics for the discoveryof nucleic acid medicines with potential indications for intractable and rare diseases in the field of central
nervous system disorders.
We believe that this will enable us to apply nucleic acid medicine technology to the central nervous systemand deliver new medicines to patients suffering from intractable or rare diseases.

I would now like to explain the features of NS-87, Vyxeos, which received manufacturing and marketing
approval in March 2024.
Please see page 16 of the slides.
Vyxeos is a liposomal formulation containing cytarabine and daunorubicin, which are conventionally used in
the treatment of leukemia, in a 5:1 ratio for the best antitumor effect. It is known to reach the bone marrowin liposome form and remain there for a long time. When taken up by leukemic cells and the liposomes aredegraded within the cells, the active ingredient is released, and the drug takes effect.

Please see page 17 of the slides.
Conventional 7+3 therapy requires continuous intravenous infusion for five to seven days in one cycle, but
Vyxeos requires administration for three days on days one, three, and five in the first cycle, and from thesecond cycle onwards, if necessary, it will be administered intravenously for 90 minutes on days one and three.
This may reduce the burden of treatment for both patients and healthcare professionals.

Please see page 18 of the slides.
Here are the results of the overseas Phase III study.
The subjects were 309 untreated high-risk AML patients aged 60-75 years. Overall survival with conventional
7+3 therapy was 5.95 months, compared to 9.56 months with Vyxeos, significantly prolonging overall survival.
Overseas, it is approved for the treatment of therapy-related AML and AML with myelodysplastic changes inmore than 30 countries or regions, including the United States and Europe. In May 2022, an orphan drug
designation was granted to this drug by the Ministry of Health, Labour and Welfare in Japan.
We believe that we can contribute to the treatment of high-risk AML by appropriately delivering this drug topatients and healthcare professionals who need it.
This concludes the overview of our R&D activities.

FY2023 Financial Results Presentation Q&A (Summary)May 14, 2024
NO

Questions

Answer.

1

Is the discontinuation of development of NS-018 due to the competitive

NS-018 began development targeting patients with myelofibrosis with platelet

environment of other JAK2 inhibitors and other drugs?

counts of less than 50,000, anticipating less competition. In contrast with our
expectations, a competing product was approved for use in patients withplatelet counts of less than 50,000, resulting in lower than expected patient
enrollment in our trials. After considering the marketability of the product basedon the speed of development, we decided to discontinue further development
of NS-018.

2

We understand that NS-018 was discontinued based on a strategic

We thought that the asset could be out-licensed or developed in-housedecision due to delays in patient recruitment. Depending on the results ofdepending on the results of the overseas P2b clinical trial, but after looking at

the clinical trial, could it have been licensed out?

the data, without waiting for the study to be completed, we decided to
discontinue it.

3

Why was NS-580 temporarily suspended?
resume the trial?

Also, what is required to

We reviewed the long-term non-clinical data for NS-580 and compared therisk-benefit in the two target diseases, which led to our decision to temporarilysuspend the trial. We will continue to review the results of the P2b clinical trial
for endometriosis and other studies, but we believe that it will take about six
months to determine whether the data from animal studies can be extrapolated
to humans.

4

Why are there more announcements of discontinuation and temporary

We have been in an expansionary phase so far and the number of oursuspension of development at this year’s earnings announcements?

development pipeline has been increasing rapidly. Looking ahead, we willprioritize investments with a high probability of success from a risk-benefit
perspective, with a view to medium-to-long-term growth.
Every discontinuation or temporary suspension has different reasons. It is theresult of strategic thinking and removing inferior items from the prioritization
process.

5

The share price is falling. Is there anything you are trying to change in the

As there are limits to what we can do on our own, we are thinking of finding afuture, such as being more proactive in partnering?

partner and out-licensing from an early stage. We have been thinking ofcollaborating with partners who have global development and sales capabilities
after completing POC in-house. Our policy on a product-by-product basis will
be announced later.

6

In the supplemental material, the FPI of NS-050 has not changed in 1H

For NS-050, there was a gradual dose escalation in the P1 study, which2024. Why has the end date the trial been moved back one year from thelengthened the duration of the study and pushed back the completion date ofprevious date?

it.

7

When do you expect to start clinical trial of NS-051?

As announced previously, the trial of NS-051 will begin during this fiscal year.

8

Sales of Viltepso of the last fiscal year were lower than expected, perhaps

There was no real impact from gene therapy. The pace of patient acquisition isdue to.

steady; we explained at the Q2 FY23 results meeting that there was a bit ofIs gene therapy the reason for the weak sales of Viltepso in the last fiscal

market turmoil in the first half of the year, but since then it has recovered. The

year?

number of new patients on a monthly basis has returned to normal.

9

Is the U.S. bridge program specific to Viltepso? Or is it also specific to CAP-

Bridge programs provide drugs free of charge to patients during gaps in their

1002 and other exon skipping drugs? Is such a program essential toinsurance coverage. In the U.S. patients must renew or switch their insurance

penetrate in the U.S. market for Duchenne Muscular Dystrophy (DMD)

plans each year, which can result in a gap in coverage at the beginning of thedrugs?

year. The reason why sales of Viltepso declined on a YoY and dollar basis in
the 4Q of the last fiscal year was because more cases than expected were
provided free of charge in the bridge program in February to fill the gap andthere were no sales for those cases. We believe that a similar program will benecessary for CAP-1002.

10What is the duration of the insurance gap for U.S. patients in the event of

It is about one month.

insurance renewal？
11

When will you release the P3 clinical trial data for Viltepso?

We are preparing to release the data soon.

12

The P3 clinical trial of Viltepso completed more than 6 months ago. Have

This will be explained in the P3 clinical trial data release.

you already discussed the data with the authorities?
13

If the results of the P3 clinical trial for Viltepso are positive, will it be

There is no change from the previous announcement. In China, based on thepossible to submit an NDA in Europe, China and other Asian

results of the P3 clinical trial, we will prepare for an NDA again. In Europe,countries/regions?

based on discussions with the authorities on the Pediatric Investigation Plan,
we expect the drug to be approved for a limited age group of patients who are
eligible for exon 53 skipping. The number of patients eligible for the drug is
expected to expand as our other clinical trials of Viltepso proceed.

14What are your plans for Europe and China, including partnering?

We will commercialize Viltepso in China by utilizing our own local subsidiary. In
Europe, we will consider strategic options: building our own sales presence,
partnering, and collaborating with other companies.

15

What if the P3 clinical trial results for Viltepso are unfavorable?

I would like to answer after the P3 clinical trial data is released.

16

How will sales of Viltepso's unapproved drug contribute significantly to the

The situation varies from country to country. If the drug is approved, it will becompany's revenues? Will it be sold at higher unit prices if the drug ismarketed in that country. If the drug is not approved in the country where a P3

officially approved in the future?

clinical trial participant resides, it will be provided free of charge under our EAP(Expanded Access Program). Other examples are that some patients pay forthe drug themselves, and some countries have charitable funds.

17

In an interview with an industry newspaper, President Nakai said that the

Capricor Therapeutics is conducting a P3 clinical trial called HOPE-3 in two

DMD pipeline, including CAP-1002 and other exon skipping drugs, would

groups, Cohort A and Cohort B. We had previously informed you that theallow for future growth. Do you still think the same?

timeline for CAP-1002 was to proceed with an application after reviewing theresults of these studies. Capricor Therapeutics has announced that there will
be a pre-BLA Type B meeting in May of this year, and they believe that a rolling
submission will be made based on the results of that meeting.
We have heard that the timing of the readout of the ongoing P3 clinical trial isscheduled for the end of this year, and that the previous Type B meeting with
the FDA confirmed that the FDA will approve the drug based on the Cohort A
results alone. In light of these factors, the timing of approval and launch may
be earlier than we had previously indicated. In light of this, the costs associatedwith CAP-1002's launch preparation are included as NS Pharma's SG&A
expenses in our forecast of SG&A expenses for this fiscal year.

18

You mentioned that SG&A expenses for CAP-1002 were a factor in theincrease in SG&A this quarter. How much is the actual amount?

We would like to refrain from disclosing specific amounts.

19

Does Capricor Therapeutics have the capacity to manufacture the volume

Capricor

Therapeutics's

current

manufacturing

capacity

is

to

make

to meet the market’s demand after the successful completion of the trial

investigational drugs for 61 patients in Cohort A and 44 patients in Cohort B,

of CAP-1002?

for a total of around 100 patients. The investigational drug manufacturing site
for Cohort B in San Diego will make the commercial formulation, but to meet
GMP standards, Capricor Therapeutics is currently investing intensively. They
have the capacity to meet the needs of the patients identified at this time, but
recognize that they need to scale up for the entire DMD patient population andare moving forward with plans to increase manufacturing volumes to supply
CAP-1002 to patients.

20

According to Clinicaltrials.gov, Top-line data of CAP-1002 is scheduled for

We understand that Capricor Therapeutics was able to successfully

disclosure in December, will it be for Cohort A only?

demonstrate to the authorities the non-clinical comparative equivalence ofboth sites A and B, which allowed the company to market the productmanufactured in Cohort B with the data from the Cohort A study.

21

Will there be any further development in the partnership with Capricor

We have a very friendly and close working relationship with Capricor

Therapeutics?

Therapeutics, and we will continue to monitor the development progress of
CAP-1002 and consider every option within our overall strategy.

22

What is the sales forecast for Vyxeos (NS-87) this fiscal year?

Although we do not disclose sales forecasts, we are eager to deliver the productto as many patients as possible. Pirtobrutinib, for which we have entered into
an alliance agreement with Eli Lilly Japan, is also scheduled for launch thisfiscal year, and as a company that is highly recognized in the blood cancer fieldin Japan, we hope to make up for the decline in Vidaza sales with these two
products.

23Will pirtobrutinib in the alliance agreement with Eli Lilly Japan be recorded
as sales or co-promotion?

The sales will be recorded on our own.

24

Is the new Medium-term Management Plan for a period of five years? Will

I will discuss the details during the briefing on the Medium-term Management

the pipeline decline due to the discontinuation or temporary suspension ofPlan, but we are considering a five-year period in principle, through FY2028. I

some candidate products affect future dividend payout ratios?

hope to be able to introduce what the company will be like in FY2028 after thepatent cliff of Uptravi, and what it aims to be like beyond that.

25

I think the fact that the dividend forecast is the same as the previous year

The dividend forecast is based on the company's decision to use cash for long-is having a negative impact on the stock price. Are plans for majorterm growth investments rather than returning it to shareholders in the short

investments, including M&A, keeping the dividend level low?

term. We will focus more on growth investments such as the acquisition ofsales/distribution networks and in-licensing to complement the Uptravi’s
patent cliff. All growth opportunities will be considered.