Outline of Consolidated Financial Results
for the 1st Quarter Ended June 30, 2024
August 7, 2024
NIPPON SHINYAKU CO., LTD.

1Q FY2024 Summary（Million yen）

1Q FY2023
Results
Ratio

1Q FY2024
Results
Ratio

YoY Change
Amt%

Revenue

37,012100.0%

39,131100.0%+2,118+5.7%(Pharmaceuticals)(30,870)(83.4%)(33,738)(86.2%)(+2,868)(+9.3%)(Functional Food)(6,142)(16.6%)(5,393)(13.8%)(-749)(-12.2%)

Cost of sales

12,96235.0%

12,636

32.3%

-326

-2.5%

SG&A expenses

8,418

22.7%

9,221

23.6%

+803

+9.5%

R&D expenses

5,911

16.0%

7,497

19.2%

+1,586+26.8%

Other income

1,572

4.2%

1,507

3.9%

-64

-4.1%

(Foreign exchange gain)(1,422)(3.8%)(1,211)(3.1%)(-210)(-14.8%)Other expenses

129

0.3%

204

0.5%

+74

+58.0%

Operating profit

11,16330.2%

11,078

28.3%

-85

-0.8%

Finance income

298

0.8%

363

0.9%

+65

+21.8%

Finance costs

21

0.1%

31

0.1%

+9

+42.0%

Profit before tax

11,44030.9%

11,411

29.2%

-29

-0.3%

Income tax expense, etc

2,690

7.3%

1,146

2.9%

-1,544-57.4%

Profit attributableto owners of parent

8,749

23.6%

10,26426.2%+1,514+17.3%

2

Segmental Review - Pharmaceuticals Ethical drugs 20,496 million yen(＋1,042 million yen, ＋5.3%, YoY)

（Million yen）

30,870 Sales growth of Uptravi and Viltepso, etc.
 Decrease in sales of Vidaza, Tramal/Onetram, etc.
+1,042+1,674

Revenues from the industrial property rights
10,779 million yen(＋1,674 million yen, ＋18.4%, YoY) Royalty revenue growth due to overseas sales ofUptravi+151

33,738

Profit in co-promotion 2,461illion yen( + 151 million yen, + 6.5%, YoY)
 Sales growth of Opsumit and Erleada

3

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

Viltepso

1Q FY2023

1Q FY2024

Results

Results

YoY Change
Amt

%

Japan

1,051

1,175

+124

+11.8%✓The number of patients currently receiving the drug is more than two-thirds ofthe peak number of 128 patients in the data by the Central Social Insurance
Medical Council (Chuikyo) .

U.S.

3,113

4,275

+1,162+37.3%✓The number of patients receiving or wishing to receive the drug is increasing.
✓Phase III Trial (RACER53 Study) is under additional analysis.

total

4,165

5,450

+1,285+30.9%

1Q FY2023

1Q FY2024

Actual rate

Actual rate

137.5yen155.9yenExchange rate
1USD

Japan

（Million yen）

U.S.

4

Segmental Review - Functional Food （Million yen）

6,142

Protein preparations 3,463 million yen(-821 million yen, -19.2%, YoY)
 Decline in unit sales prices

-821
+63

+87

-45

-32

5,393

Preservatives 800 million yen(+63 million yen, +8.6%, YoY)
 Recovery in food service-related business due toactive tourism and excursions
Supplements 552 million yen(+87 million yen, +18.8%, YoY) Growth in both the sports and anti-aging care fields

Health food ingredients 260 million yen(-45 million yen, +14.8%, YoY)

5

Operating Profit（Million yen）

Revenue 39,131 million yen(+2,118 million yen, +5.7%, YoY)

11,163+2,118+326-803-1,586-64-74

11,078

 Increase in revenue from industrial property
rights and the U.S. sales of Viltepso.
Cost of sales 12,636 million yen(-326 million yen, -2.5%, YoY)
The ratio was improved by 2.7 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 9,221 million yen(+803 million yen, +9.5%, YoY)
 Increase in promotional expenses in NS Pharma Increase in commission for promotional activities
of Uptravi
R&D expenses 7,497 million yen(+1,586 million yen, +26.8%, YoY)
 Increase in cost of investigational products

6

Business Forecast for FY2024 (consolidated)
FY2023（Million yen）

1Q
Results

FY2024FY
Results

1Q
Results

Progressfor 1H

1H
Forecasts

FY
Forecasts

Revenue

37,012

148,255

39,13151.6%

75,800154,000(Pharmaceuticals)(30,870)(125,105)(33,738)(51.7%)(65,300)(132,500)(Functional Food)(6,142)(23,150)(5,393)(51.4%)(10,500)(21,500)

Cost of sales

12,96250,234

12,63625,40051,000

SG&A expenses

8,418

34,9599,221

18,60038,700R&D expenses

5,911

31,676

7,497

16,80032,400

Other income

1,572

3,163

1,507

350

500

Other expenses

129

1,252

204

350

400

Operating profit

11,16333,295

11,07815,00032,000Finance income

298

650

363

250

600

Finance costs

21

329

31

50

100

Profit before tax

11,44033,616

11,411

15,20032,500

Income tax expense, etc

2,690

7,764

1,146

1,700

3,500

Profit attributableto owners of parent

8,749

25,851

10,264

13,50029,000Exchange rate
1USD

1Q FY2023
Actual rate
137.5yen

73.9%

75.1%

76.0%

1Q FY2024
Actual rate
155.9yen

FY2024
Forecast rate
140.0yen7

Segmental Forecast - Pharmaceuticals FY2023（Million yen）

FY2024

YoY Change

1Q
Results

FY
Results

1Q
Results

FY
Forecasts

Amt%

19,45676,143

20,49681,300+5,157+6.8%

Revenue from the licensing ofindustrial property rights

9,104

40,304

10,77942,100+1,796+4.5%

Profit in co-promotion

2,310

8,658

2,461

9,100

+442

+5.1%

Revenue

30,870125,105

33,738132,500+7,395+5.9%

Ethical drugs

8Segmental Forecast - Functional Food FY2023（Million yen）

FY2024

YoY Change

1Q
Results

FY
Results

1Q
Results

FY
Forecasts

4,284

15,6003,463

Preservatives

736

3,105

Supplements

464

Health food ingredientsAmt%

13,000-2,600-16.7%

800

3,200

+95

+3.1%

1,905

552

3,100

+1,195+62.7%

306

1,248

260

1,100

-148

-11.9%

Others

349

1,290

316

1,100

-190

-14.7%

Revenue

6,142

23,1505,393

21,500-1,650-7.1%

Protein preparations

9

R&D PIPELINE

10

R&D Updates（1/2）

For updates since May 14, 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)

March 2024

LaunchNS-87（daunorubicin /cytarabine）

Vyxeoshigh-risk acute myeloid leukemia

May 2024

Approval

LY3527727(piltobrutinib)

Jaypirca

patients with relapsed or refractory mantle cell
lymphoma who are resistant or intolerant to other
BTK inhibitors

June 2024

In application

NS-304（selexipag）

Uptravi

pediatric pulmonary arterial hypertension

April 2024

Start of P3

GA101（obinutuzumab）

Gazyvasystemic lupus erythematosus without nephropathy

October 2023

NS-089/NCNP-02（brogidirsen）

ー

Duchenne muscular dystrophy

February 2024

NS-229ーeosinophilic granulomatosis with polyangiitis

June 2024

In-license(Vicore Pharma, Sweden)

C21

ーidiopathic pulmonary fibrosis

Contract signed
in February 2024

Alliance Agreement(Eli Lilly Japan)

LY3527727(piltobrutinib)

Jaypirca

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

Contract signed
in March 2024

Temporarily suspended

NS-580ー

endometriosis
chronic prostatitis / chronic pelvic pain syndromeーStart of P2

11

R&D Updates（2/2）
Recent status/event
Preliminary analysis
results
Conference Presentations
Manuscript

Orphan Drug Designation

Code No.
(Generic name)
NS-065/NCNP-01(viltolarsen)
NS-065/NCNP-01(viltolarsen)
NS-089/NCNP-02（brogidirsen）
NS-089/NCNP-02（brogidirsen）
NS-401(tagraxofusp)
NS-229

Alliance(MiNA Therapeutics)ーFor updates since May 14, 2024,
see highlighted text in red.

Product
nameIndications and topics

Schedule

Viltepso

global Phase 3 trial (RACER53 Study)

May 2024

Viltepso

Phase 2 trial (Galactic53 trial): 2024 Muscular
Dystrophy Association Clinical & Scientific Conference

March 2024ー

non-clinical data: (Molecular Therapy Nucleic Acids)

October 2023

ー

Duchenne muscular dystrophy

December 2023 (EU)ーblastic plasmacytoid dendritic cell neoplasm

August 2023 (Japan)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

12

REFERENCE MATERIALS

13

Sales By Product in Pharmaceutical Segment（Million yen）
Brand nameIndications

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

1Q
FY2023

1Q
FY2024

Results

Results

YoY Change

FY2024
Forecasts

4,165

5,450

+30.9%

20,600(1,051)(1,175)(+11.8%)(4,600)(3,113)(4,275)(+37.3%)(16,000)

Uptravi

pulmonary arterial hypertension/
chronic thromboembolic pulmonary hypertension

3,255

3,855

+18.4%

15,400Vidazamyelodysplastic syndrome/acute myeloid leukemia

2,871

1,462

- 49.1%

4,800

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

1,220

1,254

+2.8%

5,100

Tramal/Onetram

cancer pain, chronic pain

1,104

778

- 29.5%2,700

Defitelio

sinusoidal obstruction syndrome

631

709

+12.4%

2,300

Cialiserectile dysfunction

654

647

- 1.1%

2,700

Zalutia

urinary disorder caused by benign prostatic hyperplasia

614

493

- 19.6%

1,600

Adcircapulmonary arterial hypertension

631

480

- 23.9%1,700

Erizasallergic rhinitis

334

281

- 15.7%

2,100

Profit in co-promotion

2,310

2,461

+6.5%

9,100

Revenues from the licensing of industrial property rights

9,104

10,779+18.4%

42,10030,87033,738+9.3%

132,500

Revenue

14

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

（Million yen）

1Q FY2023

1Q FY2024

YoY Change

Results

Ratio

Results

Ratio

4,284

69.7%

3,463

64.2%

-821

- 19.2%

13,000Preservatives

736

12.0%

800

14.8%

+63

+8.6%

3,200

Supplements

464

7.6%

552

10.3%

+87

+18.8%

3,100

Health food ingredients

306

5.0%

260

4.8%

-45

- 14.8%

1,100

Others

349

5.7%

316

5.9%

-32

- 9.2%

1,100

Revenue

6,142

100.0%

5,393

100.0%-749- 12.2%21,500Protein preparations

Amt%

FY2024
Forecasts

15

Consolidated Balance Sheet
End of

End ofChangeEnd of

End ofChangeFY2023

1Q FY2024

Amt

FY2023

1Q FY2024

Amt

263,404

265,828+2,423Current assets

164,285

159,973-4,311

Non-current assets

99,119105,854+6,734（Million yen）

AssetsTotal aseets

263,404

265,828+2,423Liabilities

42,87038,365-4,504Current liabilities

37,33632,826-4,510Non-current liabilities

5,533

5,539

+5

Equity220,534

227,462+6,928

Total liabilities
and equity

263,404

265,828+2,423Liabilities and Shareholders' Equity

AssetsCash and cash equivalents- 3,621

Trade and other payables- 3,057

Intangible assets+ 4,316

Other current liabilities＋ 1,684

Other financial assets (non-current)+ 2,016

Retained earnings＋ 6,08816

Pipeline（1/2）*Schedule is based on trial end dates, etc. from jRCT or
Clinical Trials.gov

Stage

Code No.
(Generic name)

OriginApplication
type

Indications

Schedule

Country

LaunchP3

NS-065/NCNP-01

In-house

NME

Duchenne muscular dystrophy―

Japan/U.S.

Approval ：June in 2024

Japan

Preparingfor launch

LY3527727(pirtobrutinib)

Alliance
agreement

NMEpatients with relapsed or
refractory mantle cell
lymphoma who are resistant or
intolerant to other BTK
inhibitors

NDA filing

NS-304(selexipag)In-house

New dose
New indication

pediatric pulmonary arterial
hypertension

Study Completion：FY 2025
Application：April in 2024

Japan

ZX008
(fenfluraminehydrochloride)

Distribution
partnership

New indication

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

Duchenne muscular dystrophy

Topline data：end of 2024

U.S.

mantle cell lymphoma

―

Japan

chronic lymphocytic leukemia

―

Japan

GA101
(obinutuzumab)

In-license

New indication

P3
CAP-1002(deramiocel)

LY3527727(pirtobrutinib)

Partnership

Alliance
agreement

NME

New indication

17

Pipeline（2/2）*Schedule is based on trial end dates, etc. from jRCT or
Clinical Trials.gov

Stage

Code No.
(Generic name)

OriginApplication
type

Indications

Schedule

Country

NS-304(selexipag)In-house

New indicationarteriosclerosis obliterans

Study Completion：FY2024

Japan

endometriosis

Temporarily suspendedJapan

chronic prostatitis/
chronic pelvic pain syndrome

Temporarily suspendedJapan

NS-580In-house

NME

P2
NS-089/NCNP-02（brogidirsen）

In-house

NME

Duchenne muscular dystrophy

Study Completion：FY2025
FPI：February in 2024

Japan/U.S.

NS-229In-house

NME

eosinophilic granulomatosis
with polyangiitis

Study Completion：FY2026

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

18

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

・Japan : Launch・U.S.
: Launch・Global : P3 open-label extension study 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

19

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.

20

LY3527727(pirtobrutinib)- Treatment for Mantle cell lymphoma, Chronic lymphocytic leukemia -Development Phase・ Preparing for launch(for patients with relapsed or refractory mantle cell lymphoma whoare resistant or intolerant to other BTK inhibitors)
・ P3 (MCL and CLL)

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.

21

CAP-1002 (deramiocel)- 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.

22

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 cytotoxicity23

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

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

OriginNippon Shinyaku

Development

Nippon Shinyaku

Mechanism of action

Selective IP receptor agonist

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

Dosage form

TabletFeatureLong-acting oral drug24

NS-580- 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 control25

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

26

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

Global：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 EGPA27

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

28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 activity29

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 AML30

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－31

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－32

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.
Financial Results Briefing for the 1st Quarter Ended June 30, 2024
August 7, 2024

Presentation
Edamitsu: I am Takanori Edamitsu, Nippon Shinyaku Co., Ltd. Director, General Manager, Business
Management & Sustainability Division.
Thank you very much for taking time out of your busy schedule to participate in our financial results briefing
today. I appreciate it very much.
I will now explain our business results for Q1 FY2024 and the progress of our R&D activities, in accordancewith the presentation materials posted on our website.

Please see page two of the slide. As an overview of the results for Q1 FY2024, consolidated revenue was
JPY39,131 million, operating profit was JPY11,078 million, profit before taxes was JPY11,411 million, and profit
attributable to owners of the parent was JPY10,264 million.

1Please see page three of the slide. In the pharmaceuticals business, consolidated net sales increased 9.3% YoY
to JPY33,738 million despite the effect of NHI price revision and generic products, due to growth in sales ofViltepso, a treatment for Duchenne muscular dystrophy, and Uptravi, a treatment for pulmonary arterial
hypertension and chronic thromboembolic pulmonary hypertension, and royalty income from overseas salesof those products.

Please see page four of the slide. Here we show sales of Viltepso, a treatment for Duchenne muscular
dystrophy, which is marketed in Japan and the United States. Sales in both Japan and the US increased YoY,
totaling JPY1,175 million in Japan and JPY4,275 million in the US.

2

Please see page five of the slide. In the functional foods business, sales of supplements and other products
increased, but sales of protein preparations and other products decreased, resulting in consolidated net salesof JPY5,393 million in the functional foods business, down 12.2% from the same period last year.

3Please see page six of the slide. The cost-to-sales ratio improved by 2.7 percentage points YoY to 32.3%, due
to factors such as the sales mix, in addition to an increase in industrial property rights and other revenues.
Selling, general, and administrative expenses increased to JPY9,221 million, up 9.5% from the same period of
the previous year, due to an increase in sales promotion expenses of the US subsidiary NS Pharma andoutsourcing fees for sales promotion activities for Uptravi.
R&D expenses totaled JPY7,497 million, up 26.8% YoY, due to an increase in manufacturing costs including
investigational drugs, etc. As a result, operating profit was JPY11,078 million, down 0.8% YoY.

4

Please see page seven of the slide. The Company has revised its consolidated earnings forecast for the fiscal
year ending March 31, 2025, from that announced on May 10, as royalty income from overseas sales ofUptravi and sales of new products such as Viltepso exceeded the Company's forecast. Consolidated revenue
is expected to be JPY154,000 million, consolidated operating profit JPY32,000 million, and profit before taxes
JPY32,500 million for the full year.
In addition, the Company expects net income attributable to owners of the parent of JPY29 billion due to the
expected decrease in income tax expense as a result of the consideration of the recoverability of deferred tax
assets at consolidated subsidiaries in the US and the reflection in the estimated effective tax rate.

5

Please see page eight of the slide. In the pharmaceutical business, we forecast revenue of JPY132.5 billion, an
increase of 5.9% YoY. Despite the effect of NHI price revision and generic products, the Company expects anincrease in revenues due to growth in sales of Viltepso, Uptravi, and other new product lines, as well as growth
in royalty income associated with overseas sales of Uptravi.

6

In the functional foods business, sales revenue is expected to be JPY21.5 billion, down 7.1% from the previous
year due to a decrease in sales of protein preparations.
Next, I will explain the progress of R&D items. This section describes the items that have been updated since
the release of the FY2023 financial results.

7

Please see page 11 of the slide. NS-87, Vyxeos, a treatment for high-risk acute myeloid leukemia, was launchedin May 2024.
The reversible, non-covalent BTK inhibitor LY3527727 (piltobrutinib), for which we entered into an allianceagreement with Eli Lilly Japan, obtained marketing approval in June 2024 for the treatment of "patients withrelapsed or refractory mantle cell lymphoma who are resistant or intolerant to other BTK inhibitors."
A global Phase II study of NS-229 for the treatment of eosinophilic granulomatosis with polyangiitis was
initiated in June 2024.

8

In May 2024, we announced the preliminary results of the analysis of the global Phase III study of NS065/NCNP-01, Viltepso for the treatment of Duchenne muscular dystrophy.
Additional analysis is currently underway to advance discussions with the regulatory authorities.
This concludes my explanation of the results for Q1 FY2024 and the progress of R&D.

9

Q1 FY2024 Financial Results Presentation Q&A (Summary)August 7, 2024
NO

Questions

Answer.

1

You have increased the sales revenue forecast by 4 billion yen for the full

The remaining increase will be the sum of Fintepla and Vyxeos sales, Viltepso's

year, although Viltepso sales and royalty income from Uptravi increased

increased sales to other regions than Japan and U.S., and other products that

only about 1 billion yen in total. I would like to ask about the remaining 3

are expected to increase in sales gradually. It is certain that Vyxeos sales havebillion yen increase. What are the sales of products not disclosed at thissignificantly exceeded the original plan.

time other than Fintepla and Vyxeos sales?
2

Other than Viltepso sales and Uptravi royalties, are the large majority of

Vyxeos is the major contributor to such sales.

the approximately 3 billion yen in either Vyxeos sales in Japan, or Viltepso
sales to the rest of the world?
3

Will Vyxeos sales not be disclosed?

It will be disclosed when it meets our disclosure criteria.

4Will revenue from sales of an individual product disclosed when the annual

Once a product is expected to reach that level, we will disclose its specificsales forecast for that product reaches 2 billion yen?

revenue.

It seems that costs will be increased in the second half of the year. Do you

The delayed recognition of some SG&A and R&D expenses in the second half

anticipate that costs will be shifted from the original plan to the secondof the year has been taken into account in the revised FY2024 financial

half?

forecast.

R&D expenses for the first half of the year were shifted to the second half

In addition to delays in the timing of manufacturing costs recognition, theof the year. Is this largely due to the shift in the timing of the accrual of

impact of outsourced research costs is also included in those R&D expenses.

56

expenses for nucleic acid API?
7

Previously, I heard that CAP-1002-related costs would be included in the

We assume that expenses will be incurred in 4Q.

U.S. SG&A expenses. Has this cost been incurred at all yet? Or will it beincurred intensively in the second half or 4Q or gradually in the future?
8

I think the company's exchange rate assumption was originally 140 yen to
the US dollar. What is the rate in the revised FY2024 forecast?

We continue to assume 140 yen to the U.S. dollar.

9Viltepso's U.S. sales have been revised slightly upward this time. Is the

Even on a U.S. dollar basis, Viltepso's U.S. sales have exceeded our internalsales performance in line with the plan, excluding the impact of foreign

projections.

exchange rates?
10

I understand that the foreign exchange assumptions have not changed in

The full-year forecast for FY2024 is expected to be generally in line with thethe revised FY2024 financial forecast. Will you not change your forecast for

initial plan.

the second half of the fiscal year?
11

To what extent did the upward effect of foreign exchange rates affect sales

The actual foreign exchange in 1Q was about 155 yen to the U.S. dollar, about

and operating income in the 1Q of this fiscal year? The company has

15 yen higher than the company's assumption of 140 yen to the U.S. dollar. We

revised its earnings forecast based on the assumption of 140 yen to thebelieve that the foreign exchange impact can be calculated considering thedollar. If the actual level of foreign exchange continues from the 2Q onward,

above difference of 15 yen in the 1Q results of Viltepso sales results in the U.S.

will the effect of foreign exchange decrease compared to the 1Q?

and revenues from industrial property rights.
The forecast exchange rate remains unchanged at 140 yen from the 2Q onward,
so if the actual exchange rate remains close to 140 yen, the results will be close
to the plan. On the other hand, if the exchange rate were to fall below 140 yen,
our performance would be affected. However, since Viltepso is slightly
exceeding the plan even on a volume basis, we do not think this will be a major
problem.

12

The extent to which foreign exchange has had an impact in the upwardWe will not disclose specific numbers.

revision of sales will be clear if we do the calculations you mentioned. How
much is the impact of foreign exchange on operating income? I think U.S.
costs also have an impact.
13

I estimate that the company's effective tax rate is about 10.8% for FY2024.

We expect the negative tax expense to be temporary and the tax rate to return

What is your forecast for FY2025 and beyond?

to normal next fiscal year.

14

Could you please elaborate on the status of your communication with the

The situation has not changed significantly since the last presentation. In-

authorities regarding Viltepso? At the May 28 presentation on the 7th Mid-house data analysis is largely complete, and we will now move on to

Term Management Plan, I heard that the P3 (RACER 53 Study) results may

discussions with the FDA, which are expected to take place in this fall. We

have been affected by steroids. What is the current situation?

expect the results of the discussions around the end of this year.

15

Do you assume that the P3 (RACER 53 Study) will be redone?

Yes.

16

Regarding the P3 (RACER 53 Study) of Viltepso, at the May 27th

At the last presentation, we were not talking about projections based onpresentation on the 7th Mid-Term Management Plan, you used a variety of

specific data. Since then, additional analysis has provided data that confirms

expressions to describe the probability that the results of this trial would

efficacy in certain populations, and we would like to continue in the direction

result in full approvals, the risk of withdrawal of conditional approvals, andof maintaining the approvals.

so on. Now that the in-house analysis is almost complete, have there been
any new findings that might change those expressions?
17

Previously you told that the results of the discussions with the authorities

We are considering making a public announcement at about the conclusion ofwill be disclosed by the end of this year. Do you have a policy or expectation

our discussions with the authorities.

for the disclosure of specific data?
18

How do you perceive the reaction in the medical community to the results

At this point in the U.S., Viltepso continues to be used. There has not been aof Viltepso's P3 (RACER 53 Study) so far? There were only qualitative

single case of a patient discontinuing treatment with Viltepso and switching tocomments on top line data, no specific data. How would this affecta competing product in response to the results of Viltepso’s P3 (RACER 53

physician and patient perceptions? How about the willingness of treated

Study). In Japan, Viltepso is the only available treatment option for DMD. We

patients to continue treatment or the willingness of patients not yet

have explained top-line data to physicians, and Viltepso continues to be usedtreated?

in Japan too. The pace of growth in the number of new patients is slightbecause the drug has been penetrated well before the P3 (RACER 53 Study)
results were available. The exact impact in the U.S. is difficult to determine,but given that Viltepso and Vyondys 53 have been on the market for some time
and the number of new patients has been gradually decreasing, we do notbelieve the impact of P3 (RACER 53 Study) data is significant.

19

I would like to know the number of new Viltepso patients in the US in 1Q.

As originally planned, the number of new patients has been increasing eachIs the number growing QonQ? Has the number been affected sincemonth by several.

Sarepta’s gene therapy received the full FDA approval in June this year?
20

Are there about 90 patients receiving Viltepso in Japan?

It’s approximately 90 plus.

21

Please tell us about the situation surrounding Viltepso in the U.S. market,

Since July, no patients receiving Viltepso have switched to gene therapy. Sales

especially since this past July.

of Elevidys (Sarepta's gene therapy) are strong, but it appears that patients
who cannot use exon-skipping drugs are using Elevidys first. Prior to the full
FDA approval, Elevidys was limited to patients aged 4 through 5 years old, so
Viltepso patients did not immediately switched to gene therapy. Now that
Elevidys has been approved and its age restriction has been removed, we need
to watch carefully its impact in the future. We will continue to collectinformation in the US market.

22

At the IR Meeting (Q2/FY2023) on November 15, 2023, President Nakai

We have heard that both drugs may be reimbursed in combination, but we havementioned “There are some insurance policies in the U.S. that can be

yet to hear of a case where Viltepso was actually administered after a dose of

combined with Viltepso and Elevidys, but we have not yet heard of any

Elevidys.

actual cases where they have been reimbursed that way”. Are there any
patients who are using Viltepso after Elevidys?
23

For now, it is less than a couple of months from the full FDA approval of

From the sales of Elevidys, it can be inferred that the number of patients is

Elevidys. Do Viltepso and Elevidys coexist in different US markets?

increasing rapidly. However, to date, no patients receiving Viltepso havereceived Elevidys.

24

I believe NS-050 has only one clinical trial site and has not yet been

We have found some candidate patients and expect to begin the clinical trial inadministered to patients. Is it likely to be started in the first half of the year?

the first half of this year.

25

Clinical trial information for NS-051 has not yet been disclosed. Do you

We are in discussions with the FDA to coordinate the interpretation of non-need to coordinate anything with the FDA regarding animal testing for NS-

clinical data to initiate clinical trials.

051?
26
27

Has the rolling BLA for CAP-1002 already started? I understand that the

We do not disclose such information as an update. Please check an

meeting with FDA will take place in July-September.

announcement from Capricor Therapeutics.

At the May 28 presentation on the 7th Mid-Term Management Plan,

Currently, our recruitment efforts are underway in the U.S., but have not yet

President Nakai mentioned the possibility of hiring a CMO (Chief Medical

been completed.

Officer) with a medical license. What is the current situation?
28

When do you expect to be able to hire a CMO in the U.S.?

We are doing our best but cannot say how long it will take to find a qualified
medical doctor to suit the position.