Outline of Consolidated Financial Results
for the 3rd Quarter Ended December 31, 2023
February 9, 2024
NIPPON SHINYAKU CO., LTD.

3Q FY2023 Summary◆ Revenue： 112,728 million yen （＋2.6% ）◆ Operating profit：

30,450 million yen （＋8.8% ）◆ Profit before tax：

30,973 million yen （＋9.0% ）Profit attributable：

24,002 million yen （＋5.9% ）◆ to owners of parent

Revenue

Operating profit

Profit before tax

Profit attributable toowners of parent(Million yen)2

Segmental Review - Pharmaceuticals 93,095-3,847
Revenues from the licensingof industrial property rights+6,290-930+1,511

94,606（Million yen）
Ethical drugs
Revenues from the licensing ofindustrial property rights

Profit in co-promotion
Revenue

3Q FY2022

YoY Change

3Q FY2023

Results

Ratio

Results

Ratio

Amt

%

62,853

67.5%

59,005

62.4%-3,847-6.1%

22,60724.3%

28,89730.5%+6,290+27.8%

7,634

8.2%

6,703

7.1%

-930

-12.2%

93,095100.0%

94,606100.0%+1,511+1.6%

Despite the effect of price revision by MHLW* and generic products, revenue of consolidated
pharmaceuticals segment increased by 1.6% due to increase of sales of “Viltepso” and “Uptravi”,
and royalty revenue from Uptravi’s overseas sales.
*MHLW : Ministry of Health, Labour and Welfare3Segmental Review - Functional Food 16,824
＋736
+130
＋374
＋105
-50
＋1,29618,121（Million yen）

3Q FY2022

3Q FY2023

YoY Change

Results

Ratio

Results

Ratio

Amt

%

Protein preparations

11,58368.8%

12,319

68.0%

+736

+6.4%

Preservatives

2,253

13.4%

2,383

13.1%

+130

+5.8%

Supplements

1,091

6.5%

1,466

8.1%

+374

+34.3%

851

5.1%

957

5.3%

+105

+12.4%

Others

1,044

6.2%

994

5.5%

-50

-4.8%

Revenue

16,824100.0%

18,121100.0%+1,296+7.7%

Health food ingredients

Revenue of consolidated functional food segment increased by 7.7% throughsales increase of protein preparations and supplements.
4Operating profit
27,987＋2,808+3,942-949-4,364＋394+631+2,46230,450（Million yen）

3Q FY2022

3Q FY2023

YoY Change

Results

Ratio

Results

Ratio

Amt

%

109,919100.0%

112,728100.0%+2,808+2.6%(Pharmaceuticals)(93,095)(84.7%)(94,606)(83.9%)(+1,511)(+1.6%)(Functional Food)(16,824)(15.3%)(18,121)(16.1%)(+1,296)(+7.7%)

Cost of sales

42,556

38.7%

38,613

34.3%-3,942-9.3%

SG&A expenses

24,791

22.6%

25,741

22.8%

+949

+3.8%

R&D expenses

15,13513.8%

19,50017.3%+4,364+28.8%

Other income

1,492

1.4%

1,887

1.7%

+394

+26.4%(998)(0.9%)(1,361)(1.2%)(+362)(+36.3%)

Other expenses

941

0.8%

309

0.3%

-631

-67.1%

Operating profit

27,98725.5%

30,45027.0%+2,462+8.8%

Revenue(Foreign exchange gain)

5

Profit attributable to owners of parent
22,674+2,462＋78+9-1,222
24,002（Million yen）

3Q FY2022

3Q FY2023

Results

Results+1,328YoY Change
Amt%

Operating profit

27,98730,450+2,462+8.8%

Finance income

533

611

+78

+14.8%

Finance costs

98

89

-9

-9.7%

Profit before tax

28,42230,973+2,550+9.0%

Income tax expense, etc

5,748

6,970

+1,222+21.3%

22,67424,002+1,328+5.9%

Profit attributable toowners of parent

6

Business Forecast for FY2023
FY2022(Million yen)

FY2023

YoY Change

3Q

FY

3Q

FY

Results

Results

Results

Forecasts

Revenue

109,919

144,175

112,728(Pharmaceuticals)(93,095)(121,988)(Functional Food)(16,824)

Operating profit
Profit before tax
Profit attributable toowners of parent

Exchange rate
(JPY)
1USD

Amt

％

147,000+2,825+2.0%(94,606)(125,000)+3,012+2.5%(22,187)(18,121)(22,000)

-187

-0.8%

27,98730,04930,45033,500+3,451+11.5%

28,42230,48930,97334,000+3,511+11.5%

22,674

22,812

24,00226,000+3,188+14.0%

3Q

FY

3Q

2H

Actual rate

Actual rate

Actual rate

Forecast rate

136.5 yen

135.5 yen

143.3 yen

140.0 yen

FY2022

FY2023

Revenue and each profit have progressed
toward achievement of FY forecasts.
7

R&D Pipeline

R&D Pipeline (Domestic)
Code No.
(Generic name)<Origin>Application
type

Indications

NME

Duchenne muscular dystrophy<in-house>
NS-87（daunorubicin /cytarabine）<in-license>New
combi-(fenfluramine<Distribution

High-risk acute myeloid leukemia

nationZX008
hydrochloride)

Lennox-Gastaut syndrome<in-license>

PI/II

for PI/II

Preparing

PII

for PIIPIIPIII

NDA filing

LaunchPII

Around the spring of FY2024

PIII

P3 data presentation

analyzing

Application：FY2023
Approval (expected)：FY2023

Application：FY2023
Approval (expected)：FY2023

New

CDKL5 deficiency disorder

Lupus nephritis

GA101

Preparingindication

partnership>（obinutuzumab）

PI

PI

NS-065/NCNP-01(viltolarsen)

Schedule

New
indication

Pediatric nephrotic syndrome

Extra renal lupus

Study Completion：FY2026

Expansion of indications：from 2026 onward

Expansion of indications：from 2026 onward

Expansion of indications：from 2026 onward

Schedule : study completion date described in jRCT or ClinicalTrials.gov

9

R&D Pipeline (Domestic)
Code No.
(Generic name)<Origin>Application
type

New
NS-304indication

Indications

Schedule

Arteriosclerosis obliterans

Study Completion：FY2024

PI

Preparingfor PI/II

PI/II

Preparingfor PIIPIIPIII

NDA filing

Launch(selexipag)<in-house>
New dose

Pediatric pulmonary arterial
hypertension

Endometriosis
NS-580<in-house>

Study Completion：FY2025

Study Completion：FY2023

NME
Chronic prostatitis / Chronic
pelvic pain syndrome

Study Completion：FY2024

NS-089/NCNP-02(brogidirsen)

NME

Duchenne muscular dystrophy

Study Completion：FY2025<in-house>

NS-229<in-house>

NME

NS-401(tagraxofusp)

NME<in-license>

NS-050/NCNP-03<in-house>

NME

Eosinophilic granulomatosis withpolyangiitis

Blastic plasmacytoid dendritic cell
neoplasm

Duchenne muscular dystrophy

Study Completion：FY2025

Study Completion：FY2026

Study Completion：FY2026

Schedule : study completion date described in jRCT or ClinicalTrials.gov

10

R&D Pipeline (Domestic)
Code No.
(Generic name)<Origin>Application
type

NS-917(radgocitabine)

NME<in-license>

NS-161<in-house>

NS-025<in-house>

NS-863<in-house>

Indications

Relapsed/refractory acute
myeloid leukemia

Schedule

PI

Preparingfor PI/II

PI/II

Preparingfor PIIPIIPIII

NDA filing

LaunchStudy Completion：FY2024

NME

Inflammatory diseases

Study Completion：FY2024

NME

Urological diseases

Study Completion：FY2024

NME

Cardiovascular diseases

Study Completion：FY2024

Schedule : study completion date described in jRCT or ClinicalTrials.gov

11

R&D Pipeline (Overseas)
Code No.
(Generic name)<Origin>Application
type

Indications

NS-065/NCNP-01(viltolarsen)

CAP-1002

PI

Preparingfor PI/II

PI/II

Preparingfor PII

PII

PIII

Around the spring of FY2024

PIII

P3 data presentation

analyzing

NME

Duchenne muscular dystrophy

NME

Duchenne muscular dystrophy

Topline data：End of 2024

NME

Myelofibrosis

Study Completion：FY2024（TBD）

NME

Duchenne muscular dystrophy

Study Completion：FY2025<in-house><partnership>

Schedule

NDA filing

LaunchNS-018(ilginatinib)<in-house>
NS-089/NCNP-02(brogidirsen)<in-house>

NS-229<in-house>

NS-050/NCNP-03<in-house>

NME

NME

Eosinophilic granulomatosis withpolyangiitis

Duchenne muscular dystrophy

Study Completion：FY2025

Study Completion：FY2026

Schedule : study completion date described in jRCT or ClinicalTrials.gov

12Positioning in the three DMD treatments

Apr-Dec（￥million）

2022

2023

AnnualYoY
ChangeProgress
for FY

2022

2023(estimated)

Viltepso

10,717

13,225

23.4%

72.3%

14,341

18,300（Japan）(3,188)(3,332)(4.5%)(69.4%)(4,139)(4,800)（U.S.）(7,528)(9,892)(31.4%)(73.3%)(10,201)(13,500)• We believe that an optimal combination is selected from the three treatments (Nucleic acid drug,
cell therapy, and gene therapy) depending on the patient‘s genetic background and stage of thedisease.
• Despite the launch of gene therapy in the U.S. in 2023, sales of U.S. Viltepso increased.
13

Reference Materials

Consolidated Balance Sheet
End of

End of 3Q

YoY Change

End of

End of 3Q

YoY Change

FY2022

FY2023Amt

FY2022

FY2023

Amt(Million yen)

Assets237,451

247,830+10,378

Current assets

157,873

160,901+3,028Non-current assets

79,57886,928+7,350Liabilities

41,51832,513-9,004Current liabilities

35,18327,193-7,989Non-current liabilities

6,334

5,319

-1,014

195,933

215,316+19,383

237,451

247,830+10,378

EquityTotal assets

237,451

247,830+10,378＝Assets＝Total liabilities
and equity＝Liabilities and equity＝Other financial assets (NCA)＋6,037

Income taxes payable

Other current assets＋3,504Retirement benefit liability

Trade and other receivables＋2,821Retained earnings-5,435-617+16,02315

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

・Japan : Launch・USA : Launch・Global : PIII in progress

OriginCo-development :National Center of Neurology and Psychiatry

Development

Nippon Shinyaku

Mechanism ofactionExon 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

16

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

Japan : NDA filing

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.

17

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

Japan : Launch (Dravet syndrome)
Japan : NDA filing（Lennox-Gastaut syndrome)
Japan：PIII (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-Gastautsyndrome and CDKL5 deficiency disorder patients
refractory to existing treatment options
・ZX008 can be used in combination with other drugs, asstandard of care for intractable epilepsy based on
combination therapy
18

CAP-1002- Treatment for Duchenne muscular dystrophy Development Phase

USA : PIII

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

Development

Capricor Therapeutics, Inc.

Mechanism ofactionExosomes 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 improvementof motor and cardiac functions
・Its broad applicability makes it suitable for patients
regardless of the type of genetic mutation
19

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

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

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

Development

Co-development : Chugai Pharmaceutical Co., Ltd.

Mechanism ofactionAnti-CD20 monoclonal antibody

Indication

Lupus nephritis (LN)
Pediatric nephrotic syndrome (PNS)
Extra renal lupus (ERL)

Dosage form

Injection

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

18

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

Japan : PIIb（ASO）
Japan : PII （Pediatric PAH）

OriginNippon Shinyaku

Development

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

Mechanism of action

Selective IP receptor agonist

Indication

・Arteriosclerosis obliterans (ASO)
・Pediatric pulmonary arterial hypertension (Pediatric
PAH)

Dosage form

TabletFeatureLong-acting oral drug21

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

Japan : PIIb (Endometriosis)
Japan : PIIa (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 control22

NS-018 (ilginatinib)- Treatment for myelofibrosis Development Phase

Global : PII

OriginNippon Shinyaku

Development

Nippon Shinyaku

Mechanism of action

JAK2 inhibitor

Indication

Myelofibrosis

Dosage form

TabletFeature・Potent and highly selective JAK2 inhibitor
・High efficacy and safety are expected for myelofibrosis(MF) patients with low platelet count23

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

Global：Preparation for PII

OriginCo-development :National Center of Neurology and Psychiatry

Development

Nippon Shinyaku

Mechanism ofactionExon 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

24

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

Global：Preparation for PII

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 EGPA25

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

Japan : PI/II

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

Development

Nippon Shinyaku

Mechanism of action

Induction apoptosis of cells by inhibiting protein synthesisby specifically 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
26

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

Global：Preparation for PI/II

OriginCo-development :National Center of Neurology and Psychiatry

Development

Nippon Shinyaku

Mechanism ofactionExon 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 activity27

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

Japan : PI

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 AML28

NS-161- Treatment for inflammatory diseases Development Phase

Japan : PI

OriginNippon Shinyaku

Development

Nippon Shinyaku

Mechanism of action－

Indication

Inflammatory diseases (to be determined)

Dosage form

Oral agent

Feature－29

NS-025- Treatment for urological diseases Development Phase

Japan : PI

OriginNippon Shinyaku

Development

Nippon Shinyaku

Mechanism of action－

Indication

Urological diseases (to be determined)

Dosage form

Oral agent

Feature－30

NS-863- Treatment for cardiovascular diseases Development Phase

Japan : PI

OriginNippon Shinyaku

Development

Nippon Shinyaku

Mechanism of action－

Indication

Cardiovascular diseases (to be determined)

Dosage form

Oral agent

Feature－31

Safe Harbor Statement• Materials and information provided during this presentation may contain so-called “forward-looking
statements.” These statements are based on current expectations, forecasts and assumptions thatare subject to risks and uncertainties which could cause actual outcomes and results to differ materiallyfrom these statements.
• Risks and uncertainties include general industry and market conditions, and general domestic andinternational economic conditions such as interest rate and currency exchange fluctuations. Risks anduncertainties 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 bycompetitors; 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 a result 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 statements in the two versions, the statements in theJapanese version shall prevail.

Nippon Shinyaku Co., Ltd.
Financial Results Briefing for the Third Quarter Ended December 31, 2023
February 9, 2024

Presentation
Edamitsu: I am Takanori Edamitsu, Director and General Manager of the Business Management &Sustainability Division at Nippon Shinyaku Co., Ltd.
Thank you very much for taking time out of your busy schedule to attend our financial results presentation
today. I really appreciate it.
I would first like to explain our business results for Q3 of FY2023 and the progress of our R&D activities, inaccordance with the presentation materials posted on our website.

A summary of results for Q3 of FY2023 is as follows: consolidated revenue of JPY112,728 million, operating
profit of JPY30,450 million, profit before tax of JPY30,973 million, and profit attributable to owners of parentof JPY24,002 million.

In the pharmaceuticals business, despite the effects of NHI price revisions and generics, growth in the sales ofViltepso, a treatment for Duchenne muscular dystrophy, and Uptravi, a treatment for pulmonary arterial
hypertension and chronic thromboembolic pulmonary hypertension, as well as growth in royalty income fromthe overseas sales of Uptravi, our consolidated net sales of the pharmaceutical business increased by 1.6%
YoY to JPY94,606 million.

In the functional food business, sales of protein preparations and sports supplements increased, andconsolidated net sales in the functional food business increased by 7.7% YoY to JPY18,121 million.

The cost of sales ratio improved by 4.4 percentage points YoY to 34.3%, due to factors such as the sales mix,
including an increase in revenues from the licensing of industrial property rights.
SG&A expenses increased by 3.8% YoY to JPY25,741 million, mainly due to US marketing expenses and anincrease in sales promotion fees associated with increased domestic sales of Uptravi.
R&D expenses totaled JPY19,500 million, up 28.8% YoY, mainly due to an increase in contract researchexpenses.
As a result, operating profit was JPY30,450 million, up 8.8% YoY.

Profit before tax was JPY30,973 million, up 9% YoY, and profit attributable to owners of parent company was
JPY24,002 million, up 5.9% YoY.

The consolidated earnings forecast for FY2023 remains unchanged from the revised plan announced on
November 13, 2023, with consolidated revenue of JPY147 billion, operating profit of JPY33.5 billion, profit
before tax of JPY34 billion, and profit attributable to owners of parent of JPY26 billion.
I will continue with an explanation of the progress of R&D items.

First, let me explain the development situation in Japan.
The Duchenne muscular dystrophy treatment drug NS-065/NCNP-01, Viltepso that skips exon 53, waslaunched in May 2020. The global Phase III study has now been completed and analysis is underway.
The Phase I/II study for NS-87, a treatment for high-risk acute myeloid leukemia, was completed and an
application for approval was submitted in June 2023, which was approved by the Second Committee on Drugs
on February 5, 2024.
In June 2023, UCB submitted a partial change application for ZX008, a drug for the treatment of intractableepilepsy, for an additional indication for Lennox-Gastaut syndrome. In addition, UCB is conducting a Phase III
study for CDKL5 deficiency starting in July 2023.
For GA101, a Phase III study for lupus nephritis and a Phase III study for pediatric idiopathic nephrotic
syndrome are being conducted in collaboration with Chugai Pharmaceutical Co., Ltd.
In addition, a Phase III study for extra renal lupus is ongoing from October 2023.

A Phase IIb study of NS-304 for the indication of arteriosclerosis obliterans is being conducted by Nippon
Shinyaku on its own. In addition, a Phase II study for pediatric pulmonary arterial hypertension is underway
in collaboration with Janssen Pharmaceutical K.K.
A Phase IIb study of NS-580 for the treatment of endometriosis is underway. In addition, a Phase IIa study forchronic prostatitis and chronic pelvic pain syndrome is ongoing from June 2023.
A global Phase II study for Duchenne muscular dystrophy drug NS-089/NCNP-02 that skips exon 44 is being
prepared.
A global Phase II study for NS-229, a treatment for eosinophilic granulomatosis with polyangiitis, is being
prepared.
A Phase I/II study of NS-401 for the treatment for blastic plasmacytoid dendritic cell neoplasm is ongoing.
A global Phase I/II study for Duchenne muscular dystrophy treatment drug NS-050/NCNP-03 that skips exon
50 is being prepared.

Phase I study are underway for NS-917, a treatment for relapsed/refractory acute myeloid leukemia.
Phase I studies are underway for NS-161, which is being developed for inflammatory diseases, NS-025, which
is being developed for urological diseases, and NS-863, which is being developed as a treatment for
cardiovascular diseases.

I will continue with an explanation of the status of overseas development.
The Duchenne muscular dystrophy treatment drug NS-065/NCNP-01, Viltepso, was launched in the US in
August 2020. The global Phase III study has now been completed and analysis is underway.
For CAP-1002, a treatment for Duchenne muscular dystrophy, we entered into a marketing collaborationagreement with Capricor Therapeutics in the US in January 2022 and in Japan in February 2023. Capricor
Therapeutics is currently conducting a Phase III study in the US.
A global Phase II study is underway for NS-018, a treatment for myelofibrosis.
Global studies of the NS-089/NCNP-02, NS-229, and NS-050/NCNP-03 is being prepared.

Finally, I will explain about our approach to treatment in the DMD area.
In regard to each of the three therapies, namely, nucleic acid medicine, cell therapy, and gene therapy, we
believe that the optimal treatment will be selected according to the patient’s genetic background and diseaseprogression and that these three therapies will coexist in the future.
In the US, where gene treatment was launched in 2023, sales of Viltepso continued to grow, with cumulative
US Viltepso sales in Q3 up 31.4% YoY.
By offering a lineup of therapeutic agents in multiple modalities for DMD, Nippon Shinyaku will work to ensurethat as many patients as possible receive optimal treatment.
This concludes my explanation.

Q3 FY023 Results Briefing (Q&A Summary)

Held on February 9, 2024
NO

Questions

Response

1

P3 study for Viltepso is under analysis now, so when will topline data be published?

It will be published in this spring.

2

Have you decided on the conference presentation of the study for Viltepso targeting ambulant and non-ambulant

It has been determined. The data is expected to be released by the end of FY2023.

patients completed in June 2023?
3

Sales of U.S. Viltepso are up more than 19% QoQ. It was pointed out on the IR meeting for 2Q that the shipments

That is correct.

were delayed. Is it correct to understand that Viltepso originally scheduled to be shipped in 2Q was actuallyshipped in 3Q?
4Regarding Sales of U.S. Viltepso, I think its sales in 3Q increased QoQ. Is there any special factor in 3Q, such as

There is not any major special factor.

delayed shipment in 2Q?
5

It can be thought that sales of U.S. Viltepso in 4Q will increase QoQ due to the fact that sales in 3Q increased

We think that it is possible from the situation in January.

QoQ. Based on the current situation, do you think that you are able to achieve its forecast, which was lowered in
November last year.
6

The dollar based sales forecast of U.S. Viltepso was lowered by 2Q. The reason was that doctors and patients

In the IR meeting for 2Q, we explained the situation between AdCom in May last year and release of P3 data released.

were concerned about whether to choose gene therapy or nucleic acid, and the acquisition of new patients was

During this period, doctors and patients had high expectations for gene therapy and they struggled with a decision ofdelayed compared to the initial expectation. I think that its sales in 3Q are inline to the revised forecast. Please

their treatment. Therefore, the number of patients enrolled and administered in 1H was lower compared to the initialtell us whether the situation explained in the IR meeting for 2Q has changed by focusing on the new patientplan.

acquisition.
7

After the data of the gene therapy was published, patients who are positive towards treatment with nucleic acidThat is right.

drugs have appeared again. That kind of patients are new patients for you, so the contribution to sales has notbeen seen at present. However, does this situation suggest the possibility of sales of U.S. Viltepso returning to theoriginal track in the future?
8

It was around the end of October that P3 results for ELEVIDYS were published. Have there been significant
shifts in Viltepso sales between October and November to December?

1

There is no change.

9

One of the payers has a policy of reimbursement for exon skipping drugs after using gene therapy. Is there any

Such patients have not been reported yet.

patient who has been given exon skipping after gene therapy?
10

Is there any patient switched from Viltepso to gene therapy?

There is not such patients.

11

I am interested in why patients who have been administered Viltepso do not switch to gene therapy. I would like

We do not have personal information of patients administered Viltepso in the U.S. such as their ages. It has been a while

to know the background, such as whether there is no need for gene therapy because the patient's condition issince Viltepso was launched in the U.S. and we can assume that the number of patients aged 4 to 5 is small now, but it

stable with Viltepso, or whether target age group of Viltepso and gene therapy is non-competitive.

is unclear.

Is there any reason why the new patients choose nucleic acid drug rather than gene therapy?

There has been no significant impact on the acquisition of new patients of Viltepso after the accelerated approval of

12gene therapy, so we are not sure of the reason.
13

Are new patients of Viltepso both switching from Sarepta’s drug and drug-naive?

That is correct.

14

It seems that the market penetration rate of Viltepso in the U.S. is increasing considerably. Do you think there is

We believe there is still room for growth in 53 exon skipping drugs.

still room for its growth?
15

As stated in the document of Central Social Insurance Medical Council, the number of patients of Viltepso in

Viltepso has been administered to about 100 patients, including those who discontinued the treatment.

Japan at the peak would be 128. How many progress have you made toward 128 patients, including the numberof patients who have discontinued the treatment?
16

Is the number of patients of Viletepso increasing? Will the number of patients be fixed to some extent in the

We assume that the number of patients will increase because patients develop DMD every year.

future?
17

18

Sales of Viltepso in Japan appears to be low progress. I remember that there was a comment in the IR meeting

Although the administration of Viltepso has been delayed a little than expected, candidate patients for administrationfor 2Q that you had an outlook on the patients administered Viltepso in 2H. Is it possible to achieve the forecast?

have been confirmed.

I would like to know if there is any information on how DMD specialists in Japan asess gene therapy.

Regarding to the way of thinking towards gene therapy of domestic DMD specialists, we are not aware of doctors fully
supporting gene therapy. Gene therapy has not yet approved in Japan, and there is no doctor actively making a comment
on gene therapy as far as we are aware.

19

In the situation where no doctor strongly supports gene therapy, which is expected to be launched in Japan in the

In the U.S. gene therapy targets ambulatory patients aged 4 to 5 with accelerated approval, while CAP-1002 is expected

future, do you think it will be difficult for CAP-1002 to penetrate the market in Japan when it is newly launched?

to target non-ambulatory patients. Therefore, we consider that CAP-1002 can be differentiate from gene therapy. If P3
topline data is published by Capricor Therapeutics in the future, it is expected that CAP-1002 will be smoothly
introduced in Japan as the new treatment option.

20

You invested in Capricor Therapeutics. Is this recorded in the balance sheet?

Yes. It is recorded as other financial assets.

2

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22

The interim analysis of CAP-1002 led to the decision to continue P3 study and you need to pay milestone toSince the interim analysis was released in December last year, we did not pay milestone in cash in 3Q. However, weCapricor Therapeutics. Are you going to pay it after January?

have recorded it in the balance sheet.

Is the recruiting of NS-089/NCNP-02 going well? When is the FPI of NS-050/NCNP-03?

We originally planned for FPI of NS-089/NCNP-02 in 1H FY2023, but patients expected to be enrolled were not eligible
for criteria in screening. Currently nine sites are opened and there are several patients awaiting screening there, so it ispossible to expect FPI immediately. We assumes FPI of NS-050/NCNP-03 in 1H FY2024, partly because it took longer
than originally planned to open the sites.

23

24

You mentioned that FPI of NS-050/NCNP-03 is in 1H FY2024, but the range in 1H is as wide as 6 months. Could

There are several candidates for NS-050/NCNP-03, but the timing of FPI can be changed depending on the results ofyou tell us more detailed schedule?

the screening. Therefore, we will set FPI in 1H FY2024.

Is any study for a new pipeline going to be started next fiscal year? Is NS-050/NCNP-03 followed by NS-

NS-051/NCNP-04 is also expected to start next fiscal year.

051/NCNP-04 scheduled to start next fiscal year?
25

What is the target of the amount of dystrophin protein expression for NS-051/NCNP-04?

Regarding NS-051/NCNP-04, we expect the amount of dystrophin protein expression as much as of NS-089/NCNP02, which showed an increase in dystrophin protein expression to more than 10% in the investigator-initiated clinical
trial.

26

At the R&D meeting in December, it was explained that the entry for P2b study of NS-580 had been completed.

I think it will be completed as scheduled.

Will the study be completed in the end of February this year?
27

If the data from P2b study of NS-580 is as expected, will you look for a global partner?

We would like to consider this possibility.

28

Regarding the progress of the performance in 3Q, I think that the progress of R&D expenses is well toward theAlmost all of expenses are generally going according to plan.

forecast, but is there any expense that can unachieve forecast? Dose everything progress as planned?
29

What is the actual exchange rate between 1Q and 3Q?

The actual rate is ¥143.3 to USD.

30Regarding to sales and profit in the functional food business, sales in 3Q tend to be higher than the other quarters

We were expected that sales would decline because of drop in the raw material prices and a stronger yen. Raw material

in recent years. However, in this year sales in 3Q is less than in 1H. On the other hand, profit is higher than in

prices were not lower than expected due to the depreciation of the yen against assumed exchange rate, but sales fall

1H. What are these factors?

slightly due to the fact that raw material prices were passed on to sales prices. Nevertheless, when raw material prices
fall, profit tends to increase due to a slight time lag in passing through to sales prices.

31

Is the period of the next medium-term management plan five years? Will you explain the next medium-term

I think it will probably be five years, though we are considering it now. In addition, we also plan to explain how tomanagement plan, including how to overcome the patent cliff?

overcome the patent cliff.

3