Outline of Consolidated Financial Results
for the 1st Quarter Ended June 30, 2023
August 10, 2023
NIPPON SHINYAKU CO., LTD.

1Q FY2023 Summary◆ Revenue：

37,012 million yen （ + 3.9% ）◆ Operating profit：

11,163 million yen （ + 8.6% ）◆ Profit before tax：

11,440 million yen （ + 8.8% ）◆ to owners of parent：

8,749 million yen （ + 6.1% ）Profit attributable

Revenue

Operating profit

Profit before tax

Profit attributable toowners of parent(Million yen)2

Segmental Review - Pharmaceuticals 30,407-1,445
Revenues from the licensingof industrial property rights+2,201-292
30,870（Million yen）
Ethical drugs
Revenues from the licensing ofindustrial property rights

Profit in co-promotion
Revenue

1Q FY2022+462
YoY Change

1Q FY2023

Results

Ratio

Results

Ratio

Amt

%

20,90068.7%

19,45463.0%-1,445

-6.9%

6,903

22.7%

9,104

29.5%

+2,201+31.9%

2,603

8.6%

2,310

7.5%

-292

-11.2%

30,407100.0%

30,870100.0%

+462

+1.5%

Despite the effect of price revision by MHLW* and generic products, revenue ofconsolidated pharmaceuticals segment increased by 1.5% due to increase of sales of
Ethical drugs such as “Viltepso” and “Uptravi”, and royalty revenue from Uptravi’s
overseas sales.
*MHLW : Ministry of Health, Labour and Welfare 3

Segmental Review - Functional Food 5,211

＋679
+10

＋129
＋91

＋20

＋931

6,142
（Million yen）

1Q FY2023

1Q FY2022

YoY Change

Results

Ratio

Results

Ratio

Amt

3,605

69.2%

4,284

69.8%

+679

+18.9%

Preservatives

726

13.9%

736

12.0%

+10

+1.4%

Supplements

335

6.4%

464

7.6%

+129

+38.7%

Health food ingredients

214

4.1%

306

5.0%

+91

+42.7%

Others

330

6.4%

350

5.6%

+20

+6.2%

Revenue

5,211

100.0%

6,142

100.0%+931+17.9%

Protein preparations%

Revenue of consolidated functional food segment increased by 17.9% throughsales increase of Protein preparations and Supplements.
4Operating profit
10,276-1,173-80-111,163（Million yen）
Revenue

1Q FY2022
Results

1Q FY2023

Ratio

Results+1,393
+965
-217

+887

YoY Change

Ratio

Amt

%

35,619100.0%

37,012100.0%+1,393+3.9%(Pharmaceuticals)(30,407)(85.4%)(30,870)(83.4%)(+462)(+1.5%)(Functional Food)(5,211)(14.6%)(6,142)(16.6%)(+931)(+17.9%)

Cost of sales

13,92839.1%

12,962

35.0%

-965

-6.9%

SG&A expenses

8,200

23.0%

8,418

22.7%

+217

+2.7%

R&D expenses

4,738

13.3%

5,911

16.0%

+1,173+24.8%

Other income

1,652

4.6%

1,572

4.2%

-80

-4.8%

Other expenses

128

0.3%

129

0.3%

+1

+1.0%

Operating profit

10,27628.9%

11,163

30.2%

+887

+8.6%

5

Profit attributable to owners of parent
8,249
+887
+31
+6
-425
+500

8,749
（Million yen）

1Q FY2022

1Q FY2023

Results

Results

YoY Change
Amt%

Operating profit

10,276

11,163

+887

+8.6%

Finance income

266

298

+31

+12.0%

Finance costs

28

21

-6

-23.1%

Profit before tax

10,514

11,440

+925

+8.8%

Income tax expense, etc

2,265

2,690

+425

+18.8%

8,249

8,749

+500

+6.1%

Profit attributable toowners of parent

6

Business Forecast for FY2023(Million yen)

FY2022

FY2023

1Q

FY

1Q

Progress

1H

FY

Results

Results

Resultsfor 1H

Forecasts

Forecasts

35,619144,175

37,01251.8%

71,500145,000(Pharmaceuticals)(30,407)(121,988)(30,870)(51.0%)(60,500)(123,500)(Functional Food)(5,211)(22,187)(6,142)(55.8%)(11,000)(21,500)

Operating profit

10,27630,049

11,16369.8%

16,00032,000

Profit before tax

10,51430,48911,44070.6%

16,20032,5008,249

22,812

8,749

70.6%

12,40025,000

Revenue

Profit attributable toowners of parent

Revenue and each profit have progressed towardachievement of 1H, FY forecasts.

7

R&D Pipeline

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

Indications

NS-065/NCNP-01(viltolarsen)

NME<in-house>
NS-87（daunorubicin /cytarabine）<in-license>New
combination

ZX008(fenfluraminehydrochloride)<Distribution（obinutuzumab）<in-license>

for PI/II

PI/II

Preparation
for PIIPIIPIII

NDA filing

LaunchPIIIin progress

High-risk acute myeloid
leukemiaNew
indication

CDKL5 deficiency disorder

Lupus nephritis
New
indication

Pediatric nephrotic syndrome

New
NS-304

Preparation

Lennox-Gastaut syndrome

partnership>

GA101

Duchenne muscular dystrophyPI

indication

Arteriosclerosis obliterans(selexipag)<in-house>

New dose

Pediatric pulmonary arterial
hypertension：changes from the Fiscal Year Ended March 31, 2023 9

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

Indications

PI

Preparation
for PI/II

PI/II

Preparation
for PIIPIIPIII

NDA filing

LaunchEndometriosis
NS-580<in-house>

NME

Chronic prostatitis / Chronic
pelvic pain syndrome

NS-089/NCNP-02(brogidirsen)

NME<in-house>
NS-229<in-house>

NME

NS-401(tagraxofusp)

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

NME

NS-917(radgocitabine)

NME<in-license>
NS-161<in-house>

NS-025<in-house>

Duchenne muscular dystrophy

Eosinophilic granulomatosis withpolyangiitis

Blastic plasmacytoid dendritic
cell neoplasm

Duchenne muscular dystrophy

Relapsed/refractory acute
myeloid leukemia

NME

Inflammatory diseases

NME

Urological diseases：changes from the Fiscal Year Ended March 31, 2023

10

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

Indications

NS-065/NCNP-01(viltolarsen)<in-house>
CAP-1002<partnership>

NME

Duchenne muscular dystrophy

NME

Duchenne muscular dystrophy

NME

Myelofibrosis

NME

Duchenne muscular dystrophy

PI

Preparation
for PI/II

PI/II

Preparation
for PIIPIIPIII

NDA filing

LaunchPIIIin progress

NS-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：changes from the Fiscal Year Ended March 31, 2023

11

Reference Materials

Consolidated Balance Sheet(Million yen)

End of

End of 1Q

ChangeEnd of

End of 1Q

ChangeFY2022

FY2023Amt

FY2022

FY2023Amt

237,451

237,408-42

Current assets

157,873

154,771-3,101

Non-current assets

79,57882,636+3,058Assets41,51834,609-6,908Current liabilities

35,18328,655-6,527

Non-current liabilities

6,334

5,953

-381

195,933

202,799+6,866

237,451

237,408-42

Liabilities

EquityTotal assets

237,451

237,408-42

Inventories
Other financial assets

and equity＝Liabilities and equity＝＝Assets＝Cash and cash equivalents

Total liabilities-4,795-840＋2,810Trade and other payables-4,177

Income taxes payable-3,848Retained earnings+4,91013

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

14

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・The enhancement of antitumor activity and reducing
adverse events are expected by NS-87 accumulated inbone marrow

15

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
16

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
17

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

Japan：PIII（LN）
Global : PIII（PNS）

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)

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 drug19

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 control20

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 count21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

22

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 EGPA23

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
24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 activity

25

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 AML26

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－27

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－28

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 1st Quarter Ended June 30, 2023
August 10, 2023

Presentation
Edamitsu: I am Takanori Edamitsu, Nippon Shinyaku Co., Ltd. Director, General Manager, Business
Management and Sustainability Division. Thank you very much for taking time out of your busy schedule toparticipate in our financial results briefing today. I appreciate it very much.
I will now explain our business results for Q1 of FY2023 and the progress of our R&D activities, in accordancewith the presentation materials posted on our website.

As an overview of our performance in Q1 of FY2023, we reported consolidated revenue of JPY37,012 million,
operating profit of JPY11,163 million, profit before tax of JPY11,440 million, and profit attributable toowners of the parent of JPY8,749 million.

In the pharmaceuticals business, consolidated net sales increased 1.5% YoY to JPY30,870 million despite theeffect of price revision by MHLW and generic products, due to growth in sales of Viltepso, a treatment for
Duchenne muscular dystrophy, and Uptravi, a treatment for pulmonary arterial hypertension and chronic
thromboembolic pulmonary hypertension, and royalty income from overseas sales of Uptravi.

In the functional foods business, sales of protein preparations and supplements increased, and consolidated
net sales in the functional foods business increased 17.9% YoY to JPY6,142 million.

The cost to sales ratio improved by 4.1 percentage points YoY to 35%, due to factors such as the sales mix,
including an increase in revenues from the licensing of industrial property rights. SG&A expenses increased
2.7% YoY to JPY8,418 million, mainly due to an increase in sales promotion fees in line with increased
domestic sales of Uptravi.
R&D expenses totaled JPY5,911 million, up 24.8% YoY, mainly due to an increase in contract researchexpenses in line with progress in clinical trials.
As a result, operating profit was JPY11,163 million, up 8.6% YoY.

Profit before tax was JPY11,440 million, up 8.8% YoY, and profit attributable to owners of the parent was
JPY8,749 million, up 6.1% YoY.

The consolidated earnings forecast for FY2023 remains unchanged from that announced on May 11, withconsolidated revenue of JPY145 billion, operating profit of JPY32 billion, profit before tax of JPY32.5 billion,
and profit attributable to owners of the parent of JPY25 billion.

Next, I will explain the progress of R&D items.
First, I would like to explain the development situation in Japan. Exon 53 skipping, NS-065/NCNP-01,
Viltepso, for the treatment of Duchenne muscular dystrophy, was launched in May 2020 and is currently inglobal Phase III trials.
The Phase I/II study for NS-87, a treatment for high-risk acute myeloid leukemia, was completed and an
application for approval was filed in June 2023.
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.
A Phase III study of GA101 for lupus nephritis is in Phase III trials in collaboration with Chugai
Pharmaceutical Co.
In addition, a Phase III study for pediatric nephrotic syndrome is underway.
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.

Phase IIb study is underway for NS-580, an endometriosis treatment.
In addition, a Phase IIa study for chronic prostatitis and chronic pelvic pain syndrome was initiated in June
2023.
A global Phase II study for exon 44 skipping, NS-089/NCNP-02, a treatment for Duchenne muscular
dystrophy, is in preparation.
A global Phase II study for NS-229, a treatment for eosinophilic granulomatosis with polyangiitis, is inpreparation.
A Phase I/II study of NS-401 for the treatment of blastic plasmacytoid dendritic cell neoplasm is underway.
A global Phase I/II study for exon 50 skipping, NS-050/NCNP-03, a treatment for Duchenne muscular
dystrophy, is in preparation.
A Phase I trial for NS-917 for the treatment of relapsed/refractory acute myeloid leukemia is underway.
Phase I trials are underway for NS-161, which is being developed for the treatment of inflammatory diseases,
and for NS-025, which is being developed for the treatment of urological diseases.

Next, I will explain the status of overseas development.
NS-065/NCNP-01, Viltepso, for the treatment of Duchenne muscular dystrophy, was launched in the US in
August 2020 and is currently in a global Phase III study.
We have signed a partnership agreement for commercialization for CAP-1002 for the treatment of
Duchenne muscular dystrophy with Capricor Therapeutics, Inc. in the US in January 2022 and in Japan in
February 2023. Capricor Therapeutics, Inc. is currently conducting Phase III trials in the US.
A global Phase II study for NS-018, a drug for myelofibrosis, is underway.
To reiterate, we are currently preparing global trials for NS-089/NCNP-02, NS-050/NCNP-03, and NS-229.
This concludes the overview of our R&D activities.

FY2023 1Q Financial Results Briefing Q&A (Summary)

Held on August 10, 2023
NO

Questions

Answers1

We felt that the progress of the profit in 1Q was fairly good. Describe the reasons for the good progress of the

Revenue progressed steadily overall. Furthermore, sales of U.S.Viltepso and royalty income from Uptravi grew more

profit to 1H forecast and the prospects for full year forecast.

than expected due to the yen's depreciation. As a result, overall sales are strengthening. With regard to expenses, theprogress of the marketing service expenses is as planned, but progress of R&D expenses has been slightly delayed due
to a shift in the schedule for the manufacture of bulk nucleic acid pharmaceuticals to the second half. We think expenses
will progress as planned for the full fiscal year.

2

What are the actual and assumed exchange rates?

The actual result is ¥137.5 to USD and the assumption is ¥130 to USD.

3

Other income is about 1.5 billion yen, but does this include a foreign exchange gain? Alternatively, is it another

It is the exchange gain. It was about 1.5 billion yen in the previous fiscal year and about 1.4 billion yen in the current

component?

fiscal year.

Tell us about the situation of domestic Viltepso. In contrast to the full-year sales increase forecast, 1Q sales haveIn 1Q, none of the patients were interrupted, and there was acquisition of the new patients and resumption of

declined YoY, and the progress rate does not seem to be very high. What is the factor behind this? And what do

administration. In the future, we believe that this increase of patients receiving this drug will lead to sales increase.

4

you think about the future outlook?
5

Tell us about the situation of domestic Viltepso. The material of Central Social Insurance shows that the number

In 1Q, several new patients have been started and 90 patients plus-alpha have been treated. There were no dropouts inof patients administered at the peak will be 128.As of the end of the previous year, it has been heard that the drug

1Q. Regarding the progress to 128 patients at the peak in the material of Central Social Insurance Medical Council,

has been administered to about 90 patients. Although sales of 1Q are almost leveling off, what is the current

many patients who we have grasped at present have not been administered because many of them are older, and sosituation and how many patients are treated? Tell us how many patients increase each year.

only 90 patients have been administered. On the other hand, there are more new patients than dropouts, and we thinkthat the number of patients receiving them will gradually increase.

6Tell us about the situation of Viltepso in the United States. In the United States, gene therapy has been received

Gene therapy has been received accelerated approval in the United States, and Sarepta recently announced that one

accelerated approval and its promotion has been started. I think the interest of patients and doctors in gene therapy

patient had been treated. In the future, the administration of gene therapy will be advanced in the U.S. We believe thatis growing. Tell us how the reputation of gene therapy and its impact on Viltepso are currently being viewed.

the effects of Viltepso should be adequately appealed as we did, so that it has no major impact. We believe that we willknow whether or not it is affected in the future. We need to look at the results of gene-therapy P3 study which will comewithin the year.

7

Tell us about the situation of Viltepso in the United States. We think Viltepso Sales in the US is strong. We have1

Regarding the Quick Start Program, the change in the contractor for HUB servicing was smoothly completed and it has

heard that the Quick Start Program has been started in June, but there is still no contribution of the Quick Start

been started since June, so we expect it to gradually become effective from June onward.

Program to 1Q's performance. We think that the contribution will accelerate from 2Q.Is it a correct perception?
8

In the United States, is there any information about whether there are new patients who are preparing for Viltepso

We haven’t received such information.

and are much interested in gene therapy?
9

I think Galactic53 study, a long-term P2 study of Viltepso, is ended in June. Will the results be disclosed in the

Data QC and analysis times are also needed, and we would like to publish the data at the appropriate timing in medical

fall?

conferences. So the result will be disclosed in the next year

10Will the results of Viltepso P3 study be disclosed within the year?

Not yet. The study will be completed in December 2024.

11

We have heard FPI of NS-089 and NS-050 is in 1H. Tell us more specifically about the developmental status of

The preparation of NS-089 P2 study is being promoted to achieve FPI in 1H. NS-050 is also moving toward FPI in 1H

NS-089, NS-050 and NS-051.

as planned, but there is a slight possibility that it will not fit in 1H due to concordance with the institution. NS-051 isunder discussion with FDA to begin the study within this fiscal year as originally planned.

12NS-089 received breakthrough therapy designation. Tell us about the implications and impacts. Also, is it possible

Until now, the discussion with FDA has only been for certain meetings, but in the future we can get support from FDA

to receive breakthrough therapy designation for other nucleic acid products currently under development?

as appropriate for the development of NS-089. FDA is also committed to the review. We believe that it is of greatsignificance to receive full support from FDA. With regard to breakthrough therapy designation for other items, wewould like to challenge if there was an opportunity, but we think that it will depend on the situation in the future whether
all items can be ridden on the same rail in general because the situation is different for each item.

13

14

15

We think that the interim analysis of CAP-1002 will be within this year. Despite the remainder of this year is

The study has progressed as planned, and the interim analysis will be conducted by December as planned. At present,

more than 4 months, ClinicalTrials.gov says the patient is still being recruited. Tell us about the current situation.

48 cases have been entered against the target sample size of 68 cases.

Tell us about CAP-1002. I have heard that the approval of drugs with unclear mechanisms, such as cellular

Although it is a cell therapy, we do not believe that it negatively affects the review. Since ongoing P3 study is atherapies, by the U.S. and Japanese regulatory authorities is quite difficult. CAP-1002 is an allogeneic CDC

confirmatory study, if the efficacy can be clearly confirmed in this study, it will undoubtedly be approved regardless oftherapy and the mechanism seems to be slightly obscured. I think the mechanism is not clear and FDA's review

the cell therapy. Regarding to mechanism, it is not unexplained, because anti-inflammatory and anti-fibrotic effects, etc.

may not be smooth, even if CAP-1002 test outcomes are good in the future. What do you think about that?

are clearly explained.

NS-018 has obtained European orphan designations. what is the recruitment status of the patient? I think the

Although the start of the study was delayed, the number of study sites in Europe has increased, and patients have begun

number of clinical trial sites is increasing in Europe and Asia, but is there no change in the schedule for study

to enter relatively smoothly. In the future, we will determine how soon we can catch up with our initial schedule.

completion date, April of the next year?
16

For NS-229, the indication has been disclosed as eosinophilic granulomatosis with polyangiitis (EGPA). Please

This disease is an autoimmune disease and designated as an intractable disease. The information on the patient number

explain the numbers of patients and why JAK1 inhibitors are effective against this disease.

is about 6000 persons in Japan. Because of autoimmune diseases, steroids have been used as a base treatment, but

2because of repeated relapses, there is a medical need. JAK1 has immunosuppressive effects and is expected to be used
to reduce steroidal use.
17

Are JAK1 inhibitors like NS-229 developed by other companies?

JAK1 inhibitor for EGPA is only our product.

18

On NS-229, I’m interested in the indication extension to other diseases, because it is JAK1 inhibitor. Is it correct

As a mechanism of NS-229 is immune suppression immunity, we are considering expanding indications to otherto think that P2 studies will be conducted for the present diseases and then will the indications be expanded? We

inflammatory diseases in addition to EGPA.

believe that JAK1 has safety issues. Tell us about the potential for future expansion of indications.
19

We think that candidates for expanded indications of NS-229 are atopic in inflammatory diseases, and ulcerative

With regard to areas where other antecedent JAK1 inhibitors have already been launched, we think that the treatment

colitis and Crohn's disease in autoimmune diseases. From the current data, what disease is suitable for expanded

need is already met and will not enter the market. We think that there is a need for our product more in diseases such as

indications?

EGPA where JAK1 inhibitors has not used and unmet medical needs exist. Expansion of indications to such diseases is
considered.

20

Regarding NS-580, P2a study for chronic prostatitis/chronic pelvic pain syndrome has started since June. BecauseYour understanding is correct.

P2a study has ended for the indication of endometriosis, is our understand correct that POC to stop the pain is
confirmed?
21

Tell us why chronic prostatitis/chronic pelvic pain syndromes for NS-580 indications was chosen.

Although there were several candidates for indications, one reason is that we have strengths in the urology field. In
terms of the nature of the disease, it has been confirmed that NS-580 is effective for pain. Although there are othermedications for short-term pain control, there are no medications that can be used for long periods of time, so we
decided to develop chronic prostatitis/chronic pelvic pain syndrome with chronic pain and discomfort.

22

I think other companies have also developed membrane-associated prostaglandin E synthase-1. Is myYour understanding is correct.

understanding correct that you are the top batter currently?
23

Do you think that NS-580 should be licensed out abroad?

The possibility is also considered.

24

Tell us about the status of functional food business. In the full-year forecast, sales will decrease due to factors

Although raw material prices have declined and stabilized, there is some uncertainty about selling prices because theysuch as a decline in sales prices, but from 1Q results, there is no sign of decrease. Are Sales and prices declining

are affected by the impact of exchange rates. In the future, if the exchange rate is shifted to strong yen, we think thatfrom 2Q , or are current status changing?

the sales price will decrease. On the other hand, because the effect of coronavirus is being resolved and sports events
have been held and the demand for inbound has recovered, the actual increase in quantity is considered to have a larger
effect.

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