Outline of Consolidated Financial Results
for the 1st Quarter Ended June 30, 2022
August 9, 2022
NIPPON SHINYAKU CO., LTD.

1Q FY2022 Summary◆ Revenue：

35,619 million yen （ - 13.1% ）◆ Operating profit：

10,276 million yen （ - 37.9% ）◆ Profit before tax：

10,514 million yen （ - 37.3% ）Profit attributable：

8,249 million yen （ - 34.5% ）◆ to owners of parent

Revenue

Operating profit

Profit before tax

Profit attributable toowners of parent(Million yen)2

Segmental Review - Pharmaceuticals 36,889＋1,517Revenues from the licensingof industrial property rights-8,310+310-6,48130,407（Million yen）
Ethical drugs
Revenues from the licensing ofindustrial property rights

Profit in co-promotion
Revenue

1Q FY2021

YoY Change

1Q FY2022

Results

Ratio

Results

Ratio

Amt%

19,382

52.6%

20,90068.7%+1,517+7.8%

15,214

41.2%

6,903

22.7%

-8,310-54.6%

2,292

6.2%

2,603

8.6%

+310

+13.6%

36,889100.0%

30,407100.0%-6,481-17.6%

Sales of Ethical drugs including “Viltepso” and “Uptravi”, revenues from the licensing of industrial
property rights containing royalty revenue from Uptravi’s overseas sales grew.
However, due to price revision by MHLW and backlash from the loss of sales revenue from thepriority review voucher booked in 1Q FY2021, net sales decreased by 17.6%.
3

Segmental Review - Functional Food 4,079＋894+80-57＋214＋1,132

5,211（Million yen）

1Q FY2021

1Q FY2022

YoY Change

Results

Ratio

Results

Ratio

Amt

2,710

66.4%

3,605

69.2%

+894+33.0%

Preservatives

645

15.8%

726

13.9%

+80+12.5%

Health food ingredients

272

6.7%

214

4.1%

-57

-21.2%

Others

450

11.1%

665

12.8%

+214

+47.6%

Revenue

4,079

100.0%

5,211

100.0%+1,132+27.8%

Protein preparations%Net sales increased by 27.8% through sales from Protein preparations including
milk proteins and Supplements such as “WINZONE Protein”.
4Operating profit
16,559-5,349-1,029-518-1,000＋1,517＋98-6,283

10,276（Million yen）

1Q FY2021

1Q FY2022

YoY Change

Results

Ratio

ResultsRatio

Amt%

40,968100.0%

35,619100.0%-5,349-13.1%(Pharmaceuticals)(36,889)(90.0%)(30,407)(85.4%)(-6,481)(-17.6%)(Functional Food)(4,079)(10.0%)(5,211)(14.6%)(+1,132)(+27.8%)

Cost of sales

12,89831.5%

13,92839.1%+1,029+8.0%

SG&A expenses

7,681

18.8%

8,200

23.0%

+518

+6.8%

R&D expenses

3,737

9.1%

4,738

13.3%

+1,000+26.8%

Other income

135

0.3%

1,652

4.6%

+1,517+1,120.9%

Other expenses

226

0.5%

128

0.3%

-98

-43.4%

Operating profit

16,55940.4%

10,276

28.9%-6,283-37.9%

Revenue

5Profit attributable to owners of parent
12,589-6,283＋32-5＋1,916-4,339

8,249（Million yen）

1Q FY2021

1Q FY2022

ResultsResults

YoY Change
Amt%

Operating profit

16,55910,276-6,283-37.9%

Finance income

234

266+32+13.7%

Finance costs

2328+5+23.4%

Profit before tax

16,77010,514-6,256-37.3%

Income tax expense, etc

4,181

2,265-1,916-45.8%

12,5898,249-4,339-34.5%

Profit attributable toowners of parent

6

Business Forecast for FY2022FY2021(Million yen)

FY2022

1Q

FY

1Q

Progress

1H

FY

Results

Results

Resultsfor 1H

Forecasts

Forecasts

40,968137,484

35,61953.2%

67,000134,000(Pharmaceuticals)(36,889)(120,650)(30,407)(53.0%)(57,400)(115,000)(Functional Food)(4,079)(16,834)(5,211)(54.3%)(9,600)(19,000)

Operating profit

16,55932,948

10,27670.9%

14,50027,000

Profit before tax

16,77033,301

10,51471.5%

14,70027,50012,58924,986

8,249

73.0%

11,30021,500

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) ①
Code No.
(Generic name)<Origin>Application
typeIndicationsPI

NS-065/NCNP-01(viltolarsen)

PI/II

PI

NME

Duchenne muscular dystrophy

NME

Iron deficiency anemia

NME

Dravet syndrome

NME

Lennox-Gastaut syndrome<in-house>

PIII

PII

NDA

Preparation

Launchfor launch

PII

PIII
in progress

NS-32(ferricderisomaltose)<in-license>
ZX008(fenfluraminehydrochloride)<in-license>
ZX008(fenfluraminehydrochloride)<in-license>
GA101（obinutuzumab）<in-license>

New
indication

Lupus nephritis：changes from the Fiscal Year Ended March 31, 2022

9

R&D Pipeline (Domestic) ②
Code No.
(Generic name)<Origin>Application
typeNew
NS-304indication

Indications

PIPI/II

PII

PIII

NDA

Preparation

Launchfor launch

Arteriosclerosis obliterans(selexipag)<in-house>
New dose

NS-580<in-house>
NS-87（daunorubicin /cytarabine）<in-license>

NME

New
combination

NS-401(tagraxofusp)

NME<in-license>

NS-229<in-house>

NME

NS-917(radgocitabine)<in-license>

NME

Pediatric pulmonary arterial
hypertension

Endometriosis

Secondary acute myeloid
leukemia

Blastic plasmacytoid dendritic
cell neoplasm

Inflammatory diseases

Relapsed/refractory acutemyeloid leukemia：changes from the Fiscal Year Ended March 31, 2022

10

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

PIPreparation
for PⅡ

PII

PIII

NS-065/NCNP-01(viltolarsen)

NME

Duchenne muscular dystrophy

NME

Duchenne muscular dystrophy

NME

Myelofibrosis<in-house>

CAP-1002<partnership>

LaunchPIII
in progress

NS-018(ilginatinib)<in-house>：changes from the Fiscal Year Ended March 31, 2022

11Reference Materials

Consolidated Balance Sheet
End of

End of 1Q

ChangeEnd of

End of 1Q

ChangeFY2021

FY2022Amt

FY2021

FY2022Amt(Million yen)

Assets219,943

219,684-259

Current assets

149,724

149,284-439

Non-current assets

70,21970,399+179

Liabilities

39,05734,456-4,600Current liabilities

32,02927,616-4,412

Non-current liabilities

7,027

6,839

-188

180,886

185,227+4,341

219,943

219,684-259

EquityTotal assets

219,943

219,684-259

Total liabilities
and equity＝Liabilities and equity＝＝Assets＝Cash and cash equivalents-1,651

Trade and other payables-5,821Inventories-1,403

Income taxes payable-1,017

Other financial assets＋473

Retained earnings+4,27513

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 ofaction

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

14

NS-32 (ferric derisomaltose)- Treatment for iron deficiency anemia Development Phase

Japan : Preparation for launch

Origin[Dec. 2016]
Licensed-in from : Pharmacosmos A/SDevelopment

Nippon Shinyaku

Mechanism of action

IronIndication

Iron deficiency anemia

Dosage form

IV bolus injection or IV drip infusion

Feature・Can be administered in high doses allowing full ironcorrection in the majority of patients・Good safety profile with no dose dependent ADRs
・Minimal potential toxicity from release of labile iron due
to tight iron binding in a matrix structure of interchanging
isomaltoside and iron・No profound hypophosphatemia15

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

Japan : NDA filing (Dravet syndrome)
Japan : PⅢ （Lennox-Gastaut syndrome)

Origin[Mar. 2019]
Commercial rights from : UCB S.A. (former Zogenix, Inc.)

Development

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

Mechanism of action

Serotonin agonistIndication

Dravet syndrome and Lennox-Gastaut syndrome

Dosage form

Oral liquid agent

Feature・Effective for Dravet syndrome and Lennox-Gastautsyndrome 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 :Capricor Therapeutics, Inc.

Development

Capricor Therapeutics, Inc.

Mechanism ofaction

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 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 Development Phase

Japan : PIII

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

Development

Co-development : Chugai Pharmaceutical Co., Ltd.

Mechanism ofaction

Anti-CD20 monoclonal antibody

Indication

Lupus nephritis

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 agonistIndication

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

Dosage form

TabletFeatureLong-acting oral drug19

NS-580- Treatment for endometriosis Development Phase

Japan : PIIb

OriginNippon Shinyaku

Development

Nippon Shinyaku

Mechanism of action

Inhibition of membrane-associated prostaglandin E
synthase-1

Indication

Endometriosis

Dosage form

Oral agent

FeatureTreatment for endometriosis without hormonal effect andwith possible analgesic potency20

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

Overseas : Preparation for 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-87 (daunorubicin / cytarabine)- Treatment for secondary acute myeloid leukemia Development Phase

Japan : PI/IIOrigin[Mar. 2017]
Licensed-in from: Jazz Pharmaceuticals plc

Development

Nippon Shinyaku

Mechanism of action

Liposomal combination of daunorubicin and cytarabineIndication

Secondary acute myeloid leukemia (secondary AML)

Dosage form

InjectionFeature

・NS-87 is the first therapy for the treatment of
secondary AML in Japan・The enhancement of antitumor activity and reducing
adverse events are expected by NS-87 accumulated inbone marrow22NS-401 (tagraxofusp)- Treatment for blastic plasmacytoid dendritic cell neoplasm Development Phase

Japan : PI/IIOrigin[Mar. 2021]
Licensed-in from: The Menarini GroupDevelopment

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

InjectionFeature・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
23NS-229- Treatment for inflammatory diseases Development Phase

Japan : PIOrigin

Nippon ShinyakuDevelopmentNippon Shinyaku

Mechanism of action

JAK1 inhibitor

Indication

Inflammatory diseases (to be determined)

Dosage form

Oral agentFeature

・Potent and highly selective JAK1 inhibitor
・High efficacy and good safety profiles are expected inthe treatment for inflammatory diseases24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 atlow dose continuous infusion
・Tolerable safety profile available to elderly patients withr/r AML

25

Safe Harbor Statement• Materials and information provided during this presentation may contain so-called “forward-lookingstatements.” 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, 2022
August 9, 2022

Edamitsu: I am Edamitsu in charge of the Business Management & Sustainability Division. Thank you verymuch for joining our financial results briefing today. I would like to express my greatest gratitude.
I would now like to explain our business results and R&D progress for Q1 of FY2022 in accordance with thepresentation materials posted on our website.

Please refer to slide two.
As an overview of our performance in Q1 of FY2022, we reported consolidated revenue of JPY35.619 billion,
operating profit of JPY10.276 billion, profit before tax of JPY10.514 billion, and profit attributable to ownersof parent of JPY8.249 billion for the quarter.

1

Please refer to slide three.
In the pharmaceuticals business, although sales of VILTEPSO, treatment for Duchenne muscular dystrophy,
and UPTRAVI, treatment for pulmonary arterial hypertension and chronic thromboembolic pulmonary
hypertension, and royalty income from overseas sales of UPTRAVI grew, consolidated revenue of thepharmaceuticals business decreased 17.6% YoY to JPY30.407 billion due to the absence of the one-time
income from the sale of priority review voucher that existed in the same period of the previous year.

2Please refer to slide four.
In the functional food business, sales of protein products such as milk protein and sports supplements
centering on WINZONE Protein increased, resulting in consolidated revenue of JPY5.211 billion, up 27.8% YoY.

3

Please refer to slide five.
The cost of sales ratio worsened by 7.6 percentage points YoY to 39.1% due to factors such as the sales mix,
led by decrease in revenues from the licensing of industrial property rights.
SG&A expenses increased 6.8% YoY to JPY8.2 billion, mainly due to sales promotion fees associated withincreased domestic sales of UPTRAVI and increased sales expenses in the functional food business.
Research and development expenses increased 26.8% YoY to JPY4.738 billion due to an increase in contract
research expenses and nucleic acid investigational new drug manufacturing costs in line with progress inclinical trials.
As a result, operating profit was JPY 10.276 billion, down 37.9% YoY.

4

Please refer to slide six.
Profit before tax was JPY10.514 billion, down 37.3% YoY, and profit attributable to owners of parent was
JPY8.249 billion, down 34.5% YoY.

5

Please refer to slide seven.
The consolidated earnings forecast for FY2022 remains unchanged from that announced on May 11, 2022,with consolidated revenue of JPY134 billion, operating profit of JPY27 billion, profit before tax of JPY27.5
billion and profit attributable to owners of parent of JPY21.5 billion.

6

I will continue with an explanation of the progress of R&D items.
Please refer to slide nine. First, I will explain the development situation in Japan.
A treatment for Duchenne muscular dystrophy, NS-065/NCNP-01, was approved in March 2020 and launchedin May. A global Phase III study is currently underway.
In March this year, we obtained manufacturing and marketing approval for NS-32, a treatment for irondeficiency anemia, and are currently preparing for launch.
As for ZX008, a treatment for intractable epilepsy, UCB Japan Co., Ltd. filed an application for Dravet syndromein December 2021. In addition, UCB S.A. is conducting a Phase III study for Lennox-Gastaut syndrome.
In June of this year, we initiated a Phase III study of GA101 for lupus nephritis in collaboration with Chugai
Pharmaceutical Co.

7

Please refer to slide 10.
Nippon Shinyaku has been conducting Phase IIb study of NS-304 for arteriosclerosis obliterans since Februaryof this year on its own. In addition, Phase II study for pediatric pulmonary arterial hypertension is ongoing incollaboration with Janssen Pharmaceutical K.K.
As for NS-580, a treatment for endometriosis, we completed Phase IIa study and started Phase IIb study inJune this year.
As for NS-87, a treatment for secondary acute myeloid leukemia, Phase I/II study is ongoing.
In July of this year, we initiated a Phase I/II study of NS-401, a treatment for blastic plasmacytoid dendritic cellneoplasm.
As for NS-229, JAK1 inhibitor, Phase I study is ongoing for inflammatory diseases.
As for NS-917, a treatment for relapsed/refractory acute myeloid leukemia, we have been conducting Phase
I study since February this year.

8Please refer to slide 11. I will continue with an explanation of the overseas R&D situation.
NS-065/NCNP-01, a treatment for Duchenne muscular dystrophy was approved in the US in August 2020 andlaunched. A global Phase III study is currently underway. It received Orphan Drug Designation in Europe inJune 2020.
With regard to CAP-1002, a treatment for Duchenne muscular dystrophy, we concluded a sales collaborationagreement with Capricor Therapeutics Inc. in the US in January 2022. In July of this year, Capricor Therapeutics
Inc. initiated Phase III study in the US.
We are currently preparing for the next global clinical trial for NS-018, a treatment for myelofibrosis. We arecurrently in the process of implementing the trial procedures at medical institutions and have begun recruiting
patients.
As for NS-089/NCNP-02 treatment for Duchenne muscular dystrophy, we have been in discussions with theFDA regarding the protocol, and the FDA has requested additional information regarding the interpretationof the IND application data. We are currently preparing to provide information and working to reach anagreement with the FDA by the end of this fiscal year.
As for NS-050/NCNP-03, a treatment for Duchenne muscular dystrophy, we expect to start clinical trials in thefirst half of the next fiscal year, following a request for additional non-clinical trials in discussions with the FDA.
That is all for overview of our research and development activities.

9