Outline of Consolidated Financial Results

for the 3rd Quarter Ended December 31, 2021
February 10, 2022
NIPPON SHINYAKU CO., LTD.

3Q FY2021 Summary◆ Net sales：

106,218 million yen （+15.7%）◆ Operating profit：

29,321 million yen （+44.0%）◆ Ordinary profit：

30,563 million yen （+45.4%）

Profit attributable：

22,606 million yen （+49.9%）◆ to owners of parent(Million yen)2

Segmental Review - Pharmaceuticals 80,243+3,789
Revenues from the licensing ofindustrial property rights+9,176+415
93,625（Million yen）
Ethical drugsRevenues from the licensing ofindustrial property rights

Profit in co-promotion
Net sales

3Q FY2020＋13,3813Q FY2021

YoY Change

Results

Ratio

Results

Ratio

Amt%

56,23070.1%

60,02064.1%+3,789+6.7%

17,63722.0%

26,81428.6%+9,176+52.0%

6,375

7.9%

6,791

7.3%

+415

+6.5%

80,243100.0%

93,625100.0%+13,381+16.7%Net sales increased by 16.7% through growth of ethical drugs such as “Vidaza”,“Uptravi”, ”Viltepso”, and revenues from the licensing of industrial property rights which contains ofroyalty revenue from Uptravi’s overseas sales and gain on sales from the priority review voucher.
3

Segmental Review - Functional Food 11,594＋412+139+6+439＋998

12,593（Million yen）

3Q FY2020

3Q FY2021

YoY Change

Results

Ratio

Results

Ratio

Amt%

Protein preparations

7,745

66.8%

8,158

64.8%

+412

+5.3%

Preservatives

2,008

17.3%

2,147

17.1%

+139+6.9%

774

6.7%

781

6.2%

+6+0.9%Others

1,066

9.2%

1,506

11.9%

+439

+41.2%Net sales

11,594100.0%

12,593100.0%+998+8.6%

Health food ingredients

Net sales increased by 8.6% through sales from functional food products
such as protein preparations, preservatives, etc.
4Operating profit
20,362+13,381＋998+294-1,469-4,247+8,95929,321（Million yen）

3Q FY2020

3Q FY2021

YoY Change

Results

Ratio

Results

Ratio

Amt%

91,837100.0%

106,218

100.0%+14,380+15.7%(Pharmaceuticals)(80,243)(87.4%)(93,625)(88.1%)(+13,381)(+16.7%)(Functional Food)(11,594)(12.6%)(12,593)(11.9%)(+998)(+8.6%)

Operating expenses

71,475

77.8%

76,89772.4%+5,421+7.6%

Cost of sales

38,14641.5%

37,851

35.6%-294-0.8%

SG&A expenses

21,86923.8%

23,33822.0%+1,469+6.7%

R&D expenses

11,45912.5%

15,70614.8%+4,247+37.1%

Operating profit

20,36222.2%

29,321

27.6%+8,959+44.0%Net sales

5Profit attributable to owners of parent
15,085＋8,959＋185+394-2,018+7,521

22,606（Million yen）

3Q FY2020

3Q FY2021

Results

Results

YoY Change
Amt%

Operating profit

20,362

29,321+8,959+44.0%

Non-operating income

1,599

1,785

+185

+11.6%

Non-operating expenses

938

543-394-42.1%

Ordinary profit

21,02330,563+9,539+45.4%

Income taxes, etc

5,938

7,956

+2,018+34.0%

15,08522,606+7,521+49.9%

Profit attributable toowners of parent

6

Business Forecast for FY2021
FY2020

FY2021

3Q

FY

3Q

Progress

FY

Results

Results

Resultsfor FY

Forecasts

91,837

121,885

106,21878.7%

135,000(Pharmaceuticals)(80,243)(106,478)(93,625)(78.5%)(119,300)(Functional Food)(11,594)(15,406)(12,593)(80.2%)(15,700)

Operating profit

20,362

26,13429,321104.7%

28,000Ordinary profit

21,023

26,76030,563107.2%

28,50015,08520,702

22,606107.7%

21,000(Million yen)

Net sales

Profit attributable toowners of parent

Sales of pharmaceuticals and functional food,and each profit have shown strong progress.

7

R&D Pipeline

R&D Pipeline (Domestic) ①
Code No.
(Generic name)<Origin>Application
type

Indications

Preparation fordevelopment

PIPI

PII

PIII

NDA

LaunchPII

NS-065/NCNP-01(viltolarsen)

Preparation for

NME

Duchenne muscular dystrophy

NME

Iron deficiency anemia

NME

Dravet syndrome

NME

Lennox-Gastaut syndrome<in-house>

PIII
in progress

NS-32(ferric derisomaltose)<in-license>
ZX008(fenfluraminehydrochloride)<in-license>
ZX008(fenfluraminehydrochloride)<in-license>： Changes from 2nd Quarter 2021

9

R&D Pipeline (Domestic) ②
Code No.
(Generic name)<Origin>Application
type

Indications

Preparation for

Preparation fordevelopment

PIPI

PII

PIII

NDA

LaunchPIII

New
NS-304indication

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

NS-580Pediatric pulmonary arterial
hypertension

NME

Endometriosis（daunorubicin /New

Secondary acute myeloidcytarabine）

combination

leukemia

NME

Inflammatory diseases<in-house>
NS-87<in-license>
NS-229<in-house>

NS-917(radgocitabine)

NME<in-license>
NS-401(tagraxofusp)<in-license>

NME

Relapsed/refractory acute
myeloid leukemia

Blastic plasmacytoid dendritic
cell neoplasm

10

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

NS-065/NCNP-01(viltolarsen)

NME

Duchenne muscular dystrophy

NMEDuchenne muscular dystrophy

NME

Myelofibrosis<in-house>

CAP-1002<partnership>PI

Preparation for
PⅡ

PII

Preparation for
PIII

PIII

LaunchPIII
in progress

NS-018(ilginatinib)<in-house>： Changes from 2nd Quarter 2021

11Reference Materials

Consolidated Balance Sheet
End ofEnd of 3Q

YoY Change

End of

End of 3Q

YoY Change

FY2020

FY2021Amt

FY2020

FY2021

Amt(Million yen)

Assets197,028

204,795+7,766

Current assets

139,090

148,535+9,444Non-current assets

57,93756,259-1,677

Liabilities

34,48527,555-6,929Current liabilities

31,51424,508-7,005

Non-current liabilities

2,970

3,046

+75

162,543

177,240+14,696

197,028

204,795+7,766

Net assets
Total assets

197,028

204,795+7,766＝Assets＝Total liabilities
and net assets＝Liabilities and Net assets＝Notes and accounts receivable＋9,027

Income taxes payable-3,780Inventories+2,105

Provision for bonuses-1,615Investments and other assets-2,553Retained earnings+15,80413

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

・Japan : Launch・USA : Launch・China : NDA filing・Global : PIII in progress

OriginCo-development:
National Center of Neurology and PsychiatryDevelopment

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: NDA filing

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: Zogenix, Inc.

DevelopmentZogenix, Inc.

Mechanism of action

Serotonin agonist

Indication

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 oncombination therapy
16CAP-1002- Treatment for Duchenne muscular dystrophy Development Phase

USA : Preparation for PIII

Origin[Jan. 2022]
Commercial rights from: 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 mutation17

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 (PediatricPAH)

Dosage form

TabletFeature

Long-acting oral drug18

NS-580- Treatment for endometriosis Development PhaseJapan: PIIa

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 potency

19

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

USA: 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 count, for whom QOL
improvement can’t be obtained because no treatment isavailable

20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 cytarabine

Indication

Secondary acute myeloid leukemia (secondary AML)

Dosage form

Injection

Feature・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 marrow21NS-229- Treatment for inflammatory diseases Development Phase

Japan: PIOrigin

Nippon Shinyaku

Development

Nippon Shinyaku

Mechanism of action

JAK1 inhibitor

Indication

Inflammatory diseases (to be determined)

Dosage form

Oral agent

Feature・Potent and highly selective JAK1 inhibitor
・High efficacy and good safety profiles are expected inthe treatment for inflammatory diseases22NS-917 (radgocitabine)- Treatment for relapsed or refractory acute myeloid leukemia Development Phase

Japan: Preparation for 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 withr/r AML23

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

Japan: Preparation for clinical development

Origin[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

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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 clinicaltrials; claims and concerns about product safety and efficacy; regulatory agency’s examination, obtaining
regulatory approvals; domestic and foreign social security reforms; trends toward healthcare costcontainment; 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.