Outline of Consolidated Financial Results
for the 3rd Quarter Ended December 31, 2019
February 5, 2020
NIPPON SHINYAKU CO., LTD.

3Q FY2019 Summary◆ Net sales：

88,113 million yen （+1.3%）◆ Operating profit：

15,466 million yen （-5.9%）◆ Ordinary profit：

16,277 million yen （-5.6%）

Profit attributable：

11,886 million yen （-6.4%）◆ to owners of parent(Million yen)2

Segmental Review - Pharmaceuticals 75,798＋1,092
Revenues from the licensing ofindustrial property rights-766+540+867

76,665（Million yen）
Ethical drugs
Revenues from the licensing ofindustrial property rights

Profit in co-promotion
Net sales

3Q FY2018

3Q FY2019

YoY Change

Results

Ratio

Results

Ratio

Amt%

59,93679.1%

61,02979.6%+1,092+1.8%

11,96315.8%

11,197

14.6%

-766

-6.4%

3,898

5.1%

4,438

5.8%

+540

+13.9%

75,798100.0%

76,665100.0%

+867

+1.1%

Although revenues from the licensing of industrial property rights decreased
due to the absence of milestone revenue from Uptravi, net sales increasedby 1.1% through growth of new products and profit in co-promotion.
3Three Main Fields of Focus(million yen)

PAH

Hematology

20,00023,000YoY Change
Approx. +14%

UrologyYoY Change
Approx. +33%

21,00010,00011,50010,500000

FY20183Q

FY20193Q

FY2019Forecast

FY20183Q

Uptravi (domestic)

VidazaOpsumit (Profit in co-promotion)

GazyvaAdcirca

Others

FY20193Q

FY2019Forecast

YoY Change
Approx. +3%

FY20183Q

FY20193Q

FY2019Forecast

Zalutia
Cialis
Eviprostat
OthersSales in the focus fields increased over the same period of the previous year,and have smoothly progressed toward FY forecasts.
4Segmental Review - Functional Food 11,221+110+70-5+51（Million yen）

3Q FY2018

11,448+227

3Q FY2019

YoY Change

Results

Ratio

Results

Ratio

Amt%

Protein preparations

7,439

66.3%

7,550

65.9%

+110

+1.5%

Preservatives

1,814

16.2%

1,884

16.5%

+70+3.9%

855

7.6%

850

7.4%

-5-0.6%

Others

1,112

9.9%

1,164

10.2%

+51+4.6%

Net sales

11,221100.0%

11,448100.0%+227+2.0%

Health food ingredients

Net sales increased by 2.0% through growth of protein preparations and preservatives.
5Operating profit
16,435+867+227-1,592-1,187+716-969

15,466（Million yen）

3Q FY2018

3Q FY2019

YoY Change

Results

Ratio

Results

Ratio

Amt%

87,019100.0%

88,113100.0%+1,094+1.3%(Pharmaceuticals)(75,798)(87.1%)(76,665)(87.0%)(+867)(+1.1%)(Functional Food)(11,221)(12.9%)(11,448)(13.0%)(+227)(+2.0%)

Operating expenses

70,58481.1%

72,64782.4%+2,063+2.9%

Cost of sales

39,09144.9%

40,68346.2%+1,592+4.1%

SG&A expenses

19,76522.7%

20,95223.7%+1,187+6.0%

R&D expenses

11,72713.5%

11,011

12.5%-716-6.1%

Operating profit

16,43518.9%

15,466

17.6%

-969

-5.9%

Net sales

6Profit attributable to owners of parent
12,698-969+224-221+154-811

11,886（Million yen）

3Q FY2018

3Q FY2019

Results

Results

YoY Change
Amt%

Operating profit

16,435

15,466-969-5.9%

Non-operating income

1,208

1,433

+224+18.6%

Non-operating expenses

399

621

+221+55.5%

Ordinary profit

17,24416,277-966-5.6%

Income taxes, etc

4,546

4,391-154-3.4%

12,698

11,886-811-6.4%

Profit attributable toowners of parent

7

Business Forecast for FY2019
FY2018FY20193Q

FY

3Q

Progress

FY

Results

Results

Resultsfor FY

Forecasts

87,019114,716

88,11376.0%

116,000(Pharmaceuticals)(75,798)(100,223)(76,665)(75.8%)(101,100)(Functional Food)(11,221)(14,492)(11,448)(76.8%)(14,900)

Operating profit

16,435

20,644

15,46673.6%

21,000Ordinary profit

17,244

21,54016,27774.0%

22,00012,69816,302

11,88672.0%

16,500(Million yen)

Net sales

Profit attributable toowners of parent

We look for operating profit, ordinary profit and profit
attributable to owners of parent to increase year on year.

8Status of Product Pipeline

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

Indications

NS-065/NCNP-01(viltolarsen)<in-house>
NS-73(defibrotide)<in-license>
NS-32(ferric derisomaltose)<in-license>
ZX008<in-license>

Preparation fordevelopment

PIPIIPI
NMENewindication

NMENMEPIII

NDA filingPII
Preparation
for PIII

Duchenne muscular dystrophy

Sinusoidal obstruction syndrome (prevention)

Iron deficiency anemia
Dravet syndrome
Lennox-Gastaut syndrome
Chronic thromboembolic pulmonary hypertension

NS-304(selexipag)<in-house>New
indication

Arteriosclerosis obliterans
Lumbar spinal stenosis

NS-580<in-house>
NS-17(azacitidine)<in-license>
NS-87<in-license>
NS-917<in-license>

NME
New
indication
New
combination
NME

PIII
PIII

Endometriosis

Acute myeloid leukemia

Secondary acute myeloid leukemia
Relapsed/refractory acute myeloid leukemia

10R&D Compounds (Overseas)
Code No.
(Generic name)<Origin>prulifloxacin<in-house>

Application
typeIndications

NME

Bacterial infections

NME

Duchenne muscular dystrophy

New

Chronic thromboembolic pulmonary

indication

hypertension

NME

Myelofibrosis

Preparation fordevelopment

PI

PII

PIII

NDA filing

NS-065/NCNP-01(viltolarsen)<in-house>
NS-304(selexipag)<in-house>
NS-018<in-house>

Preparation
for PIII11

Reference Materials

Consolidated Balance Sheet
End of

End of 3Q

YoY Change

End of

End of 3Q

YoY Change

FY2018FY2019Amt

FY2018FY2019Amt(Million yen)

Assets168,763172,719+3,956

Current assets

110,720

116,707+5,986

Non-current assets

58,04256,012-2,030Liabilities

33,57230,914-2,658Current liabilities

25,40623,807-1,599Non-current liabilities

8,165

7,106

-1,059135,190

141,805+6,615

168,763

172,719+3,956

Net assets
Total Asset

168,763

172,719+3,956＝Assets＝Total liabilities
and net assets＝Liabilities and Net assets＝Cash and deposits-2,902

Notes and accounts payable+3,984

Notes and accounts receivable+2,684

Accounts payable-1,722Inventories+5,619

Income taxes payable-3,126

Retained earnings+6,228Investment securities-961

Deferred tax assets-1,34013

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

・Japan: NDA filing・USA : NDA filing・Preparation for global PIII

OriginCo-development: National Center of Neurology and Psychiatry

Development

Nippon Shinyaku

Mechanism of action

Exon 53 Skipping

Indication

Duchenne muscular dystrophy

Dosage form

Injection

Feature・Improvement in symptoms and prevention of the diseaseprogression by recovery of dystrophin protein expression・Morpholino based oligonucleotide with possible high safety profileand maximized activity

14

NS-73 (defibrotide)- Prevention of hepatic sinusoidal obstruction syndrome Development Phase

Japan : PIII (prevention)

Origin[Mar. 2017]
Licensed-in from : Jazz Pharmaceuticals

Development

Co-development in Japan :Jazz Pharmaceuticals

Mechanism of action

Protective action on vascular endothelium, normalization ofthe coagulation/fibrinolysis balance

Indication

Sinusoidal obstruction syndrome (SOS)

Dosage form

Injection

Feature･SOS is a life-threatening complication which develops inpatients following HSCT, and in severe cases, leading todeath at high rates.
･NS-73 is the only drug for the treatment of SOS (EU/USguideline). It has been launched in various countries.
･NS-73 is the only drug for the prevention of SOS.
15

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

Japan: PIII

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 iron correctionin the majority of patients・Good safety profile with no dose dependent ADRs
・Minimal potential toxicity from release of labile iron due to tightiron binding in a matrix structure of interchanging isomaltosideand iron・No profound hypophosphatemia
16ZX008- Treatment for rare intractable epilepsy Development Phase

Japan: PIII

Origin[March. 2019]
Commercial rights from: Zogenix, Inc.

Development

Zogenix, 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-Gastaut syndrome
patients refractory to existing treatment options
・ZX008 can be used in combination with other drugs, asstandard of care for intractable epilepsy is based on
combination therapy.
17

NS-304 (selexipag)- Treatment for pulmonary hypertension, arteriosclerosis obliterans, lumbar spinal stenosis -Development Phase<CTEPH> Japan: PIII
Overseas: PIII
<ASO>
Japan: PIIb
<LSS>Japan: PIIa

OriginNippon Shinyaku

Development

・[Apr. 2008] Licensed-out to (outside Japan):
Actelion Pharmaceuticals Ltd. (Switzerland)
・Co-development in Japan:Actelion Pharmaceuticals Japan Ltd. (CTEPH)
・Overseas: Johnson & Johnson (CTEPH)
・Nippon Shinyaku (ASO)
・Nippon Shinyaku (LSS)

Mechanism of action

Selective IP receptor agonist

Indication

・Chronic thromboembolic pulmonary hypertension (CTEPH)
・Arteriosclerosis obliterans (ASO)
・Lumbar spinal stenosis (LSS)

Dosage form

TabletFeatureLong-acting oral drug18

NS-580- Treatment for endometriosis Development Phase

Japan: PIIaOrigin

Nippon Shinyaku

Development

Nippon Shinyaku

Mechanism of action

Inhibition of membrane-associated prostaglandin E synthase-1

IndicationEndometriosis

Dosage form

Oral agent

FeatureTreatment for endometriosis without hormonal effect and withpossible analgesic potency

19NS-17 (azacitidine)- Treatment for acute myeloid leukemia Development Phase

Japan: PII

Origin[Oct. 2006]
Licensed-in from: Celgene CorporationDevelopment

Nippon Shinyaku

Mechanism of action

Inhibition of DNA methylation, Cytotoxic effects

Indication

Acute myeloid leukemia

Dosage form

Injection

Feature・Standard Treatment for acute myeloid leukemia thatineligible for intensive chemotherapy
・Improvement for life prognosis

20NS-87- Treatment for secondary acute myeloid leukemia -Development Phase

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

Development

Nippon Shinyaku

Mechanism of action

Liposomal combination of cytrabine and daunorubicin

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 in bone marrow.

21NS-917- Treatment for relapsed or refractory acute myeloid leukemia -Development Phase

Japan: Preparation for Clinical Development

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 unique mechanism ofaction from other nucleoside analogs at low dose continuous
infusion・Tolerable safety profile available to elderly patients with r/r
AML22

Prulifloxacin- Quinolone antibacterial Japan
Licensee・Meiji Seika Pharma Co., Ltd.

Development phase・Launch (Dec. 2002) / Sword® Tablets

Overseas
LicenseeDevelopment phase

・Angelini (Italy)・Approval (Sep. 2004)
・Launch in Italy (Nov. 2004)
・Approval in European countries (Apr. 2005)

・Lee’s Pharmaceutical Holdings Ltd. (Hong Kong)

・NDA filing in China

・Algorithm (Lebanon)

・Launch in Lebanon (Jan. 2012)23

NS-018- Treatment for myelofibrosis Development Phase

Overseas (USA): PI/IIOrigin

Nippon Shinyaku

Development

Nippon Shinyaku

Mechanism of action

JAK2 inhibitor

Indication

Myelofibrosis

Dosage form

TabletFeature

・Highly selective for active form of JAK2
・Possibly best-in-class treatment for myelofibrosis

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 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 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, whichinclude, 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.