EXTERNALNON-CONFIDENTIAL

Corporate Presentation
September 2024 | Nxera Pharma Co., Ltd. (TSE: 4565)

Disclaimer

The material that follows is a presentation of general background information about Nxera Pharma Co., Ltd and its subsidiaries (collectively, the “Company”) as of the date of thispresentation. This material has been prepared solely for informational purposes and is not to be construed as a solicitation or an offer to buy or sell any securities and should not
be treated as giving investment advice to recipients. It is not targeted to the specific investment objectives, financial situation or particular needs of any recipient. It is notintended to provide the basis for any third-party evaluation of any securities or any offering of them and should not be considered as a recommendation that any recipient should
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The information contained herein is in summary form and does not purport to be complete. Certain information has been obtained from public sources. No representation or
warranty, either express or implied, by the Company is made as to the accuracy, fairness, or completeness of the information presented herein and no reliance should be placedon the accuracy, fairness, or completeness of such information. The Company takes no responsibility or liability to update the contents of this presentation in the light of new
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This presentation contains “forward looking statements,” as that term is defined in Section 27 A of the U S Securities Act of 1933 as amended, and Section 21 E of the U S
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measures should be viewed in addition to, and not as a substitute for, analysis of the Company’s results reported in accordance with IFRS.
(c) Nxera Pharma Co, Ltd, 2024. Nxera and the Nxera logos are trademarks of Nxera Pharma Co. Ltd.

2

Agenda

01

02

03

04

05

06

Business Overview
Strategic Roadmap
Our Medicines and Pipeline
Our NxWaveTM Platform
Financial Results
Appendix3

Business Overview

01

OVERVIEW

STRATEGIC ROADMAPPRODUCT & PIPELINEPLATFORM

FINANCIALS

APPENDIX

Leading the Next Era of Medicine. From Japan, for Japan, and the world
World-leading NxWave

platform (UK), coupled with Japan’s most effective development and commercial organization

Our Mission

Our Vision

Our Values

To accelerate thedevelopment oflife-changing medicines,
by investing in scienceand technology.

To lead the next era of
medicine.

• Patients come first• Innovation and teamwork• Focus• Speed and agility• Operational excellence

From Japan, for Japan,
and the world.

5

OVERVIEW

STRATEGIC ROADMAPPRODUCT & PIPELINEPLATFORM

FINANCIALS

APPENDIX

We are Nxera Pharma
A technology-powered biopharma in pursuit of new specialty medicines to improve the lives of patients

Cutting-edge Science

Programs by Design

Real Human OutcomesWORLD-LEADERS IN GPCRSTRUCTURE-BASED DRUG DESIGN

30+ ACTIVE PROGRAMSPROTECTING LIVES EVERYDAY

Strong focus on GPCR targets – solved 375+
molecular structures

TSE: 4565

CNS

GI

39%

33%

IMM

Other

9%

18%

350+ FTE Employees

Tokyo Stock Exchange Prime

6

10,300+ patients have received Pivlaz®
(Japan and shortly South Korea)+4 other partnered marketed products

5 Global Locations

Revenue-Generating

Tokyo, Cambridge, London, Seoul& Basel$350m+ Cash in hand(Dec-2023)

OVERVIEW

STRATEGIC ROADMAPPRODUCT & PIPELINEPLATFORM

FINANCIALS

APPENDIX

Global Corporate StructureOver 350 team members employed across Japan, South Korea, UK and Switzerland

Nxera Pharma Co., Ltd.
Group Operations | ~50 people

Nxera Pharma UK Limited

Nxera Pharma Japan Co., Ltd.

(formerly “Heptares Therapeutics”)
Cambridge | ~170 staff
Research & Drug Discovery

Nxera Pharma Korea Co., Ltd.

(formerly “IPJ” and “Sosei Co.”)Tokyo | ~130 staff

Drug Development & Commercial Operations(formerly “IPK”)
Seoul | ~6 staff

Drug Development & Commercial Operations•

NxWaveTM – SBDD Platform•

Clinical Development•

Clinical Development•

Drug Discovery•

Regulatory Affairs•

Regulatory Affairs•

Translational Medicine•

Marketing Authorisation Holder•

Marketing Authorisation Holder•

Early Clinical Development•

Commercial Sales (direct and via partners)•

Commercial Sales (via partners)•

Business Development

7

OVERVIEW

STRATEGIC ROADMAPPRODUCT & PIPELINEDavid Roblin
Clin Dev

Kuniaki KagaClin Dev

Eiko Tomita
Reg Affairs

PLATFORM

FINANCIALS

APPENDIX

Agile and decisive leadership team
BOARD OF DIRECTORS
Chris Cargill
CEO

Shinichi Tamura
ChairmanTomohiro Toyama
LegalRolf Soderstrom
Finance

Noriaki Nagai
Compliance

Miwa Seki
Tech/ESGEXECUTIVE MANAGEMENT

Kieran Johnson
Chief Accounting Officer

Group Operations
Tokyo / London

Hironoshin Nomura
Chief Financial Officer

Toshihiro Maeda
Chief Operating Officer
Candelle Chong
Chief of Staff

Chris Cargill
Chief Executive Officer

UK Research & Development Operations
Matt Barnes
President of Nxera Pharma UK

Kazuhiko Yoshizumi
Chief Compliance Officer

Japan Development & Commercial Operations
Research &Early Development
Discovery / Preclinical / Phase I

8Development &Commercialization
Phase II / III / IV

Satoshi Tanaka
President of Nxera Pharma Japan

Strategic Roadmap

02

OVERVIEW

STRATEGIC ROADMAPPRODUCT & PIPELINEPLATFORM

FINANCIALS

APPENDIX

Our History
Strategic steps taken to build Nxera over the last two decades
2000s

2015

2023

Launched a public company
dedicated to bringing innovation toJapan

Out-licensed several programs to
global pharma to generate profit, acash reserve and a larger market
valuation

Elevated our status in the Tokyo
Stock Exchange, improving access toinstitutional investors✓

IPO on TSE (MOTHERS) in 2004✓

Made strategic acquisitions to bringsteady revenue throughgroundbreaking medicines✓✓$186m acquisition of Arakis Limited in
2005
Royalty revenues from Breezhaler®
medicines from 2012 to present

15+ partnered programs that generate
upfront and milestone revenue (plusfuture royalties)

Invested in research-focusedcompanies that could generate acontinuous pipeline of new
medicines✓✓✓

Promotion to TSE (PRIME) segment in
2023
First public healthcare investment by
the Japan Investment Corporation in
2023

Acquired a commercial-stage
pharmaceutical company which
provided an integrated platform foreven greater sustainable revenue
growth$400m acquisition of Heptares
Therapeutics Limited in 2015✓✓

$466m acquisition of Idorsia
Pharmaceuticals Japan and Korea
Rapidly growing revenues from sales of
PIVLAZ®

JAPAN
KOREA
10

2024

Launched new corporate
branding:

Nxera Pharma Co
With a vision to lead thenext era of medicine.
From Japan, for Japan, andthe world.

OVERVIEW

STRATEGIC ROADMAPPRODUCT & PIPELINEPLATFORM

FINANCIALS

APPENDIX

Building a fully integrated biopharma from Japan
Accelerating growth to achieve our mission by leveraging business platform in Japan and UK
2000s

2015

2023

2024
JAPAN
KOREA

Discovery / Preclinical

Phase 1

Clinical Trials
Phase 2Phase 3

Approved
Commercialized Medicines

United Kingdom

Invest in our NxWaveTM platform
2 to seed programsJapan

1 Acquire or in-license multiple de-risked medicines for Japan

11

OVERVIEW

STRATEGIC ROADMAPPRODUCT & PIPELINEPLATFORM

FINANCIALS

Our mission is clear
Accelerate the development of life-changing medicines

1

2

3

Acquire or in-license
multiple de-risked
medicines for Japan

Invest in our
NxWaveTM platformto seed programs

Build a first-classtechnology
environment

Focusing on these three areas is how we plan to make our mission happen as fast as possible
12

APPENDIXOVERVIEW

STRATEGIC ROADMAPPRODUCT & PIPELINEPLATFORM

FINANCIALS

APPENDIX

Japan will serve as our base to expand across APAC markets
Japan is an attractive, established market with strong volumes

Japan is the second
largest pharma
market (ex-China)

Tailwinds from nearterm regulatorychangesMarket size (USD bn)“ Japan Phase 1 Drug

(2021)

580
200

169

150
85

100
50

65

42

0
US

China

High quality clinicaland regulatoryenvironment✓ Excellent access toDoctors/HCPs who evaluate
novel drugs

Clinical Trials No
Longer Needed for
Global Clinical Trials”

Japan Germany France✓ Typically achievestrong patient uptake✓ Reduces drug loss and druglag for Japan patients

Source: IQVIA Market Prognosis, Sep 2022; IQVIA Institute, Nov 2022.
APAC (ex-China) territory includes South Korea, Australia, Brunei, Cambodia, Indonesia, Laos, Malaysia, Myanmar, New Zealand, Philippines, Singapore, Taiwan, Thailand andVietnam

13

APAC is the second
highest growth
pharma market
Market growth (CAGR %)
10.0%
8.0%
6.0%
4.0%

(2019 - 2027)

8.5%
7.0%

6.0%

5.5%
4.0%

2.0%
0.0%
Latin
APAC Africa & Europe North
America
MiddleAmerica
East

OVERVIEW

Priority objectives
for FY2024

STRATEGIC ROADMAPPRODUCT & PIPELINEPLATFORM

FINANCIALS

01

JPY 16 billion+ NHI sales for PIVLAZ®

ON-TRACK

02

JNDA approval for daridorexant in Japan

ON-TRACK03

Acquire/in-license at least one late-stage
medicine for Japan/APAC (ex-China)

ON-TRACK

04

Execute at least one new major partnership,
and initiate at least one new in-house Ph.1 study05

PMI investment in new brand concept, plus systems
and applications for efficiency and scalability
14

P

P

EP4 ag.

ON-TRACKAPPENDIXOVERVIEW

STRATEGIC ROADMAPPRODUCT & PIPELINEPLATFORM

FINANCIALS

APPENDIX

Achievements and several potential catalysts in 2024 and early 2025
Potential catalysts of in-house and out-licensed programs (excluding new business development transactions)
PROGRAM

P
P
P
P
P
P
P

TIMINGEVENT

EP4 AgAchieved (Mar. 2024)

Ph.1 start

GPR35 Ag

Achieved (Mar. 2024)

Program reversion

GPR52 Ag

Achieved (Mar. 2024)

Option-to-license agreement

NBI-568 (M4 Ag)

Achieved (Apr. 2024)

Long-term TOX study completed

NBI-567 (M1 pref. Ag)

Achieved (May 2024)

Ph.1 start

ORX750 (Ox2 Ag)

Achieved (May 2024)

Ph.1 startNBI-568 (M4 Ag)

Achieved (Aug. 2024)

Ph.2 topline data

Cenerimod

2H 2024

Exclusive opt-in decision

Lucerastat

2H 2024

Exclusive opt-in decision

Daridorexant (Sth Korea)

2H 2024

New Partnership & Ph.3 start

2H 2024

NDA Approval & Launch

ORX750 (Ox2 Ag)

2H 2024

Ph.1 completion & POC data

TMP-301 (mGlu5 NAM)

2H 2024

Ph.2 startNBI-568 (M4 Ag)

1H 2025

Ph.3 start

PIVLAZ® (Sth Korea)

1H 2025

New partnership (achieved) & Launch

Daridorexant (Japan)

PARTNER

1

1 Co-development and co-promotion agreement with Mochida15

Our Pipeline and Medicines
Programs by Design

03

OVERVIEW

STRATEGIC ROADMAPPRODUCT & PIPELINEPLATFORM

FINANCIALS

APPENDIX

Active Pipeline Overview
Discovery – Preclinical

IN-HOUSE

GPR35 ag. NXE’477
IBD

M1 pref. ag. NBI’567

Phase 1EP4 ant. NXE’732
Advanced
solid tumours

Phase 2Phase 3EP4 ag. NXE’744

Cenerimod

GPR52 ag. NXE’149

Lucerastat

IBD

Schizophrenia

CXCR4 mAb KY1051
Immuno-oncology
EDS in neurology

15+ PROGRAMSPARTNEREDMultiple

Multiple

Neurology

GI

Diabetes/Metabolic

Cerebral vasospasm

Oravi®

Mouth candidiasis

Seebri® Breezhaler®

M1 pref. ag. NBI’567

Enerzair® Breezhaler®

mGlu5 NAM TMP-301: Exclusive license-out option

GLP-1 ag. PF’522
Metabolicdiseases

OX2 ag. ORX750
Narcolepsy

Neurology

Neurology

Substanceabuse

AI-driven

PAR2 peptide Gut-brain axis

APAC*

M4 pref. ag. NBI’569

Schizophrenia

Technology collabs. (on-going)
AI-driven

PIVLAZ®

MC4 ant. PF’669

Neurology

M4 ag. NBI’568

APAC*

M1M4 ag. NBI’570

Malnutrition

Previous and ongoing
discovery collabs.

Fabry disease

APAC*

Commercial

CCR6 ant. PF’894
IBD

OX2 ag. ORX142

Daridorexant

Systemic lupus erythematosus Insomnia

Neurology

10+ PROGRAMS

APAC*

Filed

Neurology& Autoimmune

Note: Seebri®, Ultibro®, Enerzair® and Breezhaler® are registered trademarks of Novartis AG.
17
Pref. ag.: Preferring agonist*APAC (ex-China) territory includes South Korea, Australia, Brunei, Cambodia, Indonesia, Laos, Malaysia, Myanmar, New Zealand, Philippines, Singapore, Taiwan, Thailand and Vietnam

Ultibro® Breezhaler®
COPD

COPD

Asthma: Exclusive opt-in rightOVERVIEW

STRATEGIC ROADMAPPRODUCT & PIPELINEPLATFORM

FINANCIALS

APPENDIX

Our product: PIVLAZ®
Our first commercially available medicine protecting Japanese lives every day
Cum. patients to have received PIVLAZ®

JP GUIDELINES INCLUSION FOR MANAGEMENT OF STROKE1•

Aug '23: Authorized and recommended by the Japanese Stroke
Society•

Excellent efficacy demonstrated for prevention of cerebral
vasospasm following Subarachnoid Hemorrhage (SAH)•

Provides confidence to neurosurgeons to prescribe PIVLAZ® as
a new standard of care for SAH based on strong evidence it
can prevent delayed cerebral ischemia and poor outcomes

Q1

Q2

Dec '23: Received Marketing Approval in South Korea•

Early 2025: Commercially available to patients

Q4

NHI-based Sales
FY2022
JPY 7.5 Bn

MARKETING APPROVAL FOR SOUTH KOREA
•

Q3

1,846

Q1

Q2

Q6

FY2023
JPY 13.4 Bn
3,477

3,326
2,365

Q5

Q7

4,537

Q4

Q5

3,071

Q6

PIVLAZ® rapidly building real world evidence mitigating the risk of cerebral vasospasm
1 Japanese Stroke Society Guideline 2021 for the Management of Stroke (Revised Version 2023)

18

Q8

Q9

FY2024 (YTD)
JPY 6.5 Bn
3,747
2,752

2,322

Q3

14,036Q7Q8Q9OVERVIEW

STRATEGIC ROADMAPPRODUCT & PIPELINEPLATFORM

FINANCIALS

APPENDIX

In-house pipeline: Daridorexant
On-track for JNDA approval in H2 2024
About daridorexant•

Dual orexin receptor antagonist (DORA)
for the treatment of chronic insomnia•

Approved in the US, Europe, Canada(2022) – marketed as QUVIVIQ®

•

Positive results in Japan Phase 3 trial
reported in Oct 2022, and JNDA filingsubmitted in Oct 2023•

Market exclusivity until 2038 (Japan andSouth Korea)

DORA: rapidly establishing its position in insomnia treatment
Lemborexant (Dayvigo)
Suvorexant (Belsomra)(JPY Bn)

DORA share (patient base)

80

Sales (NHI-base)and market shareof DORAs

20

Source: Nikkei Medical (2022/7/23, 2024/4/13), IQVIA, Encise* Estimation

19

26.4
29.4

33.3
28.7

33.1

2019

1.4
2020

0

Most frequently
prescribed
sleeping pills

22%*

14%

12%

Jul. 2022
Apr. 2024

Others

19%

17%

60
40

ZolpidemEszopiclone

19%

16%
37%

DORA

27.6

11.3
2021

2022

25%

14%

12%

17%

11%

41.5

2023
26%
22%

OVERVIEW

STRATEGIC ROADMAPPRODUCT & PIPELINEPLATFORM

FINANCIALS

APPENDIX

Our track record of major licensing transactions speaks for itself…
Income from licensing provides a great source of non-dilutive financing to support our growthBalance of potential milestone income fromexisting license agreements1

Top 15 pharmaceutical/biotech companies by license value2(cumulative total since 2015)(USD million)(USD million)

12,0001,200(USD million)
0

10,000

Ionis Pharmaceuticals

8,000

10,00015,00020,000

Sosei Group

800

Eisai

600

Precision Biosciences
Galapagos

4,000

400

CytomX Therapeutics

5,000+
2,000

Hanmi Pharmaceutical
Skyhawk Therapeutics

200

Arrowhead Pharmaceuticals
Sangamo Therapeutics

00
FY15/3

FY16/3

Dicerna Pharmaceuticals

FY17/3 FY18/3 FY18/12 FY19/12 FY20/12 FY21/12 FY22/12
Commercial milestone(left axis)
Development milestone(left axis)

Genmab

1 Balance as of the end of the fiscal year of only those currently under contract. TEVA and Abbvie (formerly Allergan), for which compounds were returned, are excluded from the balances from FY2018 and FY2021, respectively.
2 The figures are based on ‘Licensing’ category on third party's (EvaluatePharma's) proprietary database and therefore do not completely match the amounts shown in the LHS chart.

Source: Company’s data (LHS) and EvaluatePharma (as of 2023/2/6) (RHS)

30,000

3rd out of 2,914

Daiichi Sankyo
PeptiDream

6,000

25,000AstraZeneca

1,000
5,000+

5,000

20

OVERVIEW

STRATEGIC ROADMAPPRODUCT & PIPELINEPLATFORM

FINANCIALS

APPENDIX… hundreds of millions of dollars received, billions of dollars in potential milestones to comeNew collaboration and exclusive option to license agreement recently executed with Boehringer Ingelheim
Partner

Execution

Program

Therapeutic Area(s)Upfront and Initial
Milestones

Potential Total
Milestone1

March 2024

Collaboration and exclusive option-tolicense agreement for GPR52 agonist

Schizophrenia

EUR25m

EUR670m

December 2022

Multi-target Collaboration

Diabetes and Metabolic

$37m

$800m

August 2022

Multi-target Collaboration

Neurological disorders$80m$1.2bnDecember 2021

Collaboration and license agreement for
M4, M1 and M1/M4 dual agonist

Neurological disorders$100m$2.6bnDecember 2020

Collaboration and license agreement for
GPR 35Gastrointestinal, immunology

$44m

$480m

December 2020

Collaboration and license agreement for
CGRP portfolio

Neurology

$10m

$380m

June 2020

Discovery Collaboration andOption to License2

$32m

$400m

August 2019

Multi-target Collaboration$26m$1.2bnJuly 2019

Multi-target Collaboration

Multiple

$26m

$1bn

November 2015

Multi-target Collaboration

Multiple-$1.8bnInflammatory andAutoimmune
Multiple; Initial focus on
Gastrointestinal

1Potential option fees, development, regulatory and commercial milestone payments agreed at the time of transaction. Nxera is also eligible to receive tiered royalties ranging from high single digit to mid-teen percentage on future net sales of any products developed

under the partnership. 2 AbbVie has the option to expand the collaboration by an additional three targets

21

Our NxWaveTM Platform
Cutting-edge Science04

OVERVIEW

NxWaveSTRATEGIC ROADMAPPRODUCT & PIPELINEPLATFORM

FINANCIALS

APPENDIX

platform is focussed on drugging GPCRs

GPCRs are the largest family of drug discovery targets – comprising 1/3 of all FDA approved drugs~400

GPCR targets
active indiseases2~34%

of FDAapprovals
target GPCRs1

27%

of global sales
are GPCR
drugs1~400 GPCR targets in the body active in disease2

NEUROLOGICAL DISORDERS

BEST-IN-CLASSFIRST- OR
BEST-IN-CLASSGASTROINTESTINAL DISEASES
Drugged27%

IMMUNOLOGY/ONCOLOGY

In trials
17%

METABOLIC DISORDERS
As yet undrugged
56%

CARDIOVASCULARRESPIRATORYFIRST-INCLASSGPCRs are active in a wide range of disease areas,
and offer broad therapeutic potential

Significant opportunity to target new first-in-class
and/or improved best-in-class GPCR medicines

Sources: 1 “Unexplored opportunities in the druggable human genome”, Nature Reviews, 2016 ; 2 “Trends in GPCR in Drug Discovery – new agents, targets and indications”, Nature Reviews, 201723

OVERVIEW

NxWaveSTRATEGIC ROADMAPPRODUCT & PIPELINEPLATFORM

FINANCIALS

platform enables faster, cheaper and more precise drug discovery

World-leading science and platform enables efficient drug discovery against difficult targets

Conventional drug discovery

Our drug discovery

Approach

Empirical design

Rational design (computer-based)

MethodHigh Throughput Screening (HTS1)

Proprietary NxWave Platform

Period2

4.5 years on average

3.0 years on average

Costs2$15 million$5 million

Features 3

Difficult to design drugs precisely– high development attrition rate

Execute more precise drug design– lower development attrition rate

Target 3

Difficult for GPCRs with unstable structures

Best for GPCRs with unstable structures

1 HTS/High Throughput Screening is a method to find drug candidates by reacting tens of thousands to millions of compounds with drug targets using large machines and human hands.
2 The period from target selection to preclinical testing. For conventional drug discovery, figures are taken from NATURE REVIEWS Drug Discovery (MARCH 2010).

3 Precise drug design make clear the binding site of target, make easier to improve compound, create backups and redo – potentially increase the success rate. GPCR is most popular drug target which account for 30% of current drug target.

24

APPENDIXOVERVIEW

STRATEGIC ROADMAPPRODUCT & PIPELINEPLATFORM

FINANCIALS

APPENDIX

Our platform enables to design precise GPCR models
Only by performing detailed structural analysis can we design great drugs.
Imprecise GPCR model: Standard Medicine

Precise GPCR model: Optimized Medicine

Poorly understood GPCRs (locks) led to suboptimal drugs(keys) being designed

High selectivity enables to optimize efficacy and minimizeside effects

Drug

Efficacy

Target- GPCR

Drug

Unintended side effects

Off-target

Optimal Efficacy

Target- GPCR

Off-target

25

No/minimal side effects

Off-target

Off-target

OVERVIEW

NxWaveSTRATEGIC ROADMAPPRODUCT & PIPELINEPLATFORM

FINANCIALS

APPENDIX

platform has proven to be more productive than conventional approaches

Trends in the number of programs per stage (cumulative)*

Number of programs* 2022 vs 2023

40

2022

2023

Drug discovery

20+

20+

Lead optimization

7

8

25

Preclinical

9

6

20

Clinical - Phase 1

7

11

15

Clinical – Phase 2

3

3

10

Clinical – Phase 3

0

0

5

Approval application

0

0

Approved

00

Lead optimization

35

Preclinical30

Clinical and beyond

0
2012

2013

2014

2015

2016

2017

2018

2019

2020

2021

2022

2023

* The number of programs here represents the number of all drug candidates generated to date from our NxWaveTM drug discovery platform by stage and includes programs that are not currently being actively developed by us or our partners due to lower priority.

26

OVERVIEW

STRATEGIC ROADMAPPRODUCT & PIPELINEPLATFORM

FINANCIALS

APPENDIX

Exposed to the fastest growing areas of medicine
Advancing with world-leaders in neuropsychiatry, metabolic disease, and sleep disorders
Neuropsychiatry• Oral, selective muscarinic M4
agonist (NBI-568) for
Schizophrenia. Phase 2 POC1
data readout expected Q3
2024• Option to licensecollaboration with BI for
FIC GPR52 agonists(NXE-149) advancing
through Phase 1• Most comprehensive portfolioof muscarinic agonists indevelopment globally, sourced
from Nxera• Potential tosimultaneously address
positive, negative andcognitive symptoms of
Schizophrenia

Metabolic disease• NxWaveTM SBDD
used by Pfizer (PFE)• Oral small
molecule GLP-1
agonist (PFE-522)
for Type 2 Diabetes
Mellitus

Sleep disorders• NxWaveTM SBDD
used by Eli Lilly &Co (LLY)• NxWaveTM SBDD used byCentessa
Pharmaceuticals (CNTA)• Multiple next-gen
oral small moleculetargets ongoing in
discovery• Oral small molecule
orexin 2 agonist (ORX750)
for Narcolepsy ongoing inPhase 1

Perfectly positioned with the best partners in the hottest areas of medicine
1. POC = Proof Of Concept27

Financial Results

05

OVERVIEW

STRATEGIC ROADMAPPRODUCT & PIPELINEPLATFORM

FINANCIALS

APPENDIX

Financial summary for 1H FY2024
Another period of successful business execution with a new collaboration and development milestone events
Revenue of ¥12,720m ($84m) vs. ¥2,146m ($16m) in the prior comparative period.

01

Revenue is higher primarily due to (i) the inclusion of PIVLAZ® sales in Japan (ii) a new ‘option to license’ transaction with
Boehringer Ingelheim signed in March 2024 (iii) $15m M4 long term tox milestone from Neurocrine (iv) $4.6m milestone
from Centessa and (v) $10m milestone from AbbVie

02

Core Operating Profit of ¥1,176m ($8m) vs. Loss of ¥2,720m ($20m) in the prior comparative period.
The change from Core Operating Loss to Profit is due to the increase in revenue per above, partially offset by an increase incosts, including additional core costs totaling ¥ 4,988m ($33m) relating to the inclusion of NPJ/NPK* in the scope of
consolidation in July 2023.

03

Operating Loss of ¥3,654m ($24m) vs. ¥4,168m ($31m) in the prior comparative period.
Amortization charge on intangible assets, PIVLAZ® inventory adjustment and integration costs were recorded as non-core
costs

04

¥51bn ($317m) cash balance as at June 30, 2024.
We have maintained a sufficient cash balance and investment capacity.

* NPJ: Nxera Pharma Japan Co., Ltd., NPK: Nxera Pharma Korea Co., Ltd.

29

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APPENDIX

Key financial indicators
Quarterly revenues are substantially higher than prior comparative quarters due to the inclusion of PIVLAZ® product sales,
revenue from the new Boehringer Ingelheim deal and several development milestones.
EXPLANATION
Latest result(JPY million)
12,000Upfront1

1,392

◼
◼

◼

No milestone events in 1H 2023.
$15m M4 development milestone from Neurocrine (April).
$4.6m milestone from Centessa (May).
A part of $10m milestone from AbbVie recognized as revenue (June).

Royalty / Other◼

Royalty income from product sales decreased

Product Sales◼

Includes PIVLAZ® sales.

◼

Inclusion of PIVLAZ® product supply costs.
Additional non-cash CoS charge relating to PIVLAZ® inventory.

◼

9,000

Revenue

4,872

6,000

◼

Milestone2

1,063

3,000

1,547
1,577

512
1,865

667
1,415

1H FY211H FY22 80

1H FY23 64

0

5,393

1H FY24(JPY million)
2,000

OP

1,000

Core OP

1,176

No new deals in the prior comparative period.
Option-to-license deal signed with Boehringer Ingelheim.

◼

Cost of Sales

◼

R&D

◼

0

Operating
Profit / Loss

(1,000)(2,000)(3,000)(802)(1,849)(4,000)(2,378)(3,804)(5,000)1H FY211H FY22◼

(2,720)(4,168)1H FY23◼

SG&A(3,654)
1H FY24

1 Upfront fee revenue recognised at deal inception

2 Milestone revenue recognised at milestone event + deferred revenue releases

30

◼
◼

Increase in investment in R&D activities
Inclusion of NPJ/NPK related R&D costs.
Inclusion of NPJ/NPK related SG&A costs.
Integration costs (incl. company name change).
Amortization of intangible assets (PIVLAZ®).

OVERVIEW

STRATEGIC ROADMAPPRODUCT & PIPELINEPLATFORM

FINANCIALS

APPENDIX

Breakdown of 1H 2024 result
Impact of Non-cash/Non-recurring costs on full-year result is more significant in 2024 due to theinclusion of Idorsia businesses(JPY million)

Revenue
Cost of Sales
+
SG&A
R&D

Legacy Business＋
*1

7,327

=

NPJ / NPK*2

5,393

Consolidated
Non-recurring
Non-cash
costs
=
＋
＋
P&L (Core)
Costs
12,720(4,290)(7,329)(4,143)(699)(4,842)Other income

626

1

627

OP/Core OP

771

405

Core OP 1,176

Idorsia &Integration
related
Costs
Other

12,720(1,619)A(3,039)Consolidated
P&L (IFRS)

B

(681)

Amortization - Product IP

PIVLAZ® inventory adjustment

C

(563)

(11,514)Integration

D(1,323)

D

(645)

Other

(5,487)627
OP(3,654)A Additional CoS charge for current PIVLAZ® stock. This impact will continue until around 3Q 2024.
B Amortization of intangible assets (currently relates to PIVLAZ®). Annual charge to increase to c. JPY 1,800m per year from 2025.
C Integration costs including IT system integration and Corporate rebranding.
D Amortization of other intangible assets (e.g. IP), depreciation (e.g. laboratory equipment), share-based payments and other restructuring costs.

*1 = Nxera Pharma Co. Ltd. (formerly Sosei Group Corporation) + Nxera Pharma UK Ltd (formerly Heptares Therapeutics Ltd.) + Sosei K.K*2 = Nxera Pharma Japan (formerly Idorsia Pharmaceuticals Japan) + Nxera Pharma Korea (formerly Idorsia Pharmaceuticals Korea)

31

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FINANCIALS

APPENDIX

Full year cost guidance
Incremental investment designed to deliver greater returns over the medium to long term
LATESTGUIDANCE(JPY million)
15,000R&D expenses (IFRS basis)¥12,000 to ¥14,000m

14,000~12,00010,0005,000

10,075
3,793

5,931

7,454

0
FY20

FY21

FY22

FY23

FY24

Inclusion of NPJ1/NPK2 cost◼

Inclusion of NPJ/NPK R&D costs for a full year

Strengthening capability◼

Investment in discovery and translational medicine capabilities◼
◼

At least 1 clinical trial initiation
Advancing in-house programs further in the clinic will deliver
higher out-licensing revenues

Advancing priority programs

S&M + G&A expenses (IFRS basis)(JPY million)¥18,000 to ¥20,000m

25,00020,00020,000~15,00018,00010,000
5,000
0

◼
◼

Inclusion of NPJ/NPK cost◼

Post-merger
integration

◼

9,965
3,435

3,940

4,377

FY20

FY21

FY22

1. Nxera Pharma Japan 2. Nxera Pharma Korea

FY23

FY24

32

Inclusion of NPJ/NPK SG&A costs for a full year
Increase in amortization charge (c. JPY 700 mil.)Increase in support for PIVLAZ® to drive growth,commercialization of Daridorexant in Japan and preparation forlaunch of PIVLAZ® in South Korea (c. JPY 2,000m)
Costs relating to the acquisition of NPJ/NPK (post-merger
integration) are expected in 2024 (c. JPY 1,000m)

Appendix

06

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STRATEGIC ROADMAPPRODUCT & PIPELINEPLATFORM

Japan will serve as our base to expand across APAC markets
APAC is one of the most rapidly growing markets in the world
Established market with strong volumes
Second largest pharma
market (excl. China)
580

Attractive market for newcomers

85

Large, ageing population

65

42

0
US

APAC*

China

Japan Germany France

Stable, pro-innovation market

One of the fastest growing pharma regions globally
Second highest growth
pharma market
2019-2027, CAGR(%)

10.0%

Relatively weak incumbents

2021, Billion USD
169

200

Universal health care system

8.5%

7.0%

6.0%

5.5%

5.0%

4.0%

0.0%
Latin
America

APAC

Africa &Middle East

EuropeNorth
America

Significant population growth
Developing GDP/economies
Attractive market for newcomers
Large, ageing population
Accessible via other regulatory approvals

Source: IQVIA Market Prognosis, Sep 2022; IQVIA Institute, Nov 2022.
*APAC (ex-China) territory includes South Korea, Australia, Brunei, Cambodia, Indonesia, Laos, Malaysia, Myanmar, New Zealand, Philippines, Singapore, Taiwan, Thailand and Vietnam

34

Japan

FINANCIALS

APPENDIXOVERVIEW

STRATEGIC ROADMAPPRODUCT & PIPELINEPLATFORM

FINANCIALS

APPENDIX

Utilizing Japan's high quality clinical data in development and marketing
Expanding into APAC by leveraging clinical innovations based on Japan's high-quality data
Quality Clinical Development

Deep
understanding of
disease andtreatment by
Doctors/HCPs

High quality datafrom clinicalstudies throughto Post Marketing
Surveillance

Quality Regulatory Environment

High penetration
in of patient
population
duringcommercial
phase

Excellent access to Doctors/HCPs
who evaluate novel drugs
Typically achievestrong patient uptake
Reduces drug loss/lag
for Japan patients

35

Reasonable NHI
price forreimbursement
supported byhigh quality
clinical trial and
PMS data

Prolongation ofpatents via
extended clinicaldevelopment

Regional
optimization
makes clinical
trials cheaperand faster to
executeOVERVIEW

STRATEGIC ROADMAPPRODUCT & PIPELINEPLATFORM

FINANCIALS

APPENDIXPIVLAZ®
Positive top-line results from Japan specific registration program
RESULTS OF TWO PIVOTAL PHASE 3 STUDIES IN JAPAN1•

Event rate (%)

PIVLAZ® (clazosentan) demonstrated significant reduction ofvasospasm-related morbidity and all-cause mortality in patients
following aSAH (primary endpoint)p=0.0001

50

Clazosentan showed a numerical reduction of all-cause morbidity andmortality in both studies. The effect of clazosentan on this endpoint wassignificant (p<0.05) in a pre-planned pooled analysis

40•

Encouraging positive trends were observed on long-term measures ofclinical outcome (GOSE and mRS) at week 12

20•

There were no unexpected safety findings•

Results published in the Journal of Neurosurgery: Endo H, et al. April 01,
2022; DOI: 10.3171/2022.2.JNS212914•

CLIPPING STUDYCOILING STUDY

30

39.6

p=0.0055
28.8

16.2

13.6

10
0

Placebo(n=111)

Clazosenta
n 10mg/h(n=103)

Placebo(n=106)

Clazosenta
n 10mg/h(n=105)PIVLAZ® significantly reduced vasospasm-related morbidity and all-cause morbidity and mortality in domestic Phase
3 trials. It is a highly impactful medicine used to prevent death and disability after aSAH.
1 Two prospective, multicenter, double-blind, randomized, placebo-controlled, pivotal Phase 3 studies assessing the efficacy and safety of clazosentan in reducing vasospasm-related morbidity and all-cause mortality events in adult Japanese patients post-aSAH,

were conducted in parallel in 57 neuro surgical centers in Japan. Patients were randomized 1:1 to receive continuous infusion of either 10 mg/hr of clazosentan or placebo within 48 hours of the onset of aSAH for up to a cumulative maximum of 15 days after aSAH.
Protocols were identical, each study enrolling 221 patients, except for the securing intervention, which was either endovascular coiling (JapicCTI-163369; the “coiling study”) or surgical clipping (JapicCTI-163368; the “clipping study”)

36

OVERVIEW

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FINANCIALS

APPENDIX

Daridorexant – Global And Japan-Specific Program
Positive Japanese Phase 3 study; in-line with US study as published in The Lancet1
TWO PRIMARY ENDPOINTS FULLY MET IN GLOBAL PHASE 3 TRIALRESULTS OF GLOBAL AND JAPANESE PIVOTAL TRIALS1

Placebo•

A Japanese Phase 3 trial1 in 490 adult and elderly patients met both primary andsecondary efficacy endpoints, with similar results to the global study published inLancet Neurology

The rate of adverse events was comparable between placebo and daridorexant•

In the global trial, daridorexant also demonstrated significant improvement indaytime sleepiness, which means patients reported feeling less mentally andphysically tired, less sleepy and more energetic during the day•

Month 1

Daridorexant significantly improved total sleep time (sTST, p<0.001 for 50 mg dose)and significantly improved latency to sleep onset (sLSO, p<0.001 for 50 mg) v
placebo at 28 days

•

50mg

WAKE AFTER SLEEP ONSET (WASO)

Submission to the PMDA based on the global and Japanese data is planned for 2H
2023

Mean of observed values (min)

•

25mg

Month 3Month 1Month 3

-6.2
-11.1
-19.1
-18.4

-23.1
-23.0

-28.2

p<0.0001

-31.2
-29.0

p<0.0001

1The global study published in the Lancet Neurology is Mignot E, et al. Lancet Neurol 2022; 21: 125–39. The Japanese study(JRCT2031200452) was a randomized, double-blind, placebo-controlled, Phase 3 study to investigate the efficacy and safety of
daridorexant. 490 randomized adult and elderly patients (30.1% ≥ 65 years) with insomnia disorder received receive 50 or 25 mg
doses of daridorexant or placebo once daily for 28 days.

LATENCY TO PERSISTENT SLEEP (LPS)

p<0.0001

p<0.0001

-30.7

-29.4

-34.8
P=0.0005
p<0.0001

P=0.0015
p<0.0001

Daridorexant significantly improves wake after sleep onset, latency to persistent sleep, subjective total sleep time,and next-day sleepiness/daytime functioning (as measured by IDSIQ sleepiness domain) compared to placebo
37

OVERVIEW

STRATEGIC ROADMAPPRODUCT & PIPELINEPLATFORM

FINANCIALS

APPENDIX

Cenerimod and Lucerastat
Exclusive opt-in rights for two potentially exciting product opportunities

Cenerimod

Lucerastat

Indication

Systemic Lupus Erythematosus (SLE)

Indication

Fabry Disease

MoA

Selective S1P1 receptor modulator

MoA

Glucosylceramide synthase inhibitor

Stage

Global Ph3 studies ongoingStage

Number of Patients~120,000 in Japan

Major therapies*(Japan)Total Market Size : c.300 Oku JPY• Benlysta (GSK, 50~100 Oku JPY est. peak sales)• Saphnelo (AZ, 50~100 Oku JPY est. peak sales)• Plaquenil (Sanofi, ~50 Oku JPY)

• Phase 3 (MODIFY) study primary endpoint (neuropathicpain) not met, however, renal function andechocardiography secondary endpoints were positive• Open Label Extension (OLE) study ongoing

Number of Patients~1,000 in Japan• Potential to be the first oral, disease-modifying SLEtherapy that acts by reducing circulating T and B cells
early in the immune cascade• S1P1 modulation is a well-established mechanism inother diseases, such as MS (Gilenya, Zeposia)• Broadly-applicable mechanism means potential toexpand to other autoimmune diseases

Major therapies*(Japan)Total Market Size : c.300 Oku JPY• Replagal (ERT, Takeda, ~140 Oku JPY)
• Fabrazyme (ERT, Sanofi, ~100 Oku JPY)
• Galafold (PCT, Amicus, ~46 Oku JPY)

Value proposition• Potential to provide a broadly-applicable oral
monotherapy option as an alternative to IV enzyme
replacement therapy (Galafold is currently the only
available oral therapy, and applicable to patients withcertain rare mutations)

Value proposition

Small opt-in fee to license each program, with Nxera responsible for all development plans and future costs in the territory. If
successfully commercialized, Nxera is obligated to pay tiered single digit royalties to Idorsia for each product.
Source: *Estimate from Evaluate Pharma; JMDC; Datamonitor
ERT: Enzyme replacement therapy; PCT: Pharmacological chaperone therapy

38

OVERVIEW

STRATEGIC ROADMAPPRODUCT & PIPELINEPLATFORM

FINANCIALS

APPENDIX

Exclusive Opt-in Rights And ROFN/ROFR1
Option to develop up to seven clinical programs for Japan and APAC (ex-China) from Idorsia

Exclusive
Opt-in Right

ROFR/ROFN1

ProgramMechanism of Action

Indication

Stage

Cenerimod

S1P1 receptor modulator

Systemic lupus erythematosus

Phase 3

Lucerastat

Glucosylceramide synthase inhibitor

Fabry disease

Phase 3Selatogrel

P2Y12 antagonist

Suspected acute myocardial infarction

Phase 3*

ACT-1004-1239

ACKR3 / CXCR7 antagonist

Multiple sclerosis andother demyelinating diseases

Phase 2*

ACT-1014-6470

C5aR1 antagonist

Immune-mediated disorders

Phase 1*IDOR-1117-2520

Undisclosed

Immune-mediated disorders

Phase 1*

ACT-777991

CXCR3 antagonist

Recent-onset Type 1 diabetes

Phase 1*

1 ROFN/ROFR - Right of first negotiation / Right of first refusal

2 Territories include Japan, South Korea, Australia, Brunei, Cambodia, Indonesia, Laos, Malaysia, Myanmar, New Zealand, Philippines, Singapore, Taiwan, Thailand and Vietnam* Global Phase

39

RegionAPAC(ex-China)2

OVERVIEW

STRATEGIC ROADMAPPRODUCT & PIPELINEPLATFORM

FINANCIALSCore Operating Profit - Definition
Core Operating Profit/Loss – a financial indicator closer to the reality of our business
Operating Profit

Operating Profit“Core”◼ Core Operating Profit/ Loss is a key

financial indicator that highlights theunderlying recurring cash generating
capability of our business.
◼ Core Operating Profit/Loss isdefined as IFRS Operating Profit +material Non-cash costs + material
non-recurring costs◼ Material Non-cash Costs include
depreciation, amortization, sharebased payments and impairment.
◼ Material Non-recurring Costs
include restructuring costs, M&A
related professional fees and othermaterial one-off items.

“IFRS”+ Material Non-cash Costs(Depreciation, Amortization, Share based payments, Impairment...etc.)+ Material Non-recurring Costs(Restructuring costs and Other material one-off items...etc.)

Non-cash(Material)

Cash
Recurring

Costs under“Core”

Non-recurring(Material)

Costs under “IFRS”

40◼ Financial results recorded andprepared in accordance with
International Financial ReportingStandards (IFRS)APPENDIXOVERVIEW

STRATEGIC ROADMAPPRODUCT & PIPELINEPLATFORM

FINANCIALS

APPENDIX

Estimation of potential market size
Multi-billion USD annual peak sales potential for our post-pre-clinical pipeline
Category

Neurologicaldisorders

Immunologicaldisorders

Indication2

Number ofPatients

Our Candidates
Market Size

Individual Products

Dementia~55 million$7.3 billion (2010)$3.9 billion (2009/Aricept)

M1 agonist, M1/M4 agonist

Schizophrenia~20 million$20.7 billion (2011)$5.7 billion (2013/Abilify)

M4 agonist, M1/M4 agonist, GPR52 agonist--

mGlu5 NAM

Substance use disorders~10.4 million1--

Narcolepsy~3 million$2.3 billion (2022)$1.7 billion (2020/Xyrem)

OX2 agonist

Other

-

-

-

CGRP antagonist, GPR52 agonist

Cancer~42 million$178.9 billion (2022)$21.0 billion (2022/Keytruda)

A2a antagonist, EP4 antagonist, CXCR4 mAb

IBD~10 million$23.5 billion (2022)$7.5 billion (2022/Humira)

CCR6 antagonist, GPR35 agonist, EP4 agonist

Atopic Dermatitis~13.3 million$8.1 billion3 (2022)$7.0 billion (2022/Dupixent)

H4 antagonist, PAR2 mAb

T2DM/Obesity~420 million$58.3 billion (2022)$8.8 billion (2022/Ozempic)

GLP1 agonist

Anorexia~10 million-

MC4 antagonist

-

-

Other

Total

-

-

~$299 billion/year~$56 billion/year

Source (Number of patients)：World Health Organization, Evaluate Pharma, The European Federation of Crohn‘s & Ulcerative Colitis Associations (EFCCA), Narcolepsy Network, Inc., GBD 2015 Disease and Injury Incidence and Prevalence Collaborators (October2016). “Global, regional, and national incidence, prevalence, and years lived with disability for 310 diseases and injuries, 1990-2015: a systematic analysis for the Global Burden of Disease Study 2015”. Lancet. 388 (10053): 1545–1602 1 The number of patients withdrug addiction
Source (Peak Sales):Sales of each indications are extracted form Evaluate Pharma’s data of sales by disease and sales by individual products (as of 30 June. 2022). 2 Nxera may target one segment in the market for specific diseases. 3 Since there is no applicableindication category, the market size of “Eczema” is stated. Current market size for Atopic Dermatitis may be larger than stated above.

41

OVERVIEW

STRATEGIC ROADMAPPRODUCT & PIPELINEPLATFORM

PCC

Ph2

FINANCIALS

APPENDIX

Partnered pipeline (1/2)
CompoundTarget / Mechanism of Action

Modality

Indication

Partner

Disc.

Ph1

Partnered
Seebri® Breezhaler®

LAMA

SME

COPD

Ultibro® Breezhaler®

LAMA+LABA

SME

COPD

Enerzair® Breezhaler®

LAMA+LABA+ICS

SME

AsthmaORAVI®

Antifungal agent miconazole

SME

Oropharyngeal candidiasis

Imaradenant1

Adenosine A2a ant. combo

SME

mCRPC

NBI-1117568

Muscarinic M4 agonist

SME

Schizophrenia

NBI-1117569

Muscarinic M4 preferring agonist

SME

Neurology diseases

NBI-1117570

Muscarinic M1/M4 agonist

SME

Neurology diseases

NBI-1117567

Muscarinic M1 preferring agonist

SME

Neurology diseases

PF-070815322

GLP-1 agonist

SME

T2DM/ObesityPF-07054894

CCR6 antagonist

SME

Inflammatory bowel disease

PF-07258669

MC4 antagonist

SME

Malnutrition

PF-06954522

GLP-1 agonist

SME

Metabolic diseases(Not disclosed)

CGRP antagonist

SME

Neurology diseases(Not disclosed)

Multi target

SME/LME

Multiple indications(Not disclosed)

Multi target

SME/LME

Gastrointestinal and other(Not disclosed)

Multi target

SME

Neurology(Not disclosed)

Multi target

SME

Diabetes/Metabolic

Note: SME = small molecule. LME = large molecule. Seebri®, Ultibro®, Enerzair® and Breezhaler® are registered trademarks of Novartis AG. 1 AstraZenenca have removed the A2a program from their clinical pipeline as at Q3 2021 2 Pfizer decided not tocontinue develop lotiglipron (PF-07081532) in Q2 2023.

42

Ph3

App

Mkt

OVERVIEW

STRATEGIC ROADMAPPRODUCT & PIPELINEPLATFORM

PCC

Ph2

FINANCIALS

APPENDIX

Partnered pipeline (2/2)
CompoundTarget / Mechanism of Action

Modality

Indication

Partner

KY1051CXCR4 mAb

mAb

Immuno-oncology(Not disclosed)

PAR-2

PeptideInflammatory diseases(Not disclosed)

AI-Augmented Drug Discovery

SME

Neurology diseases(Not disclosed)

Multi target AI-powered

SME/LME

Immune diseases(Not disclosed)

Gut-brain axis drug discovery

SME

Gastrointestinal disorders

Co-development

Co-owned companies
TMP301mGlu5 NAM

SME

Substance use disorders

ORX750OX2 agonist (Oral)

SME

Narcolepsy

ORX142OX2 agonist (Oral)SME

EDS in neurology

Note: SME = small molecule. LME = large molecule

43

Disc.

Ph1

Ph3

App

Mkt

OVERVIEW

STRATEGIC ROADMAPPRODUCT & PIPELINEPLATFORM

PCC

Ph2

FINANCIALS

APPENDIX

In-house pipeline
CompoundTarget / Mechanism

Modality

Indication

Originator

PIVLAZ®

ETA antagonist

SME

Cerebral vasospasm

Daridorexant

Dual Orexin antagonist

SME

Insomnia

NXE’149

GPR52 agonist

SME

Neurology diseases

NXE’732

EP4 antagonist

SME

Immuno-oncology

NXE’744

EP4 agonist

SME

Inflammatory bowel disease

NXE’477 2

GPR35 agonist

SME

Inflammatory bowel disease(Not disclosed)

Muscarinic M1 agonist (JP)SME

Neurology diseases(Not disclosed)1

H4 antagonist

SME

Atopic Dermatitis(Not disclosed)

SARS CoV-2 Mpro

SME

Coronaviruses

Multiple programs

Not disclosed

SME/LME

Neurology diseases

Multiple programs

Not disclosed

SME/LMEGI and Inflammatory diseases

Multiple programs

Not disclosed

SME/LME

Immunology diseases

In-house Programs

In-house Programs (No longer internally funded. Targeting academic / industrial partnership)
NXE’310

SSTR5 agonist

PeptideHypoglycaemic disorders

NXE’097

GLP-1 antagonist

PeptideHypoglycaemic disorders

NXE’023

Dual GLP-2/GLP-1 agonist

PeptideIntestinal failure/NASH(Not disclosed)

Apelin agonist

PeptidePulmonary Arterial Hypertension

NXE’641

Dual orexin antagonist

SME

Insomnia and sleep disorders(Not disclosed)

PAR-2 mAb

mAb

Atopic Dermatitis/Pain

Note: SME = small molecule. LME = large molecule. 1 Due to changes of strategy, we deprioritized until we will find another indication opportunity 2 : Exclusive license-out option

44

Disc.

Ph1

Ph3

App

Mkt

OVERVIEW

STRATEGIC ROADMAPPRODUCT & PIPELINEPLATFORM

FINANCIALS

APPENDIX

Glossary
Basic Terminology/Technology
GPCR

G Protein-Coupled Receptor

There are about 800 types of GPCRs in the human body. While 400 of them are known to be potential drug targets, about 300 of them are not yet drugged

StaR

Stabilized Receptor

Nxera’ proprietary technology to stabilize a GPCR by engineering a small number of single point mutations outside of the ligand-binding site. It enables to identify the structure of GPCRs to be used
for SBDD drug discovery as well as antibody drug discovery as antigens

SBDD

Structure-Based Drug Design

A method to design drugs on a computer base based on the analysis of the three-dimensional structure of the drug target (e.g., protein receptor)

TPD

Targeted Protein Degradation

Drugs that promote the degradation of target proteins (e.g., receptors) in cells and aim for therapeutic effects by reducing disease-causing proteins

PAM

Positive Allosteric Modulator

A regulator that binds to unusual active sites (allosteric sites) on the receptor to increase the affinity and effect of the agonist

NAM

Negative Allosteric Modulator

A regulator that binds to an unusual active site on the receptor (allosteric site) and reduces the affinity and effectiveness of the agonist

Ag

AgonistA therapeutic drug that binds to a receptor and activates an intracellular signaling system similar to biological substances

Ant

Antagonist

A therapeutic drug that suppresses biological reactions by binding to receptors and preventing them from binding to biological substances

PK

Pharmacokinetics

Research and testing on the relationship between drug dosage and blood concentration. Mainly describes the rate process of ADMEPD

Pharmacodynamics

Research and testing on the relationship between drug concentration and pharmacological effects

ADME

Absorption, Distribution, Metabolism and Excretion

A series of process in the absorption of drugs into the body, distribution within the body, metabolism in the liver and other organs, and excretion in the kidneys and other organs

POM

Proof of Mechanism

Proof of mechanism of action, mainly through biomarkers. It can suggest the possibility of efficacy in fewer cases than POC

POC

Proof of Concept

Proof of a therapeutic concept, primarily through clinical efficacy and safety

AchAcetylcholine

A neurotransmitter released from the peripheral parasympathetic and motor nerves to transmit nerve stimuli

IND

Investigational New Drug

Information packages for development candidates to be submitted to the U.S. Food and Drug Administration (FDA) at the time of initiation of clinical trials

Ph1

Phase1

A study in humans. The main purpose is to confirm the safety of the drug candidate mainly by healthy volunteers.

Ph2

Phase2A study in humans. The main purpose is to confirm the efficacy of the drug candidates on a small scale (however, the number of patients varies greatly depending on the disease)

Ph3

Phase3A study in humans. The main purpose is to determine the efficacy of the drug candidates on a large scale (however, the number of patients varies greatly depending on the disease)

NDA

New Drug Application

An application to the U.S. Food and Drug Administration (FDA) for approval to market a new drug

LAMALong Acting Muscarinic Antagonist

An inhalant that dilates bronchial tubes and improves respiratory function by inhibiting the action of acetylcholine receptors (M3), which increase parasympathetic nerves.

LABA

Long Acting Beta2-Agonist

An inhalant that improves respiratory function by stimulating sympathetic beta2 receptors to dilate the bronchi.

ICS

Inhaled Corticosteroid

An inhalant that suppresses airway inflammation to prevent coughing attacks and other symptoms caused by asthma, also promotes the action of beta 2 stimulants and improve airway
hyperresponsiveness.

mCRPC

Metastatic Castration–Resistant Prostate Cancer

Cancer that has spread (metastasized) beyond your prostate gland and for which hormone therapy is no longer effective in stopping or slowing the disease.

COPD

Chronic Obstructive Pulmonary Disease

A group of diseases that causes damage to the bronchi and lung due to smoking or inhalation of toxic substances, resulting in breathing problems.

AD

Alzheimer’s Disease

Alzheimer's disease is a progressive neurologic disorder that causes the brain to shrink (atrophy) and brain cells to die, the most common cause of dementia .

DLB

Dementia with Lewy Bodies

Protein deposits, called Lewy bodies, develop in nerve cells in the brain regions involved in thinking, memory and movement (motor control), the second most common type of dementia.

Disease/Drug45

Locations

Midtown East,
9-7-2 Akasaka
Minato-ku
Tokyo 107-0052

F17, 410 TeheranRo
GangHam-Gu
Seoul 06192

Steinmetz Building
Granta Park,
Cambridge
CB21 6DG

Spaces Grosspeter
Tower,
Grosspeteranlage
29,4052 BaselJapan

South Korea

United Kingdom

Switzerland46

Thank you

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