JCR Pharmaceuticals : FY2023 Results Briefing Session -Material-（1,196KB）

FY2023 Results Briefing Session

May 13th, 2024

JCR Pharmaceuticals Co., Ltd.

[Securities code]4552, Prime. TSE

Copyright © 2024 JCR Pharmaceuticals Co., Ltd. All rights reserved.

Key Messages

FY2023

Revenue and earnings growth from increasing sales of core products

FY2024

Strengthen organizational structure to support global clinical development and increase R&D investment

• Expand clinical development team and establish an integrated structure of experts in Japan, US, Europe and Brazil with JCR Europe playing a lead role
• JR-141：Complete patient enrollment for an interim analysis of the global Phase III
• JR-441：Phase I/II study on track (First patient dosed in Oct 2023)
• JR-446：Initiate Phase I/II study in 1st half of FY2024
• JR-171：Discussions on licensing-out ongoing

	Copyright © 2024 JCR Pharmaceuticals Co., Ltd. All rights reserved.	FY2023_4Q_1

Progress of Developmental Pipelines

Anne Bechet

Development (Acting Officer) General Manager, JCR Europe B.V.

Copyright © 2024 JCR Pharmaceuticals Co., Ltd. All rights reserved.

FY2023_4Q_2

JR-141 (pabinafusp alfa: BBB-penetrating ERT for MPS II)

Global Phase III study（JR-141-GS31): STARLIGHT study Overview

Overview

	1.	To assess the efficacy of JR-141 on CNS signs and symptoms in
Objectives		MPS-II subjects relative to standard ERT
2.	To assess control of somatic signs and symptoms by JR-141
		relative to standard ERT
	• Changes in HS in CSF, CNS symptoms (cognitive, behavior,
Endpoints		attention)
	• Control of systemic sign and symptoms
Clinical Trails.gov		Identifier : NCT04573023

Copyright © 2024 JCR Pharmaceuticals Co., Ltd. All rights reserved.

Current Status

Recruiting

Number of Clinical trial sites (as of Apr 2024):

• US: 5
• Europe: 12
• Latin America: 4

Trial is on going in 10 countries

• Further sites to open in EU, US, Latin America to accelerate recruitment

Achievements

Oct -2018ODD by FDA

Feb -2019ODD by EMA

Feb -2021 Fast Track Designation by FDA Oct -2021 PRIME Designation by EMA

Feb -2022 First Patient dosed in JR-141-GS31

Dec -2022 Rare Pediatric Disease Designation by FDA

• Completed enrollment of all 20 eligible patients in Cohort B
• Enrollment in Cohort A on track

FY2023_4Q_3

JR-441(BBB-penetrating ERT for MPS IIIA)

July 2023 - Started Global Phase I/II study (JR-441-101) in Germany

JR-441-101 study overview

Treatment	Drug
Extension period
period	holiday

Achievements and next milestones

Jan -2022

EC grants Orphan Drug Designation

Jul -2023

Dose 2

Screening

Dose 1

Baseline

6	Optimal dose *
weeks

• Optimal dose will be determined after all patients' 27-week data are obtained and analyzed.

week

53

59

261

Approval of Global Ph I/II Clinical Trial in Germany

Oct -2023

First Patient First dosed

Dec -2023

FDA grants Orphan Drug Designation

1st Half -FY2024

Last Patient In

2nd Half -FY2025

Overview

Objectives	Safety, dose finding, exploratory efficacy
No. of subjects	12 subjects (≥1 year and ≤18 years)
Clinical Trails.gov	Identifer : NCT06095388

1-year clinical data is expected

• The Phase I/II study is progressing well
• Started recruiting pediatric patients after approval from the independent data monitoring committee

	Copyright © 2024 JCR Pharmaceuticals Co., Ltd. All rights reserved.	FY2023_4Q_4

R&D Pipeline

Code

Indication

Status

Upcoming Milestones

				• Q1 FY2024: All patients enrolled
JR-141	MPS II (Hunter syndrome)	Global Ph3		necessary for interim analysis
				• ~FY2027: Approval in US, EU, Brazil
JR-171	MPS I (Hurler syndrome etc.)	Global Ph1/2 completed	• Extension study is ongoing
•	Licensing out under negotiation
JR-142	Pediatric GHD	Ph2 (Analysis completed)	•	FY2024: Phase 3
JR-031HIE	Hypoxic ischemic encephalopathy in neonates	Ph1/2 (Analysis completed)	•	TBD
				• 1st Half FY2024: LPI
JR-441	MPS IIIA (Sanfilippo syndrome type A )	Global Ph1/2	•	2nd Half FY2025: 1-year clinical data is
					expected
JR-446	MPS IIIB (Sanfilippo syndrome type B )		Pre-clinical	• Under preparation for clinical trial
	•	1st Half FY2024: FPI in Phase 1/2
JR-471	Fucosidosis		Pre-clinical	•	TBD

※Only high-priority projects in the clinical stage or soon to be in the clinical stage are listed in the above table.

	Copyright © 2024 JCR Pharmaceuticals Co., Ltd. All rights reserved.	FY2023_4Q_5

Next Wave of Technology Innovation

Hiroyuki Sonoda, Ph. D.

Vice President

Executive Director, Research Division

Copyright © 2024 JCR Pharmaceuticals Co., Ltd. All rights reserved.

FY2023_4Q_6

History of Technological Innovation

Advanced technology and know-how in biopharmaceuticals

developed since the company's formation in 1975

Stock solutions for COVID-19

Vaccine (AZD1222)

J-Brain Cargo® Tech

Biosimilars

Stem Cell

Therapies

Recombinant Proteins

Human-derived therapeutics

Development of proprietary

JCR

technologies

	Copyright © 2024 JCR Pharmaceuticals Co., Ltd. All rights reserved.	FY2023_4Q_7

Applying J-Brain Cargo® to Gene Therapy

Treatment with AAV expressing

JBC-fusion protein

AAV capsid

JBC	X
	Therapeutic
Gene cassette	protein X

Treatment with modified AAV

expressing JBC tag for direct brain penetration

X

JCR proprietary gene therapy technology applying J-Brain Cargo® Treatment strategies can be tailored depending on the disease characteristics

	Copyright © 2024 JCR Pharmaceuticals Co., Ltd. All rights reserved.	FY2023_4Q_8

Characteristics of Gene Therapy used J-Brain Cargo®

Treatment with AAV expressing	Treatment with AAV	General AAV gene therapy
JBC-fusion protein	carrying JBC surface tag

• Transgene product, JBC-X, can reach the CNS
• Mechanism of action has been established in enzyme replacement therapy

AAV/JBC-X

JBC

X

• JBC-AAV can cross the blood-	X	• High doses are
	required to penetrate
brain barrier	X
the CNS
• Applicable to a membrane
protein since treatment
protein can be expressed in
every organ including the
CNS		Blood-Brain Barrier

JBC-AAV/X	AAV/X

JBC

X

X

X

	Copyright © 2024 JCR Pharmaceuticals Co., Ltd. All rights reserved.	FY2023_4Q_9