Extracted MPSII-related information in the Japanese version of “20221114 – 2 nd

Quarterly Financial Report 2023”

Date of discovery : 15 Nov 2022
Date of report : 18 Nov 2022
_____________________________________________________________________
Note: Please refer to the PDF version of Japanese report, attached together with this
Word document in the email. In the attached PDF of Japanese report, the parts being
translated are highlighted in yellow, and extracted and shown here in figures.

A. Operating results （ please refer to page 3/27 in the Japanese Report for the
original version, extracted part is shown in Figure 1）

Figure 1. Part A in the Japanese report

1. Operating results for the year under review

Net sales amounted to JPY 15,581 million (decreased by 45.1% HoH).
Sales of GROWJECT®, a recombinant natural human growth hormone, were
affected by the NHI price revision in April 2022. Although sales of renal anemia
treatments, which were also subject to NHI price revisions, declined significantly,
total sales of mainstay products were at the same level as in the same period of the
previous year, mainly due to the significant contribution of IZCARGO® for
intravenous infusion, which was included in the NHI price list in May 2022. Total
sales of non-mainstay products decreased HoH, mainly due to a decline in contract
revenue and the termination of AstraZeneca K.K.'s contract for the domestic
production of the active ingredient of vaccines against COVID-19, as planned.

2. Main Sales
From 1st Apr 2021 From 1st Apr 2022 Change
to 30th Sep 2021 to 30th Sep 2022
Sales (Million JPY) Sales(Million JPY) %
Growth Hormone 6,689 6,083 -9.1
MPS II Therapy
985 2118 114.8
Izcargo I.V. Infusion
Renal Anemia Therapies 3,011 2,157 -28.4
Epoetin Alfa BS Inj. [JCR] 1,512 1,392 -8.0
Darbepoetin Alfa BS Inj.
1,498 765 -48.9
[JCR]
Regenerative Therapy
1,717 1,701 -0.9
Temcell HS Inj.
Fabry Disease Therapy
Agalsidase Beta 323 521 61.0
BS I.V. Infusion
Contract Revenue 7,557 1,010 -86.6
AZD1222 Active Ingredient 8,046 1,931 -76.0
B. Status of Research and Development (please refer to page 4/27 in the Japanese
Report for the original version, extracted part is shown in Figure 2)

Figure 2. Part B in the Japanese report

Pabinafusp alfa (development number: JR-141), an enzyme preparation for the

treatment of Hunter syndrome that crosses the blood-brain barrier, was launched in
Japan in May 2021 (product name: IZCARGO® for intravenous infusion). In the US, it
received Fast Track (*1) designation from the US Food and Drug Administration (FDA)
in February 2021, and in Europe (EU), it received PRIME designation from the
European Medicines Agency (EMA) in October 2021. In February 2022, the first
patient was dosed in a global Phase III clinical trial. In December 2020, an application
for marketing authorization was submitted to the Brazil National Health Surveillance
Agency (ANVISA), but the application was not approved in August 2022. A new
application is planned to be submitted using the results of the global Phase III clinical
trial currently underway.
C. Research and Development Activities (please refer to page 6/27 in the
Japanese Report for the original version, extracted part is shown in Figure 3)

Figure 3. Part C in the Japanese Report

Total research and development expenditure for the first half of the year under
review amounted to JPY 4,216 million (previous half of the year: JPY 3,520 million).
The status of the Group's research and development activities in the first half of
the year under review is described in (1) Operating results.
Development No. Phase Indication Remarks
(Generic Name)
JR-141 Global Phase 3 MPS II Use Enzyme
(Recombinant (Hunter Replacement
blood-brain barrier Syndrome) Therapy [J-Brain
crossing iduronate Cargo]
2-sulfatase)
Omitted Below Omitted Below Omitted Below Omitted Below