Chugai Pharmaceutical : Script (including Q&A)

CHUGAI PHARMACEUTICAL CO., LTD.

Information Meeting on EVRYSDI

September 27, 2021

Event Summary

[Company Name]	CHUGAI PHARMACEUTICAL CO., LTD.
[Company ID]	4519-QCODE
[Event Language]	JPN
[Event Type]	Special Announcement
[Event Name]	Information Meeting on EVRYSDI
[Fiscal Period]
[Date]	September 27, 2021
[Number of Pages]	52
[Time]	15:00 - 16:29
	(Total: 89 minutes, Presentation: 51 minutes, Q&A: 38 minutes)
[Venue]	Dial-in
[Venue Size]
[Participants]
[Number of Speakers]	2
	Hideto Kodaira	EVRYSDI Lifecycle Leader
	Dr. Kayoko Saito	Professor of Special Appointment, Institute
		of Medical Genetics, Tokyo Women's
		Medical University
[Analyst Names]*	Fumiyoshi Sakai	Credit Suisse Securities (Japan) Limited
	Seiji Wakao	JPMorgan Securities Japan Co., Ltd.
	Kazuaki Hashiguchi	Daiwa Securities Co. Ltd.
	George Zhou	Goldman Sachs Japan Ltd.
	Shinichiro Muraoka	Morgan Stanley MUFG Securities Co., Ltd.

*Analysts that SCRIPTS Asia was able to identify from the audio who spoke during Q&A.

Support
Japan	03.4405.3160	North America	1.800.674.8375
Tollfree	0120.966.744	Email Support	[e-mail]

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Presentation

Sasai: Thank you very much for joining us for today's information meeting on Evrysdi, a new treatment for spinal muscular atrophy.

I am Sasai of Chugai's Corporate Communications Department, and I will be your host for today's session. Thank you.

Due to the ongoing coronavirus pandemic, today's session will be conducted in the form of a conference call. You can also view the materials and video of this presentation through the URL included in your email invitations.

The audio will come from the conference call system, so please do not disconnect your phones during the presentation.

The agenda for today's meeting can be found on the web page and on the first page of the presentation materials. Today's presentation will follow that listed in the agenda.

Today, we have invited Professor Kayoko Saito, Professor of Special Appointment to the Institute of Medical Genetics, Tokyo Women's Medical University. An introduction to Professor Saito's work is included in today's presentation materials.

We will take your questions after the presentations have been completed.

First, I will hand over to Mr. Kodaira, Chugai's Lifecycle Leader for Evrysdi, who will give a summary of the treatment.

Support
Japan	03.4405.3160	North America	1.800.674.8375
Tollfree	0120.966.744	Email Support	[e-mail]

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Kodaira: Hello everyone. My name is Kodaira, and I am the Evrysdi Life Cycle Leader at Chugai.

Today, I would like to give an outline about Evrysdi Dry Syrup 60 mg.

The indication for this drug is spinal muscular atrophy. The name of the product is Evrysdi Dry Syrup 60 mg. The drug is available in a white box as shown below.

The product is delivered in the form of a dry syrup, in this brown bottle. The box includes an adapter and 2 syringes.

Evrysdi itself is a low molecular compound. It can be taken orally, and distributes throughout the body.

The drug can be mixed with water at pharmacies and medical institutions, dissolved, and then taken by patients.

Support
Japan	03.4405.3160	North America	1.800.674.8375
Tollfree	0120.966.744	Email Support	[e-mail]

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In the next few slides, I would like to explain the pathogenesis of SMA and the mechanism of action of Evrysdi.

SMA is a disease caused by a defect or mutation in the SMN1 gene. In healthy individuals, this gene functions normally, resulting in the production of functional SMN protein. Unfortunately, in patients with SMA, malfunction of the SMN1 gene means that this cascade does not work properly. As a result, patients with SMA need to depend on the SMN2 gene.

Humans have both the SMN1 and SMN2 gene. In the absence of treatment, the SMN2 gene undergoes a phenomenon called 'skipping.' When skipping occurs, the pre-mRNA is skipped during the translation process.

Support
Japan	03.4405.3160	North America	1.800.674.8375
Tollfree	0120.966.744	Email Support	[e-mail]

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