Sakura Star Study

Chugai NMO Clinical Trial Webinar

8:00 a.m. PST, 11 a.m. EST

Friday, 12/12/14

Webinar Format

Session is 45 minutes in length (Welcome, Intro, Presentation, Q&A)

Attendees may submit questions in the Q&A window online - please be as succinct as possible if you choose to submit a question

After the presentation is complete, the presenters & panelists will address as many questions as time allows

Thank you for understanding that not all questions will be addressed

Webinar slides and audio will be posted on the foundation Website for later viewing/audio.

Athos Gianella-Borradori, M.D.Chief Medical OfficerChugai Pharma USA

Michael Yeaman, Ph.D.Advisor, The Guthy-JacksonCharitable Foundation

Brian Weinshenker, M.D.Expert NMO ClinicianMayo Clinic

Presenter Panelist Moderator

Presentation Format Content is solely that of the respective industry or its representative

Presenters are afforded a maximum of 15-20 minutes total time

Questions / answers are then afforded up to 20 minutes of time

The webinar is being recorded for purposes of future distribution

All perspectives are offered only for stakeholder self-education

The Guthy-Jackson Charitable Foundation does not perform clinical trials nor does it endorse any particular clinical trial design or drug

CONFIDENTIAL

Chugai Pharmaceuticals

Study SA309-JG: anti IL-6 receptor antibody

SA237 in subjects with NMO/NMOSD12-Dec-2014

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CONFIDENTIAL

Anti IL-6 Receptor Antibody SA237, Background

• Maintenance therapy has become standard practice for prevention of

relapses of patients with NMO/NMOSD.

• IL-6 is a growth factor for cells producing antibodies that contribute to

neuromyelitis optica symptoms.

• Small studies show activity of anti IL-6 receptor antibody tocilizumab

(Actemra) in preventing NMO/NMOSD relapses.

• SA237 is an anti IL-6 receptor antibody

− Designed to improve IL-6 receptor blocking activity of tocilizumab

− Tocilizumab has been used for many years by subjects with arthritis and

other autoimmune diseases.

− SA237 is an investigational agent currently being tested in clinical studies

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CONFIDENTIAL

SA237 is a Recycling Antibody

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SA237

IL-6 receptor

Inside the cell SA237 releases

IL-6 receptor which is

degraded

Recycled to the plasma

instead of being

degraded inside the cell

Investigator’s meeting of SA-309JG study

SA237 circulates in the blood

and binds to the IL-6 receptor

on cell surface

CONFIDENTIAL

Sakura Star Study: Eligible Participants

• Key Inclusion Criteria

1. NMO or NMOSD

2. Subjects of age 18 to 74 years

3. At least 1 documented relapse in last 12 months prior to screening

4. Disability score from 0 to 6.5 inclusive at screening

• Exclusion Criteria for General Safety

1. Pregnancy or lactation

2. Evidence of other demyelinating disease such as MS or progressive multifocal

leukoencephalopathy

3. Active infection within 4 weeks prior to baseline

4. Recent treatments with strong immunosuppressive agents

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CONFIDENTIAL

Sakura Star Study Design: Key Elements

• Screening Period: up to 28 days for evaluation of study candidates

• Randomization: 2:1 SA237 single agent to “pure” placebo

• Duration of Study Treatment Periods:

− Double blind: lasting until subject experiences a relapse

if no relapse – approx. 1 year

− Open label SA237: starting immediately after first relapse

for patients without relapse – approx. after 1 year

• Frequency of Dosing and Study Visits:

− Weeks 0, 2, 4, and every 4 weeks thereafter

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CONFIDENTIAL

Efficacy Outcome Measures

− Time to 1st relapse (TFR) in subjects receiving SA237

compared to subjects receiving placebo

Subjects will be closely monitored and symptoms of a

relapse will be rapidly assessed and treated

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CONFIDENTIAL

Efficacy Outcome Measures (cont.)

− Change in score for Pain

− Change in Fatigue Score

− Change in scores for Health Status

− Change in timed 25-foot Walk

− Change in severity of Disability

Evaluations to better understand NMO/NMOSD and how SA237 works,

performed at some selected sites:

− MRI scans of the brain, optic nerve, and spinal cord

− Immunological evaluations in blood and spinal fluid

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CONFIDENTIAL

Sakura Star Study Recruitment Objective

12

Num

ber

of P

atients

70 patients in 40 clinical

study sites in the US and

Canada

CONFIDENTIAL

Summary Notes for Patients

• SA237 is administered under the skin (sub-cutaneous

injection)

• 2:1 randomization of participants to SA237 or “pure” placebo

until relapse, thereafter SA237 for all participants

• Greenphire ClinCard system used for payment: similar to a

credit card that patients and caregivers can use for study

related expenses such as travel, meals, accommodations.

• For participants who complete the study, SA237 will be

made available free of charge up to FDA authorization to

market.

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CONFIDENTIAL

“SA237 and SAkura Star Study”: Name and Logo

• Name of the investigational agent is SA237, which was

discovered in Chugai Pharmaceutical laboratories in Japan,

• “Sakura” (桜), is the Japanese name of the Japanese

Cherry Blossom, which is Japan’s national flower,

• Yearly blooming of Sakura trees symbolises the return of

life and is taken as an image for the return of SA237

recycling to the blood

For more info about the SAkura Star clinical trial and SA237

go to http://clinicaltrials.gov or ask your doctor

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CONFIDENTIAL

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Questions

Learn More

This webinar will be hosted on the

foundation website at:

http://nmotion.guthyjacksonfoundation.org/series-one-nmo-

clinical-trial-update/

Conclusion

Content is solely that of the respective industry or its representative

Webinar slides and audio will be posted on the foundation website for later viewing / audio

All perspectives are offered only for stakeholder self-education

The Guthy-Jackson Charitable Foundation does not perform clinical trials nor does it endorse any particular clinical trial design or drug

We hope this webinar has been informative to all stakeholders