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© 2018 F. Hoffmann-La Roche Ltd. All rights reserved. GPT/051618/0074
Roche/Genentech Anti-Myostatin Adnectin
RG6206 Development Program in Duchenne
This compound and its uses are investigational and have not been approved by the U.S. FDA. This information is
presented only for purposes of providing a general overview of clinical trials and should not be construed as a
recommendation for use of any product for unapproved uses.
Michelle Krishnan MD, PhD.
Translational Medicine Leader, Roche
GPT/051618/0074
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© 2018 F. Hoffmann-La Roche Ltd. All rights reserved. GPT/051618/0074
Disclaimer
This session contains general information about our Duchenne program as is not intended as
specific medical advice.
RG6206 is an investigational (not approved) medicine that is being studied for the treatment of
people with Duchenne. RG6206 has not been approved by the Food and Drug Administration
(FDA). The effectiveness and safety of RG6206 are currently being studied.
You should talk with your healthcare provider for information and advice about your condition,
including any current or potential treatments.
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© 2018 F. Hoffmann-La Roche Ltd. All rights reserved. GPT/051618/0074
Agenda
• Introduction to Roche/Genentech
• What is Myostatin?
• Overview of our anti-myostatin development program:
• What is RG6206?
• Overview of RG6206 Clinical Studies
• Status to date and next steps
• Q&A
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© 2018 F. Hoffmann-La Roche Ltd. All rights reserved. GPT/051618/0074
What is Myostatin?
• Myostatin is a naturally occurring protein that is produced
primarily in skeletal muscle cells
• Its function is usually to stop muscles growing too large
• Limiting myostatin has been shown in some studies to
increase muscle size
• We hypothesize that blocking myostatin could increase
muscle growth and muscle strength in children with
Duchenne
Myostatin
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© 2018 F. Hoffmann-La Roche Ltd. All rights reserved. GPT/051618/0074
Introduction to RG6206 program
• In early 2017, Bristol-Myers Squibb (BMS) entered into an agreement to fully license BMS-
986089 (now RG6206) to Roche
• RG6206 is an investigational anti-myostatin adnectin
• Phase 2/3 study is underway in boys with ambulatory Duchenne
• Recruitment has now restarted following a temporary pause due to an issue with study drug
availability
This compound and its uses are investigational and have not been approved by the U.S. FDA. This
information is presented only for purposes of providing a general overview of clinical trials and should
not be construed as a recommendation for use of any product for unapproved uses.
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© 2018 F. Hoffmann-La Roche Ltd. All rights reserved. GPT/051618/0074
• RG6206 is an investigational molecule being developed
for people with Duchenne
• RG6206 is designed to bind to a protein called
myostatin and to potentially limit its function
• Per study protocol, RG6206 is administered at home
once weekly by subcutaneous injection (injection under
the skin) into the abdomen (tummy) after initial training
at the site
RG6206
What is RG6206?
This compound and its uses are investigational and have not been approved by the U.S. FDA. This
information is presented only for purposes of providing a general overview of clinical trials and should
not be construed as a recommendation for use of any product for unapproved uses.
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© 2018 F. Hoffmann-La Roche Ltd. All rights reserved. GPT/051618/0074
Clinical Studies of RG6206
Clinicaltrials.gov/show/ 1.NCT02145234 2. NCT03100630
3. NCT02515669 4. NCT03039686 (May 2018)
Phase 2/3Phase 1 Phase 1b/2
Safety, tolerability and efficacy of
RG6206 in ambulatory boys with
Duchenne4
Safety and tolerability of RG6206 in
ambulatory boys with Duchenne3
Recruiting
Recruitment completed. 103 adult volunteers
Recruitment completed. 43 ambulatory
boys with DuchenneStudy
WN40226
Study
WN40227
Study
WN40225
‘Spitfire’
This compound and its uses are investigational and have not been approved by the U.S. FDA. This
information is presented only for purposes of providing a general overview of clinical trials and should
not be construed as a recommendation for use of any product for unapproved uses.
Studied the safety of RG6206 in healthy
adults1,2
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© 2018 F. Hoffmann-La Roche Ltd. All rights reserved. GPT/051618/0074
Roche Anti-Myostatin Adnectin
RG6206 Development Program in Duchenne
This compound and its uses are investigational and have not been approved by the U.S. FDA. This information is
presented only for purposes of providing a general overview of clinical trials and should not be construed as a
recommendation for use of any product for unapproved uses.
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© 2018 F. Hoffmann-La Roche Ltd. All rights reserved. GPT/051618/0074
Clinicaltrials.gov/show/NCT02515669 (Accessed May 2018); Wagner KR et al. Dev Med Child Neurol 59;S34 - Presented at BPNA 2018.
Current status:
• 43 ambulatory boys with Duchenne (5-10 yrs old) enrolled in the
study
• Over 24 weeks, each received a weekly subcutaneous dose of
either RG6206 or placebo
• Multiple Ascending Subcutaneous Dose Study
• Participants in this study are now all receiving RG6206 in an
open-label phase
• Data from the WN40226 study were first presented at the British
Pediatric Neurology Association conference earlier in 2018Study WN40226
Phase 1b/2
Phase 1b/2 Study of RG6206 in Boys With Duchenne Assessing the Safety and Tolerability of RG6206
This compound and its uses are investigational and have not been approved by the U.S. FDA. This
information is presented only for purposes of providing a general overview of clinical trials and should
not be construed as a recommendation for use of any product for unapproved uses.
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© 2018 F. Hoffmann-La Roche Ltd. All rights reserved. GPT/051618/0074
Phase 2/3 Study Investigating RG6206Assessing the Safety, Tolerability and Efficacy of RG6206
Phase 2/3
Study WN40227
Approximately 159 boys with
Duchenne who are ambulatory
Double-blind period
48 weeks
Open label period
48 weeks
• Patients will be randomly allocated to a
treatment
• Neither the patient, not the investigator
will know if they are receiving RG6206 or
placebo
• 2:1 randomization means that twice as
many people receive RG6206 versus
placebo
• Two doses of RG6206
RG6206 is given to all participants
This compound and its uses are investigational and have not been approved by the U.S. FDA. This
information is presented only for purposes of providing a general overview of clinical trials and should
not be construed as a recommendation for use of any product for unapproved uses.Clinicaltrials.gov/show/NCT03039686 (Accessed May 2018).
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© 2018 F. Hoffmann-La Roche Ltd. All rights reserved. GPT/051618/0074
A Randomized, Double
Blind, Placebo-Controlled,
Study to Assess the
Efficacy, Safety, and
Tolerability of RG6206 in
Ambulatory Boys with
Duchenne Muscular
Dystrophy
Primary ObjectiveCompare the change from baseline in 4 Stair Climb (4SC) velocity at Week 48 in
RG6206 vs placebo treated participants
Secondary ObjectivesCompare the change from baseline at Week 48 in RG6206 vs placebo treated
participants in:
North Star Ambulatory Assessment (NSAA) total score
Stand from supine velocity
10 m walk/run velocity
Pediatric Outcomes Data Collection Instrument (PODCI) transfers & basic
mobility subscale score
Proximal lower extremity flexor strength measured using manual myometry
6 Minute Walk Distance
Compare number of new or worsening lab abnormalities, Serious Adverse EventAEs,
& AE leading to discontinuation
Phase 3 Study Investigating RG6206 WN40227: Study Rationale & Objectives
This compound and its uses are investigational and have not been approved by the U.S. FDA. This
information is presented only for purposes of providing a general overview of clinical trials and should
not be construed as a recommendation for use of any product for unapproved uses.Clinicaltrials.gov/show/NCT03039686 (Accessed May 2018).
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© 2018 F. Hoffmann-La Roche Ltd. All rights reserved. GPT/051618/0074
Key Inclusion Key Exclusion
• Any behaviour or mental issue that will
affect the ability to complete the required
study procedures
• Previously or currently taking medications
like androgens or human growth hormone
• Use of a ventilator during the day
• Unable to have blood samples collected or
receive an injection under the skin
• All DMD mutations are eligible
• Diagnosis of Duchenne, confirmed by
medical history and by genotyping
• Ambulatory without assistance
• Boys 6–11 years of age, inclusive
• Weight ≥ 33 lbs
• Corticosteroid use
• 4 stair climb ≤ 8 seconds
Phase 2/3 Study Investigating RG6206WN40227: Key Inclusion & Exclusion Criteria
This compound and its uses are investigational and have not been approved by the U.S. FDA. This
information is presented only for purposes of providing a general overview of clinical trials and should
not be construed as a recommendation for use of any product for unapproved uses.Clinicaltrials.gov/show/NCT03039686 (Accessed May 2018).
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© 2018 F. Hoffmann-La Roche Ltd. All rights reserved. GPT/051618/0074Clinicaltrials.gov/show/NCT03039686 (Accessed May 2018).
US
CanadaBelgium
France
Spain
UK
Sweden
Argentina
Germany
Italy
Japan
Australia
Netherlands
Currently recruiting (as of May 2018)
Phase 3 Study Investigating RG6206WN40227: Countries Planned
This compound and its uses are investigational and have not been approved by the U.S. FDA. This
information is presented only for purposes of providing a general overview of clinical trials and should
not be construed as a recommendation for use of any product for unapproved uses.
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© 2018 F. Hoffmann-La Roche Ltd. All rights reserved. GPT/051618/0074
,
,
–
NeuromuscularResea rch Center,
Arizona
David Geffen Schoolof Medicine UCLA,
CaliforniaUniversity of Florida,
Florida
University of Iowa,Iowa
Stanford University,California
University of KansasMedical Center,
Kansas
Saint LouisChildren's Hospital,
Missouri
University ofCalifornia DavisMedical Center
California
Cincinnati Children'sHospital Medical
Center, Ohio
Nemours Children'sHospital, Florida
Rush UniversityMedical Center
Illinois
Kennedy KriegerInstitute, Maryland
Children's Hospitalof Philadelphia,Pennsylvania
Seattle Children'sHospital
Washington
Center forIntegrative Rare
Disease Resea rch(CIRDR), Georgia
Las Vegas Clinic,Nevada
NationwideChildren's Hospital,
Ohio
Yale UniversitySchool of Medicine,
Connecticut
Currently recruiting (as of 8th May)
Phase 3 Study Investigating RG6206WN40227: US sites
Clinicaltrials.gov/show/NCT03039686 (Accessed May 2018).
This compound and its uses are investigational and have not been approved by the U.S. FDA. This
information is presented only for purposes of providing a general overview of clinical trials and should
not be construed as a recommendation for use of any product for unapproved uses.
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© 2018 F. Hoffmann-La Roche Ltd. All rights reserved. GPT/051618/0074
For more information about the ongoing RG6206 trial
Visit Clinicaltrials.gov
We would like to thank the patients and their families who take part in
clinical trials
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© 2018 F. Hoffmann-La Roche Ltd. All rights reserved. GPT/051618/0074
Q & A
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© 2018 F. Hoffmann-La Roche Ltd. All rights reserved. GPT/051618/0074