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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

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

© 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.

© 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

© 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

© 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.

© 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.

© 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

© 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.

© 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.

© 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).

© 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).

© 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).

© 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.

© 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.

© 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

© 2018 F. Hoffmann-La Roche Ltd. All rights reserved. GPT/051618/0074

Q & A

© 2018 F. Hoffmann-La Roche Ltd. All rights reserved. GPT/051618/0074