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Roche — Technical Presentation (October 2022)
16 slides from Roche's technical presentation, October 2022
Slide 1: GENERATION HD2, a Phase II dose-finding study of tominersen
GENERATION HD2, a Phase II dose-finding study of tominersen Peter McColgan, MD, PhD F. Hoffmann-La Roche Ltd, Basel, Switzerland To access this presentation go to: M-XX-00011398 Date of preparation: October 2022 https://bit.ly/3Tco0zb
Slide 2: Disclosures
Disclosures I am an employee of F. Hoffmann-La Roche Ltd Tominersen is an investigational drug that has not been approved by any health authority. The intent of this presentation is to provide a scientific update on the clinical trial programme of tominersen and the information included should not b
Slide 3: Outline of today's presentation
Outline of today’s presentation • CAP, CAG-age product; HD, Huntington’s disease; iDMC, independent Data Monitoring Committee. GENERATION HD2: Study design overview GENERATION HD2: Inclusion and exclusion criteria, endpoints and participating countries Roche’s commitment to HD HD, Huntington’s disea
Slide 4: Study design overview
GENERATION HD2: Study design overview 4
Slide 5: Testing a refined hypothesis
GENERATION HD2: Testing a refined hypothesis GENERATION HD1 Focused patient Lower dosing Safety, biomarkers exploratory post hoc population and efficacy trends findings GENERATION HD2 will Potential benefit in focus on adults with GENERATION HD2 will We will evaluate safety, younger adults with prod
Slide 6: Overview of GENERATION HD2
Overview of GENERATION HD2 A study to evaluate the safety, biomarkers and efficacy of two dose levels of tominersen in participants with prodromal (~20/arm) and early manifest (~100/arm) HD (HD-ISS Stage 2 and 3) versus placebo Tominersen 100 mg every 16 weeks (Q16W) N=120 Screening R 1:1:1 Tominers
Slide 7: Key aspects of the GENERATION HD2 design
Key aspects of the GENERATION HD2 design Common close design Blinded treatment period (16+ months*) Optional OLE study start† All Blinded treatment period (16 months) participants (except last Last participant) participant • Minimum 16-month treatment period (seven clinic visits with four interim ph
Slide 8: Inclusion and exclusion criteria, endpoints and participating countries
GENERATION HD2: Inclusion and exclusion criteria, endpoints and participating countries 8
Slide 9: Key inclusion/exclusion criteria
GENERATION HD2: Key inclusion/exclusion criteria Inclusion criteria Exclusion criteria Prodromal HD Early manifest HD • Current or previous use of an ASO, (n=~20 per arm) (n=~100 per arm) Broadly equivalent Broadly equivalent siRNA or HTT-lowering therapy to HD-ISS Stage 2 to HD-ISS Stage 3 • Anti-p
Slide 10: HD2 endpoints: comparing tominersen to placebo
GENERATION HD2 endpoints: comparing tominersen to placebo SAFETY BIOMARKERS EFFICACY* Primary Primary Primary Incidence and severity of AEs Percentage change from baseline in Change from baseline in cUHDRS Change in CSF leucocytes CSF mHTT at 9 months (non-US) and TFC (US) and proteins Secondary at
Slide 11: Expected country participation
GENERATION HD2: Expected country participation CANADA • We plan to run the GENERATION HD2 DENMARK study in 15 countries* POLAND UK • Specific study sites GERMANY USA FRANCE will be listed on AUSTRIA clinical trial registries SPAIN ITALY SWITZERLAND once a site is nearly PORTUGAL ready to enrol parti
Slide 12: Study design summary
GENERATION HD2: Study design summary GENERATION HD2 is a new study that will test two doses of intrathecally administered tominersen (60 mg and 100 mg) given Q16W Every participant will receive tominersen or placebo for at least 16 months, and will continue until the last participant has completed t
Slide 13: Roche's commitment to HD
Roche’s commitment to HD HD, Huntington’s disease. 13
Slide 14: Roche is pursuing multiple potential therapeutic approaches in parallel
Roche is pursuing multiple potential therapeutic approaches in parallel Preclinical Phase I Phase II Phase III Tominersen (IT, ASO) – Ionis Non-allele- specific HTT lowering Splice modifier (oral) SNP- Allele-specific targeting HTT lowering (IT, ASO) – Ionis Additional early HD research efforts acro
Slide 15: A commitment to advance scientific understanding and drug development in HD
A commitment to advance scientific understanding and drug development in HD through continued collaborations Scientific research and collaborations Clinical trial networks HD • Sharing data and learnings from the tominersen program in a timely and specialists Scientific & collaborative manner philan
Slide 16: Thank you
THANK YOU A big THANK YOU to the HD community for their ongoing contributions, especially study participants, families, investigators and site staff, and the tominersen steering committee HD, Huntington’s disease.
Roche Holding — Technical Presentation (October 2022) on 6ix