SOUTH SAN FRANCISCO, California – (BUSINESS WIRE) – Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced the decision to discontinue dosing in the Phase III GENERATION HD1 study of tominersen in overt Huntington’s disease (HD). The decision was based on the results of a pre-planned review of the data from the Phase III study conducted by a non-blind Independent Data Monitoring Committee (iDMC). IDMC made its recommendation based on the potential benefit / risk profile of experimental therapy for study participants. No new or emerging safety signs were identified for tominersen in the review of the data in this study. Genentech intends to continue following the participants for safety and clinical results, without the dosage of the experimental drug or placebo. Once the complete data from the Phase III study is available and analyzed, Genentech will share the lessons and future plans with the HD community.
Dosing will be interrupted in tominersen’s open extension study (GEN-EXTEND) while the data is carefully analyzed to inform the next steps of this study.
“This is very unfortunate news to deliver in the Phase III study of tominersen and we know that it will be especially difficult for people with Huntington’s disease to hear. The HD community currently has no treatments to stop or slow the progression of this rare neurodegenerative disease that affects families over the generations, ”said Levi Garraway, MD, Ph.D., medical director and head of Global Product Development. “GENERATION HD1 is the largest clinical trial in Huntington’s disease to date and we know that the data generated will significantly increase our understanding of huntingtin reduction as a potential treatment approach. We would like to thank all individuals and families who participated in the study for their contribution, as well as the wider HD community for their commitment and collaboration. ”
The Phase I PK / PD (GEN-PEAK) study by tominersen and the observational study of natural history Genentech and Roche HD will continue.
About tominersen and clinical trials
Tominersen, formerly IONIS-HTTRx or RG6042, is an experimental antisense therapy designed to reduce the production of all forms of the huntingtin protein (HTT), including its mutated variant, mHTT. In December 2017, Roche licensed the experimental molecule from Ionis Pharmaceuticals.
Tominersen is being investigated in HD in the following clinical studies:
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GENERATION HD1: a randomized, multicenter, double-blind, placebo-controlled Phase III clinical trial that evaluated the efficacy and safety of tominersen treatment in people with overt HD for more than 25 months. Study participants were randomized to 120 mg every 2 months or 120 mg every 4 months with intrathecal injections of tominersen or placebo. The study recruited 791 participants from 18 countries around the world.
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GEN-EXTEND: an open extension study for participants from any study by Genentech and Roche HD. Participants receive 120 mg of tominersen every 2 months or every 4 months in the study.
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GEN-PEAK: a phase I study to better understand the pharmacokinetics of tominersen and how tominersen affects levels of mHTT and other markers in spinal fluid and blood, which studies a range of doses from 30 mg to 120 mg tominersen in two administrations.
About Huntington’s disease
Huntington’s disease is a rare and progressive genetic disease that causes nerve cells in the brain to rupture, causing problems with a person’s ability to think, move and function, leading to increased disability and loss of independence. It has a devastating impact on people living with the disease and the inherited nature of HD means that it profoundly affects entire families for generations. Survival varies from approximately 10-20 years after the motor onset of the disease. There is no known cure for HD or approved therapies to treat the underlying cause.
About Genentech in Neuroscience
Neuroscience is the main focus of research and development at Genentech and Roche. Our goal is to seek innovative science to develop new treatments that help to improve the lives of people with chronic and potentially devastating diseases.
Genentech and Roche are investigating more than a dozen drugs for neurological diseases, including multiple sclerosis, stroke, Alzheimer’s disease, Huntington’s disease, Parkinson’s disease, Duchenne muscular dystrophy and autism spectrum disorder. Together with our partners, we are committed to expanding the limits of scientific knowledge to solve some of the most difficult challenges in neuroscience today.
About Genentech
Founded more than 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and markets drugs to treat patients with serious and potentially fatal medical conditions. The company, a member of the Roche Group, is headquartered in South San Francisco, California. For additional information about the company, visit http://www.gene.com.