Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in RNA-targeted therapeutics, today announced that its partner Roche, also known as Genentech in the United States, has completed enrollment for GENERATION HD1, a global Phase 3 study evaluating the efficacy and safety of tominersen (previously IONIS-HTTRx or RG6042), an investigational antisense therapy for people living with Huntington’s disease (HD).
“Completion of the enrollment of this Phase 3 study is an important landmark for the clinical development of tominersen and for families affected by Huntington’s disease. While there is much work ahead of us, we are now closer to potentially providing a treatment for people living with this devastating disease. We are grateful to Huntington’s disease patients, their families and healthcare providers for their courage and resilience, particularly in the current challenging environment,” said Brett P. Monia, Ph.D., Ionis’ chief executive officer. “At Ionis, knowing that sick people depend on us fuels our passion for discovering and delivering novel antisense medicines like tominersen, the first and only therapy in pivotal trials targeting the underlying cause of HD.”
GENERATION HD1 is evaluating the efficacy and safety of tominersen treatment administered once every two months (eight weeks) or every four months (16 weeks) over a period of 25 months, compared to placebo. The study has completed enrollment with 791 patients across approximately 100 sites around the world.
HD is a devastating, and ultimately fatal, hereditary disease resulting in deterioration in mental abilities and physical control. Currently, there is no approved disease-modifying treatment for HD. There are approximately 3 to 10 per 100,000 people worldwide affected by HD. In the U.S. alone, there are approximately 40,000 people with symptomatic HD and more than 200,000 people at risk of having inherited the gene that causes HD.
Tominersen, previously IONIS-HTTRx or RG6042, is an investigational antisense therapy designed to reduce the production of all forms of the huntingtin protein (HTT), including its mutated variant, mHTT. Tominersen is the first therapy in pivotal trials targeting the underlying cause of HD. In December 2017, Roche licensed the investigational molecule from Ionis.
In the Phase 1/2 study, 46 people with early stage HD were treated with tominersen or placebo for 13 weeks. The data demonstrated significant, dose-dependent reductions in mHTT in the cerebrospinal fluid (CSF) of treated participants with a favorable safety and tolerability profile.
Tominersen is being investigated in a Phase 3 study (GENERATION HD1), an open label extension study in HD patients and a Phase I pharmacokinetics and pharmacodynamics study (GEN-PEAK). These studies, in addition to the non-interventional HD Natural History Study, are important elements of the clinical program to thoroughly evaluate the potential of tominersen to be the first disease-modifying medicine for the treatment of HD. The Phase 3 GENERATION HD1 study is expected to complete in 2022. The timing for this study’s completion remains unchanged.
Additional information about tominersen clinical trials may be found at https://clinicaltrials.gov/ct2/show/NCT03761849.