Roche to Evaluate Tominersen, an Antisense RNA Therapy, in a New Phase 2 Trial for Huntington’s Disease Disease-Modifying Treatment

News on January 19, 2022 /BioValleyBIOON/ -- Ionis Pharmaceuticals recently announced that its partner, Roche, is designing a new Phase 2Clinical Trial`, Evaluation`Antisense RNA Drug Tominersen (Formerly: IONIS-HTTRx, RG6042) for the Treatment of Huntington's Disease (HD)Roche will be in the future of scienceMeetingShare more details about this new Phase 2 trial design.

Exploratory post-hoc analyses after stopping dosing in the Phase 3 GENERATION HD1 study suggest that tominersen may benefit younger adult patients with a lower disease burden. These results need to be confirmed in a randomized, placebo-controlled study.

Tominersen is an antisense RNA drug derived from Ionis Pharmaceuticals, used for the treatment of Huntington's disease (HD). In December 2017, Roche obtained the license for tominersen from Ionis.As an antisense RNA drug, tominersen aims to reduce the production of all types of huntingtin (HTT), including the production of toxic mutant huntingtin (mHTT). mHTT is a protein produced by the Huntington's disease-related gene and is the root cause of HD.

HD is aGeneticsNeurodegenerative diseases currently have no approved disease-modifying therapies. Tominersen is a disease-modifying therapy that targets the underlying cause of Huntington's disease (HD) by reducing the production of toxic mutant huntingtin protein (mHTT). In the EU and the United States, tominersen was granted orphan drug designation (ODD) for the treatment of Huntington's disease in May 2015 and December 2015, respectively. Additionally, in August 2018, the EU granted tominersen priority medicine (PRIME) status for the treatment of Huntington's disease.

Huntington's Disease (HD), also known as Huntington's chorea, is an autosomal dominantGeneticsNeurodegenerative disorder characterized by psychiatric symptoms, cognitive changes, and involuntary movements, primarily chorea. This disease was discovered by American physician George Huntington in 1872, hence the name.

HD is primarily caused by a mutation in the huntingtin gene on chromosome four, which produces the mutant protein mHTT. mHTT gradually accumulates within cells, forming large molecular aggregates that build up in the brain and impair neuronal function. Typically, patients develop symptoms in middle age, characterized by chorea-like movements. As the disease progresses, patients gradually lose the ability to speak, move, think, and swallow. The condition usually progresses over 10 to 20 years and ultimately leads to death.

Due to the lack of a definitive treatment effective for the disease, patients usually receive symptomatic treatment for involuntary movements and psychiatric symptoms. Currently, there are no effective drugs for treating HD, and the products currently on the market only focus on managing the symptoms of the disease.

Tominersen offers a unique approach to treating all HD patients, regardless of their respective HTT mutations.. The I/II phase clinical study published in early 2019 showed,Tominersen significantly reduced mHTT levels: After 3 months of treatment with the two highest doses of tominersen, the levels of specific HTT in the cerebrospinal fluid (CSF) of HD patients decreased by an average of 40% (up to 60% at the highest). In the most recent measurement, the mHTT levels in the CSF of the majority of patients (approximately 70%) were still continuing to decline, and RG6042 was well tolerated. (Bioon.com)

Source of the original text:Ionis' partner to evaluate tominersen for Huntington's disease in new Phase 2 trial