Ionis Pharmaceuticals’ IONIS-HTTRx is designed to address the cause of Huntington’s disease by reducing the production of the toxic mutant huntingtin protein responsible for this genetic neurodegenerative disorder. IONIS-HTTRx received orphan drug designation in Europe from the European Medicines Agency (EMA) in May 2015 and from the FDA in January 2016 for the treatment of Huntington’s disease.

A small Phase I trial involving just 46 patients was recently completed, revealing groundbreaking positive results for this incurable disease. This is the first time a gene-silencing drug has entered human brains, lowered the concentration of the harmful protein, and in proportion with the strength of the dose.

Meeting an unmet need

IONIS-HTTRx is being developed by California-based Ionis Pharmaceuticals, in collaboration with Roche. The drug is administered by injection into the spinal fluid to improve its delivery to the brain, where it reduces production of the mutant huntingtin protein. As such, this is considered to be a ‘gene-silencing’ drug that is able to modify the progression of this rare disease. While the trial results are promising, the fact that this gene silencing approach could potentially be applied to other neurodegenerative disorders holds even more promise, as this approach can be tailored to modify the production of any target protein.

Despite the availability of a wide array of symptomatic treatments, there is no way to cure or halt the progression of Huntington’s disease. GlobalData anticipates that, should IONIS-HTTRx reach the market, it would fulfil a significant unmet need in the treatment of Huntington’s disease, and would likely justify premium pricing compared to the currently-marketed therapeutics.

According to GlobalData’s OpportunityAnalyzer: Huntington’s Disease – Opportunity Analysis and Forecast to 2024, the market is expected to see solid sales growth through 2024, reaching $2.4bn in sales. Once early-stage drugs like IONIS-HTTRx launch, the value of the market is expected to see a significant boost in sales. Key opinion leaders interviewed by GlobalData have expressed enthusiasm about the development of IONIS-HTTRx and its potential therapeutic benefit in the treatment of Huntington’s disease. As IONIS-HTTRx has not shown any safety and tolerability issues so far, the industry will be eagerly monitoring its efficacy in its Phase II trial, which is due to start in 2018 in a larger number of patients.

Related Reports

GlobalData (2016). OpportunityAnalyzer: Huntington’s Disease – Opportunity Analysis and Forecast to 2024, May 2016, GDHC056POA