Ionis Pharmaceuticals, Inc. announced that its license agreement with Roche to develop and commercialize IONIS-HTTRx for the treatment of people with Huntington's disease (HD) has received clearance under the Hart-Scott-Rodino Antitrust Improvements Act. Based on Roche's decision to license the drug and this approval, Ionis has earned a $45 million license fee from Roche.
Roche is now responsible for all IONIS-HTTRx development, regulatory and commercialization activities and costs. The two companies will work together to transition the ongoing open-label extension study to Roche, which will be responsible for managing this study and all future studies.
IONIS-HTTRx is an antisense drug in development for the treatment of HD. IONIS-HTTRx is designed to reduce the production of all forms of the huntingtin (HTT) protein, which in its mutated variant (mHTT) is responsible for HD. As such, IONIS-HTTRx offers a unique approach to treat all patients with HD, irrespective of their individual HTT mutation. IONIS-HTTRx has been granted orphan drug designation by the US Food and Drug Administration (FDA) and by the European Medicines Agency (EMA) for the treatment of patients with HD.
HD is a rare, genetic, progressive, neurodegenerative disease resulting in deterioration in mental abilities and physical control. In the US, there are approximately 30,000 individuals with symptomatic HD and more than 200,000 people at risk of having inherited HD. HD is referred to as a triplet repeat disorder and is one of a large family of genetic diseases in which certain gene sequences are mistakenly repeated. In HD, the trinucleotide sequence in the gene that encodes for the HTT protein is repeated more than 36 times. The resulting mHTT protein is toxic and gradually damages neurons in the brain. Symptoms of HD usually appear between the ages of 30 to 50 years and continually worsen over a 10- to 25-year period. Ultimately, the weakened individual succumbs to pneumonia, heart failure or other complications. Presently, there is no effective disease-modifying treatment for HD, and current products focus only on managing disease symptoms.
Roche and Ionis are collaborating to develop antisense drugs to treat HD. The alliance combines Ionis' antisense expertise with Roche's knowledge in clinical development of anti-neurodegenerative therapeutics. In total, Ionis has generated $100 million in up-front, milestone and license payments and is eligible to receive an additional $275 million in milestone payments as the drug progresses in development. In addition, Ionis is eligible to receive tiered double-digit royalties up to the mid-teens on sales of IONIS-HTTRx if it is commercialized.
Ionis is the leading company in RNA-targeted drug discovery and development focused on developing drugs for patients who have the highest unmet medical needs, such as those patients with severe and rare diseases.