Ionis Licenses Huntington’s Disease Drug to Roche After Promising Results

Rare Daily Staff

Ionis Pharmaceuticals said that Roche has exercised its option to license IONIS-HTT_Rx following the completion of a phase 1/2 study of the experimental therapy to treat Huntington’s disease, a rare, genetic, progressive, neurodegenerative disease.

As part of the two companies ongoing collaboration around Huntington’s disease, Roche will pay a $45 million license fee to Ionis for IONIS-HTT_Rx, the first drug to target the underlying cause of disease. Roche will now be responsible for all development and commercial activities for IONIS-HTT_Rx. To date, Ionis has earned $55 million in upfront and milestone payments from its relationship with Roche and is eligible to earn additional milestone payments as the drug progresses in development, as well as royalties on sales of IONIS-HTT_Rx if it is commercialized. 

Roche is now responsible for all IONIS-HTT_Rx 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.

Symptoms of Huntington’s disease 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.

In the United States, there are approximately 30,000 individuals with symptomatic Huntington’s and more than 200,000 people at risk of having inherited Huntington’s. The conditions are part 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.

IONIS-HTT_Rx is an antisense drug in development for the treatment of Huntington’s. The drug is designed to reduce the production of all forms of the huntingtin (HTT) protein, which in its mutated variant (mHTT) is responsible for Huntington’s.

In the Phase 1/2a study, dose-dependent reductions of mHTT were observed in participants treated with IONIS-HTT_Rx. In addition, the safety and tolerability profile of IONIS-HTT_Rx observed in the Phase 1/2a study supports continued development, the companies said.  Ionis and Roche plan to present results from this study at medical conferences in the first half of 2018 and plan to submit the study results for publication in a peer-reviewed medical journal. Ionis and Roche have also recently initiated an open-label extension study for patients who completed the Phase 1/2a study.

“The results of this trial are of ground-breaking importance for Huntington’s disease patients and families,” said Dr. Sarah Tabrizi, professor of clinical neurology, director of the University College London’s Huntington Centre and the global lead investigator on the Phase 1/2a study. “For the first time, a drug has lowered the level of the toxic disease-causing protein in the nervous system, and the drug was safe and well tolerated. The key now is to move quickly to a larger trial to test whether IONIS-HTT_Rx slows disease progression.”

The U.S. Food and Drug Administration and the European Medicines Agency have granted IONIS-HTT_Rx orphan drug designation.

December 11, 2017