Dive Brief:
- Swiss pharma Roche AG will license a experimental Huntington's disease drug developed by Ionis Pharmaceuticals Inc., assuming responsibility for all future development and commercialization in exchange for a $45 million licensing fee paid to Ionis.
- Roche's decision follows positive results from a Phase 1/2a dose escalation study of IONIS-HTTRx in people with the neurodegenerative disease.
- Ionis reported a dose-dependent reduction in the mutant huntingtin protein, which is the root cause of the disease, and indicated the drug's tolerability profile supported continued development. An open-label extension of the Phase 1/2a study is currently ongoing.
Dive Insight:
The original 2013 deal between Roche and Ionis — then known as Isis Pharmaceuticals — set up a collaboration to develop treatments for Huntington's, which progressively weakens an individuals' physical and mental capabilities. Potentially worth up to $362 million, the deal combined the biotech's antisense oligonucleotide technology with Roche's delivery platform to enable systemic administration.
A number of companies are developing drugs to treat the symptoms of Huntington's disease. Teva Pharmaceutical Industries Ltd.'s Austedo (deutetrabenzine), for example, was approved this year to treat chorea associated with the disorder. Elsewhere, Vaccinex Inc. has a monoclonal antibody in Phase 2 that aims to block neuroinflammation, while Omeros Corp. is working on a small molecule that targets PDE10
What makes IONIS-HTTRx different is that it is the first therapy in clinical development that tackles the underlying cause of Huntington's disease, designed to reduce production of the mutant huntingtin protein.
"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," said Sarah Tabrizi, professor of clinical neurology and director of the University College London's Huntington Centre and the global lead investigator on the Phase 1/2a study, in a statement. "The key now is to move quickly to a larger trial to test whether IONIS-HTTRx slows disease progression."
In recent months, Ionis has licensed out pipeline assets to Dynacure SAS and Janssen Biotech Inc., monetizing slices of its extensive pipeline of more than two dozen potential treatments. Narrowing its focus will also help Ionis prioritize its efforts around inotersen, a treatment for hereditary TTR amyloidosis that the biotech hopes just might be able to compete with Alnylam Pharmaceuticals Inc.'s promising rival patisiran.