RARE Daily

Ionis Collaborates with Roche on RNA-Targeted Programs for Huntington’s and Alzheimer’s

September 27, 2023

Rare Daily Staff

Ionis Pharmaceuticals has entered an agreement with Roche for two undisclosed early-stage programs for RNA-targeting experimental medicines for the treatment of the neurodegenerative conditions Huntington’s disease and Alzheimer’s disease for $60 million up front.

Under the agreement, Roche gains exclusive worldwide rights and will be responsible for clinical development, manufacturing, and commercialization of the medicines if they receive regulatory approval. The companies will leverage Ionis’ expertise discovering medicines that target the root cause of central nervous system diseases and Roche’s global experience developing and commercializing therapies for nervous system disorders. Ionis is eligible to receive development, regulatory and commercial milestone payments, and tiered royalties.

“With this new collaboration, we are joining forces to accelerate the discovery and development of novel therapies for people living with Alzheimer’s disease and Huntington’s disease globally.,” said Brett Monia, CEO of Ionis. ” Collaborating on these two programs enables Ionis to advance our wholly owned programs, including those in neurology, aligned with our strategic priorities.”

Ionis and Roche have been strong partners for the past 10 years, starting with their collaboration in 2013 for tominersen, an Ionis-discovered investigational medicine for HD. Tominersen, designed to reduce the production of all forms of the huntingtin protein, is currently being evaluated by Roche in a phase 2 proof of concept study assessing safety, biomarker, and efficacy trends in people with prodromal or early manifest HD. In addition to tominersen, both companies are working together on a pre-clinical stage mutant HTT selective antisense oligonucleotide for HD. The companies entered another collaboration in 2018 to develop IONIS-FB-LRx targeting Factor B for the treatment of IgA nephropathy and geographic atrophy. IONIS-FB-LRx is currently in a phase 2 study for GA, led by Ionis, and a phase 3 study for IgAN led by Roche.

Huntington’s disease (HD) is a rare, genetic, neurodegenerative disease characterized by a triad of cognitive, behavioral, and motor symptoms leading to functional decline, progressive loss of independence, and impacting families across generations. It is caused by the expansion of the cytosine-adenine-guanine (CAG) trinucleotide sequence in the HTT gene. The resulting mutant HTT protein is toxic and gradually destroys neurons. Symptoms usually appear between the ages of 30 and 50 and worsen over a 10 to 25-year period. There is no effective treatment or cure for the disease and currently available medicines only mask the patient’s symptoms but do not slow down the underlying loss of neurons.

Alzheimer’s disease (AD) is the most common cause of dementia and, according to the World Health Organization, more than 55 million people have dementia worldwide. During the course of the disease, protein plaques appear in the brain, leading to the death of brain cells. No single factor has been identified as the cause of Alzheimer’s disease.

Photo: Brett Monia, CEO of Ionis

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