Kariko and Weissman
Roche’s most advanced attempts to treat both diseases have struggled in the clinic. (iStock / Getty Images Plus)
When Roche’s gantenerumab failed a pair of phase 3 studies last year, the pharma giant said it would look for “external collaborations and partnerships” as an avenue to treat Alzheimer’s disease. With today’s agreement with longtime partner Ionis, it seems the drugmaker is sticking to its word.

The Swiss pharma is paying $60 million upfront for the exclusive worldwide rights to two undisclosed, preclinical RNA-targeting programs in the notoriously tricky indications of Alzheimer’s and Huntington’s diseases.

Ionis will take the programs through preclinical studies before Roche takes over sole responsibility for human trials and potentially commercialization.

As well as the upfront cash, the Big Pharma agreed to pay development, regulatory and commercial milestones. But the companies are keeping the amount of biobucks at stake under wraps.

“Our lasting partnership with Ionis, a leader in RNA-targeted therapeutics, is a great example of two collaborators mutually benefiting from their relationship by complementing and learning from each other,” James Sabry, Ph.D., global head of Roche Pharma Partnering, said in this morning’s release.

“By expanding our alliance, we bring together the companies’ combined knowledge of the science in Alzheimer’s disease and Huntington’s disease with Roche’s proven capabilities in the development and commercialization of innovative treatments in neuroscience,” Sabry added.

Roche has already been burned attempting to treat both diseases. Its major Alzheimer’s contender, gantenerumab, failed to improve cognitive and functional decline in the two much-anticipated trials from the GRADUATE program in November of last year, leading the Big Pharma to cut most of the program.

However, Roche does have a number of other potential Alzheimer’s therapies in its pipeline, including the AC Immune-partnered anti-amyloid-beta antibody called crenezumab, which has also come up short in the clinic.

Keeping it company in phase 2 are the UCB-partnered anti-tau antibody bepranemab, the AC Immune-partnered anti-tau antibody semorinemab and a different formulation of gantenerumab called trontinemab.

Unlike those antibody therapies, today’s Ionis alliance means Roche could soon add an RNA approach to its Alzheimer’s portfolio.

It’s not the company’s first push into this area, however. In 2021, the Big Pharma signed a pact with Shape Therapeutics to use its RNA-editing platform to explore next-generation gene therapies for Alzheimer’s, Parkinson’s and rare diseases.

When it comes to Huntington’s, Roche and Ionis have previous experience due to their collaboration on tominersen for the past decade. The partners have pressed ahead with a phase 2 trial of the antisense drug in a subgroup of patients despite a late-stage study providing to be one of the highest profile flops of 2021.

“Collaborating on these two programs enables Ionis to advance our wholly owned programs, including those in neurology, aligned with our strategic priorities,” Ionis CEO CEO Brett Monia, Ph.D., added in today’s release.

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