Roche and Ionis Forge New Collaboration to Advance RNA-Targeted Therapies for Alzheimer’s and Huntington’s Diseases

On September 27, Ionis Pharmaceuticals announced that it had reached a collaboration agreement with Roche on two undisclosed RNA-targeting early-stage investigational drugs for the treatment of Alzheimer's disease (AD) and Huntington's disease (HD). Roche will obtain global exclusive rights to the two programs and will be responsible for the clinical development, manufacturing, and commercialization of the two drugs.

Ionis to Receive $60 Million Upfront Payment from Roche, Eligible for Development, Regulatory and Commercial Milestone Payments and Tiered Royalties.

Ionis and Roche's first collaboration began in 2013 with Ionis' discovery of the research drug tominersen for the treatment of HD. Over the past decade, the two companies have been strong partners. Tominersen is designed to reduce the production of all forms of huntingtin protein, and Roche is currently conducting a Phase II proof-of-concept study (GENERATION HD2) on this drug.

In addition to tominersen, the two companies are collaborating to develop an antisense oligonucleotide therapy for the treatment of HD with HTT mutations, which is currently in the preclinical stage. In 2018, the two companies entered into another collaboration to develop IONIS-FB-LRx, an antisense therapy targeting Factor B, for the treatment of IgA nephropathy (IgAN) and geographic atrophy (GA). IONIS-FB-LRx is currently in Phase II studies for the treatment of GA (led by Ionis) and in Phase III studies for the treatment of IgAN (led by Roche).

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