The Swiss pharmaceutical firm will obtain exclusive global rights to develop, manufacture, and market the medicines by making a $60m upfront payment to Ionis, along with milestone payments and tiered royalties in future


Ionis Pharmaceuticals headquarters building entrance. (Credit: Ionis Pharmaceuticals)

US-based Ionis Pharmaceuticals and Swiss pharmaceutical firm Roche have teamed up for two undisclosed early-stage programmes for RNA-targeting investigational medicines for Alzheimer’s disease (AD) and Huntington’s disease (HD).

Under the terms of the agreement, the US-based biotechnology company will be responsible for advancing the two programmes through pre-clinical studies.

The Swiss pharmaceutical firm will obtain exclusive global rights for the clinical development, manufacturing, and marketing of the medicines, subject to regulatory approval.

It will make a $60m upfront payment to Ionis, along with development, regulatory and commercial milestone payments, and tiered royalties in future.

Ionis will leverage its expertise in discovering medicines for central nervous system diseases, while Roche its experience in developing and marketing therapies for neurological disorders.

Ionis chief executive officer Brett P Monia said: “We are excited to expand our partnership with Roche, a global leader in developing and delivering innovative treatments to patients.

“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.

“Collaborating on these two programmes enables Ionis to advance our wholly owned programmes, including those in neurology, aligned with our strategic priorities.”

Ionis and Roche have been working together for the past 10 years, starting with their collaboration for antisense oligonucleotide medicine tominersen in 2013.

Discovered by Ionis, tominersen is an investigational medicine designed to reduce the production of all forms of the huntingtin protein, to treat HD.

Roche is currently developing the drug in a Phase 2 proof of concept study, dubbed GENERATION HD2, in people with prodromal or early manifest HD.

In addition to tominersen, the two companies are working together on a pre-clinical stage mutant HTT selective antisense oligonucleotide for HD.

In 2018, Ionis and Roche entered another collaboration to develop IONIS-FB-LRx targeting Factor B for the treatment of IgA nephropathy (IgAN) and geographic atrophy (GA).

The 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.