Ionis Licenses Investigational Alzheimer's Therapy

Ionis Pharmaceuticals announced Biogen, a collaboration partner for neurological diseases, has licensed IONIS-MAPTRx, an antisense therapy designed to selectively reduce production of microtubule-associated protein tau (MAPT), or tau, in the central nervous system. MAPT is believed to contribute to or cause several neurodegenerative diseases, including Alzheimer's disease (AD) and some forms of frontotemporal degeneration (FTD).

"The licensing of IONIS-MAPTRx, currently in a Phase 1 clinical study in mild AD patients, is an important milestone for the program. It brings us another step closer to potentially delivering a therapy to patients who have few or no effective treatment options," said Brett P. Monia, Ph.D., Ionis' chief operating officer.

Tau pathology is a hallmark of AD and other tauopathies and is widely hypothesized to contribute to clinical decline in these neurodegenerative diseases. In preclinical studies, MAPT-targeted antisense treatment demonstrated prevention and reversal of pathology.

Ionis earned a $45 million license fee from Biogen and is eligible to earn up to $155 million in additional milestone payments. Ionis is also eligible to receive royalties on sales of the medicine in the low- to mid-teens. Under terms of the collaboration, Ionis will be responsible for the Phase 1 clinical study of IONIS-MAPTRx in patients with mild AD that was initiated in 2017 and a one-year long-term extension study that began this year. Biogen will have responsibility for all subsequent studies and any further development, including regulatory filings, and commercialization.

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