Ionis Licenses IONIS-MAPTRx Antisense Therapy for Alzheimer’s to Biogen

Ionis Licenses IONIS-MAPTRx Antisense Therapy for Alzheimer’s to Biogen
Ionis Pharmaceuticals has licensed IONIS-MAPTRx (BIIB080), its investigational antisense therapy for the treatment of Alzheimer’s disease, to Biogen. IONIS-MAPTRx is an antisense therapy — an approach to fighting diseases using short DNA-like molecules called antisense oligonucleotides — that has been designed to reduce the production of the microtubule-associated protein tau (MAPT), or tau. Tau is thought to cause, or at least contribute to the development of several neurodegenerative diseases, including Alzheimer’s and some forms of frontotemporal degeneration (FTD). The therapy works by selectively targeting and preventing the messenger RNA (mRNA) containing information to produce tau from being translated into a functional protein. "The licensing of IONIS-MAPTRx, currently in a Phase 1 clinical study in mild AD [Alzheimer’s disease] 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," Brett P. Monia, PhD, Ionis' chief operating officer, said in a press release. Ionis will receive an upfront $45 million license fee from Biogen and will be eligible to receive up to $155 million in royalty payments for each developmental milestone achieved. In addition, the company will be eligible to receive further royalty payments based on product sales.  Under the terms of the licensing agreement, Ionis will be responsible for carrying out the placebo-controlled Phase 1/2 trial (NCT03186989) launched in 2017 to investigate the safety, tolerability,  and pharmacokinetic and pharmacodynamic properties of IONIS-MAPTRx in patients with mild Alzheimer’s. Ionis also will carry out the study’s one-year, long-term, open-label e
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