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 extension that started in 2019.
Pharmacokinetics and pharmacodynamics essentially refer to the interactions between the body and a specific compound. These interactions are analyzed to understand how a medication is absorbed, distributed, metabolized, and then eliminated from the body.
The Phase 1/2 trial is still recruiting in Canada and across several sites in Europe. Enrollment information can be found here.
Biogen will be responsible for all future clinical studies testing the therapy, as well as product regulatory filings and marketing.
With the new licensing of Ionis’ IONIS-MAPTRx, Biogen is now expanding its Alzheimer’s product pipeline, which includes aducanumab (BIIB037). Aducanumab is an investigational human monoclonal antibody that binds to toxic beta-amyloid aggregates, or clumps, and is thought to reduce the number of amyloid plaques in the brain.
Both companies have collaborated in the past to develop new therapies for a wide range of neurological disorders. Spinraza (nusinersen), the first commercial drug that resulted from this collaboration, became the first disease-modifying therapy to be approved to treat children and adults with spinal muscular atrophy (SMA).
Ionis and Biogen also are working together to develop tofersen (BIIB067) and IONIS-C9Rx (BIIB078), both therapies for amyotrophic lateral sclerosis (ALS), and ION859 (BIIB094) for Parkinson’s disease.
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