IONIS-MAPTRx is an antisense drug designed to selectively reduce the production of microtubule-associated protein tau (MAPT), or tau protein, in the brain. Tau misfolding leads to the accumulation of neurofibrillary tangles inside brain cells. These tangles are a hallmark feature of a spectrum of neurodegenerative diseases known as tauopathies, which include AD and some forms of frontotemporal dementia (
"In contrast to amyloid plaques that may begin to deposit in the brain for up to 20 years before the onset of AD, tau deposits are spatially and temporally associated with the brain regions where atrophy occurs and neurocognitive deficits originate. IONIS-MAPTRx is designed to reduce the production of all of the many forms of tau in all regions of the brain," said
The three-month randomized, placebo-controlled, dose escalation Phase 1/2a clinical study will evaluate the safety and activity of IONIS-MAPTRx in approximately 44 patients with mild AD. In the study, IONIS-MAPTRx is administered as a once-monthly intrathecal injection directly into the cerebral spinal fluid, similar to the way SPINRAZA® (nusinersen) is administered. Biogen has an option to develop and commercialize IONIS-MAPTRx.
"IONIS-MAPTRx is another exciting program within our strategic collaboration with Biogen," said
ABOUT IONIS AND BIOGEN
Ionis and Biogen have a broad strategic collaboration combining Biogen's expertise in neurology with Ionis' leadership in antisense technology to develop novel therapies to treat neurological disorders. SPINRAZA, an antisense drug developed with
Ionis is the leading company in RNA-targeted drug discovery and development focused on developing drugs for patients who have the highest unmet medical needs, such as those patients with severe and rare diseases. Using its proprietary antisense technology, Ionis has created a large pipeline of first-in-class or best-in-class drugs, with over three dozen drugs in development. SPINRAZA® (nusinersen) has been approved in global markets for the treatment of spinal muscular atrophy (SMA). Biogen is responsible for commercializing SPINRAZA. Drugs that have successfully completed Phase 3 studies include inotersen, an antisense drug Ionis is developing to treat patients with TTR amyloidosis, and volanesorsen, an antisense drug discovered by Ionis and co-developed by Ionis and
This press release includes forward-looking statements regarding Ionis' strategic relationship with Biogen and the development, activity, therapeutic potential, commercial potential and safety of IONIS-MAPTRx.
Any statement describing Ionis' goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, particularly those inherent in the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. Ionis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Ionis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended
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