CARLSBAD, Calif., Dec. 19, 2019 /PRNewswire/ — Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in RNA-targeted therapeutics, announced today that 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.
ABOUT THE IONIS AND BIOGEN COLLABORATION
Ionis and Biogen have a broad collaboration combining Biogen’s expertise in neurology with Ionis’ leadership in antisense technology to develop novel therapies to treat neurological disorders. SPINRAZA, the first commercial drug from this collaboration, is currently a global foundation of care for treatment of patients with spinal muscular atrophy (SMA). Ionis and Biogen are also developing tofersen (BIIB067), which Biogen licensed in 2018, and IONIS-C9Rx (BIIB078), both for amyotrophic lateral sclerosis (ALS), and ION859 (BIIB094) for Parkinson’s disease.
ABOUT IONIS PHARMACEUTICALS, INC.
As the leader in RNA-targeted drug discovery and development, Ionis has created an efficient, broadly applicable, drug discovery platform called antisense technology that can treat diseases where no other therapeutic approaches have proven effective. Our drug discovery platform has served as a springboard for actionable promise and realized hope for patients with unmet needs. We created the first and only approved treatment for both children and adults with spinal muscular atrophy as well as the world’s first RNA-targeted therapeutic approved for the treatment of polyneuropathy in adults with hereditary transthyretin amyloidosis. Our sights are set on all the patients we have yet to reach with a pipeline of more than 40 novel medicines designed to treat a broad range of disease, including cardiovascular, neurological, infectious and pulmonary diseases and many more.
To learn more about Ionis visit www.ionispharma.com and follow us on twitter @ionispharma.