Ionis' neurological franchise marks year of achievement
In the news release, Ionis' neurological franchise marks year of achievement, issued
Ionis' neurological franchise marks year of achievement
- Potential breakthrough therapies for Huntington's, Alzheimer's, ALS and Parkinson's among numerous programs advancing in the clinic
- Neurological R&D team received several prestigious honors
- Advances in neurological research highlighted in leading peer-reviewed journals
Through its partnerships, Ionis' neurological franchise achieved important milestones during the year, including:
- Enrolling the first patient in a pivotal study of IONIS-HTTRx (RG6042) for patients with Huntington's Disease (HD) by
- Advancing tofersen, a drug designed to reduce production of superoxide dismutase 1 (SOD1) in patients with amyotrophic lateral sclerosis (ALS), to a phase 3 pivotal trial by Biogen
- Licensing of IONIS-MAPTRx (BIIB080), a program currently in a Phase 1/2 clinical study in patients with mild Alzheimer's disease (AD) by Biogen
- Advancing an ongoing study by Biogen of IONIS-C9Rx (BIIB078) targeting the C9orf72 mutation in ALS
- Initiation of a clinical study of ION859 (BIIB094) as the first antisense drug to potentially treat people with Parkinson's disease
- Advancing six medicines that Ionis expects to enter the clinic in 2020, including a treatment for Angelman Syndrome.
- Advancement of 4 undisclosed neurological disease targets into drug discovery under Ionis' collaboration with Biogen
- Completion of a Phase 1 study of Ionis' TTR LICA drug and initiation of a Phase 3 study in patients with TTR amyloidosis with polyneuropathy
- Continued commercial success of SPINRAZA ®. In first nine months of 2019, sales of the blockbuster increased nearly 25 percent from the same period in 2018. Marketed by Biogen, SPINRAZA is the first and only approved medicine for treatment of all forms of spinal muscular atrophy (SMA) and is a global foundation of care, administered to more than 9,300 patients in 40 countries
Promising new Ionis-owned therapies advancing into the clinic include ION283 for Lafora disease, ION373 for a form of leukodystrophy and ION716 for prion diseases.
"It's been a privilege to see Ionis' neurology program mature from its early days as a nascent project into the industry's leading neurological disease pipeline, one that is having a meaningful impact on patients," said
Dr. Bennett, a founding member of Ionis and a 2019 Breakthrough Prize laureate for his contributions to the discovery and development of SPINRAZA, was included on the 2019 "Highly Cited Researchers" list of the world's most influential researchers, for being among the top 1 percent of most-cited authors in their field.
Ionis' neurological franchise team also received the inaugural 2019 Sean M. Healey International Prize for Innovation in ALS. The global prize rewards excellence in research for exceptional discoveries leading to a transformative advance in therapy development in ALS. Other recipients of this year's prize included teams led by
Groundbreaking neurological research being done at Ionis was also highlighted in the scientific literature. In 2019, members of Ionis' neurological franchise team contributed 16 articles to leading peer-reviewed journals, including Nature Medicine, Nature Neuroscience, the
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.
Ionis' Forward-looking Statement
This press release includes forward-looking statements regarding Ionis' business, financial guidance and the therapeutic and commercial potential of SPINRAZA (nusinersen) and Ionis' technologies and products in development. 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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Ionis Pharmaceuticals Investor Contact: D. Wade Walke, Ph.D., Vice President, Investor Relations, 760-603-2741; Ionis Pharmaceuticals Media Contact: Roslyn Patterson, Vice President, Corporate Communications, 760-603-2681