Press Releases
Ionis Pharmaceuticals Highlights Antisense Drugs to Treat Neurological Diseases at the AAN Meeting
At the meeting, Dr.
Ionis' Huntington's Disease (HD) drug, IONIS-HTTRx, will be highlighted in the session 'Contemporary Clinical Issues Plenary' in an oral presentation titled 'Discovery and
In total, Ionis' neurological disease programs will be highlighted in nine presentations and five posters. Complete abstracts for the presentations can be found on the AAN website at www.aan.com. In addition to the two presentations noted above, Ionis and its collaborators will present the following presentations:
- 'Targeting TAU in Mouse Models of Dementia Reveals a Toxic Tau Isoform Amendable to Therapeutic Intervention'; oral presentation on
April 16 at6:30 a.m. PDT . - 'Antisense Oligonucleotide to LRRK2 Ameliorate Alpha-synuclein Pathology and Behavioral Deficit Induced by Pre-formed Alpha-synuclein Fibrils' oral presentation on
April 16 at5:00 p.m. PDT and poster presentation onApril 20 at8:30 a.m. PDT . - 'Silencing of Myotonic Dystrophy Protein Kinase (DMPK) Does Not Affect Cardiac or Muscle Function in Mice' oral presentation on
April 17 at5:05 p.m. PDT and onApril 20 at7:30 a.m. PDT . - 'IONIS-DMPK-2.5Rx in
Healthy Volunteers : A Placebo-controlled, Randomized, Single Ascending-dose Phase 1/2 Study' poster presentation onApril 18 at8:30 a.m. PDT . - 'Study Design of a Phase 1/2a Trial with IONIS-DMPK-2.5Rx for the Treatment of Myotonic Dystrophy Type I' poster presentation on
April 18 at8:30 a.m. PDT . - 'Targeting ATXN2 Using Antisense Oligonucleotides as a Treatment for Spinocerebellar Ataxia Type 2 (SCA)' oral presentation on
April 19 at2:30 p.m. PDT . - 'The Effect of Bolus Volume and Mechanical Forces on the Biodistribution of ASOs (Antisense Oligonucleotides)
Following Lumbar Intrathecal Administration in Cynomolgus Monkeys' oral presentation onApril 20 at8:00 a.m. PDT . - 'Kinetics of ASO (Antisense Oligonucleotides) Distribution and Pharmacodynamics in the CNS After an Intrathecal Bolus Dose in Rat' oral presentation on
April 20 at8:15 a.m. PDT . - 'Snca Targeted Antisense Oligonucleotides Mediate Progression of Pathological Deposition in Alpha Synuclein Rodent Transmission Models of Parkinson's Disease' poster presentation on
April 21 at8:30 a.m. PDT .
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ABOUT
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 a dozen drugs in mid- to late-stage development. Drugs currently in Phase 3 development include volanesorsen, a drug Ionis is developing and plans to commercialize through its wholly owned subsidiary, Akcea Therapeutics, to treat patients with either familial chylomicronemia syndrome or familial partial lipodystrophy; IONIS-TTRRx, a drug Ionis is developing with GSK to treat patients with all forms of TTR amyloidosis; and nusinersen, a drug Ionis is developing with Biogen to treat infants and children with spinal muscular atrophy. Ionis' patents provide strong and extensive protection for its drugs and technology. Additional information about Ionis is available at www.ionispharma.com.
This press release includes forward-looking statements regarding Ionis' strategic relationship with Biogen, the discovery, development, activity, therapeutic and commercial potential and safety of nusinersen for the treatment of spinal muscular atrophy and the discovery, development, activity, therapeutic potential, safety and commercialization of drugs in Ionis' neurological disease franchise. 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
In this press release, unless the context requires otherwise, "Ionis," "Company," "we," "our," and "us" refers to
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SOURCE
D. Wade Walke, Ph.D., Vice President, Corporate Communications and Investor Relations, 760-603-2741; Amy Williford, Ph.D., Associate Director, Corporate Communications, 760-603-2772