News Releases

Ionis Pharmaceuticals Highlights Antisense Drugs to Treat Neurological Diseases at the AAN Meeting
Ionis and its collaborators to present more than 12 presentations and posters on Ionis' neurological disease programs
Webcast to provide update on nusinersen Phase 2 open-label study in infants with SMA and to review Ionis' other neurological disease programs scheduled for Thursday, April 21 at 2:00 p.m. EDT

CARLSBAD, Calif., April 15, 2016 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that Ionis and its collaborators will present a dozen presentations and posters on programs from Ionis' neurological disease franchise at the American Academy of Neurology (AAN) meeting in Vancouver. 

Ionis Pharmaceuticals (PRNewsFoto/Ionis Pharmaceuticals, Inc.)

At the meeting, Dr. Claudia Chiriboga, associate professor of clinical neurology and pediatrics at Columbia University and chief of pediatric neurology at Harlem Hospital Center, will present an update on Ionis' ongoing open-label Phase 2 clinical study on nusinersen in infants with spinal muscular atrophy (SMA).  The presentation titled 'Interim Results of a Phase 2 Clinical Study of Nusinersen in Patients with Infantile-Onset Spinal Muscular Atrophy' will be given at the Neuromuscular and Clinical Neurophysiology poster session on Wednesday, April 20 at 8:30 a.m. PDT and during a short 'Data Blitz' presentation at noon PDT.  Ionis plans to conduct a webcast the following day, April 21 at 11:00 a.m. PDT /2:00 p.m. EDT to review these data in more detail.

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 Early Clinical Development of IONIS-HTTRx, the First HTT-Lowering Drug to be Tested in Patients with Huntington's Disease' on Saturday, April 16 at 9:00 a.m. PDT.  IONIS-HTTRx is the first drug to enter clinical development that is designed to directly target the cause of HD.  The Phase 1/2 study was initiated in July of 2015 and continues to enroll patients with HD. 

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  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 at 6:30 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 at 5:00 p.m. PDT and poster presentation on April 20 at 8:30 a.m. PDT.
  • 'Silencing of Myotonic Dystrophy Protein Kinase (DMPK) Does Not Affect Cardiac or Muscle Function in Mice' oral presentation on April 17 at 5:05 p.m. PDT and on April 20 at 7:30 a.m. PDT.
  • 'IONIS-DMPK-2.5Rx in Healthy Volunteers: A Placebo-controlled, Randomized, Single Ascending-dose Phase 1/2 Study' poster presentation on April 18 at 8:30 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 at 8:30 a.m. PDT.
  • 'Targeting ATXN2 Using Antisense Oligonucleotides as a Treatment for Spinocerebellar Ataxia Type 2 (SCA)' oral presentation on April 19 at 2:30 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 on April 20 at 8:00 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 at 8:15 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 at 8:30 a.m. PDT.

At 11:00 a.m. PDT /2:00 p.m. EDT, April 21, 2016, Ionis will conduct a webcast to discuss the latest data presented at the AAN meeting, including the nusinersen Phase 2 study data.  A live audio webcast of the presentation will be available on the "Investors & Media" section of the Company's website,  Interested parties may listen to the call by dialing 877-443-5662.  A replay will be available for a limited time.  The slides presented on the webcast will be available on Ionis' website at at the time of the webcast and for a limited time after.

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

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 December 31, 2015, which is on file with the SEC.  Copies of this and other documents are available from the Company.

In this press release, unless the context requires otherwise, "Ionis," "Company," "we," "our," and "us" refers to Ionis Pharmaceuticals and its subsidiaries.

Ionis Pharmaceuticals™ is a trademark of Ionis Pharmaceuticals, Inc.  Akcea Therapeutics™ is a trademark of Ionis Pharmaceuticals, Inc. 

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SOURCE Ionis Pharmaceuticals, Inc.

For further information: 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