Positive clinical data at AAN 2019 reinforces Ionis' commitment to delivering life-changing medicines to patients with neurological diseases
Data and topics covered in the platform presentations and posters will include:
- The therapeutic potential of tofersen (also known as IONIS-SOD1Rx and BIIB067) in a single and multiple dose Phase 1/2 study in patients with amyotrophic lateral sclerosis (ALS)
- The strategy for identifying the optimum dose and dosing schedule for the RG6042 pivotal program to potentially maximize clinical benefit for patients with Huntington's Disease (HD)
- The benefits SPINRAZA® (nusinersen) provides for individuals with spinal muscular atrophy (SMA) across a broad range of the disease from infants to older patients, reinforcing SPINRAZA as the trusted standard of care for all patients with SMA
- An update on the long-term efficacy and safety analyses of the NEURO-TTR open-label extension study in patients living with polyneuropathy caused by hereditary transthyretin amyloidosis (hATTR)
- The observed survival benefit associated with prion protein (PrP) reductions in animal models with prion disease following treatment with PrP-targeting antisense oligonucleotides
Following is a schedule of Ionis and collaborator presentations (All times listed are in Eastern Time):
Emerging Science Session:
Tuesday, May 7, 11:45 a.m.-12:20 p.m.'Safety, PK, PD, and exploratory efficacy in single and multiple dose study of a SOD1 antisense oligonucleotide (BIIB067) administered to participants with ALS'
Sunday, May 5, 4:03 p.m.-4:14 p.m.'Development of antisense oligonucleotides for prion disease' Monday, May 6, 1:44 p.m.-1:55 p.m.'Translational Pharmacokinetic/Pharmacodynamic (PK/PD) Modeling Strategy to Support RG6042 Dose Selection in Huntington's Disease (HD)' Tuesday, May 7, 1:00 p.m.-1:11 p.m.'Nusinersen in Infants Who Initiate Treatment in a Presymptomatic Stage of Spinal Muscular Atrophy (SMA): Interim Efficacy and Safety Results from the Phase 2 NURTURE Study' Tuesday, May 7, 1:33 p.m.-1:44 p.m.'Interim Report on the Safety and Efficacy of Longer-Term Treatment with Nusinersen in Infantile-Onset Spinal Muscular Atrophy (SMA): Updated Results from the SHINE Study' Tuesday, May 7, 2:17 p.m.-2:28 p.m.'Long-Term Efficacy and Safety of Inotersen for Hereditary Transthyretin Amyloidosis: NEURO-TTR Open-Label Extension 2-Year Update' Tuesday, May 7, 2:28 p.m.-2:39 p.m.'Association of Phosphorylated Neurofilament Heavy Chain (pNF-H) Levels with Motor Function Achievement in Individuals with Spinal Muscular Atrophy (SMA) Treated with Nusinersen'
Sunday, May 5, 11:30 a.m.-6:30 p.m.'Preliminary Reliability and Validity of a Novel Digital Biomarker Smartphone Application to Assess Cognitive and Motor Symptoms in Huntington's Disease (HD)' Sunday, May 5, 11:30 a.m.-6:30 p.m.'Interim Report on the Safety and Efficacy of Longer-term Treatment with Nusinersen in Later-onset Spinal Muscular Atrophy (SMA): Results from the SHINE Study' Tuesday, May 7, 11:30 a.m.-6:30 p.m.'Responsiveness of Neuropathy Symptom and Change (NSC) Score Components in Inotersen Treatment of Hereditary Transthyretin Amyloidosis Polyneuropathy' Tuesday, May 7, 11:30 a.m.-6:30 p.m.'Quality of Life (QOL) in Patients with Transthyretin Amyloidosis Accompanied by Peripheral Neuropathy (PN)' Tuesday, May 7, 11:30 a.m.-6:30 p.m.'The Impact of Hereditary Transthyretin Amyloidosis (hATTR) on Work: The Patients' Perspectives'
Complete abstracts, details on presentation times and changes to presentation dates can be found on the AAN website. The above listed dates are subject to change. Please check www.aan.com for the latest information.
Ionis Neuro Franchise Webcast
Ionis will hold a webcast in conjunction with the 2019 AAN Annual Meeting on
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 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 diseases including cardiovascular diseases, neurological diseases, infectious diseases, pulmonary diseases and cancer.
To learn more about Ionis follow us on twitter @ionispharma or visit www.ionispharma.com.
Ionis' Forward-looking Statement
This press release includes forward-looking statements regarding Ionis' business, and the therapeutic and commercial potential of SPINRAZA®, TEGSEDI™ (inotersen), WAYLIVRA™ (volanesorsen) and Ionis' technologies and products in development, including the business of
In this press release, unless the context requires otherwise, "Ionis," "Company," "we," "our," and "us" refers to
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Ionis Pharmaceuticals Investor Contact: D. Wade Walke, Ph.D., Vice President, Investor Relations, 760-603-2741; or Ionis Pharmaceuticals Media Contact: Roslyn Patterson, Vice President, Corporate Communications, 760-603-2681