Can you provide a brief description of your Company?
As the leader in RNA-targeted drug discovery and development, Ionis has created an efficient, broadly applicable, proprietary antisense technology platform with the potential to 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 – such as children and adults with spinal muscular atrophy (SMA). We created SPINRAZA®(nusinersen)* and are proud to have brought new hope to the SMA community by developing the first and only approved treatment for this disease.
Our sights are set on all the patients we have yet to reach with a pipeline of more than 40 drugs with the potential to treat patients with cardiovascular disease, rare diseases, neurological diseases, infectious diseases and cancer. We created TEGSEDI™ (inotersen) the world's first RNA-targeted therapeutic approved for the treatment of polyneuropathy of hereditary transthyretin (TTR) amyloidosis (ATTR) in adult patients that our affiliate Akcea Therapeutics is commercializing. Together with Akcea, we are also bringing new medicines to patients with cardiometabolic lipid disorders.
To view a PDF file of the annual reports from 2015-2017 click here. If you would like to receive a hard copy of any of Ionis' annual reports, please click here. Please note that if you are requesting that we send the annual report to an address outside of the U.S. (including Canada and Mexico) you must include your telephone number. The US Postal Service requires a phone number for shipments out of the U.S.
SPINRAZA Demonstrated Motor Function Improvements in Infants on Permanent Ventilation; No Increase in Risk of Adverse Events in Children with Scoliosis
Biogen Highlights SPINRAZA Data at the Cure SMA 2017 Annual SMA Conference
June 29, 2017 07:30 AM Eastern Daylight Time
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Biogen (NASDAQ: BIIB) will present robust efficacy and safety data from Phase 2 and 3 SPINRAZA® (nusinersen) studies at the Cure SMA 2017 ...
CARLSBAD, Calif., June 22, 2017 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) today announced the completion of enrollment in the Phase 1/2a randomized, placebo-controlled, dose escalation study of IONIS-HTTRx in patients with Huntington's disease (HD). Dosing in the final patient cohort continues, and Ionis plans to report top-line results from this study around year-end 2017. The safety and tolerability profile of IONIS-HTTRx in the completed cohorts of the Phase 1/2a study suppor...
CAMBRIDGE, Mass., June 20, 2017 /PRNewswire/ -- Akcea Therapeutics, Inc. today announced that it has launched the proposed initial public offering of its common stock.
Akcea is offering 9,620,000 shares of common stock and the underwriters will have a 30-day option to purchase up to an additional 1,443,000 shares of common stock. The initial public offering price is currently expected to be between $12.00 and $14.00 per share. Akcea has applied to list the s...
First Approved Treatment in EU for SMA
Approved to Treat Broad Range of People with SMA
Ionis Earns $50 Million Milestone
CARLSBAD, Calif., June 1, 2017 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that the European Commission (EC) has granted marketing authorization for SPINRAZA® (nusinersen) for the treatment of 5q spinal muscular atrophy (SMA). 5q SMA is the most common form of the disease and represents approximately 95% of all SMA cases. SPINRAZA is th...