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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 45 drugs in development. SPINRAZA® (nusinersen) has been approved in global markets for the treatment of spinal muscular atrophy (SMA). Biogen is responsible for commercializing SPINRAZA. TEGSEDI (inotersen) and WAYLIVRA (volanesorsen) are two antisense drugs that Ionis discovered and successfully advanced through Phase 3 studies. TEGSEDI is approved in the E.U. for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis, or hATTR, and is currently under regulatory review in the U.S. and Canada. WAYLIVRA is under regulatory review for marketing approval in the U.S., EU and Canada for the treatment of patients with familial chylomicronemia syndrome, or FCS. WAYLIVRA is also in a Phase 3 study in patients with familial partial lipodystrophy, or FPL. Akcea Therapeutics, an affiliate of Ionis focused on developing and commercializing drugs to treat patients with serious and rare diseases, will be responsible for commercializing TEGSEDI and WAYLIVRA. Ionis' patents provide strong and extensive protection for its drugs and technology.
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...