Ionis Q1 2020 financial results and business updates

    • Commercial revenue from SPINRAZA® (nusinersen) royalties increased by more than 10 percent to $66 million compared to Q1 2019
    • Product sales from TEGSEDI® (inotersen) and WAYLIVRA® (volanesorsen) more than doubled to $15 million compared to Q1 2019
  • First quarter results in line with projections to be meaningfully profitable this year
    • Operating loss and net loss of $61 million and $48 million, respectively, on a GAAP basis
    • Non-GAAP operating loss and net loss of $20 million and $15 million, respectively
  • Cash position of $2.4 billion provides substantial financial strength to continue executing on strategic goals
  • Roche completed enrollment in the global, GENERATION HD1 Phase 3 study in patients with Huntington’s disease
  • Initiated the CARDIO-TTRansform Phase 3 clinical trial for AKCEA-TTR-LRx in patients with TTR-mediated amyloid cardiomyopathy
  • Two medicines granted Fast Track Designation by the U.S. FDA
    • AKCEA-APO(a)-LRx for the treatment of cardiovascular disease due to elevated Lp(a) levels
    • IONIS-C9Rx for the treatment of C9orf72-ALS
  • Ionis generated more than $20 million as numerous partnered medicines advanced
    • $10 million from AstraZeneca for ION532, targeting APOL1 for the treatment of kidney disease
    • $7.5 million from Biogen for IONIS-MAPTRx for the treatment of Alzheimer’s disease
    • $5 million from Dynacure for IONIS-DNM2-2.5Rx for the treatment of centronuclear myopathies
  • Ionis and Akcea reported positive topline results for AKCEA-APOCIII-LRx and vupanorsen (AKCEA-ANGPTL3-LRx)
  • Results from the Phase 2 study of AKCEA-APO(a)-LRx in patients with Lp(a)-driven cardiovascular disease, highlighting the favorable safety and tolerability profile and the potential to address a major area of unmet need, were published in the New England Journal of Medicine
  • Initiated a Phase 1 study of ION224, an Ionis-owned medicine in development for the treatment of NASH
  • Initiate the Phase 3 study of AKCEA-APOCIII-LRx in patients with FCS
  • Refile WAYLIVRA new drug application for U.S. marketing authorization
  • File for WAYLIVRA marketing approval in Brazil with PTC Therapeutics
  • Report clinical proof-of-concept results for four or more programs
  • Initiate a first-in-human study of ION541 in patients with sporadic ALS, conducted by Biogen
  • Advance five or more new medicines into development, including several Ionis-owned medicines