Ionis Reports Solid 1Q Results and Pipeline Progress; Slightly Increasing Our FVE
Ionis' first-quarter results were on track with our expectations. We're increasing our global probability of approval for rare disease drug Tegsedi from 70% to 80%, following the recent European approval, and plan to increase our assumed probability of approval for ALS drug tofersen following recent early data. We expect to slightly raise our current $52 per share fair value estimate as a result. Overall, Ionis' antisense technology is gaining traction and could lead to the creation of a moat, b...Ionis Pharmaceuticals is engaged in discovering and developing RNA-targeted therapeutics. The company has two commercial medicines, SPINRAZA and TEGSEDI. SPINRAZA (nusinersen) injection for intrathecal use is a survival motor neuron-2, directed antisense oligonucleotide indicated for the treatment of spinal muscular atrophy in pediatric and adult patients. TEGSEDI (inotersen) injection is a Generation 2+ antisense medicine and an approved subcutaneous RNA-targeting medicine designed to treat people with polyneuropathy caused by hereditary transthyretin amyloidosis.
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