Safety Concerns From Ionis' Pivotal Amyloidosis Trial Bolsters Alnylam's Program Outlook
Safety concerns from Ionis’ pivotal NEURO-TTR study evaluating inotersen (formerly IONIS-TTRRx) in familial amyloid polyneuropathy--a rare, hereditary disease that causes toxic buildup in the nervous system--sent Alnylam’s and Ionis’ stocks ricocheting in opposite directions. While Alnylam’s amyloidosis drug patisiran, which is also in pivotal testing and is expected to release top-line results this September, has its own side effect issues, we believe the string of severe adverse events with Io...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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