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Ulefnersen, an RNA-targeted therapy being developed by Ionis Pharmaceuticals and Otsuka Pharmaceutical, improved measurements of functional impairment and survival in patients with a rare, genetic ALS subtype in a Phase 3 trial. The partners plan to approach the FDA with the results to determine potential pathways for approval.

A late-stage test of Roche and Ionis Pharmaceuticals’ sefaxersen showed that the drug reduced protein in the urine, which suggests the RNA-targeting therapy could improve kidney function and delay transplant.

Zanvastro, an antisense oligonucleotide therapy, represents the first independent neurology launch for Ionis Pharmaceuticals, which intends to leverage the foothold for more approvals in rare neurodegenerative diseases, including Angelman syndrome.

The approval of Ionis Pharmaceuticals’ Tryngolza for severe hypertriglyceridemia could spur “substantial growth” for the product, according to William Blair.

With Phase 3 data showing what analysts called a “potentially best-in-class profile,” Arrowhead Pharmaceuticals plans to submit for FDA approval of plozasiran by the end of the year—hoping to follow to market Ionis Pharmaceuticals’ Tryngolza, which got the regulator’s nod in severe hypertriglyceridemia last month.

Roche’s decision to discontinue the Ionis-partnered trials came soon after the biotech sustained a late-stage failure in ATTR-CM.

The failure of AstraZeneca and Ionis’ Wainua in a late-stage study of ATTR-CM casts doubt on Alnylam’s next-generation candidate but is good news for others in the space, including BridgeBio and Intellia Therapeutics.