Analyst Says Ionis' Additional Hereditary Angioedema Study Data Supports 'Highly Competitive Profile'

Ionis Pharmaceuticals Inc IONS announced additional interim data from a Phase 2 open-label extension (OLE) study of donidalorsen for hereditary angioedema.
Interim data presented in November 2022 showed that treatment with donidalorsen resulted in an overall sustained mean reduction in HAE attack rates of 95% from baseline.
In the latest update, patients treated for one year with donidalorsen showed a clinically meaningful 24-point mean improvement in their Angioedema Quality of Life (AE-QoL) total score relative to baseline, with improvements observed in all domains.
An improvement of 6 points or more is considered clinically meaningful.
Related: Ionis Regains Rights To Thrombosis Candidate From Bayer.
William Blair notes donidalorsen showed improvements in all domains, which include functioning, fatigue/mood, fears/shame, and food.
The analyst views these additional interim results as highly encouraging for donidalorsen, and believes they continue to support a highly competitive profile in an increasingly crowded space.
It reiterates Outperform rating.
The analyst says that donidalorsen is a potentially differentiated and wholly-owned growth driver for Ionis if the Phase 2 dataset can be replicated in the larger OASIS studies expected to read out in 2024.
Ionis continues to advance a robust mid- and late-stage pipeline while generating significant non-dilutive funds through research collaborations to support internal efforts, which limits capital risk in this uncertain macro environment.
Price Action: IONS shares are down 0.18% at $38.53 on the last check Tuesday.

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