- 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.
© 2024 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.
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