Benzinga - by Vandana Singh, Benzinga Editor.
Friday, Ionis Pharmaceuticals Inc (NASDAQ:IONS) announced results from the Phase 3 OASIS-HAE and OASISplus studies of donidalorsen in patients with hereditary angioedema (HAE).
Donidalorsen had a favorable safety and tolerability profile across both studies, including when self-administered via an autoinjector.
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Based on these data, Ionis is pursuing regulatory approval of donidalorsen as a potential treatment for HAE, a condition characterized by recurrent attacks of severe swelling (angioedema) in various parts of the body, including the hands, feet, genitals, stomach, face, and/or throat.
- In the Phase 3 OASIS-HAE study, patients were treated with donidalorsen (80 mg) via subcutaneous injection every four weeks (Q4W) or every eight weeks (Q8W) or placebo over 24 weeks.
- The study met its primary endpoint, demonstrating an 81% lower monthly HAE attack rate with donidalorsen Q4W compared to placebo over weeks one to 25 and a 55% reduction with Q8W.
- In weeks five to 25, donidalorsen Q4W significantly reduced mean monthly HAE attack rates by 87% vs placebo.In the same time frame, treatment with donidalorsen Q4W reduced severe to moderate attacks per month by 89%.
- Donidalorsen Q4W also reduced HAE attacks that require acute therapy by 92%.
- Donidalorsen Q8W had a similar benefit as Q4W dosing over time on attack rate reduction and quality of life measures.
- Donidalorsen was well-tolerated.
Patients who switched to donidalorsen from prior prophylactic treatment also showed a 62% further reduction in mean monthly HAE attack rates from baseline, and 84% of patients who switched reported a preference for donidalorsen.
Ionis plans to file an FDA marketing application this year.
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William Blair says, “Ultimately, we see donidalorsen’s profile as generally superior to current prophylactic options, with less frequent dosing and at-home autoinjector dosing potential being further differentiators.”
William Blair writes that the next 12-18 months could be a period of significant growth for Ionis and maintains its Outperform rating.
Price Action: IONS shares are up 3.15% at $37.70 at last check Friday.
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| Mar 2022 | Guggenheim | Initiates Coverage On | Buy | |
| Mar 2022 | Citigroup | Initiates Coverage On | Sell | |
| Feb 2022 | SVB Leerink | Maintains | Market Perform |
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