Ionis Pharmaceuticals Reveals Late-Stage Donidalorsen Data, Analyst Says Results Show Competitive Hereditary Angioedema Profile

Friday, Ionis Pharmaceuticals Inc 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.

The OASIS-Plus open-label extension study results showed that Q4W patients and Q8W patients achieved 93% and 92% improvement from baseline in attack rates.

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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