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

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.

Also Read: Tale Of Two Ionis-Partnered Neurology-Focused Early Stage Assets-Biogen Discontinues One, Decides Not To Exercise Licensing Option For Another.

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.

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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