Ionis Pharmaceuticals (IONS) Present Positive Two-year Results from Phase 2 Study of donidalorsen

November 9, 2023 12:43 PM EST

Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced positive results from an ongoing Phase 2 open-label extension (OLE) study evaluating the safety and efficacy of its investigational prophylactic medicine, donidalorsen, in patients with hereditary angioedema (HAE), a rare and life-threatening genetic disease. Over the two years, patients treated with donidalorsen via subcutaneous injection showed an overall sustained mean reduction in HAE attack rates of 96% from baseline, from 2.70 to 0.06 attacks per month, across all dosing groups. Furthermore, all patients treated with donidalorsen reported a clinically meaningful improvement in quality of life as measured by the Angioedema Quality of Life Questionnaire (AE-QoL) over two years. Treatment with donidalorsen was well tolerated in the studies, and there were no serious adverse events.

"Hereditary angioedema is a significant healthcare challenge for which there is an ongoing need for long-term, sustained prophylactic treatment offering patients significant efficacy and tolerability that is easy to use," said Richard S. Geary, Ph.D., executive vice president and chief development officer at Ionis. "We are very encouraged by the demonstrated safety, efficacy, and quality of life profile of donidalorsen. The two-year OLE results further support donidalorsen as a potentially compelling prophylactic treatment option for patients with hereditary angioedema. We look forward to reporting pivotal topline Phase 3 results in the first half of next year."

Following the 13-week blinded, placebo-controlled Phase 2 study, patients were eligible for enrollment in the OLE study. Of the 20 Phase 2 study participants, 17 entered the OLE study and were on a fixed 13-week dosing period where they received donidalorsen 80 mg every four weeks. From week 17 through two years, patients entered a flexible dosing period where they either received donidalorsen 80 mg every four weeks, 80 mg every eight weeks, or 100 mg every four weeks.

In a subset analysis also presented at the American College of Allergy, Asthma & Immunology (ACAAI) Annual Scientific Meeting, 62.5% of patients receiving donidalorsen 80 mg every eight weeks remained attack-free over the two-year period and had a sustained mean reduction in HAE attack rates of 83% from baseline.

Injection site (IS) discoloration and IS reaction were the only study drug-related treatment-emergent adverse events (TEAEs) reported in more than one patient (n=2, 11.8% each). No serious adverse events were reported in the OLE study, and no TEAEs led to study discontinuation.

The poster presentations about the Phase 2 Open-Label Extension Study and the sub-analysis of the eight-week dosing arm are available on the ACAAI website.

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