Ionis Pharmaceuticals (IONS) Present Positive Two-year Results from Phase 2 Study of donidalorsen
- Wall St eases as investors brace for inflation data
- Stocks slip as traders hold fire ahead of US inflation data
- Oil gains on potential OPEC+ cuts, curbed by jump in U.S. crude stocks
- US market now accounts for 60% of total world investable equity market value - UBS
- Dollar bounces before inflation data
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
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.
Serious News for Serious Traders! Try StreetInsider.com Premium Free!
You May Also Be Interested In
- Applied Therapeutics (APLT) Announces FDA Acceptance and Priority Review of New Drug Application for Govorestat for the Treatment of Classic Galactosemia
- Palatin Technologies (PTN) Reports Results of PL9643 MELODY-1 Phase 3 Trial
- Vivani Medical (VANI) Announces Positive NPM-115 Preclinical Weight Loss Data Comparable to Ozempic/Wegovy and Discloses NPM-139 as Semaglutide as Strategy Shifts to Prioritize Obesity Portfolio
Create E-mail Alert Related CategoriesCorporate News, FDA
Sign up for StreetInsider Free!
Receive full access to all new and archived articles, unlimited portfolio tracking, e-mail alerts, custom newswires and RSS feeds - and more!