Ionis Pharmaceuticals, Inc.’s (IONS - Free Report) wholly owned subsidiary, Akcea Therapeutics, announced positive data from an interim analysis of a phase I/IIa study on its pipeline candidate, IONIS-ANGPTL3-L RX. Data were presented at the American Heart Association Meeting.
In the study, patients who received multiple doses of ANGPTL3-L RX (10 mg, 20 mg, 40 mg and 60 mg) experienced dose-dependent and statistically significant mean reduction of up to 83%, 66% and 35% in angiopoietin-like 3, triglycerides and LDL-cholesterol, respectively.
The candidate demonstrated a favorable safety and tolerability profile with neither any discontinuation due to adverse events nor a clinically meaningful decline in platelets.
The encouraging study results support the development of IONIS-ANGPTL3-L RX for the treatment of multiple diseases such as severe dyslipidemias and nonalcoholic steatohepatitis.
We note that IONIS-ANGPTL3-L RX is being developed utilizing LIgand Conjugated Antisense (LICA) technology.
Ionis has a deep pipeline with several candidates in the mid or final stages of development. The most advanced candidate in the company’s pipeline is Spinraza, which is currently under review in the both the U.S. and the EU for the treatment of patients with spinal muscular atrophy. Other promising candidates include IONIS-TTRRx (phase III – transthyretin amyloidosis) and volanesorsen (phase III – familial chylomicronemia syndrome and familial partial lipodystrophy) among others.
Ionis currently carries a Zacks Rank #3 (Hold).
IONIS PHARMACT Price
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