Ionis, Akcea boosted by a positive PhII for their Novartis castoff cardio drug — and they plan to push ahead into pivotals
Late last year Novartis abandoned a cardio drug from Ionis’ spinoff Akcea just after the pharma giant snapped up inclisiran, going the RNAi way in guarding against heart disease in the $9.7 billion Medco buyout.
Now the pharma goliath — which is headed down the PCSK9 road with a drug it believes can be used in a mass population — can get a clearer picture of just what they gave up.
Akcea $AKCA and the mother company $IONS put out a statement early Wednesday saying that their Phase II study of AKCEA-APOCIII-LR delivered solid efficacy data, with the high dose clearly outperforming placebo in significantly reducing triglycerides as a means to cutting the risk of cardiovascular disease. In addition, investigators concluded that the drug slashed apoC-III, very low-density lipoprotein and remnant cholesterol while boosting “good” HDL levels.
Specifically, in the high dose 50 mg arm of the study, “more than 90% of patients achieved serum triglycerides of ≤ 150 mg/dL, compared to less than 5% of patients in the placebo group; mean triglyceride levels of patients at baseline was 285 mg/dL.”
Louis O’Dea, the chief medical officer at Akcea, says the data confirmed the potential for this drug, leaving them on course to pursue development for familial chylomicronemia syndrome, or FCS, as well as other rare and common cardio ailments.
Akcea shares surged 17% ahead of the bell on Wednesday.
AKCEA-APOCIII-LRx is a ligand conjugated antisense drug designed to reduce the production of apolipoprotein C-III.
Stifel’s Paul Matteis sees some upside here, but not in FCS.
Akcea plans to rapidly pursue FCS, a disease where we see a high probability-of-success (at least on a surrogate), though we are skeptical surrounding the size of the market opportunity. Thus, for the stock, we think this program becomes more/most impactful if/when Akcea/Ionis formulate plans to go into broader populations (ie. maybe TG of >500mg/dl?); this was mentioned as under consideration in the PR.
This drug was originally part of a $1.65 billion deal that Novartis signed off on 3 years ago. And over the last year, its exit on this drug was just one of a number of disruptions that hit Akcea. With its stock suffering on poor marketing performance, the top 3 execs hit the exit at Akcea in September.