- An experimental drug developed by Akcea Therapeutics and parent company Ionis Pharmaceuticals succeeded in a mid-stage study, lessening the sting of a decision last month by partner Novartis to move on without licensing the treatment.
- Akcea plans to "rapidly pursue development" of the drug, an RNA-targeting therapy aimed at reducing triglycerides in the blood, in light of the positive data, which Akcea and Ionis reported Wednesday.
- The companies' next focus is a larger trial in a rare disorder known as familial chylomicronemia syndrome, or FCS. It's a disease Akcea and Ionis know well, having earlier developed the drug Waylivra, which won approval in Europe but was rejected by regulators in the U.S.
Losing a potential pharma partner is rarely a positive development, but Akcea and Ionis have options after their triglyceride drug succeeded in a 114-patient study.
The treatment, now wholly owned, significantly reduced levels of triglycerides versus placebo across all tested doses and improved other cardiovascular risk factors, the companies said.
At the highest once-monthly dose, more than 90% of patients on the drug saw their triglyceride levels decline below 150 milligrams per deciliter, a level considered a threshold for elevated heart risk. Fewer than 5% of placebo patients achieved similar reductions.
As a result, Akcea and Ionis are moving the therapy into Phase 3 testing in people with FCS, which leads to dangerous build-ups of fat particles known as chylomicrons. Approximately 3,000 to 5,000 people have FCS globally, a small market that Akcea is already targeting in Europe with Waylivra (volanesorsen).
"Given the robust triglyceride lowering reported for LICA-APOCIII we certainly understand that FCS makes sense, though for the stock, we continue to believe that the market size here is uncertain, could be fairly small, or could at the very least take some time to build," wrote Stifel analyst Paul Matteis in a Jan. 22 note to clients.
Waylivra and AKCEA-APOCIII-LRX work similarly. Both block production of apolipoprotein CIII, a liver protein that regulates triglyceride levels.
But Waylivra posed safety risks that led to the drug's rejection by the Food and Drug Administration in the summer of 2018.
Akcea and Ionis designed their second-generation drug so that it could be dosed in lower amounts and less frequently. So far, the new formulation appears to have resulted in an improved benefit/risk profile, as treatment with AKCEA-APOCIII-LRX was not associated with any safety signals related to platelet counts, liver or kidney function.
Further development in FCS should be manageable for Akcea and Ionis, given its rare nature. The companies also hinted at the drug's potential in "other rare and common diseases associated with elevated triglycerides," but the larger studies that would entail might push the companies to seek another partnering opportunity for the drug.
Such discussions, if they materialize, could benefit both from the increased certainty of AKCEA-APOCIII-LRX's potential as well as renewed interest in heart drugs. Novartis recently spent nearly $10 billion to buy The Medicines Company and its cholesterol drug inclisiran, and Amarin's success in proving out Vascepa has stirred investor excitement.
Vascepa, notably, was initially approved for use in patients with high triglyceride levels before the REDUCE-IT study showed its broader benefit in reducing heart risks.
Shares in Akcea rose by more than 10% Wednesday, while Ionis traded higher by about 4%.