What: Isis Pharmaceuticals (ISIS +4.25%) is up 11% today after presenting data from two trials of the company's lipoprotein(a), or Lp(a), reducing drugs at the American Heart Association Scientific Sessions on Sunday. The biotech also released its third quarter earnings today, but there wasn't anything in the release to get too excited about; ISIS technically had an income on a pro forma basis, but that was only because of payments from partners for licensing and reaching milestone.
So what: Lp(a) is a combination of LDL-cholesterol and apolipoprotein(a). High levels of Lp(a) are associated with an increased risk of coronary heart disease, atherosclerotic plaque formation, and calcific aortic valve stenosis.
In a phase 2 trial, ISIS-APO(a)Rx was able to reduce Lp(a), up to 94%, with a mean reduction of 71% in patients with high levels of Lp(a).
Isis also has a stronger version of the drug called ISIS-APO(a)-LRx that's being tested in a phase 1/2a trial. At the highest dose tested, 80 mg, patients saw reductions in Lp(a) of up to 97% and a mean reduction of 79%. The drug has a long effect: 90 days after the injection Lp(a) levels were still nearly 50% of what they were before the injection, potentially supporting quarterly injections.
The lower doses might also be an option because after patients received multiple injections of the lower doses -- 10 mg, 20 mg, or 40 mg of ISIS-APO(a)-LRx -- their Lp(a) went down by up to 99% with a mean reduction of up to 92%.
Now what: The L in ISIS-APO(a)-LRx stands for Ligand Conjugated Antisense (LICA). Beyond ISIS-APO(a)-LRx, ISIS has seven other LICA drugs in its pipeline, so some of the excitement today isn't about ISIS-APO(a)-LRx specifically, but about the fact that the new platform appears to be working as advertised.
It would be difficult to design an oral version of ISIS' antisense molecules because the stomach likes to chew up oligonucleotides that the drugs are made of, but the LICA technology makes them last so long that patients may only have to be injected four times a year -- or potentially even less often. While most people don't like injections, four injections a year is a good trade-off for not having to remember to pop a pill every day.
