Isis Pharmaceuticals (ISIS) has had a long, tough history developing antisense-based therapeutics. I've lost count of the number of promising candidates they've had (and promising deals they've signed). But the latest one seems to be progressing: mipomersen, designed to block production of the ApoB lipoprotein.
That should lower LDL, and help with several other cardiovascular risk factors at the same time. Isis and their partner Genzyme (GENZ) have just announced that a trial of the drug in patients with homozygous familial hypercholesterolemia showed significant LDL reductions (25 per cent). These people are already maxed out on statin therapy, and still have huge LDL levels, so this does seem to represent an advance.
And Genzyme knows all about getting drugs through for very small patient populations (and charging accordingly) - they're definitely a good partner for this sort of drug. But both they and Isis would like for mipomersen to be used more widely. The next target are patients with the heterozygous form of hypercholesterolemia, and then they'll try to move on to various other statin-intolerant patients with risky LDL levels.
Isis could use a success. They were the first to get an antisense therapy approved (Fomiversen), but it really has never brought in much revenue. Mipomersen, as an injectable, is never going to go out and take over the world like the stating drugs, but it could still be a winner in its own (larger) niche.