Tom3 schreef op 31 december 2019 08:28:
Gelezen op de AKCEA Yahoo site:
For anyone to estimate the size of APOCIII-Lrx market, below is the quote from Ionis website:
"ApoC-III is an important emerging target linking hypertriglyceridemia with cardiovascular disease (CVD). In several studies, apoC-III levels are an independent risk factor for CVD. Further, its presence on lipoproteins may increase their atherogenicity. A study in the New England Journal of Medicine reported that out of a sample of over 100,000 people, individuals with an apoC-III loss-of-function mutation had a reduced risk of clinical coronary heart disease. Each decrease of 1mg/dL in plasma levels of apoC-III was associated with a 4% decrease in the risk of incident coronary heart disease. Triglycerides may also play a role in cardiovascular risk. In two separate studies encompassing nearly 20,000 patients, as triglyceride levels increased, so did the risk of a cardiovascular event. In summary, apoC-III impacts triglyceride levels and may also increase inflammatory processes. This combination of effects makes apoC-III a valuable target for reducing the residual CVD risk in patients already on statin therapy, or for whom triglycerides are poorly controlled."
In two months, we will hear the outcome of phase II study, in this study 115 patients were enrolled (randomized, double-blind dose ranging study). Primary outcome measure was reduction in serum triglyceride levels from baseline. Positive outcome and no serious side effects are expected. Novartis had the right to this drug but returned the right to Akcea. Words cannot describe what this means. What I write here will be seen as hyperbole for sure. I've mentioned this earlier, this drug alone will change the fortune of Akcea (and Ionis). I've written several times on this board that market for cardiovascular diseases is nearly unlimited. As an example, Pfizer earned over $110 billions during the epic ten year period on Lipitor alone and it is still earning $2 billion a year from generic Lipitor (atorvastatin). APOCIII-Lrx market won't be as large as Lipitor but for a company like Akcea many investors will retire on the day of FDA approval. Most drugs fail to get FDA approval, here lies difference between Ionis drugs vs. rest of the drugs, Ionis drugs (not like other drugs) are targeting etiologies with outsized efficacy outcome, now with breakthrough LICA chemical engineering side effects are pretty much eliminated. Phase III study will be difficult because in this study clinical outcome will have to be tested and proven but given all the facts we have my prediction is "handwriting is on the wall", nothing is 100% for sure but expect a fortune if you are patient enough.