RE:RE:RE:RE:RE:RE:RE:RE:RE:Unfortunate timing - Nicholls article on RVX - 09/15The first in class issue is more of a safety concern of this new class of drug, which are the BET inhibitors. There is always a chance that with treatment of greater than 6 months, that something unexpected may pop up. However, with BETonMACE being designed around MACE (including death), it would not be unexpected to see some deaths occuring in both treatment arms. The questions would be are there more deaths in the RVX-208 group than the control, or are there deaths due to causes unrelated to diabetes/kidney disease/pre-existing cardiovascular disease?
One reason I bring this up is because of what happened with Zafgen (ZFGN) this week and their obesity drug beloranib. They had a death in a patient treated with their drug beloranib. This trial was in patients with a rare genetic condition called Prader Willi syndrome. These patients already have a very high chance of death. So a death of any patient was not entirely unexpected. But the market wiped out over 2/3 of their market cap this week and FDA put a halt on their trial for now.
My point is that new, first in class therapies carry the risk of encountering the unexpected. So far, the only problem has been the transient elevation in liver enzymes in a small number of patients. Michael Sweeney attributed this to increased bile acid production. Bile acids are made from cholesterol in the liver, so this may just be a side effect of ehanced RCT in some patients. Nothing to worry about, but with larger trial, longer trial and sicker baseline patients, these unexpected adverse events are definitely something that could affect the future of RVX-208.
See, I can be doom and gloom sometimes too! That's why we have clinical trials!
And regarding the orphan drug program vs. orphan drug designation, I look at this more positively than you do onlytruthhere. Personally, I find it very positive that they can begin looking at an entirely new class of complement pathway mediated disorders that RVX-208 and follow on molecules could treat. But to be clear, they do not have orphan drug designation. But it is true that they are entering an entire new area a biology separate from the diabetes/cardiovascular disease space. That in itself is exciting and promising in my view.
Best,
BDAZ