Well...If you really want to know. This is old news.
The problem with VTS270 has to do with the trial design as required by the FDA. MNK sent out a video a few weeks ago explaining the problem. The trial was a 2/1. That is, for every 3 people in the trial , 2 get the drug and one gets sham. The trial also had a "rescue protocol". That is, if someone in the sham group declined rapidly, they could be "rescued" and given the drug. Decline (disease progression) was based on a point system.
I don't know the point system, but I will give an example. Lets say for description purposes, a decline of 20 points triggered the rescue protocol. If the patient dropped 20 points, they were taken out of the sham group and put "on drug".
Here is the problem: The FDA treats the patients on the rescue protocol as "missing data". As such, they assigned the patient a universal decline in points (for this example, 5 points). So... If a patient fell 22 points and was put "on drug", the data would reflect that the patient fell only 5 points. Essentially, that data was treated as "missing". Let's say the patient dropped 30 points before the rescue protocol...same result, the data would show only a drop of 5 points. 25 point decline...5 points. 35 point decline...5 points. And so forth.
Bottom-line result: The patients that dropped the most points, showing decline while not on the drug, is hidden. While decline was significant, it only shows up as a minor drop. Because the data on the rescue protocol is buried, it looks as if there is no difference between the sham and the "on drug" group. NPC1 progresses in a stair-up fashion. That is, you progress rapidly, then stabilize for a while (sometimes years), the you progress rapidly again. The trial was not design to capture this.
This system put rare disease drug developers in a very difficult situation. They can chose not to have a rescue protocol, so that their data is true. However, they would have to watch trial participates sustain additional disease progression or worse, death. "Sorry your kid can't talk any more, but we got good data!". The other option, and more moral option, is to include a rescue protocol but risk that your data will not be perfect.
If VTS270 doesn't work, neither does CTDH's drug. If you believe this, you should sell both stocks immediately.
My child is on VTS270. The drug works. CTDH's drug works.
If CTDH was smart, they stop focusing on very difficult to prove claims of neurological benefit from IV cyclo. Instead, they should focus on the benefit to the liver and other organs. Make neurological benefit a secondary endpoint. They can prove liver benefit without a sham group. If they want to prove neurological benefit, they will be in the same boat as MNK.