I expect a great day today... late yesterday someone/people really wanted in....we should have continuation today. (News tomorrow?)
VA-- a lot of NIH trials are run at Walter Reed. I think you'll find it impossible to find the stats you're looking for
The UC Davis Mind institute is not government owned, nor are the researchers government employees. They just use some government grant money. Big difference. I used to work in a lab at Harvard, where government grants were often obtained for trials. It had no hearing on results. It would be different if this were at Walter Reed, run by government employees in a govt lab. Just my opinion. But do I do agree with your comment on a private company's additional goal of FDA approval vs science only for science's sake.
Even if FX results are negative and the stock price takes a hit, it'll recover within weeks anyway. Some are waiting for FX results to be behind us before buying.
Depression is a huge market. If SAGE's trial is encouraging, maybe Marinus will also go down that route. And the planned outside Ganax trial for MDD will get more attention.
Hey Andre... Nice to see you here again! I forgot about SAGE's trial... Thanks
Another catalyst and reason to hold through FX results. It's pretty much de-risked. Failure of FX trial is already pretty much priced in.. (and, obviously, I'm one of the only people in the world who's optimistic about FX results lol)
To be clear, the odds are against positive FX results, as something like 80% of all drug trials fail. So I'm not predicting success. All one can do as look at the company's current market cap, the risk/reward, and chances of success for a given trial--based on the science and the trial design--and then decide if it's worth risking your money. Bio says no, I say yes. The answer is different for everyone.
Hey Bio, regarding the FX trial, I myself am not concerned too much that it's an independent trial. The best, most experienced researchers in the field-- such as Dr Hagerman--are running the trial. And the Belgian half is under the auspices of Dr. Kooy, who discovered the association of GABA dysfunction with Fragile X. If anything, that would create a positive bias. Nobody wants positive trial results to prove his theory more than him.
Ganaxolone has been uniquely shown to have no developed tolerance to the drug, in many pre-clinical and animal studies (both for fragile x and epilepsy). In fact, there is no such tolerance attributed to allopregnanolone. So while the glutamate antagonists failed in this regard, it is possible that Ganax could become more effective with time. And then there is Kooy's argument, that Fragile X disease is primarily a disease of lack of neuron inhibition, as opposed to over-excitation, so targeting GABA instead of glutamate may be the correct approach.
One other note of interest... his lab discovered that Ganax does not caused developed tolerance, but those taking Ganax do develop cross-tolerance to the benzodiazepines (Valium etc). This could have interesting implications, meaning that eventually many of the indications for Benzo drugs (like epilepsy) could be replaced by Ganax, which would treat the conditions similarly but without developed tolerance, addiction, and/or dependence on having to use increasingly higher dosages.
Tech-- in a previous post I said I am here for the science, so I will talk science. Fragile X has a been a tough nut to crack. But there are many well-studied aspects of the disease. The exact genetic mutation is known. And it well-documented that a hallmark of this disease is overstimulation of neurons in the brain (similar to other diseases, such as many epilepies, Retts syndrome, autism, tics, savant syndrome, etc). The major stimulators brain receptor is glutamate, whereas the GABA receptor is main inhibitory one. Since Fragile X syndrome was mostly viewed as disease of over-stimulation (for example, with epilespy as a common symptom), the big focus was on glutamate receptor blockers (antagonists). There were two big pharma drug trials based on this hypothesis (Roche and someone else, I forgot who, maybe Novartis). Unfortunately, both failed. In fact, the drug arms performed worse than the placebo arm. It is widely assumed that a big part of the failure was due to developed tolerance to the drug which became apparent as the trial progressed, which could have actually worsened the symptoms of the disease toward trial's end. So much for glutamate antagonists...
So now here we are, on he other side of the equation. Now, instead of blocking the stimulatory receptor (glutamate), we are enhancing the inhibitory GABA receptor, with Ganaxolone (a GABA agonist). This is a very complex receptor, with many subunits (including various forms of GABAa, GABAb, and even benzodiazepine/opiod receptor). The neurosteroids (such as Ganax) are unique in their selectivity for GABAa receptor-- most important in FX syndrome--and for both intra- and extra- synaptic effect. Perhaps strong enough to overcome the lowered amount of GABA receptors present...
Ok, you ask, so why could Ganax work, when the glutamate blockers did not? The answer perhaps lies in my next post, to be continued...
Bio-- one for factor to consider... Point72 dumped a huge number of their biotech holdings in the first quarter ----look at holdingschannel-- including big and small (allergan and Idera, for example). A major washout of their biotech positions. (They nailed CPXX though.) I honestly don't think they gave Marinus, a tiny position, more than 10 minutes of thought.
Thanks for your opinion. I agree that people shouldn't invest more than they can afford!
In my case, I will remain fully vested here because I think that there's a real possibility that Ganax will work in Fragile X, and then the other indications, solely based on my review of the science. I understand your logic though about "following the money", but I can't go solely by that. And both BB and Cohen have lost big on trial failures recently (as well as FMR). But they certainly have more winners than losers.
I forgot to mention that the major Fragile X conference being held in 2 weeks (FRAXA Gordon Research Conference), lists ANAVEX as one of their sponsors. Very interesting.
In addition to Trofenetide and 2-73, you have to add Ganaxolone (from MRNS) and a new drug being developed by SAGE, both GABA receptor agonists. MRNS' trial results for Fragile X and due any day now. Look out for it. There is significant overlap in Rett's and Fragile X syndrome, both in etiology and clinical presentation. And btw Trofenetide had positive phase 2 results for Fragile X... Very interesting. Personally, I think that in the end there may be a cocktail of all or some of the above, which includes 2-73...
Hey, Daw, I realize that a lot can go wrong in blinded trials, but I (as the eternal optimist) expect positve Fragile X results. And accelerated approval soon afterwards. It may actually completely de-risk holding through adult focal results. Wouldn't that be something.
As frustrating as it is to watch them walk down the price on 100 share lots, it is actually bullish. If insiders wanted to dump, they'd walk it up instead, before selling. Again, it's all MM's tossing a few shares around.