Adult focal is likely to be positive--like phase 2-- but these are difficult patients to treat who have tried and often failed with other regimens. So even a 20-40% response rate would be good.
Fragile X, on the other hand, is regarded as a much higher risk trial, but I don't think so. There are numerous parameters and endpoints which, if enough of them are met, will lead to a phase 3 or early approval. And there is a lot of scientific literature supporting a potentially positive outcome in not just Fragile X, but perhaps the whole spectrum of autism. The science behind this drug in this disorder is compelling (a bullseye actually, if you research it), more so than practically any other experimental drug that I've ever studied. (And it's a plus that this isn't a cancer drug-- my yield on betting on cancer drugs this far is 0%!)
Oh, and I forgot to mention female pediatric infantile seizures. For that I think Ganax is a slam dunk. GABA receptor dysfunction is a hallmark of that condition.
Definitely took it in jest!!! No prob VA
We are fighting an uphill battle, trying to swim upstream against the down current in biotech land! Fortunately, MRNS is so undervalued that there is decent upside potential if trial results are good. Eventually, it should be comparable to SAGE. There are very few stocks that I see with that potential. Gale and AVXL come to mind, if trial results are good.
Thanks jr...I'm not going to trade by T.A. because we might be only days away from a fundamental binary event, Fragile X results, through which I've decided to hold my entire position.
Plus, with the few shares available it's so easy for MM's to scare retailers by dropping it below key support levels on the charts They know the charts and the game too.
Well, after fragile x and then adult focal results we'll either be in double digits or be down to $2 or less. We aren't stayin from $4-$7 much longer!!'
Good decision, Prospern.
These seem like violent moves but in reality are not. With only 19.5 mil outstanding shares, our market cap dropped by only $6-7 million today. In fact, all of these moves between $4-$7 per share are peanuts in regards to the market cap, which could very soon be in the $200-$500 mil range or more if we get good results.
It seems like a lot of small cap promising biotechs have collapsed down to the $100 mil market cap range ($90-$110 mil). Perhaps IDRA is headed there. It's like they're all being slammed back there in a holding pattern. I still think there will be individual winners if they have good trial results (like CPXX).
I don't know what to make of this collapse. I'm not sure you can blame it all on sector-wide dumping alone. I think some major institution/s are shedding their shares.
For that matter, Dr. Hagerman's own trial for Minocycline showed some benefit too for Fragile X. Another positive: she has experience getting positive trial results, without screwing up the control arm with investigator bias/enthusiasm.
Even some late stage pipeline biotechs have been languishing. Look at ARRY. Makes no sense.
The only thing that seems to make sense to a good stock price is the potential for buyout. IDRS is too early and unproven. ARRY has too many partnerships and moving parts. I guess investors are now only rewarding biotechs that could get bought out. Like MDVN. Now CPXX. Even SAGE, with its high market cap, has been mentioned as a buyout candidate.
True, since Marinus has to supply the drug.
Boy, the MM's are playing this stock like a yo-yo on few shares.
Hey VA... Thanks for your input. Trying to back the investigators could make sense if they were MRNS' own scientists running the trial for MRNS. Since this was a totally independent trial with outside scientists, I don't think it would be likely here.
I still can't seem to get out of my mind the fact that, for the first time that I can recall, the company talked about FX results first and the results are "clinician and parental-validated". It's the parental validated statement that I'm dwelling on. They didn't have to say that. No need to if results are poor. Why say that unless you're laying the groundwork for great things to come?
A hint to retail investors? Trail of breadcrumbs perhaps?
A sobering post. But I don't think it's dire for every biotech stock. There is still a reward for successful trial results. Look at CPXX. And if GALE has positive results (which I'm skeptical of), it could go to the moon. Maybe the way to invest in the current environment is to dollar cost average into your favorite beaten up biotech stocks (such as IDRA) by buying a certain dollar amount every month, instead of trying to time the exact bottom, which is almost impossible to do.
Thanks Tau. Great post. I hope you have a decent position in MRNS.
Tomorrow will be an interesting day to say the least! I wonder if today was the beginning of the shorts exiting like rats running off a sinking ship.