"... Trial presentation in June, that may be when we get answers" - wrote that feb., Mrns not getting data this month. The results have been in hand (Kooy and co.) since Jan.
Witholding results from Mrns for the past 6 months,--- only one way for 'them' to make it right. An announcement that the FDA has agreed to breakthrough therapy, accelerated approval, an open label extension trial and compassionate use for patients. I want it all.
- A contrarian is a person who takes up a contrary position, especially a position that is opposed to that of the majority, regardless of how unpopular it may be ..."
1) Biomarkers are use to develop therapies. If ganax trended towards efficacy, there would be no need for the biomarker project.
2) No mention of an open label extension study. In double blind trials, when there is an obvious improvement, those patients are then given the choice to receive the drug via 'open label'.
3) "Allopregnanolone is a natural substance, ganax is a derivative of Allo. It's possible they detected an improvement using ganax, but would like to see if Allo can be more effective. I can't get past moving on to Allo if ganax was showing efficacy." my post - 2/15/16
Dr. Hagerman made a professional decision. She could not walk away from the gabaA agonists without at least trying both compounds.
I believe ganax will show favorable activity in FX but a primary endpoint miss in June will be a sell down/buying opportunity.
(ps: I've never wanted to be more wrong)
"It may take a week, a month, a year (seriously) but 'the gap always fills' has a truth to it. Close of 5.62, open 6.05... wow. My guess, within a month" 4/20 - - One week ??? jeeze, that was quick. Next up, double digits. Glta.
Held for two years, sold last Nov. ($3.40), largest bio loss all time : ( . Thanks to an abstract posted by "guti" last Jan, my doubts about the science were confirmed.
" . . .the therapeutic effect of IL-12 produced within the tumor microenvironment could NOT BE mimicked with high doses of EXOGENOUSLY provided IL-12 . ... . "
Sage over 1B market cap. ($40/sh.) Imo, Mrns one of the most undervalued stock in bios. We could easily hold a 200M market cap. ($10) - Quick backfill followed by double digits (?) I'll take it.
It may take a week, a month, a year (seriously) but 'the gap,always fills' has a truth to it. Close of 5.62, open 6.05... wow. My guess, within a month : (
ranger, along the same line of thinking ... Dr. Hagerman may have seen an obvious improvement and wanted to bring the trial to an end. Belgium was needed to quickly move into 'fully enrolled' status. ..... ps- not skeptical, Hopeful. Gltu
'turning lemons into lemonade' : )
Coincidentally, AF wrote an article just this morning about the insignificance of announcing an FDA orphan drug designation. Good read. Looking at the Mrns pr's of last year, they held the high road, no announcement of FDA decision. (bio,I missed the 15' also)
Institutional ownership a paltry 14%. Today's science is not ready to develop a 'vaccine' against cancerous cells. Sitting on a bio longshot, Gltu.
`A spade is a spade.
" .. in some settings ganaxolone appeared inferior to SAGE-547 (and both drugs were inferior to SAGE-217)." - That would be the oral neurosteriod.
" ... Sage is significantly de-risked based on its early clinical data in SRSE and a high likelihood of success ........ Marinus, on the other hand, is a high risk bet with a lower likelihood of success but the company has a lot of catalysts in the coming year . .... "
Here for the potential high percentage return, the catalyst being low price/ high risk.
Sage has impressive clinical results, and with a 52 week high of $90/$3B market cap wall street took notice. Currently with three shots on goal (pipeline) and Mrns having only one, we are definitely in a high risk investment. Also the PPD results gave them added (speculative) value.
I do agree. 20% for them and 2% for us ... I'll type in symbols save yahoo the trouble #@* !!!
Acad completed a phase III, the FDA let them bypass a Ph III confirmatory.
' . .. . Its lead product candidate, NUPLAZID, has completed the Phase III pivotal trials for the treatment of Parkinsons disease psychosis . . . "
Getting an FDA nod with only 60 patients in a Ph II is not likely, unless the data is stellar. With nothing specific to treat FX that may open the door to your call, accelerated approval.
Gale ? uh oh. jr, super high risk, Imo... if I were sitting on a profit no way I hold thru news. Not telling you to sell... but... I expect similar to Cldx. - -- - Order in; 5.43, the usual 100.
". ...generation of TIL's in a subset of patients." When a co. relies on subgroup data, the chances of success are slim to none. Gltu
Not done buying, hoping to see a temporary pullback- that being said, with about 10k shares bought yesterday and 14k today, is this MM price manipulation (?) because it sure as hell isn't demand for shares (ha). Sage did 540k shares today at $32, that's $$$ !
now, another subject, your bullish call proved out. Cool.
No question, listening to Dr. Tsai and then reading the abstracts ... we have a shot. Being a binary event, double blind, placebo controlled; the upside/downside will be significant. Fingers crossed on this one.
Looking to add 500 shrs , trying something a little different ... 100 share lots. The volume yesterday and today, 32k and now 28k today. Pitiful, lol !!
bio, . . techs, . .... you guys are sharing the MVP award for 1st Q.
From the conference video- "up to 20 to 30% of Fragile X patients also exhibit seizures .... "
From the contract - "Ganaxolone has shown its anticonvulsant effects in diverse animal models . .. . . . . However, this is the first time that the efficacy of ganaxolone in the treatment of audiogenic seizures in the fragile x syndrome is demonstrated."