Primary Endpoint - The Clinical Global Impression Improvement scale (CGI-I) requires the clinician to rate how much the patient's illness has improved or worsened relative to a baseline measurement.
Secondary Endpoint - Swanson, Nolan, and Pelham-IV Questionnaire (SNAP-IV)
"the rating scales .... may indicate that your child has a behavioral difficulty that warrants a visit to a clinician. . .Rating scales can serve as one component of an evaluation. They can help determine if your child is making progress if your child begins medication or behavioral therapy."
I'm sure ! lol . 1) there will be a p.r. from Mrns containing topline data before conf. 2) it is a gap and it will fill. "I expect that I'll be ..." - Good post, speaks to all Mrns investors.
previous close $5.01. Open $5.07... days low $5.07 hmm - here we go again ranger.. prolly fills today Lol !!!! .. $6+ before news (??) jr, I noticed you took little break, your TA always appreciated. Tia
uh oh,,, just saw yesterday's close and today's open. Gap. (shh !) Hopefully a quick backfill, allowing the uptrend to continue. . . .. .
"The low is in" hahaha, you're killing me ranger , lol ! we'll see : ) , can't do another bet, I missed you when you did the "5 days no posting" HA - it's all good - . .. got to run, late: work
The difficulty with trial results is that sciencewise we could have positive results but the market might only be interested in the overall drug efficacy for FX. Putting extra cash in my scottrade acc. this week, if we see new lows, buying.
The San Antonio conference: Ganax/Cortical function in fragile X , this could be a breakthrough leading to new market opportunities similar to Sage with PPD.
Many bio investors (funds too?) were waiting on the Trvn (expected) fail to buy at the lows, powering a quick price recovery, maybe we see the same.
There is one minor detail (lol) that can't be overlooked, Trvn has a non-opiod drug (painkiller) that hit an FDA approvable P value in a Ph III trial.
" .... For many trials, approximately 30% respond well, but this may not be adequate to demonstrate overall efficacy, which prohibits FDA approval for marketing." From the 11 PAGE pdf. - - - - Safe does not decide approval. Could probably find 10 drugs approved that say in fine print "take it, but you might not wake up in the morning" - too many posters believe 'safe' is good enough, not true.
Overall efficacy. The only criteria wall street looks for. FX trial result is binary event. A ph II double blind placebo controlled trial with the primary endpoint in play.
" Hagerman has started a new Allo trial in FXTAS. Not sure why she does that if she thinks Ganax washed out in FXS." techs, you're way smarter than that. We don't know what she thinks.
"Allo 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."
All this speculation (me included), because Kooy and Co. decided to embargo results for six long months. Bull !!... getting angry doesn't help.. a part of me says it HAS to be good news.
imo, I don't care for the list of presentations for this trial, it makes me think 'data mining'.
and finally, I have no clue on results at all, seriously. GLTA
the (grumpy) weasl..- cause I continue to fall asleep in the fourth, missing game endings.
" ... Effect of Ganaxolone on Cortical Function in Fragile X . .. "
- Cortical Areas: The cortex is commonly described as comprising three parts: sensory, motor, and association areas.
- Individuals with FXS may present anywhere on a continuum from learning disabilities in the context of a normal intelligence quotient (IQ) to severe intellectual disability, .....
- Of those with fragile X syndrome, prevalence of concurrent autism spectrum disorder (ASD) has been estimated to be between 15 and 60%, .. ....
- A child with autism spectrum disorder may have problems in three different areas -- socializing, communicating, and behavior.
c'mon Mrns, start work on your NDA (new drug application)
Day only order last night. 100 shrs @ 4.52 Today's low 4.53. Intentional on the mm's part, called them 'sneaky' - paybacks a #$%$ : )
sorry, this txt message style of writing not my strong point : (
Several times over the years I've seen a similar pattern when nearing data readouts. (if tutes know of bad news) The sneaky mm's let the tutes out causing a price drop then on low volume price starts going up, up. Retail is fooled into buying as share price looks to be getting away before official announcement.
Of course only on news leak (upside if good data)
Most times the market (tutes) and retail is unaware of results till a co. pr.
" Somebody always knows ahead of time." From another board:
The key, as a retail long, is to remember . . . . (a PR regarding sharing the data at a major medical conference)
followed by days of decline when the stock logically should have risen. The type of decline is also important as evidenced by the large volume spikes on sharp declines (that's Trevena's financiers unloading) followed by a rising share price as optimistic retailers buy seemingly bargain priced shares on lower volume.
If news gets out before the topline readout we'll see large upside in volume, ...... maybe price too (?) . Gltu
- The understanding of the neurobiology of FXS has led to the development of targeted treatments for the core behavioral features of FXS, which include social deficits, inattention, and anxiety.
- While two medications have been developed specifically for GABAergic modulation – arbaclofen (Seaside Therapeutics, Cambridge, MA, USA) and ganaxolone (Marinus Pharmaceuticals, Inc., New Haven, CT, USA) this paper will also address GABAergic medications such as riluzole, gaboxadol, .. . . . ..
- GABAA receptors allow the flow of chloride ions across the membrane, which hyperpolarizes the neuron’s postsynaptic membrane and minimizes the effect of any coincident synaptic input. (huh ?? lol !)
-GABAergic agonists can be used for specific phenotypes including anxiety, autistic behavior, epilepsy, and cognitive impairment. GABA agonists have shown very limited efficacy in preliminary studies for these symptoms, but they have been well tolerated.
- Ganaxolone should increase and normalize GABAAmediated signaling – by boosting the signaling capacity of existing receptors – and improve behavior, particularly anxiety and attention.
Excerpts from an 11 page PDF.
* Link courtesy of: prospern23, FRX data.
" . . . and I forgot to mention female pediatric infantile seizures. For that I think Ganax is a slam dunk. " 5/5/16.
my bad, you were referencing a decrease in seizures within the trial. My understanding of the abstract , , - we are in a high risk trial similar to the 'vaccines'.
" ...further studies are required to determine whether PCDH19 plays a role in the regulation of GABAA receptors and whether these interactions may contribute to the pathophysiology of PCDH19-
"...... Interestingly, PCDH19 has been shown to directly interact with at least one GABAA receptor subunit, alpha 1."
Does ganax have to upregulate alpha 1 ? .. limited knowledge of CNS drugs and how they work. Techs ??
"... 43 gender-biased genes that are associated with PCDH19-FE. To gain some insight into the pathways associated with the gene alterations in PCDH19-FE, the authors examined an annotated list of these genes using the Database for Annotation, Visualization and Integrated Discovery (DAVID) pathway and Ingenuity Pathway Analysis (IPA). These methods identified cell adhesion and integrin-related signaling pathways, consistent with the role of protocadherin 19 as a cell adhesion molecule. However, none of the pathways identified included gender-biased genes."
43 genes associated with PCDH19-FE. Someone said this trial is a slam dunk, after reading this abstract .... I don't know about that.
- "Form 13F is required to be filed within 45 days of the end of a calendar quarter." SEC regulation for the 'tutes'. VA your posts make for an excellent read, the OT thread: nice.
Read SEC form 8-K, ... publicly owned companies have four days to report "information that shareholders should know about."
"you don't want to be the first" - me, 2/4/16
Several yrs. ago a poster explained the science of neuvax, correctly calling the failed trial. The upcoming ph III will be no different : (
Athx also missed, last year - "April 17 (Reuters) - Athersys Inc's stem-cell therapy, its only product to reach human trials, failed a mid-stage study testing it as a treatment for a type of stroke, wiping off more than 40 percent of the company's market value.. . . . . . . "
Current institutional ownership of what ??? 8% !!
Just as with Cldx ( " long odds for Cldx.... the potential for a futility readout is very real..") me, 2/17/16) - .. .. . .. .. . . . . . . . .
I'm not suggesting sell but ...... wow, you own some bigtme bio longshots. GL: I mean that sincerely.
the (helpful?) weasl.
Melbourne, Australia, 7 December 2015: Neuren Pharmaceuticals (ASX: NEU) today announced top-line results from its Phase 2 clinical trial in Fragile X syndrome. The trial has successfully established proof of
concept and provides a strong rationale for Neuren to move forward with developing trofinetide for Fragile X syndrome.
• Positive top-line results provide a strong rationale to move forward with development of trofinetide for Fragile X syndrome • Primary endpoint achieved - both dose levels were well tolerated and no safety concerns were identified
Randi Hagerman MD, Medical Director of the University of California Davis MIND Institute and Director of the Fragile X Research and Treatment Center, commented: “Trofinetide has a unique mechanism of action very different from any other molecule that has been tested before in Fragile X syndrome. Its derivation from a naturally occurring neurotrophic factor makes it a promising candidate to treat a wide range of the core symptoms of Fragile X syndrome and potentially other neurological disorders. The results of this trial and the clinical improvements that investigators observed are an exciting first step”.
* - "you don't want to be the first" (ominous) - just as in 'the gap fills - both high percentages.
* - the numerous vaccine fails come to mind, AML trials another . . .. .. .
With trofinetide meeting primary in FX, 'you don't want to be the first' is no longer in play : )
Neuren Pharm: NCT01894958