Who knows may be the drug works may be it doesn't but it certainly helped him even if it were the placebo effect.
He had the most dramatic response from the nurown treatment
The market does not believe in the drug, so didn't the executives who sold their stock in December. Like the neurologist said, the easiest way to lose your reputation as a neurologist is to try to find a cure for ALS.
I must say these guys scammed everyone to believing that their drug was effective and produced more of the protein. I personally was mislead to believe that with all of the hoopla the prior boss was touting this was a much much more respectable number. How do you even discern the difference of this kind in so few patients and claim that your drug is effective is beyond me. Another biotech BS story.
He hates BCLI for doing this presentation and thinks it is a stock promotion stunt and he thinks his friend Tony Fiorino is no longer assoiated with BCLI, but I see he presented for BCLI in October of last year with video available online in Youtube. There has been no news or sec filing that Tony is not with this outfit. it would be interesting who presents on Monday and it would be interesting to see the JAMA article. Everyone on twitter is speculating that this is old data.
Hmmm! As if we do business in China...
Seven patients (Table 1) were included (ages 1 to 20 years old). All patients had been treated with low-dose FFA (5 to 15 mg daily) with a mean treatment duration of 22 months (SD 20.45 months). During the follow-up period, all the echocardiographic examinations remained normal without any signs of valvulopathy, pulmonary hypertension, or other abnormalities (Table 2). Statistical analysis showed no significant changes related to the cumulative dose or treatment duration with FFA for all the echocardiographic parameters.
In this prospective pilot study, we did not see any clinical cardiovascular side effects or echocardiographic abnormalities in DS patients treated with FFA. By limiting the daily dose to a maximum of 20 mg per day, FFA seems to be a potentially safe anti-epileptic drug patients with this refractory epilepsy syndrome. Brabant Pharma and Zogenix, Inc. funded the study and its analysis. Medical writing assistance was provided by PharmaWrite (Princeton, NJ) and was funded by Zogenix Inc.
Seven patients have enrolled (5 male, 2 female) to date. Mean age at the start of FFA was 12.2 years (range, 1 to 20 years). All had a confirmed mutation in SCN1A. All patients were receiving at least 2 other antiepileptic drugs at study entry, including valproate (n=7), topiramate (n=6), clobazam (n=3), stiripentol (n=2), ethyl loflazepate (n=2), bromide (n=1) and levetiracetam (n=1). The median starting FFA dose was 10 mg per day (range, 5 to 10 mg). During the 3-month baseline observation prior to starting FFA, the median frequency of tonic/clonic (TC) seizures was 3.0/month (range 0.4 to 39.7). All patients demonstrated a decrease in the frequency of TC seizures during FFA treatment. At the 6-month evaluation, the median frequency of TC seizures was 1.2/month, representing a decline of 73%. Over the entire observation period (median 0.9 year; range, 0.22 to 3.91 years) the median frequency of TC seizures was 0.9/month representing an 84% reduction compared to the 3-month baseline period (Figure). Most common treatment-related adverse events were mild to moderate somnolence (n=6) and anorexia (n=4). One patient, who was hospitalized 11 times for seizures during the pre-FFA period, experienced 4 separate episodes of status epilepticus with fever due to respiratory infection.
Years ago I had a friend, who worked for Burroughs and after the merger with Sperry, another great company and the subsequent name change and downfall to unisys, was so frustrated that he used to call any company that ended with sys substitute with sheet, i.e unisheet, infosheet, linksheet etc and he was never wrong, every company ending with sys has ended with a disaster and mosheet has been no exception.
Adam Feuerstein just posted a study on CBD, on his twitter, that shows it is pretty ineffective and the effect if any does not last. I recall 60 minutes made a similar sensational program on resveretrol that proved to be a complete and utter failure.
reverse split and more dilution is coming. lets face it minor movements on the scale is not going to impress anyone in an open label phase one study in which 20% of the subjects did not even feel the placebo effect.
Without the company announcing liquidation accumulation of shares at cheap would not have been possible. Were the management coerced in announcing liquidation so the accumulation for hostile take over could take place? Shareholders lost big time on this deal, because it is the end of the year tax loss season and a lot of share holders sold when they saw the liquidation announcement. The management could have negotiated a better deal for the share holder if they had not announced the last quidation.