Member Info for mallettp

I agree with your sentiments - small pharma is fraught with risk and most are money pits that never deliver. To turn EBITDA positive is an achievement in itself.

My reading of the FDA draft guidance is that the voucher "can only be issued after the date approval of the rare pediatric disease drug".

Possibly, it's starting to dawn that if the results of the Ease trial are "just good enough" for the FDA, as was the case for the EMA in the partial thickness wounds trial of Episalvan, then the prospects for a $100M dollar windfall improve considerably. If Joe Wiley's strategy pays off, it may even have been worth the extraordinarily long wait for all those who have been in since the Fastnet days.

Indeed, it would be very helpful to have an unbiased comparison of the two approaches. According to Clinical Trials, the JCyte 2b closed recruitment a while ago and results were due this summer although this has now been moved back by a year (according to the change records on the website). I suspect whatever they have must be effective in order to justify a $50M upfront payment.

I emailed Rene asking what competative advantage they believe they have and how the procedures differ. If JCyte's is one that can be performed in a non-specialist setting then possibly this is what makes it attractive. I'd also be interested to know how market exclusivity works in the case of cell therapies?

This gives some idea of the potential value of Rene's RP program

Do you mind sharing where you saw that Amryt is entering stage 2 trials for Radiation dermatitis for AP101?

Thanks

Ultimately it will all boil down to the results of the phase Ii trial for Cogane. If you trawl the PD literature on the Net, you will find a study (circa 2003) in which GDNF was delivered directly into the brain using a pump based system. The sample size wasn't large but the results were statistically significant. So it seems that GDNF does have neurorestorative properties when delivered into the human brain in certain circumstances. The paper is published here: http://www.psychiatry.wustl.edu/Resources/LiteratureList/2003/April/Gill.PDF The big question is whether Cogane stimulates GDNF production in humans in sufficient levels to have an effect. The previous trial in non-human primates (monkeys) showed good results - the research was sponsored by the Michael J Fox PD foundation. The results were impressive and Cogane delivered orally lead to very high levels of recovery in animals with drug induced PD symptoms. Here's a summary of the research: https://www.michaeljfox.org/foundation/grant-detail.php?grant_id=392 So the question is whether the same results will be seen in humans.. Given the fact that GDNF has been shown to be beneficial to humans with PD and the animal research which suggests Cogane does have a neurorestorative effect in the animal model, there's clearly some hope that the trial will be a success. Given that Phytoparm raised sufficient capital in the market to fund the trail, I imagine that a lot of Pharma investors must have agreed with their optimism.