Contd, i take that back , fda accepted bmrn nda on same day in late june that srpt had filed their nda. So fda must have seen some of wms data, but probably not all of it. What data from wms was completed already in late june when nda was filed?
remind me, the fda accepted bmrn's nda before they had seen any of the new key etep data? Right? Whats your opinion, if they had already seen the etep data presented at wms before bmrn filed drisa nda, would the fda have rejected bmrn nda??
I think bmrn may have cooked drisas goose by not starting confirmatory trial already (like srpt has). How can fda give conditional approval if new data is not being produced to continually evaluate and potentially cause conditional to be rescinded? Isnt that the whole point of conditional? How can conditional be given to srpt on different terms than bmrn? Makes zero sense
He indicated Etep will be approved, hopefully investors look beyond the headline and read the actual article
(S)He makes 9 excellent points. The last 3 are kind of silly but if the average (biotech) investor could understand the first 9 pts fully imo SRPT pps would be much higher.......
"with the other anti-sense oligonucleotides", were they referring to only Etep or were they referring to Etep and other exons ? That wording confuses me
I think its funny the article came out after the market close, ie nobody hi profile is pumping this stock during trading hrs. I guess the pros are still accumulating, or they are sticking to their shorts
To me the Zacks article that just came out epitomizes why srpt shares are so cheap (risk adjusted): if a lay person read that article they might infer that drisa has a better shot at approval over etep and thus has better data. The average analyst gives NO actual relative analysis of the two clinical data sets. Even AF has come up short lately on this this. If that is the case, how could the average investor value srpt properly.....
I think in most peoples opinion almost the entire market cap of SRPT is based on DMD- ie Etep (vast majority) and then the exon analogs related to Etep. Imo progress on any new disease could add considerable value to SRPT over time
Contd esp when you know there has been tox w drisa and chance for rescind is very real
Well they accepted their nda so i wouldnt go too far. I have assumed in the past that drisa will be approved (simply because the fda has been approving most ndas lately) but the more i look at all of srpts data and all of prosensas data, i am beginning to think drisa should get CRL.....i stll can not understand how you give conditional approval if there are no trials ongoing in a timely manner in order to scrutinize that data for possible rescind of conditional approval. Makes ZERO sense
what will be interesting from this collaboration is if all the diseases Wilton targets are gene on diseases, like DMD. SRPT claims they can either turn a gene on (ie like w Etep, hence dystrophin is produced) or turn a gene off (ie for antivirals) with their PMOs. Will be interesting to see if Wilton goes after some gene off diseases as opposed to all gene on diseases.....
Wilton is no dummy, obviously he knew DMD and lack of dystrophin production was a tough nut to crack and he saw that PMOs were the best class of compounds to do the job. And its safe to say he saw them as far superior than Prosensas oligos w a different backbone due to half life, bioavailabilty, tox etc etc. what works for dystrophin production can obviously be applied to many other protein targets. Their platform is strong, just build out the PMO backbone and vary the base (targeting the rna) depending on the disease. My guess is Wilton will even tell them what base sequences he wants to see and then SRPT will make it in automated fashion fairly quickly, with all related analogs. If it works for dystrophin/Dmd it will work with other diseases imo. Time will tell