We should not be disappointed, if we don't get top line data associated with the Nice conference.
IMO the results so far are astounding! Long ago we speculated the expectation would be for a slowing of DMD. What we are seeing is a stabilization and it appears in some cases an improvement. The big question is for how long.
You may be right, but it's not fair or realistic for the FDA to hold out on SRPT in order to answer the "for how long" question. The results are so impressive, that these kids deserve to get it as soon as it can be produced. Anything less, will cause needless loss of lives and function. People hoped for a miracle, and it came in the form of Eteplirsen and exon skipping with PMO's. Why there is so much resistance escapes me.
I am concerned that expectations have become lavish and may not be met by walk distance that merely indicates stabilization. Some personal accounts not withstanding, these patients will always have MD, just a milder form that may keeps them ambulant, not make athletes out of them. I do not expect the stock to surge unless there is clear evidence of improvement across the board. Options IV is down also so 144 wk data may not be as big a binary event as we think. Progress toward clinical trial initiation and NDA submission will have much greater effects on stock.
The question, at least for me, is that, if some boys actually increase the distance covered in 6 minutes which goes beyond stabilization, what does that mean at the myosome level? Are we really just converting to Beckers type MD or something else? Is Etep having a effect that no one thought possible? Watch Billy's twitter video from yesterday. His ability to push that fully loaded travel cart at the pace he achieved was outstanding in my eyes.
I agree with you to a point. A right-leaning bell curve on the performance of our 10 ambulants is probably to be expected. But at what point do "improvements" start to open eyes and raise eyebrows? Two boys? Three or four? If you offered every Duchenne family in the world a chance to morph into Beckers, they would jump at the deal. And I truly see etep as the agent for cutting such a deal. As for our pps, I think the NDA is already built into the street calculus. Risk remains, and it centers on our long-term efficacy and safety profile. And I think our 144 numbers will essentially answer those concerns, not only for etep and DMD but also for our platform down the pike. THAT's why I think new highs will be reached at 144, and if anyone shorts after that event, well, good luck with that.
Sentiment: Strong Buy