It's a excellent point about more severe cases having better responses, but much greater data is needed for broad insurance reimbursement, especially when the goal is to create a billion dollar drug.   The reason we allow such high margins is in part because we have high regulatory barriers.   FDA won't rush this to approval when it's not life threatening and still in dose escalation.  Efficacy based on prior surgeries is good, but a somewhat flawed barometer because it is scheduled.   

 

Path forward IMO is PGEN and FDA will agree on a pivotal trial that ...

- Establishes a pre-defined approval level of surgery reduction that they will likely meet

- Bunch of sites; because why not

- Possibly randomized between DL2 and DL3; safety data too good to rush DL2 to approval

- Is there a way for this be Rare Pediatric Disease voucher eligible

  

This appears to be an approvable product.  Just not short term.  Hence the raise.