RE:RE:RE:RE:Q about BTD Enriquesuave wrote:
"Nice post Wildbird. I believe that the company has stated that after 25 patients they would file for BTD, and recently Vera commented "soon" . So clearly they can now as they have perhaps received data from patients 24 and 25 who have hit at least 90 days. We have strong efficacy signals and also also pointing towards strong durable efficacy results IMO. That may be all that the FDA wants to see for this novel One and Done treatment option. I'm hoping they will submit application before year end so we get BTD within 60 days to coincide with more data on the pending 7 patients. All IMo time will tell."
For general followers of this stock, I believe the 25 patient number was reported per FDA guidance, not a number the company recommended. In general, a Ph 2 gauges efficacy while a Ph 3 confirms it. Because of this particular indication (that has a highly unmet need for better treatment options & currently a substandard level of care or SOC), our Ph 2 is essentially acting as a Ph 3. A request for BTD generally takes place before the initiation of the phase (or early on in the phase) that will serve to demonstrate efficacy at any point in time...not durability. The durability outcome measure/endpoint (or duration of CR at 360 days post initial CR) is meant more for determining final drug approval.
As enrique mentioned, I also believe we are very near having enough (may currently have) data to submit an app for a BTD. Hopefully, we have a lot more reason to cheer in Q1...all imo.