Before granting a Breakthrough designation, and considering that one of the endpoints of the Ph. 2 is a "durable response" (@450 days), the FDA clearly needs some 450-days data in order to be on a solid ground to justify this Breakthrough designation. No doubt.
We know we have some 450-days data. But not that many durable responses so far, given the underdosed patients (12).
On the other hand, we had, until Nov. 29, 70% (7 out of 10) patients that had consecutive 3-months cystoscopies that showed CR (complete response @90-days), since their initial treatment.
If we continued, since Nov. 29, this winning sequence, and that
"... at an interim analysis of approximately 20 to 25 patients enrolled and successfully treated"
means something halfway through 450 days, then we might have support from the FDA, considering that "there's no standard of care for the situation of our Ph. 2b patients" as these patients are at the crossroad of "you do nothing and it migrates from NMIBC to MIBC" (and that means metastasis and death over time) and "you get your bladder removed" (and that involves a 6-7 hours operation that can leave you dead because of complications).
So what this "interim data" and "successfully treated" mean fir the FDA, that's the 5G$ question. I think it leaves latitude to the FDA, depending on the case she has in front of her.
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May 29, 2020
“In a 3Q2019 conference call with the FDA, it was discussed and agreed between the FDA and the Company, that Theralase would potentially be eligible for Fast Track Approval (“FTA”) post receipt of the FDA IND authorization, based on the clinical study data collected to date. It was further discussed and agreed that Theralase would potentially be eligible for Breakthrough Therapy Designation (“BTD”) and / or Accelerated Approval (“AA”), if Theralase can demonstrate clinically significant results (high safety profile and high efficacy response), similar to the safety and efficacy results observed in the Phase Ib NMIBC clinical study (high safety profile and 67% CR) at an interim analysis of approximately 20 to 25 patients enrolled and successfully treated.”