Characterizing Disscussions with Regulators Around Clinical Data

Pharmaceutical Executive: How would you characterize the discussions with regulators around clinical data?
Helen Sabzevari: So, the journey for approval of papzimeos. Really, it started from the when we first submit our indies for a phase one. And you can imagine this was done in 2021, during pandemic, for that matter, and then followed by our phase two, which was done in 2022 it started in 2022 and in throughout this, we have had discussions with the FDA, and basically in the summer of 2023 we received the accelerated path by December of 2024. We submitted our rolling BLA and basically in August and two weeks in advance of our per due for date, we received a full approval. Originally, the, as you mentioned, the accelerated path actually goes hand in hand with a conditional approval, which requires a confirmatory trial. However, after we continue to submit our data, both based on safety, efficacy and especially durability of response and the ease of administration in the discussions the FDA, it's decided that this will be granted a full approval, which we are very thankful to the FDA, and we are excited about that, and based on the efficacy of this data, If you look at the clinical data for papzimeos, the Phase I 12, patients in a phase one, they showed 50% response rate. In a phase II 23, patients, they showed 52% response rate. You cannot come closer in the clinical data than that. And not only this science was and this was associated with immunological responses, which is the mechanism of action of these drugs, very well correlated, as well as a very favorable safety and the durability of response that which I mentioned, we have been following these patients now. The medium has been two years, and some have passed the three years, and they are still in a full response. And that, I think, collectively, in the discussions that we had with the FDA led to the decision of the FDA to move for the full approval, as opposed to a conditional approval, which eliminates the requirement for a confirmatory trial, as you are aware of.