I agree with your overall point that the FDA is not going to rush approval of 2012.

 

I am expecting Precigen to have some clarity whether they are going to need to run a phase 3 trial in the next several months. By the end of April, Precigen should have the 6 month data from the first 11 out of 25 phase 2 patients. I have a difficult time believing that the FDA is going to say at that time, "We are ok with phase 2 being registrational based on the data from 12 phase 1 patients plus 11 phase 2 patients at the 6 month mark". 

 

The last thing Helen wants to do it slow this train down. The phase 1 trial took approx 22 months from enrollment to full 12 month patient data. Phase two with 25 patients is looking like approx 27 months. If I was Precigen I would get a roll over phase 3 approval from the FDA on another 23 patients which will bring the N to a total of 60. I agree with having multiple sites in addition to the NIH so that enrollment can be complete by end of 2023. Imo, Precigen is not going to file for a BLA on 2012 until the data is complete on phase 2 which should occur end of q2 '24.  If the data is spectacular and confirms the phase 1b data, Precigen can always make the decision with guidance from the FDA to file a BLA based upon phase 2 and potentially include any phase 3 at a later time. If the FDA expresses the desire to see more data after seeing the phase 2 results, Precigen will already be well into data collection on the phase 3 patients.

 

Where I am going to disagree is your thought that a pivotal trial will be possibly randomized with a higher dose level that has zero safety data.