So here is combo of bad news
1. The feedback was received in late October - this means FSA met their deadline of 75 days to give comments. Co decided to hold that news till Good news related to Siemens came out and they had to give it in 10-q
2. Surrogate end point changed - this means they have to add some specific end point - this may add to duration of trial
“. In its comments, the FDA seemingly is departing from its earlier implied potential support of the use of progression to varices as a surrogate endpoint for accelerated approval. “
3. The protocol is being redesigned with help of experts - this means resubmission of new protocol - the 75 day clock starts again
4. I mis-read about new trial but the effect is the same. The new design will be conducted as 2 part trial
Part A - Adaptive portion with interim futility analysis allowing early termination of trial in case of not good interim results
Part B - this is being now called as real phase 3 study and will have parameters adapted based on results of part A. It will have more criteria, more inclusion, 2 year dosing for at least 500 patients.
If the part B or P3 shows good results, it could go for approval or need another P3 study.
4. Co expects to dose 1st patient in Q1-2020 but we do not know what is the patient numbers and duration required for part A of study meaning it is really 2 studies.
All this mess- co is screwed up now. Assuming Q1-2020 start, the interim analysis will be for at least 1/3rd patients (170-200) to complete at least 24-36 weeks of treatment - so we are looking at 18 months for interim futility analysis. Then if positive results continue all patient enrollment and 104 week treatment - total time 3+ years.
This is a very very long path - unless someone buys this company, not a good candidate for investment till interim analysis.
Welcome year end tax losses.
