Marker Therapeutics Inc (MRKR)

[Phantom Lord]

I know you didn’t ask me, but I am going to expand on this since I’m assuming it was my post you are referring to where I mentioned that the PII AML trial is pivotal which means it can skip PIII.

A pivotal trial is basically just the trial that intends to provide all evidence necessary for the FDA to grant approval. Obviously, this is usually a PIII but PII trials can be designed in a way to provide the FDA with all they need to grant approval. That is what Marker has done with the PII AML trial and they worked with the FDA in the trial design to make sure that it will show everything the FDA wants to see. They can still request a PIII be done but as of right now the intention is for the PII to provide all the evidence necessary for an approval to be granted.

They have ONLY 172 participants in the Phase 2 trial, which seems very, very small for the FDA to declare that a Phase 3 trial is not needed (if the Phase 2 results are good)!

AML is relatively rare with ~20,000 new cases reported annually. HERE is a source for that. Marker estimates that ~3,500 of those patients receive HSCT. HERE is their corporate presentation. Their estimates start on slide 17. I don’t know where they get this number but HERE is a study from 2010 that estimates 25,000 total HSCTs are performed each year with ~33% of those being in AML. That would be about 8,000 HSCTs done for AML patients each year. I couldn't find a specific stat for this but the number probably falls somewhere between 3,500 and 8,000. The point is that AML is already rare. When you account for the fact that not even half of AML patients get an HSCT Marker’s targeted patient population becomes even smaller. 172 patients are definitely enough to collect the data necessary for the FDA to grant an approval.

Kite provides a real-world example. HERE is the ZUMA-1 trial that Yescarta was initially approved on for lymphoma. They ran three multiple trials concurrently totalling 307 patients. One of those being a pivotal PII that enrolled 101 patients. HERE is the PR from when they were granted approval that states, “The approval of Yescarta is supported by data from the ZUMA-1 pivotal trial. In this study, 72 percent of patients treated with a single infusion of Yescarta (n=101) responded to therapy…”

Depending on the severity and overall occurrence of the target indication therapies can absolutely be approved without the need for a PIII depending on trial design and with a smaller patient sample size.