Hi Shell,
"It would have been nice to have seen a higher number of PTCL patients but I guess, it is what is, a rare condition and cohorts hard to come by probably"
I think it would have been difficult to accommodate more in the mixed cohort in my mind.
Despite the primary objective being to discover the safety profile of PTX-100, it was a mixed cohort to also discover what cancers could garner a response, if possible to coach that data out of the basket study.
Unless we expanded the original basket cohort to say 24 patients it would have proved difficult, not to mention the cost of doubling the scope of the trial...Had Steven known the response PTX-100 was going to get in PTCL, I'm sure he'd have recruited many more PTCL patients.
But I think Steven & the team certainly got what they had come for with a perfect safety profile & a number of promising responses in the PTCL/TCL.
If the so far perfect safety profile continues, it would be very unlikely in my opinion for the FDA not to award a registrational trial on even just a further few more responses across the expanded cohort.
After all, with a perfect safety profile demonstrated...What would the FDA then have to lose on awarding a registrational trial? Given the urgency & unmet need of PTCL/TCL?
Keep up the great posting Shell. Makes for great reading and love the constructive conversation that births from yours and others participation.
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