I forgot to thank you,
@hottod, for the links regarding the FDA's "change of tune" on the Accelerated Approvals Program. I had read a bit about it previously and see the need for an overhaul due to those biotechs that have been abused the program. It doesn't belie the underlying worthiness of the program. The FDA are cognisant to that fact because of how reliant the industry is on small biotechs for innovation.
The main rectifier (as I see it) to the perceived abuse of the program, is to make the
timing of Confirmatory Trials mandatory and not just discretionary. I believe that is as it should have always been... and, therefore, should not present as a big problem for PTX. Its stated that:
"“The major thing that we’re really interested in is companies having a comprehensive plan about what their confirmatory study is … to discuss this earlier on,” Pazdur said during a discussion with FDA Commissioner Robert Califf, and M.D., at the Friends of Cancer Research annual meeting Thursday.
So, heads down bums up, PTX to get PTX100 into Phase 2 trials and then pursue a trial in lung cancer (combo or otherwise) in parallel because that would go down well with the FDA.
I have spent many hours "boning up" on RAS pathways, KRAS, its "children" and lung cancer, thanks to "Brighthope"! Fascinating stuff, albeit, a head crusher and destroying of a Saturday night and Sunday morning!!! Lol. Whilst I have s better understanding of why PTX100 has "potential" in the broad landscape of RAS pathway and how inhibition of the Rho-child has (obviously) emerged as one to go after in the TCL indication cancer types... I'd love to know the particulars of why lung cancer has been mentioned by SYC.
I've learnt that the RAS oncogene mutates differently in different cancer types. Will PTX100 perform differently in the RAS oncogene of lung cancer patients and disrupt the pathway in which overactivation of cell production leads to proliferation of mutant cells? If so, which of the Rho, Rac and Ral pathways will it serve to disrupt and benefit the lung cancer patient? That will make for an interesting study given that most biotechs are fixated on KRAS-GL12C.
It seems that SYC is intent on pursuing the under-explored and under-serviced sectors of the industry. I just hope that in the process, that the poor under-medicated, over-invested SH of diminishing patience are bestowed with some over-appreciation and rememberance soon, shortly, imminently in the so-distant-future!!!
