brilacidin / defensin-mimetics, will be the Drug (drug class) that Keeps on Giving imo --- link from the company blog. note the potential in IBD given Defensin deficiency. Humira-like potential, said with a straight face.
as to MOA and Prurisol, an Unknown -- so be it. like lots of other drugs....
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3396511/ Notwithstanding their key roles in therapy and as biological probes, 7% of approved drugs are purported to have no known primary target, and up to 18% lack a well-defined mechanism of action.
... including Kevetrin, which i think the IPIX is starting to get a better handle on... the different molecular targets and pathways.
P: know it's an immunumomodulator and affects PRINS and keratinocytes. what matters most of all is:
does it work?
seemed to in the P2 200mg arm -- good separation from placebo, w yes a small N..... the P2b will put sample size to rest and i'm thinking it will support the signal. hoping to match if not better Otezla (30% PASI 75) but really hoping it encroaches into biologics territory.
and let's not forget non-genotoxic Kevetrin's potential - yes, needs better formulation, to leverage half-life/clearance, but if it's (one of, if not) the first p53 drug approved (only a few in the running... mid-phase trials) -- well, Moonshot delivered.
all this talk of CAR-T, Mania -- Big Rx Buyer (and unfortunately, Patients) beware. some real Qs as to safety not just cost and how limited its reach.
below: recent study showing p53 in MCL. the way FDA is moving, approving meds by by genetic signature... bodes well for Kevetrin and the 50%+ of cancer implicated in dysfunctional p53.
TP53 Mutations Have Independent Prognostic Value in MCL
What were the significant findings?
Patients who had TP53 mutations did far worse than patients who were unmutated. We showed median overall survival of patients with TP53 mutations of only 1.8 years versus the median OS of unmutated patients were above 12 years. It is a pretty big difference and outweighed all of the other known prognostic markers in MCL.
[...]
How do these findings impact the treatment landscape of MCL? Understandably, people need to feel certain that this information is valid—but we are not the only ones showing this. TP53 has been associated with poor outcomes in MCL in different cohorts. We have now validated these findings in younger patients who are treated by what is currently the standard of care in MCL.
What we think the data suggests is that you should measure TP53 mutational status upfront when you diagnose a patient with MCL, and then, if there are any experimental trials, to include patients with TP53-mutated MCL. So far, they aren’t around, but there is a big European study called the Triangle trial that has 2 arms that include ibrutinib (Imbruvica), which has [has shown a] better effect, at least in patients with CLL with TP53 mutations. So, that is a potential option.
What would you like community oncologists to take away from these findings?
The main message is that TP53 mutations are associated with very poor outcomes in MCL, even though patients are treated very heavily with chemotherapy.
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