Morning Wasa, given the impressive data of PTX200 with virtually 4 Complete Responses to date, I was surprised to learn that 200 is taking a backseat to 100. However, I think Steve explains the scenario very well... plus there is another possible reason to focus more PTX100 in light of our OmniCAR trial coming up in AML.
We can't underestimate the changing landscape nor SYC's ability and tenacity to adapt to it. He states that AML is becoming a "crowded space" plus the cost of pursuing two registrational or pivotal trials ourselves would be doubly expensive. Both PTX100 and 200 present well as combination drugs. To pursue a drug as a monotherapy through to commercialisation for a small biotech such as ourselves probably wouldn't be the best use of funds when more and more (potentially viable) therapies in that indication are increasing.
Whilst the market may appear to be shrinking or becoming more competitve, it may also mean there is greater capacity there for PTX200 (as a targeted therapy) to be combined with other tech, such as a CarT therapy or alternatively farmed-out as SYC alluded. We are going to be trialling AML as one of our OmniCAR in-house CarT programs, anyway. In that trial, everything but the kitchen sink will be thrown at AML. It won't be just a single drug therapy being trialed like PTX200.
The other thing with PTX200 is, as Hottod highlighted, is that out CellPryme Adjuvant is partly based on its particular mechanism of action. PTX200 is a PH domain inhibitor that inhibits an important tumor survival pathway known as Akt, which inhibits the tumor survival pathway known as Akt. And, our CellPryme-A therapy in a way encapsulates the wonders of PTX200 (as exhibited in our AML patients) but in a therapy with broad application potentially across all cancer types and indications. It will likely be used in our OmniCAR trials too.
With all that said, I am not phased that our PTX200 in AML won't end in the "bang" we hoped for... I think the stellar safety profile and efficacy results to date speaks for itself and has provided the ammunition we need in our far more important bigger picture items such as CellPryme-A and OmniCAR. That is the platform which will unltimately encompass all the successful technology and assets this company is developing. If PTX200 can be farmed out... fantastic. Its becoming clearer that PTX100 needs to be pursued as a monotherapy given that our results have exceeded expectations and what's on the market for PTCL and CTCL sufferers is abysmal. Its a less competitve space and, therefore, a far more viable route to commercialisation.
The main game here is to get best value for money out of our assets, build our monetisable platforms and commercialise what we can for the best ROI. Thus far, IMO, our management team appear to be pursuing exactly that. We don't want to be a slave to academia and pursuing a therapy in an indication where the landscape is becoming crowded with competition. Our OmniCAR trial in AML is clearly an exception. With our CarT therapy, Spy-Catcher/SpyTag, MDA's TCR binder and CellPryme-M and A all in combo... that will be an AML trial worth pursuing! I'm hoping it will trump all AML trials out there and, more importantly, showcase the power of OmniCAR.
Hope my take on it is perceived for what it is and not just a "silver-lining" perspective. The only downside to what our PTX200 trial has highlighted is that perhaps recruitment might prove challenging for our upcoming OmniCAR trial in AML given the landscape. However, with the FDA changes coming on line and the fact that our trial incorporates Universal Receptor Imumotherapy platform, I see a far more favourable landscape for us.
Sorry, Wasa, I know you were probably hoping for something brief! Lol
