@Slideup and @Nnyck777 clearly, part of the strategy is registering NNZ-2591 for indications where they can maximise value - i.e. orphan designations. There are several levers to pull:
- Orphan indications get 7 years exclusivity from FDA irrespective of patent protection on the molecule
- Orphan indications get higher pricing, an order of magnitude or more beyond non-orphan indications; pricing on non-orphan indications is more competitive
- They will eventially need to trade-off market reach when they move beyond orphan status indications and patent protection - for that you need to be a global major, with all the global channels to market. Hard for $NEU to build to that - not sure how long they've got - so there might be further commercial deals over time. The key near term (next 12 months) decision, is whether to licence NNZ-2591, or build out $NEU capability in sales and marketing. (The PM and PH Census databases are a key enabler for going it alone, I think)
- I also don't know what the patent suite around the molecule looks like. Formulation/delivery patents can build out the time-frame beyond the original patent for the chemical entity. But at this stage, it will be years before this is on investors radar screen. That said, the orginal patents for trofinetide are very old, but perhaps there are more recent patents around the formulation and injection delivery. I know these can be important factors, but I am pretty clueless as to the specifc facts as it impacts $NEU. (Something for future investigation)
- Of course, all these indications, whether orphan or not, whether 5, 10, 20 or 100 will require clinical trials.
So it is a complex, multi-dimensional matrix, which must be challenging for a small firm like $NEU to navigate.
The good news for $NEU shareholders is that just with PM and PH indications, they've selected 1 very high and 1 modest indication which, if both make it through Phase 3 will more than double the value of the company (IMHO). Rationale: as I read it, NNZ-2591 looks like it might have better tolerability than trofinetide and if that's true it will have higher persistency. The efficacy of PM looks like it might be relatively strong compared with trofinetide for Retts and NNZ-2591 for PH. (This is a very subjective and unscientific not to say premature comparison given the small sample sizes at Phase 2 and the different conditions ... so I probably shouldn't have written it, and will be torpedoed by someone who checks the stats). There is a lot to understand here.
So overall, while all this discussion of a "platform" is interesting and - in time - might be very important, from a shareholder perspective I see the key value-drivers in order as:
- PM
- Daybue US
- Daybue RoW
- PH (or maybe this is 3)
- Anglemans
- ... not sure what to make of PW
Of course, from the perspective of overall human health benefit, if this is a major platform it could be very significant. The point of this post is just to highlight that this doesn't necessarily translate into a huge commercial success for the firm that registers the first indications. There is a lot of skill to develop the optimum commercial strategy for a platform, and its beyond my capability even to describe it fully. But I see enough indicators here to know if might be relevant.