@Strawman I've just finished watching the recording. (Happens every year, but the $NEU meeting was the first victim of diary mismangement on my part AEDT/AEST!!)
I think Jon repeated my thesis, which is essentially "two-for-the-price-of-one".
On valuation, I think I will go back and look at my valuation once the 3Q is in from $ACAD. But I have potentially undervalued NNZ-2591. The source of my error is that I haven't fully accounted for the enhanced value capture that $NEU will be able to achieve via a post-Phase 3 licensing deal, versus post-Phase 2 (trofinetide, North America). Because the numbers will look more like DAYBUE for RoW (which was post-Phase 3), the commercials should be much more attractive. I've assumed more of a DAYBUE look-alike, just discounted for time.
There are other factors that make NNZ-2591 potentially higher value than DAYBUE:
- Efficacy looks like it MIGHT be better than DAYBUE based on Phase 2 (obviously, wide confidence intervals still at this stage)
- Safety/Tolerability appears significantly better. NNZ-2591 doesn't seem to have the significant GI issues of trofinetide. (This could be a big factor in persistency (akak churn in SaaS terms),... persistency for DAYBUE appears to be flattening out around 50%. Higher persistency makes a big difference... so what if NNZ-2591 gets 60%, 70%, 80%?... we won't know for a couple of years until initial Phase 3 patients roll on to open label extension. But it could be material.)
- The market is larger for PMS than Rett, and the you can add PH later in addition. (Angelman might not get commercialised) But we don't know price. Rett in US is considered to be 6,000-9,000 patients, whereas PMS is 17,000-32,000 and PH is 7,000 - 9,000 ..... the combination is 24,000 - 41,000, which is 4-5x Rett.
- Intangible, at this stage, but once announcements are made on the additional indications under pre-clinical investigation, there could be a further uplift (platform effect)
Of course, to balance the positives, there are further factors:
- Development risk. Generically, at this stage, once Phase 3 endpoints are agreed, you might say 50% CoS. But with the larger safety dataset across the three phase 2 trials, and the significant, positive 11 of 14 endpoints, and the related mode of action to trofinetide, perhaps its more like 60-70%?
- Time value: With Phase 3 in 2025 to perhas mid-26, and approval in end 2026/early 2027, we're taking 2.5-to-3 years until revenue.
Other than that, thanks to @Scoonie for your question about the FDA meeting --- it was great hearing Jon's response. What a high stakes one hour!
On valuation, I'm still happy around $24 with a wide band, but almost totally skewed to upside from today (notwithstanding my likely low-balling of NNZ-2591)
I'll revisit the valuation once the following are in:
- Q3 DAYBUE sales - because that will give a better view of the trajectory into 2025
- Resolution of Phase 3 endpoints for NNZ-2591. On this second point, the tension is that $NEU want as low a bar as possible (the seller) whereas FDA wants a higher bar to ensure a beneficial product worth its price is entering the market (the buyer). As a totally new therapy area, it was hardly surprising there'd be clear water between these positions, particularly given the subjective nature of the endpoints.
If both these points are positive, I'll consider adding to my RL position (now 4.2%) - provided the market doesn't run away. Hopefully, we'll see a little bit of "once bitten, twice shy" so the agile might be rewarded. Ironically, from my perspective, the best news would be if DAYBUE just scrapes in at the low end of guidance. Too strong a result, might create a premature relief rally - but I think the chances of a very strong result are minimal, given the language used in the September conferences!
For now, there is nothing to do but wait.
Disc: Held in RL and SM