Eaglenebula, I've moved my reply to the questions you posed on...

Eaglenebula, I've moved my reply to the questions you posed on the chart and technical analysis thread. I know it bugs some chartists when non-chart discussion hogs their thread!

I note Rett Syndrome has a prevelance of 1:10-15000 girls ie 1:20-30000 population. Our likely first commerical product is RP11 around a third to a quarter of that at 1:100000. So I’m making a basic possibly poor assumption that Rett was a 3-4 billion market as opposed to RP11 1 billion.

You’re right that the Rett population in the United States is commonly estimated to be ~20,000 and that was the figure provided to NEU investors over the years. But as trofinetide came closer to market, Acadia spelt out that there were just 4,500 registered Rett patients in the US. In order to qualify for DayBue reimbursement, patients must be clinically diagnosed with proof of gene mutation. So, while technically there’s an addressable US patient population of ~20,000, in reality less than 25% of that figure is currently eligible for treatment. But that figure might grow now that a treatment is available.

While RP11 is less prevalent than Rett syndrome, you might well find that the percentage of those that suffer from the condition who are formally diagnosed is considerably higher. RP11 often runs in families so is more likely to be on the radar, whereas the gene mutation that causes Rett syndrome is almost always random. For this reason, I wouldn’t assume that the identified US population with RP11 is only <1,500 (i.e. a third of US Rett registered patients).
In terms of what the “addressable market” is for Rett, estimates vary considerably. I’ve seen estimates that range from US$350m – US$3.5bn, but most fall under US$1.5bn.

More often, you’ll see estimates of “peak sales” for a particular drug – that is, the maximum annual sales that the drug gets to. I’ve seen peak sales estimates for trofinetide in Rett ranging from US$250m to US$2bn. At the time of launch, Acadia was estimating peak sales of US$500m in Rett. Given a higher than expected price and faster than expected take up of the drug, numerous analysts now estimate likely peak sales for Daybue at US$1bn. But that is sometimes based on US sales only.

As you will see from all this, estimates of addressable markets and peak sales can almost become a game of “pick a figure”.


If PYC did a deal on this product is it likely to give us enough momentum to allow the other products to develop relatively unimpeded financially? Looking at PYC timetable that would be ADOA followed by PKD then PMS. Glaucoma also gets a mention in our latest presentation.

In my opinion, it is highly unlikely that a licensing deal for VP-001 (RP11) would be sufficient to allow full development of the following programs without further (substantial) funding being required. A good licensing deal though could certainly boost PYC’s share price, especially if a large upfront is attached.

While glaucoma has been mentioned by the Company and is an indication that I believe can piggyback to a degree on the ADOA program, I don’t see PYC as having the bandwidth to tackle this indication simply because of the very large trial sizes that would be required. But a glaucoma therapy could be an excellent early-stage asset for licensing to a large pharma, imo.