Share Price, page-614

I hope to get this one finally posted:
DavidJJ: " Neuren.......does a great deal including BIG cash upfront for ROW trofinetide, with focus then on developing and looking to do deals with NNZ 2591."

DavidJJ/others: Yes, INDEED, I think this is the BEST possible way forward for NEUREN!!

I did a very quick (and NOT in-depth) look at the four rare pediatric diseases that NEU plans to follow-up upon with NNZ 2591, i.e.:

1) Phelan-McDermid syndrome
2) Angelman syndrome
3) Pitt Hopkins syndrome
4) Prader-Willi syndrome
Right now it is stated that FDA review is pending but for Prader-Willi NEU plans to start in mid-2022.

What I was interested in - in my brief and fast checking - is IF there is any BIG PHARMA's around already researching in those respective fields...

Interestingly, for PRADER-WILLI, I did find the following:
- Somatropin (r-DNA) for injection (Brand name: Genotropin) - manufactured by PFIZER.
FDA approved indication: Long-term treatment of pediatric patients who have growth failure due to Prader-Willi syndrome (PWS).
(Genotropin is NOT appropriated for all children with PWS, so please talk to your doctor).
MY TAKE: A BIG PHARMA here already - that might be interested in what NEUREN has to offer...

For ANGELMAN I did also find some interesting matters as follows:
a) Hoffmann-La Roche - Angelman Syndrome
Drug: RO7248824
Study start: 19.8.2020
Estim. Primary completion as well as estim. study completion: 13.9.2022
ROCHE is yet another BIG PHARMA already involved in the field...
b) GENETEX and ULTRAGENYX (RARE on the Nasdaq exchange)
Together they informed about their joint efforts as follows:
27.9.2021: Orphan drug status for Angelman syndrome by the FDA
26.10.2021: Press release: Interim phase 1/2 data positive.
To me, ULTRAGENYX was a totally 'unknown' company. Therefore, I was a bit astonished to find out that this company's market cap is US$ 5.7bio!

For both PHELAN-McDERMID and PITT HOPKINS I could not find out any advanced competitive activities - BUT, as I DID mention, my
"research" was done very quickly and not too much "in-depth"....

Others might enlighten on those two indications and/or also improve on my aforementioned a bit !!

All the best to all NEU holders and buyers.

wasa