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Saturday, 02/09/2019 5:09:32 PM

Saturday, February 09, 2019 5:09:32 PM

Post# of 21541
Not sure if this has been posted here before, concerning rare diseases:

- Starting last September, NTRP has added a comment about rare diseases in the "About Neurotrope" section of news releases ("Neurotrope has also conducted preclinical studies of Bryostatin-1 as a potential treatment for rare diseases...including Fragile X syndrome").

- A disease qualifies as a rare disease or rare pediatric disease in the US if it affects fewer than 200,000 persons. Fragile X affects approximately 80,000 in the US, beginning in childhood.

- If the FDA grants "Rare Pediatric Disease designation" (usually in phase 3) to a drug, and the drug is approved, then the sponsor company is eligible to receive a priority review voucher.

- Priority review vouchers can be sold or transferred to another company. The going price for a priority review voucher in recent years has been in the range of $150M-$300M.

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Of note, the recent news release concerning CD-22 + lymphoma and B-cell cancer is in Pediatric and young adult patients. It seemed a bit odd to me that this was only being pursued in pediatric patients.

In light of this most recent news release, and the pursuit of Fragile X (a rare pediatric disease), perhaps a component of Neurotrope's ongoing development strategy is to target rare pediatric diseases, in order to qualify for lucrative Priority Review Vouchers (as well as Orphan Drug Designation)?

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