Very interesting. This would be aside form the SBIR funding that the CEO mentioned may be expected in the December yahoo news interview. https://www.sbir.gov/node/1206133
The rare pediatric disease vouchers do have value. Even though you have to have a drug approved for an indication to receive the voucher, they are traded in anticipation of eventual approvals. Neuren was able to include such anticipation of Rett approval into their partnership agreement last year even though they were completing a P2 trial. We very well could do similar with Fragile X or like you say somehow work it into pediatric cancer although I'm not really sure how at this point. Regardless this is from Neuren's website as far as valuing the vouchers.
"About Rare Pediatric Disease Priority Review Vouchers Under Section 529 to the Federal Food, Drug, and Cosmetic Act (FD&C Act), the US Food and Drug Administration (FDA) will award priority review vouchers to sponsors of rare pediatric disease product applications that meet certain criteria. A sponsor may choose to request rare pediatric disease designation from the FDA prior to submitting a New Drug Application (NDA), however an application for the voucher itself must be submitted with the NDA and the voucher is awarded on approval of the NDA. A voucher can be redeemed to receive a priority review of a subsequent marketing application for a different product, which can reduce the target review time for that product to 6 months, compared with the standard 10 months. A voucher can be sold to another sponsor. The market value of the voucher depends on supply and demand. The value derives from three potential factors: shifting sales earlier, longer effective patent life due to earlier entry, and competitive benefits from earlier entry relative to competitors. Earlier market entry can be worth hundreds of millions of dollars to the voucher holder. In 2014, the first voucher was sold for US$67.5 million. In 2015, a voucher was purchased for US$350 million. In 2017, five vouchers sold for prices between US$110 million and US$150 million."