BPN14770, a drug being developed by Tetra Discovery Partners for the treatment of Fragile X, was granted Orphan Drug Designation by the FDA this week. Tetra plans to soon commence Phase 2 trials of BPN14770 in both Fragile X in adults and Alzheimer’s disease.
Preclinical studies of BPN 14770 in mouse models suggested it has the potential to improve the quality of connections between neurons as well as multiple behavioural outcomes. Further Phase 1 studies have also demonstrated promising safety, oral bioavailability, and preliminary cognitive benefit in elderly subjects.
Sounds good for Tetra, but GlobalData Healthcare (a leading pharma intelligence company) questions whether it will be enough.
Why?
Well, they agree that there is a huge unmet need. There is currently no cure or approved therapies available for Fragile X. They note that key opinion leaders (KOLs) are of the belief that any drug which receives approval as a first-line therapy for Fragile X would quickly gain extensive market share.
However, GlobalData says it has one major reservation about Tetra’s drug…
…by the time this drug launches, there could be a handful of new drugs already approved for FXS. Trofinetide is one of those that also targets cognitive problems in addition to behavioural and psychiatric problems associated with FXS, and has received enthusiasm from KOLs interviewed by GlobalData. GlobalData expects trofinetide to launch in the US in Q2 2021, Europe in Q1 2022, and Japan in Q2 2023, indicating that BPN14770 will not be the first-to-market cognitive therapy for FXS, placing a commercial disadvantage on the drug.
https://www.pharmaceutical-technolo...n-drug-designation-fxs-treatment-will-enough/
https://www.globaldata.com/healthcare/about-us/why-globaldata/
