“There's a huge need for medication specifically approved for FXS treatment”


Tetra’s fragile X syndrome drug must demonstrate significant therapeutic benefits to secure market share, says GlobalData

BPN14770, which is being developed by Tetra Discovery Partners for the treatment of fragile X syndrome (FXS), was granted orphan drug designation by the FDA in April 2018.

However, by the time this drug launches, there could be a handful of new drugs already approved for FXS, according to GlobalData, a leading data and analytics company.

Tetra is planning to initiate Phase II trials in adults with FXS and in patients with Alzheimer’s disease in Q2 2018 after receiving investigational new drug approval in March. The company intends to expand its evaluation of BPN14770 in FXS to paediatric patients later this year.

BPN14770 is a novel therapeutic agent that enhances early and late stages of memory formation by selective inhibition of phosphodiesterase-4D (PDE4D) and has the potential to improve cognitive and memory function in various devastating neurological disorders.

FXS is a genetic disease whose market was worth $64m across the seven major markets in 2016. It is expected to reach $245.6m by 2026, at a compound annual growth rate of 14.4%. This rare and under-researched genetic condition is more common and generally more severe in males.

Dr Hayley Chan, Pharma Analyst at GlobalData, said: “There is no cure and no approved therapies for FXS. Thus the treatment landscape consists of widely genericized, off-label drugs, which are primarily for the treatment of symptoms. There is a huge need for medication specifically approved for FXS treatment that targets the etiology of the disease directly, rather than treating each individual symptom.”

Key opinion leaders (KOLs) interviewed by GlobalData were convinced that should any drug receive approval as a first-line therapy for FXS, many of their patients would be switched over from their current treatments, all of which are symptomatic and administered off-label.

Chan continued: “The fact that symptoms of FXS can go untreated by the available options and that these options rarely improve quality of life in severe cases, are shared beliefs among KOLs, meaning that any new drug specifically approved for this indication would quickly gain extensive market share.”

Trofinetide targets cognitive problems as well as behavioural and psychiatric problems and faces competition from other early-stage pipeline drugs with similar indications. GlobalData expects trofinetide to be launched in the US in Q2 2021, in Europe in Q1 2022 and in 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.

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Chan added: “This competition means BPN14770 needs to demonstrate significant therapeutic benefits to secure market share and commercial success in the FXS market.”