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 on 3 April. 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 (IND) approval earlier, in March. Additionally, 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). This distinctive mechanism of action has the potential to improve cognitive and memory function in various devastating neurological disorders including FXS, Alzheimer’s disease, and other forms of dementias and schizophrenia. Preclinical studies have shown BPN14770’s potential to improve the quality of connections between neurons as well as multiple behavioral outcomes in mouse models. Promising safety, oral bioavailability, and preliminary cognitive benefit in elderly subjects were also demonstrated in three Phase I clinical trials.

Unmet need in Fragile X syndrome

FXS is a genetic disease results from the silencing of the X-linked, Fragile X mental retardation-1 gene. This rare and under-researched genetic condition occurs in both genders but is more common and generally more severe in males. There is currently no cure, nor are approved therapies available for FXS; thus the treatment landscape consists of widely genericized, off-label drugs, which are primarily for the treatment of symptoms, such as seizures, mood swings, or other neuropsychiatric issues. As such, 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, a large number of their patients would be switched over from their current treatments, all of which are symptomatic and administered off-label. KOLs share the opinion that symptoms of FXS can go untreated by current available options, and that these options rarely improve quality of life in severe cases, meaning that any new drug specifically approved for this indication would quickly gain extensive market share.

However, 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.

Apart from this, the candidate also faces challenges competing with other promising early-stage pipeline drugs with similar indications, including bryostatins and AMO-01, both of which are proposed to improve memory and cognitive aspects of FXS patients. This fierce competition would result in the need for Tetra to demonstrate significant therapeutic benefits of BPN14770 to secure market share and gain commercial success in the FXS market.

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Related Reports

GlobalData (2016). OpportunityAnalyzer: Fragile X Syndrome – Opportunity Analysis and Forecasts to 2026, GDHC081POA.