Swiss biotechnology company Asceneuron has announced the US Food and Drug Administration (FDA) has granted its lead drug ASN120290 orphan drug designation for the treatment of rare neurodegenerative disease progressive supranuclear palsy (PSP).
ASN120290’s therapeutic potential was been demonstrated in pre-clinical studies. The drug, which is a selective inhibitor of the O-GlcNAcase enzyme, caused a significant reduction in the build-up of toxic aggregations of tau protein into neurofibrillary tangles in the brain.
The accumulation of tau and neurofibrillary tangles are recognised as key causes of PSP and other neurodegenerative diseases. PSP causes severe problems with walking, balance, speech, swallowing and vision, however, it is commonly misdiagnosed as it has similar symptoms to Parkinson’s disease.
Asceneuron founder and chief executive officer Dirk Beher said: “PSP is a rare neurological condition for which there is currently no treatment available. ASN120290 is an orally bioavailable molecule that has the potential of treating the root cause of the neurodegeneration. The granting of orphan drug designation for ASN120290 by the FDA is an important milestone for the team and the company. It strengthens our commitment to serving this important unmet medical need and bringing this molecule to patients.”
The company has also recently completed a randomised, double-blind, placebo-controlled phase 1 study to assess the safety and tolerability of single and multiple doses of ASN120290 in healthy patients. The results of this study will be presented at the Alzheimer’s Association International Conference in Chicago on 22-26 July.
The FDA’s orphan drug designation is granted to drugs and biologics that have the potential to diagnose or treat rare diseases. The designation includes a seven-year period of market exclusivity in the US following approval, FDA assistance in trial design and exemption from FDA user fees.
If Asceneuron’s ASN120290 gains FDA and other regulatory authority approval it would become a first-in-class treatment for PSP.
The company’s long-term aim is to use a similar mechanism of action to treat other taupathies, a group of neurodegenerative conditions associated with the aggregation of tau proteins in the brain, including Alzheimer’s, subacute sclerosing panencephalitis and corticobasla degeneration.