Evgen Pharma PLC (LON:EVG) announced progress for its lead asset on two fronts in the field of cancer.
In the US, it has applied to the Food & Drug Administration for orphan drug (OD) status for SFX-01 in the area of glioma, a brain cancer with a very poor prognosis.
A separate announcement, it said that SFX-01 has shown some very early promise as a potential leukaemia treatment.
The orphan drug application first: it is hoped the American regulator will make its decision by the end of the year.
Orphan status could potentially shorten the route to market for SFX-01, as well as providing a longer period of exclusivity over the drug.
It is granted to companies developing treatments for rare diseases where there the patient population is fewer than 200,000 in the US.
In parallel, an investigational new drug dossier for SFX-01 is being prepared for submission to the FDA around the end of the year.
This would support a phase II trial in glioma/glioblastoma that is set to get underway in the first half of next year.
Evgen chief executive Dr Huw Jones said: "Given the progress with our glioma programme and our intent to commence trials in 2022, we are seeking orphan drug status to maximise our intellectual property protection, commercial potential and partner appeal in this devastating disease.
“The incentives offered by OD designation mean that rare diseases like glioblastoma are more likely to be researched in the clinic.''
In the second of the two announcements, the company said preclinical data demonstrated a significant reduction of cell proliferation and increased apoptosis (cell death) in a mutated form of leukaemia.
The study was carried out by Dr Eleni Louka and Professor Adam Mead of the MRC Weatherall Institute of Molecular Medicine at Oxford University who investigated the effect of SFX-01 on cells from tissue donated by patients with juvenile myelomonocytic leukaemia.
Their work follows on from a pre-clinical evaluation in a similar vein by Professor Philip Eaton, of Queen Mary University of London.