A drug that targets a rare form of leukaemia has received £2.8 million in development funding.

Developed by a team of researchers at Cardiff University, tefinostat now has backing from the Cancer Research Technology Pioneer Fund – a money pot started by the commercial arm of Cancer Research UK intended to advance the development of promising new cancer therapies – for use in cases of chronic myelomonocytic leukaemia (CMML).

CMML is considered an orphan disease, affecting just 4,000 people in the UK, US, France, Italy, Germany, Japan and Canada every year.

Tefinostat is an oral histone deacetlyase (HDAC) inhibitor which affects the wrapping of DNA around structures called histones. By inhibiting this process, DNA becomes less stable and can lead to cell death.

Tefinostat is specifically designed to target histones in two types of immune cells: macrophages and monocytes. Monocytes in particular are present in high numbers in CMML.

The money invested will now go towards a phase 2 study called MONOCLE which will aim to determine the tolerability and safety of tefinostat.

The trial will initially recruit 19 patients, a number which could increase to 40 based on results.

“We believe tefinostat has great potential in treating chronic myelomonocytic leukaemia and has the possibility for rapid market authorisation in a very well-defined patient population where there is a significant clinical need and little in development,” said Dr Robert James, managing partner of Sixth Element Capital – the Pioneer Fund’s managing investment fund. “The start of the phase 2 trial is a significant milestone and our funding will help drive forward this potential new treatment to approval in the next few years.”

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