Antisoma was given a boost last week on news that regulators on both sides of the pond have awarded orphan drug status to AS1411 for treating patients with acute myeloid leukaemia (AML).

AS1411 belongs to a new group of drugs called aptamers, which are essentially short strands of DNA or RNA that fold into various three-dimensional structures that are able to target certain proteins on cells, including on tumours.

Promising Phase II data - published at the 2008 ASH and 2009 ASCO meetings – showed that adding AS1411 to cytarabine substantially boosted response rates without significantly increasing side-effects in patients with relapsed or refractory AML, a disease in which the bone marrow makes abnormal and immature blood cells ultimately resulting in bone marrow failure.

The UK drugmaker has now confirmed that the first of Phase IIb trials are expected to start early next year, and if results are positive it anticipates “rapid progress into a registration trial”. Speedy development and approval of the drug will be good news for patients given that this form of cancer has a very poor prognosis, with the potential to be fatal in just six-12 months.

AS1411 has already been granted orphan drug status in the US and European Union for the treatment of renal cancer, in which initial Phase II data are expected before the end of the year, Antisoma said.