ApoPharma, the innovative drug development arm of Canadian generics firm Apotex, has been boosted by two studies showing superiority of its iron-chelating agent Ferriprox (deferiprone) over the current gold standard treatment Desferal (deferoxamine).

The studies comparing the two treatments in preventing cardiac complications of beta-thalassaemia major were presented this week in Dubai at the 10th International Conference on Thalassaemia and Haemoglobinopathies and will be published in the leading haematology journal Blood. The papers are currently available online. Cardiac outcomes in thalassaemia are of concern to clinicians since over 70% of patients die prematurely from heart failure caused by iron overload.

In one study, led by Caterina Borgna-Pignatti of University of Ferrara, there were no events or deaths among 157 Ferriprox-treated patients while 14% of 359 patients treated with Desferal experienced cardiac events including 15 deaths.

A second study led by Professor Dudley Pennell of Imperial College, London, showed Ferriprox was faster and more effective than Desferal in reducing cardiac iron and improving left ventricular function among 61 patients with asymptomatic iron overloaded heart problems.

Ferriprox is currently approved in Europe as second-line therapy for patients with thalassaemia and transfusion-related iron overload. It is also recently been introduced in Turkey and the middle east. ApoPharma President Michael Spino said talks will begin with the US Food and Drug Administration (FDA) next month with a view to making the drug available in the USA. The new data support first-line use, he suggested.

ApoPharma faces competition from Novartis’ new once-daily oral-chelating agent Exjade (deferasirox) launched in the USA in December and currently under review by the European Medicines Agency (EMEA). However whether or not Exjade achieves the same cardioprotective outcomes as Ferriprox has yet to be established through clinical trials.

Professor Pennell is currently investigating Exjade’s activity in the heart. He believes Ferriprox is effective in removing iron from cardiac tissue on account of its smaller uncharged molecular structure. “A head to head study of the two agents is needed”, he suggested.

ApoPharma has several other iron chelators in development as well as a wound-healing agent for leg ulcers. Dr Spino believes there could be a wider role for iron chelation treatment in more prevalent condtions such as Alzheimer’s and Parkinson’s Disease. The privately-owned company may go public in future to raise funds needed to develop pipeline products that are showing promise in pilot studies, he said. Currently the Apotex Group has an R&D budget of C$1.3 billion ($1.1bn) to cover the next 10 years.