New clinical trial data presented at the 2011 American Society of Hematology (ASH) Annual Meeting and Exposition in San Diego, California, further showcase the leading role played by Novartis in the treatment and care of patients with rare cancers and blood conditions.
These data, presented for the first time at the congress, underline the value and potential of current and pipeline haematology products from Novartis for patients and clinicians in the UK.
Among the results are data for nilotinib, a novel treatment for Philadelphia chromosome-positive chronic myeloid leukaemia (Ph+ CML) in chronic phase. These include: 36-month follow-up results from the Phase III ENESTnd study, which had five participating centres in the UK.
The study continues to show that significantly fewer patients treated first-line with nilotinib 300mg twice daily (bd) progressed to accelerated phase (AP) or blast crisis (BC) stages of disease vs. imatinib (p=0.0059)1. In addition, significantly more patients treated with nilotinib 300mg bd achieved deeper responses vs. imatinib (log3 73% nilotinib 300mg bd vs. 53% imatinib 400mg once daily; p=0.0001)1.
Also presented at the congress were new data from two Phase III trials looking at pipeline compound, ruxolitinib (INC424) in myelofibrosis – a life-threatening blood cancer characterised by bone marrow failure, enlarged spleen and debilitating symptoms: COMFORT-II evaluated patient-reported health-related quality of life (HRQoL) measures for ruxolitinib vs. best available therapy (BAT).
The results showed that myelofibrosis-related symptoms and HRQoL improved significantly from baseline for patients receiving ruxolitinib but remained the same or worsened for patients receiving BAT2. Four UK trial centres participated in COMFORT-II[v].
COMFORT-I evaluated the impact of ruxolitinib vs. placebo on symptom improvement, spleen volume reduction and overall survival (OS). Results show that patients receiving ruxolitinib had higher response rates, based on reductions in spleen volume and Total Symptom Score at week 243. Moreover, the OS analysis suggested a benefit with ruxolitinib therapy over placebo (hazard ratio=0.499; p=0.0395)3.
Breakthrough data for the thalassaemia treatment, deferasirox, in the treatment of non-transfusion-dependent thalassaemia (NTDT) were also presented. NTDT patients are at risk of serious health complications associated with accumulating excess iron[vi].
THALASSA, the first placebo-controlled study examining deferasirox in this setting shows that a 10mg/kg/day starting dose can significantly reduce iron overload in patients with NTDT from baseline, as measured by liver iron concentration (LIC): at 1 year, LIC had significantly decreased in the 10 mg/kg/day treatment arm, vs. the placebo arm, in which there was actually an increase in LIC (p<0.001)4.
The THALASSA results will be used as the basis for regulatory submission (an application for an extension to the current licence) for deferasirox in this setting in the U.S. and Europe. Deferasirox is approved under the trademark deferasirox and is already authorised for the treatment of chronic iron overload in patients with beta thalassaemia major[vii].
“The data presented at ASH this year are testament to Novartis’ commitment to investing in the development of medicines that improve outcomes for patients with rare cancers and blood conditions, who may otherwise have limited treatment options,” said Mr Panos Alexakos, Oncology General Manager, Novartis UK & Ireland.
“It is only through continued research like this that we can gain a clearer picture of the full role and value of these compounds and in doing so, help to ensure patients receive the best possible care.”