Novartis blood cancer drug impresses in late stage trial

pharmafile | December 22, 2010 | News story | Research and Development, Sales and Marketing INC424, Novartis, blood cancer, myelofibrosis, orphan drug 

Novartis has released positive phase III results of its myelofibrosis drug INC424 showing it met its primary endpoint of reducing spleen size.

Myelofibrosis is a rare, life-threatening blood cancer characterised by bone marrow failure and an enlarged spleen (splenomegaly).

The phase III COMFORT trial showed treatment with INC424 provided a statistically significant reduction in spleen size in patients with primary myelofibrosis (MF) and several other forms of the disease.  

The study also met its secondary endpoint of improving symptoms as measured by a bespoke Myelofibrosis Symptom Assessment Form Diary.

The JAK family of enzymes is a key player in a number of important biologic processes, including the regulation of immune function and the formation and development of blood cells.

INC424 is a potent, selective inhibitor of the JAK1 and JAK2 signals, potentially halting JAK mutations common to MF.

Hervé Hoppenot, president of Novartis oncology, said: “Throughout its clinical development, INC424 has demonstrated the potential to fill a critical need for patients with myelofibrosis, who currently have limited treatment options.

“This promising JAK inhibitor is an important part of our rich pipeline of innovative new therapies that address unmet needs in hematology and cancer treatment.”

Current MF treatments include allogeneic stem cell transplantation to cure the disease, but the procedure is associated with significant morbidity rates and is usually appropriate only in younger patients.

Both the FDA and the EMA have granted INC424 orphan drug status for MF and Novartis plans to file the drug for regulatory approval next year.

In December last year Novartis paid US, Delaware-based Incyte Corporation $150 million for the rights to two of its oral investigational haematology-oncology therapies, including INC424.

Ben Adams

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