Second Phase III study of Novartis JAK inhibitor INC424 meets primary endpoint in patients with myelofibrosis

COMFORT-II data show INC424 provides marked clinical improvement in patients with myelofibrosis, measured by reduction in spleen size at 48 weeks compared to best available therapy

17-Mar-2011 - Switzerland

Novartis announced that a pivotal Phase III trial of the investigational Janus kinase (JAK) inhibitor INC424 (ruxolitinib) has met its primary endpoint of significantly reducing spleen size in patients with myelofibrosis (MF). INC424 is a potent, selective inhibitor of the JAK1 and JAK2 tyrosine kinases.

The European study, called COMFORT-II (COntrolled MyeloFibrosis Study with ORal JAK Inhibitor Therapy), showed treatment with INC424 provided a statistically significant reduction in spleen size in patients with primary MF, post-polycythemia vera myelofibrosis (PPV-MF), or post-essential thrombocythemia myelofibrosis (PET-MF), when compared with best available therapy, administered at doses and schedules determined by the investigator. The safety profile of INC424 was consistent with previous studies. Complete study data will be submitted to an upcoming medical meeting.

These results support findings from another large Phase III clinical trial (COMFORT-I) conducted by the collaboration partner, Incyte Corporation, in the US, Canada and Australia comparing treatment with INC424 to placebo in patients with MF at 24 weeks. In addition, a Phase I/II study published in the September 16, 2010 issue of The New England Journal of Medicine showed that treatment with INC424 resulted in marked, fast and durable clinical benefits in patients with MF. These benefits included alleviation of debilitating symptoms and reduction of spleen size, an accepted measurement of clinical improvement in MF.

Results of the COMFORT-II and COMFORT-I clinical trials will form the basis of worldwide regulatory filings, planned to begin in the second quarter of 2011. The INC424 investigational studies constitute the largest clinical trial program in MF to date.

Myelofibrosis is an uncommon, life-threatening blood cancer characterized by bone marrow failure, enlarged spleen (splenomegaly), debilitating symptoms including fatigue, night sweats and pruritus, poor quality of life, weight loss and shortened survival. Myelofibrosis has a poor prognosis and limited treatment options. Although allogeneic stem cell transplantation may cure MF, the procedure is associated with significant morbidity and mortality and is usually appropriate only in younger patients. The five-year survival rate after transplantation is approximately 50%.

Novartis licensed INC424 from Incyte for development and potential commercialization outside the US. Incyte has retained rights for the development and potential commercialization of INC424 in the US. Both the European Commission (EC) and the US Food and Drug Administration (FDA) have granted INC424 orphan drug status for MF.

https://www.bionity.com/en/news/131391/second-phase-iii-study-of-novartis-jak-inhibitor-inc424-meets-primary-endpoint-in-patients-with-myelofibrosis.html