In order to expand the mast cell franchise that started with severe systemic mastocytosis in adults, AB Science has decided to initiate in 2015 two new phase 3 studies in Mast Cell Activation Syndrome (MCAS) and in pediatric mastocytosis.  Dr. Afrin is the catalyst behind these new trials. He will be conducting trials at U of MN.  Expect more information by the middle of 2015.

Mastocytosis in children gives urticaria pigmentosa and is primarily due to mutations of c-Kit that are efficiently inhibited by masitinib. The target population in children with mastocytosis is estimated at 20,000 in the USA and in the EU.

MCAS targets a much broader population than mastocytosis. MCAS is a common disease caused by activation of mast cells and associated with the same symptoms as in mastocytosis, such as chronic fatigue, pain, depression, diarrhea, irritable bowel, pruritus, flush and anaphylaxis, but it affects around 5 to 10% of general population. MCAS could include part of diseases such as multiple sclerosis, Crohn's disease, inflammatory bowel disease, Duchene muscular dystrophy, chronic fatigue syndrome, when such diseases exhibit clear signs of mast cell hyperactivity (such as increased tryptase in blood, increased histamine in urine, dermographism). Because masitinib has been designed to be a potent inhibitor of mast cell activation (through its action against c-Kit, Lyn and Fyn tyrosine kinases), it is a suitable drug candidate in MCAS. As for systemic mastocytosis, MCAS symptoms can be mild, moderate, or severe. The proportion of severe forms where masitinib could be developed is unknown but the prevalence of severe forms of MCAS is estimated by experts to be up to a 100 times larger than in mastocytosis on the basis of their own clinical experience. In MCAS, the mutations of c-Kit or other genes driving the activation of mast cells are unknown but different from the ones driving mastocytosis. The phase 3 clinical study that AB Science will launch in 2015 will be used to identify those mutations.



Read the whole release here:

https://globenewswire.com/news-release/2014/11/10/681375/10107287/en/AB-Sci ence-Masitinib-in-Mastocytosis-Summary-of-Web-Conference.html