CYT387

CYT387 is a novel oral JAK1/JAK2 inhibitor initially targeting the treatment of a group of hematological disorders known as myeloproliferative neoplasms (MPNs), also known as myeloproliferative disorders. Additional potential treatment indications for CYT387 include other MPNs, cancer (solid and liquid tumors), graft-vs-host disease, and inflammatory conditions.

Potent, selective JAK1/JAK2 inhibitor

 

CYT387 is a potent, selective JAK1/JAK2 inhibitor designed to suppress the over-activity of the mutant JAK2. The compound possesses an excellent selectivity and safety profile with minimal off-target activities, favorable pharmacokinetic properties, a clean toxicological profile and the prospect of limited drug/drug interactions. Preliminary data using samples derived from MPN patients have shown promising activity in suppressing the over-activity of the JAK2V617F mutant enzyme.


CYT387: Mechanism of Action



JAK1/2 in myeloproliferative neoplasms

MPNs are debilitating, and potentially fatal, diseases of blood cell production. MPNs such as polycythemia vera (PV), essential thrombocythemia (ET) and myelofibrosis (MF) are disorders for which there are limited and poorly efficacious therapeutic options. A genetic mutation in the JAK2 enzyme, which results in constitutive activity of the enzyme, has been shown present in >95% of patients with PV, ~50% of patients with ET and ~50% of patients with MF. In an in vivo model of MPNs, CYT387 showed excellent activity, essentially reversing a number of the hallmarks of the disease.

JAK1/2 in cancer

JAK2 and its related biochemical pathways are now known to play a key role in the proliferation of certain types of cancer cells such as prostate, breast, head & neck, lung, ovarian, renal cell, glioma, pancreatic and liver cancers, and hematological cancers such as multiple myeloma, lymphoma and leukemia. Preclinical research is underway to examine the activity of CYT387 in these indications.

 

Clinical trials with CYT387

 

The compound is currently being studied in a Phase I/II clinical trial and Phase II BID clinical trial in patients with myelofibrosis.

 

Availability of CYT387

 

CYT387 is available through participation in ongoing clinical trials.