Mirati's Mocetinostat For Lymphoma Designated As Orphan Drug
Mirati Therapeutics announced that the U.S. Food and Drug Administration (FDA) has designated Orphan Drug status to mocetinostat for the treatment of diffuse large B cell lymphoma (DLBCL).
Mocetinostat is a spectrum selective, orally bioavailable HDAC inhibitor being developed as a monotherapy for patients with diffuse large B-cell lymphoma (DLBCL) and bladder cancer with certain genetic mutations in Histone Acetyl Transferases (HATs). The drug works by reversing aberrant acetylation caused by the HAT mutations, which is expected to stop tumor growth and reduce tumor burden. The orphan designation is mocetinostat’s second as it also received the same distinction in June for the treatment of myelodysplastic syndrome (MDS).
DLBCL is an aggressive form of lymphoma that is commonly found in the lymph nodes but which can also invade areas outside the lymphatic system such as the thyroid, breast, skin, gastrointestinal tract, bone, or brain. Symptoms include painless, rapid swelling in the neck, groin, or armpit.
Dr. Charles Baum, president and CEO of Mirati, said, “We have identified genetic alterations in histone acetylation pathways (CREBBP and EP300) in approximately one third of DLBCL and bladder tumors. Nonclinical tumor models exhibiting these mutations are particularly responsive to mocetinostat. Among other benefits, orphan designation provides seven years of market exclusivity to target these genetically defined patients with unmet medical need in the event we achieve regulatory approval.”
The company said mocetinostat is currently undergoing Phase II studies as a combination therapy together with Vidaza for the treatment of intermediate and high-risk MDS. Initial data from the studies are expected by the end of 2014. Mirati also plans to conduct additional Phase II studies of the drug as a single agent for the treatment of patients with mutations in histone acetyl transferases in bladder cancer and DLBCL. The drug has previously completed 13 clinical trials in different hematologic malignancies and solid tumors.