The month of May saw major announcements in breakthrough therapies by companies working on the Beta-Thalassemia curatives - LentiGlobin BB305 and Luspatercept. Beta-Thalassemia is a life-threatening, blood disorder where the body fails to produce sufficient haemoglobin to thrive.
This inherited disorder is caused by mutations in the beta-globin gene (HBB gene). Currently, the standard protocol for treatment of Beta-Thalassemia major is lifelong blood transfusions along with regular iron chelation therapy either through infusion or oral medications. Bone marrow transplant with a suitable match is the only available cure currently.
LentiGlobin BB305Bluebirdbio (HQ: Cambridge, Massachusetts) is a clinical-stage company working on potential curatives for genetic disorders like Beta-Thalassemia. On May 19, 2015, the company issued a press release announcing its meeting with European and United States regulatory authorities to discuss its plan to take the LentiGlobin BB305 product forward. LentiGlobin BB305 is a potential curative for Beta-Thalassemia.
The LentiGlobin BB305 introduces a fully functional human beta-globin gene into bone marrow cells extracted from the patient. The modified cells are then re-transplanted into the same patient. The functional gene enables production of normal hemoglobin levels thereby cutting out the need for lifelong blood transfusion and other therapies. Clinical data from the Northstar study and HGB-205 study were very encouraging leading to a "breakthrough therapy designation" from the FDA in February 2015.
The LentiGlobin BB305 is a one-time gene transfer therapy, which promises a permanent cure for Beta-Thalassemia. According to David Davidson, M.D., chief medical officer, the company is looking forward to two planned open-label studies each with a 15-patient sample size.
LuspaterceptAnother breakthrough announcement was made on May 18, 2015 when the FDA granted fast-track status to the product Luspatercept for patients with transfusion-dependent as well as non-transfusion-dependent Beta-Thalassemia.
Luspatercept is currently being jointly developed by Acceleron Pharma Inc. (a clinical stage biopharmaceutical company, HQ: Cambridge, Massachusetts) and Celgene Corporation (an American biotech company, HQ: Summit, New Jersey). Blood cells undergo several stages in the bone marrow before they mature and are released into the blood. Luspatercept (ACE-536) is a compound which regulates late-stage blood cell differentiation and maturation. This corrects the anemia and cuts out the need for blood transfusions. Luspatercept has been tested in human patients in phase 1 and phase 2 clinical trials.
According to Jacqualyn A. Fouse, President of hematology/oncology at Celgene, Celgene and Acceleron are now working towards initiating a phase 3 clinical trial in 2015.
With two potential curatives being developed, the outlook for patients with Beta-Thalassemia across the globe seems to be positive.