Positive news flowed in for Celgene CorporationCELG and Acceleron Pharma Inc. XLRN when the FDA granted fast track designations to pipeline candidate luspatercept for two separate indications – firstly, for the treatment of patients with transfusion-dependent beta-thalassemia and secondly, for the treatment of patients with non-transfusion-dependent beta-thalassemia.
The FDA grants fast track status to therapies that are considered capable of addressing unmet medical needs and possess the potential to treat serious or life-threatening disease conditions in order to facilitate its development and expedite the review procedure.
Luspatercept, currently in phase II, is jointly being developed by Celgene and Acceleron. The companies plan to initiate a phase III program in 2015 on luspatercept for the treatment of patients suffering from beta-thalassemia. The candidate is also being evaluated for myelodysplastic syndromes (phase II).
Apart from luspatercept, the companies are jointly developing sotatercept to promote increases in bone mineral density. Sotatercept is being evaluated for the treatment of final-stage chronic kidney disease.
While Celgene is responsible for fully funding the development cost for both programs (effective Jan 2013), Acceleron is entitled to $560.0 million of potential development, regulatory and commercial milestone payments, and will co-promote both products upon approval in North America. Acceleron will also receive a royalty on net sales in the low-to-mid 20% range upon the commercialization of the candidates following a potential approval.
We note that Acceleron currently does not have any approved products in its kitty. Hence, successful development and commercialization of these candidates will provide a big boost to the company.