Baxter International has acquired AesRx, a private U.S. biopharmaceutical company focused on orphan drug targets, including the development and commercialization of Aes-103, an investigational prophylactic treatment for sickle cell disease (SCD).

Aes-103 is a first-in-class, oral, small molecule compound (5-hydroxymethylfurfural). Early studies indicate the compound may work by binding to hemoglobin and increasing oxygen affinity and stabilization, thereby reducing the sickling of red blood cells which, in turn, may reduce sickling-related outcomes such as vaso-occlusive crisis, pain, severe anemia and fatigue. Aes-103 has received Orphan Designation from the FDA and is eligible for Orphan Designation in Europe.

The Aes-103 program currently is in a phase II clinical trial as part of an ongoing collaboration with the NIH's National Center for Advancing Translational Sciences (NCATS) through its therapeutics for rare and neglected diseases program. Data previously have been presented from an escalating dose phase I/IIa safety study involving administration of a single dose among stable SCD patients.

''Sickle cell disease was the first disease to ever have its molecular cause discovered, and now a potential treatment based on that discovery at last has been developed,'' said Christopher P. Austin, M.D., NCATS director. ''This success validates the NCATS model, which is based on a novel collaborative approach that de-risks intervention development programs to enable private-sector investment.''

Baxter made an initial payment to acquire the company and may make additional future payments based on specified development, regulatory and commercial milestones. Specific terms of the agreement have not been disclosed.