Baxter International Inc. has acquired AesRx, LLC, a private US biopharmaceutical company focussed on orphan drug targets, including the development and commercialisation 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 haemoglobin and increasing oxygen affinity and stabilisation, 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 programme is currently in a phase 2 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 (TRND) programme. Data has previously been presented from an escalating dose Phase I/2a safety study involving administration of a single dose among stable SCD patients. The compound, originally patented by Virginia Commonwealth University, was developed by a team from the VCU Institute for Structural Biology and Drug Discovery, an interdisciplinary research center spanning the university's Schools of Medicine and Pharmacy.

"Sickle cell disease was the first disease to ever have its molecular cause discovered, and now a potential treatment based on that discovery has at last been developed," said NCATS Director Christopher P. Austin, M.D. "This success validates the NCATS model, which is based on a novel collaborative approach that de-risks intervention development programmes to enable private-sector investment. We look forward to applying this model to the thousands of rare diseases that are currently untreatable, so that we realise the NCATS mission of getting more treatments to more patients more quickly."

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

"With this acquisition, we gain a compound that has the potential to address an extremely high unmet clinical need in a community with inadequate treatment options and no recent major clinical developments," said Ludwig Hantson, Ph.D., president of Baxter BioScience. "This programme is complementary to our established experience in hemophilia and supports our goals to raise the bar for care of patients with a range of blood-related disorders."

"By becoming a part of Baxter's organisation, we believe this will provide Aes-103 the needed resources and expertise to complete a robust development and commercialisation programme," commented Stephen Seiler, AesRx's Founder and former chief executive officer. "Baxter has a demonstrated track record in the treatment of hematological orphan diseases and is an ideal company to take this important programme forward."