Program Carries Potential to Address Significant Unmet Patient Need
July 9, 2014
Baxter International Inc. today announced the acquisition of AesRx, LLC, 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 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) program. 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 programs 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 realize 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 program 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 organization, we believe this will provide Aes-103 the needed resources and expertise to complete a robust development and commercialization program,” 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 program forward.”
About Sickle Cell Disease
Sickle cell disease (SCD) is a recessive disorder of the hemoglobin which can cause red blood cells to deform into rigid sickle shapes that block capillaries and other small blood vessels. This blockage can lead to a wide range of serious, sometimes life-threatening, conditions including chronic hemolytic anemia, chronic pain and acute painful crisis, stroke, acute chest syndrome, and cumulative damage to tissues and organs.
More than 13 million individuals worldwide suffer from SCD, with an estimated 100,000 people affected in the United States and 40,000 affected in Europe. SCD affects predominantly people of African descent but also occurs in certain other ethnic groups, including those of Mediterranean or Middle Eastern descent. Severe SCD is associated with chronic pain causing reduced quality of life and reduced life expectancy.
About Baxter International Inc.
Baxter International Inc., through its subsidiaries, develops, manufactures and markets products that save and sustain the lives of people with hemophilia, immune disorders, cancer, infectious diseases, kidney disease, trauma and other chronic and acute medical conditions. As a global, diversified healthcare company, Baxter applies a unique combination of expertise in medical devices, pharmaceuticals and biotechnology to create products that advance patient care worldwide.