AesRx – Biopharmaceutical Company Targeting Sickle Cell Disease

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AesRx Presents
Phase 1/2a Trial Data of Anti-Sickling Agent Aes-103 at ASH


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AesRx (ǽs-r-ex) is a biopharmaceutical company dedicated to the development of two novel drugs, each of which targets an orphan disease.

AesRx’s lead program, Aes-103, is a potential breakthrough in the treatment of sickle cell disease (SCD). AesRx has commenced a Phase 2 clinical trial of Aes-103 as part of an ongoing collaboration with the NIH. AesRx and the NIH have previously successfully completed a Phase 1 trial of Aes-103 in healthy volunteers and a Phase 1/2a trial in stable sickle cell patients. AesRx holds the IND for Aes-103.

Sickle cell disease 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 world-wide suffer from SCD, with over 100,000 of these in the US and more than 40,000 in Europe. Sickle cell is recognized in the US as an orphan disease and current treatment options for sickle cell patients are inadequate.

Aes-103 is an orally bioavailable small molecule (Da 126) discovered by researchers at Virginia Commonwealth University. It works by stabilizing sickle hemoglobin in the high oxygen-affinity R-state thus directly preventing sickling. It has also been shown to stabilize sickle red blood cells against the shear stress associated with SCD. This stress leads to red blood cell death (hemolysis) resulting in anemia in many patients.

AesRx’s second development program, Aes-210, is targeted to treat certain inflammatory diseases of the lower intestine, including ulcerative colitis, distal ulcerative colitis, pouchitis and radiation induced proctitis. These diseases can have a devastating impact on the lives of those afflicted. Existing therapies are inadequate and many patients do not respond. The Company estimates 2,500,000 individuals suffer from these diseases world-wide. Aes-210 is currently undergoing a randomized, double-blind, placebo-controlled Phase 2 study for the treatment of pouchitis which is an orphan disease. Initial data from that study are highly encouraging.

AesRx is named after Aesculapius (ǽs-kyә-'la-pē-us), son of Apollo. According to Greek mythology, he learned the healing arts from the centaur Chiron and was the first human physician.