The U.S. Food and Drug Administration (FDA), has designated Aes-103 as an orphan drug for the treatment of sickle cell disease. Aes-103 is intended to reduce blood cell sickling, a condition in which the normal doughnut-round shape of a red blood cell is distorted in to a sickle-shape because of an inherited defect in the oxygen-carrying protein hemoglobin.
Aes-103 is believed to be the only clinical-stage therapeutic that directly targets cell sickling.
Sickle cell disease is a recessive disorder of the hemoglobin which 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 are afflicted with sickle cell disease.
TRND and AesRx are currently collaborating to complete all pre-clinical development activities needed for the FDA to review an investigational new drug (IND) application for Aes-103. Once the IND goes into effect, clinical studies in healthy volunteers and sickle cell patients will be conducted next year by the intramural research program of the NHLBI. Researchers will conduct the trials in the NIH Clinical Center on the Bethesda campus. The Aes-103 development program has previously benefitted from two NIH grants, a Rapid Access to Interventional Development (RAID) grant and a Small Business Innovation Research (SBIR) grant.
“We are delighted to have the opportunity to work with the NIH,” said Stephen R. Seiler, AesRx’s Chief Executive Officer. “Aes-103 represents an attractive development opportunity in our view because its mechanism of action has been widely studied and X-Ray crystallography indicates that it binds a target of interest on sickle hemoglobin. Aes-103 could be a breakthrough in the treatment of sickle cell disease. We are pleased that the NIH has chosen to collaborate with us to move it into the clinic.”
“Seeing Aes-103 reach sickle cell patients would be very exciting,” added Lanetta B. Jordan, M.D., M.P.H., M.S.P.H., chief medical officer of the Sickle Cell Disease Association of America and a member of AesRx’s Strategic Advisory Board. “A therapy such as Aes-103 could provide an important new treatment option for patients afflicted with this disease.”
Aes-103 is one of the first molecules to enter development in the TRND program. Congress funded TRND (trnd.nih.gov) to speed the development of new medications that might otherwise be ignored by industry because they would not be sufficiently profitable. TRND provides drug development expertise and resources required to produce compounds that can be tested in people, including medicinal chemistry needed to turn the compound into a substance that can be used in the body and pre-clinical safety testing.
“TRND collaborates with researchers and companies to turn promising molecules into potential new drugs for rare and neglected diseases,” said NHGRI’s Christopher P. Austin, M.D., director of the NIH TRND program. “But this is the most difficult part of drug development, and the failure rate is high for any molecule that we are trying to turn into a medicine. It’s important to remember that the ultimate outcome for any compound is hard to predict.”
If the pre-clinical development and safety testing goes well enough for the FDA to issue an IND, tests of the sickle disease treatment will move to the NIH Clinical Center for trials with human volunteers.
“Patients with sickle cell disease suffer a great deal from pain and organ damage, and our research group is committed to finding effective new treatments. Based on our extensive review of the data, we believe there is value in exploring Aes-103 as a potential treatment for sickle cell patients,” said Gregory Kato, M.D., of the cardiovascular and pulmonary branch of the NHLBI’s Division of Intramural Research and the principal investigator for the planned clinical trials.
Note: References to the National Institutes of Health, its programs or its staff, should not be viewed as an endorsement or implied endorsement of AesRx, its products or services. Additional information about sickle cell disease is available at: Sickle Cell Anemia, http://www.nhlbi.nih.gov/health/dci/Diseases/Sca/SCA_WhatIs.html Sickle Cell Disease, http://www.cdc.gov/NCBDDD/sicklecell/index.html
About AesRx
AesRx is a biopharmaceutical company dedicated to the development of treatments for two orphan diseases. The Company’s lead program (Aes-103) is targeted to the treatment of sickle cell disease. Sickle cell disease is a recessive disorder of the hemoglobin which 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 are afflicted with sickle cell disease. Aes-103 works by increasing the affinity of sickle hemoglobin for oxygen. Because only red blood cells with no bound oxygen will sickle, increasing the ability of the sickle red blood cells to bind oxygen reduces the number of cells that can sickle. AesRx is completing the pre-clinical development program for Aes-103 and expects to begin human clinical trials in 2011. AesRx’s second development program, Aes-210, is targeted to treat certain inflammatory diseases of the lower intestine, including distal ulcerative colitis, pouchitis and radiation induced proctitis.
