AesRx, LLC, a biopharmaceutical company dedicated to the development of treatments for two orphan diseases, has begun a phase 1/2a clinical trial of its novel Aes-103 anti-sickling agent in patients with sickle cell disease (SCD). This trial, which marks an important milestone in the development of Aes-103 as a treatment for sickle cell disease, is part of an ongoing collaboration between AesRx and the National Institutes of Health (NIH).
The trial is being conducted at the NIH Clinical Center in Bethesda, Maryland. The AesRx/NIH collaboration involves two separate NIH components—the National Heart, Lung, and Blood Institute (NHLBI) and the Therapeutics for Rare and Neglected Diseases (TRND) programme. TRND is part of the National Center for Advancing Translational Sciences (NCATS). TRND and AesRx worked together to complete in less than a year all pre-clinical development needed for the FDA to allow Aes-103 as an investigational new drug and proceed with human clinical trials.
“The commencement of clinical trials in sickle cell patients marks a major step forward for Aes-103,” commented Stephen R. Seiler, AesRx’s Chief Executive Officer. “If successful, the Aes-103 programme will represent a breakthrough in the treatment of sickle cell disease. While sickle cell patients currently have several treatment options, the lack of therapies that can prevent cell sickling represents a major unmet medical need.”
Aes-103 has been designated as an orphan drug by the US Food and Drug Administration. It is a first-in-class, orally bioavailable small molecule for the treatment of SCD. AesRx believes it is the only drug currently in human trials that directly blocks cell sickling. While red blood cells are normally round, people who have sickle cell disease produce crescent-shaped cells, which can cause blood flow problems. The present trial will examine the safety and tolerability of Aes-103 in stable sickle cell disease patients as well as the drug’s pharmacokinetic and pharmacodynamic properties.
“This study represents a crucial effort to translate basic science findings into a new treatment for sickle cell disease,” said Gregory Kato, M.D., a hematologist with the NHLBI and principal investigator of the Aes-103 study.
“The TRND program has made significant contributions to the development of Aes-103 as a potential treatment for those suffering from sickle cell disease,” said Christopher P. Austin, M.D., director of the Division of Pre-Clinical Innovation at NCATS. “This project epitomizes both the urgent and neglected medical needs, and the innovative scientific and collaborative approaches to solving them, that are TRND’s mission.”
Sickle cell disease is a recessive disorder of the haemoglobin which can lead to a wide range of serious, sometimes life-threatening, conditions including: chronic hemolytic anaemia, chronic pain and acute painful crisis, stroke, acute chest syndrome, and cumulative damage to tissues and organs. More than 75,000 people in the United States, and 13 million individuals worldwide, are afflicted with sickle cell disease.