AesRx, LLC, a biopharmaceutical company dedicated to the development of treatments for two orphan diseases, has commenced a clinical trial of Aes-103, the company’s novel anti-sickling agent.

Aes-103, designated as an orphan drug by the US Food and Drug Administration (FDA), is a first-in-class, orally bioavailable small molecule therapeutic under investigation for the treatment of sickle cell disease (SCD). This trial will examine the safety and tolerability of Aes-103 in healthy volunteers as well as the drug’s pharmacokinetic and pharmacodynamic properties.

Aes-103’s proposed mechanism of action is targeted to reduce cell sickling. AesRx believes it is the only anti-sickling drug currently in human trials. The present trial is part of an ongoing collaboration between AesRx and two separate components of the National Institutes of Health (NIH)—the National Heart, Lung, and Blood Institute (NHLBI) and the Therapeutics for Rare and Neglected Diseases (TRND) programme. The collaboration is planned to develop Aes-103 through completion of initial proof of principle trials.

“We are obviously delighted with today’s announcement,” commented Stephen R Seiler, AesRx’s chief executive officer. “Aes-103 represents an attractive development opportunity in our view because the proposed mechanism of action has been widely studied and X-ray crystallography indicates it binds a target of interest on sickle haemoglobin. This trial marks an important milestone in the development of Aes-103. We are extremely gratified to have had support from the NIH to advance the research on this compound.”

“We are very excited to see Aes-103 being studied in a clinical trial,” added Lanetta B Jordan, MD, MPH, MSPH, chief medical officer of the Sickle Cell Disease Association of American and a member of AesRx’s Strategic Advisory Board. “If successful, it would represent a breakthrough in the treatment of sickle cell disease. Although SCD was first discovered over 100 years ago, there has never been a drug developed specifically to treat SCD and there is currently no known drug that directly blocks the sickling which causes the morbidity and mortality associated with the disease. We look forward to the further development of this important therapeutic.”

The National Institutes of Health (NIH) is the nation's medical research agency. It is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases.

The NHLBI plans, conducts, and supports research related to the causes, prevention, diagnosis, and treatment of heart, blood vessel, lung, and blood diseases; and sleep disorders.

TRND is overseen by the NIH Centre for Translational Therapeutics (NCTT), which is administered by the National Human Genome Research Institute, another component of the NIH. NCTT’s Bridging Interventional Development Gaps (BrIDGs) programme also contributed research material to develop Aes-103.