According to the company, the study found a positive safety and tolerability profile for aCT1 for treatment of corneal injury.
Xequel Bio Inc. has announced positive results from its Phase 1b clinical trial with aCT1 ophthalmic solution (iNexin) for the treatment of corneal injury in patients with dry eye disease. The study demonstrated that iNexin was safe and well-tolerated at the doses tested, and early efficacy signals were observed.
According to the company this Phase 1b trial was a single-center, randomly assigned, double masked, vehicle-controlled clinical study to assess the safety and exploratory efficacy of aCT1 for the treatment of corneal injury in patients with dry eye disease. There were no serious adverse events or dose-limiting toxicities associated with aCT1 treatment. Improvements in both signs (corneal staining) and symptoms (ocular discomfort) of dry eye disease were observed within two weeks of treatment in both the environment and Ora’s controlled adverse environment (CAE) model, which exacerbates signs and symptoms of dry eye disease in a safe and standardized manner for greater precision in predicting treatment efficacy.
Jerry St. Peter, CEO of Xequel, noted that the results support the company’s belief that aCT1 ophthalmic solution can improve patient outcomes by regulating the inflammatory response to promote improved ocular healing.
“We are extremely pleased that the key goals of this study were met, providing clinical evidence of safety, tolerability, and also early signs of efficacy in patients with corneal injury,” he said in a statement. “This data provides a strong basis to further refine our therapeutic focus and dosing regimen as we progress our ophthalmic development program, including designing our Phase 2 clinical trial.”
Moreover, Gail Torkildsen, MD, board certified ophthalmologist at Andover Eye Associates, noted that this study provides an initial look at the safety, tolerability, and potential biological activity of aCT1 in treating dry eye disease, with a mechanism of action suggesting additional application in treating ocular injury and inflammation.
“The results are encouraging and indicate the potential for aCT1 to restore Connexin43, which plays a critical role in the inflammatory cascade,” Torkildsen said in a statement. “iNexin could represent an innovative solution to treat patients with corneal injuries by promoting healing and rebuilding healthy tissue through accelerated re-epithelialization.”
This Phase 1b trial was a single-center, randomly assigned, double masked, vehicle-controlled clinical study to assess the safety and exploratory efficacy of aCT1 compared to Vehicle in patients with dry eye disease. A total of 36 participants, with a subject-reported history of dry eye disease in both eyes, were enrolled and randomly assigned in a 2:1 allocation for each of three concentrations of aCT1 (0.08%; 0.4%; 2.0% aCT1) to Vehicle (eye drop formulation without aCT1), administered bilaterally twice a day for 14 days. The primary objective of the trial was to evaluate the safety and tolerability of aCT1. The secondary objective was to compare the efficacy of aCT1 to Vehicle for the treatment of the signs and symptoms associated with dry eye disease.
This trial was managed and monitored by Ora, Inc., a leading full-service ophthalmic drug and device development firm, and was supported by the Department of Defense Defense Health Program, Congressionally Directed Medical Research Program through Vision Research Program Award W81XWH-20-1-0879.
Xequel’s aCT1 technology platform
Xequel Bio’s aCT1 (alpha-Connexin carboxyl-Terminal 1 peptide) technology platform is designed to develop drugs that will enable physicians to better manage a variety of indications involving inflammation and the body’s response to injury. aCT1 is a patented new chemical entity, based on the C-terminal sequence of Connexin43, designed to selectively and reversibly inhibit protein binding of endogenous Connexin43 to key binding partners.
The company noted that Connexin43 plays critical roles in multiple aspects of the injury response, including spread of injury signals, extravasation of immune cells, granulation tissue formation and fibrosis. aCT1’s unique, targeted mechanism of action has been demonstrated to restore the coordination of cellular communication, reinforce junctional integrity and temper excessive inflammatory responses in injured tissues for optimal injury response and tissue repair. aCT1 is currently in clinical development for multiple indications across dermatology and ophthalmology, as well as in ongoing preclinical research in pulmonology.