Xequel Bio, Inc., a clinical stage biopharmaceutical company developing ophthalmic and dermatologic therapeutics utilizing its patented new chemical entity aCT1 (alpha-Connexin carboxyl-Terminal 1 peptide), announced positive results from its Phase 1b clinical trial with iNexin™ (aCT1 ophthalmic solution) 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.
This Phase 1b trial was a single-center, randomized, double masked, vehicle-controlled clinical study to assess the safety and exploratory efficacy of iNexin for the treatment of corneal injury in patients with dry eye disease. There were no serious adverse events or dose-limiting toxicities associated with iNexin treatment. Improvements in both signs (e.g., corneal staining) and symptoms (e.g., 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.
“These results support our belief that iNexin, our novel aCT1 ophthalmic solution, has the potential to improve patient outcomes by regulating the inflammatory response to promote improved ocular healing,” said Jerry St. Peter, Chief Executive Officer. “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. 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.”
Dr. Gail Torkildsen, MD, board certified ophthalmologist at Andover Eye Associates, said, “This study provides an initial look at the safety, tolerability, and potential biological activity of iNexin 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. 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, randomized, double masked, vehicle-controlled clinical study to assess the safety and exploratory efficacy of iNexin 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 randomized in a 2:1 allocation for each of three concentrations of iNexin (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 iNexin. The secondary objective was to compare the efficacy of iNexin 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.
About 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. 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.
About Xequel Bio, Inc.
Xequel Bio, Inc. is a clinical stage biopharmaceutical company advancing its proprietary aCT1 (alpha-Connexin carboxyl-Terminal 1 peptide) technology platform 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 currently in development for multiple indications. The Company’s lead clinical programs include Granexin® gel in dermatology and iNexin™ ophthalmic solution in ophthalmology. The Company also has ongoing preclinical research in pulmonology. For more information, please visit www.xequel.com.
About Ora, Inc.
Ora is the world’s leading full-service ophthalmic drug and device development firm. For over 40 years, their expert teams have proudly helped clients across the globe earn more than 55 product approvals. Ora’s unique methodologies and strategies have been proven across thousands of programs to maximize the likelihood of success and efficiently bring new products from concept to market. For more information, please visit www.oraclinical.com.