US-based start-up Cystetic Medicines has announced the initiation of a Phase 1 SAD/MAD trial of its CM001 dry powder “molecular prosthetic” in healthy volunteers. The company is developing CM001 for the treatment of cystic fibrosis and said that it plans to evaluate the DPI in CF patients later this year. CM001 is based on technology developed by Martin Burke of the University of Illinois at Urbana-Champaign using a small molecule that “can form ion channels that serve as replacements for the CFTR protein in cultured primary airway epithelia from people with CF caused by mutations that cannot benefit from modulators.”
Cystetic Chief Technology Officer Jeffry Weers explained, “The concept of molecular prosthetics has the potential to alter the treatment landscape for cystic fibrosis in a profound way; in theory, it should work for all people dealing with the disease, regardless of the type of mutation they present. Our team is particularly optimistic with regard to the proprietary inhaled formulation CM001, which allows for efficient targeted delivery directly to the lungs, thereby potentially avoiding side effects and enabling optimal dosing regimens.”
Burke commented, “The initiation of our Phase 1 trial for CM001 represents an important step forward in our mission to find a treatment for everyone with CF, especially the final 10 percent of people with CF who cannot benefit from CFTR modulators. Based on encouraging pre-clinical results and initial biomarker studies in the noses of people with CF not on modulators, we are excited about the potential for this molecular prosthetic approach to restore anion channel function in a way that is independent of CFTR.”
Read the Cystetic Medicines press release.
