Novel approach could treat the final 10% of people with CF that cannot benefit from CFTR modulators
Data from Phase 1 study expected in Q4 of 2023
CHAMPAIGN, Ill., June 26, 2023 – cystetic Medicines, Inc., a clinical-stage biotechnology company developing small molecule channels that serve as molecular prosthetics for missing or dysfunctional CFTR protein channels, announced today that the first healthy volunteer has been dosed with CM001 in a Phase 1 clinical trial. CM001 is a molecular prosthetic intended to treat people with cystic fibrosis (CF).
“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,” said Martin Burke, M.D., Ph.D, co-founder of cystetic Medicines. “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.” cystetic Medicines was co-founded in 2020 by Burke, a chemist at the University of Illinois at Urbana-Champaign, and Michael Welsh, M.D., a physician-scientist at the University of Iowa.
Emily Kramer-Golinkoff, Founder of Emily’s Entourage stated, “I am profoundly proud and excited to see that the strategic investment made by Emily’s Entourage to enable key early-stage research into the molecular prosthetics approach has yielded this promising new drug candidate that might benefit everyone with CF, including those of us in the final 10 percent. We are deeply grateful for the tireless work, passion, and talent of the team at cystetic Medicines that made this possible.”
The randomized, double-blind, placebo-controlled study will evaluate the safety, tolerability, and pharmacokinetics of single-ascending and then multiple-ascending doses of CM001 administered with a portable dry powder inhaler. Studies have begun in healthy volunteers, and the company intends to begin treating people with CF in the second half of this year. The trial is being conducted in New Zealand and is supported by DevPro Biopharma LLC.
CF is caused by loss-of-function mutations in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR) anion channel, which contributes to the chronic lung infection and inflammation that is characteristic of the disease. While currently available CFTR modulators can partially restore mutated CFTR protein function in many people with CF, approximately 10 percent of this community has mutations that result in little to no CFTR protein production and thus cannot benefit from these treatments. Recent studies have demonstrated that the active pharmaceutical ingredient in CM001 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. And an investigator-initiated clinical study demonstrated that this same compound can change nasal potential difference in people with CF not on modulators in a manner that was similar to the effects seen with modulator therapy in responsive individuals.
“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,” said Jeffry Weers, Chief Technology Officer of cystetic Medicines. “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.”
CM001 is an investigational small molecule therapeutic that self assembles to form ion channels in airway epithelial cell membranes. CM001 is intended for people with cystic fibrosis who are not currently being treated with cystic fibrosis transmembrane regulator (CFTR) modulator therapies. To minimize treatment burden, CM001 has been developed as a dry powder for inhalation.
About Cystic Fibrosis (CF)
CF is a progressive, autosomal recessive chronic disease that affects approximately 80,000 people worldwide. It is cause by mutations to the cystic fibrosis transmembrane conductance regulator (CFTR) gene, that produces a key protein responsible for moving anions from the inside of cells to the outside. CF is characterized as a progressive disease that causes severe damage to the pulmonary system. Symptoms of CF are debilitating and include the buildup of mucus in the lungs, persistent infections, and decreasing lung function.
The majority of CFTR mutations are processing or gating mutations that result in misfolded or dysfunctional protein. CFTR ‘modulators’ and ‘correctors’ currently exist with the ability to improve lung function in the individuals with these types of CFTR mutations. However, there remain about 10% of people with CF who have Class 1 or other mutations that result in an inability to synthesize full length CFTR and, therefore, lack the substrate for modulators or correctors. There is currently no disease-modifying treatment for these individuals. For more information, please visit www.emilysentourage.org.
About cystetic Medicines
cystetic Medicines is a clinical-stage biotechnology company pioneering the development of small molecule anion channel prosthetics or “molecular prosthetics” to restore protein function in people with CF. The company is based on the work of Dr. Martin Burke of the University of Illinois at Urbana-Champaign and Dr. Michael Welsh at the University of Iowa and is currently focused on the development of CM001, a novel inhalable small molecule therapeutic designed to restore ion balance and thus host defenses in the lungs to treat cystic fibrosis. The approach utilizes a state-of-the-art proprietary inhaled dry powder formulation that enables improved dose consistency and high efficiency delivery to the airways. Learn more at www.cysteticmedicines.com.
Investor Contact: cystetic Medicines, Inc., IR@cysteticmedicines.com
Media Contact: Anthony Karamourtopoulos, APCO Worldwide, email@example.com