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First Person With Cystic Fibrosis Dosed In Phase 1 Trial Of Investigational Therapy Cm001, Designed To Form Ion Channels To Help Restore Airway Function

CHAMPAIGN, Ill., Oct. 30, 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 it has dosed the first person with cystic fibrosis (CF) as part of an ongoing Phase 1 clinical trial (NCT05802264) of its investigational inhalable small molecule therapy CM001, designed as a molecular prosthetic to restore ion channels in the airway tissues of people with CF. Martin Burke, M.D., Ph.D, co-founder of cystetic Medicines, will also report single-ascending dose data of the ongoing CM001 Phase 1 trial at an oral podium presentation Nov. 3 at the North American Cystic Fibrosis Conference (NACFC) in Phoenix, AZ, "Session S-14: CM001, a clinical stage molecular prosthetic for CFTR that may benefit all people with CF."

"Dosing the first person with CF represents a critical milestone in our mission to help find a treatment for everyone with CF, including the final 10 percent of people with CF who cannot benefit from CFTR modulators," said Dr. Burke. "We are grateful to all the volunteers who participated in the Phase 1 trial and look forward to continuing to work with the CF community as we transition our focus to studying CM001 in people with CF."

cystetic Medicines will also present both clinical (Phase 1 Part A – SAD in healthy volunteers) and preclinical data on CM001 via two posters at the conference: Poster #255 entitled: "Amphotericin B Cystetic for Inhalation (CM001): A randomized, double-blind, placebo-controlled, single ascending dose study in healthy volunteers" on November 3, 2023, from 11:40 pm to 12:40 pm, and Poster #282 entitled "Understanding the role of cholesterol in optimizing molecular prosthetics for cystic fibrosis" will be presented on November 3, 2023 from 12:40 pm to 1:40 pm.

The randomized, double-blind, placebo-controlled Phase 1 study (Parts A and B) evaluated the safety, tolerability, and pharmacokinetics of single-ascending (Part A) and then multiple-ascending (Part B) doses of CM001 administered with a portable dry powder inhaler to healthy volunteers. All participants in the Phase 1 Parts A and B portion of the trial, which was conducted in New Zealand, have completed enrollment. The Phase 1 Part C clinical trial will continue in Australia and New Zealand with a multiple dose component in people with CF. These studies are being conducted with the support of DevPro Biopharma LLC.

Emily Kramer-Golinkoff, MBE, Co-Founder of Emily's Entourage, stated, "We are thrilled with the completion of the Phase 1 trial of CM001 in healthy volunteers and excited to see it progress to people with CF. Emily's Entourage is proud to have helped launch this molecular prosthetics approach with the potential to benefit everyone with CF, including those of us in the final 10 percent. We are deeply grateful for the tireless efforts and passion of the team at cystetic Medicines to lead this important work."

About Cystic Fibrosis (CF)

CF is a progressive, autosomal recessive chronic disease that affects approximately 80,000 people worldwide. It is caused by loss-of-function mutations to the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which produces a key protein responsible for moving anions from the inside of cells to the outside. CF is 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.

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. There is currently no disease-modifying treatment for these individuals.

About CM001

CM001 is an investigational small molecule therapeutic that self assembles to form ion channels in airway epithelial cell membranes. CM001 has been developed as a dry powder for inhalation.

Preclinical 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. 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 similar to the effects seen with modulator therapy in responsive individuals.

CM001 is currently in clinical development to test the hypothesis that targeted delivery of the molecular prosthetic to the lungs will correct anion transport independently of CFTR mutation status and therefore has the potential to reverse the underlying pulmonary defect in CF, including in those 10% of people with CF who lack the CFTR substrate to respond to modulator drugs.

About cystetic Medicines

cystetic Medicines is a clinical-stage biotechnology company pioneering the development of small molecule anion channel molecular prosthetics to restore pulmonary function in people with CF. The company was founded in 2020, 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 CF. 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.

SOURCE cystetic Medicines, Inc.

Posted: October 2023

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