amphotericin B cystetic for inhalation (CM001) / cystetic Medicines - LARVOL DELTA

Study to Assess Amphotericin B Cystetic for Inhalation (ABCI) Doses in Healthy Volunteers & People with Cystic Fibrosis (clinicaltrials.gov) - P1 | N=108 | Recruiting | Sponsor: Cystetic Medicines, Inc. | Trial completion date: Dec 2024 ➔ Dec 2025 | Trial primary completion date: Dec 2024 ➔ Dec 2025

Trial completion date • Trial primary completion date • Cystic Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

Molecular prosthetics and CFTR modulators additively increase host defenses in cystic fibrosis airway epithelia (NACFC 2024) - "Two days before experimentation, CuFi-1(ΔF508/ΔF508) or CuFi-4 (G551D/ΔF508) cells were pretreated with one of four conditions: perfluorocarbon-72 (vehicle); a combination of elexacaftor (3 µM or 1 µM), tezacaftor (3 µM or 1 µM), ivacaftor (1 µM or 0.33 µM), and forskolin (1 µM or 0.33 µM) (ETI/fsk); the optimized formulation of AmB (ABCI-003, 2 µM) currently in clinical trials; or a combination of ETI/fsk and ABCI-003... Combined treatment with molecular prosthetics and CFTR modulators may provide additive benefits in restoring airway host defenses in PwCF."

Cystic Fibrosis • Genetic Disorders • Immunology • Respiratory Diseases

Inhaled amphotericin shows good safety and tolerability in Phase 1 single-ascending-dose and multiple-ascending-dose studies in healthy subjects (NACFC 2024) - "CM001 was safe and well tolerated in healthy subjects, with no dose-limiting toxicities observed in up to 28 days of dosing. Pharmacokinetic results support the potential for once-daily administration and indicate that inhaled CM001 is associated with low circulating levels of AmB while achieving concentrations in the ASL above those Table 1 (abstract 286): TEAEs observed for inhaled AmB in Phase 1 SAD/MAD studies in required for ion channel activity. Based on these data, CM001 was advanced for further evaluation in adults with CF (Part C of Study CM001001)."

Clinical • P1 data • Cystic Fibrosis • Genetic Disorders • Hematological Disorders • Immunology • Respiratory Diseases • CFTR

Study to Assess Amphotericin B Cystetic for Inhalation (ABCI) Doses in Healthy Volunteers & People With Cystic Fibrosis (clinicaltrials.gov) - P1 | N=84 | Recruiting | Sponsor: Cystetic Medicines, Inc. | Phase classification: P1b ➔ P1

Phase classification • Cystic Fibrosis • Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

CYSTETIC MEDICINES DOSES FIRST PERSON WITH CYSTIC FIBROSIS IN PHASE 1 TRIAL OF INVESTIGATIONAL THERAPY CM001, DESIGNED TO FORM ION CHANNELS TO HELP RESTORE AIRWAY FUNCTION (PRNewswire) - "cystetic Medicines...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...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...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'...and Poster #282 entitled 'Understanding the role of cholesterol in optimizing molecular prosthetics for cystic fibrosis'..."

P1 data • Preclinical • Trial status • Cystic Fibrosis

Amphotericin B Cystetic for Inhalation (CM001): A randomized, double-blind, placebo-controlled, single ascending dose study in healthy volunteers (NACFC 2023) - "The last patient, last visit for this study is anticipated in August 2023."

Clinical • Hematological Disorders • CFTR

Study to Assess Amphotericin B Cystetic for Inhalation (ABCI) Doses in Healthy Volunteers & People With Cystic Fibrosis (clinicaltrials.gov) - P1b | N=84 | Recruiting | Sponsor: Cystetic Medicines, Inc. | Phase classification: P2a ➔ P1b

Phase classification • Cystic Fibrosis • Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

cystetic Medicines Initiates Phase 1 Clinical Trial of a Molecular Prosthetic for Cystic Fibrosis (PRNewswire) - "Data from Phase 1 study expected in Q4 of 2023...cystetic Medicines...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)...Studies have begun in healthy volunteers, and the company intends to begin treating people with CF in the second half of this year."

P1 data • Trial status • Cystic Fibrosis