4D-710 / 4D Molecular Therap - LARVOL DELTA

4DMT Reports Third Quarter 2024 Financial Results, Operational Highlights and Expected Upcoming Milestones (GlobeNewswire) - "4D-710 for Cystic Fibrosis (CF) Lung Disease: Enrollment continues in AEROW Phase 1 clinical trial....Interim data and program update from AEROW clinical trial is expected in mid-2025."

Enrollment status • P1/2 data • Cystic Fibrosis

AEROW: 4D-710 in Adult Patients With Cystic Fibrosis (clinicaltrials.gov) - P1/2 | N=40 | Recruiting | Sponsor: 4D Molecular Therapeutics | N=24 ➔ 40 | Trial completion date: Jan 2027 ➔ Jan 2030

Enrollment change • Gene therapy • Trial completion date • Cystic Fibrosis • Gene Therapies • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

4DMT Announces Presentation at the 2024 North American Cystic Fibrosis Conference (GlobeNewswire) - "4D Molecular Therapeutics...announced a poster presentation at the 2024 North American Cystic Fibrosis Conference (NACFC) in Boston, Massachusetts on September 26-28, 2024....Preclinical data for 4D-710 for cystic fibrosis lung disease in combination with CFTR modulators to be presented in a poster presentation."

Preclinical • Cystic Fibrosis

Transgene expression and CFTR channel function in human bronchial epithelial cells after addition of CFTR modulators and 4D-710 (NACFC 2024) - "Background: Treatment with elexacaftor/tezacaftor/ivacaftor (ETI) improves lung function in people with cystic fibrosis (PwCF), but ~20% of PwCF with ΔF508-minimal function genotypes have a suboptimal response [1]. Addition of 4D-710 to ETI results in robust transduction and transgene expression in HBE cells and confers an additive effect on CFTR channel function in CF HBE ALI cultures. These findings support further evaluation of 4D-710 as a therapeutic option for PwCF who have a suboptimal response to CFTR modulators."

Cystic Fibrosis • Genetic Disorders • Immunology • Respiratory Diseases

4DMT Presents Positive Interim Data from Phase 1/2 AEROW Clinical Trial of Aerosolized 4D-710 for Modulator-Ineligible/-Intolerant Cystic Fibrosis at 47th European Cystic Fibrosis Conference (GlobeNewswire) - P1/2 | N=24 | AEROW (NCT05248230) | Sponsor: 4D Molecular Therapeutics | "4D Molecular Therapeutics...announced positive interim clinical data from the 4D-710 Phase 1/2 AEROW clinical trial for the treatment of cystic fibrosis (CF) lung disease...10 participants with CF lung disease who are ineligible for or intolerant of CFTR modulator therapy have been enrolled in the Phase 1 Dose Exploration stage across four dose cohorts....4D-710 was well tolerated, with no 4D-710–related adverse events after administration, no dose-limiting toxicities, and no SAEs...Biomarker analyses demonstrated robust, consistent and widespread CFTRDR transgene mRNA and CFTR protein expression throughout all lung biopsy samples from all participants at all four dose levels: Dose-dependent CFTR∆R transgene RNA expression, with the mean % of airway epithelial cells testing positive ranging from 14% to 53%....Advancing to AEROW Phase 2 Dose Expansion stage (n= up to 9), enrollment to begin in H2 2024."

Enrollment status • P1/2 data • Cystic Fibrosis

CFTR transgene expression in airway epithelial cells following aerosolized administration of the AAV-based gene therapy 4D-710 to adults with cystic fibrosis lung disease (ECFS 2024) - "Administration of 4D-710 (1x10 15 or 2x10 15 vg) to adults with CF lung disease resulted in widespread transgene delivery and robust CFTR protein expression in all major airway cell types. CFTR protein levels exceeded levels in normal lung samples, supporting exploration of lower doses. Enrolment has been initiated for cohort 3 (5x10 14 vg)."

Clinical • Gene therapy • Cystic Fibrosis • Gene Therapies • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases • CFTR

4DMT to Present Interim Data from Aerosolized 4D-710 Phase 1/2 AEROW Clinical Trial for Cystic Fibrosis at 47ᵗʰ European Cystic Fibrosis Conference (GlobeNewswire) - "4D Molecular Therapeutics...announced that interim data from the Phase 1/2 AEROW clinical trial evaluating aerosolized 4D-710 for treatment of cystic fibrosis lung disease will be presented at the 47th European Cystic Fibrosis Conference, taking place in Glasgow, UK. The Company will host a webcast to discuss the data in detail and provide a program update on Thursday, June 6 at 8:00 a.m. ET."

P1/2 data • Cystic Fibrosis

4DMT Receives Rare Pediatric Disease Designation from FDA for Aerosolized 4D-710 for Treatment of Cystic Fibrosis Lung Disease (GlobeNewswire) - "4D Molecular Therapeutics...announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) for the Company’s product candidate aerosolized 4D-710 for treatment of cystic fibrosis (CF) lung disease. 4D-710 is being evaluated in the Phase 1/2 AEROW clinical trial in people with CF who are not eligible for, or cannot tolerate any of, the currently approved CFTR modulators....'We continue to enroll our AEROW clinical trial and work with the CF Foundation and regulators to identify the most efficient path to advance this therapy, with preliminary feedback expected this quarter.'"

Enrollment status • FDA event • Cystic Fibrosis • Genetic Disorders

4DMT Announces Update on Regulatory Interactions and Development Path for 4D-710 for Treatment of Cystic Fibrosis (GlobeNewswire) - "Phase 2 Expansion Cohort dose selection is expected in Q2 2024 based on all clinical and lung biopsy data; the Company anticipates enrolling a total of six to nine pwCF in the Phase 2 Expansion Cohort. The Company submitted an AEROW trial amendment to the Cystic Fibrosis Therapeutics Development Network (TDN) as follows: 1) to enroll pwCF with lower baseline percent predicted forced expiratory volume in 1 second (ppFEV1) (50-90%) to assess ppFEV1 response to 4D-710, and 2) to add a second lung biopsy procedure at a longer-term timepoint (12 months or later) to study long term durability of 4D-710 CFTR transgene expression and optimal timing for redosing....4DMT is preparing for initiation of a Phase 3 clinical trial in H2 2025....'Our goal is to initiate Phase 3 development in H2 2025 with 4D-710 suspension GMP process clinical trial material. We look forward to sharing interim clinical data from the AEROW Phase 1/2 clinical trial at the ECFS conference in June 2024.'"

Clinical protocol • New P3 trial • P1/2 data • Trial status • Cystic Fibrosis

4DMT Reports Full Year 2023 Financial Results and Operational Highlights (GlobeNewswire) - "4D-710 for Cystic Fibrosis Lung Disease: Update on FDA feedback on development plan for monotherapy and approved CF modulator combination regimens expected in Q1 2024. Interim update from Phase 1/2 AEROW clinical trial expected in mid-2024."

FDA event • P1/2 data • Cystic Fibrosis

4D-710 in Adult Patients With Cystic Fibrosis (clinicaltrials.gov) - P1/2 | N=24 | Recruiting | Sponsor: 4D Molecular Therapeutics | Trial completion date: Aug 2025 ➔ Jan 2027 | Trial primary completion date: Aug 2024 ➔ Dec 2026

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

4D-710: “No clinically significant AEs during aerosol administration of 4D-710”; Cystic fibrosis (4D Molecular Therapeutics) - Aerosolized 4D-710 for the Treatment of Cystic Fibrosis (CF) Lung Disease: “No inflammation observed in airway biopsies 4-8 weeks following 4D-710 dosing”

P1/2 data • Cystic Fibrosis

4DMT Presents Positive Interim Data from Phase 1/2 AEROW Clinical Trial of Aerosolized 4D-710 for Cystic Fibrosis at 2023 NACFC (GlobeNewswire) - P1/2 | N=21 | AEROW (NCT05248230) | Sponsor: 4D Molecular Therapeutics | "4D Molecular Therapeutics... announced interim data from the Phase 1/2 AEROW clinical trial evaluating aerosolized 4D-710 for treatment of cystic fibrosis lung disease. Results will also be presented at the 2023 North American Cystic Fibrosis Conference (NACFC)....Aerosolized 4D-710 was generally well tolerated with no inflammation in any lung biopsy across Cohorts 1 and 2 (1E15 & 2E15 vg; n=7) with up to 17 months follow-up....Promising, reproducible, CFTR expression significantly above normal levels across all participants and all lung tissue samples collected (n=34), substantially exceeding target profile....FDA feedback on 4D-710 development path as monotherapy and in combination with approved CF modulator therapies expected in Q1 2024....Next interim Phase 1 data update expected mid-2024."

FDA event • P1 data • Cystic Fibrosis

4DMT to Present Interim Data from Aerosolized 4D-710 Phase 1/2 AEROW Clinical Trial for Cystic Fibrosis at 2023 NACFC (GlobeNewswire) - "4D Molecular....today announced that interim data from the Phase 1/2 AEROW clinical trial of aerosolized 4D-710 for treatment of cystic fibrosis lung disease will be presented at the 2023 North American Cystic Fibrosis Conference (NACFC) and that it will host a live webcast to discuss the data in detail and provide a program update on Wednesday, November 1, 2023 at 4:30 p.m. ET....The interim data will include additional safety and clinical activity follow-up on Cohort 1 (n=3) at the 1E15 vg dose level and initial safety and biomarker data from Cohort 2 (n=3-6) at the 2E15 vg dose level."

P1/2 data • Cystic Fibrosis

4D Molecular Therapeutics Reports Second Quarter 2023 Financial Results and Operational Highlights (GlobeNewswire) - "4D-710 for Cystic Fibrosis Lung Disease: Update regarding FDA discussion on pivotal endpoints expected Q1 2024 after anticipated initial discussion with FDA in Q4 2023."

FDA event • Cystic Fibrosis • Genetic Disorders

4D-710: “Single-dose administration of 4D-710 was well tolerated”; Cystic fibrosis (4D Molecular Therapeutics) - ECFS 2023: “Clinically meaningful improvements in CFQ-R respiratory symptom score”

P1/2 data • Cystic Fibrosis

ReCode, Vertex and 4DMT Seek to Help Untreated Cystic Fibrosis Patients (BioSpace) - "The currently available CF drugs, called CFTR modulators, are designed to bind to and correct aberrant CFTR protein, said Jennifer Taylor-Cousar, who treats cystic fibrosis...'Ultimately, I think gene editing will be what will cure CF,' Taylor-Cousar said because it would allow for the possibility of permanently correcting cells' DNA, whereas other therapies are not permanent. 'But that's a long way away.'"

Media quote

4DMT Presents Positive Interim Data from Aerosolized 4D-710 Phase 1/2 AEROW Clinical Trial in Patients with Cystic Fibrosis at the ECFS 46th Annual Meeting (GlobeNewswire) - "Upcoming Expected Milestones for 4D-710: Phase 1 Dose Exploration stage (Cohort 1 & 2) interim data expected to be presented at the North American Cystic Fibrosis Conference in November 2023, Dose selection for and initiation of Phase 2 Dose Expansion stage expected in H2 2023, FDA discussion on pivotal endpoints expected in Q4 2023; 4D-710 in combination with CFTR modulators: Development plan update expected in Q4 2023."

FDA event • P1 data • Trial status • Cystic Fibrosis • Genetic Disorders

4DMT Presents Positive Interim Data from Aerosolized 4D-710 Phase 1/2 AEROW Clinical Trial in Patients with Cystic Fibrosis at the ECFS 46th Annual Meeting (GlobeNewswire) - P1/2 | N=21 | AEROW (NCT05248230) | Sponsor: 4D Molecular Therapeutics | "4D Molecular Therapeutics...announced positive interim clinical data from the 4D-710 Phase 1/2 AEROW clinical trial for the treatment of cystic fibrosis lung disease. 4D-710 is comprised of our targeted and evolved vector, A101, and a codon-optimized CFTR∆R transgene and is designed for aerosol delivery to achieve CFTR expression within lung airway epithelial cells....Following aerosol administration, 4D-710 was well tolerated in all 3 participants (9-12 months follow up) with no 4D-710 related adverse events, dose-limiting toxicities or serious adverse events....CFTR protein expression was observed by immunohistochemistry (IHC) in 92 to 99% of airway epithelial cells, including in all major epithelial cell types, including basal cells, goblet cells and ciliated columnar cells."

P1/2 data • Cystic Fibrosis • Genetic Disorders

AAV mediated gene therapy for cystic fibrosis: interim results from a phase 1/2 clinical trial (ECFS 2023) - P1/2 | "Initial data from a Phase 1/2 clinical trial evaluating 4D-710 adults with CF lung disease demonstrated evidence of tolerability and successful transgene delivery and expression in the lung. Additional data from cohort 2 will be presented."

Clinical • Gene therapy • P1/2 data • Cystic Fibrosis • Fibrosis • Gene Therapies • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases • CFTR

4DMT to Present Interim Data from 4D-710 Phase 1/2 AEROW Clinical Trial for Cystic Fibrosis Lung Disease at the European Cystic Fibrosis Society (ECFS) 46th Annual Meeting (GlobeNewswire) - P1/2 | N=21 | AEROW (NCT05248230) | Sponsor: 4D Molecular Therapeutics | "4D Molecular Therapeutics....today announced an oral presentation of the interim data from the 4D-710 Phase 1/2 AEROW clinical trial for the treatment of cystic fibrosis lung disease at the European Cystic Fibrosis Society 46th Annual Meeting held in Vienna, Austria on June 7-10, 2023....Clinical data will focus on safety, tolerability, pulmonary function testing (percent predicted FEV1) and respiratory-related quality of life assessments (Cystic Fibrosis Questionnaire-Revised or CFQ-R, Respiratory Domain Score). The presentation will also include biomarker data on expression of the cystic fibrosis transmembrane regulator (CFTR) transgene protein from the endobronchial lung biopsies & brushings collected at week 4-8 after dosing."

Biomarker • P1/2 data • Cystic Fibrosis

4DMT Announces Presentation on Aerosolized 4D-710 for Treatment of Cystic Fibrosis at the American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting (BioSpace) - "The oral presentation will be given by Jennifer L. Taylor-Cousar, M.D...The presentation will include biomarker data on expression of the cystic fibrosis transmembrane regulator (CFTR) transgene protein from the endobronchial lung biopsies & brushings collected at week 4-8 for patients in Cohort 1 (n=3; dose 1E15 vg) of the Phase 1/2 AEROW clinical trial. In situ hybridization biomarker data, demonstrating transgene RNA expression from lung samples from Cohort 1 patients, was previously presented by Dr. Taylor-Cousar at the North American Cystic Fibrosis Conference (NACFC), November 3, 2022."

4DMT Announces Presentation on Aerosolized 4D-710 for Treatment of Cystic Fibrosis at the American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting (GlobeNewswire) - "4D Molecular Therapeutics...today announced a presentation on 4D-710 for the treatment of cystic fibrosis lung disease at the American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting held in Los Angeles, California on May 16-20, 2023....The presentation will include biomarker data on expression of the cystic fibrosis transmembrane regulator (CFTR) transgene protein from the endobronchial lung biopsies & brushings collected at week 4-8 for patients in Cohort 1 (n=3; dose 1E15 vg) of the Phase 1/2 AEROW clinical trial....Additional clinical and biomarker data for Cohort 1 patients (n=3; 9-12 months follow-up) are expected to be presented at the European Cystic Fibrosis Conference in June 2023."

Biomarker • P1/2 data • Cystic Fibrosis • Genetic Disorders

AAV mediated gene therapy for cystic fibrosis (4D-710) (ASGCT 2023) - No abstract available

Gene therapy • Cystic Fibrosis • Fibrosis • Gene Therapies • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

4D Molecular Therapeutics Reports Full Year 2022 Financial Results and Operational Highlights (GlobeNewswire) - "4D-710 for Cystic Fibrosis Lung Disease: Present interim data from the Dose Exploration stage (Cohorts 1 and 2) of the Phase 1/2 clinical trial at a scientific conference in Q2. Update on development plan for modulator combination in 2H."

P1/2 data • Cystic Fibrosis • Genetic Disorders