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.
4D MOLECULAR THERAPEUTICS PHONE TRIAL
Taylor-CousarĮMERYVILLE, Calif., J(GLOBE NEWSWIRE) - 4D Molecular Therapeutics (Nasdaq: FDMT, 4DMT, or the Company), a clinical-stage biotherapeutics company harnessing the power of directed evolution for targeted genetic medicines, today announced positive interim clinical data from the 4D-710 Phase 1/2 AEROW clinical trial for the treatment of cystic fibrosis lung disease.
Dose selection and initiation of Phase 2 Dose Expansion stage expected in H2 2023 additional interim data including for cohort 2 participants (2E15 vg dose) expected at the North American Cystic Fibrosis Conference in November 2023.Bronchoscopy sample results demonstrated widespread and consistent expression of the cystic fibrosis transmembrane conductance regulator (CFTR) transgene protein in 92-99% of lung airway cells, at levels significantly above normal control lung tissues.Percent predicted forced expiratory volume in one second (ppFEV 1 ) meaningfully improved in participant with moderate ppFEV 1 impairment at baseline ppFEV 1 maintained stable in participants with normal or mild impairment at baseline.Quality of life outcomes measured by Cystic Fibrosis Questionnaire-Revised respiratory symptom score (CFQ-R-R) meaningfully improved for all three participants.Reported data from cohort 1 (n=3, 1E15 vg dose) of cystic fibrosis modulator treatment ineligible/intolerant participants with 9-12 months follow-up.For adult participants only, the contralateral eye may also be dosed with 4D-125 as a single dose, IVT injection provided the subject is eligible and provides consent. Once the dose level has been selected, the Phase 2 Dose Expansion Cohort will be opened to dose an additional 6-12 adult patients, and includes a pediatric sub-population of up to 6 patients. Any cohort may be expanded by an additional 3 subjects (to a maximum of 12 patients) to provide additional safety information and/or to confirm the selected dose for expansion. The cohort will be expanded in the event of a dose limiting toxicity (DLT). In Phase 1, up to 2 dose exploration cohorts will be enrolled each cohort will initially recruit up to 3 patients to receive a single uniocular IVT injection of 4D-125 in a standard 3+3 design. Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information Secondary endpoints will assess preliminary efficacy measures over time after 4D-125 administration. After receiving 4D-125, patients will be followed for 24 months with continued safety follow-up and 36 additional months of long-term follow-up. The study will also evaluate the safety and tolerability, as assessed by frequency and severity of ocular and systemic adverse events, as well as preliminary clinical efficacy of a single intravitreal (IVT) injection of 4D-125 at two dose levels in this patient population in one or both eyes (the contralateral eye dose provided the subject is eligible and provides consent).ĤD-125 has been developed as a gene replacement therapy for XLRP. This Phase 1/2 study will gather data in an observational phase Natural History Cohort to further characterize and evaluate natural disease progression in male patients with genetically-confirmed X-linked retinitis pigmentosa (XLRP) caused by mutations in the gene encoding retinitis pigmentosa GTPase regulator (RPGR). Why Should I Register and Submit Results?.
0 kommentar(er)
