4D Molecular Therapeutics (4DMT), a leading biotechnology company, is gearing up for a breakthrough phase 3 trial of its inhaled gene therapy, 4D-710, as a drug may result in patients with cystic fibrosis (CF) refractory to CFTR modulators. This eagerly anticipated trial, scheduled to begin in late 2025, could pave the way for regulatory approval of this novel treatment.
In recent years, CFTR modulators have revolutionized the treatment of CF for many patients. But there is still a small group of individuals whose disease-causing mutations do not respond to these drugs or who experience intolerable side effects Enter 4D-710, a promising gene therapy intended for consumption address this unmet medical need.
CF is caused by mutations in the CFTR gene, resulting in abnormally thick and active urine that causes a variety of symptoms and complications 4D-710 aims to cause a sitting copy of the CFTR gene apples will enter lung cells, allowing them to produce a functional CFTR protein. This new approach holds great potential for individuals who cannot benefit from existing therapies.
David Kirn, MD, co-founder, and CEO of 4DMT, expressed hope for fruitful dialogue with legislators in the United States and Europe. This discussion has provided a clear road map for 4D-710 and its acceptability. Kirn believes 4D-710 could be a transformational treatment for people with significant unmet medical need in CF lung disease.
The phase 3 trial, which will enroll 60 to 80 CF patients with primary lung function, will investigate the effect of 4D-710 on the predicted percent forced expiratory volume per second (ppFEV1), 1999; on the primary measure of pulmonary function. Additionally, the trial will use the Cystic Fibrosis Questionnaire Revised Respiratory Domain (CFQ-R-RD) to assess changes in patients’ quality of life and monitor the frequency of wheezing exacerbations.
4DMT has already established facilities to manufacture 4D-710 for further testing, and pursues construction of options for commercialization if the treatment receives Company-mandated regulatory approval for successful development of 4D-710 his commitment to meeting the immediate needs of CF patients.
Notably, 4D-710 recently received FDA pediatric rare drug designation, underscoring its potential to meet critical therapeutic needs in pediatric CF patients. In addition, 4DMT is exploring the possibility of expediting approval for 4D-710, which would allow the therapy to be commercialized based on promising preliminary clinical data, and additional trials are needed to confirm efficacy.
Encouraging early results from the Phase 1/2 AEROW clinical trial showed encouraging results. Preliminary biopsy data from seven patients treated with 4D-710 showed a significant increase in CFTR protein levels, with no serious safety concerns reported During the course of the trial, 4DMT has been in order to present interim data on all patients treated with 4D-710 at the upcoming 47th European Cystic Fibrosis Meeting in June is, as well as updates on treatment progress and as it can be applied to CF patients receiving modulator therapy.
In conclusion, 4D-710 represents a promising advance in the treatment of CF, offering hope to patients tired of conventional therapies. With Phase 3 trials looming and initial results positive, 4DMT is poised to make significant strides in addressing the unmet treatment needs of individuals with CF.
Join us at the Cell & Gene Therapy World Summit 2024, a vital component of the Cell and Gene Therapy World Series, taking place on June 11th-12th, 2024, in London, UK. Cell and gene therapy hold tremendous promise for addressing unmet medical needs, but to fully realize their potential, the industry must overcome persistent challenges. This summit brings together industry leaders to discuss best practices in R&D and manufacturing, showcasing the latest innovations and tackling issues such as immunogenicity, automation, and commercialization. Don’t miss this opportunity to be part of shaping the future of transformative therapies.
Source: https://cysticfibrosisnewstoday.com/news/phase-3-trial-cf-gene-therapy-ad-710-planned-late-2025/
