New preclinical data for TAVT-135, an experimental medication for the pan-genotypic treatment of cystic fibrosis (CF), support its effectiveness and utility in the treatment of patients who are ineligible for or do not respond well to cystic fibrosis transmembrane conductance regulator (CFTR) modulators, Tavanta Therapeutics announced.

The company shared the new data at both the 2023 American Thoracic Society International Conference and the European Cystic Fibrosis Foundation Basic Science Conference held recently in Dubrovnik, Croatia.

“While many people with CF benefit from mutation-specific therapies that can restore the function of the defective CFTR protein, some people do not respond to these modulators due to ineligible mutations, side effects, or lack of access,” said Lynne Powell, chief executive officer of Tavanta Therapeutics.

“As demonstrated by the data reported at American Thoracic Society International Conference and the European Cystic Fibrosis Foundation Basic Science Conference, TAVT-135 may represent an effective treatment option for people with CF, regardless of CFTR mutation status.”

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According to the recent TAVT-135 presentations, patch clamp measurements showed that TAVT-135 effectively stimulated intracellular chloride ion transport across plasma membranes in Xenopus laevis oocytes.

Moreover, dose-dependent increases in short circuit current were achieved in chamber electrophysiological studies of human bronchial epithelial cells with nonfunctional CFTR mutations.

The investigational medication also amplified airway surface liquid and periciliary layer height in a human CF bronchial epithelial cell line homozygous for F508del, which is the most frequent mutation associated with CF.

TAVT-135 may be able to restore chloride ion balance in the airways and prevent mucus thickening and accumulation, as well as limit the progressive deterioration of lung function in patients with CF. The drug acts as an artificial chloride ion transporter across cell membranes.

Reference

Tavanta Therapeutics announces new preclinical data for its cystic fibrosis candidate TAVT-135 at two prominent scientific conferences. News release. Tavanta Therapeutics; May 24, 2023.