First Wave Biopharma Inc. is taking apart the top-line data from its phase II study of adrulipase for treating nutrition deficiencies in cystic fibrosis patients and seems unsure exactly what it found. The company said that while the initial data found the enhanced enteric microgranule delivery formulation of adrulipase was safe, well-tolerated and was an improvement over other formulations, the preliminary data also indicate that “it is likely the primary efficacy endpoint was not achieved.”
ΔF508 is the most prevalent mutation detected in patients with cystic fibrosis (CF), and it causes a loss of F508 within CFTR’s first nucleotide binding domain (NBD1). Researchers from Sionna Therapeutics Inc. recently reported the discovery and preclinical evaluation of novel small-molecule CFTR NBD1 stabilizers and CFTR assembly correctors as potential new agents for the treatment of CF.
Anoat Therapeutics has raised €2 million (US$2.17 million) in seed funding to develop novel therapies for cystic fibrosis. The financing comes from a seed funding partnership between Adbio Partners SAS and Inserm Transfert SA.
At the recent ATS meeting, Tavanta Therapeutics Inc. introduced TAVT-135, a cell-penetrating peptide (CPP) conjugate that acts as a chloride ion (Cl-) transporter. The product is currently being investigated for cystic fibrosis (CF), regardless of CF transmembrane conductance regulatory (CFTR) gene mutation status.
30 Technology Ltd. has divested its wound care business, leaving it to focus on pharmaceutical applications of its patented nitric oxide-generating chemistry in the treatment of antibiotic-resistant respiratory infections.
An investor has seen promise in First Wave Biopharma Inc.’s targeted, systemic gastrointestinal disease biotherapeutics, offering $4 million in a private placement as enrollment picks up pace in a phase II trial for the company’s lead candidate, adrulipase.
Lung Therapeutics Inc. has divulged conjugates comprising epithelial sodium channel (ENaC) blockers (particularly, amiloride and benzamil) covalently linked to peptides to retain in lung tissues reported to be useful for the treatment of cystic fibrosis, idiopathic pulmonary fibrosis, chronic obstructive pulmonary disease, asthma, primary ciliary dyskinesia, pulmonary emphysema, pneumonia and bronchiectasis.
Vertex Pharmaceuticals Inc. has received clearance from the FDA for its IND application for VX-522, a messenger ribonucleic acid (mRNA) therapy targeted at treating the underlying cause of cystic fibrosis (CF).
Sionna Therapeutics Inc. has received FDA clearance of its IND application for SION-638, a small molecule designed to target the first nucleotide-binding domain (NBD1) of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. A phase I study is now dosing healthy volunteers.
