News

The Cystic Fibrosis Foundation is giving Laurent Pharmaceuticals $2 million to conduct a Phase 2 clinical trial of Laurent’s cystic fibrosis therapy LAU-7b. The additional funding comes on top of $3 million that the foundation has already given the company. “We are truly grateful for this additional support from Cystic Fibrosis…

Key results of a Phase 1/1b clinical trial of Pulmatrix’s lead treatment for fungal infections in asthma and cystic fibrosis (CF) are expected by mid-2018, the company reports. The trial, which started in February, is focusing on pulmazole’s safety and patients’ ability to tolerate it. Pulmatrix expects the results to…

The U.S. Food and Drug Administration has granted Breakthrough Therapy Designation to Proteostasis Therapeutics’ cystic fibrosis therapy PTI-428 — a move aimed at accelerating its development. Mutations of the CFTR gene cause CF. The most common is F508del, which leads to faulty CFTR protein. Normal CFTR protein is required for transporting chloride ions…

Dozens of diverse types of the bacteria Staphylococcus aureus were identified in the airways of cystic fibrosis (CF) patients, including five new types and one commonly associated with livestock, a study reports. Findings were reported in the study, “Emergence and spread of worldwide Staphylococcus aureus clones among cystic fibrosis patients, published in the…

Anthera Pharmaceuticals is discontinuing the development of Sollpura (lipromatase) as a treatment for cystic fibrosis-related digestive problems. This follows the announcement of the latest data from the RESULT Phase 3 clinical trial (NCT03051490), which failed to demonstrate Sollpura’s effectiveness. The treatment was unable to significantly improve fat…

Ibuprofen does a good job of fighting bacteria in cystic fibrosis (CF) patients’ lungs, including two key threats, Pseudomonas aeruginosa and Burkholderia, a study shows. Researchers said the findings support ibuprofen’s use as an add-on therapy against bacterial infections in CF. The study, “Antimicrobial Activity of Ibuprofen against Cystic Fibrosis-Associated Gram-Negative…

JHL Biotech plans to start a Phase 1 clinical trial this month of its cystic fibrosis (CF) therapy JHL1922, a biosimilar to Pulmozyme (dornase alfa) that is designed to improve lung function. The trial will be in the Netherlands, the company announced. The Dutch Healthcare Authority approved the trial…

Prepubescent children with cystic fibrosis (CF) have lower bone density compared to healthy children, and findings are comparable using two distinct bone density scanning methods, researchers report. The study that supports that conclusion, “Peripheral quantitative computed tomography detects differences at the radius in prepubertal children with cystic…

Verona Pharma’s nebulized RPL554 can significantly improve lung function in patients with cystic fibrosis, according to top-line data from a Phase 2a clinical trial. The latest results revealed that single administration of both high and low doses of the investigative drug significantly increased patients’ average forced expiratory volume in…

Caregivers of people with rare diseases face emotional, financial, physical, and social strain and receive little outside support, a U.S. study found. The findings will be presented to Congress this spring along with policy recommendations to help support those caring for people with rare diseases. The report is titled “…