News

CF treatment Alyftrek shows lung benefits, per new data

Vertex Pharmaceuticals presented new data highlighting additional clinical benefits and improved outcomes in people with cystic fibrosis (CF) treated with the triple combination Alyftrek (vanzacaftor/tezacaftor/deutivacaftor) or other CFTR modulators. The data also suggest that Alyftrek may lead to fewer pulmonary exacerbations (flare-ups) and less antibiotic usage than…

New Sionna trial tests NBD1 stabilizer SION-719 with Trikafta

Sionna Therapeutics has launched a clinical trial to test its experimental therapy SION-719 in combination with Trikafta (elexacaftor/tezacaftor/ivacaftor), which is currently the standard-of-care treatment for cystic fibrosis (CF). According to a company press release, the Phase 2a study (NCT07108153) will enroll adults with CF who…

ARCT-032 well tolerated in CF trial with early positive signs

ARCT-032, Arcturus Therapeutics’ experimental inhaled therapy for cystic fibrosis (CF), has been well tolerated so far in a clinical trial. Early imaging data also suggest it may help clear mucus from the lungs, according to interim results announced by the company. “These findings suggest that ARCT-032 may be…

Trial for CF treatment BX004 nears top-line results in Europe

Despite a recent hold by the U.S. Food and Drug Administration (FDA) on a BX004 clinical trial in the U.S., the experimental cystic fibrosis (CF) lung infection treatment is still advancing in Europe. According to BX004’s developer Biomx, top-line results are expected in early 2026. “Patient enrollment and…

Close monitoring urged for weight gain with Kaftrio in CF patients

While treatment with Kaftrio (elexacaftor/tezacaftor/ivacaftor) helps people with cystic fibrosis (CF) gain weight, a tendency toward unhealthy diets may pose new risks for patients — warranting “close monitoring and interventions to prevent excessive weight gain” — a study from Norway reports. People with CF in the European nation who…

A CF patient advocate’s mission is to improve clinical trials

As a child, Ella Balasa was no stranger to hospital stays. Diagnosed with cystic fibrosis (CF) at 18 months, she faced persistent lung infections that often required weeks-long courses of antibiotics, multiple times a year. “I was always in the hospital needing treatment,” Balasa, now 33 and living in…