Therapy targeting lung scar tissue gains important FDA designation
CAL101 from Calluna Pharma being tested in Phase 2 AURORA trial
The U.S. Food and Drug Administration (FDA) recently granted orphan drug designation to Calluna Pharma‘s CAL101 as a potential treatment for idiopathic pulmonary fibrosis (IPF).
This designation is intended to incentivize the development of treatments for rare diseases that affect fewer than 200,000 people in the U.S. This status provides regulatory benefits like tax breaks, fee waivers, and the potential for seven years of market exclusivity if the therapy is ultimately approved.
“Orphan drug designation speaks to the importance of developing new treatments for debilitating rare diseases like IPF, for which treatment options are limited,” Margrethe Sørgaard, Calluna’s senior vice president of clinical operations and pharmacovigilance, said in a company press release.
AURORA trial to assess how CAL101 affects lung scarring, breathing
A Phase 2 clinical trial is testing CAL101 in people with IPF. The study, dubbed AURORA (NCT06736990), aims to enroll 150 adults with IPF, ages 40 and older. The trial is open to participants who are not currently on IPF treatment or who are on a stable dose of approved IPF therapies Ofev (nintedanib) or Esbriet (pirfenidone). Recruitment is ongoing at sites across the U.S., European Union, United Kingdom, Turkey, and South Korea.
Participants will be randomly assigned to receive CAL101 or a placebo through monthly intravenous (into the vein) infusions for six months. The main goal is to measure the therapy’s impact on forced vital capacity (FVC) — a standard measure of lung function that assesses how much air a person can blow out after a deep breath. The study will also assess the therapy’s effects on fibroblasts, which are cells that contribute to lung scarring in IPF.
“Our ongoing IPF study (AURORA) aims to demonstrate that CAL101 prevents the disease-specific activation of fibroblasts that lead to decreased lung function, and the progressive decline these patients face,” Sørgaard said.
IPF is marked by inflammation and fibrosis (scarring) in the lungs. Fibroblasts — structural cells that help produce scar tissue — become abnormally active in IPF, which contributes to fibrosis. CAL101 is an antibody-based therapy designed to block the activity of S100A4, a protein that promotes activation of fibroblasts and fibrosis. In early studies with healthy volunteers, the therapy was well tolerated, with no serious side effects reported.
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