BOSTON — Calluna Pharma AS, a clinical-stage biotechnology company developing first-in-class antibodies for inflammatory and fibrotic diseases, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to CAL101, its lead clinical candidate currently being evaluated in patients with idiopathic pulmonary fibrosis (IPF).
CAL101 is a first-in-class monoclonal antibody targeting S100A4, a damage-associated molecular pattern (DAMP) protein linked to severe and life-threatening fibrotic diseases. The drug is being investigated in the ongoing Phase 2 AURORA study, which is enrolling patients with IPF at clinical sites across the United States, United Kingdom, European Union, Turkey, and South Korea.
“Orphan drug designation speaks to the importance of developing new treatments for debilitating rare diseases like IPF, for which treatment options are limited,” said Margrethe Sørgaard, Calluna Pharma’s Senior Vice President of Clinical Operations and Pharmacovigilance. “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.”
The FDA’s Orphan Drug Designation program supports the development of therapies for rare diseases affecting fewer than 200,000 people in the U.S. The designation provides benefits such as eligibility for expedited review, tax credits for clinical testing, waiver of certain regulatory fees, and seven years of market exclusivity upon approval.
The AURORA trial is a randomized, double-blind, placebo-controlled study designed to assess the efficacy and safety of CAL101 in patients with IPF. The trial will enroll approximately 150 participants across more than 50 global sites. Following a 28-day screening period, participants will be randomized to receive seven monthly intravenous infusions of CAL101 or placebo in a 3:2 ratio. The study’s primary endpoint is lung function, measured by changes in forced vital capacity (FVC) from baseline.
