CAL101 is a first-in-class monoclonal antibody targeting S100A4, a DAMP protein implicated in severe, life-threatening fibrotic diseases
CAL101 being investigated in Phase 2 IPF Study; enrollment ongoing at sites in the USA, UK, EU, Turkey and South Korea
OSLO, Norway & BOSTON–(BUSINESS WIRE)–Calluna Pharma AS, a clinical stage biotechnology company pioneering first-in-class antibodies to treat inflammatory and fibrotic diseases, today announced that it has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for CAL101, its lead clinical candidate currently being evaluated in patients with idiopathic pulmonary fibrosis (IPF).
“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 grants Orphan Drug Designation to support the development and evaluation of new treatments for rare diseases, conditions affecting fewer than 200,000 people in the USA. Benefits of this designation include potential eligibility for expedited review pathways, tax credits for qualified clinical trials, fee waivers, and seven years of market exclusivity after approval.
AURORA is a randomized, double-blind, placebo-controlled trial designed to evaluate the efficacy and safety of CAL101 in patients with IPF. The study aims to enroll 150 individuals with IPF across more than 50 sites, primarily in the USA, UK, EU, Turkey and South Korea. After an initial 28-day screening period, patients will be randomized to receive seven monthly intravenous infusions of CAL101 or placebo at a randomization ratio of 3:2, respectively. The study’s primary endpoint is lung function, measured by forced vital capacity, or how much air can forcibly be exhaled, versus an individual’s baseline.
About CAL101
CAL101 is a systemically administered monoclonal antibody targeting the DAMP protein S100A4. S100A4 is activated when tissue is stressed or injured, triggering multiple downstream pathways. It is associated with pathological activation and proliferation of fibroblasts (the key effector cells driving progression of fibrosis), and pro-fibrotic immune response connected to fibrotic disease. Targeting S100A4 has the potential to re-establish tissue homeostasis by switching off the downstream pathways involved in the persistent and maladaptive scar tissue formation characteristic of IPF.
A randomized, double-blind, placebo-controlled Phase 1 study of CAL101 demonstrated a favorable safety profile, with balanced frequency and types of AEs, compared to placebo, across all dose levels. Preclinical studies have shown CAL101 to prevent and treat fibrosis and modify the disease-specific activation of fibroblasts.
About IPF
Idiopathic pulmonary fibrosis (IPF) is a progressive lung disease where an inappropriately activated wound-healing response causes the lung tissue to become thickened and scarred, making it difficult to breathe. The exact triggers of IPF are unknown, but it is believed to be a combination of genetic and environmental factors. Over time, the scarring in the lungs worsens, leading to respiratory failure and ultimately death, with a 3-5-year median survival rate. Primarily found in older adults, the disease impacts approximately 233,000 people in the USA and EU.
About Calluna Pharma www.callunapharma.com
Calluna Pharma is a global clinical stage company pioneering a breakthrough approach to treating inflammatory and fibrotic diseases by leveraging the body’s innate immune system. The Company’s therapeutic approach targets upstream amplifiers of disease, offering potential applicability across a diverse array of medical conditions. Calluna Pharma has a pipeline of selective antibodies targeting immunological diseases with enhanced efficacy and tolerability.
Calluna Pharma is incorporated in Oslo, Norway and operates globally.
Contacts
Media Contact:
Jason Glashow
Glashow Strategic Communications
Email: Jason@glashowstrategic.com
Tel: +1 617-510-1800
