#USA #FDA Approval #Juno Beach #Aqualung Therapeutics #ALT-100 mAb

Aqualung Therapeutics Achieves FDA Clearance for Phase 2a Study on Lung Fibrosis

Aqualung Therapeutics, a pioneering company specializing in clinical-stage immunotherapeutics, has made headlines with the announcement that the Food and Drug Administration (FDA) has granted clearance for its Phase 2a clinical study involving ALT-100 mAb. This groundbreaking therapy targets Progressive Pulmonary Fibrosis (PPF), a severe and life-threatening lung condition characterized by inflammation and scarring of lung tissue.

This pivotal FDA approval represents Aqualung's second Investigational New Drug (IND) application clearance, following its successful 2022 submission aimed at addressing acute respiratory distress syndrome (ARDS). The forthcoming multicenter trial, dubbed the PAISANO study—short for Preventing the Advance of Lung Fibrosis via ALT-100 Antibody—will examine various aspects, including the safety and efficacy of ALT-100 mAb across participating sites.

Given the critical nature of the medical need in treating PPF, the FDA's decision is somewhat unprecedented. They waived the prerequisite for a prior study on idiopathic pulmonary fibrosis (IPF), citing compelling preclinical data and established clinical safety evidence that Aqualung provided. This decision underscores the confidence in Aqualung's innovative approach to tackle the pressing challenges posed by lung fibrosis.

The condition affects patients grappling with underlying lung diseases that are either idiopathic—meaning the causes are unknown—or associated with autoimmune disorders. With limited therapeutic options available, the need for effective solutions is dire. ALT-100 mAb is a humanized monoclonal antibody designed to neutralize extracellular nicotinamide phosphoribosyl transferase (eNAMPT), a protein identified by Aqualung's CEO and Founder, Dr. Joe GN Garcia.

eNAMPT functions as a master regulator of the immune system but can also lead to excessive inflammation and the worsening of fibrosis in critical organs. By targeting eNAMPT, ALT-100 mAb serves to curb the immune response that contributes to the pathogenesis of PPF. “The Aqualung team is delighted that the FDA cleared our PAISANO study design,” mentioned Stan Miele, the company's President and Chief Business Officer. “With there being so few treatment options available, we are addressing a significant gap in medical care.”

Dr. Garcia highlights the advancements made, referencing a successful Phase 1a study that involved 32 healthy adult volunteers, as well as a Phase 2a study focused on 15 patients with moderate to severe ARDS. Coupled with rigorous toxicology studies and numerous peer-reviewed publications—over 20 validating the preclinical usefulness of ALT-100 mAb—Aqualung is poised to make a substantial impact in the field.

“Our dramatic disease-attenuating effects of the ALT-100 mAb in experimental models of lung fibrosis, combined with initial evidence of the drug's ability to mitigate innate immune activation and disease severity, provide us with substantial confidence. We believe this medication will be instrumental in meeting the unrealized needs of patients with PPF,” said Dr. Garcia.

About Aqualung Therapeutics

Aqualung Therapeutics stands at the forefront of early-stage biotechnology, spearheading an immunotherapeutics platform focused on eNAMPT. Their Next Gen platform, eNamptor™, encompasses ALT-100 mAb along with a plasma-based biomarker panel aimed at assessing disease risk and severity, and a genotyping assay that helps in identifying genetic risks associated with disease development, severity, and mortality.
The approval of this IND marks a significant step forward for Aqualung Therapeutics as it pursues human clinical trials to meet the urgent therapeutic needs of patients suffering from serious inflammatory and fibrotic diseases, as well as certain cancers. For further details about Aqualung's ALT-100 mAb therapeutic trials, you can visit their official website at aqualungtherapeutics.com.