Aqualung Therapeutics is a biotechnology company based in Arizona focused on developing novel therapies that target inflammation and fibrosis. The team at Aqualung is working to advance treatments for critically ill patients, including those living with conditions such as Acute Respiratory Distress Syndrome (ARDS).
How Aqualung Therapeutics is Making an Impact
Aqualung Therapeutics is developing a novel therapeutic approach targeting the eNAMPT pathway, which plays a major role in inflammation and fibrotic organ injury. The company’s lead drug candidate, ALT-100mAb, targets the eNAMPT protein, a biomarker that becomes highly expressed during acute lung injury & other conditions like progressive pulmonary fibrosis.
In a Phase 2a study involving patients with moderate to severe Acute Respiratory Distress Syndrome (ARDS), patients received ALT-100mAb within six hours of being placed on mechanical ventilation. Among the first 15 patients enrolled in the study, researchers observed a dramatic decrease in days spent on mechanical ventilation, significantly improved SOFA scores (Sequential Organ Failure Assessment), and a substantial reduction in inflammatory cytokines. Importantly, the safety profile of the drug was equivalent to that of the placebo group.
With FDA IND approvals for both ARDS and Progressive Pulmonary Fibrosis (PPF), Aqualung has the opportunity to continue clinical development across multiple disease areas. Preclinical studies and more than 15 publications have also demonstrated potential applications of the therapy in additional conditions, including prostate cancer, inflammatory bowel disease, pulmonary hypertension, chorioamnionitis, respiratory distress syndrome, SAVI syndrome, and interstitial lung disease with pulmonary hypertension.
The Health Challenge Aqualung Therapeutics Is Addressing
The root cause of many different disease states is centered on both inflammation and fibrosis. Aqualung scientists first identified a novel protein called eNAMPT, which binds to a ligand in the body called TLR4. When cell damage or disease occurs, the eNAMPT/TLR4 signaling pathway becomes a major driver of the innate immune response and the severity of inflammatory and fibrotic organ injury.
By targeting a pathway that may be upstream of those targeted by existing therapies like Humira, Aqualung believes its approach could lead to improved treatment outcomes. Ultimately, reducing time in the ICU for critically ill patients with ARDS could lead to lives saved and significant financial savings for hospitals.
Researchers initially identified eNAMPT through screening methods using both animal and human tissues from patients placed on mechanical ventilation. They found that eNAMPT is expressed three to ten times higher in the blood during acute lung injury and is one of the most highly expressed biomarkers in patients requiring mechanical ventilation.
Today, Aqualung’s lead focus is on Moderate to Severe Acute Respiratory Distress Syndrome (ARDS), a condition with approximately 40% mortality and currently no FDA-approved therapies beyond supportive care. Despite decades of research, ARDS has often been described as a “graveyard” for failed therapies, particularly during the height of COVID-19 when many patients required mechanical ventilation.
Aqualung believes many previous therapies failed because treatments were administered too late and patients were not screened to determine whether they had the right biological profile to respond. To address this, the company has developed both a biomarker blood test to measure eNAMPT levels and a genotyping risk score to identify patients most likely to respond to therapy, an approach aligned with precision medicine.
Powered by Arizona’s Bioscience Ecosystem
Arizona’s bioscience ecosystem has played an important role in supporting Aqualung Therapeutics’ growth. AZBio has helped connect the company with talent and expand its reach to a broader investor audience.
Local research institutions have also provided critical resources. Much of Aqualung’s early preclinical work was conducted at Dr. Joe GN Garcia’s laboratory at the University of Arizona, where access to facilities and collaborative research environments helped advance the company’s foundational discoveries.
“I am honored to be working with Dr Garcia. When we met 6 years ago there was an idea and a target. It is rewarding to see the accomplishments over the years to being in a P2 study. The journey of drug development is long but can be very rewarding and we are on a path of saving lives if ALT-100mAb gets across the finish line.” – Stan Miele, President and Chief Business Officer, Aqualung Therapeutics
Looking Ahead
Aqualung Therapeutics is currently raising a $25 million Series A2 round to support the next phase of development. Funding will help complete the Phase 1b/2a study in Progressive Pulmonary Fibrosis (current lead indication) and complete the Phase 2b in moderate to severe ARDS.
The company also plans to initiate investigator-initiated studies exploring the therapy’s potential in SAVI syndrome, respiratory distress syndrome in neonates, and interstitial lung disease with pulmonary fibrosis.
For the Aqualung team, the mission is deeply personal. As founder and CEO, Joe GN Garcia, MD, often says:
“Before I hang up my cleats I want to get a novel therapeutic across the finish line that can save lives and improve human health.”
With promising early clinical data and a precision-medicine approach designed to deliver the right therapy to the right patient at the right time, Aqualung Therapeutics is working toward improving survival for critically ill patients and advancing new treatment options in pulmonary and inflammatory diseases.