Aqualung Awarded $4.8M From NIH to Develop Monoclonal Antibody

ALT-100 is designed to prevent inflammation in severe inflammatory diseases

Somi Igbene, PhD avatar

by Somi Igbene, PhD |

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The National Institutes of Health (NIH) has granted Aqualung Therapeutics two three-year awards totaling $4.8 million to develop ALT-100, a humanized monoclonal antibody, for the treatment of pulmonary arterial hypertension (PAH) and inflammatory bowel disease (IBD).

ALT-100 is designed to address and prevent inflammation in severe inflammatory diseases. It works by blocking the binding of eNAMPT to the TLR4 protein receptor, a mechanism that amplifies unchecked inflammation. According to Aqualung, ALT-100 is a “potential game-changing therapy” for many inflammatory disorders that also include acute respiratory distress syndrome (ARDS) — which can develop in patients with respiratory infections such as COVID-19 — and pulmonary fibrosis.

“It is evidently clear that the utility of ALT-100 now goes well beyond the initial indication of ARDS, and the published pre-clinical evidence of ALT-100 demonstrates how this novel therapeutic has both anti-inflammatory and anti-fibrotic [anti-scarring] properties,” Stan Miele, president and chief business officer at Aqualung, said in a press release.

“We believe ALT-100 will play a pivotal role for those … looking for more of a curative therapy for the condition of PAH,” Miele added.

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Lung inflammation is a crucial component of the disease process in PAH. It promotes changes in the function and structure of lung blood vessels (vascular remodeling), eventually causing heart failure. Available treatments do not reverse vascular remodeling or significantly prolong survival in patients with PAH. Moreover, according to Aqualung, the median survival of patients with idiopathic (no known cause) PAH is approximately 2.8 years without effective treatment. Therefore, new therapies are needed.

By inhibiting inflammation, ALT-100 could prevent the damaging blood vessel changes characteristic of PAH. Notably, the therapeutic potential of targeting NAMPT activity has been shown in rat models of pulmonary hypertension.

The NIH fast-track awards will help Aqualung to confirm the efficacy of subcutaneous (under-the-skin) delivery of ALT-100 and the appropriate dosing in animal models of PAH and IBD. It will also facilitate pharmacological and safety studies necessary to obtain permission to conduct clinical trials.

“We are pleased the NIH echoes our belief that ALT-100 and the target of eNAMPT is central to IBD and PAH,” Miele said.

ALT-100 is currently in a Phase 1 trial (NCT05426746) in Australia to test its safety, tolerability, and pharmacological profile in healthy adults. Aqualung recently announced the U.S. Food and Drug Administration has cleared its Investigational New Drug application, enabling the company to continue this trial, called the P1A study. The trial has dosed two of the four dosing groups and is expected to be completed by mid-2023.

After successfully completing the P1A study, Aqualung will initiate P2A (PUERTA – Pioneering the Utility of eNAMPT Reducing Therapies in ARDS/VILI) to test ALT-100 in approximately 90 people with moderate to severe ARDS. The study will be conducted in about eight U.S.-based hospitals.

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