LTX-109. Broad spectrum anti-viral therapeutics for infectious diseases with epidemic potential

Airborne viruses can be quite dangerous, especially for groups of risk (e.g. immunocompromised patients), as they can replicate and migrate from the upper (nose/nasopharynx) into lower respiratory tract (lungs) and lead to severe conditions such as pneumonia. Indeed, the nose is the main site for initial viral infection and replication. Viral load titers in patients (nasal and pharyngeal swabs) have been shown to correlate with level of infectivity and mortality. This suggests that new therapeutic strategies are needed to reduce viral load in the nose early in the disease, which is highly relevant to reduce complications for risk patients, but also as treatment strategy in pandemic context where repercussions can be catastrophic. PH is developing a novel small peptide-like drug, LTX-109, which binds to the membrane/envelope of microorganisms, leading to fast-acting membrane/envelope lysis and cell death/inactivation. The main goal of this project is to complete comprehensive studies on characterization, stability, safety, tolerability, and efficacy of the novel LTX-109, against a panel of selected virus with pandemic potential. These include a series of in silico (e.g. through AI tools), in vitro and in vivo studies (e.g. safety and efficacy), as well as first-in-man and Proof-of-concept (PoC) clinical trials of the drug. Moreover, we would like to further investigate the potential effect of LTX-109 in the activation of an immune response against virus-associated antigens as LTX-109's mode of action may also represent an innovative "post exposure in situ immunization" approach. Thus, the intense research in this project will not only advance knowledge on this drug, but also bring into light key understanding on novel anti-viral modes of action and approaches, which can have a true impact for the scientific and clinical communities towards the development of broad spectrum anti-viral therapeutics against infectious diseases with epidemic potential.