Ventaleon GmbH, a biotechnology company focusing on the development of inhaled treatments against viral infections, today announced the initiation of a human challenge trial for its flu treatment candidate. The study will assess the safety, tolerability and efficacy of Ventaleon’s lead therapy candidate, an inhaled formulation of LASAG (D,L-lysine acetylsalicylate • glycine), in healthy volunteers infected with the H3N2 subtype of the influenza A virus. Topline results are expected in late 2017. Following a previous Phase II study and based on consultation with the regulatory authorities, this Phase Ib challenge trial was designed to show the causal effect of LASAG inhalation on the influenza virus and is an important step in preparing for pivotal trials. Ventaleon is going to present data from the Phase II study at an upcoming scientific conference.
In the phase Ib study initiated now, approximately 80 volunteers will be randomized in a 2:2:1 ratio into three study arms: 1) 800 mg inhaled LASAG plus oral placebo; 2) inhaled placebo plus oral placebo or 3) inhaled placebo plus oral ASA (acetylsalicylic acid). Treatment will be administered in three daily doses over five days. The primary endpoints include the effect on viral shedding as well as safety. Secondary endpoints include the effect on peak viral titers as well as on alleviating clinical signs and symptoms. Additionally, three exploratory endpoints will be evaluated: serum levels of ASA and SA (salicylic acid), the viral target sequence to assess resistance development and correlations between pharmacokinetics, viral shedding, clinical influenza symptoms and other endpoints. The AKITA® Jet inhalation system (Vectura group plc, United Kingdom) will be used for all inhaled administrations.
In preclinical studies, LASAG showed effectiveness in preventing viral replication by stabilizing nuclear pores through NFkb inhibition, so that the newly generated viral genomes could not leave the nucleus and would not be available for particle assembly. This mode of action without involvement of viral proteins could prevent development of resistance against LASAG-based treatments.
“Influenza is a common, potentially life-threatening infection with lengthy recovery times and a high economic burden. Current treatments are often ineffective as new flu strains frequently develop resistance. Previous studies with inhaled LASAG support that it could be a faster, more effective treatment of influenza, including strains that are resistant to current treatment, addressing a major unmet medical need,” said Dr. Ulrich Dauer, CEO of Ventaleon. “It is Ventaleon’s primary goal to provide highly effective, inhaled therapies against viral infections to address these needs.”