Lead candidate Apta-1 in sepsis
Apta-1, an RNA therapeutic, is currently one of few pharmaceutical candidates for sepsis. In a number of preclinical studies, Apta-1 has shown unique anti-inflammatory, anti-thrombotic and immunomodulating properties, which indicates that Apta-1 could be the complete solution that today’s sepsis treatment is lacking. The candidate is currently in early clinical phase.
The company has performed the preclinical studies required by the authorities to take Apta-1 into clinical phase. These preclinical studies have shown that Apta-1 does not show any signs to cause serious side-effects and that it has a multi-functional effect on severe inflammatory conditions.
Today, there is no approved treatment for sepsis or septic shock, only the SoC (Standard of Care) with antibiotics, different anti-coagulant and anti-inflammatoric treatments including supportive treatment to maintain healthy blood pressure and improve oxygenation to prevent organ failure. The medical need to develop an effective treatment for sepsis and septic shock is thus enormous given its high death rates.
Apta-1 in clinical phase
Aptahem has performed a phase 1 study, a so called First in Human (FIH), which started in December 2022 and finalized in June 2024 after submitting the final report to the authority. The study was performed by the Centre for Human Drug Research (CHDR) in the Netherlands. CHDR was chosed based on their long experience from clinical trials in early phase using a specific methodology for clinical research within inflammation and sepsis.
The results from the clinical phase 1 study show that Apta-1 is safe and well tolerated by the healthy volunteers, and Aptahem chose to close the study 9 November 2023. The phase 1 study in healthy volunteers also showed indications on drug efficacy as one individual had elevated inflammatory values prior to dosing. In this case, Apta-1 dosing was associated with rapid reduction of inflammatory parameters.
Strengthened by these findings, Aptahem now plans for further clinical studies of Apta-1. Aptahem plans for a phase 2 study in patients where the potential drug efficacy of Apta-1 will be studied.
Scientific collaborations
Aptahem collaborates with academic groups around the world to understand how the company’s drug candidate Apta-1 functions in different conditions related to sepsis or other acute inflammatory conditions, as well as further explore its inherent potential.
Aptahem also builds a network of Key Opinion Leaders (KOLs) to have access to the latest finding in their respective scientific areas, and to strenghthen the company’s position in the scientific sphere.