This project aims to complete a phase Ia and b study for the TB drug BTZ043. Supported by a previous Flex Fund we have nearly completed all preclinical toxicology and safety studies and reviewed the results with the regulatory authorities during a kick-off meeting. So far BTZ043 has been a well-tolerated drug candidate in rats and mini-pigs up to plasma levels that exceed the MTB MIC90-level several hundred fold. We propose now to start producing the drug substance while we develop a suitable solid drug formulation. The project is structured into 3 parts - each defined by a verifiable milestone. In Q2 we will review all preclinical and drug substance data in a scientific advice with the Federal Institute for Drugs and Medical Devices (BfArM). In Q4 2017/Q1 2018 we aim to enroll the first patients for a combined phase I a/b safety and tolerability study of different doses of BTZ043. Aim will be to design the phase I study as stringent as possible. Up to 40 volunteers will be enrolled. From Q3 2019 onwards, we plan a phase IIa study that will enroll up to 80 TB patients. Funding for this phase IIa study has already been secured through EDCTP. Additional toxicology and work that needs to be accomplished during phase I is supported by funds provided through additional funders.

This project is part of the EDCTP2 programme supported by the European Union.