E!10687 GT-002 for the treatment of schizophrenia.

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The aim of this project was to advance the new drug candidate GT-002 through phase 1 clinical trials in healthy volunteers. The project has succeeded in this. A single ascending dose study in healthy volunteer males has been completed. GT-002 was found to be safe and well tolerated in healthy male volunteers. This has been a major development towards further studies in patients. The results that have been generated in the study will form the basis for the planning and implementation of the subsequent clinical studies including Multiple Ascending Dose (MAD) studies in which GT-002 will be given repeatedly for a limited time to evaluate safety and pharmacokinetics. A study in patients including efficacy evaluation is planned where valuable data will be collected regarding GT-002 effect on CNS disorders.

There are 4 main pillars in the project: For WP4, healthy volunteers will be randomized in a double-blind, placebo-controlled Phase 1 first-in-human study to investigate the safety, tolerability and pharmacokinetics of ascending single oral doses of GT-002, including an assessment of food interactions with drug absorption. WP4 will thus clarify whether GT-002 is safe and well-tolerated as a single oral dose in healthy volunteers and whether it has favorable pharmacokinetic properties for further development. In WP5, healthy volunteers will be randomized in a double-blind, placebo-controlled Phase 1 study to investigate the safety, tolerability and pharmacokinetics of multiple ascending oral doses of GT-002. WP5 will thus clarify if repeated doses of GT-002 are safe and well-tolerated in healthy volunteers and whether its pharmacokinetic properties remain favorable after multiple dosing. WP6 is composed of exploratory PET and EEG single-dose studies. WP6 will give information about the regional extent of the brain GABAA receptor occupancy regarding GT-002 binding and information about the sedative effects of GT-002. The aim is to demonstrate target engagement at a non-sedative dose level of GT-002. The overall objective of WP7 is to reach a business agreement with a financially strong pharma/biotech company to ensure further clinical development until the end of Phase 3, or alternatively license out the GT-002 marketing rights based on the results obtained in this Eurostars project, either to a biotech partner or a pharmaceutical company. In addition, minor work packages will ensure that the manufacturing and formulation development process are performed according to cGMP (WP2), that the pre-clinical studies are performed according to cGLP (WP3) and regulatory science including regulatory affairs and presenting and discussing a regulatory strategy with relevant EU authorities in connection with Scientific Advice (WP8).