GT-002 / Gabather - LARVOL DELTA

GT-002, a novel positive allosteric modulator of the GABAA receptor (GABAA PAM): preclinical and clinical characterization (Neuroscience 2025) - "Strategic partnering opportunities are being explored to support later-stage trials and commercialisation pathways. Overall, GT-002 represents a promising CNS drug candidate with a novel mechanism, favourable receptor selectivity, and strong translational biomarker support."

Preclinical • Alzheimer's Disease • CNS Disorders • Cognitive Disorders • Depression • Psychiatry • Schizophrenia

Gabather Joins Forces with the Centre for Neuropsychiatric Schizophrenia Research in Denmark to Conduct a Clinical Phase II Study with GT-002 (PRNewswire) - "Gabather AB...announces that the Company has signed a Collaborative Agreement with the Centre for Neuropsychiatric Schizophrenia Research at the Psychiatric Centre in Glostrup to conduct a so-called clinical phase II study with GT-002 in patients diagnosed with schizophrenia....Funded by an Investment Agreement from the Innovation Fund Denmark (IFD), it will now be possible to conduct the first study with GT-002 in patients with schizophrenia. The study is a double-blind randomised so-called phase II study investigating the effect of GT-002 compared to placebo and an active comparator."

Licensing / partnership • New P2 trial • CNS Disorders • Schizophrenia

[VIRTUAL] Ex vivo expanded NK cells exhibit potent cytotoxicity against multiple myeloma. (EACR 2021) - "We aimed to investigate whether our GTA002 NK cell product, expanded and differentiated ex vivo from cord-blood derived CD34+ hematopoietic stem cells, will in vitro effectively kill target various MM cell lines...Representative model MM cell lines were chosen based on their sensitivity/resistance to lenalidomide, bortezomib (BTZ) and CD38 surface expression, latter associated with responsiveness to daratumumab treatment: MM.1S, LP1, NCI-H292, OPM2, RPMI-8226, U266B1, KMS11 and KMS11/BTZ...Receptor and ligand expression of effector and target cells, respectively, will be determined by flow cytometry Results and Discussions We observed cytotoxicity of GT002 at E:T ratio 10:1 in all model MM cell lines irrespective of their chemoresistance status or CD38 surface antigen density...More importantly, we observed high killing efficiency also in U266B1 cells with low CD38 expression, which is indicative of the refractoriness to daratumumab therapy. Conclusion Our experimental..."

IO biomarker • Preclinical • Hematological Disorders • Hematological Malignancies • Multiple Myeloma • Oncology • CD34 • CD38 • NCAM1 • PTPRC

[VIRTUAL] Ex vivo expanded NK cells exhibit potent cytotoxicity against multiple myeloma. (EACR 2021) - "We aimed to investigate whether our GTA002 NK cell product, expanded and differentiated ex vivo from cord-blood derived CD34+ hematopoietic stem cells, will in vitro effectively kill target various MM cell lines...Representative model MM cell lines were chosen based on their sensitivity/resistance to lenalidomide, bortezomib (BTZ) and CD38 surface expression, latter associated with responsiveness to daratumumab treatment: MM.1S, LP1, NCI-H292, OPM2, RPMI-8226, U266B1, KMS11 and KMS11/BTZ...Receptor and ligand expression of effector and target cells, respectively, will be determined by flow cytometry Results and Discussions We observed cytotoxicity of GT002 at E:T ratio 10:1 in all model MM cell lines irrespective of their chemoresistance status or CD38 surface antigen density...More importantly, we observed high killing efficiency also in U266B1 cells with low CD38 expression, which is indicative of the refractoriness to daratumumab therapy. Conclusion Our experimental..."

IO biomarker • Preclinical • Hematological Disorders • Hematological Malignancies • Multiple Myeloma • Oncology • CD34 • CD38 • NCAM1 • PTPRC

[VIRTUAL] Ex vivo expanded NK cells exhibit potent cytotoxicity against multiple myeloma. (EACR 2021) - "We aimed to investigate whether our GTA002 NK cell product, expanded and differentiated ex vivo from cord-blood derived CD34+ hematopoietic stem cells, will in vitro effectively kill target various MM cell lines...Representative model MM cell lines were chosen based on their sensitivity/resistance to lenalidomide, bortezomib (BTZ) and CD38 surface expression, latter associated with responsiveness to daratumumab treatment: MM.1S, LP1, NCI-H292, OPM2, RPMI-8226, U266B1, KMS11 and KMS11/BTZ...Receptor and ligand expression of effector and target cells, respectively, will be determined by flow cytometry Results and Discussions We observed cytotoxicity of GT002 at E:T ratio 10:1 in all model MM cell lines irrespective of their chemoresistance status or CD38 surface antigen density...More importantly, we observed high killing efficiency also in U266B1 cells with low CD38 expression, which is indicative of the refractoriness to daratumumab therapy. Conclusion Our experimental..."

IO biomarker • Preclinical • Hematological Disorders • Hematological Malignancies • Multiple Myeloma • Oncology • CD34 • CD38 • NCAM1 • PTPRC

[VIRTUAL] Ex vivo expanded NK cells exhibit potent cytotoxicity against multiple myeloma. (EACR 2021) - "We aimed to investigate whether our GTA002 NK cell product, expanded and differentiated ex vivo from cord-blood derived CD34+ hematopoietic stem cells, will in vitro effectively kill target various MM cell lines...Representative model MM cell lines were chosen based on their sensitivity/resistance to lenalidomide, bortezomib (BTZ) and CD38 surface expression, latter associated with responsiveness to daratumumab treatment: MM.1S, LP1, NCI-H292, OPM2, RPMI-8226, U266B1, KMS11 and KMS11/BTZ...Receptor and ligand expression of effector and target cells, respectively, will be determined by flow cytometry Results and Discussions We observed cytotoxicity of GT002 at E:T ratio 10:1 in all model MM cell lines irrespective of their chemoresistance status or CD38 surface antigen density...More importantly, we observed high killing efficiency also in U266B1 cells with low CD38 expression, which is indicative of the refractoriness to daratumumab therapy. Conclusion Our experimental..."

IO biomarker • Preclinical • Hematological Disorders • Hematological Malignancies • Multiple Myeloma • Oncology • CD34 • CD38 • NCAM1 • PTPRC

GAB-001: Study of Ascending Single Oral Dose of GT-002 in Healthy Volunteers (clinicaltrials.gov) - P1; N=32; Completed; Sponsor: Gabather AB; Recruiting ➔ Completed

Clinical • Trial completion

Preclinical and clinical studies with GT-002, a novel GABAA receptor ligand with putative antipsychotic and pro-cognitive effects (Neuroscience 2019) - "The compound shows potent antipsychotic effects in rodent in-vivo models comparable to those of clozapine. GT-002 has been tested in 28-day GLP tox study in dogs without adverse effects. In summary, GT-002 has a profile that is distinct from other GABAA receptor modulators such as Diazepam."