Actuate Therapeutics

Actuate Therapeutics, Inc. is a clinical-stage biopharmaceutical company developing novel therapeutic agents for patients with cancer or inflammatory diseases. Its lead candidate, elraglusib (previously identified as 9-ING-41), is a small-molecule inhibitor of glycogen synthase kinase-3 (GSK-3), specifically GSK-3β. The company positions elraglusib as acting on multiple molecular pathways implicated in tumor growth and in resistance to conventional cancer drugs such as chemotherapy, and describes it as also acting as a mediator of anti-tumor immunity through inhibition of multiple immune checkpoints and regulation of immune cell function. The stated development goal is to slow, stop, or shrink tumors in hard-to-treat cancers with minimal adverse effects.

The scientific rationale centers on GSK-3β, whose activity and overexpression have been linked to cancer, Alzheimer's disease, bipolar disorder, type II diabetes mellitus, and inflammatory conditions leading to fibrosis. In oncology, GSK-3β has been implicated in chemo- and radioresistance in tumor cells, and aberrant nuclear accumulation of GSK-3β is described as a marker specific to malignant rather than benign cells, supporting a targeted-therapy approach.

The clinical program has been anchored by the Actuate 1801 study, a Phase 1/2 histology-agnostic trial of 9-ING-41 as a single agent and in combination with chemotherapy in patients with refractory hematologic malignancies or solid tumors (NCT03678883), with seamless extension into six different 9-ING-41-based chemotherapy combinations. Company-associated records list 10 trials involving 14 active ingredients, including 9-ING-41 alongside gemcitabine, carboplatin, irinotecan, paclitaxel, docetaxel, oxaliplatin, fluorouracil, leucovorin, doxorubicin, lomustine, losartan, retifanlimab and ruxolitinib, and experimental indications spanning pancreatic, lung, breast, colorectal, kidney and bone neoplasms, glioblastoma, glioma, meningioma, lymphoma, neuroblastoma, metastatic disease and primary myelofibrosis.

Founding story

Actuate was founded in 2015 to develop therapeutics targeting glycogen synthase kinase-3β, a target implicated in cancer, neurodegenerative and psychiatric conditions, diabetes and fibrotic inflammatory disease, and was backed by venture investors including Bios Partners and Kairos Ventures, which invested in 2018.

Business model

Actuate develops proprietary small-molecule drug candidates in-house and states it is open to partnerships with academic and scientific organizations; it has no marketed drug or approved NDA to date.

Traction

The Actuate 1801 Phase 1/2 trial dosed its first patient at Brown University/Rhode Island Hospital in January 2019 and enrolled over 70 patients by November 2019, recruiting at Brown University Rhode Island Hospital, UCSF Helen Diller Family Comprehensive Cancer Center, Miami Cancer Institute, Mayo Clinic and Sanford Research. Later milestones reported by investor Kairos Ventures include positive Phase 1 results in pediatric sarcomas with complete and partial responses (July 2025), publication of positive Phase II data for elraglusib with platinum chemotherapy (December 2025), and a Nature Medicine publication reporting a doubling of 12-month survival with elraglusib plus chemotherapy in previously untreated metastatic pancreatic ductal adenocarcinoma (April 2026).