Our Research

Our focus is on the development of compounds for combination use in high impact carcinomas including glioblastoma, ovarian, breast, and pancreatic cancers. We have completed studies of our lead compound, 9-ING-41, in preclinical patient-derived xenograft models of cancers, including both drug sensitive and drug resistant carcinomas, results of which are quite compelling, including complete and durable regressions of disease without recurrences, and significant increases in overall survival. Our compounds have been well tolerated and have demonstrated a favorable pharmacokinetic profile.

In addition, we are advancing research for the development of our compounds for the treatment of inflammation leading to fibrosis and neurodegeneration, where results of early studies in animal models are promising.

The Company has completed pharmacology and toxicology studies and our lead drug candidate demonstrated very favorable pharmacokinetic and tox profiles in preclinical and clinical studies. A groundbreaking Phase 1/2 trial across multiple histologies (Clinical Study 1801 (Clinicaltrials.gov)) is currently enrolling patients in leading institutions in the US and the EU.

The Actuate 1901 study (Clinicaltrials.gov) will establish the value of 9-ING-41 as a single agent or in combination with Ruxolitinib in patients with advanced myelofibrosis.

The Actuate 1902 study (Clinicaltrials.gov) is a Phase 1 study of 9-ING-41 as a single agent or in combination with Irinotecan in pediatric patients with refractory cancers. This study is being conducted at multiple leading US sites with a focus on neuroblastoma as a tumor which multiple investigators have confirmed in diverse models is extremely sensitive to 9-ING-41.

View Key GSK-3 Publications