rogocekib (CTX-712)

Although most toxicities were manageable, two treatment related deaths were observed, underscoring the need for vigilant safety monitoring. Overall, rogocekib demonstrated evidence of target engagement in most patients with solid tumors and preliminary antitumor activity, warranting further clinical investigation.

Treatment with CTX-712 reduced the phosphorylation of serine- and arginine-rich proteins in a dose-dependent manner, leading to potent in vitro cell growth suppression and in vivo antitumor activity in lung cancer NCI-H1048 xenograft model. These findings highlight the promise of CTX-712 as a novel CLK inhibitor and its potential as a therapeutic for cancers, particularly those characterized by RNA splicing alterations.

This study was registered on the Japan Registry of Clinical Trials under jRCT2080224127. Currently, a Phase I/II Study of rogocekib in relapsed/refractory AML and higher risk MDS is ongoing in the United States (NCT05732103).

P1/2, N=225, Recruiting, Chordia Therapeutics, Inc. | N=170 --> 225 | Trial completion date: Apr 2028 --> Feb 2029 | Trial primary completion date: Apr 2026 --> Jun 2028

Use of azole antifungals that are not strong CYP3A4 inhibitors (e.g., isavuconazole) is permitted. The phase 2 part of the study will commence after the RP2D has been identified and confirmed and aims at evaluating therapeutic activity in R/R AML (Cohort 2a) or R/R HR-MDS (Cohort 2b), in addition to confirmation of the safety profile. Clinical trial registry number: NCT05732103

Assessment of CTX-712 safety profile for hematologic malignancies is ongoing. CTX-712 showed preliminary anti-tumor efficacy for both AML and MDS.