Coiled Therapeutics completes enrolment in two Phase I AO-252 cohorts testing a new lipid-based formulation at 120 mg BID and 160 mg QD.
Written By: Creola Gonsalves, MS Biotech
Reviewed By: Pharmacally Editorial Team
Coiled Therapeutics has completed enrolment across two initial cohorts evaluating a new lipid-based softgel formulation of AO-252, an oral, brain-penetrant TACC3 protein-protein interaction inhibitor. The Phase I study (NCT06136884) has enrolled three patients at 120 mg twice daily (BID) and three at 160 mg once daily (QD), with all six patients progressing through the protocol-specified assessment period.
The new formulation follows an earlier clinical signal from the tablet formulation, which produced an 80% clinical benefit rate (CBR) among five patients receiving BID dosing, compared with 40% among five patients receiving QD dosing. The softgel formulation is being evaluated to improve absorption consistency and produce more predictable systemic drug exposure.
Mechanism and Formulation Rationale
AO-252 inhibits TACC3, a protein involved in cellular processes including mitosis and the DNA damage response. By disrupting TACC3 protein interactions, the compound is being developed as a potential treatment for genetically defined cancers.
The Phase I programme is evaluating adults with advanced solid tumours, including patients with or without brain metastases. The broader development programme focuses on genetically defined disease, with planned expansion cohorts in ovarian and prostate cancer.
The new formulation uses a pre-dissolved, lipid-based softgel system intended to reduce variability in oral absorption, improve bioavailability and support more predictable, dose-proportional exposure. This approach allows investigators to assess sustained dose levels while refining the relationship between dose and systemic drug exposure.
Phase I Cohort Setup and Dosing
The ongoing Phase I study is evaluating the safety, tolerability, pharmacokinetics (PK) and preliminary clinical activity of AO-252.
The newly enrolled patients are being assessed in two dose cohorts:
- 120 mg BID: 3 patients
- 160 mg QD: 3 patients
The two regimens were selected based on clinical and pharmacologic observations from earlier tablet dosing. The previous 80% CBR observed in the BID cohort provided an early signal supporting further investigation of exposure and dosing frequency.
However, these efficacy findings came from only five patients in each dosing group and remain preliminary. Larger cohorts and longer follow-up will be needed to establish the reproducibility and clinical relevance of the observed activity.
All six patients enrolled with the new formulation are currently progressing through the protocol-defined assessment period. The company expects initial safety and PK data from the new formulation by the end of 2026.
Expansion Plans and Milestones
Coiled Therapeutics expects to dose up to 30 patients with the new formulation by the end of 2026 as the programme advances towards planned Phase I/II dose-expansion cohorts.
The expansion programme is expected to focus on ovarian and prostate cancer. Data from the current formulation cohorts will help inform dose selection, dosing frequency and the exposure profile for subsequent clinical development.
Sridhar Vempati, chief executive officer of Coiled Therapeutics, said enrolment across both the 120 mg BID and 160 mg QD cohorts marks progress in the company’s dose-escalation strategy. He also highlighted the objective of achieving more consistent drug exposure with the new formulation while maintaining the development timeline towards the planned expansion cohorts.
The immediate priorities are completion of the protocol-specified assessment period, generation of initial safety and PK data, and continued enrolment towards the company’s target of up to 30 patients with the new formulation by year-end.
If the formulation produces the expected exposure profile and remains clinically tolerable, the data will support dose selection for the planned Phase I/II expansion programme in ovarian and prostate cancer.
Reference
Successful Enrolment Completed Across Two Cohorts of New AO-252 Formulation, Coiled Therapeutics, 21 September 2026
About the Writer
Creola Gonsalves (Linkedin) is an M.S. Biotechnology postgraduate with a strong interest in clinical research, evidence interpretation, and healthcare writing, with a focus on translating life-science knowledge into meaningful real-world insights.
She is trained in Good Clinical Practice (GCP), clinical research principles, and critical interpretation of randomized clinical trials, with certifications from NIH and Stanford University.
Her research background in biotechnological applications and microbial research strengthens her ability to understand scientific evidence and develop clear, accurate, and research-driven healthcare content.