FDA has cleared Acepodia's investigational new drug (IND) application for ACE723, a GPC3-targeted dual-payload antibody-drug conjugate (ADC), enabling the company to begin a phase 1 trial in patients with unresectable or metastatic hepatocellular carcinoma (HCC), the company announced September 4, 2026.¹ ACE723 is the first candidate generated from Acepodia's proprietary Antibody-Dual-Drugs Conjugation (AD2C) platform to enter clinical development, according to the US-headquartered clinical-stage biotechnology company.¹
Key facts
- Class: GPC3-targeted dual-payload antibody-drug conjugate (AD2C platform)
- Indication studied: Unresectable or metastatic hepatocellular carcinoma (HCC)
- Regulatory status: FDA IND cleared September 4, 2026; NMPA IND submitted August 2026
- Milestone: First clinical-stage candidate from Acepodia's AD2C platform
- Preclinical basis: AACR 2025 abstract (binding affinity comparable to parental antibody)
- Trial design: Phase 1 dose-escalation; safety, PK, and preliminary activity endpoints
- Disease burden: HCC accounts for 70%+ of liver cancer cases worldwide
- Pipeline context: ACE1831 (CD20) and ACE2016 (EGFR) also in phase 1
- Geography: US and China (dual regulatory filings)
"The FDA clearance of ACE723 marks an important milestone for our AD2C platform and brings our first AD2C-generated candidate into clinical development," said Sonny Hsiao, PhD, CEO and chairman, Acepodia, in a company press release.¹ "ACE723 is designed to deliver two cytotoxic payloads to GPC3-expressing tumor cells, with the goal of addressing tumor heterogeneity and potential resistance to single-payload therapies. We look forward to evaluating its safety, pharmacokinetics and preliminary anti-tumor activity in patients with unresectable or metastatic HCC."
How does ACE723 work?
ACE723 is built on Acepodia's AD2C platform, which uses bioorthogonal click chemistry, derived from Nobel laureate Carolyn Bertozzi's research, to conjugate 2 distinct cytotoxic payloads to a single antibody without requiring antibody engineering.¹ The candidate targets GPC3, a tumor-associated antigen frequently expressed in HCC, and is designed so that its dual-payload mechanism can address tumor heterogeneity and reduce the likelihood of resistance associated with single-payload ADCs. In preclinical studies presented at the 2025 American Association for Cancer Research Annual Meeting, the anti-GPC3 AD2C candidate showed binding affinity and internalization comparable to the parental antibody, supporting its advancement toward clinical testing.²
Why is a new treatment option needed for HCC?
HCC accounts for more than 70% of liver cancer cases worldwide.¹ Although immunotherapy-based combination regimens have improved first-line treatment for patients with unresectable or metastatic disease, significant unmet need remains for patients whose disease progresses or becomes resistant to treatment.
What will the phase 1 trial evaluate?
The planned phase 1 study will evaluate the safety, tolerability, dose-limiting toxicities, pharmacokinetics, and preliminary anti-tumor activity of ACE723 in patients with unresectable or metastatic HCC. Its dose-escalation portion is expected to characterize the drug's safety profile and establish a recommended dose for subsequent development and potential expansion cohorts. Acepodia also submitted an IND application for ACE723 to China's National Medical Products Administration in August 2026 as part of the program's global development strategy.¹