FDA Grants Fast Track Designation to PLT012 for Advanced Liver Cancer

The FDA granted fast track designation to PLT012 for the treatment of patients with advanced hepatocellular carcinoma (HCC), according to a news release from the agent’s developer, Pilatus Biosciences.¹ PLT012 is a first-in-class metabolic checkpoint inhibitor designed to target the CD36-fatty acid metabolic pathway, which plays a critical role in the immunosuppressive nature of the tumor microenvironment. By inhibiting CD36, the agent may restore the metabolic fitness of T cells and enhance anti-tumor immunity, offering a novel mechanism of action for patients who have progressed on standard-of-care therapies.

Previously, PLT012 received orphan drug designation as treatment for liver and intrahepatic bile duct cancers.

While the Fast Track designation highlights the potential of PLT012 to address a significant unmet medical need in advanced HCC, specific clinical efficacy data, such as objective response rates (ORR) and disease control rates (DCR), are currently being established in an ongoing phase 1 trial (NCT07337525). The designation was supported by preclinical evidence and early clinical observations indicating that metabolic reprogramming of the tumor microenvironment can yield meaningful therapeutic signals in solid tumors. Researchers are particularly focused on the agent’s ability to overcome resistance to existing immunotherapies by neutralizing the metabolic barriers that typically exhaust effector T cells in HCC.

“Receiving FDA fast track designation for PLT012 is an important milestone that reinforces the potential of our checkpoint therapy approach to transform the treatment of HCC,” stated Raven Lin, PhD, co-founder and chief executive officer of Pilatus Biosciences, in the press release.¹ “PLT012 was designed to address the metabolic adaptations that drive immune evasion in cancer. With [investigational new drug] clearance already secured and our phase 1 trial open for patient enrollment, this designation will help accelerate clinical development and advance towards delivering a novel therapeutic option for patients, both in HCC and other solid tumors where patients do not benefit from existing immunotherapies.”

The efficacy and safety of the agent are being evaluated in the previously mentioned phase 1 multicenter, open-label, dose-escalation study.² The trial is designed to determine the safety of PLT012 when administered as a monotherapy. In the dose-escalation phase, the investigators will administer the agent intravenously every 3 weeks.

Patient eligibility criteria require participants to be 18 years or older and have histologically or cytologically confirmed advanced or metastatic solid tumors. Additional requirements include an ECOG performance status of 0 or 1, at least 1 measurable lesion per RECIST v1.1, and adequate organ function. Patients with active central nervous system metastases or a history of uncontrolled autoimmune disease are excluded from the study.

Safety and tolerability serve as the primary end points for the phase 1 portion of the trial. Specifically, investigators are monitoring for the frequency, type, and severity of dose-limiting toxicities and adverse events. Secondary end points include the assessment of the agent's pharmacokinetic profile and preliminary anti-tumor activity. Early reports indicate that the metabolic targeting of CD36 with PLT012 has been manageable thus far, with ongoing monitoring to characterize the long-term safety profile in the expansion cohorts.

“Targeting CD36 represents a promising new way to reshape the tumor microenvironment. Importantly, we also start to reveal its superior activity on treating metabolic disorders. The fast track represents the beginning of an exciting chapter for Pilatus Biosciences,” added Professor Ping-Chih Ho, chair of the Scientific Advisory Board and co-founder at Pilatus Biosciences.¹ “Importantly, PLT012 has the potential to redefine how we approach the MASH-to-HCC continuum by intervening at the metabolic root of disease to treat and prevent progression.”

References

1. Pilatus Biosciences receives FDA Fast Track designation for metabolic checkpoint inhibitor PLT012 in hepatocellular carcinoma. News release. Pilatus Biosciences. February 19, 2026. Accessed February 20, 2026. https://tinyurl.com/2s3sedkw
2. A first in human study of PLT012 in participants with solid tumor cancers. ClinicalTrials.gov. Updated February 18, 2026. Accessed February 20, 2026. https://tinyurl.com/3kyhhwuz