Phase 1a Study of HF1K16 Yields Promising Results Across Solid Tumors


A phase 1a study of HF1K16 for the treatment of patients with solid tumors has been completed and deemed well-tolerated.

cancer cell: © vitanovski -

cancer cell: © vitanovski -

The phase 1a study of HF1K16 (NCT05388487), a drug-encapsulated immune modulating liposome containing all-trans retinoic acid (ATRA), in solid tumors has been completed following efficacious results and a promising safety profile.1

Among 14 patients with a variety of tumor types treated in China, HF1K16 administered as a single agent was well tolerated. Only 1 dose-limiting toxicity (DLT) was observed at the highest dose level.

Findings showed that the disease control rate was approximately 35%, and the median overall survival was 8.5 months. Further, the maximum survival period exceeded 24 months, and 5 patients have remained alive for more than 10 months.

“We were especially encouraged by the outcome being correlated to the treatment duration, with the drug being tolerated for extended periods,” said Yuhong Xu, PhD, chief executive officer of HighField Biopharmaceuticals, in a press release. “One glioma patient experienced complete remission after 10 months of treatment, remained on the treatment for 2 years and is cancer-free. Another patient, with grade IV duodenal cancer, maintained stable disease for more than 5 months.”

HF1K16 is an investigational pegylated liposome formulation and unique liposome construct of ATRA that is administered by infusion. The product passes through the bloodstream and infiltrates the tumor microenvironment. ATRA, a small molecule metabolite of vitamin A, is released and starts the maturation of myeloid-derived suppressor cells.

The phase 1, open-label, dose-escalation study of HF1K16 sought to evaluate the agents tolerability, DLTs, pharmacokinetics, and preliminary efficacy in patients with refractory solid tumors.2 Patients enrolled were treated with HF1K16 at doses of 45 mg/m2, 90 mg/m2, 120 mg/m2, 160 mg/m2, and 180 mg/m2.

Enrollment was open to patients between the ages of 18 and 75 years who have been diagnosed with a histologically and/or cytologically confirmed locally advanced or metastatic solid tumor for which there is no effective standard-of-care or the patient is intolerant to the standard therapy. For patients enrolled in cohort 5, they must have been diagnosed with glioma by histology, the disease must have relapsed or progressed after previous treatment, and there must be no effective standard treatment or the subject is intolerant to standard treatment. In cohort 5, patients must also have had at least 1 lesion that can be measured in 2 dimensions, and a Karnofsky physical fitness score ≥ 60.

Patients must have at least 1 measurable lesion, an ECOG performance status of 0 or 1, a life expectancy of > 12 weeks, and male or female patients of childbearing potential must agree to use effective contraception after signing the informed consent form until 180 days after the end of the study. Moreover, premenopausal women or patients who are within 2 years after menopause were included in the trial.

The trial enrolled patients with a variety of refractory metastatic solid tumors, including gliomas, stomach, colorectal, liver, lung, and ovarian cancer.1 Patients were treated in China with HF1K16 at escalating doses.

“Given the disease state of the patients in the study, a monotherapy outcome of 35% disease control rate is impressive,” said Xu. “Moreover, we found significant changes in the patients’ myeloid cell and T-cell profiling after treatment. Because it shows a new mechanism and excellent safety profile, our next step is to explore HF1K16 in combination with chemotherapy and other immuno-oncology therapies.”

1. HighField Biopharmaceuticals HF1K16 phase 1a data suggests the new immuno-oncology drug is safe, well-tolerated and efficacious in solid tumors. News release. HighField Biopharmaceuticals. January 8, 2024. Accessed January 9, 2024.
2. Tolerability and safety of HF1K16 injection in patients with refractory solid tumors. Updated October 10, 2023. Accessed January 9, 2024.
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