DNAJB1-PRKACA TRIFLUORACETATE

DNAJB1-PRKACA TRIFLUORACETATE is an investigational therapeutic peptide vaccine. It is not an authorised medicine. The vaccine is being developed for patients with fibrolamellar hepatocellular carcinoma (FL-HCC), a rare type of liver cancer, and for other tumor entities that carry the same oncogenic driver fusion gene.^[2]

What is DNAJB1-PRKACA TRIFLUORACETATE?

DNAJB1-PRKACA TRIFLUORACETATE is a peptide vaccine based on the DNAJB1-PRKACA fusion transcript, a specific genetic change found in fibrolamellar hepatocellular carcinoma and some other tumors. In the clinical trial records, the vaccine is referred to as Fusion-VAC-XS15. The substance is classified under digestive system diseases in the registry, reflecting the liver cancer focus of the research.^[2] It is an investigational substance with no marketing authorisation in the available records.

How does DNAJB1-PRKACA TRIFLUORACETATE work?

The vaccine is designed to teach the immune system to recognise the DNAJB1-PRKACA fusion protein, which is a driver of cancer growth in fibrolamellar hepatocellular carcinoma. By presenting peptide fragments of this fusion protein to immune cells, the vaccine aims to induce peptide-specific T-cell responses, meaning that the body's own immune cells learn to attack tumor cells carrying this fusion.^[2] In one trial, the vaccine is combined with atezolizumab, an immune checkpoint inhibitor that blocks PD-L1, to potentially enhance the immune response against the tumor.^[1]

Which conditions is DNAJB1-PRKACA TRIFLUORACETATE studied for?

• Fibrolamellar hepatocellular carcinoma (FL-HCC): a rare form of liver cancer that typically occurs in adolescents and young adults without underlying liver disease.
• Tumor entities carrying the oncogenic driver fusion DNAJB1-PRKACA: other cancer types that have the same specific genetic fusion and may therefore respond to the vaccine.

How is DNAJB1-PRKACA TRIFLUORACETATE given?

In the clinical trials, the peptide vaccine Fusion-VAC-XS15 is given by subcutaneous injection, meaning it is injected under the skin.^[2] In the combination trial, atezolizumab (Tecentriq) is given by intravenous infusion, either as an 840 mg or 1,200 mg concentrate for solution for infusion.^[1]

What do the clinical trials show?

Two Phase 1 clinical trials are authorised for DNAJB1-PRKACA TRIFLUORACETATE. Both are interventional studies in patients with fibrolamellar hepatocellular carcinoma or other tumors carrying the DNAJB1-PRKACA fusion. The first trial, FusionVAC22_02, is testing the peptide vaccine alone as adjuvant treatment in 32 participants.^[2] The second trial, FusionVAC22_01, is testing the vaccine in combination with atezolizumab in 20 participants.^[1]

What are the trials measuring?

Both trials have the same primary objectives: to assess immunogenicity, meaning whether the vaccine induces peptide-specific T-cell responses, and to assess safety and toxicity. Safety is determined using the Common Terminology Criteria for Adverse Events (CTCAE V 5.0), a standard system for grading side effects.^[2][1] The trial with the vaccine alone also evaluates first efficacy as an exploratory objective.^[2]

Who can take part in these trials?

The trials enrol patients with fibrolamellar hepatocellular carcinoma or other tumor entities carrying the DNAJB1-PRKACA fusion transcript. The vaccine-alone trial is designed as adjuvant treatment, meaning it is given after primary treatment to help prevent the cancer from returning.^[2] Detailed eligibility criteria are available in the trial registry records.

What side effects are known?

As these are Phase 1 trials, the safety and toxicity of DNAJB1-PRKACA TRIFLUORACETATE are still being evaluated. The trials assess side effects using the Common Terminology Criteria for Adverse Events (CTCAE V 5.0), which grades adverse events by severity.^[2][1] No specific side effect data from these trials is available in the registry records at this stage.