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Positive topline results from a phase II trial of antroquinonol for patients with AML

Nov 27, 2020

On November 26, 2020, a press release on topline results from a phase II trial (NCT03823352) of antroquinonol in adult patients with acute myeloid leukemia (AML) was released, showing encouraging overall response rates and a safe tolerability profile.1


  • Is an ubiquinone derivative of the fungus Antrodia camphorate, which inhibits the enzymatic activity of isoprenyltransferases, thereby interfering with the Ras/Rho-PI3K-Akt-mTOR pathway to induce apoptosis and autophagy.
  • Received U.S. Food and Drug Administration (FDA) orphan drug designation for the treatment of AML, hepatocellular cancer, and pancreatic cancer in 2015, and is also currently being tested clinically for efficacy in the treatment of COVID-19.


Study design3

  • Phase IIa, open-label, non-randomized study evaluating the safety and efficacy of antroquinonol in 12 adult patients (18–70 years) with relapsed AML or newly diagnosed AML ineligible for intensive treatment
  • Dose: 200 mg twice daily on Day 1 for 4 weeks
  • Primary outcome: Overall response rate (complete response/complete response with incomplete count recovery [CR/CRi])
  • Secondary outcomes: % of patients alive at 4 weeks and at 24 weeks

Topline Results1

  • CR/CRi: 50%
  • 80% of patients achieved a blast cell count of < 5%
  • 90% of patients did not require blood transfusions during the trial period
  • 100% of patients were alive at Week 24
  • No serious adverse effects were reported

Based on these results, plans for future trials of antroquinonol in the frontline AML setting are ongoing and may expand to other indications, such as multiple myeloma, chronic myelogenous leukemia, and pediatric leukemia.1

  1. GoldenBiotech. Breakthrough in AML treatment: GoldenBiotech reports new drug trial of antroquinonol outperforms listing drugs in relapsed acute myeloid leukemia. Published Nov 26, 2020. Accessed Nov 27, 2020.
  2. GoldenBiotech. The mechanism of action of antroquinonol. Accessed Nov 27, 2020.
  3. Evaluate efficacy and safety/tolerability profiles of antroquinonol in acute myeloid leukemia (AML) adult patients. Updated Sep 9, 2020. Accessed Nov 27, 2020.