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  • GoldenBiotech Presents Outstanding Results of Potential Treatment for Relapsed AML in ASCO 2021


Apr 07, 2021
GoldenBiotech Presents Outstanding Results of Potential Treatment for Relapsed AML in ASCO 2021

ASCO Meeting Library:
Company:Golden Biotechnology Corporation
Drug: Antroquinonol ( HOCENA)

The potential treatment of antroquinonol in adult patients with relapsed acute myeloid leukemia (AML).



The prognosis of relapsed acute myeloid leukemia (R/R AML) remains poor and the management is challenging. Despite the increasing availability of targeted agents, the lower responses still represent a major obstacle to overcome. Antroquinonol is an isoprenyltransferase inhibitor with antitumor activity in vitro and xenograft models of AML. Our aim is to evaluate hematopoietic recovery with Antroquinonol in this phase IIa study.

Here, the results from our phase IIa trial demonstrate the safety, tolerability, and activity of Antroquinonol in patients with relapsed AML.


GHAML-2-001 is a phase IIa, open-label study in adult patients with relapsed acute myeloid leukemia or at initial diagnosis when no intensive treatment is possible. Antroquinonol 200 mg BID was administered in a 28-day cycle (up to six cycles).

The primary endpoint is hematologic response rate. Secondary endpoints consist of 4-week survival, 24-week survival, duration of hematologic response and transfusion independence, and the response rates. Therapy was continued for as long as tolerated and there was continuing evidence of therapeutic benefit in the opinion of the investigator. Treatment response was assessed by the International Working Group (IWG) response criteria after each cycle.


Twelve eligible patients had a median age of 62.5 years (range, 42-69; 6 males, 6 females) and the whole process was conducted between March 2019 and February 2020.

Of the two patients who did not complete the therapy period were due to the patient’s decision to withdraw: One patient withdrew on day 7 and the other one withdrew on day 49.

Among other ten patients who finished all six cycles (168 days), Antroquinonol showed excellent safety and favorable tolerability.

At the end of the treatment, the rate of complete remission (CR/CRi) after 6-completed cycles was 50% (5/10) and the 24-week survival rate was 100%. Transfusion independence was achieved in 8 (80%) of 10 transfusion independence evaluable patients at the end of the 6th cycle treatment.


In high-risk relapsed AML patients, orally administered Antroquinonol induced CRs and was well tolerated.

This significant result suggests the potential of Antroquinonol for the therapy of this underserved population.

Clinical trial information: NCT03823352.

Clinical trial link : NCT03823352