FDA Approves GoldenBiotech’s IND Application of Antroquinonol for Phase II Trial on Relapsed Acute Myeloid Leukemia (AML)

May 27, 2022

Golden Biotechnology Corp. (GBC, GoldenBiotech, TPEX 4132), a leading Taiwanese bio-pharmaceutical company announces that Antroquinonol (HOCENA®), a new small molecule drug developed by Golden Biotech, has been officially notified by the US FDA for the approval of its IND application for Phase II clinical trial to treat relapsed acute myeloid leukemia (AML) in the USA.

On May 27, 2022, Golden Biotechnology Corp. received a letter of approval via CRO from the US Food and Drug Administration (US FDA) with IND NO: 157917.  

This small molecule new drug is independently developed by Golden Biotechnology Corp. with owned 100% patent rights. Since Antroquinonol (HOCENA®) has been granted by the US FDA for orphan drug designation (ODD) in the treatment of acute myeloid leukemia (AML) in 2015, if significant efficacy is achieved after the trial, GBC will apply for its marketing authorization.

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.

GoldenBiotech presented outstanding results of potential treatment for relapsed AML in ASCO 2021 for its Phase 2 trial (GHAML-2-001) in Russia which completed in 2020. 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).

At the end of the treatment, the rate of remission (CR/CRi) after 6-completed cycles was 50% and the 24-week survival rate was 100%. Transfusion independence was achieved (80%) in patients at the end of the 6th cycle treatment. In high-risk relapsed AML patients, orally administered Antroquinonol induced CRs and was well tolerated.

The significant results suggest the potential of Antroquinonol for the unmet needs of effective therapy in relapsed AML patients.