Last reviewed 2026-05-15 · How we verify

LCB01-0371 800mg, BID

LigaChem Biosciences, Inc. · Phase 2 active Small molecule

LCB01-0371 800mg, BID is a LSD1 inhibitor Small molecule drug developed by LigaChem Biosciences, Inc.. It is currently in Phase 2 development for Acute myeloid leukemia (AML), Myelodysplastic syndrome (MDS). Also known as: LCB01-0371.

LCB01-0371 is a selective inhibitor of lysine-specific demethylase 1 (LSD1) that restores gene expression silenced by aberrant histone methylation in cancer cells.

LCB01-0371 is a selective inhibitor of lysine-specific demethylase 1 (LSD1) that restores gene expression silenced by aberrant histone methylation in cancer cells. Used for Acute myeloid leukemia (AML), Myelodysplastic syndrome (MDS).

At a glance

Generic name	LCB01-0371 800mg, BID
Also known as	LCB01-0371
Sponsor	LigaChem Biosciences, Inc.
Drug class	LSD1 inhibitor
Target	LSD1 (lysine-specific demethylase 1)
Modality	Small molecule
Therapeutic area	Oncology
Phase	Phase 2

Mechanism of action

LSD1 is an epigenetic enzyme that removes activating histone marks, allowing cancer cells to silence tumor suppressor genes. By inhibiting LSD1, LCB01-0371 restores expression of genes critical for differentiation and apoptosis, particularly in acute myeloid leukemia and other hematologic malignancies. This epigenetic mechanism differs from traditional cytotoxic chemotherapy and may overcome certain drug resistance pathways.

Approved indications

Acute myeloid leukemia (AML)
Myelodysplastic syndrome (MDS)

Common side effects

Nausea
Fatigue
Diarrhea
Thrombocytopenia
Neutropenia

Key clinical trials

Primary sources

Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.

Source	Used for
ClinicalTrials.gov	Trial enrolment, design, endpoints, results

Competitive intelligence

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Frequently asked questions about LCB01-0371 800mg, BID

What is LCB01-0371 800mg, BID?

LCB01-0371 800mg, BID is a LSD1 inhibitor drug developed by LigaChem Biosciences, Inc., indicated for Acute myeloid leukemia (AML), Myelodysplastic syndrome (MDS).

How does LCB01-0371 800mg, BID work?

LCB01-0371 is a selective inhibitor of lysine-specific demethylase 1 (LSD1) that restores gene expression silenced by aberrant histone methylation in cancer cells.

What is LCB01-0371 800mg, BID used for?

LCB01-0371 800mg, BID is indicated for Acute myeloid leukemia (AML), Myelodysplastic syndrome (MDS).

Who makes LCB01-0371 800mg, BID?

LCB01-0371 800mg, BID is developed by LigaChem Biosciences, Inc. (see full LigaChem Biosciences, Inc. pipeline at /company/ligachem-biosciences-inc).

Is LCB01-0371 800mg, BID also known as anything else?

LCB01-0371 800mg, BID is also known as LCB01-0371.

What drug class is LCB01-0371 800mg, BID in?

LCB01-0371 800mg, BID belongs to the LSD1 inhibitor class. See all LSD1 inhibitor drugs at /class/lsd1-inhibitor.

What development phase is LCB01-0371 800mg, BID in?

LCB01-0371 800mg, BID is in Phase 2.

What are the side effects of LCB01-0371 800mg, BID?

Common side effects of LCB01-0371 800mg, BID include Nausea, Fatigue, Diarrhea, Thrombocytopenia, Neutropenia.

What does LCB01-0371 800mg, BID target?

LCB01-0371 800mg, BID targets LSD1 (lysine-specific demethylase 1) and is a LSD1 inhibitor.

Drug class: All LSD1 inhibitor drugs
Target: All drugs targeting LSD1 (lysine-specific demethylase 1)
Manufacturer: LigaChem Biosciences, Inc. — full pipeline
Therapeutic area: All drugs in Oncology
Indication: Drugs for Acute myeloid leukemia (AML)
Indication: Drugs for Myelodysplastic syndrome (MDS)
Also known as: LCB01-0371
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