A Phase 2a Study of HT-6184 in Subjects With IPSS-R Very Low, Low or Intermediate Risk MDS and Anemia

Inclusion criteria

• ≥ 18 years of age.
• Signed Informed Consent Form (ICF).
• Adequate organ function.
• A documented diagnosis of MDS or non-proliferative Myelodysplastic/myeloproliferative neoplasm (MDS/MPN).
• Less than 10% bone marrow myeloblasts.
• Refractory or intolerant of, or ineligible for treatment with an erythroid stimulating agent (ESA).
• Prior ESA treatment must have been discontinued ≥ 2 weeks prior to date of study treatment.
• Eastern Cooperative Oncology Group (ECOG) score of 0, 1, or 2.
• Subjects must have symptomatic anemia.
• Subjects with NGS (Next-generation sequencing) myeloid-specific somatic gene mutation profile with ≥ 5 % quantitation of clone size by variant allele frequency (VAF).
• Women of child-bearing potential using an acceptable double-barrier method of contraception.
• Male subjects who are using an acceptable method of contraception.

Exclusion criteria

• Other causes of anemia such as iron deficiency.
• Clinically significant anemia resulting from B12 or folate deficiencies, autoimmune or hereditary hemolysis, or gastrointestinal bleeding.
• Women must not be pregnant or breastfeeding.
• Presence of concomitant intercurrent illness which, in the opinion of the Investigator, would compromise safe participation in the study.
• Secondary MDS.
• Treatment with cytotoxic chemotherapeutic agents or experimental agents for the treatment of MDS within 4 weeks of study treatment.
• Chronic use of systemic corticosteroids for comorbid or study disease condition within last 4 weeks of study treatment.
• Prior history of malignancy other than MDS.
• Subject has undergone a stem cell, bone marrow or solid organ transplant
• Subjects with positive serology for Hepatitis B Virus (HBV), Hepatitis C Virus (HCV), or Human Immunodeficiency Virus (HIV).
• Prior treatment with disease modifying agents.
• Participation in any clinical study within 90 days before the first dose of Investigational Product.
• Loss of ≥ 350 ml of blood within 90 days before the first dose of Investigational Product.