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

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

This research is being conducted to asses if HT-6184 is effective in the treatment of Very Low, Low, or Intermediate Risk Myelodysplastic Syndrome (MDS) and Symptomatic Anemia.

The study includes a 28-day Screening Period followed by a 16- or 32-week Treatment Period.

Participants will be monitored at each cycle for drug tolerance, safety, and hematological response. A response assessment will occur after 16 weeks of study treatment.

Inclusion Criteria:

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

Exclusion Criteria:

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