MDS

• MDS IPSS High-Risk or Intermediate 2
• Intolerant of Hypomethylating Agents or not have responded to 4 cycles of decitabine or 6 cycles of azacidine
• Progressed after initation of HMA

HEMREF 48 - H3B-8800-101 H3 Biomedicine and Eisai

An Open-label, Multicenter Phase 1 trial to evaluate the safety, Pharmacokynetics and Pharmacodynamic of splicing modulator H3B-8800 for subjects with Myelodysplastic Syndromes.

• Low or Intermediate 1 MDS or non- proliferative CMML (WBC <12.000/ must have symptomatic anemia untransfused with hgb <10 within 8 weeks of registration or with RBC transfusion - dependence (>2 units/month) confirmed for a minimum of 8 weeks before randomization. Or patients with platelet < 50 and hgb > 10. / 28 day washout for all disease modifying therapy for MDS / Must not have received prior therapy with lenalidomide (for more than 2 months) nor eltrombopag / ECOG 0-3 /

MDS20 RV – MDS – P1-0645

Phase II Study of Lenalidomide and Eltrombopag in Patients with symptomatic Anemia in Low or Intermediate Myelodysplastic Syndrome.

• Newly diagnosed AML or previously treated and previous treated MDS.
• Patients need to be tested for TP53 status using NGS.
• Patients must have recovered from the previous therapy to Grade 1 or base line of significant toxicities and 5half-lives or 4 weeks.

HR 49 ALRN-6924-1-02

A phase 1/1b open label study to determine the safety and tolerability of ALRN-6924 alone and in combination with Cyterabine (Ara-C) in patients with Relapsed/Refractory Acute Myeloid Leukemia of Advanced Myelodysplastic Syndrome with Wild-Type TP53.

• First line treatment
• Low blast count

MDS 21

Phase 3, Randomized, Controlled, Open label, Clinical Study of Pevonedistat Plus Azacitidine versus single agent Azacitidine as First-Line treatment for patients with higher risk Myelodysplatic Syndrome, Chronic Myelomonocytic Leukemia, of Low Blast Acute Myelogenous Leukemia.