Hematology

A Phase 3, Randomized, Double-blind, Placebo-controlled, Multicenter Study Of Mavorixafor In Participants With Congenital And Acquired Primary Autoimmune And Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent And/Or Serious Infections

MOA: Mavorixafor is a CXCR4 antagonist that blocks the binding of the CXCR4 ligand, SDF 1α/CXC chemokine ligand 12.

Key Eligibility:

• Diagnosis of congenital or acquired primary autoimmune and
  idiopathic chronic neutropenic disorder
• Subjects must be receiving G-CSF or other active background
  therapy for the past 12 months with ongoing infections, be on a
  stable dose and schedule for at least 4 weeks before screening,
  and maintain this regimen throughout the study
• Subjects with a diagnosis of secondary neutropenia are excluded
• Subjects with any of the following diagnoses are excluded:
  • Aplastic anemia
  • WHIM syndrome
  • Certain CNs
  • Neutropenia associated with a Duffy-null phenotype

A Phase II, Nonrandomized, Single-Arm Study of Elranatamab Outpatient Administration in Patients with Relapsed/Refractory Multiple Myeloma

MOA; Elranatamab is a bispecific antibody targeting BCMA and CD3, designed to redirect T cells to eliminate malignant plasma cells in MM.

Key Eligibility:

• Documented diagnosis of relapsed/refractory MM according to IMWG criteria
  • Must have received ≥1 prior LOT that includes both
    lenalidomide and an anti-CD38 mAb(in the same or
    separate prior lines)
• Must have measurable disease at screening
• Subjects must be able and willing to receive prophylaxis for VZV and PJP
• Subjects with the following plasma cell disorders are excluded:
  active plasma cell leukemia, Waldenström’s macroglobulinemia,
  POEMS syndrome, or primary AL amyloidosis
• Subjects with active CNS multiple myeloma involvement are
  excluded

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Elritercept (KER 050) for the Treatment of Transfusion-Dependent Anemia in Adult Participants with Very Low-, Low-, or Intermediate-Risk Myelodysplastic Syndromes (MDS) (RENEW)

MOA: Elritercept is a recombinant fusion protein designed to inhibit signaling by select TGF-β superfamily ligands, including activin A, activin B, GDF8, and GDF11.

Key Eligibility

• Diagnosis of MDS with or without RS according to WHO 2016 classification that
  meets the IPSS-R classification of very low-, low-, or intermediate-risk MDS
• Transfusion dependent (16 weeks pre-randomization)
  a) LTB: 4–7 RBC units / 16 weeks
  b) HTB: ≥ 8 RBC units / 16 weeks
  • Pre-treatment Hgb levels <10 g/dL
  • At least 2 transfusion events and 1 in each of the 2 consecutive 8 weeks
    blocks over 16 weeks
• Subjects must be refractory (≥4 weeks of treatment), intolerant (any duration of
  treatment), or unlikely to respond to ESA treatment (EPO > 200 U/L)
• Subjects must have less than 5% blasts in an evaluable bone marrow aspirate
  collected at screening
• Subjects with del(5q) MDS, therapy-related (secondary) MDS, or any known
  history of AML are excluded
• Subjects with anemia due to any other known cause are excluded

NX-5948-201: A Single-arm, Phase 2, Open-label, Multicenter Study to Evaluate NX-5948 in Adults with Relapsed/Refractory (R/R) Chronic Lymphocytic Leukemia (CLL) or Small Lymphocytic Lymphoma (SLL) Previously Exposed to a Bruton's Tyrosine Kinase Inhibitor (BTKi) and a B-cell Lymphoma-2 Inhibitor (BCL-2i)

MOA: NX-5948 (bexobrutideg) is a CTM that induces the degradation of BTK in cells through recruitment of CRBN and promotes the formation of a ternary complex of CRBN, NX-5948, and BTK.

Key Eligibility Criteria:

• Confirmed diagnosis of relapsed/refractory CLL/SLL
• ECOG ≤2
• Subjects must have prior exposure to a cBTKi, ncBTKi, and BCL-2i either in separate LOT or in combination
• Subjects must have measurable disease by CT
• Subjects previously treated with a BTK degrader are excluded
• Subjects with a known or suspected prolymphocytic
  leukemia or Richter’s transformation are excluded

A Phase 2 study to evaluate the efficacy and safety of selinexor monotherapy in subjects with JAK inhibitor (JAKi)-naïve myelofibrosis and moderate thrombocytopenia

Brief Summary: The main purpose of this study with corresponding optional expansion is to evaluate the efficacy of selinexor in JAKi-naïve participants with myelofibrosis (MF) and moderate thrombocytopenia based on spleen volume reduction (SVR). Additional efficacy and safety parameters will also be assessed during the study.

Key Eligibility Criteria:

• A diagnosis of MF or post-ET or post-PV MF
• Measurable splenomegaly during the screening period 
• ECOG Performance Status less than or equal to (<=) 2
• More than 10% blasts in peripheral blood or bone marrow (accelerated or blast phase) excluded
• Previous treatment with JAK inhibitors for MF excluded
• Previous treatment with selinexor or other XPO1 inhibitors excluded