NXC-201 (formerly HBI0101) Multiple Myeloma

A Phase 1 Dose Escalation and Safety Study of NXC-201 (formerly HBI0101) CART in BCMA-Expressing Multiple Myeloma Patients

It is a phase one study with dose escalation and safety CART in BCMA- Expressing Multiple Myeloma and AL amyloidosis Patients

The intention with NXC-201 (formerly HBI0101) CART is to follow the chimeric antigen receptor T-cells (CART) approach, as for approved products, but target the B cell maturation antigen (BCMA) rather than the CD19 antigen targeted by KYMRIAHTM (tisagenlecleucel) and YESCARTATM (axicabtagene ciloleucel).

Importantly, successful results from at least three clinical trials of a BCMA targeted CAR T therapy were published (Zhao 2018, Brundo 2018, Raje 2019), with excellent results obtained for relapsed or refractory multiple myeloma (MM) patients, that validate the approach.

Inclusion Criteria:

• ≥18 years of age

  • Voluntarily signed informed consent form (ICF)

  • Eastern Cooperative Oncology Group (ECOG) performance status 0 - 2

  • Diagnosis of MM with relapsed or refractory disease and have had at least 3 different prior lines of therapy including proteasome inhibitor, immunomodulatory therapy and at least one antibody therapy.

  • Subjects must have measurable disease, including at least one of the criteria below:

    • Serum M-protein greater or equal to 0.5 g/dL
    • Urine M-protein greater or equal to 200 mg/24 h
    • Serum free light chain (FLC) assay: involved FLC level greater or equal to 5 mg/dL (50 mg/L) provided serum FLC ratio is abnormal
    • A biopsy-proven evaluable plasmacytoma
    • Bone marrow plasma cells > 20% of total bone marrow cells
    • Non secretory patient will be allowed provided they have measurable disease by PET-CT or bone marrow aspiration, as designated.

  • Women of child-bearing potential (WCBP), must have a negative serum pregnancy test prior to treatment. All sexually active WCBP and all sexually active male subjects must agree to use effective methods of birth control throughout the study

  • Recovery to ≤Grade 2 or baseline of any non-hematologic toxicities due to prior treatments, excluding alopecia and Grade 3 neuropathy

  • Ability and willingness to adhere to the study visit schedule and all protocol requirements