Phase I, open label, study of B-cell Maturation Antigen (BCMA)-directed CAR-T cells in adult patients with multiple myeloma

This is a first-in-human study to evaluate the feasibility, safety and preliminary antitumor
efficacy of autologous T cells genetically engineered with a novel B-cell Maturation Antigen
(BCMA)-specific chimeric antigen receptor (CAR) and manufactured with a new process. CAR-T
cells will be investigated as a single agent in multiple myeloma

Inclusion Criteria:

- Part A: Subjects with MM who are relapsed and/or refractory to at least 2 prior
treatment regimens, including an IMiD (e.g. lenalidomide or pomalidomide), a
proteasome inhibitor (e.g. bortezomib, carfilzomib), and an approved anti-CD38
antibody (e.g. daratumumab), if available, and have documented evidence of disease
progression (IMWG criteria)

- Part A: ECOG performance status that is either 0 or 1 at screening

- Part B: Subjects with newly diagnosed multiple myeloma (NDMM) who have received a
minimum of 4 and up to 6 cycles of standard induction therapy with VRd, D-VRd, or
D-Rd, and have achieved a response of PR or better. One cycle of CyBorDex is allowed
prior to induction.

- Part B: ECOG performance status that is either 0,1 or 2 at screening

- Measurable disease as defined by the protocol

- Adequate hematological values

- Must have a leukapheresis material of non-mobilized cells accepted for manufacturing

Exclusion Criteria:

- Prior administration of a genetically modified cellular product including prior BCMA
CAR-T therapy. Patients who have received prior BCMA-directed bi-specific antibodies
or antibody-drug conjugates (ADC) are not excluded.

- Autologous HSCT within 6 weeks prior to enrollment or any prior history of allogeneic
hematopoietic stem cell transplant (HSCT)

- Chemotherapy or any concomitant anti-cancer therapies (other than protocol prescribed
lymphodepletion (LD) chemotherapy) within 2 weeks prior to apheresis

- Treatment with small molecule targeted antineoplastics within 2 weeks of apheresis
collection or 5 half-lives whichever is shorter

- Have received antibodies or immunotherapies (other than daratumumab) within 4 weeks
prior to apheresis collection. Daratumumab within 3 weeks prior to apheresis
collection.