Pipeline Program

ANN-01M

ANN-01M is a bispecific antibody in pre-clinical development for BCMA-positive multiple myeloma.

Indication: Multiple Myeloma Status: Late Research and Development / Pre-clinical
ANN-01M
Concept illustration of the ANN-01 platform in development.

Multiple Myeloma Disease Landscape

Multiple myeloma is a malignant plasma cell disorder that accounts for ~10% of hematologic cancers and ~2% of all cancers. The American Cancer Society estimates ~36,110 new U.S. diagnoses and ~12,030 deaths in 2025—highlighting substantial unmet need despite progress with proteasome inhibitors, IMiDs, anti-CD38 mAbs, and BCMA-directed therapies.

Patients typically experience cycles of remission and relapse, cumulative toxicity, and end-organ damage (bone, renal). Outcomes are especially poor in triple-class-refractory disease, where survival can be measured in months. Differentiated, off-the-shelf mechanisms that do not depend on T-cell fitness remain a priority for later-line care.

Incidence Snapshot

U.S. annual cases (2025 est.) ~36,110 new diagnoses; ~12,030 deaths (American Cancer Society).
Global burden GLOBOCAN 2022: ~188k new cases; ~121k deaths; ~539k 5-year prevalent cases worldwide.
Median age at diagnosis 69 years; most diagnoses occur at ages 65-74 (SEER, 2018-2022).

BCMA as a Precision Anchor

BCMA (B-cell maturation antigen) is found on mature B cells and plasma cells, where it promotes survival signals that myeloma cells exploit.

While BCMA-directed CAR-T and bispecific therapies have proven the target’s value, they face challenges like T-cell exhaustion and limited accessibility. ANN-01M offers an immune-independent approach that preserves BCMA as a target while reducing systemic toxicity and simplifying development.

ANN-01M Program & Research Plan

Mechanism & Rationale

  • ANN-01M bispecific escorts endogenous ApoL1 into BCMA+ myeloma cells; post-internalization lysosomal pore formation delivers immune-cell–independent killing.
  • Designed as an off-the-shelf, redosable option suitable for heavily pre-treated, immunosuppressed patients.

Current Progress (pre-clinical)

  • In-vitro: BCMA-dependent binding, rapid internalization, ApoL1-redirected killing in BCMA+ lines; no activity in BCMA-negative controls.
  • Selectivity & Safety: Early off-target screens;

Translational Models (in flight / planned)

  • Emory/Winship access to myeloma PDX and primary samples for benchmark comparisons.
  • Transgenic Human ApoL1-capable NSG mouse models to assess efficacy and safety in a relevant background.
  • γ-secretase inhibitor in-vivo studies to evaluate mBCMA vs sBCMA effects on exposure and response.

Regulatory Workstreams

  • Focused on MoA validation, early tox signals, expanded animal model choices - gates supporting an INTERACT → pre-IND path.
  • CMC readiness: sequence/format lock, pilot expression, developability & stability screens, and CDMO down-selection for GLP tox material; business development for future GMP capacity.

Near-Term Milestones

  • Complete expanded in-vitro panel and BCMA threshold analysis; initiate PDX efficacy.
  • Finalize tox strategy in humanized/NSG models; progress IND-enabling study design.
  • Deliver CMC feasibility package (titre/yield, HCP/HC DNA analytics, stability screens).

Further Reading