2. Overview
• B-Cell Maturation Antigen (BCMA): is a genetically validated, specific antigen target expressed on plasma and
Multiple Myeloma (MM) cancer cells. It is the 2nd most prevalent antigen target in cell therapy clinical trials, after
CD19.1
• Incidence: 31,000 MM patients are diagnosed annually in US and approx. 85,000 in 7MM (2019).2 Making it the 2nd
most common hematological malignancy, after Non-Hodgkin Lymphoma.
• Standard of Care: Chemotherapy, proteasome inhibitors, immunomodulating thalidomide derivatives & CD38-
targeted antibodies.
• Unmet need: Despite significant improvements in median survival (3.5yrs to 8yrs, 2005-12)3, nearly all patients will
relapse after initial treatments, illustrating high unmet need for longer lasting, ultimately curative therapy.
• New Modalities: Novel therapeutic modalities in early and late-stage clinical development: CAR-T cell therapy;
Bispecific Antibodies and Antibody Drug Conjugates.
3. Near Term Milestones
2019 2020 2021
BLA = Biologics License Application; ORR = Overall Response Rate; CR = Complete Response Rate; PR = Partial Response; VGPR = Very Good Partial Response; DoR =
Duration of Response
GSK filed for FDA
marketing approval for
Antibody Drug Conjugate
(Dec’19): Belantamab
mafodotin, GSK-2857916,
on DREAMM-2 data, in the
2.5 mg/kg n=97:
• ORR: 31%
• CR: 3%
BMS/bluebird anticipate filing for
FDA marketing approval for CAR-
T (2h’20): ide-cel, bb2121, on
KARMMA data (ASH, Dec ‘19), in
highest dose n=54:
• ORR: 81%
• CR: 35%
• Median DoR: 11 months
J&J/Legend Bio anticipate
filing for FDA marketing
approval for CAR-T (2021):
JNJ-4528, on CARTITUDE-1
Ph1b data (ASH, Dec ‘19)
n=29:
• ORR: 100%
• CR: 69%
• Median DoR: 6+ months
Poseida anticipate filing
for FDA marketing
approval for CAR-T
(2h’20): P-BCMA-101,
Ph1 (ASH, Dec ’18)
n=19:
• ORR: 80%
• CR, PR, VGPR: 68%
• FDA Marketing Approval: GSK filed BLA for the 1st BCMA targeting therapy (Antibody Drug Conjugate) in Dec’19.
• BMS/bluebird CAR-T candidate, ide-cel, expected to follow in 2020; likely trailed swiftly by Poseida Tx and
J&J/Legend Bio with their CAR-T candidates. Amgen and Regeneron’s Bispecific Antibodies possible too.
4. CAR-T
Name Company Partner Designation Phase
Autologous
ide-cel (bb2121) BMS / Celgene bluebird bio Orphan, breakthrough II *BLA 2020
liso-cel (JCARH125) BMS / Celgene - Orphan I/II
JNJ-4528 Janssen Legend Bio (Genscript) Breakthrough I/II * BLA 2021
P-BCMA-101 Poseida Therapeutics - Orphan, RMAT I/II * BLA 2020
C-CAR088 Cellular Biomedicine - - I
bb21217 BMS / Celgene bluebird bio - I
FCARH143 BMS / Celgene - - I
MCARH171 BMS / Celgene - - I
Allogeneic
ALLO-715 Allogene - - I
• 9 key BCMA CAR-T candidates in clinical development (auto & allo).4 BMS/Celgene have leading position.
• >36 cell therapy BCMA agents in clinical development. 1 Multiple more in pre-clinical development.
• Autolus withdrew AUTO-2 (ph I/II) in 2019 and Gilead dropped Kite’s BCMA (KITE-585), both for competitive reasons.
5. Bispecific Antibodies
Name Company Partner Designation Phase
AMG 420 Amgen Boehringer Ingelheim Fast track I/II
REGN-5458 Regeneron Sanofi - I/II
CC-93269 BMS / Celgene - - I
JNJ-64007957 Janssen Genmab - I
PF-06863135 Pfizer - - I
TNB-383B Abbvie Tenebio Orphan I
AMG-701 Amgen - - I
• 7 key BCMA Bispecific Ab candidates in clinical development.5
• They bind BCMA on the cancer cell and a T-cell receptor, commonly CD3, on the T-cell to initiate a killing response.
• Some harmful side-effects are seen, but the therapy would be off-the-shelf - a significant manufacturing cost and
complexity advantage over personalised therapies such as autologous CAR-T.
6. Antibody Drug Conjugates
Name Company Partner Designation Phase
Belantamab mafodotin
(GSK-2857916)
GSK Seattle Genetics Orphan, breakthrough,
PRIME
II *BLA Dec 2019
MEDI22228 AstraZeneca - - I
CC12-997 Celgene Sutro Biopharma - I
• 3 key BCMA Antibody Drug Conjugates (ADC) candidates in clinical development.
• GSK is first to file for marketing approval with a BCMA targeting therapy (Dec 2019) in 4th line patients. Response
rates in DREAMM-2 trial are lower than in other programs, but GSK are moving to earlier line treatments and
combination therapy.6
• Fewer risks in terms of safety, delivery, durability & manufacturing compared to CAR-T and Bispecific Ab modalities.
7. Sources
1. Xin Yu et al. Nov 2019. ‘The Global Pipeline of Cell Therapies for Cancer’. Nature Reviews.
2. GlobalData; May 2019; Pharmaceutical-technology
3. Anderson, Kenneth. Feb 2012. ‘Bench-to-bedside translation of targeted therapies in multiple myeloma’. J Clin Oncol.
4. Updated from: Feb 2019 ‘BCMA – Biopharma Can’t Miss Action’. Informa.
5. Updated from: Mullard, Asher. July 2019. ’The BCMA Bonanza’. Nature Reviews.
6. Martz, Lauren. Dec 2019. ‘GSK looks to expand into MM subsets after submitting first anti-BCMA BLA’; BioCentury.
