50 µm50 µm 50 µm

0 140

50

100

150

200

0

5000

10000

15000

200005B-5

Days Post-BPX-601

Cells

/mcl BPX-601

(copies/µgD

NA)

Rim

7

0 140

200

400

600

0

10000

20000

300005B-2

Days Post-BPX-601

Cells

/mcl BPX-601

(copies/µgD

NA)

Rim

7

−4 −2 0 2 4

05

10

CXCL10CXCL11IDO1CXCL9GBP4CCL8CCL7IFIT3CCL5GBP1

PSCAEIF5AL1PLA2G2AFGF18NCAM1WNT11TNFRSF11BTNFRSF10CBAMBIEGF

Pa�ent 5B-5

−4 −2 0 2 4

05

10

C5TDO2ARG1HLA-DRB5IL4CXCL13SERPINA1HSDB11B1C2APOE

HLA-DPA1SPP1EROA1CCL3/L1COL17A1IL1BLAMC2MMP1TREM1WNT7B

Pa�ent 5B-4

Average Expression−4 −2 0 2 4

05

10

HLA-DQA1CDC25CHLA-DRB5ICOSOTOAP4HA2IL10CBLCFASLGLAG3

IRF8RUNX3ZAP70NCAM1FGF13PNOCJAG2ARNT2IL34EGF

Pa�ent 5B-2

Log2

FC(P

re/P

ost)G-CSF GM-CSF

IL−17A

IL−1RA

IL−1β

IL−7

IL−8 MCP−1 MIP−1β

TNF-α

VEGF

IFN-γ

IL−10

IL−5

IL−6

IP−10

IL-2

Log(

pg/m

l)-Ba

selin

e

Base

line

Day

0−Pr

eDa

y 0−

1hDa

y 0−

4hDa

y 1

Day

2Da

y 4

Day

7Da

y 7

(Rim

+1 h

)Da

y 7

(Rim

+4 h

)Da

y 8

Day

14Da

y 21

Day

28

Day

10

Base

line

Day

0−Pr

eDa

y 0−

1hDa

y 0−

4hDa

y 1

Day

2Da

y 4

Day

7Da

y 7

(Rim

+1 h

)Da

y 7

(Rim

+4 h

)Da

y 8

Day

14Da

y 21

Day

28

Day

10

Base

line

Day

0−Pr

eDa

y 0−

1hDa

y 0−

4hDa

y 1

Day

2Da

y 4

Day

7Da

y 7

(Rim

+1 h

)Da

y 7

(Rim

+4 h

)Da

y 8

Day

14Da

y 21

Day

28

Day

10

Base

line

Day

0−Pr

eDa

y 0−

1hDa

y 0−

4hDa

y 1

Day

2Da

y 4

Day

7Da

y 7

(Rim

+1 h

)Da

y 7

(Rim

+4 h

)Da

y 8

Day

14Da

y 21

Day

28

Day

10

Base

line

Day

0−Pr

eDa

y 0−

1hDa

y 0−

4hDa

y 1

Day

2Da

y 4

Day

7Da

y 7

(Rim

+1 h

)Da

y 7

(Rim

+4 h

)Da

y 8

Day

14Da

y 21

Day

28

Day

10

0.0

0.5

1.0

1.5

2.0

−0.5

0.0

0.5

1.0

1.5

0.0

0.5

1.0

1.5

0.0

0.5

1.0

−0.5

0.0

0.5

−0.5

0.0

0.5

0.0

0.5

1.0

1.5

2.0

−0.5

0.5

1.5

2.5

−0.6

−0.2

0.2

0.6

0.0

0.4

0.8

1.2

0.0

0.5

1.0

1.5

0.0

0.2

0.4

0.6

0.0

0.5

1.0

1.5

0.0

0.5

1.0

−0.4

0.0

0.4

−0.2

0.0

0.2

0.4

−1.0

−0.5

0.0

Prostate Stem Cell An�gen (PSCA) is a cell surface protein overexpressed in approximately 50-80% of pancrea�c cancers.1-3 BPX-601 is an autologous GoCAR-T cell therapy engineered to express a PSCA-CD3ζ CAR and the inducible MyD88/CD40 (iMC) coac�va�on domain. Ac�vated by the ligand rimiducid (Rim), iMC is designed to boost CAR-T performance in solid tumors. The safety and ac�vity of BPX-601 ac�vated with Rim in PSCA+ metasta�c pancrea�c cancer is currently being assessed in a Phase 1/2 clinical trial, BP-012 (NCT02744287).Phase 1 of BP-012 is a 3+3 dose escala�on of BPX-601 (1.25-5 x 106 cells/kg) administered on Day 0 with a single, fixed-dose of Rim (0.4 mg/kg) on Day 7 in subjects with previously treated PSCA+ metasta�c pancrea�c cancer. A total of 18 subjects have been enrolled and treated with BPX-601. Fourteen subjects received rimiducid on Day 7 following cell infusion. Cohorts 0 (n=3), 3 (n=3), 4 (n=3) and 5A (n=4) received single dose of Cy lymphodeple�on (LD). Cohort 5B (n=5) subjects re-ceived Flu/Cy LD over 3 days followed by BPX-601 (5 x 106 cells/kg) and Rim (0.4 mg/kg) on Day 7. The current study presents a brief biomarker summary analysis from all cohorts (n=18) of BPX-601 cell prolifera�on and persistence as well as the produc�on of several CAR-T-associated cytokines (IFN-γ, IL-6, IP-10, and GM-CSF). An in-depth biomarker analysis of Cohort 5B (n=5) was per-formed, including serum cytokine profiles (n=5), tumor infiltra�on by GoCAR-T cells and differen-�al gene expression from available tumor biopsies collected (n=3).

Stalk

Linker

VH VL

Rimiducid

QStalk

Linker

VH VL

Rimiducid

Q

BP-012 STUDY DESIGN

PATIENTS

BIOMARKER SAMPLING TIMELINE

Table 1. BP-012 Pa�ent Demographics and Clinical Characteris�cs

Cohort 0: Lead-in safety cohort with cells only and no rimiducidCohorts 3-5B: Increasing cell doses based on a 3+3 dose escala�on design followed by fixed dose of rimiducid on Day 7Cohort 5C: Maximum cell dose (5 x 106 cells/kg) followed by rimiducid on Day 7 and every 7 days therea�er un�l unacceptable toxicity or progression of disease.

Cy, cyclophosphasmide; Flu, fludaradine; PSCA, prostate stem cell an�gen; RDE, recommended dose for expansion

Current Status of Phase 1: enrollment ongoing in Cohort 5C (mul�-dose rimiducid)

Cohort 5B:5 x 106 cells/kg

PSCA+ advanced pancrea�c

cancer

Cohort 0:1.25 x 106 cells/kg

Cohort 3:1.25 x 106 cells/kg

Cohort 5C:5 x 106 cells/kg

Cohort 5A:5 x 106 cells/kg

Cohorts 3, 4, 5:

Single-doserimiducid0.4 mg/kgCohort 4:

2.5 x 106 cells/kgCohort 4:

2.5 x 106 cells/kg

Phase 2

Rou�ne safety and efficacy evalua�ons

up to 60 monthsDay 0 Day 7

Cohorts 0, 3, 4, 5ALymphodeple�on

(Cy)Apheresis

Cohort 5C:Mul�-dose rimiducid

0.4 mg/kgCohorts 5B, 5C

Lymphodeple�on(Flu+Cy)

Every 7Days

Cy: Day -3Flu/Cy: Days -5, -4, -3

RDE

Phase 1

Tumor Infiltra�on and Cytokine Biomarkers of Prostate Stem Cell An�gen (PSCA)-Directed GoCAR-T® Cells in Pa�ents with Advanced Pancrea�c TumorsJoanne Shaw,1 Brandon Ballard,1 Xiaohui Yi,1 Aditya Malankar,1 Ma�hew R. Collinson-Pautz,1 Kwame Okrah,1 Carlos R. Becerra,2 Paul Woodard,1 Aaron E. Foster11Bellicum Pharmaceuticals, Inc., Houston, TX; 2Baylor University Medical Center, Dallas, TX. Corresponding Author: afoster@bellicum.com

GoCAR-T CELLS INFILTRATED TUMOR METASTASES

• BPX-601 GoCAR-T cells exhibited enhanced survival and persistence up to 9 months.• Ac�va�on of BPX-601 GoCAR-T cells mediated upregula�on of immunomodulatory cytokines in pa�ents.• BPX-601 GoCAR-T cells infiltrated metasta�c pancrea�c tumors.• Changes in tumor microenvironment gene expression consistent with a produc�ve CAR-T cell immune response were observed in pa�ents treated with BPX-601 GoCAR-T cells ac� vated with rimiducid.• Pa�ents are currently being enrolled in BP-012 study Cohort 5C to assess the safety and efficacy of repeated BPX-601 ac�va�on with weekly rimiducid administra�on.

CONCLUSIONS

Figure 7. Differen�al gene expression in response to treatment with BPX-601 and rimiducid were evaluated in available biopsies from cohort 5B pa�ents (n=3) using the Nanostring PanCancer IO 360 panel. (A) The log fold change in gene expression between the baseline and on-treatment samples was evaluated and consistent changes were observed in all pa�ents (n=3, p < 10%). Genes highlighted in red are poten�ally associated with CAR-T ac�vity. (B) The rela�ve contribu�on from genes in defined func�onal categories to differen�al gene expression observed in all 3 pa�ents. Upregulated genes were most associated with immune cell localiza�on to tumors, while downregulated genes were most associated with myeloid cell ac�vity. (C) Individual gene expression profiles for each pa�ent evaluated (n=3). Upregula�on of interfer-on-responsive genes was observed in pa�ent 5B-5, together with genes associated with effector T cell func�on and downregula�on of PSCA which is indica�ve of a produc�ve, effector T cell response. Pa�ent 5B-5 also had the highest levels of tumor infiltra�ng BPX-601 cells (Figure 6B-C). (D) Func�onal annota�on of individual pa�ent gene expression profiles revealed a diverse pa�ern of func�onal a�ributes.

A B

C

TLR5

MFG

E8IL

16P4

HA1

HEY1

CXCR

3BN

IP3

MAG

EA1

IL6R

TNFS

F9U

BA7

TNFR

SF9

LILR

A3CD

247

TSLP

IFI3

5CD

7TN

FRSF

18M

AGEA

4ST

AT1

BLK

TWIS

T2SI

GLEC

1IL

21R

PLA1

AGZ

MB

CASP

1IL

10BP

X-60

1HS

D11B

1TD

O2

HLA−

DRB5

−2−1

01

23

4

Cohort 5B Differen�ally Expressed Genes (n=3)

Log2

FC(P

re/P

ost) Downregulated Genes

2 2. 2%

3 3. 3%1 1. 1%

1 1. 1%

2 2. 2%

Upregulated Genes

2 3. 1%

1 8. 5%

1 2. 3%1 2. 3%

1 0. 8%

7 .7%

7 .7%

7 .7%

D

Immune Cell Localization to TumorsT Cell Priming and ActivationKilling of Cancer CellsMyeloid Cell ActivityCommon Signaling Pathways

Pa�ent 5B-2

4 1. 7%

8 .3%8 .3%

1 6. 7%

2 5%

Pa�ent 5B-2

2 0. 0%

2 0. 0%1 3. 3%

1 0. 0%

1 0. 0%

1 3. 3%

1 0. 0%

Pa�ent 5B-5

3 3. 3%

1 6. 7%

1 6. 7%

2 5%

8 .3%

Pa�ent 5B-5

1 8%

1 2. 8%

2 0. 5%1 8%

1 2. 8%

1 2. 8%

Immune Cell Localization to TumorsT Cell Priming and ActivationKilling of Cancer CellsMyeloid Cell ActivityCancer Antigen PresentationRecognition of Cancer Cells by T CellsStromal FactorsTumor-Intrinsic Factors

Pa�ent 5B-4

2 2. 2%

2 2. 2%

1 1. 1%2 7. 8%

1 6. 7%

Pa�ent 5B-4

2 3. 3%

1 0. 0%

1 0. 0%2 6. 7%

1 0. 0%

1 0. 0%

6 .7%

Upregulated Genes

Downregulated Genes

CD3+ T Cell LevelsA C D

Figure 6. Biopsies taken at baseline and on-treatment (Day 14-21) from lymph node (5B-2), liver (5B-4) and omentum (5B-5) were stained for CD3 (IHC), BPX-601/CAR (RNAScope ISH), and PSCA (RNAScope ISH). (A) Percent CD3+ lymphocytes in tumor stayed rela�vely the same between baseline and on-treatment. (B) Infiltra�on of CAR+ cells into tumor �ssue was observed in all on-treatment specimens (n=3). (B) CAR+ cells were located both in the tumor and the stroma in all pa�ents (n=3). (C) The highest intensity of CAR staining was observed in pa�ent 5B-5, who also had the highest number of infiltra�ng CAR+ cells. (D) CAR+ cells were observed proximal to tumor, representa�ve images; CD3 (blue) and CAR (red). (E) PSCA expression was observed in baseline and on-treatment specimens, representa�ve images showing PSCA (red).

E

CAR+ Cell Infiltra�on Total Infiltra�ng CAR+ Cells

Baseline On Treatment(D14-D21)

0

20

40

60

80

100

% C

D3+ o

f Tot

al C

ells

% C

AR+ o

f Tot

al C

ells

Baseline On Treatment(D14-D21)

0

1

2

3

4

5

TumorStroma

CAR Expression Intensity

CAR(BPX-601) = redCD3 = blue

B

COHORTS 0-5B BIOMARKER OVERVIEW

GoCAR-T ACTIVATION INCREASED IMMUNOMODULATORY CYTOKINES

COHORT 5B BIOMARKER RESULTS

Figure 3. (A) Persistence of BPX-601 cells in cohort 5B pa�ents was evaluated by vector copy number (VCN) analysis. BPX-601 cells expanded in all pa�ents (n=5) and persisted up to 9 months 9. Four of 5 (80%) pa�ents has measure BPX-601 VCN at last �me point available. (B) Rapid reduc-�on and rebound in BPX-601 VCN was observed in all pa�ents (n=5) following rimiducid infusion.

0 140

50

100

150

200

0

2000

4000

6000

8000

100005B-3

Days Post-BPX-601

Cells

/mcl BPX-601

(copies/µgD

NA)

Rim

7

CD3+CD4+CD8+

VCN (right axis)

Tocilizumab (CRS)

Dexamethasone (Encephalopathy)

A

0 140

50

100

150

200

250

0

20000

40000

600005B-1

Days Post-BPX-601

Cells

/mcl BPX-601

(copies/µgD

NA)

Rim

7

0 140

50

100

150

200

0

5000

10000

150005B-4

Days Post-BPX-601

Cells

/mcl BPX-601

(copies/µgD

NA)

Rim

7

A

PROLIFERATION & PERSISTENCE ACTIVATED T CELL REDISTRIBUTION

Figure 4. (A) A rapid, transient reduc�on and rebound in the absolute number of total T cells (CD3) and CD4+ and CD8+ T cell subsets in cohort 5B pa�ents (n=5) was observed following rimiducid in-fusion, concurrent with the decrease in BPX-601 VCN. This suggests redistribu�on of ac�vated T cells from the blood stream poten�ally by vascular adhesion associated with T cell transmigra�on.

Rela�onship between Serum Cytokines and BPX-601 VCNCohort 5B (n=5)

A

Figure 5. Serum cytokine levels in Cohort 5B pa�ents (n=5) were evaluated at serial �mepoints using a mul�plex assay. (A) Analytes were grouped by immune func�on (Th1 proinflammatory, Th2 proinflammatory, Chemotrac�ve, Th17 proinflammatory, and Regulatory) and the sum of mean cytokine levels at each �mepoint was plo�ed in the stacked bars. The do�ed line represents the mean VCN for Cohort 5B (n=5). Serum cytokine levels increased at Day 4 following BPX-601 infusion and within 24 hours of rimiducid infusion, with peak cytokine levels observed concurrently with increasing VCN. This data supports rimiducid-mediated T cell ac�va�on. (B) Serum concentra�on of individual analytes normalized to baseline was plo�ed, with mean levels from all Cohort 5B pa�ents (n=5) shown in pink. Day 7 (pre-rimiducid) indicated by enlarged Yellow data points. Data from individual pa�ents are plo�ed (gray lines) along with the median and interquar�le ranges (boxes).

B

Cohort 0 1.25x106

(Cy)

Cohort 3 1.25x106

+ Rim (Cy)

Cohort 4 2.5x106

+ Rim (Cy)

Cohort 5A 5x106

+ Rim (Cy)

Cohort 5B 5x106

+ Rim (Flu/Cy)

101

102

103

104

105

BPX-

601

Peak

VCN

0 1 105 2 105 3 105 4 105

Cohort 0 1.25x106(Cy)

Cohort 3 1.25x106+ Rim (Cy)

Cohort 4 2.5x106+ Rim (Cy)

Cohort 5A 5x106+ Rim (Cy)

Cohort 5B 5x106+ Rim (Flu/Cy)

BPX-601 VCN AUC(copies/ g * day)

D0-D7

D7-D∞

BPX-601 VCN AUC

Figure 2. BPX-601 vector copy number (VCN) was quan�tated by qPCR from isolated PBMC samples. (A) Area under the curve (AUC) for BPX-601 VCN over �me was calculated by the linear trapezoidal method using PK Solver. Pa�ents without an evaluable terminal elimnia�on phase were omi�ed from the D7-Dinfinity dataset. (B) Peak BPX-601 VCN observed. (C) Serum cytokines were measured in pa�ent samples using a mul�plex Luminex assay. Each box shows weighted-mean fold-change serum concenta�on (pg/ml) from baseline for CAR-T-associated cytokines, pooling IFN-γ, IP-10, GM-CSF, and IL-6 together.

Peak BPX-601 VCNA B C Pooled Cytokine Profiles (IFN-γ + IP-10 + GM-CSF + IL-6)

GoCAR-T PLATFORM

BPX-601 GoCAR-T DESIGN

TARGET ANTIGEN: PSCA

•Small, GPI-anchored cell-surface protein of the Thy-1/Ly-6 family

•Expression observed in 50-80% of pancrea�c ductal adenocarcinomas1-3

•Low basal expression on normal prostate epithelium, urinary bladder, kidney, esophagus, stomach, and placenta3

•Low toxicity profile with PSCA-targeted an�bodies in prostate and pancrea�c cancer7,8

Figure 1. PSCA expression measured by qPCR in BP-012 pancrea�c tumor screening samples. Data as of October 15, 2019

PSCA-Targeted 1st Genera�on CAR+iMC-ac�vated GoCAR-T Cells:

Increase persistence, survival, and func�on of adop�vely transferred CAR-T cells4,5

S�mulate endogenous immunity (adjuvant effects): •Produc�on of immunomodulatory cytokines4,5

•Upregula�on of cos�mulatory molecules6

FKBP scFv CD3ζCD8αMyD88 CD40 2A QFKBPψ

INDUCIBLE COSTIMULATION 1st GENERATION CAR

LTR LTR

PSCA-CAR.CD3ζ

MyD88/CD40 Coac�va�on Domain

CD3ζ

Stalk

Linker

VH VL

Rimiducid

FKBP

CD40

MyD88

Q

FKBP

BACKGROUND

ProinflammatoryN-

F

Th1 IF

G-CSGM-CSF

Proinflammatory-5-7

Th2 ILIL

e0

-81

1

Chemotrac�vIP-1ILMCP-MIP-

-67A

-1

Th17 ProinflammatoryILIL-1IL

y

-10RA

RegulatorVEGFILIL-1

(right axis)BPX-601

TNF-αIL-2

Immune Cell Localiza�on to TumorsMyeloid Cell Ac�vityT Cell Priming and Ac�va�onKilling of Cancer CellsRecogni�on of Cancer Cells by T CellsRelease of Cancer Cell An�gensCancer An�gen Presenta�onTumor-Intrinsic FactorsStromal Factors

Upregulated (Top 10)

Downregulated (Bo�om 10)Effector T Cell Func�on

BPX-601 IV:5 x 106 cells/kg

PSCA+ advanced

PancCa

Rimiducid IV:0.4 mg/kg

D0(pre, 1hr, 4hr)

D7(pre, 1hr, 4hr)

Apheresis

Lymphodeple�on(Flu+Cy)

Days-5, -4, -3

Baseline(-14 to -1)

D2 D42D8x3

D4x3

D28 D35D21D17D14D10 x1/Mo.M2-6

M9, M12

EOT

x1

On-Treatment

= biopsy (IHC/ISH, Nanostring gene expression); added to BP-012 protocol star�ng for Cohort 5B

= blood (whole blood: VCN qPCR; serum: cytokine mul�plex, baseline-D42 only)

D1

BP-012 Cohort 5B Schedule Shown

F

BaselineOn-Treatment

(D14-21)

Tumor PSCA Expression (ISH)

PSCA = red

5B-2

5B-5

5B-4

BBPX-601 VCN Day 0 - 270 (M9) BPX-601 VCN Day 0 - 14 Absolute T Cell Counts and BPX-601 VCN Day 0 -14

BP-012101

102

102

103

104

105

106

107

PSCA ExpressionPancrea�c Tumors

(54.8% Posi�ve, n = 40/73)

BLQLOQ

CutoffPSCA

Cop

ies /

1e6

copi

es h

ACTB

le� axis

Pa�ent 5B-2 (Lymph node) Pa�ent 5B-5 (Omentum)Pa�ent 5B-4 (Liver)

TumorStroma

Pa�ent 5B-2

Pa�ent 5B-4

Pa�ent 5B-5

CD8A

PDL1

EOMES

GZMA

GZMB

PRF1

0

2

Effector T CellFunc�on Genes

4

Log2FC(Pre/Post)

Day

0-P

re

Day

0-1

h

Day

0-4

h

Day

1

Day

2

Day

4

Day

7

Day

7 (R

im+1

h)

Day

7 (R

im+4

h)

Day

8

Day

10

Day

14

Day

21

Day

280

50

100

150

200

102

103

104

105

Conc

. / B

asel

ine

(pg/

ml) BPX-601 copies/μg DN

A

5B-2 5B-4 5B-5

Adj. Normal

TumorStroma

Tota

l CAR

+ Cel

ls

0

100

200

300

400

500

77

82 119

161

38

19

212

238

5A-1

5B-1

0-10-20-3

3-13-23-3

4-1

4-34-2

5A-25A-35A-4

5B-25B-3

5B-55B-4

BaselineDay 0−PreDay 0−1hDay 0−4hDay 1Day 2Day 4Day 7Day 7 (Rim+1h)Day 7 (Rim+4h)Day 8

Day 14Day 21Day 28

Cohort0 3 4 5A 5B

0

5

10

Fold-Change(Baseline)

Day 10

Days Post-BPX-601

BPX-

601

(Cop

ies/µg

gDN

A)

0 10 20 30 40 50 60101

102

103

104

105

90 180 270LOQ

Rim

Days Post-BPX-601

BPX-

601

(Cop

ies/µg

gDN

A)

0 14101

102

103

104

105

LOQ

5B-1*

5B-25B-35B-45B-5^

Rim

7

0

2

4

65B-2

5B-55B-4

H-Sc

ore

(BPX

-601

)

Tumor Stroma

Cohort LD Regimena

BPX-601 Dose

(106 cells/kg)

Rim(Y/N) Age/Sex

# Prior Systemic

Therapiesb

Prior

Immunotherpy

History of Surgical PSCA

(copies)Best

Responsed

0 Cy 1.25N 0-1 50/F 1 N N 5,071 N/Ee

N 0-2 58/F 3 N Y 377,444 N/Ee

N 0-3 65/F 5 N N 15,494 N/Ee

3 Cy 1.25Y 3-1 59/F 2 DC vaccine

(WT1, mesothelin)

N 34,342 SD

Y 3-2 70/M 1 N N 7,367 SDY 3-3 58/F 1 N N 31,429 PD

4 Cy 2.5Y 4-1 71/F 2 N N 7,686 SDY 4-2 65/M 2 Anakinra Y 8,243 PDY 4-3 60/F 3 N N 354730 SD

5A Cy 5.0Y 5A-1 64/M 1 N N 19,533 N/Ae

Y 5A-2 61/M 2 Anakinra Y 14,238 PDN 5A-3 59/M 5 Pembro N 969,094 N/Ae

Y 5A-4 55/M 2 N N 52,797 SD

5B Flu/Cy 5.0

Y 5B-1 77/M 2 N Y 18,980 SDY 5B-2 56/M 4 N Y 201,277 PDY 5B-3 72/M 1 N N 21,099 SDY 5B-4 68/F 1 N N 54,877 PDY 5B-5 58/M 1 N N 149,110 SD

aCy dose = 1 g/m2 Day -3; Flu/Cy dose = Cy 500 mg/m2 + Flu 30 mg/m2 Days -5, -4, and -3bPrior lines of systemic therapy regardless of intentcHistory of pancreatoduodenectomydResponse Criteria as measured by RECIST 1.1; e

Pa�ent

Cohort 0 subjects not evaluable per RECIST 1.1; Subject 5A-1 NED at baseline; Subject 5A-3 off study prior to first scan due to AE associated with pro-gressive disease

Safety data for Cohorts 0-5B reported in Becerra et al., Annual Mee�ng 2019, American Society of Clinical Oncology

Cy, cyclophosphamide; Flu, fludarabine; N/A, not available; N/E, not evaluable; NED, no evidence of disease; Pembro, pembrolizumab; PD, progress-ive disease; PSCA, prostate stem cell an�gen; Rim, rimiducid; SD, stable disease.

An�-Cancer An�-CancerResec�onc

Inves�ga�onal

Table 2. Biomarker Sampling by Cohort

Rimiducid TCR Complexβα

CARCD3ζ

Prolifera�onPersistence

CAR-T Enhanced by iMC Co-ac�va�on

Ac�vatedImmune S�mula�on

&Cell Survival

Target-SpecificCytotoxicity

iMC

The GoCAR-T pla�orm incorporates the iMC coac�vator into CAR-T cells to provide powerful ac-�va�on of immune pathways, downstream of MyD88 (innate) and CD40 (adap�ve) signaling. Ac-�va�on of iMC with rimiducid promotes cell prolifera�on, persistence, secre�on of immuno-modulatory cytokines, and resistance to T cell exhaus�on4,5.

DIFFERENTIAL GENE EXPRESSION IN TUMOR MICROENVIRONMENT

*Pa�ent 5B-1 received cor�costeroids Day 8 for grade 2 encephalopathy. ^Pa�ent 5B-5 received tocilizumab Day 8 for grade 2 cytokine release syndrome

Cohort

Cohort 01.25 x 106 cells/kg

(Cy)

Cohort 31.25 x 106 cells/kg

+Rim(Cy)

Cohort 42.5 x 106 cells/kg

+Rim(Cy)

Cohort 5A5 x 106 cells/kg

+Rim(Cy)

Cohort 5B5 x 10

6 cells/kg

+Rim(Flu/Cy)

n=3 n=3 n=3 n=4 n=5BPX-601 Tracking(Vector Copy Number) √ √ √ √ √

Serum Cytokines √ √ √ √ √

Tumor Biopsy*(IHC, ISH, NanoString) NA NA NA NA √

(n=3/5)

# of Pa�ents

(Mul�plex)

*Baseline and on-treatment tumor biopsies added to BP-012 clinical protocoal star�ng for Cohort 5B

No Data

1. Argani P, et al. Cancer Res. 2001;61:4320–4324.2. Wente MN, et al. Pancreas. 2005;31:119–125.3. Abate-Daga D, et al. Hum Gene Ther. 2014;25(12):1003–1012.4. Mata M, et al. Cancer Discov 2017;7:1306-1319.5. Foster AE, et al. Mol Ther. 2017;25(9):2176–2188.6. Bellicum data on file.7. Morris MJ, et al. Ann Oncol. 2012;23:2714–2719.8. Wolpin BM, et al. Ann Oncol. 2013;24:1792–1801.

REFERENCES

The authors would like to acknowledge all pa�ents, their families, and caregivers for par�cipa�ng in this clinical trial, along with the inves�gators and their staff.

Presented at the American Society of Clinical Oncology Gastrointes�nal Cancers Symposium - January 23 - 25, 2020, San Francisco, CA

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NF-κB NFATc

IL-2