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| 8-K - FORM 8-K - Sorrento Therapeutics, Inc. | d548180d8k.htm |
 Sorrento Therapeutics, Inc. (STI)
Next-Generation
Targeted and Personalized
Cancer Therapeutics
Jefferies 2013 Global Healthcare Conference
June 6, 2013
Exhibit 99.1 |
 2
Safe Harbor Statement OTC
Symbol: SRNE Pro Forma for
Merged Sorrento + IGDRASOL
This presentation contains "forward-looking statements," as that term is defined under the
Private Securities Litigation Reform Act of 1995 (PSLRA), including statements regarding
expectations, beliefs or intentions regarding our business, technologies and products
strategies or prospects. Actual results may differ from those projected due to a number of
risks and uncertainties, including, but not limited to, the possibility that some or all of
the pending matters and transactions being considered by the Company may not proceed as
contemplated, and by all other matters specified in Company's filings with the Securities and
Exchange Commission, as well as risks inherent in funding, developing and obtaining regulatory
approvals of new, commercially-viable and competitive products and product candidates.
These statements are made based upon current expectations that are subject to risk and uncertainty
and information available to the Company as of the date of this presentation. The company does
not undertake to update forward-looking statements in this presentation to reflect actual
results, changes in assumptions or changes in other factors affecting such forward-looking
information. Assumptions and other information that could cause results to differ from those
set forth in the forward-looking information can be found in the Company's filings with the
Securities and Exchange Commission, including its most recent periodic report. We intend that
all forward-looking statements be subject to the safe-harbor provisions of the PSLRA.
|
 3
Oncology Product Candidates
G-MAB
®
Immuno-
therapy
mAbs
Cynviloq
™
Phase 3
Ready
AfDC
Next-Gen
ADC |
 4
TUMOR
Abraxane
®
ADC
(Adcetris
®
, Kadcyla™
)
Combo Therapy
(Abraxane
®
+ Avastin
®
)
The Target Oncology Markets
mAb
(Avastin
®
, Herceptin
®
, Yervoy
®
)
+ |
 5
Significant Value Drivers
Abraxis/Celgene
Genentech
Cambridge Antibody Technology
Seattle Genetics (SGEN): ~$4B Market Cap
Immunogen (IMGN): ~$1.5B Market Cap
Abraxane
®
MBC approval
Abraxis acq’d by
Celgene for $3B+
Abraxane
®
NSCLC approval
Abraxane
®
MBC sales:
$427M
Abraxane
®
PC
approval (exp’d)
2005
2010
2012
2013
Genentech acq’d by
Roche for ~$106B
Avastin
®
+
Herceptin
®
(~$12B)
Humira
®
(~$10B)
2009
2013
Adcetris
®
approval
2013
2011
KADCYLA
®
approval
2006
CAT acq’d by
AZ for $1.3B
Abraxane
®
mAbs
ADC |
 6
TUMOR
G-MAB
®
Cynviloq™
+ G-MAB
®
Next Generation Cancer Therapeutics
Cynviloq™
•
High-diversity, human Ab library
•
10+ lead mAb programs
•
Immunotherapies
(Oncology, non-oncology and rIVIG)
•
FTO and no stacking royalty
•
Paclitaxel
polymeric micelle
•
Phase 3-ready
•
Competitive vs.
Abraxane
®
•
Single source
•
Combination therapy
•
No stacking royalty
•
Single source
•
G-MAB
®
or biosimilar mAbs
•
Proprietary formulation
+
AfDC:
Antibody formulated Drug Conjugate
|
 7
INDICATION
INDICATION > TARGET
G-MAB
®
Metastatic Breast Cancer (505(b)(2) BE)*
Non-Small Cell Lung Cancer (505(b)(2) BE)*
Bladder Cancer (sNDA)
Ovarian Cancer (sNDA)
Pancreatic Cancer (505(b)(2) BE)*
Oncology > PD-L1, PD-1, other
Inflammation > PD-L1, CCR2, other
Infectious Disease > MRSA
Q4 2013
Q4 2013
H1 2015
H1 2015
AfDC
Oncology > c-Met, CXCR5, other
Q4 2013
H1 2015
*
Subject to EOP2 meeting with FDA scheduled for Q3 2013
Multiple Strategic Partnership Opportunities
H1 2014
2016
H1 2015
H2 2015
Pipeline
Cynviloq™ |
 8
Management Team
8
Henry Ji, Ph.D.
President, CEO & Director
•
Inventor of STI G-MAB
®
Technology
•
President & CEO of Stratagene Genomics
•
VP of CombiMatrix and Stratagene
CombiMatrix, Stratagene,
Stratagene Genomics,
Human Genome Sciences
Vuong Trieu, Ph.D.
Chief Scientific Officer
•
Founder and CEO of Igdrasol
•
Instrumental
in
the
approval
of
Abraxane
®
•
Co-inventor
of
IP
covering
Abraxane
®
•
Led and developed the pipeline for Abraxis Biosciences
resulting in its sale for $2.9B
Abraxis BioScience (ABI),
Celgene, AME (Eli Lilly),
Genetic Therapy Inc.
(Novartis)
George Uy
Chief Commercial Officer
•
Directed
the
highly
successful
launch
of
Abraxane
®
,
Xeloda
®
& Fusilev
®
•
Built commercial infrastructures and organizations in
startup companies
ABI, Spectrum Pharma,
Hana Biosciences, Roche
Jaisim Shah
Chief Business Officer
•
$430M sale of Elevation Pharmaceuticals to Sunovion-
Dainippon
•
$1.15B PDL-BMS global collaboration
•
$800M PDL-Biogen Idec collaboration
•
$200M sale iv Busulfex to Otsuka
Elevation, Roche, BMS,
Facet Biotech, PDL, Cytrx,
Zelos
Richard Vincent
CFO and Director
•
$430M sale of Elevation to Sunovion-Dainippon
•
Meritage Pharma option agreement with ViroPharma
($90M upfront + milestones)
•
$310M sale of Verus Asthma program to AZ
•
Elan: various acquisitions and divestitures with
aggregate values in excess of $300M
Elevation, Meritage,
Suneva Medical, Verus,
Women First Healthcare,
Elan, Phase Metrics,
Deloitte & Touche |
 9
Oncology Franchise
Phase 3
candidate
Cynviloq™ |
 10
What is Cynviloq™?
Proprietary Paclitaxel Polymeric Micelle
paclitaxel
Poly(ethylene glycol)
hydrophilic shell
Poly(D,L-Lactide)
hydrophobic core
Cynviloq™
10 |
 Mean size
80 nm
Cynviloq™
paclitaxel
polymeric micelle
Chemical
polymer:
Poly-lactide and polyethylene
glycol diblock copolymer
Mean size
130 nm
Biological
polymer:
Donor-derived human serum
albumin (HSA)
260 mg/m
2
Taxol
®
paclitaxel
Cremophor
EL
excipient:
Polyoxyethylated
castor
oil
Formulation
Generation
175 mg/m
2
Maximum Tolerated
Dose (MTD)
Evolution of Paclitaxel Therapy
11
1
2
3
Abraxane
®
-paclitaxel
300
mg/m
2
st
nd
rd
nab |
 12
Cynviloq™
Taxol
®
Abraxane
®
Cynviloq™
Advantage
Maximum
Tolerated
Dose
(mg/m
2
)
>300
175
260
Potential for
higher efficacy
Rapid reconstitution:
no foaming concerns
Convenience for
busy practices and
pharmacies
No donor-derived human
serum albumin (HSA)
No viral / prion
concerns
Convenient storage
conditions
No requirement for
controlled temp
storage
No microbial growth
Chemical polymer
Cremophor-free
Reduced side effects
Advantages vs. Competitors:
Taxol
®
& Abraxane
®
Taxol
®
is
a
registered
trademark
with
Bristol-Myers-Squibb.
Abraxane
®
is
a
registered
trademark
with
Celgene. |
 13
•
>300 mg/m²
(q3w) vs. 175 mg/m²
(Taxol
®
; weekly) and 275 mg/m
²
(Abraxane
®
; q3w)
•
Data suggestive of efficacy comparable to historic data for
Abraxane
®
and superior to historic Taxol
®
data or Standard-of-Care
•
Possible Phase 3 sNDA programs in these tumor types
•
GPMBC301. An Open-label, Randomized, Parallel, Phase 3 Trial to Evaluate
the Efficacy and Safety of Genexol-PM
®
Compared to Genexol
®
(Paclitaxel with
Cremophor EL) in Subjects with Recurrent or Metastatic BC; n=105
(Genexol-PM
®
); n=104 (Genexol
®
)
•
Interim analysis suggests ORR superior to Taxol
®
and comparable to historic
Abraxane
®
efficacy
•
Efficacy and safety data supportive of 505(b)(2) BE submission
•
Efficacy and safety data supportive of 505(b)(2) BE submission
Clinical Efficacy & Safety Summary
Cancer Center, Russia, S. Korea)
Trials
established
higher
MTD
(total
n=80;
in
US
-
Dana
Farber
Cancer
Inst.,
Russia, S. Korea)
Completed
trials
in
MBC,
NSCLC,
PC,
OC,
BC
(total
n=259;
in
US
–
Yale
Phase 2:
Phase 1:
Ongoing trial for MBC
(n=209; in S. Korea)
Phase 3:
Completed for MBC and NSCLC (total n=502)
PM-Safety:
Total number of patients across all trials: 946 |
 14
Comparison of Clinical Efficacy in MBC:
Phase 3 Trials
Overall
Response
Rate
(%)
US
approval
for
Abraxane
®
in
MBC
and
NSCLC
for
non-inferiority
against
Taxol
®
based
on
ORR
*
No
obvious
ethnic
differences
seen
between
ORR
in
trials
* Interim data from trial; OS and PFS analyses ongoing
United States
Cynviloq™
Taxol
®
Abraxane
®
CA012
Abraxane
®
CA201
Taxol
®
Taxol
®
China
South Korea*
p = 0.03
p = 0.001
p = 0.025
0
5
10
15
20
25
30
35
40
45
n=105
n=104
n=209
n=205
n=81
n=82 |
 15
Abraxane
®
nanoparticles
at concentrations above its critical
micelle concentration*
* Data on file
FBS = fetal bovine serum
Paclitaxel released
from
both
formulations
binds
to
albumin
in
the
blood
and
exploits
albumin-transport
pathway
into
the
tumor
Cynviloq™
polymeric micelles
at concentrations above its critical
micelle concentration*
Absence of nanoparticles at
physiological concentration
Absence of nanoparticles at
physiological concentration
Size (nm)
Nanoparticle Disintegration
Size (nm)
g/mL |
 16
Cynviloq™
data on file
*
Gardner et all, 2008
•
PK BE was calculated as 95% confidence interval (CI)
•
Ratio T/R (Cmax and AUCinf) within 80-125% (90% CI)
Comparison of mean non-compartmental pharmacokinetic parameters of
Cynviloq™
(T) and Abraxane
®
(R) @ 260 mg/m
2
with 30 min infusion time:
C
max
(ng/mL)
Ratio
C
max
(T)/C
max
(R)
AUC
inf
(ng*h/mL)
Ratio
AUC
inf
(T)/AUC
inf
(R)
Cynviloq
TM
(Simulated PK)
19486
99.6%
22198
109.2%
Abraxane
®
(Actual PK)*
19556
20324
Simulated PK Parameters Supportive
of
505(b)(2)
BE:
Cynviloq™
vs.
Abraxane
® |
 17
•
•
•
EOP2 meeting with FDA to discuss BE protocol and site selection
(Q3 2013)
•
Initiate BE studies (Q4 2013)
-
One cross-over study (n=66)
at dose of 260 mg/m
2
-
12 months duration
-
~$3M
•
NDA filing (H1 2015)
•
FDA approval for all current (H1 2016 for MBC and NSCLC) and
future Abraxane
®
(PC and MM) indications
STI’s
Business
Strategy
505(b)(2) BE Timeline
17
BE Registration Trial and Commercial Launch for MBC / NSCLC
EU Rights for Partnership |
 18
Cynviloq™
Phase 2 (Korea)*
n=34
#
Best Supportive Care (BSC)
Phase 2 (Japan) **/ Phase 3 (EU)***
n=23** / n=108***
2
line
260
mg/m
(cycle
1)
300
mg/m
(cycles
2-4)
q3w
Designed to prevent and
control the side effects of
cancer and its treatment
Overall Response Rate
21%
-
/ 0%***
Progression Free Survival
2.7 M
-
/ 1.5 M***
Overall Survival
6.5 M
2.3 M** / 4.3 M***
*
Invest
New
Drugs
(2012)
30:1984–1990
# advanced urothelial carcinoma patients refractory to gemcitabine and
cisplatin **
AUA-
San
Diego
May
4th-8
th
;
***
JCO
(2009):
4454-61
Summary:
•
High
unmet
need
with
no
FDA
approved
2
nd
line
drug
•
Generally well-tolerated and demonstrated clinical ORR
•
Phase
3-ready
for
development
as
2
nd
line
chemotherapy
in
patients
refractory to platinum-based therapy
Cynviloq™
Bladder
Cancer
-
vs.
Historic
BSC
Clinical
Experience:
2
2
nd |
 19
•
Registration Trial and Commercial Launch for BC
•
EU Rights for Partnership
STI’s
Business
Strategy
sNDA Timeline for Bladder Cancer
•
Phase 3 study preparation (Q3 2013)
-
No approved 2nd line treatment; ~15,000 deaths annually (US)
•
Orphan Drug Status Designation (Q4 2013)
-
7-year marketing exclusivity
-
Enhanced R&D credit
•
“First Patient In”
(H1 2014)
-
n=400 (~200 patients each arm) vs. BSC
-
18-24 months duration
-
$20M
•
505(b)(2) sNDA filing for 2nd line treatment (H1 2016)
19 |
 20
Therapeutic Antibody Engine
G-MAB
®
Immuno-
therapy
mAb
candidates |
 21
•
Proprietary amplification
–
RNA amplification for
variable domains
STI Technology
•
Very high diversity:
V
H
: 7.1 x 10
7
V
L
: 2.9 x 10
8
Combined: 2.1 x 10
16
•
High successful hit rate
Library Quality
•
FTO
•
Issued and pending
patents on G-MAB
®
library and individual
mAbs
•
No royalty burden
Intellectual
Property
G-MAB
®
Library
Difficult targets: GPCR |
 22
Potent
mAbs
Generated
From
G-MAB
®
Library
Difficult Targets:
Small Molecules
Low molecular weight antigens
(< 2,500 Da; “haptens”)
Validated and/or Hot Targets:
Medium-sized antigens
Soluble protein ligands
or cell surface receptors
Most Difficult Targets:
Large Complex Molecules
G protein-coupled receptors
(GPCRs)
•
Auto-Inducing Peptides (AIPs)
-
Methicillin-resistant S. aureus
(MRSA)
-
Clostridium difficile
(C.diff)
•
PD-L1
•
PD-1
•
c-Met
•
VEGFR2
•
EGFR
•
ErbB3
•
CCR2
•
CCR6
•
CXCR3
•
CXCR5
Size of Target Antigen |
 23
Days After Disease Induction
STI-B0201 (D1) -
prophylactic
STI-B0201 (D1) -
therapeutic
PBS
Inflammation: Anti-CCR2 mAb Efficacious in
Multiple Sclerosis (EAE) Model
Summary:
•
STI-B0201
identified
from
G-MAB
®
library
has
superior
binding
properties
to
CCR2
compared to competitor mAb
•
STI-B0201 stabilizes disease when given prior to or after onset of symptoms
mAb
Cell Binding -
hCCR2
(EC
50
–
nM)
STI-B0201 (D1)
0.17
MLN1202
21 |
 24
Immunotherapy: Anti-PD-L1 mAbs Exhibit Potent
Immune Modulation In Vitro
Summary:
STI
anti-PD-L1
mAbs
identified
from
G-MAB
®
library
at
least
as potent as competitor’s mAb (best listed in patent)
C)
Anti-PD-L1 mAbs trigger:
A)
Enhancement of T cell activation
B)
Increased interleukin-2 (IL-2)
C)
Increased interferon-
(IFN-
)
A)
B) |
 25
Immunotherapy: Anti-PD-1 mAbs Exhibit Potent
Immune Modulation In Vitro
Summary:
STI
anti-PD-1
mAbs
identified
from
G-MAB
®
library
at
least
as potent as competitor’s mAb (best listed in patent)
B)
C)
A)
Anti-PD-1 mAbs trigger:
A)
Enhancement of T cell activation
B)
Increased interleukin-2 (IL-2)
C)
Increased interferon-
(IFN-
) |
 26
Multiple Strategic Partnership Opportunities
ONCOLOGY
PD-L1
INFLAMMATION
ONCOLOGY /
INFLAMMATION
(GPCR)
INFECTIOUS
DISEASE
PD-1
c-Met
VEGFR2
EGFR
ErbB3
PD-L1
RAGE
CGRP
CCR2
CXCR3
CXCR5
MRSA
CCR6
(STI-A100X)
(STI-A110X)
(STI-A060X)
(STI-A020X)
(STI-A050X)
(STI-B010X)
(STI-B120X)
(STI-B150X)
(STI-B020X)
(STI-A120X)
(STI-B030X)
(STI-C020X)
(STI-B100X)
(STI-A0168)
H1 2015
H2 2015
G-MAB
®
Pipeline
H1 2015 |
 27
Antibody formulated Drug Conjugate (AfDC)
AfDC
Next-Gen
ADC |
 28
AfDC Advantage
Requires:
•
Prodrug synthesis
•
Linker chemistry
•
ADC internalization
Drug released
in cancer cell
Enables:
•
Single source
•
Drug formulation
•
mAb-payload combinations
Drug released
in TUMOR
28
ADC
AfDC
STI’s
Business
Strategy
•
Advance 1 AfDC Project/Year to Clinical Proof-of-Concept
•
Strategic Partnerships |
 29
Xenograft
model
–
5x10
6
U118 human glioblastoma cells
treatment
initiated
Days After Tumor Inoculation
STI-A0601 (A1)
STI-A0602 (E1)
STI-A0603 (H8)
PBS
Oncology: Anti c-Met mAbs Exhibit Potent
Anti-Tumor Activity In Vivo
Summary:
STI
mAbs
identified
from
G-MAB
®
library
all
demonstrate potent suppression of tumor growth.
0
100
200
300
400
500
600
700
800
900
1000
7
10
12
14
17
19
21
24
26
28
31
33
35 |
 30
AfDC
Opportunities
Multiple Strategic Partnership Opportunities
B-W-B (Bio-Way-Better) Candidates
EGFR
(Erbitux
®
)
Her2
(Herceptin
®
)
CD20
(Rituxan
®
)
VEGFR2
EGFR
c-Met
ErbB3
IGF-1R
CXCR5
CXCR3
Biosimilar
mAb
G-MAB
® |
 31
Positioning to Become an Oncology Leader
Cynviloq
™
G-MAB
®
AfDC
Next-Gen
ADC
Immuno-
therapy
mAbs
Phase 3
Ready |
 Sorrento
Therapeutics,
Inc.
(SRNE)
32 |
 33
Sorrento Therapeutics, Inc. (SRNE)
Next-Generation
Targeted and Personalized
Cancer Therapeutics
Contact:
Henry Ji
President and CEO
hji@sorrentotherapeutics.com
(858) 668-6923 |