Download Pipeline Platform - Cerulean Pharma Inc

The team at Cerulean (NASDAQ: CERU) is committed
to improving treatment for people living with cancer.
We apply our Dynamic Tumor Targeting™ Platform to
create a portfolio of nanoparticle-drug conjugates (NDCs)
designed to selectively attack tumor cells, reduce toxicity
by sparing the body’s normal cells, and enable
therapeutic combinations.
Platform
Normal Vasculature
(Small Pores)
Tumor Vasculature
(Large Pores)
Dynamic tumor Targeting
•
•
•
Selective entry into tumor tissue via large pores in
blood vessel cell wall
Transport into tumor cells
Sustained payload release via chemical hydrolysis
Potential to Improve Efficacy and Safety
•
High payload concentrations in tumors while
sparing healthy tissue because our drugs are small
enough to slip through the large pores of tumor
blood vessels, but are too large to escape the small
pores of healthy blood vessels
•
Payload release from within the tumor cells
because, once inside tumor tissue, our drugs are
taken up into tumor cells
•
Enhanced therapeutic effect because the
anti-cancer payload is released slowly from the
NDC by a linker that degrades inside tumor cells
Blood
Vessel
Blood vessel cell
NDC
•
Targeted and sustained drug exposure inside
tumor cells
Released payload
•
Low tissue distribution
Normal cell
Pipeline
PRECLINICAL
PHASE 1
Combo in Relapsed
Renal Cell Carcinoma
CRLX101
2 Combos in Relapsed
Ovarian Cancer
Combo in Non-Metastatic
Rectal Cancer
CRLX301
Solid Tumors
Tumor cell
PHASE 2
PHASE 3
Our platform is a product engine that
can expand our pipeline. We intend to
develop additional oncology products
using commercially validated payloads
with our clinically validated platform. In
addition, we can partner our platform
with biopharmaceutical companies
(a) to differentiate small molecules in
development and (b) to improve the
return on investment in marketed small
molecule products.
Our Dynamic Tumor Targeting Platform has created two clinical candidates and has the potential to create additional candidates alone or with partners
CRLX101
Management
The lead candidate from our platform, CRLX101, is an NDC with a camptothecin
payload. Camptothecin is a potent chemotherapy that was too toxic to develop
commercially. CRLX101 inhibits topoisomerase 1, a commercially validated
cancer target, and HIF-1α, a master regulator of cancer survival mechanisms for
which there are no marketed products that provide durable inhibition. HIF-1α is
associated with treatment failures, and it increases in patients who receive drugs
that cut off a tumor’s blood supply (anti-angiogenic drugs) or radiotherapy. By
inhibiting HIF-1α, CRLX101 may help anti-angiogenic drugs and radiotherapy
become more effective, which is one reason why CRLX101 is well suited for
combination therapy.
Christopher Guiffre, J.D., M.B.A.
President & Chief Executive Officer
Cancer Treatments Can Lead to HIF-Mediated Treatment Failures*
Treatment Failure Reasons
Standardof
ofCare
Care
Standard
Adrian Senderowicz, M.D.
Senior Vice President & Chief Medical Officer
Alejandra Carvajal, J.D.
Vice President, General Counsel
Tiffany Crowell
Vice President, Clinical Operations
Scott Eliasof, Ph.D.
Vice President, Research
Pamela Strode, M.S.
Vice President, Regulatory Affairs
Marc Wolfgang, M.S.
Vice President, CMC and Portfolio Management
Drug/radiation
Resistance
Financials
Radiation
SoC treatments
increase
HIF-1α
Angiogenesis
Cancer
Stem Cells
$85.5 million in cash and cash equivalents as
of 6/30/15
Contact
Anti-angiogenic drugs
Metastasis
HIF-1
• CRLX101 inhibit HIF-1α**
• There are no marketed cancer products known to durably inhibit HIF-1α
• CRLX101 appears to be combinable with other cancer treatments, creating opportunities
for synergistic benefit
*a See Nature Reviews Clinical Oncology 2012, 9(7):378 for anti-angiogenesis and HIF-1a review article.
** Based on Cerulean preclinical data.
•
Appears to be generally well-tolerated in over 250 patients to date
•
Demonstrated clinical activity in multiple tumor types as both monotherapy
and in combination with other anti-cancer agents
•
Currently being studied in four combination clinical trials:
•
Randomized Phase 2 trial in combination with Avastin® in relapsed renal cell
carcinoma with Fast Track designation– follows clinical proof of principle from
Phase 1b/2 trial
• Phase 2 clinical proof of principle trial in combination with Avastin in
relapsed ovarian cancer
• Phase 1b clinical proof of principle trial in combination with weekly
paclitaxel in relapsed ovarian cancer in collaboration with the GOG
Foundation
• Phase 1b/2 clinical proof of principle trial in combination with
chemoradiotherapy in non-metastatic rectal cancer
•
Gregg Beloff, J.D., M.B.A
Interim Chief Financial Officer
CRLX101 has Fast Track designation in combination with Avastin® in metastatic
renal cell carcinoma and Orphan Drug designation in ovarian cancer
CRLX301
The second candidate from our platform, CRLX301, is an NDC with a docetaxel
payload. Docetaxel is a commercially successful oncology product that is limited
by toxicities and lack of tumor targeting. In preclinical studies, CRLX301 delivers
up to 10 times more docetaxel into tumors, compared to an equivalent dose of
docetaxel and was similar to or better than docetaxel in 7 of 7 animal models, with
a statistically significant survival benefit seen in 5 of those 7 models. Preclinical
data also show that CRLX301 had lower toxicity than has been reported with
commercially available docetaxel in similar preclinical studies. CRLX301 is in a
Phase 1 study for the treatment of advanced solid tumors.
840 Memorial Drive
Cambridge, MA 02139
617.551.9600
[email protected]
Forward -Looking Statements
Any statements in this fact sheet about our future
expectations, plans and prospects, including statements
about the clinical development of our product candidates,
statements about our estimated research and development
expenses and sufficiency of cash to fund specified use of
cash and other statements containingthewords“anticipate,”
“believe,”“continue,”“could,”“estimate,”“expect,”“hypothesize,”
“intend,”“may,”“plan,”“potential,”“predict,”“project,”“should,”
“target,”“would,”andsimilarexpressions,constituteforwardlooking statements within the meaning of The Private
Securities Litigation Reform Act of 1995.
Actual results may differ materially from those indicated
by such forward-looking statements as a result of
various important factors, including: the uncertainties
inherent in the initiation of clinical trials, availability
and timing of data from ongoing and future clinical
trials and the results of such trials, whether preliminary
results from a clinical trial will be predictive of the
final results of that trial or whether results of early
clinical trials will be indicative of the results of later
clinical trials, expectations for regulatory approvals,
availability of funding sufficient for our foreseeable
and unforeseeable operating expenses and capital
expenditure requirements and other factors discussed
in the “Risk Factors” section of our Quarterly Report
on Form 10-Q filed with the Securities and Exchange
Commission on 8/6/15 and in other filings that we make
with the Securities and Exchange Commission.
In addition, any forward-looking statements included
in this fact sheet represent our views only as of the
date of this fact sheet and should not be relied upon
as representing our views as of any subsequent date.
We specifically disclaim any obligation to update any
forward-looking statements included in this fact sheet.
Avastin® is a trademark of Genentech, Inc.
September 8, 2015