Transcript Slide 1

Establishing Preclinical
Development Operations in China
Ming Guo, Ph.D.
VP Pharmaceutical Sciences & Manufacturing
SABPA Pacific Forum
Nov. 4, 2006
“Peer into the biotechnology industry's crystal ball, and the
shape you see might look a lot like Ascenta Therapeutics”
“Company is a model of efficiency for the drug development industry”
San Diego Union Tribune October 27, 2006
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Ascenta Profile
Private Oncology Development Company
– Founded in September 2003 in San Diego with IP from NCI & U. Michigan
– Now with >40 employees in San Diego, Shanghai and Philadelphia
Expert, efficient & economical oncology business model
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Ascenta team is highly experienced in oncology drug development
Early discovery is generated externally via academic alliances
Preclinical pipeline is progressed in a wholly-owned Shanghai subsidiary
Clinical trial enrolment optimized by US bicoastal site management organization
Ascenta’s apoptosis pipeline is rapidly advancing and highly competitive
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Product pipeline focused on Bcl-2, MDM2 and XIAP apoptosis targets
AT-101 is an oral pan-Bcl-2 inhibitor
AT-201 is an oral inhibitor of MDM2 entering Phase 1 in 2Q07
AT-301 is an inhibitor of XIAP entering Phase 1 in 2H07
Clinical Lead AT-101 is an oral pan-Bcl-2 Inhibitor
– The only oral pan-Bcl-2 inhibitor in clinical trials (entering Phase 3 in 2Q07)
– Single-agent clinical activity seen in first 3 tumors tested in Phase 2
– Synergy seen in combinations with other oral targeted agents & biologics
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Ascenta’s Global Reach and Efficient
Discovery-Development EDGE
Optimization &
Manufacturing
Early Preclinical
Early Discovery
Generated Externally
Discovery Alliance with
Shaomeng Wang, PhD
University of Michigan
30+ chemists
& biologists
San Diego Headquarters
Manages discovery alliances,
all IND-enabling preclinical
development & West Coast
clinical operations
Ascenta Shanghai R&D Ctr
A Wholly-owned subsidiary
15 FTEs, Chemists & Biologists
All data transferred to San Diego
nightly via Ascenta internet portal
Late Preclinical
& Clinical
Development
Philadelphia Site
Management Office Manages
all clinical trial sites in East &
Mid-West
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Ascenta Shanghai
Wholly-owned subsidiary
– WOFE status essential for success
– Inventions owned by Ascenta
– Secure data infrastructure with
warehousing in US
– Bilingual oversight from US and Shanghai
Chemistry Capabilities
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Ascenta
Shanghai
Key intermediate Synthesis
API batch scale-up (10-100 gram)
Analytical and formulation development
Formulation and stability testing
Biology Capabilities
– In vitro and in vivo screening
– Rodent and non-rodent PK coordinated
with local Shanghai contractors
– DMPK and tox assessments
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Ascenta Shanghai Laboratory
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Future Plans
To continue to build the preclinical function
– Chemistry and Discovery Manufacturing lab for synthesis of key
intermediates, scale-up of lead compounds, formulation and process
development
– In Vivo efficacy screening models
– DMPK Studies
– Screening Toxicology
IND enabling studies following US GLP and ICH guidelines
– IND Filing with SFDA in China
– IND Filing in Hong Kong and US
Clinical trials
– Proof of concept trials
– Part of Phase 3 multi-national trials
– Registry trials for China market
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Challenges
Acceptance by Regulatory agencies is uncertain
– The first to file will be scrutinized
– While the situation is improving, it will have to be closely watched
Patent protection improving but still a risk
– IP law and related judicial practices are in the early stages of
development
Clinical Development is less advanced than preclinical
A physical presence is necessary to realize benefits
– Working through US CROs decreases the cost-savings
– Close supervision will be essential to realize cost-savings & quality
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