Advanced Biologics and Biomanufacturing Program...Advanced Biologics and Biomanufacturing Program...
Transcript of Advanced Biologics and Biomanufacturing Program...Advanced Biologics and Biomanufacturing Program...
Advanced Biologics and Biomanufacturing Program
Lynne LeSauteur (Interim Program Director)
Health-Canadian Ecosystem Total health expenditures in Canada was $242B in 2017: 11.5% of GDP.
The health care system faces important challenges: • Demographics – the rise of chronic disease burden • The cost of medicines which limits accessibility
Canada (and NRC) needs to: • Invest in developing a pipeline of innovative medicines, • Support acceleration of commercialization of new medicines and vaccines • Establish effective public health ways to deliver new treatments to all Canadians.
SMEs do not have the wide-ranging expertise and expensive infrastructure needed for drug development. Instead, they rely on partnerships with translational organizations like NRC.
Biologics – The new generation of therapeutics Due to their high specificity, biologics offer effective treatments for diseases; adding to the arsenal of sophisticated targeted therapies. The biologics market share grew from 11% in 2008 to 24% in 2015. v However, the design, manufacturing, and characterization of biologics is far more complex than for synthetic drugs and the production of
biologics remains a critical and expensive step in the development process.
Cell and gene therapies – The next generation of therapeutics Cell and gene therapies are emerging as the next generation of very promising therapies to address many unmet medical needs, with disease modifying outcomes such as tissue regeneration, immune cell reprograming, and gene replacements. v However, technology advancements and innovative partnership models are needed to increase accessibility;
NRC can catalyze a national public health delivery model for new bed-side therapies.
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Some of the public health priorities • Cancer will affect 1 in 2 Canadians and results in
an economic burden of $4B; • Emerging and re-emerging infectious diseases
are a grave concern for vulnerable populations such as Indigenous peoples and have an estimated economic burden of $4.7B.
• Neuropsychiatric conditions affects 3.6M Canadians; it has some of the highest direct costs to the healthcare system and an economic burden of $11,4B;
*Economic burden data are from PHAC’s Economic Burden of Illness in Canada custom report (2008)
Agenda
1. NRC-Human Health Therapeutics
2. Success of collaborations
3. Advanced Biologics and
Biomanufacturing Program
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NRC organizational structure
Information and Communications
Technologies
Emerging Technologies
Aerospace
Automotive and Surface Transportation
Aquatic and Crop Resource Development
Human Health Therapeutics
Pacific Region
West Region
Engineering Life Sciences Industrial Research Assistance Program
NRC Herzberg Astronomy and Astrophysics
Security and Disruptive Technologies
Construction
Energy, Mining and Environment
Ocean, Coastal and River Engineering
Medical Devices Ontario Region
Quebec Region
Atlantic and Nunavut Region
National Office
Research Centers
DIVISIONS
Measurement Science and Standards
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NRC 2018-19 planned budget: $1B Total revenues 2017-18 $221M ∼3, 444 employees
340
65+ 25+
5experts
projects
Departments
candidates
3sites
$35Mannual invest
HHT Research Center
5 Departments: 1. Bioprocessing 2. DSP and A 3. Immunobiology 4. Translational Biosciences 5. Operations
Recognized leaders: • Biologics and Vaccines product
process development • Neurodegenerative disorders • Immune and cancer related
diseases • Emerging infections
One-stop shop from discovery to tech. transfer
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Omics Novel drug targets Molecular modeling and design Screening and functional assays Production platforms and Bioprocessing engineering In vitro and in vivo pharmacology, efficacy and safety models Biologics and Vaccines Analytical characterizaton Technology transfer
HHT enables Translation to regulatory studies
R&D environment activities
selected candidate
CMO’s Quality Management System NRC’s Quality Management System GMP environment
activities
tech transfer
IND / CTA package support
selected process
HHT CMO
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Canadian Biomanufacturing Opportunity
Ø Sustained growth trends of biologics sales
Ø Complexity of biomanufacturing → stickiness factor for local production
Ø Major worldwide investment in mammalian cell biomanufacturing, yet shortage of CMO offer
Ø Gap in mammalian cell biomanufacturing offer in Canada
Vaccine core technologies and pipeline
Adjuvants Mucosal pathogens and bio-threat Viral infections Glycoconjugate technologies Antibody engineering
CNS core technologies and pipeline
Blood-brain barrier carriers BBB-crossing therapeutics Difficult (CNS) targets Brain beyond Barriers (BBB) Carta BBB-CNS models
Biologics core technologies and pipeline Target discovery Anti-cancer antibodies and ADCs Lead optimization Biomanufacturing and characterization
Programs Strengths
HHT is a key player of business innovation Since 2012, HHT has grown its footprint in the Canadian biopharmaceutical sector by collaborating and assisting a wide array SMEs and organizations in the sector.
Success of HHT collaborators
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• Oncolytics, Reolysin, Phase II and III (NRC improved purification process) – 2013 • Alethia, AB-16B5, Phase I trials in Canada and Russia (NRC’s antibody and cell line) – 2014 • CanSino, Ebola vaccine (NRC’s cell line) – approved by Chinese FDA in 2018 • PREVENT, Group A Streptococcus vaccine (NRC’s bioprocess) – 2016 • Forbius, AVID100 IND approved by FDA (NRC co-developed) – 2017 • Forbius AVID200 IND approved by FDA (HHT-discovered and co-developed) July 2018 • VBI Vaccines, CMV vaccine in phase I (NRC’s cell line) – 2017 • Zymeworks, ZW25, a Bi-Specific Antibody, for Cancer (NRC supported the overall development) – 2017 • VBI Vaccines, VBI-1901, vaccine for Glioblastoma in Phase I/2a (NRC’s cell line) – 2018 • HIA Vaccine licensing-2018 • Kalgene, Anti-Alzheimer’s disease biologics (combines two of HHT’s technologies that were discovered at HHT,
licensed to Kalgene and co-developed ) – CTA anticipated in 2019 HHT is also licensing non-exclusively a number of core technologies, comprising its pTT vector platform, its CHO-BRI, 293-SF and BMAbE1(A549) cells; over the past five years over 150 licenses were signed.
IND application filed for FTIH clearance
from FDA
Co-development of an antibody-based drug candidate with a strategic partner
Selection of clones for 1L production
2016 2017 2018
Genetic Design of new drug candidates
Lead Selection
Development and transfer of Analytical methods
PK ELISA developed using
NRC antibodies to support clinical development
FTIH
Clone selection for 5 L production and USP data for tech transfer
DSP data available for tech transfer
Product Formulation
defined
NRC IND-enabling studies for Drug candidate
Post-tech transfer technical support
Multidisciplinary effort with more than 75 NRC members involved
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Merged Advancec Biologics and Biomanufacturing Program Goals
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Enable innovation in biologics and product process development to accelerate biologics development in Canada and address unmet needs
Need Strategic Partnerships; Assets, technologies, gaps
Leveraging Strengths and Interests
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BBB crossing CNS assets Difficult to access targets
Adjuvants Mucosal pathogens and bio-threat Viral infections
Anti-cancer Biologics Biomanufacturing and Characterization
Strengths Interests Novel immunomodulators Bites Checkpoint inhibitors Anti-cancer vaccines New ADCs Proprietary platforms Radiotherapeutics Keep existing focus based on needs Vaccines for unmet needs Difficult to access targets Personalized therapeutics Biosimilars Biobetters Improved Biomanufacturing and characterization: de-risking, faster, lower cost
NRC differentiators
1. Integrated one-stop shop from lead identification to tech transfer
2. NRC offers agility, transparency, track record and personalized collaboration
3. Specific strength for different types of molecules (Ab, non-Ab, enzymes,
ADCs, vaccines, therapeutics that cross the BBB), pipeline of assets to co-
develop
4. Availability of various expressions systems (eukaryotic, microbial)
5. NRC proprietary cell lines
6. Rapid identification and characterization of lead molecules
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Program partnerships
Over 50 targets with panels of antibodies 10 biotherapeutics in co-development Enabling technologies Gap filling
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EDNRB ADC for co-development
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Novel anti-EDNRB antibodies developed which perform as potent ADCs in melanoma xenograft model when conjugated DNA damaging agents (PNU-159682)
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Advance Biologics and Biomanufacturing Program:
BasicResearch
TargetSelec3onandValida3on
CandidateDesignandOp3miza3on
PreclinicalDevelopment
ClinicalPhaseI&II
ClinicalPhaseIII Market
NRC - HHT
Fill the pipeline Accelerate development Enable VC funding Minimize risk Transfer enabling technologies to industry Pre-IND package
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THANK YOU Lynne Le Sauteur• Director R&D, HHT NRC Tel: (514) 283-1012 • [email protected] www.nrc-cnrc.gc.ca