Circio Holding ASA · OSL:CRNA
Circio Holding ASA — Targovax — Technical Presentation (February 2018)
25 slides from Circio Holding ASA's technical presentation, Feb 28, 2018
Slide 1: CMC outsourcing in small virtual biotech company
Arming the patient’s immune system to fight cancer CMC outsourcing in small virtual biotech company 28.02.2018 www.targovax.com
Slide 2: Subjects
Subjects Introduction to Targovax and technology platforms Outsourcing to CDMOs Selection of CDMO 2 www.targovax.com
Slide 3: Subjects
Subjects Introduction to Targovax and technology platforms Outsourcing to CDMOs Selection of CDMO 3 www.targovax.com
Slide 4: Targovax
Targovax Small biotech company in antigen specific cancer immunotherapy Located in Oslo and Helsinki History www.targovax.com 4
Slide 5: Targovax has two immuno-oncology programs in clinical development
Targovax has two immuno-oncology programs in clinical development o Genetically designed adenovirus ONCOS o Makes cancer antigens visible to immune system Oncolytic virus o Induces T-cells specific to patients’ tumor TG o Cocktail of synthetic peptides RAS neoantigen o Mimics cancer causing RAS neoa
Slide 6: Targovax strategy is to boost the effect of immunotherapy
Targovax strategy is to boost the effect of immunotherapy by targeting multiple aspects of the cancer immunity cycle T-cell trafficking T-cell activation T-cell tumor infiltration Cancer antigen presentation Cancer cell TG01 identification Peptide vaccine Checkpoint inhibitors Release of cancer Yerv
Slide 7: ONCOS-102 is a cancer targeting adenovirus armed with an immune stimulating transgene
ONCOS-102 is a cancer targeting adenovirus armed with an immune stimulating transgene Enhanced cancer cell Selective Immune system infection replication in booster cancer cells ∆24 bp ∆6.7K/gp19K ∆Ad5 knob E1A E3 Fiber knob ITR ITR Transgene Ad3 knob o GM-CSF transgene o Triggers innate immune respo
Slide 8: Resected pancreatic cancer is the lead indication, but all RAS mutated cancers are potential TG targets
Resected pancreatic cancer is the lead indication, but all RAS mutated cancers are potential TG targets Pancreatic Colorectal Lung cancer All mutRAS 1 cancer (resected) 2 cancer 3 (NSCLC) 4 cancers TG01 lead indication TG02 lead indication TG02 potential TG02 + TG03 ultimate Completing phase I/II Ph
Slide 9: Overview of Targovax' full clinical program
Interim data Overview of Targovax’ full clinical program Clinical, immune and safety data 2018 2019 2020 Cancer Indication H1 H2 H1 H2 H1 Resected Pancreas Ph l/II Resected Pancreas TG Planned Phase II Planned registration program Colorectal Phase lb Melanoma Phase l ONCOS-102 Mesothelioma Phase lb/
Slide 10: Subjects
Subjects Introduction to Targovax and technology platforms Outsourcing to CDMOs Selection of CDMO 10 www.targovax.com
Slide 11: Targovax strategy for CMC outsourcing
Targovax strategy for CMC outsourcing Targovax is a virtual company and will outsource: o All development work and validation for manufacture processes and analytical methods for our IMPs. o GMP manufacture and quality control, and supply to clinical trials towards commercialization. Key CDMO and co
Slide 12: Targovax uses three IMPs in the clinical programs
Targovax uses three IMPs in the clinical programs ONCOS o Genetically designed adenovirus 5 Oncolytic virus o Produced from human cancer cell line TG o Mixture of 7-8 synthetic peptides of 17 amino acids RAS neoantigen vaccine o Lyophilized drug product GM-CSF o rHuGM-CSF expressed from E.coli Immun
Slide 13: IMP supply and development for clinical trials
IMP supply and development for clinical trials EMA and FDA Advice meetings MAA/BLA Approval CLIN Phase I/II Clinical Trial Phase III Clinical Trial Timeline CMC IMP Supply Phase I/II IMP Supply Phase III Development IMP for Phase Commercial Drug Supply CMC III/Commercial Validation & Characterisatio
Slide 14: TG peptides
TG peptides Supply of TG to Phase I/II clinical trials: DS DP DP DP Specific DS Release Release Manufacture Manufacture Release Methods Methods Methods Development & manufacture of TG for Phase III clinical trials DP DP DS DS Process Release Manufacture Manufacture Development Methods DP TG Release
Slide 15: TG peptides
TG peptides The CDMO picture for TG peptides Well known middle size CDMO for DS manufacture – Long history with Targovax – Financial strong – Located in central Europe, but global company – Strong expertise in development and manufacture of peptides – Regulatory compliant to EU and FDA GMP – Strong
Slide 16: Recombinant GM-CSF
Recombinant GM-CSF Supply of GM-CSF to Phase I/II clinical trials: Commercial drug Import & product EU QP release Chinese supplier Development & manufacture of GM-CSF for Phase III clinical trials: DP DP Formulation DS Process DS Manufacture DP Development Development Manufacture DP Release Release
Slide 17: Recombinant GM-CSF
Recombinant GM-CSF The CDMO picture for GM-CSF Well known small/middle size CDMO selected for DS manufacture – Financial strong – Located in central Europe – Strong expertise in development and manufacture of recombinant proteins – Regulatory compliant to EU and FDA GMP – Contracts with other CDMOs
Slide 18: ONCOS-102 Viral vector
ONCOS-102 Viral vector Supply of ONCOS-102 viral vector to Phase I/II clinical trials: DS Other DS Other DP DP Other DP Other DP Other DS DS Release DP Release Release Release Release Release Release Manufacture Methods Manufacture Methods Methods Methods Methods Methods Methods Development & manufa
Slide 19: ONCOS-102 viral vector
ONCOS-102 viral vector The CDMO picture for ONCOS-102 Small CDMO for DS and DP manufacture – Long history with ONCOS – Located in Finland – Strong expertise on cell culture, viruses and quality control testing – Regulatory compliant to EU GMP – Strong project management – Strong involvement from Tar
Slide 20: Subjects
Subjects Introduction to Targovax and technology platforms Outsourcing to CDMOs Selection of CDMO 20 www.targovax.com
Slide 21: Selection of CDMO
Selection of CDMO Assessment of DP CDMO for TG peptides, GM-CSF and ONCOS-102 Define the strategy and goals ▪ Strategic decision with long-term consequences Define your team: CMC, QA, Regulatory, BD ▪ Identify stakeholders Search by Internet / Networks / Consultans / Other CMOs 50 CMOs Stage I Stage
Slide 22: Selection of CDMO – Stage I
Selection of CDMO – Stage I Definition of Targovax specific requirements: Operations, Technical, Quality, Business o Location o DS and/or DP o Technology and equipment o Safety, containment, e.g. biosafety levels o Capacities o Regulatory compliance (EU, US) Availability of CDMOs, e.g. ” Directory o
Slide 23: Selection of CDMO – Stage II
Selection of CDMO – Stage II Direct contact with CDMO o Emails / calls / presentations / meetings o Request for Information (RFI) based on criteria o Technical o Confirmation of data from Stage I ▪ More detail info vs requirements ▪ Premises, technology and equipment ▪ Capacities (batch sizes, new c
Slide 24: Selection of CDMO – Stage III
Selection of CDMO – Stage III Request for proposals (RFP) o Scope of services, technical data and milestones o Proposal o Commercial proposal (breakdown in all project and manufacture costs) o Design for a competitive bidding process o Proprietary technology o Master program incl. Gantt chart o Sub-
Slide 25: CDMO learnings
CDMO learnings Building trust and good working relationship Frequently communication & site visits – transparency Be present on CDMO site Stepwise approach to CDMO services - development CDMO full responsibility of sub-contractors Master Service Agreements & QA agreements Budget with additional cost