PSTC Japan Safety Biomarker Conference Session 1. Introduction John - PowerPoint PPT Presentation

PSTC Japan Safety Biomarker Conference Session 1. Introduction John Michael Sauer, PhD Takayoshi Nishiya, PhD Executive Director, Predictive Safety Testing Consortium Senior Director, Daiichi Sankyo

Chugai Nozomi Fujisawa NIHS Yoshiro Saito RIKEN Piero Carninci Naoto Toyota Novartis Yumiko Tsujimura Masaaki Furuno C-Path Nicholas King Otsuka Yuichi Shimomura Linda Kostrencic John-Michael Sauer Masanori Hizue Todd Taylor Pfizer Daiichi Sankyo Ito Kazumi Chieko Muto Jiri Aubrecht Takeda Takayoshi Nishiya PMDA Akihiro Ishiguro Naomi Kamiguchi Mitsubishi Takuya Fujita Yasuto Otsubo Shingo Okubo Manami Miyake Taiki Sato Yusuke Sudo Toshinobu Shimuzi Thank you Planning Committee 2

Thank You to Our Host 3

Thank You to Our Co-Sponsors 4

PSTC Japan Safety Biomarker Conference Why are we here today? The objectives of the conference: • Bring together biomarker stakeholders in order to share scientific discoveries and approaches around safety biomarkers • Identify collaborative opportunities across stakeholder groups • To teach and to learn 5

PSTC Japan Safety Biomarker Conference Why are we here today? The objectives of the conference: • International alignment around the implementation of safety biomarkers in drug development • Alignment on the regulatory qualification of biomarkers in general across the three major government agencies – FDA, EMA, and PMDA 6

Agenda – Day 1 Session Chairs Time Session Proposed Session Description / Theme John Michael-Sauer (C-Path) 10:30 – 11:00 1 Introduction Takayoshi Nishiya (Daiichi Sankyo) 11:00 – 13:00 Lunch (poster session) Takayoshi Nishiya (Daiichi Sankyo) 13:00 – 15:00 2 Beyond protein biomarkers: small circulating RNA Nicholas King (C-Path) • miRNA safety biomarkers – Jiri Aubrecht (Takeda) • miRNA – Tsuyoshi Yokoi (Nagoya Univ.) • small circulating RNA – Takahiro Ochiya (Tokyo Medical Univ.) 15:00 – 15:15 Break Piero Carninci (Riken) 15:00 – 17:30 3 Cluster/network analysis of genomics for disease grouping Jiri Aubrecht (Takeda) • New diagnostic examination (biomarkers) and treatment for mental disorders by using of genomics, epigenetics and metabolomics technologies – Atsushi Takata (Yokohama City Univ.) • Modeling of the transcriptional response to multidrug treatment for prediction of positive and negative effects of combinatorial drug therapy – Erik Arner (RIKEN) • Building the Human Cell Atlas towards Medical Innovations – Jay Shin (RIKEN) • TGx-DDI – Qualification of a Preclinical Biomarker – Roland Frötschl (BfArM) 18:00 Banquet 7

Agenda – Day 2 Session Chairs Time Session Proposed Session Description / Theme Yoshiro Saito (NIHS) 09:00 – 10:30 4 Safety testing and disease diagnosis Tanja Zabka (Genentech) • GLDH clinical qualification – Jiri Aubrecht (Takeda) • Validation of drug-induced liver injury biomarkers in Japan – Noriaki Arakawa (NIHS) 10:30 – 10:45 Break Yoshiro Saito (NIHS) 10:45 – 12:00 4 Safety testing and disease diagnosis Tanja Zabka (Genentech) • Qualification of Clinical Skeletal Muscle Injury Biomarkers: An Innovative Approach – Tanja Zabka (Genentech) • Diagnostic test/tool standardization – Masato Maekawa (Hamamatsu Medical Univ.) 12:00 – 13:00 Lunch Yasuto Otsubo (PMDA) 13:00 – 15:30 5 Biomarker qualification evidentiary considerations Masanori Hizue (Pfizer • Current evidentiary considerations progress across PMDA, FDA, and EMA – Nicholas King Japan) (C-Path) • Case study: clinical and analytical validation of safety biomarkers – John-Michael Sauer (C-Path) • Survey results and perspectives on analytical validation in Japan – Yoshiro Saito (NIHS) • Qualification at PMDA – Mineo Matsumoto (PMDA) 15:30-15:45 Break Akihiro Ishiguro (PMDA) 15:45 – 16:30 6 Closing John Michael Sauer (C-Path) • Conclusions and next steps • Include all session chairs for panel discussion to summarize the conference 8

PSTC Japan Safety Biomarker Conference Since the 2017 Safety Biomarker Conference… PSTC has initiated formal interactions with PMDA to qualify the kidney • safety biomarkers for clinical use in Japan FDA qualified the kidney safety biomarker for clinical use as a composite • measure PSTC has advance the qualification of GLDH with FDA and EMA • PSTC has initiated the qualification of clinical skeletal muscle safety • biomarkers with the FDA 9

Translational Safety Biomarkers Monitorability of Drug-Induced Tissue Injury Fluid-Based Safety Biomarkers: Can be used to accurately predict drug-induced tissue injury, similar to a routine clinical pathology measure. ? 10

Gaps: The Need for Better Safety Biomarkers Current biomarker standards do not exist or they have significant limitations 11

PSTC Members and Partners Consortium Members Partners 12

Thank you www.c-path.org