GuideLineGuidelines for Selecting and Implementing Themes
and Projects
Our approach to select drug discovery themes and projects
The Program for Drug Discovery and Medical Technology Platforms (DMP) helps to construct optimal
research strategy for each theme or project required for clinical application of seeds as new
drugs and medical technologies and provides support to establish research and development plan
required for drug approval. We have guidelines (selection criteria) for the selection of drug
discovery themes and projects to be supported (Table 1).
In order to maximize the value of our future deliverable, these criteria have been set to ensure
the selection of potentially promising seeds at the start of research of themes and projects
that are deemed worthwhile as basic research with a high likelihood of successful drug
discovery. The selection of drug discovery themes is conducted by a panel of experts from RIKEN
who screen and then judge submitted proposals.
Table 1. Criteria for the selection of drug discovery themes
and projects
1. Strategic fit to DMP drug discovery
Novel drug target molecules for diseases with high unmet medical needs (e.g.
rare diseases, CNS/neurology, infectious disease, immunology), and innovative
drug discovery themes that leverage outcomes of basic research at RIKEN
Cutting-edge technologies for novel class of pharmaceutical modalities1.
modalities
: Technologies for pharmaceutical like small molecule, antibody, siRNA, proteins, gene therapy, cell therapy.
2.Novelty of research and confidence in the mechanism
Originality of mechanism or molecular target
Validity and relevance of the target mechanism to the disease
3.Likelihood of finding promising drug candidates
Concrete plan for screening cascade and methodologies for discovery
Plan for proof of concept in discovery research and also in clinical develoment
4.Differation from similar products/technologies
If there are similar products or technologies, competitive advantage and
strategy for differentiation required.
5.Leaders with strong commitment to drug discovery
Theme leaders and project leaders need to show strong commitment to drug
discovery research. Ideally the theme or project proponent is appointed as
leader, but this is not always the case, someone else being appointed where
considered appropriate.
Implementation and management of selected themes and projects
Drug discovery themes and projects selected for this program are divided into three stages—S
(seed), L (lead), and P (clinical phase), with each stage subdivided to be managed as S0 to S3,
L1 to L3, and P0 to P3. Stages S1 to L2 are referred to themes, in which selected proposals are
in the exploratory stage before candidates have not been yet identified. Theme leaders (TL)
collaborate with a portfolio manager (PFM) who manages multiple themes to move them forward.
Stages from L3 onward are for themes that have advanced far enough to be called as projects.
Project leaders are assigned to projects to drive the subsequent development of the selected
candidate compound.
Small-molecule compound:
S0: Target identification and validation
S1: Screening system designed, and screening assay conducted
S2: Hit compounds discovered and developed into seeds
S3: Promising drug discovery seeds identified and taken to lead stage
L1: Promising lead compounds discovered through animal testing and subsequently optimized
L2: Several promising compounds identified and optimized for non-GLP study
L3: Non-GLP study to select P0 candidate compounds
P0: GLP IND-enabling study to select P1 candidate compounds
P1/P2/P3: Corresponding to Phases I/II/III in clinical trials
Criteria for progressing to the next stage are also set for other modalities such as antibodies,
processed cell products, and gene therapies. Particularly for progressing to important stages,
these criteria are defined in detail. All themes and projects are subject to the same process of
promotion to the next stage based on the above requirements, but we periodically discuss all
themes and projects in the program pipeline to determine which if any are worthy of
prioritizing, and resources are allocated to the most promising themes and projects to
accelerate development.
Many requirements need to be met in the process of drug discovery to create a final drug product
(see the figure below). In DMP, we have brought together many drug discovery experts and have
established a structure to support you to pursue drug discovery themes and projects. We look
forward to working with you.
Contact:
Program for Drug Discovery and Medical Technology Platforms, RIKEN Cluster for Science,
Technology and Innovation Hub (RCSTI)
Email: pharma-support “at” riken.jp (Replace the “at” with @ when emailing us.)