New Drug Candidates for Rare Diseases

Request Number REQ4363118
Due Date February 14, 2018
Request for Proposal Details
RFP Title
New Drug Candidates for Rare Diseases
RFP Description

NineSigma, representing a Japanese pharmaceutical company specialized in rare diseases, seeks new drug candidates for the treatment of rare diseases.


Using the strength of drug discovery and clinical development in the above therapeutic area, the client strives to further strengthen its product lineup and pipeline.


Rare diseases are a therapeutic area in which unmet medical needs and a demand for new drug discovery are high. Therefore, the client has issued this open request to accelerate clinical application of new drugs in collaboration with external organizations that have developed a potential candidate compound.


Target diseases

Drug candidates for rare diseases in general are eligible. Target diseases include, but are not limited to, the following:

  • Neurological diseases
  • Cardiovascular diseases
  • Muscle diseases
  • Autoimmune diseases

However, drugs for cancer, blood diseases, infectious diseases, and ophthalmic diseases are excluded.


Key Success Criteria


The client seeks new drug candidates that meet the following requirements:

  • Modality: Low molecular compounds, peptides, proteins, nucleic acids
  • Route of administration: Oral administration is preferable
  • Has a new mechanism, which differs from that of existing drugs
    • Has higher competitive advantage than existing drugs
    • Diseases for which existing drugs do not exist
    • Drug candidates with the same mechanism of action as that of existing drugs that can be clearly differentiated from existing drugs are also eligible.
  • The action on the target molecule has been verified in an in vitro or in vivo study.
    • Preferably, an in vitro study has been conducted to predict clinical pharmacological action.
    • It is preferable that an in vivo study has been conducted.
  • Preferably, the selectivity to the target molecule has been evaluated.
  • Preferably, the safety and toxicity have been evaluated.
  • Development level:
    • Drug candidates in a preclinical to phase II study (especially, compounds in an optimization completion stage and drug candidates in a preclinical study to initial stage of clinical trial [phase I] are preferable.)

Approaches not of interest


The following drug candidates are out of the scope:

  • Known substances with poor exclusivity
  • Antibodies
  • Biologics
  • Substances and approaches that are not based on pharmacotherapy
    • Enzyme replacement therapy
    • Gene therapy
  • Drug candidates whose phase II study has been completed
  • Drug candidates for the treatment of the following diseases:
    • Cancer
    • Blood diseases
    • Infectious diseases
    • Ophthalmic diseases


Respondents should submit proposals using the attached Response Template.

The client will review submitted proposals and possibly ask clarifying questions before selecting the most suitable candidates for collaboration. The client will select the best candidate(s) through evaluations. During the selection process, the client may execute non-disclosure agreements (NDA) with selected respondent(s), seek further information disclosure, and discuss specific development targets or potential opportunities.

The client will execute necessary agreement(s) with the selected respondent(s) and move to the advanced development phase. Specifics of any collaboration will be determined through consultation with the concerned parties.


Preferred Collaboration types
  • Joint Development
  • Technology Licensing
Items to be submitted

Responses will use the Proposal Template which is linked to the “attachments” shown at the bottom of the link <REQ4363118> and include the following items:


  • Target diseases
  • Overview of the new drug candidates (modality, route of administration, mechanism of action, etc.)
  • Current performance
    • Data obtained from an in vitro or in vivo study
    • Data showing the selectivity to the target molecule
    • Data obtained from safety and toxicity studies
  • Current R&D stage
  • Future development plan
  • Request regarding the form of collaboration
  • Current status of intellectual property related to the proposed technology
  • Research achievements

Area of Interest
Request Priority