Innovative Drug Seeds for Muscle-Related Diseases

NineSigma, representing a pharmaceutical company, seeks innovative drug seeds applicable to muscle-related diseases involving muscle atrophies such as muscular dystrophy, sarcopenia, cachexia, etc. The possession of new drug candidates is ideal, but the proposal of innovative new mechanisms, concepts, and ideas is also welcomed.

The Client positions muscle-related diseases as a focus area in drug discovery, and aims to establish an R&D platform by building a mid- to long-term partnership with organizations with innovative drug seeds in this area.

Of especial interests are drug seeds based on the concept of senescence inhibition/aging control, such as the removal of senescence cells or the NAD+/Sirtuin pathway activation, either of which affects to increase muscle weight/strength, or longevity- regulatory factors which potentially affect to increase in muscle weight/strength, but the client also welcomes proposals with mechanisms of action for increasing muscle weight/strength mediated via muscle satellite cells, myoblasts, muscle fibers, etc., for ameliorating fat deposition, or for remedying fibrosis, etc.

Anticipated drug seeds

Anticipated drug seeds can be substances as new drug candidates and new drug discovery targets. The possession of in-vivo and/or in-vitro assay system to evaluate drug efficiency for the concerned drug seeds is preferred.

• Requirements for new candidate substances
  • Modality: Low-molecular compounds, peptides, proteins, nucleic acids, antibodies, etc.
  • Must be a drug candidate before clinical trials: in stages from drug discovery to preclinical studies.
  • Drug efficacy must be verified by at least in-vitro tests reflecting the treatment concept. Ideally, in-vivo validity should be confirmed in animal models of target diseases.

• Requirements for new drug target molecules/pathways
  • Must have novelty as a target molecule and pathway aiming to increase muscle weight and strength.
  • Adequacy as a drug target must be validated by in vitro tests at least and by in vivo tests ideally.

Regarding drug seeds based on new concepts such as senescence inhibition/aging control, proposals for ideas and drug discovery strategies not meeting above requirements at this point are also welcomed.

Target diseases

• Muscular dystrophy
  • Especially Duchenne muscular dystrophy (DMD)
• Sarcopenia
• Cachexia: Especially skeletal muscle atrophy.
• Disuse muscle atrophy
• Mitochondrial myopathies
• Other muscle-related diseases: muscle weight/strength increase would lead to effective treatments for them.

Aging, refractory muscle-related diseases, etc. cause morphological changes and dysfunction in skeletal muscle. Musculoskeletal dysfunction leads directly to motor functional decline, which reduces activities of daily living (ADL) and quality of life (QOL). Recent global trends in population aging indicate the number of patients suffering from muscular diseases, such as sarcopenia, will increase, and the establishment of efficient cure to attenuate skeletal muscle dysfunction is an urgent task.

In the area of muscle-related diseases, new drug discovery targets have been being discovered with the progress of science in recent years such as myostatin and mitochondria, which is responsible for energy metabolism. On the other hand, high unmet medical needs are still existing in that area.

The Client aims to develop innovative drugs to cure muscle-related diseases to meet these unmet medical needs. They decided to seek potential partners this time to accelerate R&D through collaborations with organizations with cutting edge technologies in the area of muscular diseases.

After the submission due date, the client will review all submitted proposals. NineSigma will send the review results to each proposer 6-8 weeks after the due date. The client possibly asks clarifying questions before selecting the most suitable candidates for collaboration. The client will select best candidates through evaluations. During the selection process, the client may execute NDA with selected respondents, seek further information disclosure, and discuss specific development targets or potential opportunities.

The client will execute necessary agreements with the selected respondents and move to the advanced development phase. Specifics of any collaboration will be determined through consultation with the concerned parties.

Proposal shall have clear points and should not include confidential information. Supplemental files may be submitted in addition to the proposal.

The client will evaluate all responses with the following criteria.

• Overall scientific and technical merit
• Approach to proof of concept or performance
• Economic potential of concept
• Realism of the proposed plan (action items, timeline, roles, deliverables, cost estimation)
• Potential for proprietary position
• Respondents’ capability and related experiences