R&D Strategy Support for a Novel Technology Platform

A publicly traded biopharmaceutical company with a novel technology platform requested support for their R&D strategy. The organization recently licensed one of its lead development programs to a top ten pharmaceutical company and needed to replenish its pipeline. Because the platform was broadly applicable across indications, there was a need to conduct an objective indication prioritization exercise.

Kineticos was charged with narrowing a list of over 9,400 rare diseases to the five most attractive indications from a technical and commercial perspective, which was then scored and weighted based on various criteria established collectively between Kineticos and the sponsor.

Once rank-ordered, Kineticos conducted a sensitivity analysis to identify other diseases that could potentially disrupt the original top five. Upon receiving approval on the top five diseases, Kineticos conducted a detailed review of each disease.

This review included:

• Assessing the disease state
• Current standard of care
• Competitive landscape
• Unmet medical needs
• Timing to market
• Probability of success
• Commercial potential

The detailed assessments resulted in the sponsor selecting two indications for final consideration. The last step included conducting key opinion leader (KOL) interviews with leading researchers to validate secondary findings and fill any remaining information gaps regarding the two diseases and their technology’s viability.

• Detailed assessments on the top five diseases
• KOL interviews
• A database of over 9,400 diseases with an objective ranking of the top 35 diseases.

Kineticos equipped the sponsor with a database of over 9,400 diseases with an objective ranking of the top 35 diseases. Also provided a PowerPoint deck that provided a detailed methodology outlining the filtering/scoring process, an assessment of the top five diseases, key themes and insights gained through the KOL interviews, and recommendations on potential follow on indications.