Background and challenges
Our client wanted to better understand the value of a new platform geared toward creating next-generation proteolysis-targeting chimeras (PROTACs) across different indications and therapeutic areas. Given the breadth of options associated with modality and the growing competition in the space, it was difficult to prioritize clinical investment opportunities without a robust characterization and analysis of the scientific opportunity.
The company engaged L.E.K. Consulting to develop a market overview across multiple key therapeutic areas (e.g., oncology, immunology, neuroscience) and define which indications and targets can be paired with a PROTAC strategy. Additionally, our team helped the client understand what R&D pipeline competition may exist and define where the white-space opportunities were for this modality when considering the longer-term strategy for the client.
Approach
To address the client’s questions, we took a rigorous analytical approach to define the scope of the market and identify where the client could invest to create innovative therapeutic outcomes.
- Defining PROTAC clinical strategy. We identified a range of previously “undruggable” targets that could be approached using PROTACs. In the process, we highlighted where PROTACs can outcompete previously established modalities (e.g., small molecules, monoclonal antibodies).
- Evaluating the client’s value proposition. Our analysis of the PROTAC opportunity landscape was leveraged to create a comprehensive assessment of the client’s platform strategy and value proposition, including highlighting areas where they are differentiated compared to other PROTAC platforms.
- Characterizing the competitive landscape. In parallel, we characterized which companies have publicly entered the space and specifically identified where the client may sit on a development timeline relative to these other players, further evaluating how our client can develop clinical value.
- Creating a strategic framework for opportunity assessment. We provided the client with a list of over 100 attractive near-term targets, segmented by tissue specificity and expression profiles, for R&D development. These opportunities were positioned against the different trade-offs that need to be considered when selecting a target and investing in a new drug program.
Results
The results of this analysis led to actionable near-term implications and longer-term strategies for our client, such as:
- Next step actions to de-risk future R&D investments. We helped frame key preclinical/discovery activities that the client can immediately pursue with limited investment. Based on the results of these actions, the client could then reevaluate the clinical success rate and decide on moving forward with a larger R&D investment.
- Target opportunity selection strategy. We provided immediate examples of near-term target opportunities that could be pursued based on the level of clinical validation and broader market interest, helping support a faster ramp to the clinic.
- Portfolio expansion plans. We mapped out areas of future synergies between putative drug targets and identified a potential program investment strategy that can leverage certain initial target investments to increase the potential value of other future new target investments.
- Defined milestones to support future investment decisions. We presented the clinical milestones the client will need to consider beyond the next two to three years and mapped out how each milestone would drive needs for new R&D investments and greater organizational capabilities.
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