It won’t have escaped most here that, following successful Phase 2 results in the second indication of Pitt Hopkins, NNZ-2591 is being promoted by Neuren as a validated multi-indication platform.
NNZ-2591 could alternatively be described as a “pipeline-in-a-drug”. “Pipeline-in-a-drug” is a drug development strategy in which one drug is developed for use in multiple indications.
This has been described as “the hardest strategy to pull off and the most impactful.” It is a strategy that comes with significant risk – there is a lot riding on the one drug candidate and if the drug fails in its first trial, it can spell disaster for further development. But if a company can pull it off, the strategy can maximize asset value and create a very efficient drug development business that is more about clinical execution than finding the next blockbuster. The result can be a drug which can achieve enormous patient impact and business scale.
The attractions of a pipeline-in-a-drug asset include:
- Lower development costs – savings on discovery, preclinical and Phase 1 development.
- Maximizing commercial potential – by pursuing multiple, discrete patient populations, the drug’s addressable market size can be maximized.
- Commercialization efficiencies – there are substantial sales and marketing savings and each new approval and its market uptake is often amplified by the previous approval(s).
The pipeline-in-a-drug strategy is said to reflect the evolution of the “blockbuster” ambition. Rather than relying on a single, broadly defined indication, pharma interest turned to demonstrating the efficacy of a pathway-targeted product in a range of conditions involving the implicated pathway.
It has been predicted that the “pipeline-in-a-drug” strategy will lose its attraction to pharma under the United States’ IRA price-setting legislation. It is reasoned that pharma will now be incentivized to seek approval in the broadest possible non-orphan indication to maximize sales during the pre-price negotiating window. In the case of orphan indications, pharma may be incentivized to pursue just one indication instead of multiple, to remain exempt from IRA price-setting.
However, NNZ-2591 comes with all the attractions of a pipeline-in-a-drug asset, as listed above, but none of the disadvantages that come with IRA price-setting. As discussed here recently, drugs in the paediatric orphan indications being developed by Neuren are covered by Medicaid and private health insurance, rather than Medicaire, which is the price-setter under the IRA. NNz-2591 should therefore remain outside the reach of the IRA legislation.
NNZ-2591 - what is there not for pharma to love about this "pipeline-in-a-drug"?
https://axial.substack.com/p/drugs-that-are-their-own-pipelineshttps://www.forbes.com/sites/davids...-its-a-new-blockbuster-model/?sh=dddbdbb55d13https://www.linkedin.com/pulse/inflation-reduction-act-changes-biopharma-pipeline-bd-singh-ph-d-
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