To overcome pharma's preference for new chemical entities (NCEs), Cereno created a second-generation drug by deuterating its original molecule. This modification improved the metabolite profile and, critically, made it a patentable NCE, opening doors for broader platform deals.

Related Insights

Instead of relying on finding novel targets, a key strategy in neuropsychiatry is to revisit failed compounds that showed efficacy signals. Companies use modern chemistry and delivery to engineer solutions that separate efficacy from the historical liabilities that halted development, turning past failures into new opportunities.

Many peptides are unlikely to ever receive FDA approval because their simple, easily replicated structures make them commodities. Pharma companies won't fund billion-dollar trials for drugs they can't patent, leaving them in a permanent gray market.

By reformulating existing oncology drugs, Nenology uses the streamlined 505(b)(2) regulatory pathway, de-risking and accelerating development. Simultaneously, their composition-of-matter patents provide strong intellectual property protection typically associated with entirely new chemical entities, creating a unique strategic advantage.

Biotech companies create more value by focusing on de-risking molecules for clinical success, not engineering them from scratch. Specialized platforms can create molecules faster and more reliably, allowing developers to focus their core competency on advancing de-risked assets through the pipeline.

The acquisition of Verona shows that a novel mechanism of action with a substantial clinical effect can make a company a prime M&A target. This holds true even with weaknesses like no composition of matter patent or an unfashionable drug delivery method, especially in disease areas lacking innovation.

A-muto suggests many drug programs fail due to toxicity from hitting the wrong epitope, not a flawed biological concept. By identifying and targeting a structural epitope unique to the diseased state of the same protein, these previously abandoned but promising therapies could be salvaged.

Instead of patenting a specific molecule, Alt-Pep underwent a decade-long process to patent the novel alpha-sheet protein structure itself. This unconventional IP strategy gives them a powerful, defensible platform applicable across numerous amyloid diseases, not just a single target composition.

For old molecules lacking composition-of-matter patents, a robust IP moat can be built by combining new use patents, patents on novel administration forms, and securing market exclusivity through Orphan Drug Designation for rare diseases.

Many innovative drug designs fail because they are difficult to manufacture. LabGenius's ML platform avoids this by simultaneously optimizing for both biological function (e.g., potency) and "developability." This allows them to explore unconventional molecular designs without hitting a production wall later.

R&D departments in large pharmaceutical companies often resist repurposing projects. Their leaders are rewarded for discovering new chemical entities, not for finding new applications for existing drugs, creating an internal funding barrier that business units must overcome.