Breakthrough drugs aren't always driven by novel biological targets. Major successes like Humira or GLP-1s often succeeded through a superior modality (a humanized antibody) or a contrarian bet on a market (obesity). This shows that business and technical execution can be more critical than being the first to discover a biological mechanism.

In the competitive oncology market, Step Pharma differentiates itself by highlighting its novel, "first-in-class" mechanism and excellent safety profile. This strategy attracts interest by focusing on a unique therapeutic opportunity and potential for combination therapies, rather than competing directly on incremental efficacy gains.

Ipsen's drug Icorvo, which failed in the large NASH market, found unexpected success in the rare liver disease PBC. The smaller market is growing faster than anticipated due to physicians treating patients earlier and a key competitor being withdrawn. This demonstrates how a failed asset can be repurposed for a highly successful niche application.

After years of focusing on de-risked late-stage products, the M&A market is showing a renewed appetite for risk. Recent large deals for early-stage and platform companies signal a return to an era where buyers gamble on foundational science.

The GSK3 inhibitor was developed for CNS diseases, requiring high specificity and the ability to cross the blood-brain barrier. These same pharmaceutical characteristics—potency and lipophilicity—proved highly advantageous for treating cancer, demonstrating an unexpected but effective therapeutic pivot from neuroscience to oncology.

Synthakyne operates as a specialized 'cytokine engineering shop.' It develops its own assets in high-value areas like oncology (IL-2, IL-12) while simultaneously licensing its platform for other indications, such as inflammation, through major partnerships with Merck and Sanofi. This strategy generates capital and validates the core technology.

A significant number of Eli Lilly's compelling inventions came from unsanctioned projects. The company intentionally provides budget flexibility and avoids micromanagement at its R&D sites, allowing scientists to pursue their curiosity.

The company's plan to commercialize its drug alone is based on the manageable scale of CML clinical trials. Unlike mass-market diseases like obesity, pivotal trials require only 250-400 patients, making the financial and operational burden feasible for a smaller company to handle without a larger partner.

The company's lead molecule was initially invented to treat CNS diseases like Alzheimer's. A pivot occurred when a postdoc with an interest in oncology tested the compounds against refractory tumors, uncovering their true potential and leading to the company's formation around a new indication.