Voyager CEO Al Sandrock outlines a focused strategy: remain specialists in neurology, but broaden the therapeutic modalities (gene therapy, proteins, oligonucleotides). This allows them to pursue well-validated CNS targets that are considered "undruggable" by traditional small molecules, which have historically been the only option for crossing the blood-brain barrier.

The pharmaceutical industry's focus on rare diseases has intensified, with 57% of all novel drugs approved in 2025 designated as orphan treatments. This is a continued increase from prior years, indicating a strategic shift towards smaller patient populations with high unmet needs, as exemplified by three different drugs for Hereditary Angioedema (HAE) being approved within ten weeks.

Yale spin-out Bexorg uses donated, post-mortem human brains kept molecularly active to test CNS drugs. This novel platform's key advantage is its ability to directly measure pharmacokinetics and blood-brain barrier penetration in a complex human organ, addressing a primary reason for clinical trial failure that animal and cell models cannot adequately predict.

To build investor confidence in the high-risk neuroscience field, Neurocrine employs a dual strategy. It highlights its own proven track record while simultaneously de-risking its pipeline by targeting biological pathways already validated by competitors, aiming to create superior, best-in-class medicines rather than pursuing unproven science.

Instead of diversifying across diseases, Kenai is building deep expertise in Parkinson's. Its pipeline addresses different patient needs: replacing lost cells (lead program), repairing existing damaged cells (002), and targeting inherited forms (003), creating a comprehensive disease franchise.

The DAYBREAK pivotal study focuses on third-line plus patients who have already failed both BTK and BCL2 inhibitors. By enrolling this high unmet need population, particularly those resistant to the newest non-covalent inhibitors, Neurix aims for an accelerated regulatory approval to get its drug to market faster.

Neurocrine's strategy with its M4 agonist hinges on achieving superior safety and tolerability through high selectivity. The company believes that for chronic psychiatric disorders, long-term patient adherence—driven by fewer side effects—is a more critical factor for commercial success than marginal gains in efficacy.

With patent cliffs looming and mature assets acquired, large pharmaceutical companies are increasingly paying billion-dollar prices for early-stage and even preclinical companies. This marks a significant strategic shift in M&A towards accepting higher risk for earlier innovation.

The success of Praxis's small molecule for a genetic epilepsy presents a strategic alternative to cell and gene therapies. In an era where complex modalities face funding, safety, and commercial hurdles, advanced small molecules offer a viable and potentially more practical path for treating genetic disorders.