Ripe for clinical innovation
Over the last 20 years the development of new therapies aimed at tackling the most major chronic neurodegenerative diseases (NDD) has been filled with promise and setbacks. The complexity associated with targeting neurological diseases has led to some big pharma players retreating from brain drug research, yet the recent technological and clinical advancements exhibit potential to address the ever-growing demand. Having observed notable milestones to date in neurology and potential innovative mechanisms of action, we anticipate that we may only be a few positive studies away from the launch of the next generation of breakthrough NDD medicines.
Many of us are acutely aware of the challenges associated with drug development. However, these challenges are perhaps most pertinent in discovering new medicines to treat central nervous system (CNS) disorders. CNS clinical programmes have historically experienced substantially higher rates of failure relative to most other disease areas, with the success rates of CNS drugs roughly less than half of those for non-CNS drugs. This is likely due to the complexity of CNS disorders, historical lack of understanding of disease pathologies and inappropriate trial designs that lacked clinically meaningful endpoints.
With recent technological and clinical advancements, we are now observing the emergence of new genetic targets to treat CNS diseases, effective biomarkers to de-risk programmes earlier during discovery, delivery platforms that get drugs into the brain and more sophisticated trial designs that focus on meaningful benefits to patients. We feel neuroscience drug discovery could be at the beginning of a major evolution where the breakthroughs we are seeing today, and further anticipate in the 2020s, closely resemble what drove the successes seen in oncology in the 2010s with immunoncology and checkpoint inhibitors.
While we are not able to predict the timing of the next CNS breakthrough, it is clear that it will be a collective effort. If success is to be achieved, cross-industry collaboration between pharma and biotechs will be critical. Biotechs possess the deep, specialised understanding of the disease biology in the relevant indications. Conversely, instances where underlying science is overruled by strategy, purely driven by generating the next blockbuster in the largest indications, regardless of understanding, will more likely be met with failure. While there may be great rewards in diseases such as Alzheimer’s disease (AD), a strategy that involves throwing everything at a certain indication in the hope something will stick represents what we see as an out-of-date and costly approach.