Leading biotech industry veterans have outlined key strategies for success in drug development, emphasizing the critical balance between innovation, efficiency, and market differentiation in today's challenging pharmaceutical landscape.
Streamlining the Path to Clinical Trials
The journey from drug candidate identification to human testing remains one of the most challenging aspects of drug development. Peter Flynn, CEO of Arialys Therapeutics, shared how his company accelerated this process by acquiring a pre-validated drug candidate from Astellas Pharma.
"What that means is that first $20 to $30 million worth of work had taken place, and there'd been a level of validation which allows you to accelerate quicker and with more confidence," Flynn explained. This approach stands in stark contrast to starting from early academic observations, which he noted would be "really challenging" in today's environment.
However, Andrew Miller, chairman of Progentos Therapeutics and founder of Karuna Therapeutics, cautioned that this strategy isn't universally applicable. While established fields like immuno-oncology may offer fewer hidden opportunities, Miller suggested that "emerging areas" present more unexplored possibilities for companies with the right investigative approach.
Creating Competitive Advantages
In today's crowded biotech landscape, differentiation has become paramount. Chris Bardon, co-managing partner of MPM BioImpact, highlighted how genetic insights are opening new opportunities, particularly in neurology and psychiatry. "We're seeing many examples of that right now, for example, in Alzheimer's disease or in ALS, where you're looking at genetic subcomponents," she noted.
Flynn emphasized that competitive advantages can emerge from various sources, including "approach, mechanism, speed of delivery of clinical data, or actual clinical execution." Even operational elements like manufacturing readiness can provide crucial differentiation in the market.
Balancing Platform Technology with Product Development
The panel addressed the complex relationship between platform technologies and individual drug development. While platforms offer the allure of generating multiple drug candidates, they can also become resource drains if not managed properly.
"How much time and money do you spend investing in the platform, and at what point do you kind of lock on that iteration and move it forward?" Bardon questioned, warning against indefinite iteration without progress toward actual drug candidates.
The discussion highlighted successful examples in neurology, particularly with antisense technology. "Some of the most exciting stuff that's happening in neurology is related to antisense," Bardon noted, describing how refined modifications have enabled better blood-brain barrier penetration and CNS distribution.
Financial discipline remains crucial throughout these strategies. Bardon emphasized that excessive spending, particularly in private companies, can make it "really difficult for them and their investors to actually make money." This underscores the need for careful resource allocation across all development approaches.
