Category | Small Molecule
Small molecule drug development has changed substantially in recent years. With the heightened focus on molecularly targeted therapies, small molecule active pharmaceutical ingredients (APIs) and drug products formulations are more complex and potent than ever, requiring increasingly specialized manufacturing processes and drug delivery solutions. At the same time, the competitive demand for rapid entry into clinical development—combined with accelerated review pathways—translates into compressed clinical manufacturing timelines.
These dynamics challenge sponsors to balance the need for speed with optimal execution of chemistry, manufacturing, and control (CMC) activities to ensure the quality of the finished product during all phases of development. Achieving this balance is particularly challenging for new and emerging biopharma companies who are dominating the pipeline and opting to hold onto their molecules longer, possibly through commercialization.
To balance development, manufacturing, and financial pressures, small and emerging pharma companies are increasingly relying on outsourcing partners to support various aspects of their small molecule development programs. The more distributed the development team, however, the more risk there is in advancing molecules to initial clinical studies. Spreading drug substance, drug product, and clinical strategy activities across multiple vendors can lead to workflow and process inefficiencies across the development lifecycle and can introduce vulnerabilities associated with knowledge transfers.
In contrast, working with a single partner that integrates all of these activities “under one roof” results in a more efficient development plan, minimizing risk and accelerating progress to clinic. Realizing the full potential of this integrated approach, however, requires collaborating with a strategic partner that has a deep understanding of the process and science involved in each phase of development and the impact that phase-specific decisions have down the line.
Every API is different, and each development process will have its own unique challenges and opportunities. Yet, there are a few key strategies that apply broadly and can help developers navigate the early development and manufacturing process while mitigating risk and ensuring quality. These strategies are described in a recent whitepaper, Expediting early-phase development of small molecules: An integrated approach.
According to the report, efficiently advancing small molecules from API to pre-clinical data and clinical supply requires:
As small molecule therapies become more complex and competitive pressures increase, the importance of finding new solutions to tighten development processes becomes ever clearer. The adoption of an integrated drug substance, solid-state characterization, and phase-appropriate drug product development strategy offers a way forward, by streamlining and accelerating the journey from lab to clinic.