Exploring advanced modelling, solid form selection, and scalable process design to reduce risks and enable smoother drug development for small biotechs.
Drug development is one of the most complex and high-stakes enterprises in science. The path from discovery to patient is long, costly, and fraught with challenges. Identifying a novel therapeutic molecule is an achievement in itself, but it is only the beginning of a rigorous journey. Molecules must withstand years of preclinical evaluation, formulation optimization, and manufacturing development before they ever reach a first-in-human study.
For biotechs, this journey can feel like an unforgiving obstacle course. Resources are limited, timelines are ambitious, and the pressure to deliver results to investors and patients is immense. In this environment, speed is often prioritized but without the right foundation, haste can jeopardize success.
This white paper explores how contract development and manufacturing organizations (CDMOs) such as Lonza help innovators navigate this journey, enabling smart decisions, avoiding pitfalls, and ultimately increasing the probability of success.
The illusion of speed
For many small biotech teams, the focus is on reaching clinical trials as quickly as possible. However, a poorly enabled drug form can undermine years of preclinical work, setting back timelines and consuming valuable capital.
True speed in development does not come from cutting corners but from setting realistic expectations, asking the right scientific questions, and choosing the most viable path forward.
Building the right foundations: formulation and process design
Selecting the optimal drug form is one of the most critical decisions in development. Whether formulated as a crystalline salt, co-crystal, or amorphous dispersion, each option carries implications for solubility, stability, manufacturability, and ultimately patient efficacy.
Companies that take the time to thoroughly understand their molecule’s properties can design formulations that meet the desired product profile from the outset. Investing additional effort at this stage reduces the risk of failure later and maximizes the chances of success.
Lonza applies advanced modelling tools such as physiologically based biopharmaceutics modelling (PBBM) to predict systemic exposure under physiological conditions. These models integrate in vitro data to provide insights into absorption and clearance, supporting informed decisions before costly in vivo and clinical studies.
A successful lead formulation is only the first milestone. Ensuring scalability from a few grams for animal studies to kilograms for late-stage trials is critical. Development teams must anticipate how processes will evolve at each stage, avoiding costly redesigns or delays further down the pipeline.
Quality as the cornerstone
As molecules advance into clinical trials, the rigor of manufacturing and analytical development must evolve in parallel. Robust analytical methods such as microscopy, chromatography, and spectroscopy are essential to support GMP release and ensure consistent quality.
Insufficient analytical methods are a common cause of bottlenecks in development. Establishing robust, phase-appropriate methods early ensures that clinical supply chains remain uninterrupted and reduces the risk of rework as molecules progress into later stages.
Respecting the molecule
Every molecule is unique, with its own technical challenges. As drug candidates have become more complex over the past two decades, the importance of a tailored, molecule-specific development strategy has only grown.
A standardized, one-size-fits-all approach is rarely sufficient. Success depends on combining broad technical expertise with an individualized development pathway that respects the specific properties of each molecule and our customer’s timelines and objectives.
Conclusion
Drug development success depends on balancing ambition with scientific rigor. For small biotech teams, the temptation to prioritize speed can be overwhelming, but the risks of missteps are high. With the right partner, companies can move with both speed and confidence, advancing molecules efficiently while building a solid foundation for long-term success.