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A New Age of Pharmaceutical Innovation

IPI Speaks with Dr Patrick Larcier, Senior Director, Strategy Product Development Solutions, EU & US, of PharmaLex, on Next-generation Small Molecules

Q: Can we start with a bit of an overview of what Small Molecule Excellence from PharmaLex involves, including the types of experts involved and the overarching objective?

A: Despite the high-profile advances in the field of biotechnology, small molecules continue to be the mainstay in disease treatment. Nevertheless, these new small molecule products require expertise in order to move seamlessly through the product development process. That’s where the concept of excellence comes from – it’s about having that depth of knowledge in the field and across the development life cycle. With this in mind, we involve experts focused on operational excellence in every different part of the process, meaning CMC experts, non-clinical experts, as well as clinical experts, in order to build the appropriate steps, paths and studies needed in each module of the regulatory dossier, while considering time constraints and using the most appropriate regulatory pathways at all development stages. Through this expertise and extensive experience in developing small molecules, we’re able to help companies save time and effort in the development of these products.

Q: Let’s break down some of the areas a bit more, specific to the type of support offered to small molecule innovators (for example, non-clinical/quality, strategy, marketing authorization, scope, regulatory support)

A: It’s really on a case-by-case basis, based on the experience and expertise we have. This allows us to adapt the classic development steps or recommendations to each program and provide very specific support for the development of these molecules at the CMC, non-clinical, and even clinical stages. We combine this expertise with recommendations on interactions with the health authorities. The idea is to propose smart development plans to enable successful outcomes for our customers.

Q: What is unique, or different, about the new generation of small molecules?

A: First of all, these products are typically more diverse and complex than traditional chemical entities and they target pathologies that previous generations of small molecules did not. By way of example, now we find small molecules under development in neurological conditions, such as Alzheimer’s disease and multiple sclerosis, as well as in the field of oncology.1 That’s different to what we have seen previously, with companies largely focusing on the development of what we would call “classical biologics” (or large molecules) for these diseases – such as monoclonal antibodies, fusion proteins, and so on. So now some companies are, in some ways, reversing, by turning to small molecules for these pathologies. We’re seeing that at companies like Bayer, which is targeting at the RNA level through small molecule innovation,2 and Pfizer, which has had success with breakthrough small molecule treatments for arthritis and ulcerative colitis.3 We’re also observing this trend with smaller companies, such as Agios Pharma with Pyrukynd for adults with pyruvate kinase (PK) deficiency which results in chronic anemia,4 as well as with Accent Therapeutics, Epics Therapeutics, Skyhawk Therapeutics among others.