As the complexity of brain disorders continues to challenge traditional drug development, Angelini Pharma is taking a bold, forward-looking approach. Rafal Kaminski, Chief Scientific Officer, outlines a clear shift in strategy—from treating symptoms to delivering mechanism-driven, patient-relevant innovation aimed at modifying the disease course and preserving brain function over the long-term.
At the heart of this transformation is a recognition of the unique challenges in CNS drug discovery. Brain disorders are highly complex, with significant disease heterogeneity and a lack of reliable translational models. Many preclinical systems still rely on animal models with limited relevance to human biology, while the scarcity of robust biomarkers further complicates development. These barriers demand a fundamentally different approach—one that aligns scientific discovery with clinical reality and societal needs.
Angelini Pharma is addressing this by anchoring its R&D efforts in human-relevant biology. The company is increasingly leveraging human neuronal models to better understand disease mechanisms and improve translational success. Strategic collaborations play a critical role here, including partnerships with Quiver Bioscience to explore neurological disorders using optogenetics, and with collaborators such as JCR Pharmaceuticals to develop biologics that can effectively penetrate the blood-brain barrier.
Emerging technologies are also reshaping the pipeline. Advances in AI and data science are enabling a shift from empirical experimentation to more hypothesis-driven development, powered by large, multimodal datasets. At the same time, genetics and functional biology are supporting precision medicine approaches, helping match the right therapies to the right patients, while reducing development risk through translational biomarkers such as EEG and imaging.
Collaboration is not just a tool—it is a core strategy. Angelini Pharma has entered in partnerships of collective value over $2 billion in just past few years, reflecting a deep investment in R&D and external innovation. Initiatives like Headway, launched in 2017 in collaboration with TEHA Group, bring together clinicians, researchers, policymakers, and patient groups to ensure that innovation is aligned with real-world impact, access, and long-term sustainability.
Looking ahead, Kaminski is optimistic. The convergence of biology, technology, and data is creating new opportunities to move beyond disease management and toward brain health preservation. Angelini Pharma aims not only to participate in this transformation, but to lead it—positioning brain health as a central pillar of human well-being across the lifespan.
