Rare neurology is entering a pivotal moment. Scientific understanding is accelerating, new technologies are maturing, and the patient need remains profoundly unmet. As the biopharmaceutical community convenes for the J.P. Morgan 2026 Annual Healthcare Conference, Servier is sharpening its long-term focus on rare neurological diseases. The team believes that meaningful progress will come from both in-house and external efforts, and that some of the conversations had this year in San Francisco will be another step in tackling some of the most complex neurological diseases.
Rare neurological disorders affect more than 130 million people worldwide today,1 a number expected to rise by nearly 50 percent by 2040.2 Despite their prevalence and severity, almost 90 percent of these conditions lack a therapy capable of altering the course of disease.3 Patients often move through years of uncertainty, with few or no options to slow the progression of debilitating symptoms. This scale of unmet need is exactly why Servier believes now is the moment to act with urgency and long-term commitment.
Servier’s unique operating structure differs from that of most global pharmaceutical companies. Governed by a non-profit foundation and operating outside the constraints of public shareholder markets, Servier’s sense of urgency is not driven by the market pressures or financial timelines, but by patient need. This independence enables Servier to reinvest 20 percent of its profits into R&D each year and stay focused on long-term progress for patients, without external financial pressures.
“In the U.S., we’ve stepped into neurology with purpose,” says David Lee, CEO of Servier Pharmaceuticals. “Through our dedicated work in oncology, we’ve proven we can build a franchise by combining great science with the right partners. We’re now taking those learnings into rare neurological diseases.”
This isn’t just a U.S. effort. Globally, Servier is investing in the neurology space and has hired an expert in the field to take the lead as the team strategically enters this new frontier. Nitza Thomasson, Global Head of R&D Neurology, brings a track record of building neurology companies and expanding R&D footprints across global organizations, returning to Servier this year to design and scale the newly launched franchise. She is directing an integrated effort across biology, data science, clinical development, and platform innovation to build an ecosystem that enables innovation to thrive. Integrating technology, patients, caregivers, and advocacy groups early in the process ensures the therapies Servier develops meet patients’ real-world needs.
“We’re structuring the neurology organization for where we need to be in 2030 and beyond,” Thomasson says. “This is not a short-term expansion. It is a deliberate, multi-year effort to build capabilities, align expertise, and advance therapies in areas that have been underserved for decades.”
Building on Servier’s “One Innovation Engine” approach, already established in oncology, Nitza is leveraging both the company’s global research capabilities and external partners to create an expansive pipeline. In oncology, this framework has enabled Servier to build deep expertise around IDH mutations and translate that capability across multiple tumor types. Although the scientific mechanisms in neurology differ, the same strategic principles apply: anchoring R&D around focused biological pathways enables deeper scientific insight, more efficient platform development, and repeatable progress across diseases.
In neurology, Servier is focused on life-threatening and highly debilitating rare conditions. Six areas of major interest were defined, with diseases including rare epilepsies, neuromuscular disorders, movement disorders, leukodystrophies, genetically driven autism spectrum disorder, and peripheral neuropathies. These diseases can be approached through pathways such as genetic dysfunction, ion channel disruption, repeat expansion, and neuroinflammatory signaling.
Each of these scientific areas is internally supported by a growing suite of platform technologies (small molecules, monoclonal antibodies, and antisense oligonucleotides) designed to enable precision engagement of key neurological pathways. Notably, Servier’s antisense oligonucleotide program has evolved from an exploratory initiative into a structured clinical platform.
Servier’s neurology pipeline currently includes 15 programs spanning early discovery through clinical development. The company is also expanding its proven partnership model from oncology into neuroscience, drawing on recent collaborations that demonstrate Servier’s ability to structure high-value partnerships and integrate external innovation.
Servier recently strengthened its neurology pipeline with the acquisition of KER-0193, a clinical-stage program from U.K.-based Kaerus Bioscience. The deal, valued at up to $450 million, represents Servier’s first asset acquisition in neurology and marks an important milestone in advancing its 2030 strategy. KER-0193 is being developed as a potential therapy for Fragile X syndrome, a rare condition and the most common genetic cause of autism spectrum disorder, with no approved treatment options today. Following the transaction, Servier plans to initiate a Phase 2 clinical trial in Europe and the U.S., reflecting the company’s intent to pair its internal platform capabilities with selective external innovation to bring treatment to this underserved patient population.
For Servier, rare neurology is more than a strategic growth area. It reflects a mindset centered on continuity, scientific rigor, and the belief that meaningful progress in underserved patient populations requires sustained, long-term commitment.
As the biopharmaceutical industry looks beyond today’s therapies and toward its next wave of innovation at the J.P. Morgan Healthcare Conference, Servier’s priorities are clear: build intentionally, partner meaningfully, and advance therapies for patients who have waited far too long. Rare neurology is not a short-term opportunity for Servier—it is a long-term commitment grounded in science, sustained investment, and the belief that progress in underserved diseases is both possible and necessary.
To learn more about partnership opportunities with Servier in the U.S., please visit https://servier.us/partnering/.
