Centogene N.V. together with Evotec SE have discovered a new small molecule which has the potential to treat patients with type 2 and type 3 Gaucher disease, otherwise known as neuronopathic Gaucher disease.
 
The discovery was the result of the collaborative work under the existing drug discovery partnership.
 
The Companies have now extended this collaboration for another year, granting Evotec an R&D License for the continued development activities. The amended agreement also provides Evotec with an exclusive option until March 31, 2025, to determine whether to enter a license agreement acquiring CENTOGENE’s share of the IP generated throughout the collaboration. In exchange for such a global exclusive license, CENTOGENE would receive an up-front fee, milestone payments, as well as additional royalties. Further financial details were not disclosed.
 
“This partnership is indicative of how we are able to successfully leverage our expansive know-how in rare diseases as well as the depth of the CENTOGENE Biodatabank, which in combination with Evotec’s multi-modality platform has successfully identified drug candidates in Gaucher disease,” said Dr. Peter Bauer, Chief Medical and Genomic Officer at CENTOGENE. “Importantly, together with Evotec, we have established a much deeper understanding of neuronopathic Gaucher disease than before, which we believe could accelerate the development of a new life-saving treatment for these patients worldwide.”
 
Within their collaboration, CENTOGENE and Evotec have been working together to research, discover, and develop a molecule to reduce the lysosomal molecule lyso-Gb1, which is massively increased in patients with neuronopathic Gaucher disease. The collaboration brought together Evotec’s induced pluripotent stem cell (iPSC) platform and broad drug discovery and development capabilities with the CENTOGENE Biodatabank, which contains data from over 850,000 patients, including patient-derived iPSC lines and proprietary translational biomarkers.
 
“We are excited that our highly complementary partnership with CENTOGENE has yielded a first potential treatment for Gaucher disease,” said Dr. Cord Dohrmann, Chief Scientific Officer of Evotec SE. “The identified molecule is the result of combining CENTOGENE’s real-world, data-based, global rare disease platform with Evotec’s broad drug discovery and development capabilities. We look forward to exploring further development options for the promising molecule to deliver a much-needed option for an underserved patient population.”
 
The collaboration has achieved significant success in preclinical studies. In vivo pharmacodynamics and pharmacokinetics studies in animal models have demonstrated high brain penetration and metabolic stability of the drug candidate. This new molecule is now positioned to enter the next stage of R&D, which Evotec is currently pursuing.