Orfenix provides protocol and regulatory support to Guanarep to develop a treatment for a rare but debilitating neurological disease that affects children from a young age and for which there is currently no treatment. The disease leads to severe brain damage, and eventually death.

Guanarep will develop a promising potential treatment for Vanishing white matter (VWM) disease. VWM disease, or Leukoencephalopathy, is a rare disease with severe neurological consequences. It is an inherited condition caused by a faulty gene. VWM disease is a progressive often affecting patients from an early age. Children with VWM disease have a defective protein that prevents the body from making enough myelin. Myelin is a white, fatty substance that covers nerve fibers in the brain and protects them from damage. Damage may result from trauma, infection, or fever. Without myelin, nerves throughout the body deteriorate and disappear, leading to degeneration of motor function, followed by cognitive decline and personality changes.

There is no treatment available to patients suffering from VWM disease. However, preclinical and early clinical work at Amsterdam UMC has identified a promising candidate to meet this unmet clinical need. This candidate, guanabenz, is an existing drug that is used in the treatment of high blood pressure. It is undergoing initial preclinical development to determine if guanabenz can be repurposed for the treatment of VWM disease.

Guanarep is a university spinoff that will develop guanabenz for clinical use as a treatment for VWM disease. To that end Guanrep will work towards obtaining orphan designation and generate the relevant data package to initiate further clinical trials and identify routes to market the product upon succesful conclusion of the trials. Orfenix contributes by providing clinical and regulatory support, including protocol support for the development. In addition, Orfenix is leading the venture, making sure this investigator initiated project moves forward in accordance with the principles of socially responsible licensing.

References

https://ulf.org/leukodystrophies/vanishing-white-matter-disease/

Schoenmakers, D.H., Leferink, P.S., Vanderver, A. et al. Core protocol development for phase 2/3 clinical trials in the leukodystrophy vanishing white matter: a consensus statement by the VWM consortium and patient advocates. BMC Neurol 23, 305 (2023).

https://doi.org/10.1186/s12883-023-03354-9

Hamilton EMC, van der Lei HDW, Vermeulen G, Gerver JAM, Lourenço CM, Naidu S, Mierzewska H, Gemke RJBJ, de Vet HCW, Uitdehaag BMJ, Lissenberg-Witte BI; VWM Research Group; van der Knaap MS. Natural History of Vanishing White Matter. Ann Neurol. 2018 Aug;84(2):274-288. doi: 10.1002/ana.25287. Epub 2018 Sep 6. PMID: 30014503; PMCID: PMC6175238.