On 10 February, Arkuda Therapeutics announced the completion of a USD64 million financing round that will support the further development of a new medicine for frontotemporal dementia. Arkuda Therapeutics is a biotechnology company which is developing treatments that target the lysosome, a small organelle that is responsible for breaking down and disposing of damaging proteins and material inside cells. In certain types of frontotemporal dementia (FTD), lysosomes stop functioning properly, which contributes to the brain pathology that drives FTD. Arkuda Therapeutics has already attracted the support of Pfizer, Eli Lilly and other companies. In its recent financing round, Arkuda was able to raise a further USD64 million, which will help it advance its first-in class therapy to clinical studies.
This therapy targets a protein called progranulin, which is known to play an important role in lysosomal function and inflammation in the brain. Arkuda plans to launch an early stage clinical trial in late 2023, which will recruit participants with a type of FTD that is caused by mutations in progranulin (GRN-FTD). GRN-FTD is an inherited form of dementia that is caused by a faulty copy of the progranulin gene, which leads to low progranulin levels. In their press release, Arkuda explained that this is a first step in their broader strategy of exploring the clinical potential of its compounds, as there are multiple additional neurodegenerative diseases in which genetic links to progranulin biology have been established, including Alzheimer’s Disease and Parkinson’s Disease.