Arkuda is pioneering the development of small molecule therapeutics directed at a proprietary molecular target that modulates progranulin, a protein known to play an important role in lysosomal function and innate immunity in the brain, the company said.

The initial clinical indication for Arkuda's lead program is granulin precursor (GRN)-related frontotemporal dementia (FTD-GRN), a devastating inherited neurodegenerative disease occurring in individuals with an autosomal dominant mutation in the GRN gene, which codes for progranulin. This mutation results in progranulin deficiency and subsequent lysosomal dysfunction in the brain.

Arkuda's molecules are designed to correct this progranulin deficiency, improve lysosomal function, and potentially provide therapeutic benefits to patients with the disease, the firm said. Arkuda has developed multiple potent brain-penetrating compounds that are currently undergoing advanced lead optimization.

FTD-GRN is the first step in Arkuda's 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.

The financing was co-led by Cormorant Asset Management and Pivotal Bioventure Partners, with participation from various other investors.