Arkuda Raises $64 Million to Advance Programs Targeting Lysosomal Biology for Neurodegenerative Diseases

Arkuda Therapeutics completed a $64 million series B financing to advance a small molecule platform targeting the intersection of lysosomal and microglial biology that has applications across multiple rare and common neurodegenerative diseases.

Photo: Gerhard Koenig, co-founder, president and CEO of Arkuda Therapeutics

Cormorant Asset Management and Pivotal bioVenture Partners co-led the financing, with participation from Surveyor Capital (a Citadel company), and Eli Lilly, as well as existing investors Atlas Venture, Pfizer Ventures, funds managed by Tekla Capital Management, and Mission BioCapital.

“This series B funding marks a critical milestone as we advance our first-in-class lead progranulin enhancer program to IND-enabling studies and expand our pipeline of programs targeting lysosomal dysfunction, which is a key driver of many neurodegenerative diseases,” said Gerhard Koenig, co-founder, president and CEO of Arkuda Therapeutics. “The patients we aspire to serve are in great need of therapeutic options, and this financing will enable us to accelerate our efforts to turn the promise of our emerging understanding of lysosomal biology into effective medicines with the potential to positively impact the trajectory of their disease.”

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 initial clinical indication for Arkuda’s lead program is 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. Arkuda has developed multiple potent, brain-penetrant compounds that are currently undergoing advanced lead optimization.

FTD-GRN is the first step in the company’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.

In conjunction with the financing, Raymond J. Kelleher, managing director at Cormorant Asset Management and Heather Preston, managing partner at Pivotal bioVenture Partners are joining the Arkuda Board of Directors.

Author: Rare Daily Staff