Arkuda Therapeutics

About Arkuda Therapeutics

Arkuda Therapeutics is a biotechnology firm focused on developing medicines for neurodegenerative diseases by applying new understandings of lysosomal biology. Founded in 2018 by Gerhard Koenig, PhD, and Duane Burnett, PhD, the company was seeded and incubated by Atlas Venture. Koenig, who serves as President and CEO, brings over 30 years of R&D leadership in neuroscience from roles at Bayer, FORUM Pharmaceuticals, and Quartet Medicine. Burnett, now Chief Technology Officer, has a background in medicinal chemistry at major pharmaceutical companies.

The company's core strategy is to address the root causes of neurodegeneration, specifically lysosomal dysfunction, which is considered a key driver in many of these conditions. Arkuda concentrates on developing small molecule therapeutics that modulate progranulin (PGRN), a protein vital for lysosomal function and innate immunity in the brain. By correcting progranulin deficiency, the company aims to restore lysosomal health and prevent the progression of neuron death.

Arkuda's lead program, ARKD-104, is an orally bioavailable, brain-penetrant small molecule designed to increase PGRN levels. The initial clinical focus is on GRN-related frontotemporal dementia (FTD-GRN), a genetically-defined neurodegenerative disease caused by a mutation in the progranulin gene (GRN). This condition typically affects individuals between 45 and 65. The company's broader strategy involves exploring the therapeutic potential of its compounds for other neurodegenerative diseases linked to progranulin dysfunction, such as Alzheimer's and Parkinson's disease.

The company's business model is centered on research and development, funded through significant venture capital investments. Arkuda has successfully raised substantial capital, including a $44 million Series A round in 2019 and a $64 million Series B round in 2022. Key investors include Atlas Venture, Pfizer Ventures, Eli Lilly and Company, and Cormorant Asset Management. In a significant milestone, Arkuda entered into an option and asset purchase agreement with Janssen Pharmaceuticals, a Johnson & Johnson company, in February 2024. Johnson & Johnson later exercised this option in January 2025, acquiring Arkuda's portfolio of lysosomal function enhancers for an upfront payment with potential for future development and commercial milestones.