July 18, 2023

Arkuda Therapeutics to Present Update on ARKD-104, its First-in-Class Oral Development Candidate for the Treatment of Frontotemporal Dementia and other Neurodegenerative Disorders at the Alzheimer’s Association International Conference 2023

Study demonstrates ARKD-104 has excellent CNS drug-like properties, increases PGRN in the CNS of non-human primates and increases important cofactors of
lysosomal enzymes important to neuronal health

ARKD-104 expected to enter clinical trials for Frontotemporal Dementia in H1 2024      

Watertown, MA – July 18, 2023 – Arkuda Therapeutics, a biotechnology company applying novel insights at the intersection of lysosomal biology and neuronal health to develop medicines that change the trajectory of neurodegenerative disease, will present pre-clinical data on lead development candidate ARKD-104 for the treatment of GRN-related frontotemporal dementia (FTD-GRN) at the 2023 Alzheimer’s Association International Conference (Poster P1-752).

FTD-GRN is caused by heterozygous mutations in the GRN gene encoding progranulin (PGRN) that lead to a reduction in levels of progranulin. Data presented demonstrate that ARKD-104, a novel, brain-penetrant, orally bioavailable small molecule, increases PGRN in the central nervous system (CNS) of non-human primates in a dose-dependent manner, and that the increase in PGRN levels is sustained with daily repeat dosing which returned to baseline following cessation of treatment. Further, ARKD-104 was shown to increase important cofactors of lysosomal enzymes important to neuronal function.

“We’re excited about the profile of ARKD-104 as a novel oral treatment for FTD-GRN, but also for other diseases driven by lysosomal dysfunction, and look forward to advancing ARKD-104 to the clinic in the coming year,” said Gerhard Koenig, PhD, Co-Founder, President and CEO of Arkuda Therapeutics.

Arkuda is currently conducting IND-enabling studies for ARKD-104 and expects to initiate a first-in-human trial starting in the first half of 2024.


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About Arkuda Therapeutics
Arkuda Therapeutics is a biotechnology company applying insights into lysosomal biology to drive the development of medicines to change the trajectory of neurodegenerative disease. Arkuda’s lead program ARKD-104 aims to correct progranulin deficiency and lysosomal dysfunction in patients with GRN-related frontotemporal dementia (FTD-GRN), a genetically-defined subtype of frontotemporal dementia caused by an autosomal dominant mutation in the progranulin (GRN) gene. The company is further exploring the therapeutic potential of its molecules in other neurodegenerative diseases where genetic links to dysfunction in progranulin biology have been established, including Alzheimer’s Disease and Parkinson’s Disease. Arkuda is backed by leading investors including Atlas Venture, Cormorant Asset Management, Pfizer Ventures, funds managed by Tekla Capital Management LLC, Mission BioCapital, and Eli Lilly and Company. To learn more visit

 Investor Contact:
Andy Hu
Chief Business Officer

 Media Contact:
Lissette Steele
Verge Scientific Communications


Lissette Steele
Verge Scientific Communications