News

March 9, 2021

Arkuda Therapeutics to Present Update on its First-in-Class Small Molecules for the Potential Treatment of Parkinson’s Disease with GBA1 Mutations (PD-GBA) at the AD/PD 2021 Conference

Study provides preclinical evidence of the ability of Arkuda compounds to increase activity of a key lysosomal enzyme deficient in PD-GBA patients

Compounds previously shown to increase levels of both extracellular progranulin (PGRN) and intracellular granulins in preclinical models of GRN-related frontotemporal dementia (FTD-GRN)

Watertown, MA – March 9, 2021 – Arkuda Therapeutics, a biotechnology company applying new insights into lysosomal biology to develop medicines to change the trajectory of neurodegenerative disease, will present data on the potential of their compounds for the treatment of Parkinson’s disease patients with GBA1 mutations (PD-GBA) at the 15th​ International Conference on Alzheimer’s and Parkinson’s Diseases and related neurological disorders (AD/PD™ 2021), taking place virtually March 9-14, 2021 (Poster 501, Abstract #606).

The new data demonstrate that a novel Arkuda small molecule increases the enzymatic activity of beta-glucocerebrosidase (GCase), which plays a key role in normal lysosome function and is essential for the health of cells in the central nervous system.  Mutations in the GBA1 gene result in a deficiency in GCase activity and collectively are the most common known genetic risk factors for Parkinson’s disease, occurring in an estimated 10% of patients with the disease. Therapeutics that increase GCase activity offer potential for the treatment of Parkinson’s disease, for which no disease-modifying therapies currently exist.

“With the growing understanding of the significance of reduced GCase activity in the pathophysiology of Parkinson’s disease as well as in other neurodegenerative diseases, we are encouraged to have identified potent compounds that increase GCase activity in a concentration-dependent fashion in multiple relevant cellular models,” said Gerhard Koenig, PhD, Co-Founder, President and CEO of Arkuda Therapeutics. “Importantly, these same compounds were previously shown to increase progranulin (PGRN) as a potential treatment for frontotemporal dementia, so we are excited about the broad potential clinical utility for this class of compounds aimed at improving lysosomal health and function in the brain.” Arkuda is advancing related compounds towards clinical development, with the lead program moving to IND-enabling studies and targeting entry into human clinical studies in 2022.

 

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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 aims to correct progranulin deficiency and lysosomal dysfunction in GRN-related frontotemporal dementia (FTD-GRN), a genetically-defined subtype of frontotemporal dementia caused by an autosomal dominant mutation in the progranulin (GRN) gene. Beyond progranulin, Arkuda’s compounds enhance the activity of several other key lysosomal enzymes, including GCase, and the company is further exploring the therapeutic potential of its molecules in PD-GBA and other neurodegenerative diseases. Arkuda is backed by leading life science investors including Atlas Venture, Pfizer Ventures, funds managed by Tekla Capital Management LLC, and Mission BioCapital. To learn more visit www.arkudatx.com.

 

Media Contact:
Lissette Steele
Verge Scientific Communications
202.930.4762
lsteele@vergescientific.com