Arkuda Therapeutics Raises $44 Million to Treat Rare Form of Dementia

Rare Daily Staff

Arkuda Therapeutics has raised $44 million in a series A financing to develop new medicines targeting progranulin and lysosomal biology to treat neurodegenerative diseases.

The financing was led by Atlas Venture, which co-founded, seeded, and incubated the company. Pfizer Ventures also co-led the financing, with participation from funds managed by Tekla Capital Management and BioInnovation Capital.

Arkuda will initially focus on genetically-defined early onset form of dementia with potential to delay the onset of symptoms and disease progression. Its therapeutic approach centers on improving lysosomal function and neuronal health by increasing levels of progranulin, and its functional subunits, granulins, in the central nervous system.

Arkuda’s initial therapeutic focus is a devastating genetically-defined rare disease called GRN-related frontotemporal dementia (FTD-GRN), which strikes people between the ages of 45 and 65. Individuals with FTD-GRN have an autosomal dominant mutation in one of the alleles of the GRN gene, which codes for progranulin, a protein known to play an important role in lysosomal function and innate immunity in the brain. FTD-GRN is marked by progressive and rapid nerve cell loss in the frontal and temporal lobes of the brain, impacting personality, behavior, language, memory and movement, with a typical life expectancy of 6 to 7 years following the appearance of symptoms.

“We believe that the challenges in treating neurodegeneration stem from targeting disease pathologies rather than root causes such as lysosomal dysfunction, as well as from treating patients too late in their disease when significant damage has already occurred,” said Gerhard Koenig, co-founder, president and CEO, Arkuda Therapeutics. “FTD-GRN presents a unique and exciting opportunity to address a key driver of neurodegeneration before it progresses too far.”

It is now understood that the deficiency in progranulin and subsequently, granulins in GRN mutation carriers causes lysosomal dysfunction which leads to neurodegeneration. Arkuda is harnessing new and evolving insights into the role of progranulins and granulins in lysosomal health to drive the discovery of small molecules to restore and maintain neuronal health. Using existing biomarkers to reliably identify GRN mutation carriers and those at risk of symptomatic conversion, the company’s goal is to intervene as early as possible in the disease.

“For years, companies have focused their efforts on targeting neurodegenerative disease pathologies such as amyloid beta and tau, with little success,” said Bruce Booth, Arkuda board chairman and partner at Atlas Venture. “There is growing scientific evidence showing the critical importance of lysosomes in maintaining neuronal health in FTD as well as in other diseases such as Alzheimer’s and Parkinson’s Disease. Arkuda’s unique approach in targeting progranulin, a critical driver of lysosomal function, has the potential to have a truly meaningful impact for patients and their families.”

Arkuda Therapeutics was founded in 2018 by Atlas Venture, Gerhard Koenig, and Senior Vice President of Discovery Duane Burnett.

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

X