Arkuda Therapeutics, a company leveraging new insights into progranulin and lysosomal biology to develop medicines to change the trajectory of neurodegenerative disease, today announced the completion of a $44 million Series A financing. Led by a team of drug developers and scientists with deep experience in central nervous system disorders and neurodegeneration, Arkuda's lead 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. Atlas Venture co-founded, seeded and incubated the company, and co-led the Series A financing with Pfizer Ventures, with participation from funds managed by Tekla Capital Management LLC, and BioInnovation Capital.

Arkuda's initial therapeutic focus is a devastating genetically-defined 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, PhD, 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, DPhil, 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 Duane Burnett, PhD, Senior Vice President, Discovery.

About Arkuda Therapeutics
Arkuda Therapeutics is a biotechnology company leveraging new insights into progranulin and lysosomal biology to develop medicines to change the trajectory of neurodegenerative disease. Arkuda's lead compounds aim 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 GRN gene. Arkuda launched in November 2019 with $44 million from leading life science investors include Atlas Venture, Pfizer Ventures, funds managed by Tekla Capital Management LLC, and BioInnovation Capital. To learn more visit www.arkudatx.com.

About Atlas Venture
Atlas Venture is a leading biotech venture capital firm. With the goal of doing well by doing good, the company has been building breakthrough biotech startups since 1993. Atlas works side by side with exceptional scientists and entrepreneurs to translate high impact science into medicines for patients. Our seed-led venture creation strategy rigorously selects and focuses investment on the most compelling opportunities to build scalable businesses and realize value. For more information, please visit www.atlasventure.com.

About Pfizer Ventures
Pfizer Ventures (PV), the venture capital arm of Pfizer Inc. was founded in 2004 and invests for return in areas of current or future strategic interest to Pfizer. PV seeks to remain at the forefront of life science advances, looking to identify and invest in emerging companies that are developing transformative medicines and technologies that have the potential to enhance Pfizer's pipeline and shape the future of our industry. For more information, visit www.pfizerventures.com.