Arkuda Therapeutics Raises $64 Million Series B Financing to Advance Pipeline of Programs Targeting Lysosomal Biology to Treat Neurodegenerative Diseases

Funding to support progression of lead progranulin enhancer program into IND-enabling studies

Small molecule platform targeting intersection of lysosomal and microglial biology has applications across multiple rare and common neurologic conditions

Financing co-led by Cormorant Asset Management and Pivotal bioVenture Partners; Raymond J. Kelleher, M.D., Ph.D., accomplished neurologist and managing director at Cormorant Asset Management, and Heather Preston, managing partner at Pivotal bioVentures to join Arkuda Board of Directors

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Arkuda Therapeutics

Feb 10, 2022, 07:00 ET

WATERTOWN, Mass., Feb. 10, 2022 /PRNewswire/ -- Arkuda Therapeutics, a biotechnology company applying novel insights at the intersection of lysosomal and microglial biology to develop medicines to change the trajectory of neurodegenerative disease, today announced the completion of a $64 million Series B financing. The financing was co-led by Cormorant Asset Management and Pivotal bioVenture Partners, with participation from Surveyor Capital (a Citadel company), and Eli Lilly and Company, as well as existing investors Atlas Venture, Pfizer Ventures, funds managed by Tekla Capital Management LLC, and Mission BioCapital.

"This Series B funding marks a critical milestone as we advance our first-in-class lead progranulin enhancer program to IND-enabling studies and expand our pipeline of programs targeting lysosomal dysfunction, which is a key driver of many neurodegenerative diseases," said Gerhard Koenig, Ph.D., Co-Founder, President and CEO of Arkuda Therapeutics. "The patients we aspire to serve are in great need of therapeutic options, and this financing will enable us to accelerate our efforts to turn the promise of our emerging understanding of lysosomal biology into effective medicines with the potential to positively impact the trajectory of their disease."

Arkuda is pioneering the development of small molecule therapeutics directed at a proprietary molecular target that modulates progranulin, a protein known to play an important role in lysosomal function and innate immunity in the brain. The initial clinical indication for Arkuda's lead program is GRN-related frontotemporal dementia (FTD-GRN), a devastating inherited neurodegenerative disease occurring in individuals with an autosomal dominant mutation in the GRN gene, which codes for progranulin. This mutation results in progranulin deficiency and subsequent lysosomal dysfunction in the brain. Arkuda's molecules are designed to correct this progranulin deficiency, improve lysosomal function and potentially provide therapeutic benefits to patients with the disease. Arkuda has developed multiple potent, brain-penetrant compounds which are currently undergoing advanced lead optimization.

FTD-GRN is a first step in Arkuda's broader strategy of exploring the clinical potential of its compounds, as there are multiple additional neurodegenerative diseases in which genetic links to progranulin biology have been established, including Alzheimer's Disease and Parkinson's Disease.

"We are excited to support Arkuda in their pathbreaking work to address progranulin deficiency and lysosomal dysfunction, which is increasingly recognized as key driver of neurodegeneration," said Raymond J. Kelleher, M.D., Ph.D., Managing Director at Cormorant Asset Management and newly appointed member of the Arkuda Board of Directors.

Also joining the Arkuda Board is Heather Preston, M.D., Managing Partner at Pivotal bioVenture Partners, who added, "The company's novel platform, innovative science, and seasoned leadership team with deep experience in CNS drug development give us confidence that Arkuda has the potential to impact devastating neurologic diseases that currently lack any effective treatments."

"We look forward to partnering with the teams from Cormorant and Pivotal, along with all our new and existing investors as we build Arkuda into a leading precision neuroscience company focused on progranulin and lysosomal biology," said Bruce Booth, DPhil, Chairman of the Board of Arkuda. "We are gratified to have a high quality group of investors supporting these efforts, and we are happy to welcome Ray and Heather to our Board."

About Arkuda Therapeutics
Arkuda Therapeutics is a biotechnology company applying insights into lysosomal and microglial 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 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, Pivotal bioVenture Partners, Pfizer Ventures, funds managed by Tekla Capital Management LLC, Mission BioCapital, Surveyor Capital (a Citadel company), and Eli Lilly and Company. To learn more visit www.arkudatx.com.

SOURCE Arkuda Therapeutics