Pfizer, Lilly-Backed Neuro Company Bags $64 Million Series B To Correct Progranulin Deficiency in Dementia

Arkuda Therapeutics has raised $64 million in a Series B financing round, building on the $44 million Series A raised in 2019. The Cambridge, Massachusetts-based biotech said the proceeds will support the progression of their lead progranulin enhancer program into IND-enabling studies. 

Arkuda is targeting GRN-related frontotemporal dementia (FTD-GRN), an inherited neurodegenerative disease where individuals experience rapid and severe neuronal losses starting at the age of 50. 

Cormorant Asset Management and Pivotal bioVenture Partners co-led the round, which also saw participation from Surveyor Capital (a Citadel company) and Eli Lilly, as well as existing investors Atlas Venture, Pfizer Ventures, funds managed by Tekla Capital Management LLC, and Mission BioCapital.

Joining Arkuda’s board of directors are Raymond J. Kelleher, Managing Director at Cormorant Asset Management, and Heather Preston, Managing Partner at Pivotal bioVenture Partners.

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Fixing Progranulin Deficiency to Treat Dementia 

FTD-GRN is caused by a mutation in the gene that encodes for progranulin, a highly conserved protein that regulates cell growth, survival, repair and inflammation. 

Progranulin is processed into granulins in lysosomes, which are diverse organelles that break down material inside the cell. Granulins themselves are critical for maintaining normal lysosomal function.  

Mutations in one allele of the GRN gene leads to progranulin deficiency, which subsequently causes neuronal deterioration and death. Besides frontotemporal dementia, progranulin deficiency has been implicated in multiple neurodegenerative diseases, including Parkinson’s disease, Alzheimer’s disease and amyotrophic lateral sclerosis (ALS). 

Arkuda is developing small molecules that aim to correct progranulin deficiency and subsequently improve lysosomal function. The company has mentioned that it has several brain-penetrating compounds in the lead optimization phase. 

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Monoclonal Antibodies to Elevate Progranulin Levels 

Like Arkuda, several companies are targeting progranulin deficiency in treating neurodegenerative diseases. 

Last July, GSK and Alector inked a deal worth $2.2 billion to co-develop two monoclonal antibodies, AL001 and AL101, to elevate progranulin levels. AL001 is undergoing a Phase 3 in people with FTD-GRN, while AL101 is in a Phase 1a clinical trial covering patients with more common neurodegenerative diseases. 

Passage Bio, on the other hand, is taking the gene therapy route. It is assessing a candidate that delivers a functional copy of the GRN gene to the brain to restore progranulin levels.

Author

Joy Lin

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