Escape Bio Raises $73 Million to Advance Precision Medicines for Genetic Neurodegeneration

Rare Daily Staff

Escape Bio has raised $73 million in a financing to advance development of its pipeline of experimental medicines targeting genetically defined neurodegenerative disorders including lysosomal storage disorders and Parkinson’s disease.

Wellington Management led the financing, which included new investors Avidity Partners, CAM Capital, New Leaf Ventures, Rock Springs Capital, Surveyor Capital, and Sphera Funds Management. Existing investors OrbiMed, Novo Holdings, Johnson & Johnson Innovation, Novartis Venture Fund, Osage University Partners, and Sutter Hill Ventures also participated.

“The proceeds allow us to accelerate two programs into patients who lack disease modifying treatments,” said Julie Anne Smith, president and CEO of Escape.

Escape’s lead program ESB1609 is a novel, orally administered, brain-penetrant and selective agonist of the S1P5 receptor in development for the treatment of lysosomal storage disorder Niemann-Pick type C and for Parkinson’s disease. It is currently being studied in a phase 1 randomized, double-blind, placebo-controlled, safety, tolerability, pharmacokinetic and biomarker study of escalating multiple doses in healthy volunteers.

S1P5 receptors are predominantly expressed in the central nervous system and natural killer (NK) cells. Activation of S1P5 plays a significant counteractive role in many aspects of neurodegenerative disease relevant biology inclusive of up-regulating several CNS lipid transporters. S1P5 agonism has been shown to normalize brain ceramide and sphingosine phosphate levels and promoted clearance of aggregation-prone proteins across multiple pre-clinical models of neurodegeneration.

Escape’s second program, ESB5070, is a novel, orally-administered, brain-penetrant and selective Leucine-Rich Repeat Kinase 2 (LRRK2) G2019S kinase inhibitor that spares wild type LRRK2 functionality. G2019S is the most common pathogenic mutation linked to Parkinson’s disease occurring in 1–3 percent of Parkinson’s patients. G2019S drives elevated kinase activity that perturbs the delicate orchestration of downstream signaling required for the healthy functioning of multiple cellular processes. ESB5070 is being developed specifically for the treatment of subjects carrying the LRRK2 G2019S variant and is currently in IND-enabling toxicology studies.

Photo: Julie Anne Smith, president and CEO of Escape