ESCAPE Bio Snags $73 Million to Advance Neurodegenerative Clinical Development Programs


Clinical-stage biopharmaceutical firm ESCAPE Bio has snagged a $73 million crossover financing deal, led by Wellington Management Company, which will help advance the company’s two clinical development programs of precision neurology medicines for patients with genetic neurodegeneration.

ESCAPE’s early focus was on an Apolipoprotein E-targeting program, which was geared toward tackling Alzheimer’s disease. This initiative secured the company a $63 million Series A in 2017. No mention of advancing this program was made in the announcement of the crossover financial closing.

Bluechip investors who are newly contributing to the latest crossover financing round include Avidity Partners, CAM Capital, New Leaf Ventures, Rock Springs Capital, Surveyor Capital (a Citadel company) and Sphera Funds Management. They join OrbiMed, Novo Holdings, Johnson & Johnson Innovation, Novartis Venture Fund, Osage University Partners and Sutter Hill Ventures, all of whom own a stake in ESCAPE.

“We are delighted to partner with these new outstanding investors as we advance our pipeline of precision neurology medicines,” said ESCAPE’s president and CEO Julie Anne Smith in a statement. “The proceeds allow us to accelerate two programs into patients who lack disease modifying treatments.”

ESCAPE is currently in Phase I/II investigations of ESB1609, an orally administered, brain-penetrant and selective sphingosine-1-phosphate 5 (S1P5) receptor agonist with potential utility in the treatment of neurodegenerative disorders associated with lysosomes. This unique treatment approach for lysosome disorders targets S1P5, a receptor expressed in both the central nervous system and on natural killer cells.

According to ESCAPE, activation of SIP5 can increase lipid transporter activity, essentially helping promote lipid levels in the brain while reducing neurodegeneration markers. The current study is focused on a rare neurodegenerative lipid storage disorder, Niemann-Pick Type C.

Findings from the Phase I study reported excellent safety and tolerability outcomes in healthy volunteers.

“Results from this study provide the first clinical data in support of ESB1609 and enables its evaluation as a first-in-class oral S1P5 pathway-based therapeutic for treatment of neurodegenerative diseases, including Niemann-Pick C and GBA-Parkinson’s, our initial indications of focus,” said ESCAPE Bio’s Chief Medical Officer Carrolee Barlow, MD, in a statement. “This initial clinical data suggests ESB1609 has durable CNS drug exposure with a single oral dose with excellent safety and tolerability. No cardiac adverse events related to bradycardia or atrioventricular slowing were found, consistent with the high selectivity of our compound for S1P5. The data are consistent with a once daily administration therapy. We look forward to working towards a U.S. IND filing in the coming months.”

Additionally, the California-based biopharmaceutical company is in the process of advancing a mutant-selective leucine-rich repeat kinase 2 (LRRK2) G2019S kinase inhibitor for the treatment of Parkinson’s disease (PD). This program, which is currently in IND-enabling studies, mirrors a similar initiative currently pursued in $1 billion collaboration between Denali and Biogen. This Denali-led program is also studying small molecule inhibitors of LRRK2 for PD, as mutations in this gene are key factors involved in this dopamine-deficient neurodegenerative disease.

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