ESCAPE Bio to Present at the Cowen 40th Annual Health Care Conference
SAN FRANCISCO, February 24, 2020 — ESCAPE Bio, Inc., a clinical stage company developing novel, precisely targeted therapeutics for geneticaDate: Tuesday, March 3, 2020
Time: 10:30 a.m. ET
Location: Boston Marriott Copley Place in Boston, MA
Ms. Smith will present program and corporate updates. ESCAPE recently initiated a Phase 1 multiple ascending dose study in healthy volunteers with ESB1609, a S1P5 agonist being developed for the treatment of CNS lysosomal storage disorders. The company has also initiated preclinical toxicology studies with their G2019S LRRK2 inhibitor being developed as a potential treatment for Parkinson’s disease.
About ESB1609, a S1P5 Agonist for CNS Lysosomal Storage Disorders
ESB1609 is a novel, orally-administered, brain-penetrant and selective sphingosine 1-phosphate 5 (S1P5) receptor agonist. S1P5 receptors are one of five receptors within the G-protein-coupled S1P receptor family (S1P1 – S1P5). S1P5 couples to Gi and G12 and is predominantly expressed in the central nervous system (CNS) and natural killer (NK) cells. The endogenous ligand for S1P5 is sphingosine-1-phosphate (S1P), a sphingolipid that plays a significant role in many aspects of cellular homeostasis and proliferation. Activation of S1P5 upregulates several CNS lipid transporters and has been shown to normalize brain ceramide and sphingosine phosphate levels and promote clearance of aggregation-prone proteins across multiple pre-clinical models of neurodegeneration. Multiple genetic forms of neurodegeneration cause perturbations in the sphingolipid pathway and ultimately, lysosomal dysfunction. ESB1609 is currently in a Phase 1 Multiple-Ascending Dose study in healthy volunteers.
About LRRK2 Inhibitor Program for Parkinson’s Disease
Pathogenic LRRK2 mutations co-segregate with familial Parkinson’s Disease (PD). Almost all PD patients carrying a G2019S LRRK2 variant have two versions of LRRK2 protein; one mutant variant with excessive kinase activity (up to 10-fold) and one healthy version, critical for regulating intracellular vesicular trafficking throughout the body. G2019S is the most common LRRK2 pathogenic mutation, estimated to account for 1-3% of all PD and 1% of people of Ashkenazi Jewish descent. G2019S LRRK2 PD patients experience the same progression of symptoms as idiopathic PD, with onset of tremors and rigidity at rest in their 50s, followed by deterioration of motor and cognitive function and progressive neuropsychiatric symptoms, which culminate in premature death. Pathobiological data suggest that alpha-synuclein, a protein that normally regulates dopamine, forms aggregates which can propagate from one neural cell to another and is specifically harmful to neurons that produce dopamine in the substantia nigra of the brain. There are no disease modifying therapies approved.
About ESCAPE Bio
ESCAPE Bio is a clinical stage, privately held biopharmaceutical company developing novel, precisely targeted therapeutics for genetically defined neurodegenerative diseases. ESB1609 is in a Phase 1, randomized, double-blind, placebo-controlled, safety, tolerability, pharmacokinetic and biomarker study of escalating multiple doses in healthy volunteers. ESCAPE’s pipeline includes small molecules targeting known genetic drivers of CNS disorders, including ESB1609, an S1P5 agonist for the treatment of CNS lysosomal storage disorders and GBA Parkinson’s; a G2019S-selective kinase inhibitor for Parkinson’s Disease (PD) patients with that mutation; and an Alzheimer’s disease program targeting ApoE4. For additional information, please visit www.escapebio.com.