Enterin, Inc. a CNS pharmaceutical company based in Philadelphia and developing novel compounds to treat Parkinson’s disease (PD) has enrolled the first patient in the RASMET study. The study is a Phase 1/2a randomized, controlled multi-center study involving patients with PD and taking place at 12 US sites. It will enroll 50 patients over a 9-12 month period. The first Stage is open label and involves single escalating doses in 10 patients with PD. Participating sites include Denver, Boca Raton, Tampa and Cleveland. Details relating to the study can be found at ClinicalTrials.gov. and contact information is available on Enterin’s website, www.enterininc.com.
The study will establish the safety, tolerability and efficacy of an orally administered synthetic derivative of squalamine which is not absorbed into the blood stream. The compound acts locally on the enteric nerve cells of the gut, stimulating gut motility and altering afferent neural signalling from gut to brain. It has the potential to ameliorate some or all of the non-motor symptoms of Parkinson’s disease, including constipation, fragmented sleep and REM-behavior disorder, and to modify disease progression.
Synthetic squalamine was recently shown to prevent the buildup and reduce the toxicity of alpha-synuclein, implicated in the pathogenesis and progression of Parkinson’s disease. The compound was shown to displace alpha-synuclein aggregates from the inner wall of nerve cells, and to prevent the stiffness which develops in C. Elegans worms engineered to produce alpha-synuclein in its muscles. The results were published online in the February 7th edition of the Proceedings of the National Academy of Sciences (Perni et al, PNAS Vol 114, no 6, 2017, doi: 10.1073/pnas.1610586114). Links to the article and to the press coverage can be found at the Enterin website.