Sachi Bio's lead program NI112 shows excellent Safety-Toxicity, brain-delivery, and biodistribution profile in Investigational New Drug (IND) enabling studies.
Promising results of extensive dosing studies and safety/toxicity studies carried out in large and small animals, shows brain-penetrant Nanoligomer lead NI112 crosses the blood brain barrier effectively, distributes well among different brain regions, and is safe and targeted. This study also enables us to find the ideal administration route, and dosing frequency for NI112 when changing the route of administration or between different species, as well as translational parameters for human clinical testing. The results of the study are published recently in ACS Pharmacology and Translational Science. Sachi Bio's lead molecule, NI112, is specifically designed to target neuroinflammation, offering promising therapeutic potential across various disease models such as Amyotrophic Lateral Sclerosis (ALS), Multiple Sclerosis (MS), Alzheimer's Disease and Related Dementias (ADRD), and other protein misfolding diseases.
Building on these successful studies, Sachi Bio will complete and submit the IND-application for our lead program. "IND enabling" refers to the process of preparing and submitting an Investigational New Drug (IND) application to the Food and Drug Administration (FDA) in the United States. This application is a crucial step in the development of a new pharmaceutical product, particularly in the context of clinical trials.
Advancement of Nanoligomers towards the clinic is a major step for the field of RNA therapeutics, and towards finding treatments for highly debilitating Neurodegenerative diseases including ALS, MS, and ADRD.
Following Sachi Bio's mission. we continue to be committed to bringing better, rational, accelerated and accessible treatments to patients and hope for them and their loved ones.
References:
Sadhana Sharma, Sydney Risen, Vincenzo S. Gilberto, Sean Boland, Anushree Chatterjee, Julie A. Moreno, and, Prashant Nagpal* (2024). ACS Chemical Neuroscience 15, 7, 1596–1608.
Sydney Risen, Breonna Kusick, Sadhana Sharma, Vincenzo S. Gilberto, Stephen Brindley, Mikayla Aguilar, Jared M. Brown, Anushree Chatterjee, Julie A. Moreno*, and Prashant Nagpal* (2024). ACS Pharmacol.ogy and Translational Science (In press).
Sydney J. Risen, Sean W. Boland, Sadhana Sharma, Grace M. Weisman, Payton M. Shirley, Amanda S. Latham, Arielle J. D. Hay, Vincenzo S. Gilberto, Amelia D. Hines, Stephen Brindley, Jared M. Brown, Stephanie McGrath, Anushree Chatterjee, Prashant Nagpal, and, Julie A. Moreno* (2024). ACS Chemical Neuroscience 15, 7, 1533-1547.
Devin Wahl, Sydney J. Risen, Shelby C. Osburn, Tobias Emge, Sadhana Sharma, Vincenzo S. Gilberto, Anushree Chatterjee, Prashant Nagpal, Julie A. Moreno, Thomas J. LaRocca* (2024). BioRxiv.