Disarm Therapeutics Reports Preclinical Data Demonstrating Small Molecule SARM1 Inhibitors Preserve Both Axonal Structure and Function In Vitro and In Vivo
- Category: Small Molecules
- Published on Wednesday, 23 October 2019 09:07
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Presentation at Neuroscience 2019 shows that Disarm’s novel small molecule SARM1 inhibitors reproduce the axonal protection phenotype seen with SARM1 genetic deletion
CAMBRIDGE, MA, USA I October 22, 2019 IDisarm Therapeutics, a biotechnology company creating a new class of disease-modifying therapeutics for patients with axonal degeneration, today announced preclinical data demonstrating that small molecule inhibitors of SARM1 protect axons in both in vitro and in vivo models of axonal degeneration.
“Today’s findings are the first reported demonstration of pharmacologic SARM1 inhibition replicating the axonal protection we’ve previously described in genetic knockout models,” said Alvin Shih, M.D., President and CEO of Disarm Therapeutics. “SARM1 is the central driver of axonal degeneration, and today’s data further validate that it is an important and also druggable target. This marks significant progress in our development of oral SARM1 inhibitors.”
The data, presented today in a poster at the Society for Neuroscience (SfN) Annual Meeting, show that SARM1 inhibitors protect axons, including human iPSC-derived motor axons, in vitro from multiple causes of degeneration, including traumatic, chemotoxic, and mitochondrial insults. In addition, pharmacologic inhibition of SARM1 via oral, small molecule inhibitors prevented axonal degeneration and preserved axonal structure and function in a rodent model of chemotherapy-induced peripheral neuropathy (CIPN). Protection from axonal degeneration was measured using neurofilament light chain (NfL), a downstream biomarker of axonal degeneration that can be detected in blood.
“This is a breakthrough for Disarm and for patients affected by axonal degeneration,” said Rajesh Devraj, Ph.D., Chief Scientific Officer and Founder of Disarm Therapeutics. “We are moving rapidly to develop potent, orally active inhibitors of SARM1 to address the fundamental pathological process of axonal degeneration that drives disability progression in patients with diseases such as MS, ALS, and CIPN.”
The Disarm SfN 2019 poster can be found here.
About Disarm Therapeutics
Disarm Therapeutics is a biotechnology company that is creating a new class of disease-modifying therapeutics for patients with axonal degeneration, a central driver of neurological disease-causing disability and disease progression. By inhibiting the SARM1 protein, identified by the company’s scientific founders as the central driver of axonal degeneration, these therapeutics may prevent the loss of axons in chronic and acute diseases of the central, ocular, and peripheral nervous systems. For a broad range of diseases including multiple sclerosis, amyotrophic lateral sclerosis, glaucoma, and peripheral neuropathies, the therapeutic goal is to prevent further degeneration, stabilize disease, and allow for functional recovery. Disarm was founded by Atlas Venture, Dr. Jeffrey Milbrandt and Dr. Aaron DiAntonio of Washington University in St. Louis, and a team of exceptional scientists and drug developers committed to developing a new treatment paradigm for patients with neurological diseases. For more information, please visit www.disarmtx.com.
SOURCE: Disarm Therapeutics