Rodin Therapeutics today announced the initiation of a Phase 1 clinical trial of the company’s lead candidate, RDN-929, a potent and selective HDAC-CoREST inhibitor. The compound is designed to treat synaptopathies, a group of over 100 brain diseases characterized by synaptic loss and dysfunction.
The randomized, double-blind, placebo-controlled study will assess the safety, tolerability, pharmacokinetic and pharmacodynamic profile of single ascending and multiple ascending doses of RDN-929 in healthy volunteers ranging from 18 to 80 years old.
Inhibition of the CoREST complex targeted by RDN-929 is expected to reactivate neuronal gene expression, strengthen synaptic function and promote creation of new synapses while minimizing adverse effects. RDN-929 preclinical data show pro-synaptic effects and suggest a safety profile suitable for long-term dosing as a therapeutic for neurodegenerative and neuropsychiatric diseases.
“We’re thrilled to be in the clinic with our lead compound, which represents a novel approach to treating neurodegenerative diseases such as Alzheimer’s,” said Adam Rosenberg, Rodin’s president and chief executive officer. “We believe that directly targeting and strengthening synapses will lead to meaningful improvements in clinical symptoms, and we are hopeful that this approach can help patients across a broad range of disease severities. This Phase 1 trial is an important milestone for us as we advance an ambitious clinical strategy.”
Rodin is also sponsoring an ongoing, non-therapeutic clinical trial to assess the performance of a new PET ligand that enables measurement of synaptic density in the living brain. Results from this PET study and the newly launched Phase 1 trial will help guide Rodin’s planned clinical trials of RDN-929 in patients with Alzheimer’s disease, Parkinson’s disease, frontotemporal dementia and other synaptopathies.
Rodin’s lead compound is a potent and selective inhibitor of the CoREST complex. In preclinical studies, RDN-929 has demonstrated strong pro-synaptic pharmacological effects at multiple levels, including increase in the synaptic protein SV2A; significant, dose-dependent increase in dendritic spine morphology; and greater strength of synaptic function. For more information about the trial, please visit: https://clinicaltrials.gov/ct2/show/study/NCT03668314.
About Rodin Therapeutics
Rodin Therapeutics is discovering and developing first-in-class therapeutics for synaptopathies by applying novel chemical strategies to target specific HDAC complexes and upregulate key neuronal genes. Rodin’s targeted approach to strengthening synaptic integrity, backed by a robust translational strategy, has potential across multiple diseases, such as Alzheimer’s disease, Parkinson’s disease, frontotemporal dementia and schizophrenia, all of which are characterized by impaired neuronal and synaptic function. For more information, visit https://rodintherapeutics.com/ and follow Rodin on Twitter @Rodintx.