– The study will evaluate the safety, tolerability and ability of [18F]OP-801 to cross the blood brain barrier and selectively target neuroinflammation in individuals with ALS and healthy volunteers–
REDWOOD CITY, Calif., February 6, 2023 – Ashvattha Therapeutics (“Ashvattha”), a clinical-stage company developing novel nanomedicines called hydroxyl dendrimer therapeutics (HDT), today announced that the first patient has been enrolled in a Phase 1/2 clinical study to evaluate the safety, pharmacokinetics and biodistribution of an intravenously administered dose of [18F]OP-801 in healthy volunteers and in patients with amyotrophic lateral sclerosis (ALS) (NCT05395624). [18F]OP-801 is a hydroxyl dendrimer (HD) imaging agent that selectively targets reactive macrophages and microglia – key markers of neuroinflammation.
“The initiation of the clinical trial evaluating [18F]OP-801 as an imaging agent is a significant step to unlocking the potential of our proprietary HD technology for treatment of neurological diseases,” said Jeffrey Cleland, Ph.D., chairman, CEO and president of Ashvattha Therapeutics. “With [18F]OP-801, we will be able to estimate HDT uptake in the diseased part of the brain prior to treating patients with the HDT. This approach, if successful, will reduce clinical risk by ensuring the correct amount of drug reaches the target to treat the neurological disease.”
Farshad Moaradi, M.D., Ph.D. clinical associate professor of radiology- rad/nuclear medicine at Stanford Medicine added, “An obstacle to the treatment of neurodegenerative diseases like ALS is ensuring that treatment reaches areas of disease in the brain. This study will determine if visualizing [18F]OP-801 uptake can pinpoint regions of inflammation and disease with enough specificity to provide insights into future patient selection, potentially making precision medicine even more precise.”
The study is an interventional, non-randomized open-label Phase 1/2 clinical trial to evaluate the safety, pharmacokinetics and biodistribution of a single dose of [18F]OP-801 after intravenous administration to healthy volunteers and patients with ALS. The study will also measure the biodistribution and clearance of the HD imaging agent in both populations and evaluate the uptake of [18F]OP-801 in regions of neuroinflammation in ALS patients using PET/computed tomography (CT) scans. The study is expected to enroll up to 26 participants.
[18F]OP-801 is a hydroxyl dendrimer (HD) imaging agent that tracks uptake of HDs across tissue barriers including the blood-brain barrier. [18F]-OP-801 selectively (>95%) targets reactive microglia/macrophages in regions of neuroinflammation in animal models of disease1[18F]OP-801 shows promise for visualizing the progression of neuroinflammation with high specificity and sensitivity in human neurodegenerative diseases like ALS, Alzheimer’s disease and Parkinson’s disease. A Phase 1/2 clinical trial of [18F]OP-801 is currently underway in healthy volunteers and ALS patients.
About Ashvattha Therapeutics
Ashvattha Therapeutics is a clinical-stage biotech company developing novel hydroxyl dendrimer therapeutics (HDTs) targeting unmet medical needs in ophthalmology, neurology, inflammation, and neuro-oncology. Hydroxyl dendrimers (HDs) are a targeted nanomedicine technology exclusively licensed from our founders, Kannan Rangaramanujam and Sujatha Kannan at Johns Hopkins University. HDs chemically conjugated to disease-modifying drugs create novel proprietary HDTs selectively targeting reactive inflammatory cells in disease tissue with localized sustained effects. Ashvattha has initiated multiple programs with HDTs focused on ocular neovascular disease including neovascular age-related macular degeneration (AMD) and diabetic macular edema (DME), neurology and hyperinflammation. For more information, visit: www.avttx.com.
 Henningfield, C.M., Cleland, J.L., Sharma, R., Green, K.N.Selective targeting of plaque-associated microglia through systemic dendrimer administration in an Alzheimer’s disease model. Alzheimer’s & Dementia. 2020; Volume 16. Issue S2. https://doi.org/10.1002/alz.040661