● 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 amyotrophic lateral sclerosis, Alzheimer’s disease, multiple sclerosis and Parkinson’s disease

● Preliminary data expected in 1H 2024

Ashvattha Therapeutics (“Ashvattha”), a clinical-stage company advancing a new class of nanomedicine therapeutics that traverse tissue barriers to selectively target activated cells in regions of inflammation, today announced that the first patient has been enrolled in its expanded Phase 1/2 study of ¹⁸F-OP-801, a hydroxyl dendrimer (HD) imaging agent. The study will evaluate the safety, pharmacokinetics and biodistribution of an intravenously administered dose of ¹⁸F-OP-801 in patients with amyotrophic lateral sclerosis (ALS), Alzheimer’s disease (AD), multiple sclerosis (MS) and Parkinson’s disease (PD).

“This study builds on preclinical data which demonstrated ¹⁸F-OP-801's ability to cross the blood-brain barrier and be taken up by activated microglia in regions of neuroinflammation in multiple disease models. We have already demonstrated in the first part of this clinical trial that there is no appreciable uptake in the brains of healthy volunteers, as expected. Our focus now turns to enrolling subjects with neurodegenerative conditions to determine the uptake of ¹⁸F-OP-801 in regions of neuroinflammation to support the use of this agent as a companion biomarker for one of our HD therapeutics,” said Jeffrey Cleland, Ph.D., Chairman, President, and CEO of Ashvattha Therapeutics. “We look forward to evaluating imaging data in this study over the next several months and also expect to have initial results from our Phase 2 trial of D-4517.2 in patients with wet AMD and DME in the first half of 2024.”

The Phase 1/2 study will initially enroll up to 20 subjects with ALS, AD, MS, and PD. In addition to evaluating the safety and tolerability of ¹⁸F-OP-801, major objectives of the study are to measure the biodistribution and clearance of ¹⁸F-OP-801 and to evaluate its ability to detect regions of neuroinflammation via PET imaging in subjects with disease compared to age-matched healthy volunteers. Additional objectives include determining the correlation between PET imaging signal, biomarkers of disease activity and clinical disease assessments. The study will also assess test/retest repeatability of ¹⁸F-OP-801 uptake in a subset of subjects.

About ¹⁸F-OP-801
¹⁸F-OP-801 is a hydroxyl dendrimer (HD) imaging agent that has been shown to cross the blood-brain barrier and selectively target activated microglia and macrophages in regions of neuroinflammation in multiple animal models1. These studies establish the potential to visualize the extent and progression of neuroinflammation with high specificity and sensitivity in human neurodegenerative diseases via serial imaging with ¹⁸F-OP-801. Furthermore, because all HDs distribute the same way in the body, the amount of ¹⁸F-OP-801 in the brain provides an estimate of the amount of HD therapeutic that will get to the same cells in the brain enabling an image-treat-reimage paradigm using ¹⁸F-OP-801 as a companion biomarker for treating conditions such as amyotrophic lateral sclerosis, Alzheimer’s disease, multiple sclerosis, and Parkinson’s disease.

About Ashvattha Therapeutics
Ashvattha Therapeutics is advancing a new class of clinical-stage nanomedicine therapeutics that traverse tissue barriers to selectively target activated cells only in regions of inflammation. Our targeted nanomedicine approach seeks to redefine precision medicine, empowering a new standard of care across ophthalmology, neurology, and inflammation. 

[1] 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