A 13-week GLP toxicology study with SPG302 in Beagle dogs
Abstract
There is a great unmet need for innovative new therapeutics for ALS (Lou Gehrig s disease), a degenerative disease of motor neurons in the brain and spinal cord that is almost invariably fatal within 3-5 years of diagnosis. The therapies that are currently approved for ALS provide very modest extension of life of several months and are not well tolerated by all patients. The goal of this proposal is to advance the development of a novel small molecule, SPG302, as a new potential therapeutic for patients with ALS by completing required animal toxicology studies as agreed to by the FDA. SPG302 has a unique mechanism of action wherein it induces an increase in synapses, the key connections between neurons that allow us to think, plan, remember, and control motor functions, faculties that are diminished in neurodegenerative diseases including ALS. Recent data indicate that a loss of dendritic spine synapses is an early and progressive feature of ALS pathogenesis that may contribute to both motor and cognitive symptoms. Spinogenix has developed drugs that regenerate these lost synapses. These drugs improve motor function and survival in an aggressive mouse model of ALS, as well as neural network function in ALS patient-derived induce pluripotent stem cells and respiratory motor function in a rat model of spinal cord injury. Biomarker evaluation of a synapse-specific PET imaging tracer is ongoing in preclinical models, which if successful, can easily be translated into patient trials. To our knowledge, this is the only current therapeutic under evaluation to regenerate synapses lost in ALS. Spinogenix s novel approach has the potential to demonstrate that replacing lost synapses can provide a meaningful benefit for patients with ALS. Therefore, our lead compound could be used as a monotherapy and, in principle, as a co-therapy with any among the large and diverse set of emerging therapeutics aimed at inhibiting neurodegeneration in ALS patients.
Document Details
- Document Type
- DoD Grant Award
- Publication Date
- Jan 04, 2024
- Source ID
- HT94252310580
Entities
People
- Vincent Simmon
Organizations
- United States Army