Novel Product Candidates for the Treatment of Neurological Diseases
With a deep commitment to scientific discovery and translation, we pursue any path necessary to target key biochemical checkpoints to disrupt the disease process. Our current focus is the analysis and disruption of neurodegenerative pathways in the brain and gastrointestinal tract that lead to Parkinson’s and related diseases.
Key technologies include:
- RAMP™: Re-engineering Approach with Metabolism Preserved, a method of designing novel medications by learning from clinically evaluated and/or marketed drug products to build in desirable, clinically validated safety or pharmacology characteristics from the template into new drug substances.
- Prodrug delivery methods: Novel prodrug linkers that enhance drug absorption, delivery and/or suppress undesired on-dosing side effects.
RAMP™ Drug Innovation Engine
Delivering First-in-class Small-molecule Product Candidates
Our Re-engineering Approach with Metabolism Preserved (RAMP™) drug innovation engine enables us to design and develop novel small-molecule product candidates, leveraging the clinical experience gained from currently marketed or clinically evaluated small molecule drugs that target CNS or non-CNS diseases. Using preexisting chemical entities as a template, we pursue clinically validated targets to create first-in-class small-molecule product candidates with predicted human safety profiles to treat neurodegenerative diseases.
RAMP Technology Enables:
- Safety enhancements achieved through chemical modifications that could lead. to dose reduction, enabling chronic administration with minimized side effects.
- Preservation of the favorable side-effect profile of the “template” medication, achieved through medicinal chemistry changes to preserve how the drug is metabolized in the body.
- The ability to generate multiple novel/improved drug molecules that address a range of therapeutic areas from the same clinically validated kinase inhibitor.
- Medicinal chemistry insights spanning protein kinase families.
- Medicinal chemistry and structural engineering techniques for generating kinase inhibitors with greater selectivity and optimized pharmacological properties for drugs to treat neurodegeneration.
- Discovery workflow attuned to novel pharmacological outcomes.
RAMP™ Enables the Discovery of Novel Candidates
Treating Parkinson’s and Related Diseases with Small-Molecule Kinase Inhibitors
We are utilizing our RAMP™ discovery engine to develop a new class of protein kinase inhibitors for the potential treatment of kinase-sensitive CNS diseases. These candidates could be more potent and safer than commercially marketed kinase inhibitors and be administered chronically and systemically.
We are actively developing novel kinase inhibitor treatments for additional Parkinson’s-related disorders, including multiple system atrophy (MSA), a neurodegenerative disorder that shares the hallmarks of c-Abl activation and alpha-synuclein pathology with Parkinson’s disease. Future programs will include Dementia with Lewy Body (DLB), a Parkinson’s-like disease characterized by alpha-synuclein aggregates that primarily lead to cognitive decline without a significant movement disorder component.
Our intellectual property includes patents issued or pending, as well as know-how related to the composition of matter and broad use of our technologies across neurodegenerative diseases inside and outside of the brain. We have carved out a broad and deep intellectual property position that enables the development of the Company’s internal product pipeline and supports value-creating partnerships with biotechnology and pharmaceutical companies.
Our Prodrug Delivery Technology
Our prodrug technology platform enables potentially greater control over the non-hematological side effects common to kinase inhibitor therapies, such as nausea or diarrhea, and it may improve drug delivery into the target tissue. Using a proprietary set of linkers and attachment points, our oral prodrugs are absorbed intact and then fall apart into the active ingredient and the linker. We believe this approach could lead to kinase inhibitors that are safer and better tolerated, with the potential to treat a variety of indications.
Application of RAMP™ Drug Innovation Engine
Using our drug innovation engine is the basis for discovery of our product candidates and we have successfully developed several small-molecule medications with the potential to treat multiple different diseases through the same target approach.Explore Our Pipeline