Enabled Therapeutics announced the successful completion of its pre-Investigational New Drug (pre-IND) meeting with the U.S. Food and Drug Administration (FDA) for CPT-021, a first-in-class oral therapy targeting neuroinflammation in Alzheimer's disease. The regulatory feedback confirms alignment between the company's development plan and FDA requirements for IND submission, setting the stage for potential clinical trials.
"This meeting was a critical milestone," said Sara Isbell, CEO of Enabled Therapeutics. "The FDA's response confirms our plan is on track with no hurdles. With funding, we can complete the necessary studies in 12 months and move CPT-021 into clinical trials—bringing much-needed innovation to Alzheimer's patients."
Pathotargeting: A Precision Approach to Neurological Treatment
CPT-021 employs a novel mechanism called pathotargeting through the company's proprietary Capton® platform. Unlike conventional therapies that deliver active drug throughout the entire brain, CPT-021 circulates in a precursor form and completes its synthesis into homotaurine only in brain regions where neuroinflammation is present.
The drug candidate easily crosses the blood-brain barrier and targets areas characterized by high oxidative stress and metal ions—hallmarks of Alzheimer's pathology. In these regions, CPT-021 undergoes a natural chemical reaction to become active, ensuring high drug concentrations where needed while minimizing off-target effects.
Dr. Todd Zankel, Chief Scientific Officer of Enabled Therapeutics, explained: "For decades, CNS drugs have failed because they expose the entire brain to active drug, limiting efficacy before toxicity takes over. Captons change that. With pathotargeting, we're not just improving delivery—we're rewriting the rules of neurological drug design."
This approach addresses a fundamental challenge in CNS drug development. Homotaurine has demonstrated the ability to reduce CNS inflammation by preventing immune cell invasion via the GABA system, but previous clinical trials struggled with efficacy due to dosing limitations imposed by toxicity concerns.
The Science Behind Capton® Technology
The Capton® platform leverages thiol-based chemistry to enable precise drug activation exclusively in diseased brain tissue. This chemical process, rather than biological activation, makes translation from laboratory to human trials more predictable by bypassing biological variability.
Marcel Gradidge, Chief Business Officer at Enabled Therapeutics, noted: "The future of Alzheimer's treatment isn't about plaques—it's about precision."
The technology addresses three major barriers that have historically hindered neurological treatments: blood-brain barrier limitations, systemic toxicity, and unpredictable biological activation. By ensuring that the drug forms only where needed, Captons allow for higher drug concentrations at disease sites without increasing systemic toxicity.
Regulatory Path and Development Timeline
With regulatory clarity now established, Enabled Therapeutics has outlined a 12-month plan to complete IND-enabling studies and manufacturing in collaboration with CRO and CDMO partners. The FDA has specified next steps for CPT-021, including GLP toxicology studies and clinical trial planning—key milestones toward potential Fast Track designation.
If funding is secured, the company expects to complete Phase I trial enrollment within 18 months. Under Fast Track designation, CPT-021 could potentially reach patients within three to four years, offering an affordable, disease-modifying treatment option for Alzheimer's disease.
Broader Applications in Neurology
While Alzheimer's disease represents the initial focus, the Capton® technology platform holds promise for treating various other neurological conditions, including refractory epilepsy, traumatic brain injury, glioblastoma, and rare neurological diseases.
Alzheimer's disease affects approximately 6.7 million Americans, with numbers expected to rise significantly as the population ages. Current treatment options primarily address symptoms rather than modifying disease progression, highlighting the urgent need for innovative approaches like CPT-021.
By enabling selective drug activation in diseased tissue while sparing healthy regions, the pathotargeting approach could potentially reshape treatment paradigms across neurology, offering new hope for conditions with significant unmet medical needs.
