NOVATO, Calif., Jan. 9, 2020 /PRNewswire/ — Mercaptor Discoveries (Mercaptor), a company developing therapeutic agents active exclusively at the site of injury, announced the advancement of its preclinical program in epilepsy. The program culminates in human trials, beginning in early 2022.
A preclinical-stage company, Mercaptor tackled 2019 with all eyes on lead-selection for epilepsy. Their primary candidate has demonstrated reproducible POC and efficacy in animal seizure models.
Mercaptor is answering the immense need for a new approach to treating epilepsy and other CNS disorders. According to the Epilepsy Foundation, each year, approximately 150,000 Americans will be diagnosed with this CNS disorder. Approximately 1-in-26 people in the US will be diagnosed at some point in their lifetime. The disease has global reach, with more than 65 million suffering worldwide.
The fourth most common neurological disease, epilepsy is characterized by seizures along with a wide variety of related symptoms. Patients with symptomatic disease may suffer a reduction in lifespan averaging ten years. About 1-in-1,000 epilepsy patients die from SUDEP (Sudden Unexpected Death in Epilepsy) each year.
The Global Epilepsy Market is projected to reach USD 9,509.2 million by 2023.
Many antiepileptic drug candidates (AEDs) with positive preclinical results fail in the clinic. These agents often have poor brain penetration, but are ironically active everywhere else, increasing unwanted off-target effects, providing minimal therapeutic benefit. There has been little success in targeted drug development for the CNS, with most drugs on the market showing high toxicity and low efficacy.
“High toxicity and low efficacy is a crushing scenario for patients with epilepsy,” said Dr. Todd Zankel, CSO and co-founder of Mercaptor. “We plan on flipping this relation on its head by creating highly effective drugs that are safe and tolerable by design.”
Mercaptor is developing Captons, a new class of therapeutic agents. Captons are inactive drugs that become active ONLY when encountering damaged areas inside the brain. Many drugs with documented activity against CNS disorders—including anti-inflammatory, immunosuppressant, and anti-excitatory —can be “captonized.” Captonization inactivates the drug while facilitating improved transit across the blood-brain barrier. Distributed evenly throughout the brain, exposure to damage causes Capton activation and target engagement. In animal studies, Captons are observed to distribute efficiently to the CNS and to undergo near-quantitative, seizure-dependent activation in areas of stress. A recently published paper (DOI: 10.1002/ana.25474, PMID: 30937971) demonstrated that hydrogen peroxide, a molecule involved in activating Captons, is the trigger for all focal seizures. If true, Captons would not only control seizures, but would be the cure for Epilepsy.