JUPITER, Fla., Sept. 22, 2020 /PRNewswire/ -- Jupiter Orphan Therapeutics, Inc., a clinical-stage drug platform company focused on neurological and rare diseases, announced today that the National Institute on Aging at the NIH will award it $1.76 million (over 1 year) to support a Phase I clinical study of JOTROL™, for the treatment of early-stage Alzheimer's disease.
The grant will support a Phase I study titled, "Safety and Pharmacokinetics of JOTROL™ for Alzheimer's Disease."
"We are very grateful and proud of this competitive award from the National Institutes of Health, as it underwent extensive scientific review and scored highly," said Christer Rosén, Jupiter Orphan Therapeutics Chairman & CEO. "Jupiter Orphan Therapeutics is focused on slowing the progression of Alzheimer's at an early stage of the disease."
Alzheimer's is a progressive brain disease that erodes thinking and memory, and gradually leads to dementia and death. An estimated 5.5 million Americans over age 65 may have the disorder, with many more under 65 affected. Although treatments are available to manage symptoms, no therapies on the market delay symptom progression. The illness costs the United States about $277 billion annually.
JOTROL™ is a proprietary patented formulation featuring the active ingredient resveratrol. Resveratrol is a naturally occurring plant compound found in small amounts in red wine, red grapes, berries and peanuts. Multiple clinical studies have found resveratrol can reduce markers of brain inflammation and oxidative stress, and boost production and function of cells' energy organelles, called mitochondria. Mitochondrial damage is a feature of Alzheimer's and other neurodegenerative diseases.
In addition, resveratrol has been shown to potently activate anti-aging factors called sirtuins. These are endogenous regulators of metabolism that produce beneficial, caloric-restriction-associated amelioration of diseases of aging. Studies of resveratrol suggest it may reduce age-related cognitive decline and reduces Alzheimer's disease-associated plaques. A daily dose of JOTROL™ delivers the equivalent resveratrol dose of approximately 20 bottles of red wine, without the side effect the wine might have.
However, earlier resveratrol clinical studies have demonstrated issues with brain and central nervous system bioavailability at orally tolerated doses. Such doses have been produced gastrointestinal side-effects including nausea and diarrhea.
The JOTROL™ delivery platform allows resveratrol to be consumed in an easily consumable, oral softgel in a patented micellar form. Delivered via the JOTROL™ platform, resveratrol infuses blood plasma and the central nervous system, and crosses the blood brain barrier, unlocking the compound's potential as a therapeutic for neurodegenerative and neurological diseases, without the GI issues. Early clinical studies of regular resveratrol for treatment of Friedreich's ataxia and Alzheimer's disease show it is safe and well-tolerated besides the associated GI side effects at high doses.
The formulation has been developed together with our technology partner, Aquanova AG, and studied in consultation with scientists at the University of Miami, Harvard, MIT and Georgetown University, as well as Australia-based Murdoch Childrens Research Institute, noted Marshall Hayward, Ph.D., Jupiter Orphan Therapeutics chief scientific officer.
"I am very pleased to receive this award, as it will enable us to take our JOTROL™ product through Phase I and set us up for Phase II applications," Hayward said. "We have a significant advantage in our development programs, guided by scientific publications on resveratrol, internally developed data, and the open-label studies conducted by MCRI in Friedreich's ataxia patients, and the Turner et.al clinical study in 119 Alzheimer's patients."
Jupiter Orphan Therapeutics' long-term focus is to develop a treatment for Alzheimer's Disease, while in parallel, developing one or more treatments for rare, "orphan" diseases including Friedreich's ataxia and Mucopolysaccharidosis Type-I, Rosén said. Its anti-inflammatory, oxidative-stress-reducing, and other mechanisms of action enable Jupiter Orphan's flagship product, JOTROL™, to have multiple potential disease applications, he said.
"This can potentially generate a development speed that is very unusual," Rosén said. "It means a successful Phase I study can generate the ability for initiation of Phase II trials in multiple indications, creating strong capital efficiency."