Jupiter Orphan Therapeutics Inc. (JOT) announced that David Sinclair, a Harvard Medical School professor with an interest in the dietary supplement resveratrol and its potential benefit in treating diseases such as Friedreich’s ataxia (FA), has joined the company as the co-chairman of its scientific advisory board.
“I am excited about being able to work with JOT to bring drugs to patients who are waiting for a solution to their rare disease. JOTROL opens up the possibility that resveratrol will finally realize its potential to revolutionize human health,” Dr. Sinclair said in a press release.
Resveratrol is a natural antioxidant found in red grape skins, blueberries and some other berries, Japanese knotweed and peanuts, and is believed to protect cells against environmental stress. Because it is currently considered a dietary supplement, it is not an approved medication.
Professor Martin Delatycki, with the Murdoch Children’s Research Institute (MCRI) in Australia (which entered into a development agreement with Jupiter in January), contacted Dr. Sinclair and told him of the resveratrol JOTROL product (JOT101) the company is developing. JOT101 was designed in collaboration with the MCRI to treat FA.
JOT101 was evaluated in an open-label clinical trial, led by Professor Delatycki and MCRI, in 24 human subjects. The results indicated that a high daily dose of nutritional grade trans-resveratrol can stop FA progression, but the high dosing also led to adverse side effects. More work is needed on the product’s formulation and, possibly, its delivery system.
“If we can show that JOT101, an improved formulation of resveratrol, is safe, well-tolerated and slows the progression or even reverses some of the symptoms, this would be a significant advancement for people with Friedreich’s ataxia” Professor Delatycki said in a January press release announcing the development agreement. “Our collaboration with Jupiter Orphan Therapeutics gives us the greatest chance of achieving this.”
Dr. Sinclair is currently co-director of the Paul F. Glenn Center for the Biology of Aging and a professor of genetics at Harvard. He has co-founded eight biotech companies, four of which have gone public, and he is the co-discover of Sirtuin-activating compounds (STACs), like resveratrol and SRT2104. He has authored 170 publications and owns 40 patents.
“This is a fantastic addition to our company and Dr. Sinclair’s knowledge, network and reputation is very valuable to a small company like Jupiter Orphan Therapeutics,” said Christer Rosén, chief executive officer of JOT. The pharmaceutical company specializes in rare diseases associated with single gene deficiencies, including FA.
FA is caused by a mutation in the FXN gene. The recessive defect – meaning it only occurs when someone inherits two defective copies, one from each parent – is rare even though it is the most common form of hereditary ataxia, affecting nearly 1 in every 50,000 people in the United States.