Mathew Shanley, Rare Disease Report. FEBRUARY 05, 2018
Jupiter Orphan Therapeutics, Inc. (JOT) announced this morning that it intends to submit an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for Jotrol in mucopolysaccharidosis type 1 (MPS I), among other indications, within the next few weeks.
"We will initiate the IND in MPS I and plan to cross-reference PK and safety data in follow-on indications. We are well prepared to gear up for a study in Friedreich's Ataxia (FA) and will thereafter determine if Mitochondrial Encephalopathy, Lactic Acidosis, and Stroke-like episodes (MELAS) or Leber's Hereditary Optic Neuropathy (LHON) will be the 3rd indication we approach. An IND submission for FA is planned for quarter 2 of 2018, and for MELAS and/or LHON no later than quarter 4," stated JOT Chief Security Officer Dr. Marshall Hayward in a press release.
JOT has used an isomer of the resveratrol to develop a pharmaceutical grade compound that can be properly tested in clinical trials, and successful pre-clinical data have shown that it can increase levels of frataxin.
Jotrol IND Application Submission for MPS and Friedrich's Ataxia Imminent