Jotrol IND Application Submission for MPS and Friedrich's Ataxia Imminent

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.

In August, JOT was granted orphan drug designation by the FDA for Jotrol as a potential therapy for patients with Friedrich’s Ataxia, and the company has stressed that this highly anticipated IND submission will be the first of several for the platform product.

"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.

The trans-Resveratrol formulation is projected to manufacture the elevated amount of resveratrol in blood plasma essential to reaching therapeutic conclusions, while eluding the typical gastrointestinal problems that exist with high doses of resveratrol. Reversatrol is a polyphenol compound often found in grapes, berries and nuts, and has long been believed to have anti-cancer, anti-diabetic, cardio protective, antioxidant, and neuroprotective properties.

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.

"This is a major milestone in JOT's relatively short history and marks an important inflection point. The significance of this is quite unique since the initial PK-study for MPSI is expected to lead to results that can be utilized in upcoming Phase II trials in several indications. Jotrol is being developed to address the unmet medical needs of MPSI patents, including those who are on standard of care therapies," said Chief Executive Officer, Christer Rosén.

Resveratrol, as a small molecule with high target tissue and blood-brain barrier penetration, can find utility as monotherapy or as an adjunct to biological treatment or other approaches that might not address all patient needs. While historical studies have associated the product with severe gastrointestinal (GI)- related adverse events (AEs), JOT expects, based on very successful pre-clinical data, that administration of the Jotrol formulation will deliver the necessary levels of resveratrol in plasma without generating any severe side effects.

For more from the FDA, follow Rare Disease Report on Facebook and Twitter.