Jupiter Orphan Therapeutics has submitted its IND to the FDA on March 12, 2018 for Mucopolysaccharidosis Type-I (MPSI). This will be the first of several anticipated IND submissions in 2018 utilizing JOT's platform product, JOTROL™.
Mucopolysaccharidosis I (MPSI, Hurler Syndrome, Hurler-Scheie Disease, Scheie Syndrome) is an autosomal recessive genetic disorder in which the alpha-L-iduronidase (IDUA) enzyme is greatly depleted or absent from the affected individual. The loss of IDUA results in a buildup of glycosaminoglycans (GAGs) which leads to cellular disruption and inflammation, and eventually organ failure occurs.
"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 clear unmet medical needs of MPSI patents, including those who are on the standard of care Enzyme Replacement Therapy (ERT)." said chief executive officer, Christer Rosén of JOT.
JOT’s JOTROL™ is expected to deliver the documented high amount of resveratrol in blood plasma that is required to achieve therapeutic effects. In previous studies, patients who have been given high doses of resveratrol experienced severe GI-side effects, thus limiting utilization of resveratrol in the pharmaceutical field. JOT is expecting, based on very successful pre-clinical data, that resveratrol administration in the JOTROL™ formulation will deliver the necessary levels of resveratrol in plasma without causing these GI side-effects.