IND Application Submitted for Potential MPS-I Therapy

Jupiter Orphan Therapeutics, Inc. (JOT) has submitted the first of the several highly anticipated investigational new drug (IND) applications for potential therapies that utilize Jotrol, its platform product.

The IND application submitted to the U.S. Food and Drug Administration (FDA) late yesterday is indicated for Mucopolysaccharidosis Type-I (MPSI), an autosomal recessive genetic disorder in which the alpha-L-iduronidase (IDUA) enzyme is significantly depleted or completely nonexistent from the person affected.

The loss of IDUA results in an accumulation of glycosominoglycans (GAGs), which results in cellular disruption and inflammation and eventual organ failure.

Jotrol, a patented trans-resveratrol formulation, was developed with the intention of delivering the documented high amount of resveratrol in blood plasma that is required to achieve therapeutic effects. Its true value comes in its ability to elude the typical gastrointestinal (GI) problems that exist with high doses of the plant-based polyphenol compound.

"This is a major milestone in JOT's relatively short history and marks an important inflection point,” said Chief Executive Officer, Christer Rosén of JOT in a press release. 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). ERT slows disease progression and prolongs life, however patient condition continues to deteriorate leaving an unmet need for a product like JOTROL.”

JOT has utilized an isomer of the resveratrol — which is commonly found in grapes, berries and nuts – to develop a pharmaceutical grade compound that can be properly evaluated in clinical trials. Successful pre-clinical data have exhibited its ability to increase frataxin levels.

"We plan to cross-reference PK and safety data from this study in follow on indications. It is exciting to have developed a platform product that has the potential to be a treatment for several diseases. We are well prepared to gear up for a study in Friedreich's Ataxia, in parallel to MPSI, and will thereafter start our work in mitochondrial dysfunction related diseases," stated CSO, Dr. Marshall Hayward.

While MPSI, or Hurler Syndrome, is the most frequent of the MPS and lysosomal storage disorders, only an estimated 1 in 100,000 people are affected by it worldwide. The patient lifespan rarely exceeds 20 years, with most children affected by MPSI dying before the age of 10.

In August, JOT was granted orphan drug designation by the FDA for Jotrol as a potential therapy for patients with Friedrich’s Ataxia.

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