Jupiter Orphan Therapeutics Announces IND Submission for Mucopolysaccharidosis Type-I

Jupiter Orphan Therapeutics, Inc. recently announced it had 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.

“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). ERT slows disease progression and prolongs life; however, patient condition continues to deteriorate leaving an unmet need for a product like JOTROL,” said Chief Executive Officer, Christer Rosén of JOT.

“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,” added CSO, Dr. Marshall Hayward.

JOT has developed a unique patented trans-resveratrol product called JOTROL that 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 preclinical data, that resveratrol administration in the JOTROL formulation will deliver the necessary levels of resveratrol in plasma without causing these GI side-effects.

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. While MPSI is among the most frequent of the MPS and lysosomal storage disorders, it remains a rare disease that affects approximately 1 in 100,000 people worldwide. There are some 100 mutations in the IDUA gene that can cause MPSI with a wide range in disease presentation. The loss of IDUA results in a buildup of glycosaminoglycans (GAGs) which leads to cellular disruption and inflammation. Eventually organ failure occurs. Patient lifespan rarely exceeds 20 years, with most MPSI children dying before the age of 10, depending on the severity of the disease. Visit the National MPS Society website www.mpssociety.org and www.ryanfoundation.org for more information.

Friedreich’s Ataxia (FA) is a rare inherited disease that causes damage to the nervous system as well as diminished mobility. FA usually begins in childhood and leads to impaired muscle coordination (ataxia) that worsens over time. It is caused by a defect (mutation) in the Frataxin (FXN) gene. Friedreich’s ataxia is recessive, meaning it only occurs in someone who inherits two defective copies of the gene, one from each parent. Although rare, FA in the most common form of hereditary ataxia, affecting about 1 in 50,000 people in the US. There are no approved treatments available today. Visit the FARA (Friedreich’s Ataxia Research Alliance) website, www.curefa.org, for further details on this rare disease.

Jupiter Orphan Therapeutics, Inc. (JOT) is a clinical stage specialty pharmaceutical company developing therapies for rare diseases. JOT, a Delaware Corporation with its principal office located in Jupiter, FL, was founded in the summer of 2015. In its short period of operations, JOT has assembled a very strong management and scientific team, developed JOTROL as a platform product to treat multiple rare diseases as well as collaborating with established partners in other disease areas. For more information, visit http://www.jupiterorphan.com/.