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Jupiter Orphan Therapeutics in Process to Submit IND for MPSI and Other Indications

Monday, February 5, 2018   (0 Comments)
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Jupiter Orphan Therapeutics in Process to Submit IND for MPSI and Other Indications


JUPITER, Fla., Feb. 5, 2018 /PRNewswire/ -- Jupiter Orphan Therapeutics, Inc. ("JOT"), Jupiter, FL, today announced that it plans to submit an IND to FDA within the next few weeks. 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 unmet medical needs of MPSI patents, including those who are on standard of care therapies," said Chief Executive Officer, Christer Rosén.

"We will initiate the IND in Mucopolysaccharidosis Type I (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. We are also very grateful to charitable foundations including Gene Spotlight, Inc. which has funded a portion of this vital research to help treatments for rare diseases come to market," stated CSO Dr. Marshall Hayward.

"Being involved in this project from its inception, including first identifying the potential for therapeutic use in MPSI patient cells then in rodents and finally to see the product be submitted to FDA is an outstanding achievement for our scientific team and MPSI patients and Jupiter Orphan Therapeutics who understood the importance and worked hard to develop the idea. It shows that together we hold great potential to make a meaningful impact on these terrible diseases," said Consulting VP of Scientific Research, Dr. Shaun Brothers who has received NIH and MPS foundation funding to study the disease.

"The MPS I community applauds the efforts of JOT and is excited about the announcement of an IND application for a potential new MPS I therapy," said Mark Dant, Founder and Volunteer Executive Director of the Ryan Foundation, a non-profit founded in 1992 to fund translational science to treat orphan diseases with a specific focus on MPS I and Chairman of the Board of the EveryLife Foundation for Rare Diseases, a science-based advocacy organization that works to bring lifesaving treatments to the 30 million Americans with rare diseases.

JOT has developed a unique patented formulation of trans-resveratrol called JOTROL™. JOTROL™ is expected to deliver the high amount of resveratrol in blood plasma that is believed to be required to achieve therapeutic effects. These high doses have earlier been plagued with severe GI-side effects that has limited 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 generating any severe GI side-effects. Further, as a small molecule with high target tissue and blood-brain barrier penetration, resveratrol can find utility as monotherapy or as an adjunct to biological treatment or other approaches that may not address all patient needs.

About Mucopolysaccharidosis Type I (MPSI)

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 for the severe disease variants rarely exceeds 20 years, with many MPSI children dying before the age of 10. Visit the National MPS Society website and for more information. 

About Friedreich's Ataxia (FA)

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) which 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 United States. There are no approved treatments available today. Visit the FARA (Friedreich's Ataxia Research Alliance) website,, for further details on this rare disease.

About Jupiter Orphan Therapeutics

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, USA, 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

Media contact:

Christer Rosén, +1 561 308-7780 
Alexander Rosén, +1 561 427-4447

SOURCE Jupiter Orphan Therapeutics, Inc.

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