MyeloRx LLC
The Triptolide Company ™
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Therapeutic Areas


Triptolide-based drugs have shown clinical and/or pre-clinical activity in a variety of cancer types including leukemias and some solid tumors.  The lead compound, MRx102, is being developed initially for a number of leukemias including acute myeloid leukemia (AML), chronic myeloid leukemia, multiple myeloma and myelodysplastic syndrome, with AML being the first indication.

AML is a disease that affects tens of thousands of people.  In 2010 over 12,300 patients were diagnosed with AML in the U.S. and nearly 9,000 died of this disease.  There is an unmet medical need in AML particularly in elderly patients.  The two-year survival in older patients with this disease is less than 20%.   Because AML affects a relatively small number of patients this indication is eligible for both a fast track approval as well as orphan drug status. These two designations combine to provide both a more rapid approval process and extended market exclusivity.  The strategy of using both the fast track and orphan drug designation have been of great benefit to Celgene for Revlimid ® (Lenalidomide, a thalidomide derivative for multiple myeloma) and Biomarin for Naglazyme® (galsulfase for MPS VI) among others.

Triptolide as well as our lead compound, MRx102, have also demonstrated significant activity in in vitro and in vivo preclinical melanoma models.  MRx102 has shown activity in vitro and in an in vivo nude mouse human tumor xenograft model performed by the NCI Developmental Therapeutics Program.  Based on the mechanism of action and some preclinical work with triptolide, we believe our prodrugs and analogs will be active in other solid tumors, such as pancreatic cancer.

Immune-Based Diseases

Solid organ transplantation is a well accepted procedure but could be better served with more effective and safer therapeutics.  For example, graft survival rates decline sharply over time. Indeed, the leading cause of long-term organ failure is fibrosis.  It is significant that the leading immunosuppressants calcineurin inhibitors (Neoral®, Prograf®) and the anti-proliferatives (Cellcept®, Rapamune(®) are pro-fibrogenic.  

Our first immune-based disease target is rheumatoid arthritis (RA).  Extracts of Tripterygium wilfordi Hook f, the herb from which triptolide is derived, is a major product for RA in China. In addition, an extract of Tripterygium wilfordi Hook f has been shown clinically efficacious in two Phase 2 double-blinded RA clinical studies performed in the U.S.  Our lead compound, MRx109 is a potent immunosuppressant with anti-fibrogenic properties and has demonstrated activity in a rodent RA model system.

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