20 March 2018
ORSENIX, LLC ANNOUNCES SUCCESSFUL END OF PHASE MEETING AND PLANNED REGISTRATION STUDY FOR ORH-2014, A NOVEL ORAL FORMULATION OF ARSENIC TRIOXIDE, OFFERING A POTENTIAL NEW STANDARD OF CARE FOR FIRST LINE TREATMENT OF ACUTE PROMYELOCYTIC LEUKEMIA
Atlanta, Georgia, March 20, 2018 – Orsenix, LLC, a company that is developing a novel oral form of arsenic trioxide (ATO), ORH-2014, for the treatment of acute promyelocytic leukemia (APL), today announced the successful completion of its End of Phase (EOP) meeting with the U.S. Food and Drug Administration (FDA). The Company has reached general agreement with the FDA on the key elements of the Phase III program to support a New Drug Application (NDA) for ORH-2014. Orsenix held the EOP meeting with the FDA to discuss the registration program for Orsenix’s novel oral arsenic trioxide formulation (ORH-2014) for the treatment of low- to medium-risk patients with untreated acute promyelocytic leukemia (APL). Orsenix and the FDA reached agreement on a number of parameters including registration study design, endpoints, suitability of 505(b)(2) pathway for approval, and chemistry and manufacturing control (CMC) requirements for the drug product.
Importantly, the FDA has accepted Orsenix’s proposal to utilize molecular complete remission (CR) (molecular and hematological remission) at the end of the 3rd consolidation cycle (7 months after randomization) as an approval endpoint. As suggested by the FDA, Orsenix is planning to conduct a randomized study to compare the safety of ORH-2014 to IV arsenic trioxide while efficacy evaluation is based solely on the ORH-2014 study arm. The Phase III study is likely to require less than 100 patients.
The FDA previously granted orphan drug designation to Orsenix for ORH-2014 for the treatment of APL on November 2, 2015.
“We are very pleased with the productive guidance we have received from the FDA during the recent End of Phase meeting. Orsenix expects to initiate the Phase III program of ORH-2014 for the treatment of APL, in combination with ATRA, in 4Q2018 and will continue working closely with the agency to develop ORH-2014. An all oral treatment option may become reality for patients with APL, which will significantly improve the standard of care of these patients. Assuming that the registration study is initiated in 2018, an NDA submission may occur as early as 2021.”
Kris Vaddi, Ph.D.Co-Founder and a director of Orsenix
Orsenix plans to pursue regulatory approval of ORH-2014 for the treatment of APL in the U.S. through the 505(b)(2) registration pathway.
ORH-2014 is a new oral capsule formulation of arsenic trioxide. The standard of care treatment of APL is a combination of ATRA/IV Arsenic Trioxide. Patients face daily IV treatments for up to 60 days of induction and additionally up to 4 cycles of consolidation via treatment 5 days/week for 4 weeks. The IV regimen negatively impacts the quality of life of patients with APL and places a financial burden on both the patient and the healthcare system overall. ORH-2014, an oral therapy, offers the potential to revolutionize the treatment of APL, vastly improve the quality of life of APL patients, and offer a lower cost of delivery. ORH-2014 at a dose of 10 mg daily provides equivalent drug exposure to that of the approved dose of intravenous (IV) ATO, the current standard of care for both relapsed and first line APL. Once approved, oral ATO is expected to replace IV ATO as the standard of care in both.
APL is a subtype of acute myeloid leukemia (AML) and is characterized by a chromosomal translocation involving the retinoic acid receptor alpha (RARα or RARA) gene also known as PML-RARα. APL is currently treated with Trisenox® (an IV formulation of ATO) along with oral ATRA.
Partner, Torreya Partners (Europe) LLP, London
Phone: +44 20 7451 4550
Managing Director, Torreya Partners (Europe) LLP, London
Phone: +44 20 7451 4550