Mount Freedom, NJ – November 20, 2024 – SDK Therapeutics, Inc. (“SDK Therapeutics”), announces the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development has granted SDK001 (oral solution of Arsenic Trioxide) Orphan Drug Designation (ODD) for the treatment of Acute Promyelocytic Leukemia (APL). SDK001 is being actively developed for the treatment of APL.
“FDA’s granting Orphan Drug Designation signals the benefit that SDK001 could bring to patients with Acute Promyelocytic Leukemia,” said Stephane Berthier, Chief Executive Officer of SDK Therapeutics. “SDK001 has the potential to become the new standard of care in Acute Promyelocytic Leukemia by addressing the significant treatment burden associated with the current intravenous formulation”.
Intravenous arsenic trioxide is part of the routine standard of care for treatment of newly diagnosed APL patients in the United States and other major territories. In Hong Kong, where SDK001 is currently approved, and available physicians have an oral option to treat their patients without burdensome infusion therapy. Clinical studies spearheaded by Dr. Harinder Gill at the University of Hong Kong, have demonstrated that complete remission rates of 100% and overall and relapse-free survival rates of 97% over 3 years of follow-up are obtained with SDK001 frontline treatment consistent with current intravenous formulation. 1,2 Additionally, there are many benefits associated with the oral formulation versus an infusion administration.
Reducing the burden on patients and their families derived from repetitive infusions and monitoring should provide a greater quality of life. An oral formulation has the clear potential to mitigate some toxicities that can be associated with an intravenous drug. SDK001 represents a unique opportunity for patients and health care providers to provide comparable efficacy based on extensive clinical experience in over 400 patients treated while the potential to offer a differentiated safety profile and a more favorable clinical experience.
“Receiving Orphan Drug Designation moves us one step closer to our goal of transforming the lives of people diagnosed with Acute Promyelocytic Leukemia and their caregivers,” said Danelle James, M.D., Chief Medical Officer at SDK Therapeutics. “We are committed to advancing the oral formulation, SDK001 as it represents a therapy with the potential to bring considerable improvement to the standard of care. This designation, along with our planned IND filing in December 2024, brings us closer to offering a new treatment option to patients with Acute Promyelocytic Leukemia.”
The FDA’s Orphan Drug Designation is intended to encourage the development of treatments for rare diseases, defined as those affecting fewer than 200,000 people in the United States.
Orphan Drug Designation may shorten the clinical development path through closer FDA collaboration and potential qualification for expedited development programs. This designation also provides the company with certain benefits, including tax credits for qualified clinical trials, exemption from user fees, and potential seven-year market exclusivity upon FDA approval. SDK is pursuing orphan drug designation in other territories.
About Acute Promyelocytic Leukemia
Acute promyelocytic leukemia (APL) accounts for approximately 5-10% of patients with acute myeloid leukemia (AML) and is characterized by the balanced translocation t(15;17) (q22;q12), resulting in the formation of PML-RARA fusion gene. The combination therapy of all-trans retinoic acid (ATRA) and arsenic trioxide (ATO) has shown to be highly effective, achieving remission rates greater than 80% in patients with APL. This combination therapy is the standard treatment for adult patients with de novo, non-high-risk APL.
A Potential New Standard-of-Care Therapy with the Product
Arsenic Trioxide is currently approved in North America and Europe only as an intravenous formulation, which imposes a significant burden on patients, requiring up to 140 two- to four-hour infusions over the course of induction and consolidation treatment.
SDK001 is an oral liquid formulation of arsenic trioxide that has already been approved in Hong Kong for the treatment of patients with APL. SDK001 has been extensively studied in over 400 APL patients, both newly diagnosed and relapsed, 1-3 with long-term data demonstrating its efficacy and safety. The oral solution is administered once daily and has the potential to become the new standard of care for newly diagnosed APL patients. It offers comparable efficacy to the intravenous formulation, with the added benefits of a more convenient route of administration, reduced treatment burden on patients, improved accessibility, and lower costs to the healthcare system.
1 Gill, H. et al. Oral arsenic trioxide incorporation into frontline treatment with all-trans retinoic acid and chemotherapy in newly diagnosed acute promyelocytic leukemia: A 5-year prospective study. Cancer 125, 3001-3012, doi:10.1002/cncr.32180 (2019).
2 Gill, H. et al. An Entirely Oral Regimen of Oral-Arsenic Trioxide/All-Trans Retinoic Acid/Ascorbic Acid in Newly Diagnosed Acute Promyelocytic Leukaemia (APL): Updated Results of an Ongoing Multicentre Trial. Blood 142, 157 (2023).
3 Gill, H. et al. Long-term outcome of relapsed acute promyelocytic leukemia treated with oral arsenic trioxide-based reinduction and maintenance regimens: A 15-year prospective study. Cancer 124, 2316-2326, doi:10.1002/cncr.31327 (2018).
About SDK Therapeutics LLC
SDK Therapeutics LLC is a New Jersey based biotechnology company founded in 2024 with a mission to develop and commercialize medicines in hematology, oncology, and rare diseases to improve the life of patients.
Contact: Stephane Berthier
Chief Executive Officer
+1-862-812-6042
[email protected]