Oxcia AB announced today that its lead clinical candidate OXC-101 has received orphan drug designation (ODD) from the U.S. Food and Drug Administration for the treatment of acute myeloid leukemia (AML), marking a significant milestone in the drug's development journey.
OXC-101 represents a novel approach to cancer treatment as a first-in-class mitotic MTH1 inhibitor with a dual mechanism of action. The drug exploits cancer cells' vulnerability to high endogenous oxidative stress and DNA damage, showing significant survival benefits and tumor growth reduction in preclinical AML models.
Clinical Development Progress
The company is currently conducting a Phase 1/2 expansion trial investigating OXC-101 in combination with the standard treatment idarubicin for relapsed/recurrent AML patients. This follows encouraging safety data from Phase 1 studies in advanced hematological cancer patients. The ongoing trial aims to validate preliminary efficacy signals that could support advancement to a pivotal Phase 2 trial for potential fast-track approval.
"We are delighted with the FDA's decision to grant orphan drug designation to OXC-101 for AML," said Ulrika Warpman Berglund, Oxcia's CEO. "This is a significant milestone and underscores the significant unmet need for novel medicines and the unique approach by OXC-101. We believe OXC-101 holds potential to greatly improve treatment for patients who suffer from AML."
Trial Expansion and Precision Medicine Approach
To optimize patient recruitment for this rare disease indication, Oxcia is expanding its clinical trial network. Rigshospitalet in Denmark will join the study shortly, with additional sites in Bulgaria and Serbia planned for inclusion during 2025. The company received a 3 million SEK grant from Swelife and Medtech4Health in early 2023 to support this study.
Notably, the trial incorporates precision medicine screening to identify responders and non-responders, representing an innovative approach to treatment personalization that could help reduce unsuccessful treatments and unnecessary patient suffering.
AML Disease Burden and Treatment Landscape
AML remains the most prevalent acute leukemia in adults, representing over 80% of cases. Global incidence reached 162,200 cases in 2021, with projections indicating an increase to 169,000 cases by 2027. Despite some improvements in survival rates, prognosis remains poor, with only 20% of patients in selected subpopulations surviving five years post-diagnosis.
The disease's heterogeneous nature has maintained classical intensive chemotherapy, based on cytarabine and anthracycline combinations, as the standard of care. While recent approvals have targeted specific biomarker-defined AML subsets, there remains a substantial unmet need for effective, well-tolerated therapies for the majority of patients.
Regulatory Implications
The orphan drug designation brings several advantages, including potential marketing exclusivity following approval, reduced regulatory fees, and in the European context, access to a centralized approval process. Oxcia is currently preparing a similar application for orphan designation with the European Medicines Agency, emphasizing the importance of early engagement with regulatory authorities.
