Benzopyran-Based Small Molecule Granted FDA Orphan Drug Status in AML

The FDA has granted orphan drug designation (ODD) to TRX-E-002-1 (Cantrixil) for the treatment of acute myeloid leukemia (AML).¹

TRX-E-002-1 remains in preclinical development for AML, and the designation supports its plans to advance the candidate toward a phase 1 study in this setting. Specifically, ODD, granted to investigational agents intended for treatment, prevention, or diagnosis of rare diseases affecting fewer than 200,000 people in the United States, provides the sponsor with potential development incentives and the possibility of 7 years of market exclusivity if the drug candidate receives approval for the indication.

“The [ODD] is an important regulatory milestone for the development of [TRX-E-002-1],” Erik Kinnman, CEO of Vivesto, stated in a news release. “Together with the positive preclinical results generated to date, the designation strengthens the foundation for advancing [TRX-E-002-1] toward a [p]hase [1] study in AML.”

About TRX-E-002-1 and the Unmet Need in AML

TRX-E-002-1 is a selective small-molecule drug candidate consisting of a third-generation benzopyran molecule encapsulated in cyclodextrin.² The agent has shown activity against a broad range of cancer cells, including chemotherapy-resistant tumor-initiating cells that may contribute to disease recurrence.

AML is an aggressive hematologic malignancy associated with substantial morbidity and mortality. In the United States, approximately 22,720 new cases and 11,500 deaths are projected in 2026, with a 5-year relative survival rate of 33.4%.¹ The disease's clinical course and limited long-term survival underscore the need for additional therapeutic strategies, particularly for patients who do not achieve durable disease control with available treatments. For investigational agents such as TRX-E-002-1, evaluation in AML could provide insight into whether targeting tumor cell populations associated with treatment resistance may offer another approach to disease management.

Development Pipelines

The candidate's development rationale is based on its potential to address tumor cell populations that may persist after conventional treatment. In preclinical studies, TRX-E-002-1 demonstrated cytotoxic activity at low doses, according to the sponsor; activity was also reported when the candidate was combined with other anticancer agents in several hematologic cancer cell lines. In animal models, treatment was associated with reduced tumor growth and improved survival, with reported tolerability.

TRX-E-002-1 was previously evaluated in a phase 1 clinical study involving patients with persistent or recurrent ovarian cancer, fallopian tube cancer, or primary peritoneal cancer (NCT02903771). The study investigated intraperitoneal administration of the candidate, including its safety, tolerability, pharmacokinetics, and antitumor activity.³ The sponsor has since focused the candidate's development on hematologic cancers, based on preclinical findings from blood cancer models.

REFERENCES

1. Vivesto receives Orphan Drug Designation in the U.S. for Cantrixil in AML. News release. Vivesto. November 3, 2026. Accessed November 9, 2026. https://tinyurl.com/3fysaa7n

2. Cantrixil. Vivesto. Published June 5, 2025. Accessed November 9, 2026. https://tinyurl.com/yeed8te4

3. Phase I Study of Cantrixil in Patients With Ovarian Cancer, Fallopian Tube Cancer or Primary Peritoneal Cancer. ClinicalTrials.gov. Updated September 19, 2024. Accessed November 9, 2026. https://tinyurl.com/2j83d8hb