Qurient, a South Korean biopharmaceutical company, has announced that the U.S. Food and Drug Administration (FDA) has approved its Investigational New Drug (IND) application for Adrixetinib (Q702) to proceed with a Phase 1b clinical trial for chronic graft-versus-host disease (cGVHD). The trial, approved less than a month after submission, will evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of Adrixetinib monotherapy in patients with relapsed or refractory active cGVHD.
Trial Design and Objectives
The Phase 1b trial will enroll 18 patients across five hospitals in the United States. The primary objective is to determine the recommended Phase 2 dose (RP2D) of Adrixetinib. The study will also assess the drug's impact on patients with cGVHD who have not responded to previous treatments.
Adrixetinib's Mechanism of Action
Adrixetinib is a selective triple kinase inhibitor targeting Axl, Mer, and CSF1R. This dual-action mechanism is designed to enhance the body’s immune defenses and increase the susceptibility of cancer cells to treatment. Inhibition of CSF1R is relevant for cGVHD, while Axl/Mer inhibition targets leukemia, potentially offering simultaneous benefits in treating both conditions, according to Qurient.
cGVHD Treatment Landscape and Unmet Needs
cGVHD is a severe complication that occurs in approximately 50% of patients following allogeneic hematopoietic stem cell transplantation for blood cancers. In cGVHD, donor immune cells attack the patient’s tissues, leading to systemic symptoms and significant morbidity. Current treatments often lack sufficient long-term efficacy, underscoring the urgent need for new therapeutic options.
Qurient's Broader Clinical Development Program
In addition to the cGVHD trial, Qurient is conducting Phase 1 and 2 clinical trials of Adrixetinib for acute myeloid leukemia (AML) at MD Anderson Cancer Center in Texas. This strategic expansion into cGVHD aims to leverage Adrixetinib's potential in regulating myeloid immune cells, addressing not only cancer but also complications arising from blood cancer therapies.
Competitive Advantages
While Syndax Pharmaceuticals' Niktimvo (axatilimab-csfr), a CSF1R antibody treatment, has gained attention for cGVHD, Qurient emphasizes that Adrixetinib offers the advantage of oral administration compared to Niktimvo's intravenous delivery. Furthermore, Adrixetinib has demonstrated potential in treating blood cancers in solid tumor trials, attracting interest from key opinion leaders in cGVHD treatment.
Management Perspective
Nam Ki-yean, CEO of Qurient, stated that this IND approval is a significant step for Adrixetinib to become a key drug in regulating myeloid immune cells, with potential applications beyond cancer treatment. He anticipates substantial interest from global pharmaceutical companies as the development program advances towards approval for subsequent trials.
