Panavance Therapeutics has reported encouraging interim results from its Phase 1 dose escalation trial of misetionamide (GP-2250) in combination with gemcitabine for patients with advanced or metastatic pancreatic cancer. The data, presented at the American Society of Clinical Oncology (ASCO) 2025 Annual Meeting, demonstrate promising efficacy signals and favorable safety profile for the novel therapeutic combination.
Trial Design and Patient Population
The multicenter Phase 1 dose escalation trial enrolled 55 patients with advanced/metastatic pancreatic cancer across seven US clinical sites. Misetionamide was administered as second-line therapy in combination with gemcitabine, targeting patients who had previously received treatment for their disease.
Mechanism of Action
Misetionamide represents a novel approach to cancer treatment through its unique mechanism of action. The small molecule inhibits three major transcription factors: c-MYC and NFκB, both oncogenic transcription factors, as well as HIF1α, an antiangiogenic transcription factor. Through c-MYC inhibition, misetionamide selectively disrupts the energy metabolism of cancer cells, leading to cancer cell death. The NFκB inhibition component prevents cancer cells' ability to proliferate and survive, creating a dual-target mechanism that makes misetionamide an attractive tumor cell selective agent.
Efficacy and Safety Results
The interim analysis revealed that approximately 42% of patients experienced either a partial response or stable disease when treated with the misetionamide-gemcitabine combination. According to Dr. Anup Kasi, Associate Professor of Medical Oncology at the University of Kansas Cancer Center and lead investigator of the trial, "The extent of tumor regression observed with this combination appears to compare favorably with historical outcomes from gemcitabine alone."
The safety profile proved equally encouraging, with the combination demonstrating excellent tolerability. No new or exacerbated toxicities were observed compared to those expected with gemcitabine monotherapy, suggesting that misetionamide does not add significant adverse effects to the established chemotherapy regimen.
Clinical Significance
Dr. Kasi emphasized the potential clinical impact of these findings: "These interim results—highlighting both early efficacy and a favorable safety and tolerability profile—suggest the combination of misetionamide plus gemcitabine may offer a meaningful new treatment option for patients with pancreatic cancer."
The results gain additional significance given the historically challenging nature of pancreatic cancer treatment. Greg Bosch, Chairman and CEO of Panavance, noted that "These advances bring us another step closer to providing transformative therapies for patients with pancreatic cancer, which has historically been difficult to treat."
Preclinical Foundation
The clinical results are supported by extensive preclinical data demonstrating misetionamide's broad spectrum of activity across 13 xenograft models. This preclinical foundation, combined with the novel mechanism of action, provides rationale for the observed clinical activity and supports further development of the compound.
Future Development Plans
Based on the encouraging Phase 1 results, Panavance plans to expand misetionamide's clinical development program. The company intends to initiate a clinical trial for platinum-resistant ovarian cancer treatment and another trial as first-line maintenance therapy for non-BRCA mutated pancreatic cancer patients—a population currently lacking FDA-approved therapeutic options.
The broad antineoplastic mechanism of action demonstrated in preclinical studies suggests potential efficacy in additional tumor types, including melanoma, squamous cell carcinoma, breast cancer, and colorectal cancer, potentially expanding the therapeutic's clinical utility beyond pancreatic cancer.
Company Background
Panavance Therapeutics, founded in 2021 as a carve-out from the Swiss Geistlich group, focuses specifically on developing misetionamide for oncology applications. The clinical-stage pharmaceutical company, based in Berwyn, Pennsylvania, was established to advance this novel therapeutic approach for improving outcomes and quality of life for cancer patients.
