FDA Clearance of ACT-IOP-003 Launches Phase 1b Study in Pancreatic Cancer

• ACT-IOP-003 is an implantable iontophoretic product designed to achieve high concentration of gemcitabine in the pancreas.

• The agent has previously demonstrated promising results in an orthotopic patient-derived xenograft murine model of human pancreatic cancer.

• Now, a phase 1b trial will assess the safety and tolerability of targeted delivery of gemcitabine to locally advanced unresectable (LANR) pancreatic tumors.

The FDA has cleared an investigational new drug (IND) application for ACT-IOP-003, an implantable iontophoretic product, as a potential treatment for patients with pancreatic cancer, and a phase 1b trial of the agent plans to start mid-2024.¹

ACT-IOP-003 is designed to achieve high concentration of gemcitabine in the pancreas in a safe manner. This is done through non-circulatory pathways and causes little to no systemic drug exposure or associated toxicity.²

Previously, ACT-IOP-003 has led to encouraging findings in an orthotopic patient-derived xenograft murine model of human pancreatic cancer.¹ In the model, all of the treated tumors averaged 40% volume reduction.

cancer of pancreas : © Dr_Microbe - stock.adobe.com

“Pancreatic cancer is a devastating disease with a very poor prognosis,” said William Daunch, PhD, chief technology officer of Focal, in a press release. “Once a locally advanced tumor is not resectable, treatment options are limited and these patients experience a significantly reduced survival outlook compared to resectable cases. Our thesis, which we have demonstrated in animal models of pancreatic cancer, is that localized delivery of high concentrations of gemcitabine via our implantable iontophoretic device can reduce tumor volume to a point where surgical removal may be possible, while also minimizing systemic exposure and associated toxicity. If successful, we may offer the opportunity of extended survival for the significant number of pancreatic cancer patients presenting with [unresectable] disease.”

The multi-center, open-label, modified dose-escalation, phase 1b clinical trial plans to evaluate the safety, tolerability, and clinical activity of ACT-IOP-003, a targeted therapeutic product which will deliver gemcitabine directly into the pancreas to treat patients with LANR pancreatic cancer.

Enrollment in the study will be open to up to 12 patients who will then be enrolled into 1 of 2 cohorts. In the first cohort, patients will receive treatment once a week over 8 weeks, and patients in the second cohort will receive treatment twice a week over the same duration of time.

“Clinicians who treat pancreatic cancer need new and more powerful tools in their armamentarium to address this terrible disease,” said Jen Jen Yeh, MD, Focal Medical co-founder and non-executive director, and professor and vice chair of research, department of surgery and director of the Pancreatic Cancer Center of Excellence at the Lineberger Comprehensive Cancer Center, University of North Carolina at Chapel Hill, in a press release. “The novel approach that the Focal Medical system employs to drive gemcitabine directly and selectively into the pancreas may offer new hope for patients.”

REFERENCES:

1. Focal Medical receives FDA clearance to initiate phase 1b clinical trial of ACT-IOP-003, a targeted therapeutic product in development for pancreatic cancer. News release. Focal Medical, Inc. January 9, 2024. Accessed January 9, 2024. http://tinyurl.com/52ndvtwu

2. Applications. Focal Medical Inc. Accessed January 9, 2024. https://focalmedical.co/applications/