Immuneering Corporation's IMM-1-104, a drug candidate for pancreatic cancer treatment, has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA). This regulatory milestone follows promising initial Phase 2a trial results, where IMM-1-104, in combination with chemotherapy, demonstrated complete and partial responses in first-line pancreatic cancer patients.
The orphan drug status, designed to encourage the development of treatments for rare diseases affecting fewer than 200,000 people in the United States, provides Immuneering with potential benefits, including tax credits, FDA fee exemptions, and extended marketing exclusivity post-approval. Earlier in the year, the FDA also granted Fast Track designation to IMM-1-104 for both first and second-line pancreatic cancer treatments.
Mechanism of Action and Clinical Trials
IMM-1-104 is designed to selectively target cancer cells by inhibiting the MAPK pathway, a signaling pathway frequently altered in cancers, including those with RAS mutations. The drug is administered orally once daily and is currently under evaluation in a Phase 1/2a study involving patients with advanced solid tumors.
According to Immuneering, initial data from at least one additional arm of the Phase 2a portion of their Phase 1/2a trial is expected to be released before the end of the year. The company is also exploring IMM-1-104 in combination with modified FOLFIRINOX and as a monotherapy for pancreatic cancer.
Executive Insights
Ben Zeskind, Ph.D., Co-Founder, and CEO of Immuneering, expressed optimism regarding the drug's potential to enhance the current standard of care for pancreatic cancer. "The FDA’s granting of orphan drug designation for IMM-1-104 underscores the urgent need for new therapies that meaningfully improve outcomes for pancreatic cancer patients and represents an important milestone in the development of our lead asset," said Zeskind.
About the Phase 1/2a Trial
The ongoing Phase 1/2a trial (NCT05585320) is an open-label study involving patients with advanced solid tumors, including those with pancreatic cancer. The Phase 2a portion of the study, announced in September 2024, showed that patients with pancreatic cancer (n = 5) treated in the frontline setting with IMM-1-104 in combination with modified gemcitabine and nab-paclitaxel experienced an overall response rate (ORR) of 40% and a disease control rate (DCR) of 80%.
Implications of Orphan Drug Designation
The FDA's orphan drug designation may qualify Immuneering for incentives such as tax credits for qualified clinical trials, exemptions from certain FDA fees, and additional time for post-approval marketing exclusivity.
