FDA Grants Orphan Drug Status to Immuneering's IMM-1-104 for Pancreatic Cancer

Immuneering Corporation, a clinical-stage oncology company listed on Nasdaq (IMRX), recently revealed that the U.S. Food and Drug Administration (FDA) has granted orphan drug status to its lead product candidate, IMM-1-104, for the treatment of pancreatic cancer. This significant recognition underscores the potential of IMM-1-104 in addressing the critical need for novel treatments in this challenging cancer type.

IMM-1-104 is currently under investigation in a Phase 2a clinical trial for patients with advanced solid tumors, including pancreatic cancer. The trial recently yielded promising initial data, particularly for first-line pancreatic cancer patients who were treated with a combination of IMM-1-104 and modified gemcitabine/nab-paclitaxel. This positive response is a crucial step forward in the development of IMM-1-104 as a potential new therapy for pancreatic cancer.

Dr. Ben Zeskind, Co-Founder and CEO of Immuneering, emphasized the significance of the orphan drug designation. He mentioned that this status highlights the critical need for new therapeutic options that can improve outcomes for pancreatic cancer patients. Dr. Zeskind also pointed out that the positive initial data from the trial involving IMM-1-104 in combination with modified gemcitabine/nab-paclitaxel demonstrates the potential of IMM-1-104 to enhance the current standard of care. Furthermore, the ongoing study is also evaluating IMM-1-104 in combination with modified FOLFIRINOX and as a monotherapy for pancreatic cancer. Initial data from at least one additional arm of the Phase 2a portion of the trial is anticipated by the end of the year.

Orphan drug designation by the FDA is granted to investigational therapies aimed at treating rare medical conditions affecting fewer than 200,000 people in the United States. This designation can provide sponsors with various incentives, such as tax credits for qualified clinical trials, exemptions from certain FDA fees, and extended marketing exclusivity post-approval. Earlier this year, Immuneering also received FDA Fast Track designation for IMM-1-104 for both first and second-line pancreatic cancer treatment.

The mechanism behind IMM-1-104 involves achieving universal-RAS activity that selectively targets cancer cells more than healthy cells. This is done through a process called Deep Cyclic Inhibition of the MAPK pathway, administered once daily. IMM-1-104 is being tested in a Phase 1/2a study involving patients with advanced solid tumors that have RAS mutations.

Immuneering Corporation is dedicated to developing and commercializing universal-RAS/RAF medicines for a wide range of cancer patients. Its primary focus is on creating a universal-RAS therapy that works through Deep Cyclic Inhibition of the MAPK pathway, which aims to impact cancer cells while sparing healthy cells. The company’s leading product candidate, IMM-1-104, is an oral, once-daily Deep Cyclic Inhibitor currently in Phase 2a trials for patients with advanced solid tumors, including those with RAS mutations. Another product, IMM-6-415, is an oral, twice-daily Deep Cyclic Inhibitor under Phase 1/2a trials for patients with advanced solid tumors harboring RAS or RAF mutations. Immuneering’s pipeline also includes several early-stage programs aimed at broadening their therapeutic reach.