Immuneering Granted Orphan Drug Designation for Pancreatic Cancer Treatment

Immuneering Corporation recently announced the US FDA has granted orphan drug designation to IMM-1-104 in the treatment of pancreatic cancer. IMM-1-104 is currently being evaluated in a Phase 2a clinical trial in patients with advanced solid tumors, including pancreatic cancer, in which positive initial response data was recently reported for first line pancreatic cancer patients treated in combination with modified gemcitabine/nab-paclitaxel.

“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 Ben Zeskind, PhD, Co-Founder and CEO of Immuneering. “I believe our recently announced positive initial Phase 2a data, from our arm investigating IMM-1-104 in combination with modified gemcitabine/nab-paclitaxel in pancreatic cancer, speaks to IMM-1-104’s potential to improve upon the current standard of care in this indication. Importantly, in the same trial we are also studying IMM-1-104 in combination with modified FOLFIRINOX, as well as in monotherapy for pancreatic cancer. We look forward to providing initial data from at least one additional arm of the Phase 2a portion of our Phase 1/2a trial before the end of the year.”

FDA orphan drug designation is granted to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the United States. Orphan drug designation may qualify sponsors for incentives, including tax credits for qualified clinical trials, exemptions from certain FDA fees and additional time for post-approval marketing exclusivity. Earlier this year, Immuneering was granted FDA Fast Track designation for IMM-1-104 for the treatment of both first and second-line pancreatic cancer.

IMM-1-104 aims to achieve universal-RAS activity that selectively impacts cancer cells to a greater extent than healthy cells, through Deep Cyclic Inhibition of the MAPK pathway with once-daily dosing. IMM-1-104 is currently being evaluated in a Phase 1/2a study in patients with advanced solid tumors harboring RAS mutations (NCT05585320).

Immuneering is a clinical-stage oncology company seeking to develop and commercialize universal-RAS/RAF medicines for broad populations of cancer patients with an initial aim to develop a universal-RAS therapy. The Company aims to achieve universal activity through Deep Cyclic Inhibition of the MAPK pathway, impacting cancer cells while sparing healthy cells. Immuneering’s lead product candidate, IMM-1-104, is an oral, once-daily Deep Cyclic Inhibitor currently in a Phase 2a trial in patients with advanced solid tumors including those harboring RAS mutations. IMM-6-415 is an oral, twice-daily Deep Cyclic Inhibitor currently in a Phase 1/2a trial in patients with advanced solid tumors harboring RAS or RAF mutations. The company’s development pipeline also includes several early-stage programs. For more information, visit www.immuneering.com.