Elraglusib Receives FDA Orphan Drug Designation in Pancreatic Cancer

The FDA has granted orphan drug designation to the investigational GSK-3β inhibitor elraglusib (9-ING-41) as a treatment for patients with pancreatic cancer, according to a press release from Actuate Therapeutics, Inc.

Investigators are currently assessing elraglusib in combination with gemcitabine and nab-paclitaxel (Abraxane) in patients with advanced pancreatic cancer as part of the phase 1/2 1801 trial (NCT03678883). Developers designed elraglusib as a therapy for adult and pediatric patients with refractory cancers.

“We are pleased with the FDA’s decision to grant orphan drug designation to elraglusib for the treatment of pancreatic cancer,” Daniel Schmitt, president and chief executive officer at Actuate, said in the press release. “Pancreatic cancer remains an area of high unmet medical need, with less than 10% survival in the U.S. at 5 years. The orphan drug designation furthers our ability to advance development and regulatory interactions with the FDA to bring elraglusib to patients with this highly lethal malignancy.”

Investigators of the phase 1/2 1801 study are assessing the safety and efficacy of elraglusib as a treatment for patients with advanced pancreatic cancer and those with other refractory cancers including sarcoma, non-Hodgkin lymphoma, glioma, renal cancer, and breast neoplasms. In the pancreatic cancer cohort, patients will receive elraglusib on day 1 and 4 each week as part of a 21-day cycle intravenously over 60 minutes plus 125 mg/m² of nab-paclitaxel intravenously and 1000 mg/m² of gemcitabine intravenously on days 1, 8, and 15 of a 28-day cycle. Across other treatment arms, investigators are administering elraglusib in combination with doxorubicin, lomustine, carboplatin, or irinotecan (Campto).

The primary end points include treatment-related adverse effects in parts 1 and 2 of the trial, as well as 1-year survival rate in part 3.

Patients 18 years and older with pathologically confirmed advanced or metastatic malignancy that is refractory to existing therapies or relapsed following standard therapies are eligible for enrollment on the trial. Additional eligibility criteria include having at least 1 measurable lesion per RECIST v1.1 criteria; adequate bone marrow, liver, and renal function; an ECOG performance status of 0 to 2; and no prior receipt of any other investigational agent. Patients may also receive endocrine therapy for breast or prostate cancer, or agents targeting HER2 while on the study.

Patients with a hypersensitivity to any components of elraglusib and those who have not recovered from clinically significant toxicities following prior anti-cancer treatment are not able to enroll on the trial. Patients are also ineligible for enrollment if they have significant cardiovascular impairment, a myocardial infarction within 12 weeks of beginning study treatment, symptomatic progressive brain metastases or leptomeningeal involvement, undergo major surgery within 7 days of study entry, or have a current active malignancy other than the target cancer. Having endocrine or acinar pancreatic carcinoma is also grounds for exclusion from enrollment on the trial.

According to Andrew Mazar, PhD, chief operating officer at Actuate, elraglusib is under investigation in additional phase 2 trials as a treatment for patients with pancreatic cancer. Investigators are administering elraglusib in combination with a checkpoint inhibitor for those with advanced pancreatic cancer in one phase 2 study (NCT05239182) and with folinic acid, fluorouracil, irinotecan, oxaliplatin (FOLFIRINOX) for patients with metastatic pancreatic cancer in another (NCT05077800).

Reference

Actuate Therapeutics receives FDA orphan drug designation for elraglusib for treatment of pancreatic cancer. News release. Actuate Therapeutics, Inc. August 1, 2023. Accessed August 2, 2023. https://shorturl.at/fwUY7