Biotech Bulletin: FDA Approval Delivers Vital Win for Pancreatic Cancer

February 17, 2024 - by Catie Corcoran

The FDA has approved the first new frontline treatment for metastatic pancreatic cancer in more than a decade. This week, global biopharmaceutical company, Ipsen, announced that it had received regulatory approval for Onivyde ^®plus oxaliplatin, fluorouracil and leucovorin (known as NALIRIFOX), for the treatment of metastatic pancreatic adenocarcinoma (mPDAC).

The company’s randomized, open-label, pivotal Phase III clinical trial enrolled 770 mPDAC patients, and demonstrated that the treatment boosted overall survival by 17%, and improved progression-free survival by 31%.

The pancreas, tucked away between the stomach and the spine, plays an essential role in converting food into fuel for the body’s cells. It has a dual function, producing enzymes that help to digest food, while also making insulin to control blood sugar levels. The organ’s hidden anatomical position means that early malignancies can’t be seen or felt by health care providers during routine physical exams, leading to poor survival outcomes in pancreatic cancer patients.

Pancreatic cancer is a highly aggressive, fatal disease that will be responsible for the deaths of nearly 52,000 Americans in 2024. According to the American Cancer Society, a further 66,000 Americans will receive a pancreatic cancer diagnosis this year.

Pancreatic ductal adenocarcinoma (PDAC) accounts for more than 90% of pancreatic cancers, and despite it being the seventh leading cause of global cancer death, few advances have been made to improve patient prognosis over the last two decades.

Numerous companies are working on promising treatments for pancreatic cancer, offering hope to patients living with one of the most deadly cancers. We have featured five companies that are waiting on near-term catalysts:

Pyxis Oncology (Nasdaq: PYXS)

Pyxis Oncology is developing therapeutics to target difficult-to-treat cancers, including pancreatic cancer. In 2023, the FDA granted orphan drug designation to Pyxis’ PYX-201 for the treatment of patients with pancreatic cancer. The company has initiated a phase 1 clinical trial evaluating PYX-201 in solid tumors, with preliminary data expected in the first half of this year. Pyxis’ market cap stands at $241.56 million.

AIM Immunotech (NYSE American: AIM)

This month, AIM ImmunoTech dosed the first patient in its Phase 1b/2 clinical trial combining its lead candidate, Ampligen® (rintatolimod), with AstraZeneca’s Imfinzi® (durvalumab) for the treatment of late-stage pancreatic cancer. The company expects to enroll up to 18 subjects in its Phase 1b portion, which will measure safety, and up to 25 subjects in its Phase 2 portion, which will determine the treatment’s clinical benefit rate. AIM says it expects to complete the 1b portion of the study within six months. AIM’s market cap is currently $18.51 million.

Oncolytics Biotech Inc. (Nasdaq: ONCY)

Oncolytics, an immunotherapy company, is developing pelareorep as a potential therapy for PDAC. The company has studied pelareorep in more than 1,100 patients, and it has been shown to be well-tolerated with mild side effects. Oncolytics expects to initiate the first Phase 3 study for pelareorep in pancreatic cancer this year. In September 2023, the Pancreatic Cancer Action Network awarded a $5 million grant to Oncolytics for the development of pelareorep. Oncolytics’ market cap is currently $85.05 million.

Purple Biotech (Nasdaq: PPBT)

Purple Biotech is conducting a Phase 2 trial in collaboration with Bristol Myers Squibb ($BMS) to evaluate CM24, a multi-functional immune checkpoint inhibitor, in combination with BMS’s nivolumab, plus chemotherapy, in PDAC patients. The company has enrolled approximately 60 patients in 18 centers in the U.S., Spain and Israel. An interim analysis of overall survival, the primary endpoint of the study is expected during 1H 2024 and topline analysis is expected in 2H 2024. Purple Biotech has a market cap of $19.99 million.

BioXcel Therapeutics (Nasdaq: BTAI)

Earlier this month, biopharmaceutical company, BioXcel, announced it had completed patient enrollment in the safety portion of its Phase 2 trial of BXCL701 in combination with KEYTRUDA® (pembrolizumab) in previously treated metastatic PDAC. Following an initial six-week safety study of six patients, BioXcel’s trial is expected to enroll approximately 39 patients in its efficacy phase. The primary objective is to determine the 18-week progression-free survival rate and initial study data is expected in 2024. BioXcel’s market cap stands at $106.64 million.