FDA Grants Orphan Drug Designation to MT-601 for Pancreatic Cancer Treatment


With an orphan drug designation in tow, MT-601 will soon be investigated in combination with chemotherapy in phase 1 clinical trial.

The FDA has granted orphan drug designation to MT-601 for the treatment of patients with pancreatic cancer, according to a press release by Marker Therapeutics, Inc.1

MT-601 is a multi-tumor-associated antigen (MultiTAA)-specific T cell product optimized to treat pancreatic cancer. The PRAME, NY-ESO-1, Survivin, MAGE-A4, SSX2, WT1, are tumor-associated antigens that are commonly expressed in pancreatic tumors.

“The FDA’s orphan drug designation underscores MT-601’s potential as a treatment for pancreatic cancer, a cancer typically diagnosed at an incurable advanced stage with a total overall 5-year survival rate of 10%,” said Peter L. Hoang, president, and chief executive officer of Marker Therapeutics, in a press release. “Our novel therapy has shown encouraging results in an ongoing phase 1 trial sponsored by Marker’s partners at the Baylor College of Medicine.”

In the TACOPS study, MT-601 was administered to 13 patients with multiTAA T-cells. Eight of the patients had disease control for longer than historic controls, according to data presented during the 2020 American Society of Clinical Oncology Virtual Annual Meeting. Further, there were 3 partial responses to therapy and 1 complete radiographic complete response per RECIST v1.1. MT-601 treatment did not result in any infusion-related systemic events or neurotoxicity, and it was determined that the agent is safe and achieved durable clinical benefit to patients with pancreatic cancer.2

“Our therapy has demonstrated the potential to safely produce durable responses in combination with chemotherapy as a first-line treatment option for patients with advanced or metastatic pancreatic adenocarcinoma. The results also revealed that epitope spreading was consistent in responders to Multi-TAA-specific T cells. Following MT-401 for the treatment of post-transplant acute myeloid leukemia, MT-601 is Marker’s second novel MultiTAA-specific T cell product to receive orphan drug designation and the first in a solid tumor indication, underscoring the potential of Marker’s multi-antigen targeting T cell therapy approach in both solid tumors and blood cancers,” said Hoang, in the press release.1

Based on its orphan drug designation and recent success in the clinical trial setting, MT-601 will soon be evaluated in a phase 1 clinical trial. In the study, MT-601 will be combined with chemotherapy and administered to patients with locally advanced unresectable or metastatic pancreatic cancer in the frontline setting. The study’s design will include an antigen escalation period and a dose-escalation period with approximately 20 to 25 patients included.

The developer of MT-601 also plans to submit an investigational new drug application to the FDA for MT-601 for the treatment of pancreatic cancer in 2022.


1. Marker Therapeutics receives FDA orphan drug designation for its multi-antigen targeted t cell therapy for pancreatic cancer. News release. January 19, 2022. Accessed January 21, 2022. https://bit.ly/33IVUXQ

2. Smaglo BG, Musher BL, Vasileiou S, et al. A phase I trial targeting advanced or metastatic pancreatic cancer using a combination of standard chemotherapy and adoptively transferred nonengineered, multiantigen specific T cells in the first-line setting (TACTOPS). J Clin Oncol. 2020; 38 (5): 4622-4622. doi:0.1200/JCO.2020.38.15_suppl.4622

Related Videos
Rohit Gosain, MD; Rahul Gosain, MD; and Pamela L. Kunz, MD, presenting slides
Rohit Gosain, MD; Rahul Gosain, MD; and Pamela L. Kunz, MD, presenting slides
Rohit Gosain, MD; Rahul Gosain, MD; and Pamela L. Kunz, MD, presenting slides
Rohit Gosain, MD; Rahul Gosain, MD; and Pamela L. Kunz, MD, presenting slides
Rohit Gosain, MD; Rahul Gosain, MD; and Pamela L. Kunz, MD, presenting slides
Related Content