FDA grants orphan drug designation to multitargeted T-cell therapy for pancreatic cancer
The FDA granted orphan drug designation to MT-601, an investigational T-cell immunotherapy, for treatment of advanced or metastatic pancreatic adenocarcinoma.
MT-601 (Marker Therapeutics) is an autologous, nongene-edited T-cell therapy composed of cytotoxic T lymphocyte lines that target PRAME, SSX2, MAGEA4, NY-ESO-1 and Survivin proteins on the surface of cancer cells.
“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%,” Peter L. Hoang, president and CEO of Marker Therapeutics, said in a company-issued press release. “MT-601 is Marker’s second novel [multitumor-associated antigen]-specific T-cell product to receive orphan drug designation and the first in a solid tumor indication, underscoring the potential of [our] approach in both solid tumors and blood cancers.”
The FDA based the orphan drug designation on interim results of the single-center phase 1 TACTOPS trial, which evaluated MT-601 in combination with standard chemotherapy as first-line therapy for patients with advanced or metastatic pancreatic adenocarcinoma.
Eight (61.5%) of 13 patients who received the regimen achieved improved disease control compared with historical controls, according to data presented by researchers from Baylor College of Medicine at the virtual ASCO Annual Meeting in 2020.
Three of eight patients who demonstrated disease control achieved partial response to therapy, with one patient achieving a radiographic complete response.
Researchers observed no cases of systemic toxicity or neurotoxicity.
Marker plans a multicenter phase 1 dose-escalation study to assess MT-601 in combination with chemotherapy as first-line therapy for patients with locally advanced unresectable or metastatic pancreatic adenocarcinoma. The company also plans to file an investigational new drug application with the FDA this year for MT-601 for its use in pancreatic cancer treatment.
The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.
Reference:
Smaglo BG, et al. Abstract 4622. Presented at: ASCO20 Virtual Scientific Program; May 29-31, 2020.
Read more about