University of Minnesota Developed Platform Moves to First-in-Human Clinical Trial for Leukemia

A designer molecule discovered at the University of Minnesota activates the patient’s own immune cells to attack cancer. Known as tri-specific natural killer engagers (TriKE™), they are a combination protein that bridges an immune cell and a tumor cell to drive tumor cell killing exponentially—now have entered Phase I, first-in-human study to treat leukemia. The study is opening exclusively at the University of Minnesota Medical Center and is sponsored by GT Biopharma, Inc.

The Clinical Trial Funding and Deal Summary

Funded by GT Biopharma and supported by the University of Minnesota designed and manufactured molecules called GTB-3550 to treat leukemia. Additional funding originated from Minnesota Masonic Charities. GT Biopharma secured the valuable intellectual property for $200,000 upfront payment and a series of maintenance, clinical milestone and sales milestone payments. 

AML: The Problem

An aggressive and dangerous acute leukemia with 21,000 new cases annually according to the American Cancer Society. The current standard of care is most commonly chemotherapy, however, about 50% of AML patients relapse or require additional therapies. There is a significant amount of unmet need with current therapies reports the University of Minnesota in the press release picked up by Globe Newswire.

The Study

The first-of-its-kind clinical trial is made from three separate components that bind, activate and target the patient’s own natural killer (NK) immune cells to specifically attack Acute Myeloid Leukemia (AML) tumors.

Lead Research/Investigator

The study will be led by Principal Investigator Erica Warlick, MD, Associate Professor, Division of Hematology, Oncology and Transplantation at the University of Minnesota. Dr. Warlick is also a researcher with the Masonic Cancer Center.

The TriKE™ Platform

This platform was created and brought into the clinic by Masonic Cancer Center Deputy Director Jeffrey Miller, MD, a Professor of Medicine at the University of Minnesota. Other colleagues involved include Martin Felices, Ph.D., Assistant Professor of Medicine, and Daniel Vallera, Ph.D., Professor of Therapeutic Radiology -Radiation Oncology at the University of Minnesota Medical School.

The group capitalized on their decades-long expertise in NK cell biology and protein fusion to bring GTB-3550 from the laboratory into the clinic in a cost-effective and off-the-shelf therapy for resistant or relapsing AML.

Dr. Miller noted, “GTB-3550 is a protein immune engager that binds NK cells and targets them specifically to leukemia cells.” He continued “Our team has been working on the structure for years, and we are excited to see it in clinical testing. Another important feature is that the same TriKE protein will stimulate IL-15, an NK cell growth factor, which significantly increases the number of immunotherapeutic cells targeting leukemia cells.”

GTB-3550 was produced and manufactured at the University of Minnesota’s Molecular and Cellular Therapeutics (MCT) Center, which offers full-service development and GMP manufacturing of cell- and tissue-based products, monoclonal antibodies and other therapeutic proteins, as well as active pharmaceutical ingredients for use in Phase I, II or III clinical trials. University of Minnesota Health (M Health), the clinical partner of the Masonic Cancer Center, supports the MCT in the production of these molecules.

The Commercial Sponsor: GT Biopharma Inc.

GT Biopharma is a clinical-stage biopharmaceutical venture focused on the development and commercialization of immune-oncology products based on their proprietary Tri-specific Killer Engager (TriKE™) and Multi-Target Directed Bispecific Drug Conjugate technology platforms. Their TriKE platform, developed at the University of Minnesota, is designed to harness and enhance the cancer-killing abilities of a patient’s immune system natural killer cells (NK cells). GT Biopharma secured an exclusive worldwide license with the University of Minnesota to further develop and commercialize cancer therapies using proprietary TriKE technology developed by researchers at the University. The Multi-Target Directed Bispecific Drug Conjugate platform can generate product candidates that are bi-specific, ligand-directed single-chain fusion proteins that, they believe, represent the next generation of targeted therapy.

The Deal Terms

GT Biopharma via its subsidiary Oxis Biotech included the following terms with the University of Minnesota:

Upfront Payments

  • $200,000 payable upfront–$75,000 within 15 calendar days after the Effective Date and the remaining $125,000 payable 6 months of the Effective Date.

Maintenance Payments

  • $25,000 payable on the first and second anniversary of the Effective Date.
  • $50,000 payable on the third and fourth anniversary of the Effective Date
  • $100,000 payable on the fifth anniversary of the Effective Date and on each anniversary of the Effective Date thereafter.

Clinical and Sales Milestone Payments

  • $100,000 upon dosing of the first human subject in Phase I clinical trial of the Licensed Product.
  • $250,000 upon dosing of a first human subject in Phase II clinical trial of a Licensed Product.
  • $500,000 upon dosing of a first human subject in Phase III clinical trial of a Licensed Product.
  • $500,000 upon the filing of a BLA with FDA (or EMEA or equivalent authority) in any jurisdiction for a Licensed product.
  • $1 million following the first commercial sale of a Licensed Product.
  • $500,000 for the second commercial sale of a Licensed Product.
  • $250,00 for the first commercial sale of a Licensed Product for any non-human use.
  • A one-time $50,000 payment is due upon issuance of a Licensed Patent in any of Australia, European Union, Japan, the U.S. or Canada including a valid claim to a licensed product.

Call to Action: Do you have a loved one with a leukemia diagnosis? It may make sense to monitor the TriKE platform and this study. Sign up for the TrialSite Newsletter as we will monitor the study.