Benzinga | May 12, 2021 07:03AM EDT

GT Biopharma Says Announces Update On Commencement Of GTB-3550 TriKE Monotherapy Phase 2 Clinical Trial; Says Indications Of Anti-Tumor Activity During Phase 1 Resulted In 'refocusing of the Phase 2 design of the clinical trial'

GT Biopharma, Inc. (NASDAQ:GTBP), a clinical stage immuno-oncology company focused on developing innovative therapeutics based on the Company's proprietary NK cell engager (TriKE(tm)) protein biologic technology platform, is pleased to provide an update concerning the commencement of the GTB-3550 TriKE(tm) monotherapy Phase 2 clinical trial, and certain of its solid tumor targeting TriKE(tm) product candidates.

Highlights to date from patients treated with GTB-3550 TriKE(tm) in the dose escalation Phase 1 clinical trial for the treatment of high-risk myelodysplastic syndromes (MDS) and refractory/relapsed acute myeloid leukemia (AML):

* Up to 63.7% Reduction in Bone Marrow Blast Levels seen in some patients.

* Restoration of Patient's Endogenous NK Cell Function, Proliferation and Immune Surveillance.

* No Progenitor-derived or Autologous/Allogenic Cell Therapy Required.

* No Cytokine Release Syndrome Observed.

While the design of the Phase 1 part of the GTB-3550 clinical trial was focused on evaluating safety, indications of anti-tumor activity during Phase 1 have resulted in a refocusing of the Phase 2 design of the clinical trial towards enhancing efficacy, durability of the clinical response, and overall survival with the goal to seek accelerated approval from FDA. We intend to (i) enroll patients with CD33 expression ?50% in two independent cohorts (higher-risk myelodysplastic syndrome and acute myeloid leukemia); (ii) treat patients with two cycles of GTB-3550 therapy with a rest period between cycles as opposed to the single-cycle used during Phase 1; (iii) enroll patients with fewer prior treatment lines; and, (iv) evaluate the potential use of minimal residual disease (MRD) based endpoints that may allow for accelerated approval.

Solid tumor cancers present a significantly larger market opportunity than hematologic cancer indications, and represent the majority of new cancer diagnoses annually. The Company is presently advancing three TriKE(tm) product candidates in GMP manufacturing and early clinical development. These solid tumor TriKE product candidates will target cancers expressing HER2 (GTB-6550), PD-L1 (GTB-4550) and B7H3 (GTB-5550), and will be evaluated for the treatment of multiple cancers such as breast, lung, gastric, colorectal and ovarian. We believe GTB-3550 established the importance of incorporating IL-15 directly within the TriKE protein biologic, providing NK cells with targeted cytokine stimulation simultaneously with target-directed cancer cell killing. We believe the selective expansion and proliferation of the patient's endogenous NK cells coupled with enhanced target-directed infiltration of activated NK cells into the bone marrow resulting in significantly AML cancer blast killing will translate to the solid tumor microenvironment.

"We believe GTB-3550 TriKE(tm) sets a new standard for NK cell engager therapies due to the incorporation of interleukin 15 (IL-15) directly in the protein backbone," said Anthony J. Cataldo, GT Biopharma's Chairman and Chief Executive Officer. "The flexibility and versatility of our TriKE(tm) platform allows us to change the cancer cell targeting mechanism of TriKE(tm) to attack different cancers while maintaining the core NK cell activation, proliferation and persistence attributes of the molecule," Mr. Cataldo further stated.

About High-Risk Myelodysplastic Syndromes

MDS is a rare form of bone marrow-related cancer caused by irregular blood cell production within the bone marrow. As a result of this irregular production, MDS patients do not have sufficient normal red blood cells, white blood cells and/or platelets in circulation. High-risk MDS is associated with poor prognosis, diminished quality of life, and a higher chance of transformation to acute myeloid leukemia. Approximately 40% of patients with High-Risk MDS transform to AML, another aggressive cancer with poor outcomes.

About Acute Myeloid Leukemia

Acute myeloid leukemia is a type of cancer in which the bone marrow makes abnormal myeloblasts (a type of white blood cell), red blood cells, or platelets. According to the National Cancer Institute (NCI), the five-year survival rate is about 35% in people under 60 years old, and 10% in people over 60 years old. Older people whose health is too poor for intensive chemotherapy have a typical survival of five to ten months. AML accounts for roughly 1.8% of cancer deaths in the United States.