GT Biopharma, Inc. (NASDAQ:GTBP) is a clinical-stage biopharmaceutical company at the forefront of the immuno-oncology revolution. With its proprietary Tri-specific Killer Engager (TriKE®) and Tetra-specific Killer Engager (Dual Targeting TriKE) technology platforms, the company is developing a robust pipeline of innovative therapeutics designed to harness and enhance the natural cancer-killing abilities of a patient's own natural killer (NK) cells.
Business Overview and History GT Biopharma was founded in 1965 as Diagnostic Data, Inc., a California-based company focused on diagnostic testing. In 1972, the company reincorporated in Delaware and changed its name to DDI Pharmaceuticals, Inc. The company underwent several mergers and name changes over the years, eventually becoming OXIS International, Inc. in 1994 and then GT Biopharma, Inc. in 2017.
The company's current focus on immuno-oncology began in 2016 when it entered into an exclusive patent license agreement with the Regents of the University of Minnesota to develop and commercialize cancer therapies using the TriKE technology. This platform generates proprietary therapeutics designed to target and activate a patient's NK cells, directing them to specific cancer cells for destruction.
In 2021, GT Biopharma further expanded its intellectual property portfolio by entering into an exclusive license agreement with the Regents of the University of Minnesota specific to the B7H3 targeted TriKE technology. This agreement provided GT Biopharma with exclusive worldwide rights to conduct research and develop, make, use, sell, and import the TriKE technology for the treatment of any disease, state, or condition in humans.
To support the development of its novel immunotherapy platforms, GT Biopharma entered into a Master Services Agreement with Cytovance Biologics, Inc. in 2020. This partnership enables Cytovance to perform biologic development and manufacturing services, as well as produce and test compounds used in the company's potential product candidates.
Throughout its history, GT Biopharma has faced various challenges, particularly in raising capital and managing operating expenses. For instance, in 2022, the company reported a net loss of $20.9 million and used $15.2 million in cash from operations. Despite these obstacles, GT Biopharma has continued to advance its pipeline and achieve key milestones in its development programs.
Since then, GT Biopharma has rapidly advanced its TriKE pipeline, with several product candidates in various stages of clinical development. The company's lead program, GTB-3650, is a second-generation TriKE targeting CD33, an antigen expressed on acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) cells. In June 2024, the FDA cleared GT Biopharma's Investigational New Drug (IND) application for GTB-3650, paving the way for the initiation of a Phase 1 clinical trial in the second half of 2024. The company believes GTB-3650 has the potential for enhanced potency compared to its earlier TriKE candidate, GTB-3550, based on preclinical data showing better tumor control in animal models. Enrollment for the Phase 1 trial began on January 21, 2025.
In addition to GTB-3650, the company is also developing GTB-5550, a TriKE that targets the B7-H3 antigen expressed on a broad range of solid tumors, including prostate, breast, head and neck, ovarian, lung, and gastrointestinal cancers. GT Biopharma initiated GMP manufacturing for GTB-5550 in 2023 and plans to submit an IND for GTB-5550 in the first half of 2025 and initiate a Phase 1 dose-escalation basket trial shortly thereafter.
The company's pipeline also includes GTB-7550, a TriKE that targets CD19 and is in preclinical development for the treatment of lupus and other autoimmune disorders.
Financials and Liquidity As of December 31, 2024, GT Biopharma reported cash and cash equivalents of $4.04 million, which the company believes will be sufficient to fund operations into the second quarter of 2025. The company has not generated any revenue to date, as its product candidates are still in the clinical development stage.
For the year ended December 31, 2024, GT Biopharma reported a net loss of $13.16 million, with research and development expenses of $5.80 million and selling, general, and administrative expenses of $8.57 million. The company's net loss per share for the year was $6.94. GT Biopharma used $12.90 million in cash for operations during this period.
In the fourth quarter of 2024, GT Biopharma raised $3.2 million in a registered direct offering, further strengthening its balance sheet and providing additional runway to advance its pipeline. However, the company's financial statements raise substantial doubt about its ability to continue as a going concern, as it will require additional financing to fund its planned level of operations.
For the most recent quarter, GT Biopharma reported revenue of $0, a net loss of $3.78 million, and did not disclose quarterly operating cash flow or free cash flow figures.
As of December 31, 2024, the company had no debt, a current ratio of 0.717, and a quick ratio of 0.717. GT Biopharma does not disclose any available credit lines or credit facilities.
Risks and Challenges As a clinical-stage biopharmaceutical company, GT Biopharma faces several risks and challenges common to the industry, including:
1. Uncertainty around the successful development and regulatory approval of its product candidates: The company's ability to generate revenue is dependent on the successful completion of clinical trials and obtaining regulatory approvals for its TriKE therapies, which is a lengthy and expensive process with no guarantee of success.
2. Intense competition in the immuno-oncology space: GT Biopharma operates in a highly competitive market, with numerous other companies developing novel cancer immunotherapies, which could impact the commercial potential of its products.
3. Reliance on third-party manufacturers and suppliers: The company's operations are dependent on the reliable performance of its manufacturing partners, any disruptions to which could delay the development and commercialization of its products.
4. Ability to raise additional capital: As a pre-revenue company, GT Biopharma will need to secure additional financing to support its ongoing operations and future clinical trials, which may not be available on favorable terms or at all.
5. Potential for clinical trial failures or delays: The company's clinical development programs may be subject to delays or failures, which could significantly set back its timeline and increase costs.
Corporate Governance and Legal Matters GT Biopharma has faced some challenges related to corporate governance and legal matters in recent years. In July 2024, the company filed an arbitration demand against its former CFO, Manu Ohri, alleging breach of fiduciary duties and breach of contract. Additionally, in November 2023, the company's former interim CEO, Dr. Gregory Berk, filed a lawsuit alleging discrimination and retaliation. This lawsuit was later dismissed with prejudice in December 2024 as part of a settlement.
The company has not disclosed any short seller reports or other significant corporate scandals. GT Biopharma does not provide information about performance by geographic markets, and as a small-cap company, it is likely that its operations are primarily focused on the United States.
Outlook and Conclusion Despite the challenges, GT Biopharma remains well-positioned to advance its innovative TriKE and Dual Targeting TriKE platforms and address significant unmet medical needs in cancer and autoimmune diseases. The company's lead program, GTB-3650, has entered a Phase 1 clinical trial, with initial data anticipated in the first half of 2025. Additionally, the company plans to submit an IND for its solid tumor-targeting TriKE, GTB-5550, in the first half of 2025, followed by the initiation of a Phase 1 dose-escalation basket trial.
GT Biopharma continues to leverage its research collaborations and licensing agreements with the University of Minnesota related to its TriKE technology. In 2024, the company entered into a clinical trial agreement with the university to sponsor a phase 1 trial for GTB-3650, further strengthening its development capabilities.
With a robust pipeline, a novel therapeutic approach, and a seasoned management team, GT Biopharma is poised to make significant strides in the immuno-oncology landscape. As the company continues to execute on its clinical development strategy and navigate the regulatory landscape, investors will closely watch for key milestones that could unlock the full potential of its TriKE technology and drive long-term value creation. However, the company's financial position remains a significant challenge that will need to be addressed through additional funding to support its ongoing research and development efforts.