Burzynski Research Institute is a clinical-stage company with no revenue, focused entirely on developing a cancer treatment called Antineoplastons. Antineoplastons are peptides, amino acid derivatives, and organic acids originally isolated from human blood and urine by Dr. Stanislaw Burzynski, based on the theory that these naturally occurring substances can inhibit cancer cell growth without harming normal tissue. The company provides Antineoplastons exclusively to Dr. Burzynski for use in FDA-authorized clinical trials at his Houston clinic, with a primary focus on brain cancers including glioblastoma, brainstem glioma, and DIPG. The company has no employees and no commercial operations — Antineoplastons cannot legally be sold anywhere in the world. The sole source of funding is payments from Dr. Burzynski's personal medical practice. The entire business depends on obtaining FDA approval, which remains highly uncertain. Phase II trials began in 1994 across a range of brain cancer protocols, but the program is currently under a full clinical hold imposed by the FDA in 2017 due to manufacturing deficiencies. Phase III trials, the pivotal step toward an NDA, have not begun enrollment and are also on hold pending resolution of manufacturing issues and funding. The FDA has raised additional concerns around serious adverse events, trial design, and data integrity, and has indicated it would likely require trials conducted at institutions independent of Dr. Burzynski before accepting an NDA. Antineoplastons hold orphan drug designation for brainstem glioma and gliomas, which would provide seven years of marketing exclusivity if approval is ever granted.