AZAYA Therapeutics, Inc. is an emerging technology company that is based in San Antonio, Texas. We began operations in 2003, and are a privately owned Delaware Corporation. AZAYA is using a novel, proprietary and widely applicable targeted nanotechnology platform aimed at developing safer and more effective cancer treatments for patients around the world. We received a patent for our Protein Stabilized Liposome (PSL™) nanotechnology in February, 2007. This platform solves the significant problem associated with the formulation and delivery of water-insoluble active pharmaceutical ingredients, and has the potential to be applied to numerous therapeutic agents.

Liposomes are small structures that were discovered in the early 1960s by British haematologist Dr. Alec D. Bangham. The name is derived from two Greek words: “LIPO” meaning fat and “SOMA” meaning body. They consist of an aqueous solution (a drug, for example) encapsulated by a hydrophobic membrane. Liposomes have unique properties that enable them to deliver drugs directly into targeted cells by “fusing” with the cell membrane and releasing the drug into the cell.  In treating cancer, liposomes are very effective due to a mechanism referred to as the Enhanced Permeability and Retention (EPR) effect. Healthy human blood vessels are encased by tightly bound endothelial cells that do not permit any large particles to “leak” from the vessel into surrounding tissue. In patients with cancer, the blood vessels that support the growth of tumors do not have tightly bound endothelial cells, and “leakage” from the blood vessel into surrounding tissue occurs. Liposomes smaller than 200 nm in diameter are able to move from the blood vessel into the tumor, releasing the chemotherapy drug.

During the late 1970s and early ‘80s, liposomes were re-engineered to maintain their stability so they could circulate in the blood for longer periods of time. While this was accomplished and stealth™ liposomes – ideal for delivering pharmaceutical drugs directly to cells - were developed, they remained very difficult to produce on a large scale.

Seeing the benefits that liposomal formulations of chemotherapy drugs offer to patients with cancer, but recognizing the production challenges, AZAYA identified an important opportunity. If we could develop a platform technology that reliably produced large quantities of liposomal encapsulated drugs, we would be advancing the state of cancer therapy. The AZAYA Protein Stabilized Liposome (PSL™) technology was developed, utilizing a one-step manufacturing process that offers a more efficient and economical production model.  By applying this proprietary process, active pharmaceutical ingredients are encapsulated in the lipid layer of the liposome and form active nanoparticles of less than 120 nm in size, facilitating movement from the blood vessel to the targeted cancer cells.

The PSL technology provides two additional key advances to cancer therapy. Many commonly used cytotoxic agents are insoluble, and must be combined with a solvent prior to administration to a patient. The solvents used (polysorbate-80 and Cremophor-EL™) are highly toxic to patients and require that the patient be pre-medicated with corticosteroids, antihistamines, and H₂ antagonists prior to receiving the chemotherapy agent.  Using the PSL technology enables liposome-encapsulated drugs to be administered without the need for solvents and the associated pre-medications. In addition, PSL technology appears to target key proteins that have been over-expressed in malignant cells, which results in improved response rates and outcomes.

The pre-clinical studies conducted by AZAYA have demonstrated enhanced pharmacokinetics, increased inhibition of tumor xenograft models, and evidence of increased tumor uptake of the active compound - which together promise improved therapeutic results.

At the 2011 American Society of Clinical Oncology (ASCO) Conference, AZAYA presented data from a Phase 1 study on the compound ATI-1123 - a novel serum albumin-stabilized nanoparticle docetaxel liposomal formulation – in heavily pre-treated patients with advanced solid tumors. This study established the maximum tolerated dose and dose-limiting toxicities of ATI-1123. The clinical results observed in the study were very impressive – the toxicity profile was much improved over what is typically experienced with docetaxel, and responses were seen in patients who had previously received docetaxel. Four additional Phase II studies in solid tumors are planned for 2012.

A second project is underway at AZAYA – referred to as ATI-0918 – which is to produce a generic form of liposomal doxorubicin. The patents for DOXIL®, the branded  drug, expired in late 2009, and there are currently no generics on the market. Due to manufacturing challenges, there is a severe shortage of DOXIL worldwide, and this problem is expected to continue though the next few years. Liposomal doxorubicin is an important chemotherapy agent that has shown strong efficacy in a number of malignancies, and AZAYA’s plan is to move toward commercialization as expeditiously as possible, so patients around the world can continue receiving this therapy.

AZAYA is open to partnership opportunities with other organizations to utilize the proprietary PSL technology to extend the marketing life of existing cancer treatments, and to develop new agents that will markedly change the course of cancer.

Prolonging Survival and Improving the Quality of Life Through Our Novel PSL™ Platform Technology