ST-001

Innovation: Therapeutics & drug delivery—unique nano-delivery system to enable intravenous (IV) delivery of water-insoluble cancer drug
Targeted Cancer(s): Non-Hodgkin lymphoma and small cell lung cancer
Scientific Leadership: Michael Sporn, M.D.; Michael Burns, Ph.D.; Ralph Parchment, Ph.D.
Stage of Project: Molecule is clinic-ready; approved Investigational New Drug (IND) status issued by the U.S. Food and Drug Administration (FDA) in December 2019; orphan drug designation granted in 2018

Fenretinide, a synthetic small-molecule which has been clinically tested as a breast cancer prevention drug in more than 3,000 women, has been shown to be safe after long periods of use. However, heretofore it has not been approved as a cancer therapeutic due to the challenges of delivering it in adequate doses to tumor cells. The molecule is hydrophobic, leading to solubility problems, and multiple IV delivery methods have not been able to overcome this critical hurdle. Not until now. 

A unique IV delivery system has been developed which is able to safely deliver concentrations of fenretinide, thereby prospectively achieving therapeutically effective doses without the toxic side effects observed with other methods. Combined with this proprietary drug delivery, an additional immunotherapeutic effect of the drug has been demonstrated. This reactivated natural immune response compliments the previously understood safe and direct chemotherapeutic effect, resulting in a unique, genetically directed process of cancer cell destruction (known as apoptosis).

• The treatment outcome of patients with T-cell non-Hodgkin lymphoma (T-cell NHL) and small cell lung cancer (SCLC) remains dismal.
• Within T-cell NHL cancer indications, the cutaneous variants remain chronic but incurable. The natural disease history for many patients is marked by responses and relapses to therapies with eventual progression and fatal outcomes. For the more aggressive types, the long-term survival is approximately 20%.
• The 5-year overall survival rate for SCLC patients seldom exceeds 5%.
• Studies have demonstrated that fenretinide is a promising drug candidate for the treatment of these and other types of aggressive cancers.
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• The combination of fenretinide with the proprietary delivery vehicle has led to a unique drug candidate. Known as ST-001, it is a nanoparticle suspension for IV administration, comprised of fenretinide in patented combination with specifically selected phospholipids (inactive ingredients).
• The phospholipids have been chosen because they are recognized as safe for IV use in humans and because of their unique chemical and physical properties which, when combined with those of fenretinide, yield an integrated, robust structure that contains a much higher concentration of fenretinide. Simply put, the drug candidate is protected from water by the lipid layers of the nanoparticle, which dissolve only when fused or absorbed by the cancer cell—a benefit previously unattainable.

Proof of Concept: Prior clinical trial data validates the safety and efficacy of fenretinide, which makes the case for its use in the new formulation, and the associated clinical trial will be confirmatory, not exploratory

Intellectual Property: Co-owned licenses and patents have been issued in the U.S., EU and other countries; furthermore, a patent runway expansion is under development and a new provisional U.S. patent was filed in November 2018

Clinical Development Plan: The immediate goal is to reconfirm fenretinide’s safety and efficacy in its new formulation, opening the potential for use in T-cell NHL, SCLC and other cancers