Instant Report

Date: 04-Jan-2021

Ascendis Pharma Seeks US FDA Approval To Initiate TransCon TLR7/8 Agonist Clinical Programme

Ascendis Pharma, a biopharmaceutical company, announced the filing of an investigational new drug (IND) application with the US Food and Drug Administration (FDA) to initiate the clinical programme of TransCon TLR7/8 Agonist.

TransCon TLR7/8 Agonist is a long-acting prodrug of resiquimod, a small molecule agonist of Toll-like receptors (TLR) 7 and 8. Administered as an intratumoral injection, TransCon TLR7/8 Agonist is designed to provide sustained activation of intratumoral antigen presenting cells driving tumor antigen presentation and induction of immune stimulatory cytokines in the tumor.

“The filing of our first oncology IND for TransCon TLR7/8 Agonist – which is designed to provide intratumoral, sustained release of resiquimod over several weeks from a single administration with minimal systemic exposure and potent immune response against cancer cells – is a major milestone for Ascendis,” said Juha Punnonen, M.D., Ph.D., senior vice president and head of oncology at Ascendis Pharma. “We believe TransCon TLR7/8 Agonist represents a potential paradigm shift in the treatment of cancer through sustained release of an immunostimulatory molecule over several weeks inside the tumor, thereby employing the patients’ own immune systems to destroy cancer cells with reduced risk of systemic adverse events.”

TransCon refers to “transient conjugation.” The proprietary TransCon platform is an innovative technology to create new therapies that are designed to optimize therapeutic effect, including efficacy and safety and through dosing frequency. TransCon molecules have three components: an unmodified parent drug, an inert carrier that protects it, and a linker that temporarily binds the two. When bound, the carrier inactivates and shields the parent drug from clearance. When injected into the body, physiologic conditions (e.g., pH and temperature) initiate the release of the active, unmodified parent drug in a predictable manner. Because the parent drug is unmodified, its original mode of action is expected to be maintained. TransCon technology can be applied broadly to proteins, peptides or small molecules in multiple therapeutic areas, and can be designed for systemic or localized release.