Radiopharm Theranostics gains orphan drug designation for RAD 301 to treat pancreatic cancers

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By Imelda Cotton - 
Radiopharm Theranostics ASX RAD orphan drug designation pancreatic ductal adenocarcinoma Radiopharmaceutical technology

Radiopharm Theranostics (ASX: RAD) has been granted orphan drug designation by the US Food and Drug Administration for its Ga68-Trivehexin (RAD 301) radiopharmaceutical technology for the imaging of patients with pancreatic ductal adenocarcinoma (PDAC).

Trivehexin is a proprietary peptide-based molecule which targets the αvβ6-integrin cellular marker for tumor invasion and metastatic growth.

The receptor is found in high density on most pancreatic carcinoma cells, making it an attractive diagnostic and therapeutic target.

The expression of αvβ6-integrin correlates with decreased survival in several carcinomas.

Phase 1 clinical trial

Chief executive officer Riccardo Canevari said the Orphan Drug Designation would be followed by a Phase 1 clinical trial focused on pancreatic cancers.

“The FDA’s decision highlights the significant demand for effective imaging agents for improved and earlier diagnosis of pancreatic cancer, which has one of the highest levels of unmet needs among all cancer types,” he said.

Treating rare diseases

Orphan designations are granted for drugs or biologic products with the potential to diagnose, prevent or treat rare diseases and conditions.

Recipients can qualify for benefits including tax credits for clinical trials, exemptions from user fees and a potential seven years of market exclusivity if their drug is approved.

Radiopharm holds two FDA orphan drug designations, along with the LRRC15 antibody DUNP19 for the treatment of patients with osteosarcoma.

The company has signed an exclusive licensing agreement with German radiopharmaceutical company TRIMT GmbH for the development and commercialisation of RAD 301 in the US, Australia, China, Hong Kong and Japan.

Radiation therapy

Radiopharmaceuticals deliver small doses of radiation to targeted cells for therapeutic or diagnostic purposes.

Mr Canevari said they are fast becoming a highly-promising therapeutic frontier in oncology.

“Radiopharmaceuticals offer new hope to patients who may have exhausted all other treatment options,” he said.

GenesisCare partnership

In March, Radiopharm announced a two-year partnership with Australian company GenesisCare to develop novel radiopharmaceuticals for complex, hard-to-treat cancers.

The companies will work together on Australian-based Phase 1 clinical trials focused on drug safety and tolerability in areas of high unmet oncological need.

The trials will involve a proprietary nanobody from the Nano-mAbs platform which targets the PDL1 expression in non-small cell lung cancer; a PTPu targeting peptide for brain tumors; and a PSA targeting antibody focused on free human prostate kallikrein (PSA) in prostate cancer cells.