Noxopharm granted FDA orphan drug status for CRO-67 to treat pancreatic cancer

Australian biotechnology company Noxopharm (ASX: NOX) has been granted orphan drug designation (ODD) status by the US Food and Drug Administration for its CRO-67 preclinical drug candidate to treat pancreatic cancer.

The FDA grants the status to drugs designed to prevent, diagnose or treat rare diseases or conditions.

It comes with benefits including tax credits for qualified clinical trials; exemptions from application fees; and up to seven years of market exclusivity after approval.

So far this year, only two other Australian companies have received an ODD from the FDA, from a total of 260 applications.

Positive move

Noxopharm chief executive officer Dr Gisela Mautner said the ODD designation was a positive move in the drug’s development.

“For CRO-67 to achieve an ODD is a significant milestone for us… in addition to financial benefits, it will strengthen our commercial position in a market which has seen very few new treatments over recent decades,” she said.

“Our pancreatic cancer program is a high priority and we are committed to progressing its development as quickly as possible with further studies and investigations into dosing and formulation.”

Development plan

Dr Mautner said ODD status would support Noxopharm’s development plan for CRO-67 as it continues to build a data package which will be needed for regulatory approvals.

It comes shortly after encouraging CRO-67 data was presented at the special conference of the American Association of Cancer Research last month in Massachusetts.

Pancreatic cancer is set to become the second leading cause of cancer-related deaths in the US by 2040, and has a five-year survival rate of around 9% from the time of diagnosis.

“There is an urgent need to develop innovative drugs [to treat pancreatic cancer] and therefore a major opportunity for Noxopharm to make a significant contribution in this space,” Dr Mautner said.

Novel, dual-cell candidate

CRO-67 is a novel, dual-cell pre-clinical candidate designed to target pancreatic cancer.

The disease is notoriously difficult to treat as tumours are surrounded by a dense barrier of cells which protects them from anti-cancer drugs, as well as from the body’s immune system.

In addition, the cancer typically shows a large diversity of genetic variations between individual patients.

This presents biotechnology companies with the challenge of developing a drug which works across a maximum number of variants.

In vivo research

Noxopharm’s latest in vivo research involved human pancreatic cancer tumour cells being implanted under the skin of mice.

The mice were then treated with CRO-67 for 21 days, with tumour volumes measured over the dosing period.

At the end of drug treatment, CRO-67 had been shown to significantly reduce tumour volume in vivo by an average of 56.7% versus the untreated controls.

Additionally, the drug was found to have slowed down the rate at which the tumours grew by 48%.

The median doubling time for the tumours treated with CRO-67 was 8.5 days, compared to 4.4 days for the untreated controls.