UCL School of Pharmacy


Novel UCL cancer drug given Orphan Drug Designation (ODD) in USA for pancreatic cancer

24 January 2023

A novel UCL cancer drug candidate licensed by Qualigen Therapeutics has been granted Orphan Drug Designation (ODD) status in the USA for Pancreatic Cancer.

Emeritus Professor Stephen Neidle

Pancreatic cancer is one of the deadliest of all cancers. In the UK about 11,000 new cases are diagnosed every year, and about 62,000 in the United States. The incidence, however, is on the rise worldwide. Unfortunately, treatment for pancreatic cancer has not significantly advanced over the past several years, in striking contrast to improvements for many other human cancers. It still has a dismal 6-7% 5-year survival rate in the UK. 

Professor Stephen Neidle (UCL School of Pharmacy) has pioneered a novel approach to cancer therapy. Using specially designed compounds, this approach targets the unusual signal sequences present in elevated levels in many cancer-associated genes, especially those involved in pancreatic cancer. He and his group, with support from the UCL Technology Fund and the UCL Drug Discovery Group, have utilised this approach to develop a novel compound “QN-302” for the treatment of pancreatic cancer. Other potential indications include prostate cancer, sarcomas and gastrointestinal stromal tumors (GIST).

Qualigen Therapeutics, a US-based diversified life sciences company developing treatments for adult and paediatric cancers with potential for orphan drug designation, while also commercialising diagnostics, in-licensed this cancer therapeutic from UCLB in January 2022. In just twelve months, the lead compound QN-302 has advanced to the stage of IND-enabling studies and is now on track for Investigational New Drug (IND) FDA submission in the first half of 2023 to enter its first-in-human clinical trial. 

Furthermore, Qualigen recently announced that the U.S. Federal Drug Administration has granted Orphan Drug Designation (ODD) status to Qualigen for QN-302 for pancreatic cancer. ODD status provides advantages to pharmaceutical companies who are developing investigational drugs that show promise in treating rare diseases or conditions, and also to the benefit of pancreatic cancer patients.



Profile photo courtesy of Professor Stephen Neidle.