Acelarin (NUC-1031; NuCana) has been granted Orphan Drug designation by the Food and Drug Administration (FDA) for the treatment of biliary tract cancer.

The investigational agent is derived from gemcitabine, a nucleoside metabolic inhibitor. According to the Company, the treatment is comprised of a “pre-activated nucleotide analogue and a protective phosphoramidate moiety” which enables the drug to enter the cancer cell independent of membrane transporters and protects it from degradation.

“Our phase Ib study of Acelarin combined with cisplatin showed an approximate doubling of the response rate expected with the standard of care, gemcitabine plus cisplatin, with several patients achieving significant reductions in their tumor volume as well as further tumor shrinkage over time,” said Hugh Griffith, NuCana’s Founder and Chief Executive Officer. The Company expects to begin a phase 3 trial evaluating Acelarin in combination with cisplatin as a first line treatment for patients with advanced biliary tract cancer sometime in 2019.

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The treatment is also being investigated in a phase 2 study for patients with ovarian cancer and a phase 3 study in patients with metastatic pancreatic cancer.


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If approved, Orphan Drug designation guarantees the Company 7 years of market exclusivity.

For more information visit nucana.com.