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CellAct’s targeted anti-cancer drug to initiate pivotal phase III in patients with Biliary Tract Cancers

CellAct Pharma is a Phase III clinical stage, venture backed biotech company based on science generated from Charite Univ. Hospital, Berlin, Harvard Medical School, Boston and
other international leading institutions. The company focuses on development of innovative therapeutics for the treatment of various cancers. CellAct programs are managed by an experienced management team.


CellAct’s lead product, the targeted anti-cancer drug, CAP7.1, is ready to enter Phase III. CAP7.1 is converted by Carboxylesterase 2 enzyme (CES2) in specific tissues, to an active topoisomerase II inhibitor. The conversion of CAP7.1 via CES2 allows tissue and cancer specific targeted treatment approach and patient selection.

CAP7.1 completed successfully Phase I and II studies in adults with endstage, refractory malignancies of the lung, nasopharyx, biliary tract, stomach, esophagus, ovary, testes as well as neuroendocrine tumors of the skin. The drug was also administered in children with progressive Neuroblastoma disease. CAP7.1 showed acceptable safety profile in both adults and children with heavily pretreated solid tumors. Initial efficacy data reflects CAP7.1’s potential to treat various refractory malignancies.

CAP7.1 has statistically meaningful randomized Phase II efficacy data for hard-to-treat tumors, and a benefit risk profile in patients with heavily pretreated refractory malignancies. Patient with CES2+ tumors (right panel below) demonstrated highest benefit reflected in longest survival time (left panel below) (doi:10.3390/cancers12113149).


A multinational pivotal Phase III of CAP7.1 in is scheduled for unresectable Biliary Tract Cancer (BTC), a rare and highly aggressive cancer with poor prognosis. BTC patient population is almost 40K in the US and EU, with a substantially larger patient population in China. With a strong patent portfolio and orphan drug designation in the EU and USA, the asset will be positioned for second and third line in BTC. Expansion into other solid tumor populations will follow.


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