Cellestia presents promising clinical data of its lead compound

Basel-based Cellestia Biotech AG, a clinical stage biopharma company developing innovative treatments for cancer, presented their first clinical data of its lead compound CB-103 at the virtual congress of the European Society for Medical Oncology (ESMO). CB-103, which acts as a first-in-class protein-protein interaction inhibitor of the Notch transcription complex, showed excellent safety data and no signs of severe toxicity in the clinical phase 1 study.

The presented clinical study confirmed that CB 103 is the first drug in clinical testing that can control oncogenic NOTCH pathway activation effectively and safely, in absence of any severe toxicities. In the dose escalation, 41 patients with advanced metastatic tumours have been treated at doses ranging from 15 – 600 mg once daily; CB-103 was generally well tolerated. At doses of 120mg and above, up to 90% down regulation of NOTCH target genes in surrogate tissue has been demonstrated, confirming effective and sustained target engagement, in the absence of significant side effects, notably no dose limiting diarrhoea. Target engagement coincided with long-term Stable Disease in NOTCH positive ACC (Adenoid Cystic Carcinoma) patients. The dose of 600 mg QD has been declared as safe dose and further dose escalation is possible; the study is ongoing.

Florian D Vogl, Chief Medical Officer of Cellestia commented: “We are very excited to share at ESMO for the first-time clinical results from the ongoing Phase 1 study of CB-103. Following these very encouraging results we are committed to advance our lead molecule CB-103 into Phase 2 in various indications. The necessary preparations are well in progress to timely launch our global clinical phase 2 program in the EU, the USA, and Asia.”

Cellestia's approach

By building on in-house know-how generated over more than a decade, Cellestia Biotech has developed a drug discovery platform to directly target the NOTCH transcription complex using small molecule inhibitors. Cellestia’s clinical-stage drug candidate, CB-103, acts as a first-in-class protein-protein interaction inhibitor of the NOTCH transcription complex and thereby inhibits NOTCH signalling at the most downstream event in the cascade. Due to its unique mode of action, CB-103 acts as a pan-NOTCH inhibitor and circumvents the gastrointestinal tract toxicities associated with previous generations of NOTCH inhibitors.

About Cellestia Biotech AG

Cellestia is a biopharmaceutical company specialised in research and development of first-in-class drugs targeting gene transcription factors enabling the treatment of multi-drug resistant cancers as well as a wide range of non-cancer indications. This innovative approach has successfully led to a pipeline of proprietary drug candidates. Cellestia’s lead compound CB-103 is a highly potent and selective inhibitor of NOTCH transcription factor and is currently in clinical testing. Oncogenic activation of the NOTCH pathway is typically involved in highly aggressive, metastatic and multi-drug resistant, hard-to-treat cancers. Cellestia holds a worldwide exclusive license on the intellectual property rights for CB-103 and related series of analogues, for development and commercialization. The company pursues an integrated approach combining drug and personalized medicine development for patient selection.