Presentation

Cellestia is a private, Swiss-based Biotech developing first-in-class therapies to control pathogenic gene expression by selective inhibition of previously undruggable transcription factors in the cell nucleus. CB-103 is a safe, highly selective, pan-NOTCH inhibitor targeting the “undruggable” downstream gene transcription factor complex CSL-NOTCH intracellular domain – delivering potency comparable to genetic knock-out. The drug has advanced to phase 2 in oncology, followed by a R&D pipeline of novel gene transcription factor inhibitors in oncology, auto immune and inflammatory disorders. Scientific Advisory Board chaired by David R. Epstein, former CEO of Novartis Pharmaceuticals.

Recent highlights
CB-103 has demonstrated no dose limiting toxicities (DLTs) with efficacy in a Phase 1/2a trial. First CB-103 treated patient with T-ALL (a rare, acute type of blood cancer) achieved a CR in combo treatment. Several solid tumour patients show tumour shrinkage and/or long-term disease control.

Outlook
Currently there are no FDA approved therapies targeting NOTCH. Previous upstream approaches targeting NOTCH, such as γ-secretase inhibitors, are effective but unspecific/toxic, affecting a number of physiological processes, while CB-103 is proven to be safe. The company is currently preparing for phase 2 trials in ACC (rare solid tumour) and T-ALL, followed by an upcoming phase 2 trial in ER+ breast cancer as well as a potential phase 2 trial in aGvHD (autoimmune disease after stem cell transplant). Given the rich R&D pipeline, good safety of CB-103 and demonstrated efficacies, we believe that Cellestia will be able to continue advancing the developments and unlock blockbuster potentials.