BAL101553 - Basilea Pharmaceutical

Basilea Pharmaceutica reports that it initiated a phase 2a study with its investigational oncology drug BAL101553. The study is designed to further characterise safety and tolerability, and to obtain efficacy data in adult patients with advanced or recurrent solid tumours who have failed standard therapy or for whom no effective standard therapy is available. Tumour types were selected based on clinical observations in the phase 1 study and a detailed analysis of potential patient stratification biomarkers across tumour indications. The study will also continue the extensive biomarker testing initiated in Phase 1, to further evaluate dose and patient populations most likely to respond.

BAL101553 is an intravenous and oral small-molecule microtubule-targeting agent (MTA) with a dual action against human tumours, targeting tumour cells refractory to standard MTAs as well as tumour blood supply. In preclinical studies the investigational drug demonstrated potent anti-cancer activity in a broad panel of treatment-resistant tumour models. Tumour cell proliferation was arrested and tumour cell death was induced through a destabilising effect on microtubules, an intracellular network essential for cell division. In addition, tumour-specific vascular disruption activity was observed in preclinical cancer models. First evidence of clinical antitumour activity has been seen in a recently completed phase 1 study.