Basilea Pharmaceutica Ltd. announced the start of the first clinical study with its anti-cancer drug BAL101553. The open-label phase I study will determine the safety, tolerability and pharmacokinetic profile of BAL101553, a novel microtubule-targeting agent which has demonstrated anti-cancer activity in many drug-resistant tumour models.

Cancer remains a significant challenge in healthcare despite progress in the development of agents with anti-tumour activity. Specifically, there is an increasing medical need for novel agents with new mechanisms of action that overcome tumour resistance against current therapies.

BAL101553 is a highly water-soluble prodrug of the synthetic small molecule BAL27862 that allows intravenous and oral administration without solubilizing excipients associated with adverse effects. BAL27862 arrests tumour cell proliferation and induces tumour cell death through a characteristic destabilizing effect on microtubules that is distinct from other anti-cancer agents also directed against the microtubule network. Anti-tumour activity has been demonstrated across a broad panel of solid tumour models, including those resistant against conventional microtubule-targeting drugs such as taxanes or Vinca alkaloids.

The phase I open-label, dose-escalation study investigates safety, tolerability and pharmacokinetics of intravenous BAL101553 to establish dose and administration schedule for phase II studies. The study includes adult patients with advanced solid tumours who have failed standard therapy or for whom no effective standard therapy is available.

“There is a high need for novel anti-cancer therapies to treat patients whose cancers are resistant to current agents such as taxanes,” said Dr Anthony Man, CEO of Basilea Pharmaceutica Ltd. “BAL101553 is a novel small-molecule drug active against a well-validated cancer cell target and showing broad anti-tumour activity in preclinical models. We are delighted to see this innovative anti-cancer drug move into clinical development to broaden our hospital-focused product portfolio.”

Basilea Pharmaceutica Ltd. is fully integrated research and development operations are focused on antibiotics, antifungals and oncology drugs, as well as on the development of dermatology drugs, targeting the medical challenge of resistance and non-response to current treatment options in the hospital and specialty care setting.