Madrid, Spain 4 February, 2019 – Pivotal has enrolled the first patients in a recently awarded new phase II trial in Europe, testing a targeted drug in patients with von Hippel-Lindau (VHL) disease, a hereditary disease which increases the risk to develop multiple tumours, mainly renal cell cancer, and also haemangioblastoma and retinoblastoma, among others.

The incidence of von Hippel-Lindau syndrome is estimated to be 1 in 36,000 individuals, and tumours most frequently appear during young adulthood. The disease is caused by mutations in the VHL gene, which is a tumor suppressor gene. Mutations in this gene prevent production of the VHL protein or lead to the production of an abnormal version of the protein. An altered or missing VHL protein cannot effectively regulate cell survival and division. As a result, cells grow and divide uncontrollably to form the tumours and cysts that are characteristic of von Hippel-Lindau syndrome.

The sponsor is a US-based biopharmaceutical company developing first-in-class oral medicines for patients with cancer and other serious or life-threatening conditions. The IMP is an orally active, small molecule with potential antineoplastic activity.

Pivotal is working in close partnership with the sponsor in the execution of this development programme to study the new drug. It will provide its internal European resources to increase and streamline operational efficiency, including Start-up, Regulatory, Clinical Operations, Medical Monitoring, Pharmacovigilance and other operational services in 6 countries in Europe.

“The power of partnerships, such as the one we are embarking on, brings together the best minds and capabilities to accelerate this dynamic area of research and bring therapies to patients with high unmet needs,” said Lourdes Huarte, MPham, PhD, MBA. “We are delighted to start this trial in Europe and deploy our capabilities to accelerate the development of medicines in such a difficult-to-treat area.”

The study will include up to 60 patients from US and European sites and will assess, as primary objective, the clinical benefit in terms of overall response rate in renal tumours and, as secondary objectives, duration of response, time to response, progression free survival, time to surgery, pharmacokinetics, safety and tolerability. Additionally, the trial will assess the efficacy in other disease-associated tumours. Promising results were achieved in a phase I study with this drug, showing preliminary clinical efficacy and a very good safety profile.

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