Essential adds neuroblastoma drug with Renaissance buy

Essential Pharma has bought fellow UK drugmaker Renaissance Pharma, adding its first development-stage drug candidate, a therapy for hard-to-treat rare cancer neuroblastoma.

The takeover of Renaissance comes just a few months after the company was set up to develop Hu14.18K322A (Hu14.18), a neuroblastoma therapy discovered at St Jude Children’s Research Hospital in the US.

Hu14.18 is a humanised anti-GD2 monoclonal antibody that is being tested in a phase 2 trial as an add-on to standard induction chemotherapy for the treatment of newly diagnosed high-risk neuroblastoma (HRNB). Each year, there are around 1,500 new cases of the cancer diagnosed in Europe and 800 in the US, of which around half are classed as high-risk disease.

Hu14.18 is in the same class as Y-mAbs Therapeutics’ Danyelza (naxitamab), a GD2-targeting antibody that was approved as a second-line treatment for relapsed/refractory high-risk neuroblastoma in the bone and bone marrow in 2020 and is due to report data from a first-line study in HRNB the coming weeks.

Neuroblastoma is a cancer of nerve tissue that usually occurs in children and arises in the nervous system, outside the brain, although aggressive forms can spread quickly to the central nervous system, as well as bone and bone marrow.

It accounts for 7%-10% of all childhood cancers and is the most common extracranial cancer in children, as well as the most common cancer in children under one year of age. High-risk cases are aggressive, with a five-year survival rate of around 50%.

The ongoing phase 2 trial of Hu14.18 is testing the drug as first-line therapy, as well as post-consolidation therapy for HRNB patients. Initial results showed a three-year event-free survival (EFS) rate of 73.7% and overall survival (OS) of 86%, with five-year data due shortly.

Taking on a development-stage drug candidate is something of a departure for Essential Pharma, which has built its business on the licensing of commercial generic and branded essential medicines, focusing on low-volume, difficult-to-manufacture, but clinically well-established pharmaceutical products.

Chief executive Emma Johnson said the deal was a “significant milestone” for the company, which has been steadily building its portfolio since a management buyout in 2020 supported by Gyrus Capital.

“Hu14.18 has enormous potential to help high-risk neuroblastoma patients, the majority of whom are young children,” she said. “We are now committed to developing this immunotherapy to be able to bring it to market and to patients as quickly as possible.”

Simon Ball, CEO and co-founder of Renaissance Pharma, will continue to work with Essential Pharma on Hu14.18’s development.