Takeda signs $882m rare disease alliance with Oxford biotech Evox

Evox Therapeutics is the second UK biotech to sign a big deal with a pharma giant this week, snaring an $882 million partnership with Takeda for up to five rare disease programmes.

The alliance – which includes $44 million in upfront fees, near-term milestones and research funding – is focusing on the development of protein replacement and mRNA therapies that will be delivered using the Oxford University spin-out’s exosome delivery platform.

Earlier this week, AstraZeneca agreed a $2 billion-plus deal with London-based gene-silencing specialist Silence Therapeutics – including $80m upfront – to develop up to five drugs for cardiovascular, renal, metabolic and respiratory diseases over the next three years.

Takeda’s agreement with Evox covers a preclinical-stage candidate for Niemann-Pick disease type C (NPC), a progressive genetic disorder that leads to the accumulation of fatty substances in tissues and can cause a range of symptoms, including liver and lung disease and neurological impairment.

A second programme covers an undisclosed rare disease, and Takeda has an option on up to three additional targets. The Japanese drugmaker will assume responsibility for the programmes once they reach the clinical development stage.

Evox development platform is concentrated around its use of exosomes – extracellular membrane-bound parcels or ‘vesicles’ that are used by cells to transport molecules between them – which can be used to deliver large substances like drugs made from protein or RNA into cells.

The Takeda alliance is the first with a big pharma company for Evox, although it has other partnerships in place, including a programme with medical charity Duchenne UK looking at using exosomes to deliver dystrophin, a protein deficient in people with Duchenne muscular dystrophy.

Last year, it also won a £1.5 million ($1.8 million) grant from Innovate UK to develop an exosome-based therapy to treat argininosuccinic aciduria (ASA) – a rare life-threatening metabolic disease – in collaboration with University College London (UCL).

Takeda’s head of rare disease drug discovery Madhu Natarajan said that Evox’ platform – called DeliverEX – offers both targeted and non-targeted approaches “with the potential to enhance tissue delivery for a variety of payloads.”

He said the deal ties onto Takeda’s efforts to develop cell and gene therapies, including its adeno-associated virus (AAV) platform, which it hopes will “provide transformative therapies or functional cures for people living with rare diseases.”

Takeda dramatically increased its interest in the rare disease area after it bought Shire in a $59 billion deal that closed last year.

Since then it has added to its rare disease portfolio with other licensing deals, including a $850 million partnership with Germany’s Evotec that covers rare diseases as well as cancer, gastroenterology and neuroscience projects.