Evox and Takeda sign deal to collaborate on rare disease treatments

In an $882 million deal, Evox and Takeda will partner to develop and manufacture treatments and drug delivery systems for rare diseases.


Evox Therapeutics has announced it has signed a multi-target rare disease collaboration deal with Takeda. 

Under the terms of the agreement, Evox will be eligible to receive up to $44 million in upfront, near-term milestone payments and research funding. In total, Evox is eligible to receive approximately $882 million in upfront, development and commercial milestone payments from Takeda. Evox will also receive tiered royalties on net sales of each product.

The partnership will focus on developing up to five novel protein replacement and mRNA therapies as well as exploring the targeted delivery of these payloads, including Evox’s pre-clinical programme in Niemann-Pick disease type C (NPC) and a second new programme directed at another undisclosed rare disease. As part of the deal, Takeda also has the option to select up to three additional rare disease targets.

Dr Antonin de Fougerolles, Chief Executive Officer of Evox, commented: “We are delighted to have entered into this strategically important, multi-target partnership with Takeda, a recognised leader in the development of treatments for rare diseases. We look forward to working with Takeda to advance these exosome drugs towards the clinic. Additionally, the deal significantly extends our cash runway into late 2022 and allows us to aggressively expand our own proprietary pipeline of rare disease drugs, including a urea cycle disorder programme we expect to enter the clinic in 2021.”

The partnership will focus on developing up to five novel protein replacement and mRNA therapies”

Madhu Natarajan, Head of the Rare Diseases Drug Discovery Unit at Takeda, commented: “Evox Therapeutics has developed a novel approach toward treating devastating diseases, such as Niemann-Pick Type C. The targeted and non-targeted exosomes offer a highly differentiated platform with the potential to enhance tissue delivery for a variety of payloads like mRNA and proteins. Collaborating on the Evox exosome platform also complements our expanding capabilities in cell and gene therapies, particularly with the potential to develop new delivery approaches in addition to our cutting-edge adeno-associated virus (AAV) platform, to provide transformative therapies or functional cures for people living with rare diseases.”

Evox will be primarily responsible for R&D activities for each programme until Investigational New Drug (IND)-enabling studies and for manufacturing up to and including Phase I clinical trials. Takeda will reimburse Evox for manufacturing costs incurred after the pre-clinical handover of the programmes.