Japan’s Takeda Pharmaceutical is partnering with UK biotech Evox Therapeutics to develop treatments for rare diseases. Financial terms of the agreement have not been disclosed.

The collaboration enables Evox to continue advancing its proprietary exosome-based targeting and delivery technology. The Oxford-based company is focused on harnessing and engineering the natural delivery capabilities of extracellular vesicles, known as exosomes, to develop an entirely new class of therapeutics for various severe diseases where patients have limited options.

“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,” said Madhu Natarajan, head of Takeda’s rare diseases drug discovery unit.

“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 platform, to provide transformative therapies or functional cures for people living with rare diseases," Natarajan said.

The companies will develop up to five novel protein replacement and mRNA therapies, including Evox’s preclinical program in Niemann-Pick disease type C (NPC) and a second program directed at another undisclosed rare disease.  NPC is a progressive genetic disorder that affects the body’s ability to metabolize cholesterol and lipids within cells, subsequently leading to a loss of nerve, brain and organ function.

Takeda also has the option to select up to three additional rare disease targets.

Under the terms of the agreement, Evox is eligible to receive a total of about $882 million in upfront, development and commercial milestone payments. It will also receive tiered royalties on net sales of each product.

Antonin de Fougerolles, CEO of Evox, added that the deal also significantly extends its cash runway into late 2022 and allows the company to aggressively expand its proprietary pipeline of rare disease drugs, including a urea cycle disorder program that it expects to enter the clinic in 2021.

In separate news, Takeda has signed a deal with US protein engineering company Codexis to develop novel gene therapies for certain disease indications, including the treatment of lysosomal storage disorders and blood factor deficiencies.

Using its CodeEvolver platform, Codexis will generate novel gene sequences encoding protein variants that are tailored to enhance efficacy. Takeda will combine these improved transgenes with its own gene therapy capabilities to generate novel candidates for rare genetic disorders.

 “Our CodeEvolver platform technology enables the rapid engineering of novel genetic sequences that encode more efficacious proteins. The prospects of these improved sequences for the development of differentiated gene therapies for patients with rare diseases therefore holds great promise,” said John Nicols, president and CEO of Codexis.

The parties will initially start working on three programs but Takeda may initiate up to another four programs for separate target indications.