Evox Therapeutics Signs Multi-Target Rare Disease Collaboration with Takeda

Rare Daily Staff

Evox Therapeutics has entered into a rare disease-focused partnership with Takeda Pharmaceutical focused on developing up to five novel protein replacement and mRNA therapies for rare diseases.

The collaboration focuses on targeted delivery of these therapies using Evox’s proprietary exosome technology and encompasses up to five rare disease targets, including Evox’s preclinical program in Niemann-Pick disease type C (NPC) and a second new program 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.

The partnership with Takeda enables Evox to continue advancing its proprietary exosome-based targeting and delivery technology, and its own pipeline of rare disease drugs, including a urea cycle disorder program, while also leveraging Takeda’s extensive development and clinical expertise to advance these partnered programs into the clinic.

Under their deal valued at up to $882 million, Evox will be eligible to receive up to $44 million in upfront, near-term milestone payments and research funding, and up to $838 million in development, and commercial milestone payments from Takeda, plus royalties on net sales of each product.

Evox will be primarily responsible for research and development activities for each program until IND-enabling studies and for manufacturing up to and including phase 1 clinical trials. Takeda will reimburse Evox for manufacturing costs incurred after the pre-clinical handover of the programs for clinical development and commercialization.

“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,” said Madhu Natarajan, head of the Rare Diseases Drug Discovery Unit at Takeda.

Photo: Madhu Natarajan, head of the Rare Diseases Drug Discovery Unit at Takeda