Rare Daily Staff

Nestlé Health Science has exercised its option to obtain an exclusive license for the global development and commercialization of Codexis’ orally delivered enzyme CDX-6114 for the management of phenylketonuria, a rare metabolic disorder.

The exercise of the option triggers a $3 million milestone payment. Upon exercising its option, Nestlé Health Science has now assumed all responsibility for future clinical development and commercialization of CDX-6114. Codexis originally granted Nestlé an option under agreement announced in October 2017. 

Phenylketonuria (PKU) is an inborn metabolic disorder resulting from a mutation in the gene for the enzyme that converts the essential amino acid phenylalanine, which is present in almost all dietary protein, into tyrosine. As a result of this deficiency, phenylalanine builds up to levels that are toxic in the brain, causing serious neurological symptoms including intellectual disability, seizures, and cognitive and behavioral disabilities.

To avoid phenylalanine toxicity and the most severe disease symptoms, individuals with PKU must follow a strict, lifelong diet that is low in phenylalanine and supplement their diet with a synthetic phenylalanine-free formula to provide sufficient nutrients. Maintaining a strict, lifelong diet is a challenge for individuals with PKU.

CDX-6114 is Codexis’ first internally developed biotherapeutic product candidate.

“We are proud to have discovered an enzyme that shows promise as a novel treatment for patients with PKU and are delighted that Nestlé Health Science has elected to take on its continued development,” said Codexis President and CEO John Nicols. “The exercise of this option further validates our strategy to partner biotherapeutics discovered using our CodeEvolver protein engineering platform, and to do so early in the process of clinical development.”  

Photo: John Nicols, president and CEO of Codexis