Codexis Announces Results of Phase 1a Clinical Trial

Codexis, Inc. recently announced top-line results from its Phase 1a single ascending-dose study in healthy volunteers with CDX-6114, its orally administered enzyme candidate for the potential treatment of the metabolic disorder phenylketonuria (PKU). All of the defined study endpoints were met.

The randomized, double-blind, placebo-controlled Phase 1a study evaluated the safety and tolerability of CDX-6114 in 32 healthy volunteers evenly divided into four cohorts. Each cohort was administered a single dose of CDX-6114 at increasingly higher dose levels. CDX-6114 was well tolerated at all four dose levels with no serious adverse events (SAEs) or GI-related symptoms observed. In addition, a dose-dependent pharmacodynamic response was observed, and an initial readout on pharmacokinetics was obtained with no evidence of systemic exposure.

“We have reached a key milestone for the company with the completion of our first ever clinical study on a novel biotherapeutic candidate developed using our CodeEvolver protein engineering platform,” said Codexis President and CEO John Nicols. “We are encouraged by the outcomes of this trial which, in addition to demonstrating safety and tolerability across the entire dose range, generated valuable pharmacology data to support and guide the continuing clinical development of this biotherapeutic candidate.”

PKU is an inborn metabolic disorder resulting from a mutation in the gene for the enzyme that converts the essential amino acid phenylalanine, 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, life-long diet that is low in phenylalanine and supplement their diet with a synthetic phenylalanine-free formula to provide sufficient nutrients. Maintaining a strict, life-long diet is a challenge for individuals with PKU. There are an estimated 50,000 people with PKU in the developed world.

Codexis is a leading protein engineering company that applies its proprietary CodeEvolver technology to develop proteins for a variety of applications, including as biocatalysts for the commercial manufacture of pharmaceuticals, fine chemicals and industrial enzymes, and enzymes as biotherapeutics and for use in molecular diagnostics. Codexis’ proven technology enables improvements in protein performance, meeting customer needs for rapid, cost-effective and sustainable manufacturing in multiple commercial-scale implementations of biocatalytic processes. For more information, visit www.codexis.com.