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Codexis Presents Preclinical Data Highlighting Its Homocystinuria and Maple Syrup Disease Programs at ICIEM 2021


REDWOOD CITY, Calif., Nov. 22, 2021 (GLOBE NEWSWIRE) – Codexis, Inc. (NASDAQ: CDXS), a leading enzyme engineering company enabling the promise of synthetic biology, today announced that two of the biotherapeutic programs in Company-owned CDX-6512 for the treatment of homocystinuria (HCU) and enzymes for the treatment of maple syrup urinary disease (MSUD) were presented orally at the 14th International Congress of Innate Errors of Metabolism (ICIEM) 2021 held in Sydney, Australia from November 21 to 24, 2021. Candidate enzymes are stable gastrointestinal (GI) enzymes specially designed to be highly resistant to both acidic conditions of the stomach and proteases of the upper intestines, in order to effectively degrade methionine and leucine which are released from protein digestion. High levels of these amino acids and their metabolites can lead to a variety of clinical manifestations of HCU and MSUD respectively.

“Our modified enzymes have demonstrated the potential to be first-rate oral enzyme treatments for patients with homocystinuria and maple syrup urinary disease, who currently have limited treatment options and must adhere to a restricted diet. protein throughout their life, ”said John Nicols, President and CEO of Codexis. “As we have demonstrated with our Phase 1 programs in Phenylketonuria and Pancreatic Exocrine Insufficiency, and now through our HCU and MSUD programs, food component degradation enzymes designed to be stable under gastrointestinal conditions. -intestinal have exciting potential as treatments for inborn errors of metabolism and gastrointestinal disorders. We look forward to leading the clinical development of these programs and continuing to push the boundaries of what our enzymes can do to meet unmet patient needs.

Kristen Skvorak, PhD, Translational Researcher and Patient Ambassador at Codexis, presented the “Discovery of CDX-6512, a stable gastrointestinal methionine-gamma-lyase as a potential oral enzyme therapy for homocystinuria”, which demonstrated up to 45% suppression in serum total homocysteine ​​(p

Dr Skvorak also presented the “Discovery of Gastrointestinal Stable Bacterial Leucine Decarboxylase as a Potential Orally Administered Enzyme Therapy for Urine Disease Due to Maple Syrup”, which demonstrated up to 45% suppression of leucine levels in the blood, measured as an incremental area under the curve. (iAUC), in whey-fed iMSUD mice with a single dose of leucine decarboxylase versus vehicle (p

About homocystinuria (HCU)

Homocystinuria is a rare inborn error of metabolism, most commonly due to cystathionine beta-synthase (CBS) deficiency and is characterized by high levels of homocysteine ​​in the blood and urine which when not left untreated, can lead to learning and intellectual disabilities, cardiovascular disease, osteoporosis and stroke. Homocysteine ​​is a metabolite derived from methionine, an essential amino acid that enters the body as part of dietary protein. Strict, lifelong adherence to a low methionine diet, often combined with vitamin supplementation (eg, pyridoxine, folic acid, vitamin B12, betaine), is currently the only treatment available. According to the Center for Information on Genetic and Rare Disorders (GARD), it is believed that approximately 1 in 150,000 people worldwide have HCU due to a mutation in the CBS or MTHFR gene. HCU is listed on the Uniform Recommended Screening Panel for disorders recommended by the Secretary of the Department of Health and Human Services for states to be screened as part of their universal newborn screening programs.

About maple syrup disease (MSUD)

Maple syrup disease is an inborn error in branched-chain amino acid metabolism and is characterized by a build-up of leucine and its metabolites in the brain, blood, and urine that can lead to intellectual and developmental disabilities. A diet low in leucine throughout life can promote good development and prevent serious consequences, but compliance remains a significant challenge. Orthotopic liver transplantation is an effective, but highly invasive and risky experimental therapy. Episodes of metabolic crisis require prompt medical interventions, such as dialysis, and are not intended for long-term treatment. According to the Genetic and Rare Disorders Information Center (GARD), about 1 in 150,000 to 185,000 people are born with MSUD. MSUD is listed on the Uniform Recommended Screening Panel for disorders recommended by the Secretary of the Department of Health and Human Services for states to be screened as part of their universal newborn screening programs.

About Codexis

Codexis is a leading enzyme engineering company that leverages its proprietary CodeEvolver® platform to discover and develop new high performance enzymes and new biotherapeutics. Codexis enzymes have applications in the sustainable manufacture of pharmaceutical, food and industrial products; in creating the next generation of life science tools; and as gene therapy and biological therapy. The Company’s unique performance enzymes drive improvements such as: reduced energy consumption, waste generation and capital requirements; higher returns; more accurate diagnoses; and more effective therapies. Codexis enzymes hold the promise of synthetic biology to improve the health of people and the planet. For more information, visit www.codexis.com.

Forward-looking statements

To the extent that the statements contained in this press release are not descriptions of historical fact concerning Codexis, they are forward-looking statements reflecting the current views and expectations of management in accordance with the Safe Harbor of Private Securities provisions. Litigation Reform Act of 1995. You should not place undue reliance on these forward-looking statements as they involve known and unknown risks, uncertainties and other factors which are, in some cases, beyond the control of Codexis and which could materially affect actual results. Factors that could materially affect actual results include, but are not limited to: our biotherapeutic programs are early stage, highly regulated and expensive; our ability to secure additional program development partners, advance our product candidates into clinical trials and ultimately receive regulatory approvals is highly uncertain; the regulatory approval processes of the U.S. Food and Drug Administration and comparable foreign authorities are lengthy, time consuming and inherently unpredictable, and so we are unable to obtain or maintain regulatory approval for our products and product candidates, our business will be significantly affected; the results of preclinical studies and early clinical trials of product candidates may not be predictive of the results of subsequent studies or trials; our product candidates may not perform favorably in subsequent clinical trials, if any, or may not receive regulatory approval; if any of our product candidates do not perform as expected or cause unwanted side effects, this could impede or prevent obtaining regulatory approval or the realization of commercial potential for them or our other product candidates and could significantly affect our business; and even if we obtain regulatory approval for any product that we develop alone or with collaborators, those products will remain subject to ongoing regulatory requirements, which may result in significant additional expenses. Additional information on factors that could materially affect actual results can be found in Codexis’ Annual Report on Form 10-K filed with the Securities and Exchange Commission (“SEC”) on March 1, 2021, and in the Quarterly Report Codexis on Form 10 -Q filed with the SEC on November 5, 2021, including under “Risk Factors” and in other Codexis periodic reports filed with the SEC. Codexis expressly disclaims any intention or obligation to update these forward-looking statements, except as required by law.

Investor Relations Contact:
Argot Partners
Stephanie Marks / Carrie McKim
[email protected]
(212) 600-1902