Phenylketonuria (PKU) is a rare metabolic disease that manifests at birth and is marked by an inability to break down Phe, an amino acid that is commonly found in many foods. High levels of Phe can lead to serious neurological and neuropsychological problems affecting the way a person thinks, feels, and acts. Due to the seriousness of these symptoms, infants in many countries are screened for PKU at birth to ensure early diagnosis and access to treatment that can help reduce the risk of intellectual disability and other complications. To date, Synlogic has determined that SYNB1618 can lower Phe levels in healthy volunteers and patients. The company has initiated a Phase 2 study in PKU patients called SynPheny-1 (NCT04534842) to assess the ability of two drug candidates, SYNB1618 and SYNB1934, to decrease Phe levels in the blood. Learn more about the SynPheny-1 trial is available at www.pkuresearchstudy.com.

Homocystinuria (HCU) is a rare metabolic disease that is caused by an inborn error of metabolism, specifically the loss of function of the enzyme cystathionine beta-synthase, which results in excessive accumulation of homocysteine and its metabolites in the blood and urine. People with HCU face risks of complications including thromboembolism, lens dislocation, skeletal abnormalities, developmental delay, and intellectual disability. Many patients are required to comply with a rigid methionine-restricted diet and currently approved treatment options for HCU are limited due to efficacy and tolerability. In November 2021, Synlogic announced SYNB1353, an orally-administered, non-systemically absorbed drug candidate currently in IND-enabling studies and developed as part of a research collaboration with Synlogic and Gingko Bioworks. Synlogic holds worldwide development and commercialization rights to SYNB1353, which is expected to begin clinical development and report Phase 1 data in healthy volunteers in 2022.

Enteric hyperoxaluria is a chronic, progressive disease characterized by high levels of urinary oxalate, the leading cause of recurrent kidney stones. Oxalate crystals can damage kidneys, potentially leading to damage that can include nephrocalcinosis, chronic kidney disease (CKD) and end-stage renal disease (ESRD). Enteric hyperoxaluria often occurs as a result of a primary insult to the bowel, such as inflammatory bowel disease or short bowel syndrome, or as a result of surgical procedures such as Roux-en-Y bariatric weight-loss surgery. There are no approved treatment options. SYNB88802 is a drug candidate designed to consume dietary oxalate throughout the GI tract. In a Phase 1 AB clinical trial, SYNB8802 achieved proof of mechanism in healthy volunteers during Part A of the study. The company has initiated Part B of the study to demonstrate proof of concept for lowering of urinary oxalate in patients following Roux-en-Y gastric bypass surgery.

Synlogic is also advancing research to assess the ability of novel Synthetic Biotic medicines to treat inflammatory bowel disease.