SYNB1934 for Phenylketonuria

(SYNPHENY-3 Trial)

This trial tests a new treatment called SYNB1934 for individuals with phenylketonuria (PKU), a condition where the body cannot break down the amino acid phenylalanine, leading to harmful blood levels. The trial aims to determine if SYNB1934 can manage these levels more effectively than current treatments. Individuals with PKU who struggle to control their blood phenylalanine levels, despite a strict diet and existing medications, might be suitable candidates for this trial. Participants will take SYNB1934 with meals, and the trial includes several phases to monitor effects and any side effects over time. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment for PKU.

The trial requires that you continue your current diet, sapropterin, sepiapterin, and large neutral amino acids without changes during the study. However, you cannot take Palynziq® (pegvaliase-pqpz) within 1 month of screening or any systemic antibiotics within 28 days prior to the first dose.

Research has shown that SYNB1934 has generally been well-tolerated in past studies. For example, one study found that participants taking SYNB1934 experienced significant reductions in phenylalanine levels, which is crucial for treating phenylketonuria (PKU). Although the study did not provide detailed safety information, the progression to later trial stages suggests a reasonable safety profile so far.

SYNB1934 is unique because it’s a living medicine specifically designed for phenylketonuria (PKU), a condition where the body can’t properly break down the amino acid phenylalanine. Unlike traditional treatments like dietary management or enzyme replacement therapies, SYNB1934 uses live bacteria to naturally consume phenylalanine in the gut before it enters the bloodstream. This innovative approach could offer more flexibility and effectiveness in managing PKU, potentially reducing the strict dietary restrictions currently necessary for patients. Researchers are excited because it represents a new way of tackling PKU by directly addressing phenylalanine levels with a biological approach.

Research has shown that SYNB1934, which participants in this trial may receive, produces promising results in lowering phenylalanine levels in people with phenylketonuria (PKU). In earlier studies, 60% of patients responded well, experiencing a 42% drop in phenylalanine levels. This treatment uses a specially modified type of bacteria to help break down phenylalanine, a problematic amino acid for people with PKU. SYNB1934 has also demonstrated better efficacy than previous treatments like SYNB1618. These findings suggest that SYNB1934 could effectively manage PKU.¹³⁴⁵⁶