Positive Results for Possible New PKU Oral Treatment

According to BioPortfolio, there was recently an announcement about preclinical data for SYNB1618, which is a Synthetic Biotic treatment that is being assessed right now in a Phase 1/2a clinical trial in patients with PKU. The results from the study apparently show that an oral delivery of SYNB1618 led to lower blood Phe levels in trial participants.

Phenylketonuria, abbreviated as PKU, is a rare genetic condition that results from a buildup of the amino acid called phenylalanine. The buildup of this amino acid can lead to serious health problems, and since amino acids are found in proteins, it is advised that those who are diagnosed with PKU stay away from high protein foods that may further exacerbate the problem. Symptoms of PKU include seizures, intellectual delays, developmental delays, heart defects, and decreased bone strength. Early detection of PKU is crucial to helping alleviate symptoms and treatments mainly focus on dietary changes. To read more about the condition, click here.

Phe is the abbreviation for phenylalanine, which is the amino acid that is in excess in those who have PKU. Therefore, the lower levels of Phe as reported in the study means the oral SYNB1618 treatment may be effective.

Those involved in the study are hopeful for the implications this research will have on PKU patients. This includes Synlogic’s interim president and chief executive officer, Aoife Brennan, who says, “For patients with PKU, life-long disease management can be challenging due to the protein-restricted diet required to accompany currently available treatments. We are encouraged by the preclinical data presented at ASM Microbe, which support the development of orally administered SYNB1618 as a potentially broadly applicable therapeutic option for patients.”

The chemistry behind SYNB1618 comes from the fact that the medicine contains a strain of probiotic bacterium called E.coli Nissle. This bacterium has been manipulated to metabolize Phe.

The findings from mouse models of PKU show that oral SYNB1618 leads to notable reduction in blood Phe levels, which is the goal for many who live with the disease.

There is more to come with regards to this research in the latter half of 2018, however.

“We are currently exploring production of these same biomarkers in an ongoing Phase 1/2a clinical trial in healthy volunteers and patients with PKU, and look forward to reporting interim safety data from the dose-escalation portion of this trial in the second half of 2018,” said Brennan.

For those who are a part of the PKU community, stay tuned for how this research pans out. To read more about it, click here.

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