Cary Harding, MD, Professor of Molecular and Medical Genetics at Oregon Health & Science University (OHSU), highlights the data evaluating SYNB1934 to treat phenylketonuria (PKU).
Uncontrollable levels of the amino acid phenylalanine (Phe) cause PKU, a rare metabolic disease that can lead to neurocognitive deficits. Most patients with PKU are either unresponsive to the two approved current therapies or face safety risks such as anaphylaxis. Since Phe is present in all sources of natural protein, most patients must rigorously adhere to a protein-free diet
As noted by Dr. Harding, one possible treatment that would allow patients to eat a relatively normal diet is SYNB1934.
SYNB1934 is a genetic engineering probiotic bacteria (E. coli Nissle) that a person can take orally when consuming a meal. The bacteria is engineered to break down the Phe in the gut, enabling individuals with PKU to consume protein.
A new probiotic therapy (SYNB1934) is in development that made remove the need for patients to adhere that strict diet.
Building upon promising data from Phase 1 and Phase 2 studies, a Phase 3 clinical trial will commence, aiming to enroll approximately 150 patients with PKU
The study will consist of three parts: Part 1 will be a run-in period, enabling individualized titration across three potential dose levels (3×1011, 6×1011, and 1×1012), with patients spending a minimum of three weeks at each dose. Part 2 will be a four-week, placebo-controlled, randomized study to assess change in levels of plasma Phe. Part 3 will be an open-label extension that may extend for up to three years.
More information on the Synpheny-3 study is available at pkuresearchstudy.com.
To learn more about PKU and other metabolic conditions, visit checkrare.com/diseases/metabolic-disorders/
