Phase 3 Trial Testing Probiotic to Treat Phenylketonuria (PKU) Is Enrolling Patients

Описание: Cary Harding, MD, Professor of Molecular and Medical Genetics at Oregon Health & Science University (OHSU), highlights the data evaluating SYNB1984 to treat phenylketonuria (PKU).

PKU is a rare metabolic disease caused by uncontrollable levels of the amino acid phenylalanine (Phe), which can lead to neurocognitive deficits. Despite two approved treatments, most patients with PKU are either unresponsive to current therapies or these options present unacceptable safety risks such as anaphylaxis. Given that Phe is found in all sources of natural protein, most patients must adhere to a very strict, 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. It is engineered to break down the Phe in the gut, thereby allowing the person with PKU to eat protein.

A new probiotic therapy, SYNB1934, is in development that made remove the need for patients to adhere to that strict diet.

Based on promising data from Phase 1 and Phase 2 studies, a Phase 3 clinical trial will begin and is expected 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 (3x1011, 6x1011, and 1x1012), 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.