Development of Innovative Treatment Agents for Phenylketonuria and New Diagnostic Biomarkers

We recently provided a patented mechanistic explanation and a novel therapeutic target for phenylketonuria (PKU), one of the most common among the rare inborn error of metabolism (IEM) disorders with a continuously expending market. Drugs to be developed in this path could be disease-modifying. And have the tremendous potential to help a wide range of PKU patients in the future.

Currently, no disease-modifying course of treatment is available, with the mainstay of treatment being a very strict low-phenylalanine diet. Kuvan®, today’s gold standard treatment forPKU, with annual revenues of $400-$500 million, can reduce blood levels of phenylalanine in only 30% of patients, while most patients have little or no benefit. Altogether, the development of a new therapeutic agent is required and has a high commercial potential
Our new state-of-the-art paradigm shifting concept, introducing the presence of amyloid features as a new therapeutic target in PKU, a target that was verified both in-vitroand in-vivo, may lead to a new innovative course of treatment. We have identified efficient phenylalanine self-assembly inhibitors, which resulted in the discovery of a lead compound. We successfully demonstrate the ability of the lead compound treatment to statistically improve cognitive function in PKU mice model. Moreover, we set to develop new and more accurate diagnostic tools to be used in combination of our proposed treatment
Today, the main effort is reduction of phenylalanine concentration rather than avoiding its aggregation into toxic species. We provide a completely new solution that can allow phenylalanine presence in the blood but with no adverse effect. Thus, we provide a new target, which will further lead to the development of a new therapeutic agent(s) for the treatment of PKU
New disease-modifying drug agents for the treatment of PKU

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