As the expression goes, “the journey is more important than the destination.” For Global Project Leader Neil Smith, PharmD, MBA, both are equally important, as improving the lives of other people is the ultimate destination. Smith has kept this end goal in mind throughout his journey working on PTC923 – an oral therapy in development for the potential involvement in the metabolism and synthesis of numerous metabolic products.
Originally trained as a pharmacist, Smith has worked for more than two decades in the pharmaceutical industry, stepping into various roles at both large pharmaceutical companies and small biotech firms, and building a diverse skill set along the way.
Part of Smith’s journey has included working on PTC923 since the earliest stages of its development and optimization, even before he or the compound eventually ended up at PTC Therapeutics.
PTC923 is being developed to treat hyperphenylalaninemia – a rare genetic condition that occurs when the amino acid phenylalanine (also referred to as “Phe”), a protein building block, rises to dangerous levels in the body. Hyperphenylalaninemia is often the result of a deficient enzyme, phenylalanine hydroxylase, that converts Phe to tyrosine, another protein building block. People living with hyperphenylalaninemia due to a deficiency in phenylalanine hydroxylase or phenylketonuria (PKU) are unable to break down the Phe found in high protein foods such as dairy products, meat, fish, chicken, eggs, beans, and nuts.
I was always fascinated with clinical studies and loved seeing the results to determine whether the drug being tested, PTC923 for example, was benefiting the patient. It’s really important to me to improve the lives of other people.
Improving the lives of people living with PKU has been a driving force behind Smith’s work. But the road hasn’t always been smooth – as with any rare disease, finding patients to participate in clinical trials can be difficult. Recruiting for the PTC923 trial is no exception.
“Yes, finding patients is always challenging for rare diseases, but you really can see the benefit,” he said. “It’s why I do this work.”
For a scientist who has dedicated years of his career to PTC923, remaining optimistic has led him to a major milestone, and incredible achievement, on the journey: initiation of the Phase 3 clinical trial studying PTC923 in PKU patients. And this new beginning is more than just a mile-marker. It’s a route that leads to Smith’s ultimate destination of seeking to improve the lives of those with PKU.