Member Spotlight: Q&A with PTC Therapeutics

Jan 02, 2020

Every month, MassBio spotlights a member company and the great work they’re doing to advance the life sciences industry and support the patients we serve. In January, we spoke with Stuart W. Peltz, Ph.D., Founder & CEO of PTC Therapeutics. Under his leadership, PTC has grown from a research organization to a publicly traded, global commercial organization with multiple approved products and a foundation of strong technology platforms that continues to drive a robust discovery pipeline for patients with rare disorders.

Tell us about your organization, its mission, and current initiatives

I founded PTC over 20 years ago with a clear vision of creating an exciting innovative science company to treat patients with rare disorders. It’s important to me that we remain as committed to this vision as when we first started PTC. I often say that we are just as much a cause as a company, with a strong mission to bring new therapies to improve the lives of patients and their families. I am proud that our efforts have led to the first approved product to treat Duchenne muscular dystrophy in the world. In addition to our two therapies for Duchenne, we are commercializing two other rare disorder treatments and are growing a pipeline of potential new therapies, including two products that we anticipate having regulatory approvals for next year. Our pipeline continues to expand with new potential therapies for multiple rare disorders such as AADC deficiency, Friedreich’s Ataxia, and Huntington’s Disease and we continue to explore more scientific platforms and therapeutic areas where we think we can make a difference. We have grown to be a global commercial company with over 700 employees in 20 countries worldwide and can bring therapies to patients in 50 countries. We realize that life is measured by moments; we want to help make sure that patients with rare disorders share in these moments.

How does your organization’s activities help patients now and into the future?

Our cause has always been to provide more of life’s moments to patients and families taking our medicines. Whether it’s days, months, or years, every small moment is a big win. Thus, we work to make patients the center of everything we do at PTC. From the beginning of the company, we have worked closely with patient advocacy groups, patients, caregivers, and physicians to better understand their specific situation, which helps us develop treatments and programs to address the needs of the community. They are part of our solution from the beginning, from early research to commercialization and beyond. We develop programs to try to reduce the burdens on the family. For example, we have a program called PTC Cares™ to help patients, caregivers, and prescribers navigate the prescription process and provide financial assistance for PTC products. Additionally, we also introduced a global grant program called PRIORITY which is designed to provide funding to biomedical researchers and clinicians who are interested in developing unique and collaborative research programs that will improve clinical practice for patients with rare genetic disorders. We also have a grant program for patient advocacy groups to provide funding to help them innovate new ways to help patients with rare disorders. This is the 5th year of the program with 28 organizations over the world who have been awarded grants.

What do you see as the biggest challenge facing the life sciences industry today?

I try and look at challenges in the industry in which patients are affected. One major challenge for us in the rare disorder space continues to be the regulatory path for the ultra-rare patient populations.Patient populations for these disorders can be incredibly small, and the understanding of the natural history, clinical endpoints and the best patient population to study in a rare disorder can be very challenging. This will become a bigger issue as we try to identify potential treatments for smaller and smaller ultra-rare patient populations. In a subset of these indications the patient populations will be too small to be able to execute placebo-controlled trials. Therefore, alternative approaches or regulatory pathways are needed to be able to bring products to patients. This may include a new regulatory framework, perhaps something analogous to the EMA’s exceptional circumstances pathway. We can also look to real-world evidence of a drug’s efficacy to provide clinicians, regulators and payers a true picture of patient response to treatment. We at PTC will continue to work with the rare disorder community and regulators to advocate for a process that could allow for the approval and commercialization of these medicines as quickly and safely as possible. We believe that we can find innovative ways to bring new treatments to rare disorder patients. We are committed to creating a strategy that supports access for current and future populations.

What’s next for your organization / what are you focused on in the coming year?

PTC has been growing rapidly over the last two years and we will continue to grow and expand in the upcoming year. We plan to launch four new treatments over the next year – including our first gene therapy for the treatment of AADC deficiency – by year end. We are accelerating 20 discovery and development programs over the next five years. We will also begin manufacturing our gene therapy products in our own facility in 2020. We are expanding into new scientific platforms and therapeutic areas, including Friedreich’s Ataxia, Angelman Syndrome and Huntington’s Disease. Using our new redox scientific platform, we will also be expanding into a number of mitochondrial epilepsies and neuro-inflammatory diseases. I’m pretty sure this is going to be another exciting year!

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