Genzyme, PTC Therapeutics Form Collaboration on Drug For Genetic Diseases

Genzyme has struck another big drug development partnership, this time with PTC Therapeutics, a privately-held biotech in South Plainfield, NJ. The Cambridge, MA-based biotech (NASDAQ: GENZ), the world’s largest maker of drugs for rare genetic disorders, is paying $100 million in upfront cash, plus milestone payments and royalties for the right to co-develop and co-market PTC124, an experimental drug being tested for Duchenne Muscular Dystrophy.

The deal has big dollar terms. PTC will be eligible for as much as $165 million in milestone payments from Genzyme for success in development, and another $172 million for achieving certain sales goals. The New Jersey firm will retain the right to commercialize the drug in the U.S. and Canada, while Genzyme will market it in all other countries, should it win approval; PTC will collect royalties worth a double-digit percentage of sales from countries where Genzyme sells the drug.

Why all the fuss? PTC124 is a pill with an unusual mechanism designed to block the effects of what are called “nonsense mutations” or single-point alterations in genetic code that prevent patients from making a fully functioning proteins; since these types of mutations are to blame in a number of genetic diseases, the drug could address a very broad market. The drug is currently being tested in a mid-stage clinical trial as a treament for Duchenne muscular dystrophy, and is expected to enter another mid-stage study as a treatment for cystic fibrosis, a genetic disorder that damages the lungs, by the end of the year. About 20,000 children worldwide are born each year with muscular dystrophy and few survive into their 30s, while about 70,000 people worldwide have cystic fibrosis.

“Over the past two decades, Genzyme has successfully developed four therapies for patients with severe genetic diseases. PTC124 is a powerful new approach that holds great potential to help CF and DMD patients, and many others with a variety of devastating diseases,” said Henri A. Termeer, Genzyme’s chairman and chief executive officer, in a statement. “This collaboration is an excellent strategic fit for Genzyme and will be managed within the company’s stated financial guidance.”

Genzyme’s last big deal was announced in January with Carlsbad, CA-based Isis Pharmaceuticals to co-develop mipomersen, a drug for a being tested as a treament for genetically caused form of high cholesterol that may also be used in a mass market. The PTC deal is more about going after lots of niche diseases. The compound “has the potential to address the underlying cause of disease in a subset of patients affected by more than 2,400 rare genetic disorders,” said Claudia Hirawat, PTC’s senior vice president of corporate development in a statement. We’ll dig in some more about how the companies plan to develop this drug and let you know.

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4 responses to “Genzyme, PTC Therapeutics Form Collaboration on Drug For Genetic Diseases”

  1. mario says:

    I want to Know PTC124 side effects.
    Where is it possible to find more news about this therapy ?