Regeneron's chief scientist spotlights potential blockbuster allergy drug

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Dr. George Yancopoulos

Regeneron Pharmaceuticals ($REGN) has highlighted clinical evidence of another therapy in the biotech company's venerable drug pipeline with prospects for more than $1 billion in annual sales. With one likely blockbuster on the market in Eylea and at least three others in development, Regeneron Chief Scientific Officer Dr. George Yancopoulos has plenty to keep him busy for the next several years.

He's got a fortune at stake too. Last June, Tarrytown, NY-based Regeneron's board gave Yancopoulos a stock award valued at nearly $57.3 million, making him the most highly compensated biopharma executive in the world with a total pay package of $81.6 million in 2012. The 500,000 share award vests 5 years from the granting date, meaning that Yancoupulos needs to stick around until at least June 2017 to collect the massive sum.

Dupilumab offers one of the latest examples of why Yancopoulos is the highest paid scientist in biotech. Discovered with antibody technology he co-invented, dupilumab showed some impressive signs of efficacy in a Phase IIa study of patients with allergic asthma whose respiratory ailment is poorly controlled with inhaled glucocorticosteroids and long-acting beta agonist (LABA) therapy.

"I think we have as many exciting programs as anybody in the industry," Yancopoulos said in an interview with FierceBiotech, "but I think this is characteristic of several of our programs. This could be a true blockbuster that really provides a game-changing therapy for a lot of patients with a lot of need."

As reported online today in the New England Journal of Medicine, asthma patients on Regeneron's therapy had an 87% reduction in the incidence of asthma exacerbations compared with those in the placebo arm. The 104-patient study provided evidence of improved lung function in patients on weekly injections of dupilumab. There were adverse events in 76.9% of those on placebo versus 80.8% in patients taking dupilumab, which was linked with headaches, nausea, injection site reactions and other side effects.

Dupilumab (REGN668) is one of several promising therapies in a massive collaboration between Regeneron and French drug giant Sanofi ($SNY). The companies plan to advance the therapy into a 500-patient Phase II study in allergic asthma patients next month, seeking evidence of its safety and efficacy in a much larger group of patients, according to records from ClinicalTrials.gov. The therapy has also yielded promising evidence in patients with atopic dermatitis, which appears to share similar molecular triggers with allergic asthma.

Sanofi and Regeneron have also joined forces on Phase III efforts to advance alirocumab (REGN727), an LDL-cholesterol-lowering drug that targets PCSK9, and sarilumab (REGN88) for rheumatoid arthritis.

"This is why we all go into science in the first place," Yancopoulos said. "We're hoping to understand and define a fundamental driver of disease, or in this particular case a widespread disease epidemic, and then show that impacting this fundamental driver can have incredible benefits for patients."

Dupilumab is designed to target the interleukin 4 receptor, stymieing key drivers in the Type 2 helper T cell immune response and inflammation implicated in multiple allergies, including allergic asthma and atopic dermatitis. The Th2 inflammation pathway plays a role in about half of patients with moderate to severe asthma, according to Regeneron. An estimated 25 million Americans have asthma. And atopic dermatitis affects about 7% of the population, according to the National Institutes of Health.

Of course, Regeneron and Sanofi have a lot to prove before dupilumab is crowned a blockbuster. Yet Regeneron has already proven the commercial value of its antibody technology with approvals of three drugs, including the macular degeneration therapy Eylea, which took off last year with $838 million in sales and is projected to top $1 billion in sales this year. Bayer controls sales of the eye therapy outside the U.S., where Regeneron has kept exclusive rights.

Success in biotech research is expensive. Regeneron pumped $625 million into research in 2012, a whopping 72% of its product revenue and an increase of 18% over the prior year. Yancopoulos, who joined Regeneron as founding scientist in 1989, says that his group's success and the big investments in R&D work hand in hand. 

"We believe that if you don't invest in biology, and that if you're not driven by biology, science and technology, then you're never really going to be successful in this business," Yancopoulos said. "The reason we are successful is because that's what we focus on." -- Ryan McBride (email | Twitter)