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$4.5 million to develop glutamate-regulating proteins to improve symptoms of Fragile X, a genetic condition that commonly causes autism. On September 15, Vanderbilt announced it had reached a milestone in that partnership and that drugs could be ready for human testing early next year.
Vanderbilt also has a partnership with the Michael J. Fox Foundation for Parkinson’s Research to develop molecules that act on a specific glutamate receptor. Conn says Vanderbilt is currently in discussions with companies about licensing the Parkinson’s compounds and will “probably announce a partnership by the end of the year.”
All three molecules could be multibillion-dollar opportunities, but the market potential in schizophrenia is particularly large. Sales of Astra Zeneca’s (NYSE: AZN) quetiapine (Seroquel) exceeded $3 billion last year, while Eli Lilly’s (NYSE: LLY) Olanzapine (Zyprexa) brought in $5 billion in revenues. And other companies are looking at the GlyT1 approach, including Roche and Merck (NYSE: MRK).
Harrigan says Karuna was attracted to Vanderbilt’s compounds because they show strong affinity for their targets in the brain and they modulate GlyT1 rather than shutting it down. “We believe that modulation is important to avoid toxicity,” Harrigan says. He adds that the company hopes to bring a drug into clinical trials within two years.
If the Karuna program yields an effective schizophrenia treatment, Conn says, it will bring to full-circle research that started in the 1950s. Back then, scientists noticed that drug abusers on PCP—otherwise known as angel dust—exhibited symptoms similar to schizophrenia. PCP also manipulates the glutamate system, Conn says. “But what we’re trying to do is the opposite of what angel dust does.”
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