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the pediatric setting,” he says. The FDA has granted the drug “orphan” status, which may help speed its path through the regulatory process.
Nevertheless, that process won’t be an easy one for AesRx. Sickle cell disease is an unpopular target for drugmakers because the FDA has traditionally set too high a bar for the endpoints they must prove in clinical trials, Seiler says. For example, the agency has traditionally expected drugmakers to show that their compounds prevent hospitalizations for severe episodes of pain. “That’s not appropriate because many of these patients are now treated on an outpatient basis,” meaning they never check into the hospital, Seiler says. “The treatment modality is disappearing, so the endpoint should be disappearing, too.” He says AesRx is working with the FDA to establish more reasonable endpoints for the Aes-103 trials.
If all goes well, AesRx could complete Phase 1 and 2 trials by the end of next year and start Phase 3 trials in the first quarter of 2013, Seiler says. He hopes that a Big Pharma company will show some interest in partnering with AesRx to fund the later-stage trials. Seiler was particularly encouraged on October 11, when drug giant Pfizer (NYSE: PFE) formed a $340 million development deal with GlycoMimetics, a Gaithersburg, MD-based company that’s in Phase 2 trials of a drug to treat “vaso-occlusive crisis,” the episodes of extreme pain that sickle cell can cause. “Over the last two years there’s been a huge amount of interest from companies like Pfizer in orphan diseases,” Seiler says.
For now, Seiler says, AesRx has enough money to make it through 2012. At that point, he says, the company will need to “look at the next step.” After all, he knows he can’t rely on government funding forever. “The goal of the NIH is to take you across the Valley of Death,” he says, “and no further.”
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