Stephen Seiler has been hunting for cash to advance an experimental therapy for sickle cell disease into initial human studies. And the veteran biotech executive, the former CEO of Cambridge, MA-based Idera Pharmaceuticals (NASDAQ:[[ticker:IDRA]]), has founded a startup in Newton, MA called AesRx to carry out his goal.
Seiler said he has provided an undisclosed amount of money from his own pocket to support the young startup, which was formed in November 2008. Now he’s looking to raise about $10 million in a Series A round of venture capital to advance the startup’s lead drug, called Aes-103, into a Phase I clinical trial next year for sickle cell disease. He’s making the case that the current treatments for sickle cell disease (sometimes called sickle cell anemia), which affects about 75,000 Americans, have left patients with far too few options to treat the genetic, lifelong illness that causes pain, infections, organ damage, and can often be deadly.
The firm’s drug is a small molecule called 5-hydroxymethyl-2-furfural, or 5HMF, which is derived from sugar and can be found in everything from caramel and cookies to prune juice. Based on preclinical studies, according to Seiler, there is evidence that the drug could be less toxic than previous treatments for sickle cell disease. For example, the standard treatment for the disease, called hyroxyurea, is typically prescribed only when patients are experiencing pain and other complications of sickle cell because it can cause side effects such as vomiting and lowered red blood cell production. And while bone marrow transplants have been able to cure the illness, there are challenges to making this therapy available to most patients because a matching donor is needed and it’s too expensive for them.
“Although it’s still pre-clinical, we know a lot about this drug,” Seiler said. “Therefore we believe our odds of success are better than a random pre-clinical compound.”
For now, the company is supporting some of its claims about the potential utility of its treatment with outside, peer-reviewed studies that validate the importance of the target that AesRx’s treatment binds with, as well as the drug’s intended mechanism of action. The compound was also studied extensively by researchers at Virginia Commonwealth University who first discovered its potential to treat sickle cell disease. (Seiler purchased the experimental drug after its former owner, New Jersey-based Xechem International, filed for Chapter 11 bankruptcy protection in fall 2008, he said.)
Sickle cell disease is caused by abnormal
