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: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 hemoglobin, the protein in red blood cells that carries oxygen. The abnormality causes red blood cells to change from their normal rounded-disc shape into crescent, or sickle, shapes. The rigid, sickle-shaped cells can get backed up in tiny capillaries in the body, leading to extreme pain and damage to organs. The disease is most prevalent in people with sub-Saharan African, Indus, Saudi Arabian, and Mediterranean heritage, and the average person with the disease dies in their 40s, according to AesRx. The firm’s drug is designed to improve the ability of the abnormal hemoglobin in sickle cells to carry oxygen, enabling the diseased cells to maintain their normal round shape, according to Seiler.
Still, it’s difficult to raise venture capital nowadays, especially for product-focused companies like AesRx that build their businesses around licensing or acquiring products to advance to the market rather than developing a scientific platform that could yield multiple product opportunities, such as RNA-interference. Seiler acknowledges that product-focused companies aren’t in vogue at the moment, but he also noted that they could come back into fashion with venture investors as well. Seiler definitely has experience in raising money for biotech firms with his experience as CEO of Idera, Effective Pharmaceuticals, and Access Pharmaceuticals. He was also senior vice president of business development for Irish drug maker Elan in the 1990s.
Seiler’s started strong in building AesRx’s leadership team. The headliner on his board of directors is Maggie LeFlore, a managing director of MedImmune Ventures, a venture fund owned by London drug giant AstraZeneca. Yet MedImmune Ventures is not an investor in AesRx because sickle cell disease is not a condition in which AstraZeneca has a strategic interest, LeFlore told me in an e-mail.
AesRx also isn’t a one-trick pony; it got a second drug candidate in its pipeline dubbed Aes-210 for a chronic bowel disease called ulcerative colitis. But the main focus of the company is still to get the sickle cell drug into clinical trials, Seiler said.
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