Allozyne, Developer of Multiple Sclerosis Drug in Fewer Shots, Poised to Enter Clinical Trials

Allozyne will find out soon whether it has a disruptive technology for treating multiple sclerosis. The fledgling Seattle biotech company plans to start its first clinical trial within the next six months, which will give it an early glimpse into whether it can do something that giant companies have been unable to do. It plans to develop an MS drug that works better, lasts longer, has fewer side effects, and requires less-frequent injections than the current standard of care.

Allozyne bears watching as one of the “graduates” of the Accelerator, the Seattle-based startup incubator affiliated with Leroy Hood’s Institute for Systems Biology. Almost exactly a year ago, it raised $30 million from MPM Capital, OVP Venture Partners, Amgen Ventures, Arch Venture Partners, and Alexandria Real Estate Equities. While that deal was getting done, the venture capital backers recruited Meenu Chhabra from her no-doubt lucrative job as a top dealmaker at Switzerland-based pharmaceutical giant Novartis to be the founding CEO. Hood himself, MIT biologist Harvey Lodish, and Nobel Laureate K. Barry Sharpless of the The Scripps Research Institute serve as scientific advisers, so this idea seems to have a little more shine to it than your average startup in town.

The concept has major implications for patients. Without getting too deep into the science (more on that later), Allozyne thinks it has discovered an improved treatment for multiple sclerosis. It’s a disease in which the immune system goes haywire, attacking nerve cells, and ultimately robbing patients of their vision, speech, and walking ability. More than 400,000 patients in the U.S. have this disease (including a disproportionate percentage in the Northwest).

The staple drugs for these patients are from a class of drugs called interferon beta products, which tamp down the part of the immune system that attacks nerves. The drugs are marketed as Biogen Idec’s Avonex, Merck KGaA’s Rebif, and Bayer’s Betaseron. These medicines are huge moneymakers, generating more than $3.5 billion a year in annual sales. But they are far from perfect. The drugs cause flu-like symptoms, have to be taken by injection at least once a week, and sometimes as often as daily, and don’t stop the progressive decline patients have to endure.

Allozyne’s technology aims to enable what is known as “pegylation” of an interferon beta drug. That means scientists can attach a polymer that helps the drug remain stable for a longer period of time in the bloodstream to do its job. Instead of weekly injections, Allozyne thinks its candidate can be given every other week, or even once a month, Chhabra says. By avoiding the peaks and valleys in bloodstream concentration between injections, the drug might offer improved effectiveness at reducing brain lesions in MS patients, Chhabra says.

“This can supplant interferon beta,” Chhabra says.

Of course, Allozyne isn’t the only company trying to come up with a more convenient way to treat MS. Cambridge, MA-based Biogen (NASDAQ: [[ticker:BIIB]]) is developing an oral pill called BG-12, Merck KGaA has a tablet called cladribine in the works, and Chhabra’s former employer, Novartis, has another oral pill in development called FTY-720. These drugs are further along in clinical trials, so if they succeed, they could beat Allozyne to the punch with a more convenient MS treatment.

“If the oral drugs in the pipeline fizzle out, there might be a significant market for Allozyne’s product because of its reduced frequency of administration,” says John Richert, executive vice president of research and clinical programs for the National MS Society in New York.

So how is this technology really supposed to work? Allozyne has licensed technology from the lab of Caltech chemists William Goddard and David Tirrell. They discovered a way to essentially snip out a certain amino acid found in the backbone of protein drugs (methionine), and replace it with a genetically modified amino acid that can stick like Velcro to other molecules.

This means that Allozyne should be able to efficiently attach the polymer in exactly the same place on the protein, providing the kind of consistency the FDA likes to see in a product, says Hans van Houte, Allozyne’s vice president of finance and administration.

But if this concept can be proven in MS, then the implications could be profound for other diseases, because the method from Goddard and Tirrell can be applied to attach polymers to any number of genetically engineered drugs to improve their properties, Chhabra says.

Author: Luke Timmerman

Luke is an award-winning journalist specializing in life sciences. He has served as national biotechnology editor for Xconomy and national biotechnology reporter for Bloomberg News. Luke got started covering life sciences at The Seattle Times, where he was the lead reporter on an investigation of doctors who leaked confidential information about clinical trials to investors. The story won the Scripps Howard National Journalism Award and several other national prizes. Luke holds a bachelor’s degree in journalism from the University of Wisconsin-Madison, and during the 2005-2006 academic year, he was a Knight Science Journalism Fellow at MIT.