that is often lacking when biotech companies try to improve the properties of existing drugs. One example is with pegylation, in which polymer molecules are attached to protein drugs, to make them last longer in the bloodstream so they can be taken with less frequent injections. This pegylation feature has been vital to the growth of interferon beta drugs that make up a multi-billion dollar market for Biogen Idec, Merck KGaA of Germany, and Bayer.
One of the problems, though, is that proteins that are pegylated sometimes clump together, creating a different 3-D structure that’s no longer effective and can cause side effects, like welts at injection sites. Companies have been working for years to make pegylated proteins that can avoid the aggregation problem, and which can remain stable long enough in the bloodstream to enable patients to switch from every-other-day or once-weekly injections to a greater ideal in convenience—once-monthly dosing.
So Allozyne has set a goal of making a pegylated interferon beta drug for MS that could be manufactured consistently to avoid the clumping problem, and to enable once-monthly dosing. By snipping out the amino acid (methionine), Allozyne is able to consistently make a pegylated drug that won’t aggregate, Chhabra says. A well-financed competitor, Biogen Idec, has been testing its own versions of longer-lasting interferon beta drugs that are further along in clinical trials. San Diego-based Ambrx is another venture-backed company with its sights set on making a longer-lasting MS drug, in partnership with Merck KGaA. Neither of those companies have yet presented full data at a medical meeting from the middle or late stages of clinical trials.
Allozyne’s contender, AZ-01, entered its first clinical trial in the first half of 2009. The first study enrolled 40 patients who were randomly assigned to get the experimental drug or a placebo, Chhabra says. This trial was only designed to measure safety, not effectiveness, so Allozyne can’t say for sure how well its drug might be working to reduce the progressive neurological damage patients suffer from multiple sclerosis. That kind of test will come later in development, Chhabra says.
But for now, the company learned its drug was safe, that it has potential for once-monthly dosing, and that it doesn’t cause skin damage around injection sites like other products, Chhabra says. More detailed results will come in an Allozyne press release in a couple weeks, she says, and results from a second Phase I clinical trial should arrive by the end of the year. The next step will be to run a mid-stage clinical trial, and possibly start a final-stage clinical program for AZ-01 within 24 months, Chhabra says.
The second big thing that Allozyne has done is to continue to invest in its basic platform technology, and not bet the entire company on a single compound, like many other startups have been forced to do in the downturn.
“We don’t want this to become a one-trick pony,” Chhabra says.
So Allozyne put its scientists to work on using its pinpoint protein modification technology toward one of the big challenges in biotech today—the making of “bi-specific” antibodies. This is an old idea that no one has yet solved. The concept is essentially that certain complex diseases, like excess inflammation found in rheumatoid arthritis, might
