15 Apr Madison biotech company happy to ride the coattails of competitor
Madison, Wis. – Is there cause for concern at Madison-based Quintessence Bioscience? After all, Alfacell Corp., an East Coast biotech competitor, just finished its second Phase III trial of a new class of anti-cancer therapy that is very similar to what is being developed at Quintessence, which has not even begun clinical trials on its lead candidate.
If the folks at Quintessence are worried about being behind Alfacell, it doesn’t show.
Both Laura Strong, president and chief operating officer, and Ralph Kauten, chairman and CEO, are very excited over the developments at Alfacell. Both believe that success at Alfacell will help validate the novel anti-cancer therapy under development at Quintessence. It also will help attract interest from investors as well as smooth the regulatory hurdles that all new technologies face.
Kauten, in fact, believes that Alfacell’s progress is a “great boon” for Quintessence. In other words, Alfacell is paving the way for Quintessence, but can Quintessence really reap some of the largesse of Alfacell’s success?
Novel anti-cancer therapy
The novel therapies being developed by both companies target RNA in cancer cells. While current anti-cancer therapies mostly target either the DNA or specific proteins in cancer cells, RNA, which is produced by the genes and then directs the production of a cell’s proteins, is an untapped target for killing cancer cells.
Both companies are developing therapeutic ribonucleases (RNases), which are ubiquitous enzymes that destroy RNA. RNases are important in regulating gene expression in normal cells, where their activity is tightly regulated in order to protect against indiscriminate destruction of RNA that would cause unwanted cell death. However, researchers, including the University of Wisconsin-Madison’s Ronald Raines, discovered that RNases from non-human species sometimes are not regulated inside human cells and can cause cell death.
Surprisingly, cancer cells are much more susceptible than normal cells to foreign RNases. Alfacell is taking advantage of the anti-cancer activity of an RNase purified from frog eggs – named Onconase. Meanwhile, Quintessence used genetic engineering to create a designer RNase, QBI-139, that is 95 percent human, but like the frog enzyme, it kills human cancer cells.
Studies have shown that both Onconase and QBI-139 kill a wide range of different human cancer types while sparing normal cells. Thus, these RNases offer an exciting potential to broadly treat different types of cancers, which is in distinct contrast to many other experimental therapies that narrowly target only certain types of cancer. The market potential of RNase therapy could be huge.
At this point, the anti-cancer effects of Onconase have been reported on about 300 patients with different types of cancer. Most of the data reported so far are for patients with malignant mesothelioma, a rare lung cancer that is very difficult to treat. In the first phase III trial, the most advanced cases of mesothelioma were not responsive to Onconase, but the drug did increase the life span of patients with less advanced cancer.
Alfacell will soon report the results of its just-completed phase III confirmatory trial for treating mesothelioma. Presumably, this trial focuses on treating patients with less advanced disease, so it stands a good chance of showing encouraging results.
It is interesting that in recent weeks, Alfacell has entered marketing and distribution agreements for commercializing Onconase in the U.S., Eastern Europe, and parts of Asia. The company seems quite optimistic that Onconase soon will be widely approved as an orphan drug for treating mesothelioma.
The Quintessence advantage
So why doesn’t all of this discourage folks at Quintessence? First, the science behind QBI-139 is very strong and it promises to have significant advantages over Onconase. In clinical studies, frog-derived Onconase caused allergic reactions in some patients and was poorly cleared by the kidneys, causing dose-limiting problems. In contrast, since QBI-139 is an engineered gene product that is 95 percent human, it therefore is unlikely to cause allergic or other immune problems.
Also, as Strong pointed out, animal studies reveal that QBI-139 is much less toxic than Onconase, which will allow greater dosing flexibility. Finally, QBI-139 reportedly is about 100 times more enzymatically active than Onconase. Together, these facts indicate that QBI-139 should be an efficient and flexible therapy that stands a very good chance of yielding more impressive clinical results than Onconase.
Quintessence also is developing a second generation QBI-139 product that remains longer in the blood stream, meaning that it has enhanced opportunity to attack cancer cells throughout the body. Strong is very excited about this product and recently submitted a second-level Small Business Innovation Research application to the National Institutes of Health in order to continue development of this product.
“Things are really working well,” she said.
At this point, QBI-139 is being produced for use in a Phase I clinical trial anticipated to begin this summer and end sometime in 2009. Indications are that this trial will be conducted at the University of Wisconsin Carbone Cancer Center.
Assuming that Quintessence’s Phase I trials yield encouraging results, what do they do next?
Kauten opined that it would be “hard to develop a new cancer company in Wisconsin. Obviously, we’ll need to partner and probably before Phase II (trials).”
The unspoken sentiment was that Wisconsin does not have the infrastructure needed to take a successful therapeutic drug from a mid-research stage to the market.
This highlights an unfortunate problem that hinders Wisconsin’s desire to become a major player in the biotech arena. If good biotech ideas, for which there are quite a few in Wisconsin, cannot sustain development beyond early clinical trials, much less be produced and marketed here, then Wisconsin simply becomes an incubator for West and East coast concerns. Ideas are born and tested here, but brought to fruition there.
Quintessence’s Phase II trials could begin in 2009, which means that the company stands a good chance of forming a partnership with a pharma company in the not-too-distant future. Quintessence’s success will cause it to look beyond Wisconsin’s borders.
Recent columns by Steve Clark
• Steve Clark: Understanding the science is key to predicting biotech success
• Steve Clark: Predicting success of early-stage biotechnology
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