By Jim Davis <i>HBJ Editor</i>
It’s a pivotal year for Bothell-based biotech firm OncoGenex Pharmaceuticals.
The company will finish clinical trials on one of its three cancer-fighting drugs while reaching a key milestone on another.
In biotech, development costs can run into the tens of millions of dollars before firms can get the regulatory OK to sell their products — or be forced to scrap years of work.
“Because it takes so long, when you get to the back end it gets really exciting because it ends up being successful or it’s not,” said Scott Cormack, OncoGenex’s president and CEO. “That’s why this year is so defining.”
One analyst believes there’s reason for optimism.
OncoGenex is a small, nimble company that has the respect of key industry leaders and investors, said Greg Wade, managing director of Healthcare Equity Research. He said the data on its drugs under development have been compelling.
The drug that’s finishing clinical trials in the next few months, dubbed Custirsen, treats prostate cancer and non-small cell lung cancer. In the trials, the drug is being given to 1,023 patients with advanced stage prostate cancer.
This is the final step in most cases before a drug can be taken to market.
The second drug is called Apatorsen. It is being developed to treat bladder cancer, some forms of lung cancer, pancreatic cancer and prostate cancer. The drug is expected to finish the second round of testing later this year, involving 180 patients being treated for bladder cancer.
The third drug, OGX-225, is still in initial testing.
The drugs were discovered at the Vancouver Prostate Centre in Vancouver, B.C. The idea behind the drugs is to create medicine that wipes out proteins produced by tumors to protect cancerous cells.
“If you can disable the defense, your primary strategy for offense should be more effective,” Cormack said. “It’s a paradigm shift in how we typically treat cancer.”
Usually, drugmakers are looking for new ways of killing cancer. OncoGenex reasons that if a drug can weaken a cancerous cell’s defenses, then chemotherapy and other more traditional methods of treatment should be more effective.
“You’re really getting at the defense of the tumor cell and blocking it,” said Dr. Cindy Jacobs, OncoGenex’s chief medical officer and executive vice president.
The ability to understand how cancer cells defend themselves came about just in the past 15 years with advances in both microbiology and the creation of massive databases of information on thousands of patients.
Cormack said that it appears that all tumors produce the same sorts of proteins for defense. It’s possible that drugs being developed by OncoGenex could be used to treat other types of cancer.
For Custirsen, the company, which is partnering with Teva Pharmaceuticals, is focusing on prostate and lung cancers. The company first started developing the drug in 1997-98 and started clinical trials in 2001.
With development costs running anywhere between $90 million to $140 million, the company needed to focus on just a limited number of cancers.
The company chose to focus Custirsen on advanced prostate and lung cancers because they are particularly deadly and have few treatments.
“In this case, we looked at it and said prostate cancer makes a lot of sense, because up until the last couple of years there had been no new advancements for prostate cancer for decades,” Cormack said. “So it’s an important area to go into.”
OncoGenex was a Vancouver, B.C., firm that took over the former Sonus Pharmaceuticals in 2008. Sonus was a publicly traded company and the merger allowed OncoGenex access to funding through the stock market. OncoGenex, which had a U.S. subsidiary in Pioneer Square in Seattle, moved its headquarters to Bothell.
Being in the Puget Sound area has been beneficial because it’s given the firm access to some of the leading minds in cancer treatment in the world, Cormack said. These folks have been relied upon in an advisory capacity. The area also has a large talent base to draw upon new hires.
The company has about 40 employees in Bothell who are keeping tabs on the drugs that are undergoing clinical trials at about 130 sites globally. The staff is working with urologists and oncologists who are conducting the testing. They’re also focusing on financing and working with regulatory agencies across the world.
“We don’t have a bunch of people running around in lab coats testing drugs,” Cormack said. “That part of the development cycle is done.”
In the clinical trials, OncoGenex is focusing testing on how it measures against current forms of treatment — what is called the standard of care. Cormack said that sets a higher bar.
“Our view is there is no point for the patient in developing a therapy that is simply going to displace something else,” Cormack said. “There’s no benefit for the patient whether you get a green packet or a red packet if your survival is going to be the same at the end of the day.”
The company found in the second round of testing for Custirsen that the median survival benefit for a patient was 6.9 months longer life.
Jacobs said that usually anything longer than two months is considered beneficial. The idea is that as therapies develop that doctors can use a combination of treatments to prolong a patient’s life from months to years.
“That’s where you gain ground with cancer as you get more effective therapies,” Jacobs said.
OncoGenex hasn’t been able to track the results for Custirsen during this final stage of testing. It’s so the company can’t influence the results.
It’s possible that the Food and Drug Administration could decide that the drug is not effective enough to go to market. And that means that years of research, testing and cost will be for naught.
“Unfortunately that’s the risk in the business,” Cormack said.
Healthcare Equity Research’s Wade said there are several reasons to expect a favorable outcome on Custirsen.
There’s a “mechanistic rational” for the drug, it’s unlikely to be determined to be unsafe and it’s shown a strong outcome in its previous randomized study.
And the current study is large and well powered.
Cormack said they’re hopeful that the drug will get the OK to move forward.
“Our optimism comes from what is observed in the science,” Cormack said. “The preclinical data is super strong across all of these different indications particularly in prostrate cancer.”