Vancouver-based DelMar Pharmaceuticals [DMPI] is developing new drug candidates targeting orphan cancer indications representing market opportunities in the $100s of millions in North America and potentially billions of dollars worldwide. The company’s lead candidate, VAL-083, is a “first in class” small-molecule chemotherapeutic in Phase I/II, targeted for refractory glioblastoma multiforme. Approved in China for chronic myelogenous leukemia (CML) and lung cancer, Del Mar obtained the rights to develop VAL-083 after promising NCI-sponsored Phase I studies.
Promising data announced in 2012 with follow ups scheduled at various healthcare conferences this year coincided with the company’s recent listing on the OTCQB, which commenced Q1-2013.
OneMedRadio spoke with Del Mar Co-Founder, President and CEO Jeffery Bacha, about this recent company milestone, and what lies ahead in the advancement of VAL-083. Bacha also mused on trends in the industry related to re-positioning promising data for the international markets, and the opportunities out of achieving orphan drug status.
Click to listen, and view the transcript below.
Matthew Margolis: OneMedRadio welcomes Jeffrey Bacha, Co-Founder, President and CEO of Del Mar Pharmaceuticals. Del Mar is developing and commercializing proven anti cancer therapies in new high impact often cancer indications for patients who have failed modern biological target therapies. The company recently commenced public trading, listing under the symbol DMPI. Jeffrey, thank you for joining us.
Jeffrey Bacha: Thank you Matthew. It’s a pleasure to be here.
MM: Let’s start with the company snapshot. Give us Del Mar’s brief history.
JB: We started the company in 2010 around the initial asset that we pulled out of the National Cancer Institute here in the United States. This is a drug that had been widely studied in what we would call the original war on cancer a few years ago. We saw some promising activity in the candidate, which we called VAL-083, particularly in central nervous system or tumors attached to brain cancer. In particularly, the form of brain cancer called glioblastoma, which is the most common and aggressive form of brain cancer. It affects about 1,500 patients a year in the United States.
We decided that we would move the drug back into clinical trials in glioblastoma. We began treating patients in 2011 and have been recently pleased with the results so far. Our business plan is essentially to take a [chemotherapeutic] that has some history around it, wrap new and intellectual property around it, new patent filings, for example, [that offers] drug protection where its available. [Then] to look at the mechanism of action and move it forward into a clinical indication where we believe it can have the most impact and be able to move it forward very quickly.
Our team has actually done this before. Our clinical group is responsible for the development of a drug called Synribo, which was again another older candidate which was taken through clinical trials of the FDA and that company was acquired in 2011 for $235 Million. So, a nice history of success within the team, success with the business model, and being told very attractive exit for investors.
MM: Talk about the technology behind VAL-083 and the clinical progress.
JB: It is a small molecule drug. [VAL-083] is a cytotoxic, so it’s a classic cancer drug. One of the things that intrigued us was that it crossed the blood brain area – a mechanism that the body has to keep most things outside of the brain. This drug actually gets there. It’s one of the few things that does. The other thing that we found interesting was that it works differently in terms of how it impacts the tumor compared to other chemotherapies that have had some level so success in glioblastoma. The problem with those other therapies is that they don’t work for everybody. We now understand why they don’t work. This drug, VAL-083, meets that need and having activity for other therapies fail, which really represent half of the patients that are diagnosed with the disease in a $1 BN market.
MM: Is glioblastoma considered an area of unmet medical need, and are you pursuing orphan drug status?
JB: It is definitely an area of unmet medical need. As I said, 60% or 70% of the patients who are diagnosed won’t respond to the current therapies and there is nothing for these patients unfortunately. If a patient is in that group that doesn’t respond to the current therapies, the likelihood of surviving for two years from diagnosis is less than 10%. So it is a huge unmet need, and we are very pleased and hopeful that we are developing something that will have an impact in those patients.
This is an orphan drug indication. We do have orphan drug protection in the United States and in Europe. What that means is that it helps to move things forward in a more rapid manner as well as provide some market protection for us in terms of market exclusivity following approval. We have also filed new patents but in the event that we end up without any new patent claims, we would have seven years of market exclusivity from approval by the FDA and the United States, and 10 years from approval in Europe, where we would be able to market the product without competition.
MM: VAL-083 is approved in China for the treatment of chronic myelogenous leukemia and lung cancer. So what are the next steps here?
JB: When we started out, we weren’t looking for a Chinese drug. We were interested in the drug based on data from the National Cancer Institute in the United States. We learned that the drug was approved in China when we were looking for collaborator to help manufacture the product for us. We found the Chinese approval interesting and in studying it, believe that there are ways to re-position the drug in those two indications. The nice thing is that we can create that new data – we can re-position the drug in the current market place with a partner to generate revenue.
Then we can use that data to expand the global opportunity for the product. So we are in the process of doing that in parallel with what we are doing in glioblastoma here in the United States.
MM: I want to shift gears a little bit, talk about recent news. Give us a summary of the company’s public listing, the steps that were taking to reach this milestone for example.
JB: We had accomplished what we consider two major milestones in 2012. One being [the acquisition of] the commercial rights of the product overseas and globally in fact. Two, moving forward in our glioblastoma clinical trial in the United States and actually presenting some initial data demonstrating the drug had activity in patients failing other therapies after the Society for Neuro-Oncology meeting last November.
So based on those two milestones, we decided that it would be possible to test the public markets…We are very pleased that we were able to in very short order upon launching a financing in January, complete that in just a few weeks and raised $10.5 million – the transaction was actually originally seven and a half, but they were so oversubscribed that we expanded it up to ten and a half. We are very pleased with how that went.
So now we are publicly listed, the stock has been trading for several weeks and we are very excited to have a capital now to move the project forward in a timely manner. Hopefully as we go forward and hit our milestones the market will continue to respond in a favorable manner.
MM: Talk about some of that data that you presented, as well as how that positions you for a regulatory path in the US.
JB: The data that we presented at the Society for Neuro-Oncology meeting was interim data, the first couple of cohorts or patients. What we are doing is modernizing the dosing regimen initially. We have noted what worked in terms of treatment of glioblastoma [which coincides] with the National Cancer Institute several years ago. But we’ve taken advantage of learning more about the mechanism of action of the drug and the disease itself.
So we are treating the patients more often and trying to push to a higher dose than they had done 20 years ago, and we hope to achieve an even better outcome than what was attracted activity from that original National Cancer Institute work. So we presented the first data to Society for Neuro-Oncology meeting last year, which demonstrates that the drug even at lower doses appears to have activity in some patients who have failed the front line therapy in glioblastoma, which is called temozolomide, and also have failed the second line therapy which is a drug called Avastin. That population in both first and second line therapy represent about half of the patients who were diagnosed.
That was exciting news for us, because it was lower doses than had been shown before, which suggest that maybe our modernizing dose regimen is having an impact. We have been continuing to move the product forward in clinical trials and anticipate presenting further data at the American Association for Chemotherapy Research in Washington DC in April, and ASCO in June. As well as again the Society for Neuro-Oncology meeting later in the fall. As we go forward in complete the dose modernization course of the study, we will be positioned at least in the refractory disease to move nearly directly into registration trials with the product.
The interesting thing in terms of refractory disease, whether there is no other therapy, typically approval is sought based on an open-label Phase II design, and often in less than 100 patients. There had been several other examples of these recently, including Avastin’s approval in second line therapy in glioblastoma in 2009. So this positions us to be able to move forward in a streamline manner with a study design that is capital effective, in terms of the number of patients and the cost of the study, and in terms of timing because we can move forward much more quickly to be able to bring the product to market and to help patients in the most timely manner possible.
MM: That was Jeffrey Bacha Co-Founder, president and CEO of Del Mar Pharmaceutical. Del Mar trades under the symbol DMPI. For OneMedRadio this is Matthew Margolis signing off.