Posted on January 8th, 2016
by Jeffrey Bacha
In the past 72 hours, biotech and pharmaceutical companies have filed new registrations seeking to raise more than $1.6 billion from the public markets. This is all in anticipation of and in preparation for “JP Morgan Week” in San Francisco; the annual frenetic mecca where tens of thousands of biotech and pharma executives, bankers, entrepreneurs, investors, and service providers convene to raise capital, pursue partnering opportunities and set the stage for the coming year.
Everyone’s goal is to garner attention and rise above the fray to capitalize on opportunities during such a frenzied event!
As I shared in my previous post, DelMar’s mission is to develop proven cancer medicines that have demonstrated promise in early-stage clinical trials but for various reasons, be it financial or circumstance, did not previously progress to commercialization.
Dr. Dennis Brown, DelMar co-Founder and Chief Scientific Officer, mined the archives to opportunistically identify drug candidates that had already demonstrated activity against cancer in prior human clinical trials that might have renewed value today.
We believe starting with clinical validation from prior studies conducted by the U.S. National Cancer Institute (NCI) reduces risk and gives us a real strategic edge toward success.
As the saying goes: Facts are stubborn things.
The drug hasn’t changed and the tumor hasn’t changed, so the drug will work the same way it did in the past. Our job is to determine how to bridge prior clinical validation with modern science to treat cancer patients in today’s world.
Of course, what has changed in many cases is the types of care available to patients and scientists’ knowledge about biology of the tumor.
In this era of targeted therapies, we have made great strides in the treatment of many types of cancer; however, what we have also learned is that there is no magic bullet for every patient, or even for a single cancer. Time and time again, researchers have attacked a new cancer target successfully only to find a new subset of patients or tumor cells that may not express that target. Suddenly, a “new” unmet medical need is defined in the context of an elegant array of biomarkers and genetic signatures.
To be clear: That “new” unmet need is represented by a particular tumor subtype that probably always existed, but has now been highlighted by the targeted therapy treating a separate subset of patients within what was once believed to be one single cancer diagnosis. Thus, to some extent, targeted therapies have allowed us to chip away at some phenotypic subsets while illuminating others as highly underserved.
Employing modern research techniques and tools allows us to explore genes and biological pathways to provide new clarity and point a drug that is known to work in a broad sense precisely toward the type of patient whose tumor will benefit most today.
Our lead product candidate VAL-083 fits this profile nicely, providing another edge toward success.
Published pre-clinical and clinical data suggest that VAL-083 may be active against a range of tumor types, including lung, brain, cervical, ovarian tumors and hematologic (blood) cancers. Our goal is to pinpoint the right patient population in today’s environment and build upon previous clinical experience to solve modern unmet medical needs. Our research pointed us in the direction of the most aggressive of brain cancers, glioblastoma multiforme (GBM).
GBM affects 15,000 people annually in the United States. The median survival from diagnosis is about 15 months. That figure, unfortunately, has not changed in decades.
VAL-083 was previously shown to readily cross the blood-brain barrier and accumulate preferentially in tumor tissue. There was also promising evidence of clinical activity in brain cancers, including GBM, from prior NCI-sponsored trials. A good start for sure!
The question we then asked was: Does all of that matter today?
That’s where modern understanding of tumor biology gives VAL-083 its next edge toward success:
We now know that approximately 2/3 of patients express high levels of an enzyme known as “MGMT.” MGMT is a DNA repair enzyme that makes tumor cells resistant to the chemotherapies currently used to treat GBM.
We hypothesized that because VAL-083 attacks the tumor in a slightly different way compared to the current chemotherapies it might circumvent MGMT repair and offer a new treatment paradigm for the majority of GBM patients who are underserved by current treatments.
We found this to be exactly true. VAL-083 maintains activity independent of MGMT levels. We presented these data at the American Association of Cancer Research meeting in 2012 and launched into new clinical trials shortly thereafter.
Our initial focus is on patients who have failed currently available therapies. As such, we’re treating patients who have failed to respond to both front-line therapy with temozolomide and radiotherapy and Avastin®, which was approved by FDA in 2009 as second-line therapy in GBM (although, as stated on the Avastin label, there is “No data available demonstrating improvement in disease-related symptoms or survival with Avastin” – a story for another day).
We are encouraged by our results to date. As we presented at the most recent annual meeting at of the American Society for Clinical Oncology (ASCO), VAL-083 is well tolerated at therapeutic doses and we have observed a dose-dependent survival benefit in Avastin-failed GBM patients following treatment.
These results from our Phase I study were in line with expectations based on the data already known about VAL-083 from previous NCI-sponsored clinical trials conducted and our own research. Based on these promising results, we entered into a Phase II expansion cohort, which is now fully enrolled. Interim data from this trial was presented at the Society for Neuro-Oncology (SNO) Annual Meeting in November, 2015.
During the first half of 2016, we anticipate presenting the next data from this clinical trial and conferring with the FDA to confirm the design of a registration-directed Phase III clinical trial which is targeted to begin in the second half of the year.
Now that we have defined a well-tolerated dosing regimen for GBM patients whose tumors have failed to respond to currently available treatments, we plan to leverage the opportunity in newly diagnosed GBM patients whose tumors are known to express high levels of MGMT. We expect to begin these new clinical trials soon.
Our continued research into VAL-083’s mechanism of action has begun to open new doors. We believe that VAL-083 offers promise in difficult-to-treat subsets of certain lung and ovarian cancers. We plan to begin clinical studies in lung cancer in the first half of 2016.
As you can see, DelMar has an exciting year in store in 2016 as we continue to build on the momentum of encouraging data with VAL-083 from our ongoing Phase II trial in refractory GBM and newly planned clinical studies in front-line GBM and NSCLC. We truly believe that our results so far, our approach, and the experience of our team give us an edge toward success.
I look forward to sharing that with you as our modernized medicine, VAL-083, progresses towards commercialization.
I’d like to wish all of you a happy and healthy New Year! I look forward to updating you “live” from San Francisco!
Forward Looking Statements
Any statements contained in this blog that do not describe historical facts may constitute forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995. Any forward-looking statements contained herein are based on current expectations, but are subject to a number of risks and uncertainties. The factors that could cause actual future results to differ materially from current expectations include, but are not limited to, risks and uncertainties relating to the Company's ability to develop, market and sell products based on its technology; the expected benefits and efficacy of the Company's products and technology; the availability of substantial additional funding for the Company to continue its operations and to conduct research and development, clinical studies and future product commercialization; and, the Company's business, research, product development, regulatory approval, marketing and distribution plans and strategies. These and other factors are identified and described in more detail in our filings with the SEC, including, our current reports on Form 8-K.
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