Posted on January 30th, 2017
by Jeffrey Bacha
DelMar Pharmaceuticals’ newly initiated glioblastoma multiforme (GBM) clinical trial at the University of Texas MD Anderson Cancer Center is a keystone in our strategy to expand the development of VAL-083 to target newly-diagnosed MGMT-unmethylated GBM. We believe this is where VAL-083 will have the greatest disease impact, the greatest benefit to patients, and ultimately the greatest benefit to shareholder value.
DelMar’s initial GBM clinical trials have been conducted in end-stage/refractory post-Avastin patients. This is “normal course” in drug development. Approved GBM treatment is surgery followed by adjuvant chemo-radiation with temozolomide and Avastin (bevacizumab) upon recurrence. So, DelMar was required to initiate clinical trials for GBM patients whose tumors had failed to respond to this standard of care.
GBM patients who fail Avastin have no approved treatments and are generally ineligible for clinical trials, leaving them with no real therapeutic options. DelMar’s research to date suggests treatment with VAL-083 may offer a survival benefit versus other salvage approaches for these patients.
DelMar has been encouraged to validate these initial results via a randomized controlled trial in the post-Avastin GBM population. We feel strongly that this is the right thing to do. The FDA has indicated that DelMar can seek approval for VAL-083 in the post-Avastin GBM population with a single randomized, controlled trial. Based on FDA guidance, we have proposed a single pivotal trial that will enroll 180 patients and is targeted to be completed within approximately 20 months from initiation. Indeed, a relatively small trial in the global scheme of drug development.
If our proposed Phase 3 trial is successful, it will mean in an extension of life for GBM patients for whom currently available chemoradiation and Avastin have failed – a chance to live longer; an “opdivo for GBM” if you will – but I believe that we can do better for both patients and our shareholders.
Importantly, we believe that expanding our VAL-083 development efforts beyond refractory GBM to target the MGMT-unmethylated population will give more patients a real chance to beat this horrible cancer.
We know and have been introduced to many GBM patients who had a good surgeon and who responded to adjuvant chemo-radiation with a durable complete response that has lasted more than five years. Five years “disease-free” is considered a cure for many cancers. Unfortunately, up to two-thirds of GBM patients have no chance for this outcome because their tumors are MGMT-unmethylated and exhibit high-expression of MGMT. MGMT is a DNA repair enzyme causing resistance to chemotherapies currently approved for the treatment of GBM, including temozolomide and nitrosoureas, such as CCNU (lomustine) or BCNU (carumustine).
VAL-083 is a novel chemotherapy that was active against GBM in prior clinical trials sponsored by the U.S. National Cancer Institutes (NCI). At DelMar, our research demonstrates that VAL-083 targets the tumor differently than temozolomide or nitrosoureas and therefore is not impacted by MGMT-mediated resistance mechanisms. By focusing our expanded development efforts on these patients, we believe that we will make the biggest difference in the outcome of the disease and give more patients the chance to beat GBM.
We are very pleased to be collaborating with The University of Texas MD Anderson Cancer Center to launch the first clinical trial in DelMar’s expanded effort targeting MGMT-unmethylated GBM. We plan to enroll 48 patients in an effort to demonstrate that patients with recurrent, Avastin-naive MGMT-unmethylated GBM treated with VAL-083 can achieve a survival benefit versus recently published results with CCNU. The trial is open label so we plan to continue our practice of reporting interim results at peer-reviewed scientific meetings such as AACR, ASCO and SNO.
We also plan to conduct additional clinical trials that will advance VAL-083 into newly diagnosed patients with MGMT-unmethylated GBM. Our newly initiated MD Anderson trial will conjoin with these trials to position VAL-083 as a potential replacement for temozolomide in the treatment of newly diagnosed GBM. If we are successful, this will truly be a paradigm shift in the treatment of GBM.
As we advance, our hope and goal is that the benefits that VAL-083 may deliver to patients will be mirrored by increased value to our shareholders.
The refractory Avastin-failed GBM patient population is between 5,000 and 6,000 patients every year in the United States. Our upcoming Phase 3 clinical trial in Avastin-failed GBM provides DelMar Pharmaceuticals with an opportunity in a specific patient population where we believe that we can establish a commercial presence.
Expanding our efforts toward newly diagnosed MGMT-unmethylated GBM can offer new hope in the fight against GBM to tens of thousands of additional patients annually around the world. Newly diagnosed patients would also undergo substantially longer courses of therapy compared with Avastin-failed GBM patients thereby expanding the possible market opportunity by an order of magnitude.
We continue to work hard toward a paradigm shift in the treatment of GBM. Our thoughts and prayers are always with patients and their families who are dealing with this horrible disease. Our hope is VAL-083 will bring a new treatment option to GBM patents and unlock tremendous value for our shareholders.
You can learn more about our research and our new clinical trial at MD Anderson on our website.
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