The ASCO abstract on DCVax-Direct was released last night. It contains only a description of the drug and the goals of the phase 1/2 trial. This abstract was submitted some months ago and describes the basic structure and goals of the trial. It does not give any specific patient results.
This trial is being conducted by Northwest Biotherapeutics (OTC:NWBO) in patients who have inoperable solid tumors. The primary emphasis is on colorectal cancer but it will also enroll melanoma, pancreatic, liver cancer and some miscellaneous cancers. In the treatment of solid tumors, the standard procedure is to surgically remove as much as the primary tumor mass as possible and then try to reduce or eliminate the remaining tumor tissue with drugs and radiation. In cases in which the tumor cannot be resected because the tumor has broadly metastasized or cannot be operated on because of its location, the prognosis is much worsened. This trial is being conducted in very advanced, inoperable solid tumor patients. This means that any signs of biological activity will be received with great interest by the medical and drug development community.
Because this is an open label trial, results on patients can be reported as the trial progresses. This morning, NWBO sent out a press release on results for a specific patient who is suffering from an advanced, inoperable and very difficult to treat sarcoma. I believe that over 20 patients in the first phase of the trial have received three or more injections which compares to the four injections for the sarcoma patient. As the year progresses, we should get significant information on these 20 patients and others that will give investors a pretty good insight into the potential of DCVax-Direct.
Treatment was started in February, 2014 for this sarcoma patient so that the results are very preliminary, but MRI scans, CT scans and a biopsy suggest that DCVax-Direct is killing cancer cells at the site of the primary tumor and importantly at distant metastases. It also appears to be creating an immune response against the tumor which is the goal of cancer vaccines. In this patient, the investigators noted that there was a partial collapse of the primary tumor. I am trying to clarify what this means, but I believe that this indicates that there was around 50% shrinkage in the tumor mass. If so, this qualifies as a partial response and will certainly get the attention of the medical and drug development community as this type of sarcoma has extremely poor outcomes and there is no effective drug therapy.
This is very early data and it will take several more months to begin to assess the ultimate effect of DCVax-Direct on this sarcoma patient. However, these initial results are quite encouraging. Because the 60 patient Phase I/II study is open label, the company can report results as they occur. Hence, investors can follow the progress of this sarcoma patient and I would expect there to be numerous other case reports and follow-ups in 2014 and 2015.
The commercial opportunity is huge for a product that is effective in inoperable solid tumors as there are no really effective current therapies. Also, the regulatory pathway will be much shorter if DCVax-Direct produces encouraging results. DCVax-Direct has the potential to be as or more important than DCVax-L in the NWBO investment thesis. However, at this point we have seen preliminary data on just one patient and extremely positive animal data. It is very exciting but very early.
I have written a blog on my website that provides much more detail on the sarcoma patient and the trial. It can be accessed through this link.
Disclosure: I am long NWBO. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.