A new cancer vaccine trial is now underway, as of March 27th, that is fundamentally different from any cancer vaccine trial held until now. Not only is it different, but it’s also a lot simpler, its preclinical results on solid tumors is near a 100% complete response rate, it’s cheaper, and the two components of this new vaccine approach are already approved. The combination isn’t yet, but because the components are, final approval for the combination could come as soon as two years from now, according to trial leader Dr. Ronald Levy. This is despite the fact that human trials are only now beginning phase 1.
Before we get into the science, the importance of this for investors is not that the trial is being sponsored by private industry, at least not yet. This is a Stanford University initiative, and there is no stock you can buy that will directly benefit from the success of this trial yet. Rather, the importance is that if this trial succeeds, the implications for other cancer immunotherapy companies focusing on solid tumors could be quite negative.
These include Incyte (NASDAQ:INCY), ZIOPHARM Oncology (NASDAQ:ZIOP), CAR-T companies like bluebird bio (NASDAQ:BLUE), Celldex (NASDAQ:CLDX) and others. However, since this particular vaccine only targets solid tumor cancers, even if results on humans are as good as those on mice, there will still be plenty of space in the cancer immunotherapy space to attack other diseases.
It would also be a negative for companies that earn much of their revenue from chemotherapy treatments, as no chemotherapy is involved in this treatment at all. Since the first trial is focused on lymphoma, Roche (OTCQX:RHHBY) could also be particularly affected if it succeeds, because its lymphoma drug rituximab is one of the best selling cancer drugs in the world.
The idea behind the new vaccine is this. When a solid tumor first starts developing, the immune system responds. It just gets shut down in the middle of its response. Not that immune cells don’t recognize the tumor. They just no longer have the command to attack it. What the vaccine does is restart the attack.
The vaccine’s two components are CpG oligonucleotide, a section of DNA that has been used as a vaccine adjuvant since 2011, and an antibody called BMS-986178 that binds to a protein called OX40. Together, they synergistically activate the immune cells already in the tumor.
The CpG component can recognize molecular patterns on pathogens in the body and cause T-cells to express OX40. By then binding to the OX40, the T-cells are reactivated. The mice in the study were transplanted with genetically identical tumors on opposite sides of the body, and when one was injected with the combination, the other responded as well.
87 out of 90 mice that had one solid tumor injected had complete responses both for all tumors in the body. The other three mice had relapses, but then had complete responses again after a second treatment. Meaning, the complete response rate is essentially 100% after two rounds of treatment for mice. For humans, we shall see soon.
The other advantage over engineered cancer vaccines is that this is much cheaper. CAR-T vaccines can cost over $500,000. This new vaccine, since it is off-the-shelf and doesn’t have to be specifically engineered for each patient, will probably cost much less and have much less side effects. Since the immune cells that are activated are already engineered by the patient’s own immune system to contain the correct antigens that will help the T-cells recognize the cancer, there is no need to engineer anything patient-specific.
According to the trial filing, this will be open-label with the end point of safety and objective response rate and progression-free survival. It has an estimated completion date of 2020, though results may be available before then, soon after enrollment of 15 is completed. Each patient will be treated first with radiation in order to make the tumors more visible to the immune system, and then with injections at intervals for about six months, so about six months after enrollment completion, we may have data shortly afterwards. Patients will be followed up for up to 96 weeks after initial treatment.
Returning to investment, it is possible that if the results of this first human trial are positive (and Dr. Levy says he is planning to start a second trial this year as well), then the research could be acquired. This has happened before in the CAR-T space especially, and sponsoring a pivotal trial is orders of magnitude more expensive than a small 15-patient phase 1. A large company may need to swoop in to sponsor. So if the trial succeeds and pharma moves in to buy the candidate, buying the company that does acquire it would be my move here.
It won’t be a cancer cure-all, because it isn’t designed for cancers that aren’t solid tumors. It also is limited to only certain cancers that evade the immune system in a specific way. Nonetheless, it is unquestionably exciting.
Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.
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.
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