While markets are focusing on this program, however, there’s another side to Hemispherx that’s worth keeping an eye on but that, to-date, markets aren’t paying much attention to.
And interestingly, it’s rooted in the same asset that’s under investigation as a potential treatment for the just discussed ME.
The asset is called rintatolimod and is known commercially as Ampligen.
The asset is what’s called a toll like receptor (TLR) agonist. TLR’s are one of a number of pattern recognition receptors in our bodies and have been referred to in medical journals as the “Swiss army knife of immunity and vaccine development.”
To employ the scientific term, they are transmembrane proteins expressed by cells of the innate immune system, which recognize invading microbes. On recognition, they activate signaling pathways that launch immune and inflammatory responses to destroy the invaders.
Rintatolimod is a TLR agonist, meaning it stimulates the activation of these receptors which, in turn, promotes the immune response that would normally require an antigen to initiate. Their flexibility, then, is rooted in the wide-ranging applicability of this sort of immune system activation process.
In ME, which as mentioned is the primary indication for Hemispherx’s TLR agonist Ampligen, the exact MOA is not known, but it’s thought to have something to do with the fact that the drug can promote the release of signaling molecules that can fight pathogens and (and this is the important part) prohibit the buildup of what’s called RNase L, which is thought to be correlated with ME severity.
Anyway, it’s the applications outside of ME that are the subject of this discussion and both are potentially long-term value drivers for Hemispherx.
The first is in cancer therapy.
The company announced on January 25 that it had presented data at the Immuno-Oncology Frontiers Conference in Miami, FL, which suggested that Ampligen could improve the impact of certain anticancer therapies when used as a combination type adjuvant.
Specifically, in animal models, solid tumors seemed to respond better (and when we say better here, we mean that solid tumor cells died faster and in larger numbers), when Ampligen was combined with alpha interferon and a COX-2 inhibitor, both of which are established anticancer agents in the solid tumor space.
The idea here is that the combination can translate to the immune system both recognizing the cancer cells into a higher degree and also mounting a larger scale response to the cells than would be the case without the stimulation of the TLRs that occurs when Ampligen is added to the mix.
The research was conducted in mice and points to the potential for human clinical trials with this combination type therapy in patients with a range of solid tumor cancers, starting with ovarian and colorectal cancers – two of the most difficult cancers to treat effectively using current standard of care regimens.
The second potential indication is in viral infection, with an initial focus on influenza.
On January 30, the company intends to present at the Keystone Symposia Conference on Emerging Technologies in Vaccine Discovery and Development, as part of which it will outline the potential impact of adding a TLR agonist like Ampligen to nasally-administered seasonal flu vaccines.
According to the abstract, the addition of Ampligen can potentially induce an amplified immune response (especially as relates to the part of the immune system that identifies and remembers pathogens such as the influenza virus, and, in turn, can induce a more robust immune response than would be the case for the standard vaccination on its own. Further, that the combination type therapy might be able to induce cross-strain vaccination, meaning a patient can be vaccinated against multiple version of the influenza virus with just one administration.
Again, this is early stage data, so there’s a way to go before this MOA is confirmed in a clinical trial that could underpin approval in this target indication. With that said, however, and when taken in line with the potential anti-cancer combination discussed above, it serves to strengthen Hemispherx’s pipeline considerably.