Vaxxinity, Inc. is a purpose-driven U.S.-based biotechnology company looking to develop vaccines for several hard-to-treat diseases.
The company, led by this week’s podcast guest, Mei Mei Hu, said it is committed to democratizing global healthcare.
The company is pioneering a new class of medicines aimed at disrupting the existing treatments for chronic disease, which are increasingly dominated by monoclonal antibodies.
Vaxxinity’s proprietary technology platform has enabled the innovation of novel synthetic peptide immunotherapy candidates designed to bring the efficiency of vaccines to the treatment of chronic diseases. These include Alzheimer’s disease, Parkinson’s disease, migraine, and hypercholesterolemia.
The technology is also being implemented as part of a COVID-19 vaccine program. Vaxxinity has optimized its pipeline to achieve what it said is a potentially historic, global impact on human health.
How does Vaxxinity approach differ?
“We are developing vaccines for chronic diseases or active immunotherapies and what that means is we’re teaching your body to protect itself from things like Alzheimer’s, Parkinson’s, migraine, all the major diseases that affect millions or billions of people,” Hu said.
“The main difference is in the scalability and accessibility and the model of how vaccines can work versus other drugs today that attack and treat chronic diseases. 80% of the entire globe is vaccinated. We saw in COVID that you can rapidly deploy billions of doses around the world, whereas traditional biologics are very limited by their supply and their complexity and their price. So, the real difference is the ability to what we call democratize health and get transformative treatments to everyone in the world that needs them at a price that everyone can really afford and accept.”
Hu said there are limitations to the current technologies being deployed to treat such conditions.
“They’re expensive to make, they’re complex to manufacture, and there’s limited supply. And so they have to have a different model. But when you can do it in a vaccine form, that’s when the model can change. And that’s what’s really exciting about what we’re doing.”
What is the Vaxxine Platform?
Vaxxinity’s Vaxxine Platform is designed to harness the immune system to convert the body into its own natural “drug factory,” stimulating the production of antibodies.
The technology uses synthetic peptides to mimic and optimally combine biological epitopes to selectively activate the immune system and overcome “immune tolerance.” This is the body’s tendency to avoid attacking molecules within the body.
“What we do is we select epitopes based on the targets that we want to elicit an immune response to. Everyone understands vaccines popularized by COVID, where one of the keys is that we want to get your immune system to produce antibodies. We want to activate these B cells to produce antibodies. The challenge with going after chronic diseases, though, is that your immune system is very smart. It’s probably one of the most sophisticated machines in the world. And it doesn’t like to make antibodies that attack itself. If it did that naturally, though, it’s the cause of autoimmune disease.
“So, it’s trained itself to really not do that against key proteins in your body. The problem is as we get older, some of those key proteins are actually the cause of disease, either because there’s too much of them being produced or your body can’t clear them fast enough, or in fact, they begin mutating and misfolding and then becoming toxic themselves. We literally trick your body into thinking that these things are foreign and thus induce an immune response, induce an antibody response to in fact neutralize some of these.
Different targets for different diseases
Hu said that for every disease, there is a different target. For Alzheimer’s, the lead vaccine is targeting aggregated amyloid protein. For hypercholesterolemia, it’s PCSK9.
“A lot of these targets have been validated before by drugs before them, but never in this type of approach.”
Hu explained that safety is critical, and that generating an antibody response without resulting in T-cell inflammation is center to the vaccine platform.
She noted that monoclonal antibodies are manufactured “in a giant vat externally someplace and then injected into patients. As a result, they’re very expensive, they’re very complicated, and there’s limited number of people that they can treat. Our whole approach is trying to basically democratize biologics by getting your body to be that bioreactor.”
“Imagine being able to go and get an Alzheimer’s vaccine when you’re 40 or 50… so that even though you would have maybe gotten Alzheimer’s at 60, you’re pushing it away so you don’t get it until you’re 100.”
Mei Mei Hu, CEO, Vaxxinity
How does the Vaxxinity vaccine platform work?
Hu used an analogy of a sheep in wolf’s clothing to explain the process.
“If you present a sheep to a shepherd, it’s not going to attack it because it’s part of the herd. So, what we do is with our epitope design, with our vaccine design, we put just a little bit of wolf’s clothing on the sheep. We make it have a motif that looks foreign, like measles or tetanus, something that our body recognizes is something foreign and that it needs to mount the immune response to.
“So really what we do is we may dress the sheep with a wolf’s head and that’ll trigger parts of the shepherd to say, hey, I’ve got to get on alert, and then I mount an attack to protect my herd. And that’s what we do with the immune system.”
What’s in the pipeline of Vaxxinity?
The furthest along the path is Vaxxinity’s vaccine for Alzheimer’s, which has finished two stages of trials. The next is UB-312, for Parkinson’s, which is in the clinic as well.
UB-312 is a vaccine candidate targeting pathological forms of alpha-synuclein (aSyn) for the disease-modifying treatment and prevention of Parkinson’s disease and other synucleinopathies. Preclinical data showed UB-312 elicits antibodies that preferentially recognize pathological forms of aSyn, and improve motor performance in mouse models of synucleinopathies.
Clinical data from its phase 1 trial indicate that UB-312 elicits antibodies that target aggregated aSyn. These antibodies slow the aggregation of alpha-synuclein in the cerebrospinal fluid of patients with Parkinson’s.
Hu said there are other programs, one for migraine and the other for hypercholesterolemia.
“Really what we’re showing is one, the platform works. We’re able to break immune tolerance and get virtually everyone to generate antibodies against self-antigens. Second, we’ve proved the mechanism of action. And we did that most recently in our Parkinson’s study, which means that not only can we break immune tolerance, those antibodies are getting across the blood brain barrier where they need to be, and then they’re engaging in the target. They’re actually neutralizing the target that we’re going after. Our next step is now showing proof of concept that we can find some clinical efficacy.”
Prevention or cure?
While curing existing disease is one approach, Hu said Vaxxinity is looking to prevent diseases from developing in the first place.
“Just like we give our children MMR vaccines or the chickenpox vaccine so that our kids no longer get chickenpox in the first place, imagine being able to go and get an Alzheimer’s vaccine when you’re 40 or 50, or when you show in your blood that you’re starting to have these proteins accumulate and you start getting immunized so that even though you would have maybe gotten Alzheimer’s at 60, 70, you’re now pushing it away so you don’t get it until you’re 100 or you die of something else,” Hu explained.
Can the vaccines work with other conditions?
Hu said that one benefit of vaccines is the ability to go after multiple targets at the same time.
“There are many, many indications that we can go after and our pipeline right now is just the beginning. Right now, vaccines are mostly against infectious diseases and there are a limited number of targets that are really important to go after. If you could apply the same technology but to all chronic diseases, you’re talking about being able to expand your pipeline by 40 or 50 times.”
To learn more about this topic:
Here are some links to more articles on Vaxxinity’s pipeline, as well as other companies working on using vaccines to tackle hard-to-treat diseases.