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Emerging Company Profile

Vaxxinity: supplanting mAbs with vaccines for chronic disease 

Emerging Company Profile: after its COVID-19 vaccine, Vaxxinity plans to take on mAbs for chronic diseases with vaccines against the same targets

After getting its COVID-19 vaccine on the market next year, Vaxxinity plans to take on mAbs for chronic diseases with vaccines against the same targets. 

Nov 7, 2020 | 3:40 AM GMT

Vaxxinity aims to disrupt the biologics market with a vaccine platform that replaces mAbs for chronic disease by inducing patients’ immune systems to manufacture therapeutic antibodies in vivo. But first, it intends to deliver 100 million doses of a COVID-19 vaccine by 1Q21.

Just seven months old, Vaxxinity is still in the process of being formed through the merger of its two predecessor entities, United Neuroscience Inc. and the Covaxx unit of United Biomedical Inc. The two already share a management team and have been using the same vaccine platform for different purposes.

United Neuroscience spun out of United Biomedical Inc. in 2014 with exclusive, worldwide rights to UB-311, a ß amyloid vaccine for Alzheimer’s disease. Covaxx was created in response to COVID-19 and is developing a multitope peptide vaccine, UB-612.

Both diseases are gigantic, pressing health needs, but the scope of Vaxxinity’s UBITh peptide vaccine platform is even larger, CEO Mei Mei Hu told BioCentury.

“We have the first vaccine platform that can break immune tolerance in virtually 100% of patients. When you can do that against chronic disease targets it opens up many possibilities,” including developing “vaccine versions” of marketed mAbs, said Hu.

Harnessing patients’ immune systems to produce antibodies against chronic disease targets isn’t a new idea, but it has yet to take off. 

Vaxxinity is leveraging a synthetic peptide vaccine platform — dubbed UBITh  that has a history of commercial success in animal health, via parent company United Biomedical. Hu believes the technology has generated enough early clinical data in Alzheimer’s, via United Neuroscience, to suggest that success will translate to humans.

Alzheimer’s is not Vaxxinity’s lead indication, however; COVID-19 has jumped into that spot.

Though Vaxxinity is behind the first wave of COVID vaccines in the clinic, it plans to start a Phase II/III trial of UB-612 by year-end and to be ready with 100 million doses should the vaccine receive emergency use authorization early next year. Hu said that United Biomedical’s extensive experience in manufacturing scale-up and the fact that synthetic peptide vaccines can use existing supply chain infrastructure, unlike mRNA vaccines, which need new infrastructure, mean that Vaxxinity will have production and distribution advantages. 

“Clinical risk is not the end of the story; you’ve got to manufacture it and deliver it,” she said. 

Behind COVID-19, Vaxxinity is advancing several programs against validated chronic disease targets, including CGRP for migraine and PCSK9 for hypercholesterolemia. Hu thinks these programs could end up moving faster than Vaxxinity’s more advanced vaccines for neurodegeneration.

“Alzheimer’s and Parkinson’s are big unmet needs, but they are also more difficult and have newer biology,” Hu said. “With CGRP you have five mAbs essentially competing on payer coverage. If you can come in with a vaccine and disrupt the cost-benefit ratio, you’ll get advantageous shares from the payers.” 

Neither Vaxxinity nor United Neuroscience has raised a full institutional venture round. But Hu said the platform has attracted high-profile individual investors including Peter Diamandis, general partner of Bold Capital Partners and co-founder Silicon Valley think tank Singularity University; and Bill Ackman, CEO of Pershing Square Capital Management. 

Vaxxinity interim CFO Daniel Geffken told BioCentury the company is exploring a range of fund-raising options including going public via a SPAC (special purpose acquisition company), an avenue that’s become more popular this year.

“We have to raise a lot of money for Alzheimer’s and Parkinson’s. We realize that,” said Geffken.

Head start in animal health

The UBITh peptide vaccine platform was invented by Hu’s mother, United Biomedical co-founder and Chairwoman Chang Yi Wang. The platform yielded the first synthetic peptide-based vaccine to reach the market, an infectious disease product to prevent foot and mouth disease in pigs.

United Biomedical produces about 500 million doses of the pig vaccine per year, said Hu.

The platform lends itself to efficient manufacturing and distribution, Hu said, because its peptides are small and produced via an entirely synthetic process.

For an indication with the urgency and size of COVID-19, what’s arguably even more important is the manufacturing scale-up knowledge that United Biomedical has gained via its marketed vaccines, Hu said. “Making more vaccine is often not as simple as doubling the recipe.”

Vaxxinity plans to manufacture up to 1 billion doses by YE21.

For distribution, Hu said Vaxxinity can take advantage of standard cold chain supply lines, whereas the two front-runner COVID vaccines that are based on mRNA will require liquid nitrogen to keep them at -70°C throughout the shipping process.

Another design feature that sets UB-612 apart from the majority of COVID vaccines is that it’s constructed to trigger immunity against epitopes on more viral proteins than just the spike, which could lead to a broader, more robust immune response.

UB-612 is in Phase I testing in Taiwan, where the New York-based biotech has an office. Vaxxinity has received a NT$430 million ($15 million) grant from the country to support Phase I and II studies.

The company’s upcoming Phase II/III trial will take place in Brazil where infection rates are high. “We are COVID chasing,” said Hu.

Taking aim at chronic diseases

Vaxxinity’s platform can be tuned for infectious or chronic disease applications, and although COVID-19 is its lead indication, chronic diseases are its future.

Hu said the UBITh platform can generate vaccines that selectively stimulate B cell responses and avoid inflammatory T cell responses, which is safer for chronic disease treatment. Its COVID-19 vaccine is tailored to stimulate both types of responses. 

For chronic diseases, the platform links 20-amino acid peptides against self-antigens, which are not immunogenic on their own, to equally small peptide carriers that trigger B cell-coordinating helper T cell responses by “mimicking things on measles, tetanus, yellow fever, that the body recognizes as foreign,” said Hu.

The small size of the carrier also contributes to the vaccines’ safety, she added.

“Traditional vaccines attach that peptide antigen to something really big to activate the immune system. The problem is the big thing usually gets 99% of the response, which leads to off-target effects, while the little epitope gets far less,” Hu said, citing the freuqency of reactions to flu vaccines as an example.

“There are two parts of a vaccine, the part that activates immune cells and the part that directs them. The first part, we make that very small and we make it look like something foreign, then the only thing that exists to direct the cells is the self-peptide.”

Adding to the safety picture are the fully synthetic nature of the peptides and the fact that they are free of viral vectors, Hu said. “They don’t have the biohazard risk of traditional vaccines.” 

The benefits of Vaxxinity’s vaccines over antibody therapies include more efficient manufacturing, lower cost of goods and likely less frequent dosing. The company is aiming for quarterly to annual dosing schedules with its chronic disease vaccines; whereas many mAbs require monthly infusions.

Hu cited PCSK9 inhibition as an example of where these features could come together to disrupt the market. The bimonthly injection schedules of the marketed PCSK9 mAbs are inconvenient for patients, and payers have erected barriers to usage due to the products’ perceived high prices, “even though these therapies have been shown to be effective,” she said. 

“We think we can provide the same level of efficacy on a biannual or annual basis, and when you can change the cost paradigm you can negotiate leapfrogging to front of line,” Hu said.

Vaxxinity’s CGRP and PCSK9 programs are both preclinical; the company plans to submit an IND for its CGRP vaccine next year and for its PCK9 program in 2022.

Hu said the next step for the Alzheimer’s vaccine is a Phase III trial, for which it is seeking a partner. The company announced in January 2019 that its Alzheimer’s vaccine UB-311 was safe and well-tolerated and led to a 96% response rate in a 43-patient Phase IIa trial to treat mild disease. Immunogenicity was determined by measuring change from baseline in anti-β amyloid antibody titers. Hu said Vaxxinity will publish full Phase II data next year.

The company also expects to present data next year from its Parkinson’s disease vaccine, which targets ɑ synuclein and is in Phase I testing. 

COMPANY PROFILE
Vaxxinity 
New York, N.Y. 
Technology: Vaccine platform for chronic and infectious diseases
Origin of technology: United Biomedical Inc.
Disease focus: Neurology, endocrine/metabolic disease, infectious disease
Clinical status: Phase II
Founded: 2020 by Mei Mei Hu, Lou Reese
University collaborators: University of Nebraska Medical Center, University of Southampton, Boston University, University of Texas Southwestern, Academia Sinica
Corporate partners: Dasa
Number of employees: 50-100
Funds raised: Undisclosed
Investors: Peter Diamandis, Bill Ackman, other undisclosed individual investors
CEO: Mei Mei Hu 
Patents: Undisclosed number of patents covering the vaccine platform, UB-311

TARGETS
CGRP – Calcitonin gene-related peptide
PCSK9 – Proprotein convertase subtilisin/kexin type 9 

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