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IRVINE, Calif., May 5, 2025 /PRNewswire/ -- Nuravax Inc., a biotechnology company pioneering immunotherapy for neurodegenerative disorders, has been awarded a $3 million grant from the National Institutes of Health (NIH) to support Duvax, the first dual-target Alzheimer's vaccine, in its entry into human clinical trials. Nuravax, in partnership with the non-profit Institute for Molecular Medicine (IMM), secured the award on its very first NIH grant submission, earning top-tier scores. This rare distinction highlights the scientific strength and strategic importance of the Duvax vaccine. The grant, awarded through NIH's highly competitive SBIR Fast-Track program, will fund a critical milestone in the clinical validation of Duvax. An initial portion of the $3 million grant has been allocated to initiate the Phase 1 clinical trial evaluating the safety and immunogenicity of Duvax as a secondary prevention therapy, with the remaining funds expected later in 2025. "This SBIR grant is a powerful endorsement of our mission to clinically validate and advance Duvax as a first-in-class vaccine for Alzheimer's prevention," said Roman Kniazev, CEO of Nuravax. "While IMM laid the scientific foundation, our focus at Nuravax is on translating that innovation into real-world impact. This funding enables us to move Duvax into clinical trials in the U.S. and brings us one step closer to delivering it as preventive therapy for individuals at risk – before symptoms begin." Duvax is a first-in-class vaccine that targets both amyloid-beta and tau proteins, the two hallmark drivers of Alzheimer's disease, positioning it at the forefront of Alzheimer's research. Unlike conventional treatments, Duvax is designed for use during the preclinical stage, when the disease is silently progressing but before cognitive symptoms appear. "The Phase 1 trial, supported by this SBIR grant awarded to our development and commercialization partner, Nuravax, marks a critical step in translating years of foundational research at IMM into a potential clinical breakthrough," said Dr. Michael Agadjanyan, VP of IMM and Head of Immunology. "Based on strong preclinical data, we at IMM expect Duvax to elicit a robust immune response against both Aβ and tau pathologies, an approach that could fundamentally shift the paradigm of Alzheimer's treatment from symptomatic and disease-modifying treatment (DMT) to secondary preventive therapy for cognitively unimpaired people at risk of disease." The vaccine's use is guided by highly precise and specific blood tests that detect early pathological changes, enabling timely intervention. By clearing toxic forms of amyloid-beta and tau before irreversible damage occurs, Duvax aims to shift the treatment paradigm from symptom management to secondary preventive therapy. About Nuravax Nuravax is a clinical-stage biotech company developing and advancing immunotherapeutic vaccines for neurodegenerative diseases, including Alzheimer's, Parkinson's, TBI, and CTE. Focused on early intervention, Nuravax aims to alter disease progression before irreversible damage occurs. The company leads the clinical development, regulatory strategy, and manufacturing of Duvax, a dual-target vaccine licensed from the Institute for Molecular Medicine (IMM), a nonprofit dedicated to neurodegenerative disease research. About IMM The Institute for Molecular Medicine (IMM) is a non-profit research organization dedicated to understanding, preventing, and curing chronic human diseases, with a focus on neurodegeneration. IMM is advancing MultiTEP — a universal vaccine platform technology that enables the development of diverse vaccine formats, including DNA, RNA, and recombinant protein-based designs. Disclaimer: Research reported in this publication was supported by the National Institute On Aging of the NIH under Award Number R44AG091903. The content is solely the responsibility of the authors and does not necessarily represent the official views of the NIH. SOURCE Nuravax Inc WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Topas Therapeutics, a small German biotech company attempting to quell overactive immune systems, has revealed the first data from a study of its “tolerance induction” therapy for celiac disease. The results, to be presented at the Digestive Disease Week conference on Monday, come more than six months after Topas first declared success in the study via a press release , without divulging any data. The company discussed the therapy and shared the data with Endpoints News in an exclusive interview ahead of the conference, though many questions remain about the results. The 38-person trial focused on the safety of the company’s experimental nanoparticle treatment over seven weeks, its ability to steer immune cells toward a less inflammatory state, and patient-reported changes in gastrointestinal symptoms. Topas executives told Endpoints that the therapy was successful against those goals, even though granular data on safety and patient outcomes was missing from the presentation. They conceded that bigger and longer studies are still needed, but added that any relief from celiac disease would be a breakthrough. “There is both very much a medical need and big commercial opportunity in celiac,” Topas CEO Hugo Fry told Endpoints. Celiac disease is caused by an unwanted immune reaction to gluten, which is abundant in food. There’s no treatment or cure for the condition that, by some estimates, may affect as many as 1 in 100 people. Despite that, there are relatively few drug developers working on treatments. Topas is one of three companies, along with Anokion and Takeda, developing a potential immune tolerizing therapy in a Phase 2 study. Rather than merely tamping down on inflammation, the efforts aim to retrain the immune system to ignore triggers like gluten. “Tolerization is at that disease-modifying, almost curative end of the spectrum,” Fry said. It aims to do so by presenting nanoparticles decorated with tidbits of peptides that resemble the problematic parts of proteins that set off the immune system, known as antigens. For the celiac therapy, dubbed TPM502, the company uses three nanoparticles, each with a different antigenic peptide from the gluten protein. In many ways, the goal is opposite to that of a vaccine, which presents antigens to build the body’s reaction and create an immune response. Instead, Topas’ infused nanoparticles target specific cells in the liver that serve as a calming zone for T cells, where they learn to live peacefully alongside antigens that don’t warrant being attacked. “That’s the body’s natural hub of induced tolerization,” Fry said. “And it pushes them through that natural route.” Celiac was a good place to start looking for signals that the tolerization approach was working because doctors can easily provoke the immune system to flare up by having participants consume gluten, said Topas chief medical officer Cristina de Min. The company tested four doses of its nanoparticle therapy, ranging from 0.72 micromolar to 7.2 micromolar, in 26 patients. The other 12 patients got a placebo. Participants got the drug or placebo on day 1 and day 15 of the study and then consumed gluten a week later. De Min said that “the drug was remarkably safe,” largely resulting in side effects that resemble gluten consumption in celiac disease, including headache, pain, nausea and vomiting in 27 patients. But the company didn’t break this safety data down by side effect, or between participants who got the drug versus placebo. The company also said that there were three “grade 3” adverse events, the second highest severity, but didn’t say what they were. To get an idea of whether the treatment was working, Topas measured the release of the inflammatory immune protein IL-2 in response to being “challenged” with gluten. Only the highest dose of the therapy resulted in a significant decrease in IL-2 release. But when studying the gene expression of T cells before and after the therapy, the company found that all dose levels helped turn on genes that tamped down on inflammation and reduced genes that promoted inflammation. Topas also asked patients to rate their gastrointestinal symptoms before and after the treatment. The company said that people who got higher doses of the therapy had reduced symptoms, but the presentation was light on specific details. Topas emphasized that the study was not designed to test for efficacy. Another major caveat was the study’s strict inclusion criteria. Topas screened 391 people and recruited only one-tenth of them. Many prospective participants were excluded because they didn’t produce high enough levels of IL-2 in response to gluten consumption. “We wanted to make sure that only patients with measurable response would be involved,” de Min said. Even more patients were excluded because they didn’t have the certain immune system genotype that Topas focused on in this study. Topas is planning a Phase 2b study that will include a broader range of immune genotypes, accounting for 85% to 90% of people with celiac disease. Topas also wants to develop a tolerization therapy for type 1 diabetes, Fry said. But doing so will require more funding, and Fry is setting out to raise a €70 million Series D round. The company is currently funded through 2026 and isn’t planning to start a new study before then, he said.

Though the trial missed its primary endpoint, Immunic pointed to other positive results from the study to suggest that IMU-838 could succeed in phase 3.\n Immunic’s lead oral small molecule has failed to hit the primary endpoint of a phase 2 trial in patients with progressive multiple sclerosis (PMS), but the New York biotech is pointing to other measures from the study as reason to continue developing the asset.Vidofludimus calcium, called IMU-838 by Immunic, showed only a “modest benefit” of 5% over placebo at improving the yearly rate of percent brain volume change in patients with PMS after the approximately two-year treatment period, Immunic said in an April 30 press release. This measure of brain volume change is the primary endpoint of the still-ongoing phase 2 Calliper trial, which has enrolled 467 patients with PMS. The treatment period followed patients for about 120 weeks, while an open-label extension will continue for up to eight years.The company did not disclose the statistical significance of the result, but a spokesperson confirmed that the trial missed its primary goal.During an April 30 webinar about the top-line results, Immunic CEO Daniel Vitt, Ph.D., dismissed the primary endpoint, saying the measure had fallen behind current scientific advancements.\"Nobody should care at all about whole brain volume change anymore,\" Vitt said. \"This is a little bit of a leftover of our design of a study we did five years ago.\"Though IMU-838 had a marginal effect on reducing whole brain volume loss, patients receiving the drug candidate did see a 20% reduction in loss of the thalamus, Immunic said. \"Change in thalamic volume is considered a more sensitive MRI atrophy marker,\" Immunic said in the release. \"Thalamic atrophy is prevalent in PMS and data has shown strong associations with clinical disability progression.\"Daily oral 45-mg doses of IMU-838 also reduced the risk of a patient experiencing an event that worsened their disability for at least 24 weeks by 20% compared to placebo, as measured by the Expanded Disability Status Scale (EDSS) score, Immunic reported. This figure jumped to 30% when focusing on the subset of patients with primary progressive multiple sclerosis (PPMS), whose disease did not start with the more common relapsing MS (RMS).The time to 24-week confirmed disability worsening based on EDSS is a secondary endpoint of the trial. Immunic did not disclose detailed data in its communications about the top-line results. Results for another secondary endpoint, an MRI measure of change in brain parenchymal fraction, have also not been disclosed yet.\"We ourselves have not seen all data from all endpoints yet and we will continue to receive them over the next weeks and months to come,\" Vitt said in the webinar.The frequency of adverse events in the group of patients receiving treatment was similar to the rate in the placebo group, according to the company\'s release.“We are particularly thrilled to see such a clinically meaningful effect in the PPMS population,” Vitt said in the release, noting “the relative risk of 24-week confirmed disability worsening events” would be the primary endpoint of a potential future phase 3 study.“We look forward to discussing these results with healthcare authorities to determine appropriate next steps for vidofludimus calcium in PMS,” Vitt added. Analysts from William Blair wrote April 30 that \"with usual cross-trial comparison caveats, in our view, vidofludimus calcium demonstrated a competitive risk/benefit profile\" compared to Genentech\'s Ocrevus (ocrelizumab), Novartis\' Mayzent (siponimod) and Sanofi\'s experimental tolebrutinib.\"The FDA appears to be focused on confirmed disability worsening endpoints where vidofludimus compares well in the PPMS population,\" the analysts wrote.Immunic\'s stock price dropped sharply after the data reveal, from $1.41 per share in the evening of April 29 to 99 cents per share by 2 p.m. EDT on April 30.William Blair analysts attributed this stock drop to concerns about Immunic\'s ability to fund a phase 3 trial for IMU-838; the company reported $35.7 million in cash and cash equivalents as of March 31, which is expected to fund the company through the third quarter of this year.\"We acknowledge the financing overhang is material and Immunic will likely need a partner and/or additional capital to fund future phase III studies,\" the analysts wrote.Most patients with MS have relapsing MS, where symptoms like numbness, fatigue, vision changes and brain fog periodically flare up. In progressive MS, these symptoms instead gradually worsen over time; flare-ups may still happen but are less common. Relapsing MS can develop into progressive MS over time, called secondary progressive MS, but about 10% of MS patients are diagnosed with primary progressive MS without first having any relapsing symptoms.IMU-838 is also currently in a pair of phase 3 trials in RMS, after previously hitting the primary endpoint of reducing brain lesions in a phase 2 trial. The phase 3 trials are analyzing the time to first symptom relapse as the primary endpoint.Immunic’s phase 3 RMS program recently underwent an interim futility analysis, the company said in October 2024, with the independent data monitoring committee determining that no changes to the trial protocol were needed.In addition to MS, Immunic is also testing IMU-838 in a phase 2 ulcerative colitis trial. The company has two other drug candidates in development, one clinical-stage asset for celiac disease and other gastrointestinal disorders and another preclinical gastrointestinal asset.This is, however, a more narrow focus for the biotech, which in 2023 revealed that it was deprioritizing development in psoriasis and castration-resistant prostate cancer and throwing its remaining resources to multiple sclerosis and celiac disease.