Ethris Announces Promising Phase 1 Clinical Data for mRNA Therapeutic ETH47 in Asthma Treatment
Ethris GmbH, a biotechnology pioneer in the development of next-generation RNA therapeutics and vaccines, has announced promising topline results from its completed Phase 1 clinical trial of ETH47, its lead mRNA-based therapeutic candidate, designed to treat uncontrolled asthma. The study, which involved healthy volunteers, has highlighted the potential of ETH47 to address the upstream triggers of asthma exacerbations, which are often linked to viral infections in the respiratory tract. ETH47 encodes interferon lambda (IFNλ), a protein essential for antiviral immune responses in the respiratory system.
This Phase 1 clinical trial represents a significant milestone for Ethris, as the data validates the company’s novel, targeted RNA therapeutic approach. By leveraging their SNIM® and SNaP® proprietary technology platforms, Ethris was able to successfully deliver mRNA to the respiratory tract, inducing a local production of antiviral interferon-stimulated genes (ISGs) after nasal administration. This positive clinical outcome offers the first clinical evidence for the efficacy and safety of this cutting-edge RNA-based treatment and lays the groundwork for the future development of ETH47 as a therapeutic for asthma.
Dr. Thomas Langenickel, Chief Medical Officer at Ethris, presented the promising topline results at the mRNA-Based Therapeutics Summit Europe in Frankfurt, Germany, on January 30th, 2025. The company’s innovative approach represents a significant leap forward in asthma treatment, and the early results suggest ETH47 could offer a novel, safe, and effective solution for patients.
Promising Data Highlighting the Potential of ETH47
These positive Phase 1 results reinforce ETH47’s potential as a transformative treatment for asthma patients, while also validating Ethris’ proprietary technology platforms—SNIM® and SNaP®—as effective delivery mechanisms for mRNA therapeutics to the respiratory tract. According to Dr. Langenickel, “This significant milestone demonstrates the ability of our technology to deliver precisely designed mRNA candidates to the respiratory tract, facilitating production of the encoded protein and engagement with the intended target.”
The promising results from this study are especially significant for asthma patients, a population that currently lacks effective treatments to address asthma exacerbations triggered by viral infections. In the future, ETH47 could provide a targeted and highly effective option for patients, helping to prevent viral-induced asthma attacks and improving overall disease management.
The Phase 1 study demonstrated several key outcomes that underscore ETH47’s potential as a therapeutic candidate for asthma. Dr. Langenickel further emphasized, “We are excited about advancing ETH47 into Phase 2 trials in 2025, and we are committed to exploring its broader potential to transform care for patients with asthma and other respiratory conditions.”
Study Design and Key Findings
The Phase 1 clinical trial was a dose-ranging study that evaluated the safety, tolerability, and pharmacodynamics of ETH47 after nasal administration in 40 healthy participants. This was a single-center, randomized, double-blind, placebo-controlled trial. The primary objectives of the study were to assess the safety and tolerability of ETH47 at varying dose levels, as well as to measure the ability of the therapeutic to engage the intended target and induce the desired antiviral immune response.
The results from the Phase 1 study showed that ETH47 was well tolerated across all dose levels, with no serious adverse events and no adverse events leading to study discontinuation. Importantly, the study demonstrated that ETH47 had no detectable systemic bioavailability, meaning that neither the mRNA, the produced IFNλ protein, nor the proprietary lipidoid delivery compound reached the bloodstream. This suggests that the therapy acted locally in the respiratory tract without causing systemic side effects or off-target effects.
Key results from the study include:
- Safety and Tolerability: ETH47 was shown to be generally safe and well tolerated, with no serious or severe adverse events observed. The safety profile of the mRNA therapeutic was favorable, and there were no reports of adverse events leading to participant discontinuation. The study also found no systemic exposure to the mRNA or its components, indicating a low risk of unwanted side effects.
- Target Engagement: The study demonstrated that ETH47 induced a clear, dose-dependent increase in the production of IFNλ in the nasal lining fluid, surpassing the predicted therapeutic levels. Additionally, the treatment activated the expression of interferon-stimulated genes (ISGs) in nasal brush samples. These findings confirm that ETH47 effectively engaged its target in the respiratory tract, which supports the proposed mechanism of action for the therapeutic.
- Pharmacodynamics: The ability of ETH47 to induce the local production of antiviral proteins and stimulate the immune system via ISGs was a key indication of its pharmacodynamic activity. These results suggest that the mRNA therapeutic could play a critical role in modulating immune responses to viral infections, such as rhinovirus, a major trigger of asthma exacerbations.
Based on the positive results of this Phase 1 trial, Ethris is preparing for the next phase of clinical development. The company has already filed a Clinical Trial Application (CTA) for a Phase 2a rhinovirus challenge study, which will assess the effectiveness of ETH47 in asthma patients. This Phase 2a trial, which is expected to begin in Q2 2025, will focus on evaluating the potential of ETH47 to reduce lower respiratory symptoms in asthma patients following rhinovirus infection. Rhinovirus infections are one of the most common triggers of asthma exacerbations, making this Phase 2a study a critical step in determining the therapeutic’s potential to improve asthma management.
The successful Phase 1 trial of ETH47 marks an important step in the ongoing development of RNA-based therapies for asthma and other respiratory conditions. The results demonstrate that mRNA therapeutics, delivered via novel platforms like SNIM® and SNaP®, can be an effective and safe option for targeting the respiratory tract. By leveraging this technology, Ethris has the potential to create transformative treatments for patients with asthma, chronic obstructive pulmonary disease (COPD), and other viral-driven respiratory disorders.
The planned Phase 2 trials will be an essential step in determining whether ETH47 can achieve its goal of reducing asthma symptoms in patients. If successful, ETH47 could represent a game-changing approach to treating asthma, addressing the viral infections that often trigger exacerbations and providing a targeted immune response in the respiratory tract.
Looking beyond asthma, the potential applications of ETH47 and other mRNA-based therapies extend to a wide range of respiratory diseases. By targeting the respiratory tract with RNA therapeutics, Ethris is paving the way for the next generation of treatments that could have a significant impact on respiratory health worldwide.
Ethris is committed to advancing its pipeline of mRNA therapeutics and remains focused on bringing innovative treatments to market that address the unmet needs of patients suffering from asthma and other respiratory conditions. The company’s leadership in RNA therapeutics, combined with its proprietary delivery technologies, places Ethris at the forefront of a promising new era in respiratory medicine.
As the company looks ahead to Phase 2 trials, Ethris is optimistic about the future of ETH47 and its potential to revolutionize the way asthma and other viral respiratory conditions are treated.
