BioVersys Receives EMA Orphan Designation for the Combination of Alpibectir and Ethionamide for the Treatment of Tuberculosis

Ad hoc announcement pursuant to Art. 53 LR

BASEL, Switzerland, Aug. 27, 2025 (GLOBE NEWSWIRE) --

• Orphan designation is granted to drug candidates addressing life-threatening or chronically debilitating diseases affecting fewer than 5 in 10’000 EU residents
• EMA orphan status provides key incentives, including reduced fees, research and clinical protocol support, and 10-year EU market exclusivity

BioVersys AG (SIX: BIOV), a multi-asset, clinical stage biopharmaceutical company focusing on research and development of novel antibacterial products for serious life-threatening infections caused by multidrug-resistant (MDR) bacteria, announced today that the European Medicines Agency Committee for Orphan Medicinal Products (EMA COMP) has granted orphan designation for the combination of alpibectir and ethionamide (AlpE) for the treatment of tuberculosis (TB).

Tuberculosis is one of the leading causes of death by infectious diseases globally, and many existing treatments are becoming less effective due to growing drug resistance. Alpibectir, a small molecule acting through a novel mode of action, represents a totally new concept of overcoming resistance by significantly potentiating the activity of an existing antibiotic, ethionamide (Eto). It is being developed for pulmonary and meningeal TB.

EMA’s orphan designation follows a successful clinical Phase 2a proof of concept trial and FDA’s Orphan Drug Designation (ODD) granted in 2023. The orphan designation reflects the potential for AlpE to improve treatment options for patients who have TB and to overcome resistance to TB medicines. Alpibectir was identified in a successful public-private collaboration with GSK, the Pasteur Institute of Lille and the University of Lille. Currently, alpibectir is being studied in a Phase 2 trial in pulmonary TB in combination with first line TB drugs. This trial is being run within the European Union’s IMI2 UNITE4TB project with our partner GSK. In parallel, dosing in a Phase 2 trial for meningeal TB is anticipated to start in early 2026.

Dr. Glenn Dale, Chief Development Officer of BioVersys: “The EMA orphan designation validates our mission to deliver urgently needed therapies for TB patients, who face prolonged treatment durations and unacceptably high mortality rates. We are pleased that this effort continues to be supported by a public-private partnership.”

Dr. David Barros-Aguirre, VP and Head of Global Health Medicines R&D, GSK: “We are pleased with the progress being made with our partner BioVersys on the development of alpibectir for tuberculosis. The EMA orphan drug designation is an important step towards addressing drug resistance in TB treatment. This collaboration highlights GSK's commitment to working with partners to bring essential medicines and vaccines to those who need them.”

Clinical development of AlpE has been supported by several European grants, including the EU IMI2 TRIC-TB Project, the EU IMI2 UNITE4TB project and the European & Developing Countries Clinical Trials Partnership (EDCTP2 program).

About tuberculosis (TB)
Tuberculosis (TB) is one of the leading causes of death worldwide. Its causative agent is the bacterial pathogen Mycobacterium tuberculosis (Mtb). According to the latest 2024 WHO TB report, worldwide an estimated 10.8 million people developed TB in 2023 and an estimated 1.25 million died from TB. WHO estimates that there were 400’000 new cases with resistance to rifampicin, most of them were multi-drug resistant (MDR). MDR-TB remains a public health crisis and a health security threat. Worldwide, only 68% of MDR-TB patients are currently successfully treated.¹ In the modern world of global travel, and ease with which infections spread, it is very worrying to note that two-thirds of the global total of TB cases were recorded in eight countries: India (26%), Indonesia (10%), China (6.8%), the Philippines (6.8%), Pakistan (6.3%), Nigeria (4.6%), Bangladesh (3.5%) and the Democratic Republic of the Congo (3.1%). Furthermore, 3.2% of all new and 16% of reoccurring TB cases were MDR/RR-TB.

About UNITE4TB
UNITE4TB is one of nine projects within the AMR Accelerator. Working across a global clinical trials network, UNITE4TB conducts regulatory standard Phase 2 clinical trials to accelerate clinical evaluation of novel drugs and combinations of drugs for tuberculosis (TB). Innovative adaptive trial designs, treatment response biomarkers, pharmacokinetic-pharmacodynamic models and Artificial Intelligence / Deep Learning techniques are integrated across study protocols, deploying cutting-edge methodologies to find antibiotic regimens with the highest likelihood of improved clinical efficacy.

This project has received funding from the Innovative Medicines Initiative 2 Joint Undertaking (JU) under grant agreement No 101007873. The JU receives support from the European Union’s Horizon 2020 research and innovation programme and EFPIA, Deutsches Zentrum für Infektionsforschung e. V. (DZIF), and Ludwig-Maximilians-Universität München (LMU). EFPIA/AP contribute 50% of funding, whereas the contribution of DZIF and the LMU University Hospital Munich has been granted by the German Federal Ministry of Education and Research.

About ENABLE
The ENABLE study will evaluate the 14 day EBA, safety, tolerability and dose-response of AlpE and AlpE with rifampicin (R), pyrazinamide (Z), and ethambutol (E) in adults with newly diagnosed, drug-susceptible pulmonary TB. The over-arching objective is to optimise the dose of AlpE and to evaluate the safety of the AlpE+RZE regimen for future evaluation as an alternative regimen for isoniazid mono-resistant TB. The data from the ENABLE study and the bEto study (NCT05473195) will support the evaluation of the optimal dose combination of AlpE to move forward into late phase studies.

About the Innovative Medicines Initiative
The Innovative Medicines Initiative (IMI) IMI is a partnership between the European Union and the European pharmaceutical industry, represented by the European Federation of Pharmaceutical Industries and Associations (EFPIA). It was set up to improve health by speeding up the development of, and patient access to, the next generation of medicines, particularly in areas where there is an unmet medical or social need. It works by facilitating collaboration between the key players involved in healthcare research, including universities, pharmaceutical companies, other companies active in healthcare research, small and medium-sized enterprises (SMEs), patient organisations, and medicines regulators. This approach has proven highly successful, and IMI projects are delivering exciting results that are helping to advance the development of urgently-needed new treatments in diverse areas. IMI projects are now managed by the Innovative Health Initiative (IHI), which builds on the successes of IMI and is a cross-sectoral public-private partnership involving a wider range of health industries.

• More info on IHI: https://www.ihi.europa.eu/   
• Twitter: @IHIEurope
• More info on IMI AMR accelerator: https://amr-accelerator.eu/project/tric-tb/
• Working Group of new TB drugs: https://www.newtbdrugs.org/pipeline/compound/bvl-gsk098

About EMA orphan designation
EMA orphan designation grants companies a number of benefits: reduced regulatory fees, research grants, clinical protocol support and 10-year market exclusivity in the EU. Orphan designation is granted to drugs/biologics intended to address life-threatening or chronically debilitating diseases that affect fewer than 5 in 10,000 EU residents.
See: https://www.ema.europa.eu/en/human-regulatory-overview/orphan-designation-overview

About GSK
GSK is a science-led global healthcare company. For further information please visit https://www.gsk.com/en-gb/about-us/

About BioVersys
BioVersys AG is a multi-asset, clinical stage biopharmaceutical company focused on identifying, developing and commercializing novel antibacterial products for serious life-threatening infections caused by multi-drug resistant (“MDR”) bacteria. Derived from the company’s two internal technology platforms (TRIC and Ansamycin Chemistry), candidates are designed and developed to overcome resistance mechanisms, block virulence production and directly affect the pathogenesis of harmful bacteria towards the identification of new treatment options in the antimicrobial and microbiome fields. This enables BioVersys to address the high unmet medical need for new treatments against life-threatening resistant bacterial infections and bacteria-exacerbated chronic inflammatory microbiome disorders. The company’s most advanced research and development programs address nosocomial infections of Acinetobacter baumannii (BV100, Phase 3), and tuberculosis (alpibectir, Phase 2, in collaboration with GlaxoSmithKline (GSK) and a consortium of the University of Lille, France). BioVersys is located in the biotech hub of Basel, Switzerland.

BioVersys contact 
Hernan Levett, CFO, Tel. +41 61 633 22 50; Mail: hernan.levett@bioversys.com
For media: media@bioversys.com 
Website: www.bioversys.com
https://twitter.com/Bioversys 
https://www.linkedin.com/company/bioversys-ag

¹ Global tuberculosis report 2024. Geneva: World Health Organization; 2024. Licence: CC BY-NC-SA 3.0 IGO.

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