Source reference for publication: https://advanced.onlinelibrary.wiley.com/doi/10.1002/advs.202514128
ALS (amyotrophic lateral sclerosis) is a rapidly progressive neurodegenerative disease that leads to the destruction of motor neurons. As the disease advances, affected individuals increasingly lose their ability to move and, in far too many cases, ultimately succumb to respiratory failure. Despite decades of intensive research, no therapy to date has been able to halt or reverse the course of ALS. In collaboration with Smita Saxena, InnoMedica now presents initial preclinical data on the treatment of ALS with Talineuren (TLN) in this publication. The observed beneficial effects of liposomal GM1 on mitochondrial function, synaptic integrity, inflammatory processes, and toxic protein aggregation highlight TLN’s potential as a promising candidate for future clinical investigation in ALS.
Authors of the publication: Federica Pilotto, Tristan Dellazizzo Toth, Silvano Bond, Alexander Schmitz, Rim Diab, Sara Y. Ngo Tenlep, Brian Mooney, Silvia Erni, Martina Schobesberger, Olivier Scheidegger, Camille Peitsch, Smita Saxena
Publication title: Engineered GM1 Intersects Between Mitochondrial and Synaptic Pathways to Ameliorate ALS Pathology
Advanced Science 2026, 0:e14128; doi.org/10.1002/advs.202514128
Source reference for publication: TLD-1, a Novel Liposomal Doxorubicin, in Patients with Solid Tumors: Comparative Pharmacokinetics and Final Results of a Multicenter Phase 1 Study (SAKK 65/16) | Clinical Pharmacokinetics
In collaboration with the Swiss Cancer Institute, InnoMedica conducted a dedicated clinical research programme in which a novel liposomal formulation of doxorubicin (TLD-1) was directly compared with the established liposomal reference product, Caelyx®. The study demonstrated that TLD-1 remains in systemic circulation for a longer period and releases the active substance from the liposomes in a more uniform manner than Caelyx®. These findings confirm a core hypothesis consistently advanced by InnoMedica: that differences in the size and structural properties of liposomal drug formulations can have a significant impact on patient outcomes. The resulting publication is therefore of particular scientific value, as it represents a rare and direct comparison of two liposomal nanomedicines in humans.
Authors of the publication: Marian Klose, Ilaria Colombo, Katrin Gobat, Kira-Lee Koster, Simon Haefliger, Manuela Rabaglio, Sara Bastian, Michael Schwitter, Ursina Zürrer-Härdi, Katrin Eckhardt, Stefanie Hayoz, Stefan Halbherr, Cristiana Sessa, Robin Michelet, Anna M. Mc Laughlin, Dagmar Hess, Anastasios Stathis, Charlotte Kloft, Markus Joerger
Publication title: TLD-1, a Novel Liposomal Doxorubicin, in Patients with Solid Tumors: Comparative Pharmacokinetics and Final Results of a Multicenter Phase 1 Study (SAKK 65/16)
Clinical Pharmacokinetics 2025; doi.org/10.1007/s40262-025-01588-z
Source reference for publication: PET imaging for non-invasive monitoring of 89Zr-Talidox delivery to the brain following focused ultrasound-mediated blood-brain barrier opening – ScienceDirect
This paper reports on a radioactively labelled formulation of liposomal doxorubicin, Talidox. Using state-of-the-art PET imaging, Rafael de Rosales and his team were able, for the first time, to non-invasively track the biodistribution of Talidox in vivo over several days. The findings provide important methodological and technological insights for the further development of brain-targeted drug delivery strategies. In addition, the publication highlights the potential of radioactively labelled Talidox as a tool for modern precision and personalised medicine.
Authors of the publication: Aishwarya Mishra, Chris Payne, Amaia Carrascal-Minino, Kavitha Sunassee, Stefan Halbherr, Antonios N. Pouliopoulos, Rafael T. M. de Rosales
Publication title: PET imaging for non-invasive monitoring of 89Zr-Talidox delivery to the brain following focused ultrasound-mediated blood-brain barrier opening
Journal of Controlled Release 2025; 387: 114183. doi.org/10.1016/j.jconrel.2025.114183
Source reference for publication: TLD: An optimized liposomal doxorubicin formulation with small vesicle size for improved anti-tumor efficacy – ScienceDirect
This preclinical work summarises more than a decade of research on Talidox (TLD), a liposomal formulation of doxorubicin with particularly small particle size developed by InnoMedica. The study provides a detailed analysis of how liposome structure, size, and drug loading influence pharmacological behaviour in cellular systems and animal models. Across multiple cancer models, distinct differences were observed compared with established liposomal doxorubicin formulations, both in terms of cellular uptake and tolerability. Taken together, these findings establish a robust scientific foundation for the further clinical development of Talidox and underscore its potential advantages relative to existing doxorubicin-based therapies.
Authors of the publication: Sime Brkic, Silvia Erni, Younes Louaguenouni, Marianna Carone, E. Henrik Peters, Andreas Schreiber, Andreas Zumbuehl, Alberto Gabizon, Stéfan Halbherr, Camille Peitsch
Publication title: TLD: An optimized liposomal doxorubicin formulation with small vesicle size for improved anti-tumor efficacy
European Journal of Pharmaceutical Sciences 2025; 213: 107246 doi.org/10.1016/j.ejps.2025.107246
Source reference for publication: Safety and tolerability of intravenous liposomal GM1 in patients with Parkinson disease: A single-center open-label clinical phase I trial (NEON trial) | PLOS Medicine
This publication presents the initial results of the NEON Phase I trial. The study investigated the safety and tolerability of Talineuren, a liposomal formulation of the endogenous GM1 molecule, in patients with Parkinson’s disease. Weekly intravenous infusions were generally well tolerated. Observed infusion-related reactions were temporary and predominantly mild, and could be effectively managed through adjustment of the infusion rate. In addition, the study documented the biodistribution and metabolism of Talineuren, thereby providing important foundational pharmacokinetic data to support further clinical development.
Authors of the publication: Stefan Halbherr, Stefanie Lerch, Sebastian Bellwald, Petra Polakova, Bettina Bannert, Marie Roumet, Roch-Philippe Charles, Martin A. Walter, Corrado Bernasconi, Valérie Lisa Halbherr, Camille Peitsch, Pascal C. Baumgartner, Céline Kaufmann, Vanessa Aires, Heinrich P. Mattle, Alain Kaelin-Lang, Andreas Hartmann, Michael Schuepbach
Publication title: Safety and tolerability of intravenous liposomal GM1 in patients with Parkinson disease: A single-center open-label clinical phase I trial (NEON trial)
PLOS Medicine 2025; 22(5): e1004472. doi:10.1371/journal.pmed.1004472
Source reference for the publication: Understanding the In Vitro-In Vivo Nexus: Advanced correlation models predict clinical performance of liposomal doxorubicin – ScienceDirect
This work demonstrates that the behaviour of a liposomal doxorubicin formulation in the human body can be predicted in the laboratory with remarkable accuracy. By combining advanced in vitro assays with clinical data from a Phase I study, the researchers were able to demonstrate a close correlation between in vitro drug release profiles and pharmacokinetic behaviour in the bloodstream. The findings clearly show that even small differences in liposome structure and surface characteristics have measurable effects on circulation time and drug release. As such, the study provides an important methodological foundation for the more targeted development of liposomal drug formulations and for their reliable and comparable evaluation in future research and development programmes.
Authors of the publication: Kennard Gan, Zhuoxuan Li, PhyoMaw Darli, Teresa Wong, Harshvardhan Modh, Petra Gottier, Stéfan Halbherr, Matthias G. Wacker
Publication title: Understanding the In Vitro-In Vivo Nexus: Advanced correlation models predict clinical performance of liposomal doxorubicin
International Journal of Pharmaceutics 2024; 654: 123942. doi:10.1016/j.ijpharm.2024.123942
Source reference for the publication: Population pharmacokinetics of TLD-1, a novel liposomal doxorubicin, in a phase I trial | Cancer Chemotherapy and Pharmacology
This study investigates the biodistribution and metabolism of TLD-1, a newly developed liposomal formulation of doxorubicin, in patients with advanced solid tumours. Using advanced pharmacokinetic modelling, the study demonstrates that TLD-1 remains in systemic circulation for an extended period, clearly distinguishing it from existing liposomal doxorubicin formulations. The analysis provides a detailed characterisation of the release of the active substance from the liposomes as well as of its principal degradation product. Together, these findings establish an important scientific foundation for the targeted optimisation of dosing and administration strategies in future clinical studies.
Authors of the publication: Anna M. Mc Laughlin, Dagmar Hess, Robin Michelet, Ilaria Colombo, Simon Haefliger, Sara Bastian, Manuela Rabaglio, Michael Schwitter, Stefanie Fischer, Katrin Eckhardt, Stefanie Hayoz, Christoph Kopp, Marian Klose, Cristiana Sessa, Anastasios Stathis, Stefan Halbherr, Wilhelm Huisinga, Markus Joerger, Charlotte Kloft
Publication title: Population pharmacokinetics of TLD-1, a novel liposomal doxorubicin, in a phase I trial
Cancer Chemotherapy and Pharmacology 2024; 94: 349–360. doi:10.1007/s00280-024-04679-z
Source reference for publication: TLD-1, a novel liposomal doxorubicin, in patients with advanced solid tumors: Dose escalation and expansion part of a multicenter open-label phase I trial (SAKK 65/16) – European Journal of Cancer
In this clinical study, InnoMedica, in collaboration with the Swiss Cancer Institute SAKK, evaluated a newly developed liposomal anticancer drug, TLD-1, in patients with advanced solid tumours. The active substance is encapsulated in exceptionally small liposomes, a design intended to enhance tumour targeting while limiting exposure of healthy tissues, particularly the heart. In the Phase I study, TLD-1 demonstrated a favourable safety profile and showed initial indications of clinical activity.
Authors of the publication: Ilaria Colombo, Kira-Lee Koster, Lisa Holer, Simon Haefliger, Manuela Rabaglio, Sara Bastian, Michael Schwitter, Katrin Eckhardt, Stefanie Hayoz, Anna M. Mc Laughlin, Charlotte Kloft, Marian Klose, Stefan Halbherr, Christian Baumgartner, Cristiana Sessa, Anastasios Stathis, Dagmar Hess, Markus Joerger
Publication title: TLD-1, a novel liposomal doxorubicin, in patients with advanced solid tumors: Dose escalation and expansion part of a multicenter open-label phase I trial (SAKK 65/16)
European Journal Cancer 2024;201: 113588. doi:10.1016/j.ejca.2024.113588
Source reference for the publication: Hyperlipidemia-induced cholesterol crystal production by endothelial cells promotes atherogenesis | Nature Communications
InnoMedica showed that specific liposomally formulated active compounds can slow this pathological process and improving vascular function. In a mouse model, treatment led to a marked reduction in deposit formation, pointing to a novel and promising approach for the prevention of cardiovascular disease.
Authors of the publication: Yvonne Baumer, Sara McCurdy, Tina M. Weatherby, Nehal N. Mehta, Stefan Halbherr, Pascal Halbherr, Noboru Yamazaki, William A. Boisvert
Publication title: Hyperlipidemia-induced cholesterol crystal production by endothelial cells promotes atherogenesis.
Nature Communications 2017; 8: 1129. doi:10.1038/s41467-017-01186-z
