Leriglitazone (NEZGLYAL) for cerebral adrenoleukodystrophy (cALD)
Minoryx Therapeutics, partnered with Neuraxpharm, has filed a Marketing Authorisation Application (MAA) with the EMA for leriglitazone, targeting both paediatric and adult male patients with cALD.
The EMA has validated the submission and it's now under CHMP review. This action is supported by the 96-week, open-label NEXUS study, in which all 20 evaluable patients remained clinically stable and 35% met disease-arrest criteria — a notable improvement compared to the ~10% natural arrest rate (p<0.05).
No treatment discontinuations occurred owing to adverse events. The MAA also includes data from the placebo-controlled ADVANCE study — demonstrating reductions in cerebral lesion progression in adults — as well as real-world evidence from compassionate use programmes.
Ongoing trials include the CALYX trial in adults with cALD and the TREE study in paediatric Rett syndrome.
If approved, leriglitazone would be the first pharmacological option for this devastating rare disease.
Getacatetide (CY-101) for Wnt/β-catenin–driven cancers
Cytovation has secured the INN “getacatetide” for its bifunctional peptide CY-101, which targets the Wnt/β-catenin pathway by membranolytic inhibition — exposing tumour neoantigens and overcoming immune evasion.
Phase I CICILIA data revealed early antitumour activity in dysregulated Wnt-pathway tumours.
Preclinical findings at AACR 2025 highlight its promise in adrenocortical carcinoma (ACC), colorectal cancer and hepatocellular carcinoma.
A Phase II ACC trial, scheduled for early 2026, is being launched under a partnership with Cancer Research UK and the Norwegian Cancer Society, with initial readouts expected by year-end.
Etherna’s bioreducible lipid nanoparticles for mRNA delivery
Etherna has announced a major platform breakthrough: its proprietary bioreducible LNPs can deliver mRNA efficiently to bone marrow hematopoietic stem and progenitor cells (HSPCs) and T cells — a key advancement for targeted gene therapies.
Preclinical data show more than 90% transfection of HSPCs and robust CAR‑T expression in humanised mouse models.
The platform outperformed clinical benchmark LNPs by at least 5-fold in rodent and non-human primate tests, while maintaining strong tolerability.
These findings mark an important step toward extra‑hepatic mRNA delivery, with new therapeutic potential in in vivo gene editing, protein replacement and immunotherapy outside the liver.
In a nutshell
- Leriglitazone: Poised to become the first-ever pharmacological treatment for cALD, backed by compelling trial data.
- Getacatetide: Unlocks a novel Wnt/β-catenin inhibition mechanism and is set for Phase II ACC trials under strong nonprofit collaboration.
- Etherna’s LNPs: Overcoming the traditional liver-targeting limits of mRNA therapy, enabling delivery to bone marrow and immune cells—a transformative new delivery paradigm.
These developments showcase the rapid evolution of drug research approaches— spanning small molecules, peptides and mRNA platforms — aimed at solving previously intractable medical challenges through strategic innovation and collaboration.