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Scientific Communications

ENCALS 2026 – European Network to Cure ALS

Madrid, Spain – 24-26 June 2026

“SEALS”: A Randomized, Double-Blind, Placebo-Controlled Phase 2 Study Evaluating Efficacy And Safety Of NX210c In Amyotrophic Lateral Sclerosis (ALS) Patients – Preliminary Results

Presented by Dr Emilien Bernard, Hospices Civils de Lyon, Coordinating Investigator of SEALS study, during TRICALS Satellite at the ENCALS meeting: insights in recent and upcoming trial results.

Download TRICALS Satellite slide deck

POSTER #PO108 presented by Dr Emilien Bernard, Hospices Civils de Lyon, Coordinating Investigator of SEALS study, and Dr Annette Janus (Chief Medical Officer – Axoltis), Sébastien Marie (Clinical Operations Director – Axoltis) and Juliette Ledouce (Clinical Project Manager – Axoltis) at the ENCALS meeting 2026

Download Poster #PO108

SEALS – Study Design Summary

The SEALS Phase 2 trial is a double-blind, randomized, placebo-controlled study in ALS stratifying patients by predefined prognostic biomarker level (neurofilament light, NfL) to improve differentiation of treatment responsiveness challenged by heterogeneity.
Patients were randomized (3:3:2) in three arms to receive NX210c at either of two dose levels (5 mg/kg or 10 mg/kg) or placebo, administered by 10-minute intravenous infusion 3x weekly for 4 weeks;
a core study followed patients for 3 months thereafter, with timepoints at week 4 (end of treatment), week 6, week 10 and week 16. Primary endpoints included NfL and Qalb (Quotient of albumin concentration in CSF/Blood) with secondary endpoints expanding the analysis to clinical outcomes (e.g. ALSFRS-R (the rating scale for functional assessment) and biomarker indicators.

Preliminary Results

  • Thanks to the ACT4ALS/MND French network, 82 patients were randomized at 16 investigational sites from Nov. 2024 to Nov. 2025, with all patients reaching the end of treatment timepoint.
  • To date, the safety and tolerability profile of NX210c has been good in this patient population.
  • A dose dependent trend in relative change in serum NfL from baseline level has already been identified at week 4 (End of Treatment), although a difference between groups has not yet shown statistical significance.
  • A higher proportion of patients in both active dose groups demonstrated more than 10% decrease in serum NfL at week 6 (15% in the placebo arm, 22.6% in the 5mg/kg arm, 26.7% in the 10mg/kg arm), potentially predicting an effect on survival.
  • A higher proportion of patients in each of the active dose arms also had the combination of more than 10% decrease in serum NfL AND a decrease in Qalb (5.3% in the placebo arm, 9.7% in the 5mg/kg arm, 23.3% in the 10mg/kg arm) at week 6.
  • A reduced ALSFRS-R decline rate was noticed in the active dose groups (-0.67 point/month in average at 5mg/kg, -0.9 point/month at 10mg/kg) compared to placebo (-1.14 point/month) with notable motor subscore contributions – suggesting not only an effect on biomarkers but on those of clinical relevance.

Conclusion

Encouraging week 6 safety and early efficacy signals support continued clinical development of NX210c in ALS.
The full dataset will be harnessed in the next months to:

  • Amplify the placebo group with 62 digital twins’ generation.
  • Characterize/profile the potential responder patients
  • Determine the optimal dose and therapeutic regimen (frequency of course of treatment) for the next clinical development steps.

Acknowledgments

Axoltis Pharma sincerely thanks all the participants and contributors to SEALS, first and foremost the patients and their caregivers, alongside investigators, their clinical teams, as well as the CRO and service providers, with whom we are privileged to have been able to realize SEALS. We are also very glad to have the support of ARSLA, Les Invincibles and Swiss ALS Foundation.

NX210c, A Promising Drug Candidate For Neurodegenerative Diseases

Presented by Dr Annette Janus (Chief Medical Officer – Axoltis) and the Axoltis Clinical staff. The Poster #PO101 gives Details about the Mecanism of Action and Properties of NX210c.

NX210c is a chemically synthesized Thrombospondin type 1 repeat (TSR1) analog that demonstrates blood brain barrier (BBB) restorative capacity by increasing the dominant tight junction protein expressed on neurovascular endothelium (claudin-5). It also exhibits neuroprotective properties and neurotransmission enhancement – showing functional recovery in preclinical models of CNS diseases, including ALS.

Download Poster #PO101