EQS-News: AL-S Pharma AG
/ Key word(s): Conference/Study results
AL-S Pharma Reports New AP-101 Phase 2 Weight Data and Outlines Phase 3 Clinical Trial in ALS at 2026 NEALS Annual Meeting New Phase 2 post-hoc analyses demonstrate greater body-weight preservation with AP-101 compared with placebo in patients with ALS Findings reinforce clinical relevance of body weight as a potential surrogate marker of clinical response; weight loss is closely associated with ALS disease progression and poorer outcomes Planned confirmatory Phase 3 clinical trial expected to initiate in 2027 ZURICH, Switzerland - October 26, 2026 - AL-S Pharma AG ("AL-S Pharma" or "the Company"), a clinical-stage biotechnology company discovering and developing human antibodies that target misfolded proteins implicated in amyotrophic lateral sclerosis (ALS), today announced new analyses from its global Phase 2 clinical trial (AP101-02) evaluating the company's Phase 3-ready lead program, AP-101, in patients with sporadic ALS and superoxide dismutase 1 (SOD1)-ALS. The new findings demonstrate greater preservation of body weight in patients treated with AP-101 compared with placebo. Weight loss has been associated with shorter survival, while longitudinal changes in weight may reflect several aspects of disease progression, including nutritional status, metabolic changes, and muscle loss. The results will be presented by Angela Genge, M.D., FRCP(C), eMBA, chief medical officer of AL-S Pharma, during the NEALS Affiliated Trial Session at the 2026 Network of Excellence for ALS (NEALS) Meeting taking place October 27-30, 2026, in Clearwater, Florida, and online. "Preservation of body weight is particularly relevant in ALS, where weight loss is closely associated with disease progression and poorer outcomes," commented Dr. Genge. "These new analyses add an important clinical perspective to our Phase 2 findings, demonstrating a consistent trend toward greater weight preservation with AP-101 across different ALS patient populations. The new analyses provide additional evidence that rate of weight loss is an important additional measure of clinical response alongside established functional, respiratory, and survival outcomes. We are eager to progress the confirmatory Phase 3 study with the intention of delivering a critically needed new treatment option targeting misfolded SOD1 to people living with ALS." New Phase 2 Analyses Demonstrate Greater Body-Weight Preservation with AP-101 In the sporadic ALS cohort, mixed-model repeated-measures analyses showed a progressive separation in body-weight trajectories favoring AP-101, with an adjusted between-group difference of 2.25 kg at Week 24. In the SOD1-ALS cohort, the analysis demonstrated a 4.82 kg difference favoring AP-101 at the 21-day follow-up after the randomized treatment period (p = 0.0092). Additional post-hoc analyses examined participants with sporadic ALS according to baseline cerebrospinal fluid (CSF) levels of misfolded SOD1. Among participants in the highest tertile of baseline misfolded SOD1, AP-101 treatment was associated with greater preservation of body weight compared with placebo, with a 4.59 kg difference at the 21-day follow-up (p = 0.0010). The global AP-101 Phase 2 clinical trial met its primary endpoint related to safety and tolerability. In addition to greater body-weight preservation in AP-101-treated participants, study findings provide additional evidence of clinically meaningful disease modification and prolonged survival. This is supported by reductions in key neuroaxonal injury biomarkers, serum neurofilament light chain (NfL) and cerebrospinal fluid phosphorylated neurofilament heavy chain (pNfH) after six months of treatment in the per protocol population. Adverse events were found to be similar to those in the placebo group, with no reported antibody responses induced by AP-101. Presentation Details
Planned NEALS-Affiliated Phase 3 Confirmatory Trial of AP-101 Featured in Poster Presentation The planned primary analysis will employ a joint statistical model incorporating longitudinal changes in the ALS Functional Rating Scale-Revised (ALSFRS-R) and survival-related events, defined as death or permanent assisted ventilation. This approach is intended to evaluate whether AP-101 has a significant effect on function and/or survival. Additional assessments will include body weight, respiratory function, King's staging, quality of life, safety, pharmacokinetics, immunogenicity, and CSF and plasma biomarkers. The inclusion of serial body-weight measurements will enable further prospective evaluation of the weight-preservation findings observed in the Phase 2 AP-101 clinical trial. Eligible participants will have an ALS diagnosis based on Gold Coast or revised El Escorial criteria, symptom onset within 24 months, and slow vital capacity of at least 60% at screening. Participants completing the 12-month double-blind treatment period may enter a further 12-month open-label extension. The Phase 3 trial is planned to initiate in 2027. AP-101 has received Orphan Drug designations from the U.S. Food and Drug Administration, the European Medicines Agency, and Swissmedic. Poster Details
About AP101-02 AP101-02 was conducted in the U.S., Canada, the U.K., European Union, and South Korea. More information about the clinical trial can be accessed on www.clinicaltrials.gov. About Amyotrophic Lateral Sclerosis and SOD1 About AL-S Pharma AG Media Contact(s) AL-S Pharma MC Services AG
26.10.2026 CET/CEST Dissemination of a Corporate News, transmitted by EQS News - a service of EQS Group. |
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