Additional Phase 2 Data of AL-S Pharma’s Lead Program AP-101 Further Demonstrate Clinically Meaningful Disease Modification and Prolonged Survival in ALS

Additional Phase 2 Data of AL-S Pharma’s Lead Program AP-101 Further Demonstrate Clinically Meaningful Disease Modification and Prolonged Survival in ALS

Results supported by key neuroaxonal injury biomarkers pNfH and NfL after six months of treatment 

AP-101 is an investigational human-derived antibody therapeutic that selectively targets the misfolded, toxic form of SOD1 to disrupt progressive spread of ALS 

Data featured in oral presentation at the AD/PD™ 2026 International Conference on Alzheimer’s and Parkinson’s Diseases 

Preparations underway to advance AP-101 into confirmatory Phase 3 clinical trial with initiation aimed for late 2026
 

ZURICH, Switzerland — March 19, 2026 — AL-S Pharma AG, a clinical-stage biotechnology company discovering and developing human antibodies that target misfolded proteins implicated in amyotrophic lateral sclerosis (ALS), today announced the presentation of new clinical data from the global Phase 2 clinical trial (AP-101-02) evaluating the company’s lead program, AP-101, in patients with ALS. AP-101 is an investigational human-derived antibody directed against misfolded superoxide dismutase 1 (SOD1). AP-101 is designed to inhibit the spread of SOD1 pathology in the central nervous system of people living with ALS, helping the body’s immune system clear these harmful proteins.¹

The global Phase 2 clinical trial met its primary endpoint related to safety and tolerability. New results provide additional evidence of clinically meaningful disease modification and prolonged survival 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. Adverse events were comparable to placebo, and no AP-101-induced antibody responses were reported.

The AP-101-02 clinical trial results will be featured in an oral presentation at the AD/PD™ 2026 International Conference on Alzheimer’s and Parkinson’s Diseases and related neurological disorders. The presentation will be delivered by Angela Genge, M.D., chief medical officer of AL-S Pharma, today at 8:40AM CET in Hall A2 (Session 5210). 

“These data show something we rarely see in ALS – objective evidence of clinically meaningful disease modification that tracks directly with prolonged survival,” said Dr. Genge. “The Phase 2 study results with AP-101 are consistent with the hypothesis that targeting misfolded SOD1 is a disease-modifying approach in ALS.² At AL-S Pharma, we believe AP-101 could fundamentally transform the treatment of ALS. We look forward to continue advancing the AP-101 clinical program so that we can bring a much-needed new treatment option to people living with ALS rapidly.”

The prespecified analysis of an exploratory composite endpoint revealed that early treatment with AP-101 prolonged survival and delayed ventilatory support in comparison to study participants receiving placebo for six months followed by six months of treatment with AP-101. Positive treatment effects were observed in both the sporadic ALS cohort (p = 0.013) and the SOD1 mutation carrier cohort (p = 0.036). Effects on survival were accompanied by disease stabilization as measured by King’s staging. Functional decline measured by ALSFRS-R was reduced in study participants with elevated misfolded SOD1 at baseline and in SOD1 mutation carriers.

AL-S Pharma is currently preparing for a confirmatory Phase 3 clinical trial of AP-101 in ALS aimed to initiate end of 2026. AP-101 received Orphan Drug designations from the U.S. Food and Drug Administration, the European Medicines Agency, and Swissmedic.

About AP-101-02

The AP-101-02 clinical trial (NCT05039099) was a global, randomized, double-blind, placebo-controlled Phase 2 study evaluating the safety, tolerability, pharmacodynamic markers, and pharmacokinetics (PK) of AP-101 in 73 patients with sporadic ALS and in patients with mutations in the superoxide dismutase 1 (SOD1) gene. Study participants with sporadic (n=52) or mutant SOD1 ALS (n=21) were stratified and randomized 2:1 to intravenous AP-101 or placebo every three weeks. Key assessments included survival and ventilation endpoints, slow vital capacity, neurofilament levels, misfolded SOD1, PK, and anti-drug antibodies. After 24 weeks all participants entered a 24-week open-label extension with continued AP-101 treatment, followed by a 16-week safety follow-up period. 

AP-101-02 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

Amyotrophic lateral sclerosis (ALS) is a relentless and progressive neurodegenerative disease that affects the motor neurons of the brain and spinal cord. Symptoms vary from person to person. Some forms of ALS begin with limb weakness, while others start with bulbar symptoms. All forms ultimately lead to loss of independence and a markedly shortened lifespan. Median survival remains three-to-five years, and diagnosis is often delayed.

Despite this diversity, many patients share common downstream pathologies involving axonal injury, inflammation, and protein misfolding. Superoxide dismutase 1 (SOD1) is a protective enzyme that helps cells manage oxidative stress. In ALS, structural changes can cause SOD1 to lose its proper function and misfold, taking on toxic conformations that disrupt cellular function. Such misfolded SOD1 can injure motor neurons, damage mitochondria, and impair axonal transport. Pathology can spread by the seeding of SOD1 misfolding.

Misfolded SOD1 represents a powerful therapeutic opportunity. Across different presentations of ALS, misfolded SOD1 can amplify axonal injury and accelerate disease progression, making these toxic conformations an important target for intervention.

About AL-S Pharma AG

AL-S Pharma is a clinical-stage biotech company developing AP-101 for the treatment of amyotrophic lateral sclerosis (ALS). Founded and co-owned by Neurimmune and TVM Capital Life Science, AL-S Pharma brings together a seasoned team of biotech and pharmaceutical leaders with expertise spanning drug discovery, translational research, and clinical development. AL-S Pharma is based in Zurich, Switzerland. For more information, visit www.al-spharma.com. 

Investor Contact 

AL-S Pharma
Fabian Buller, Ph.D.
Chief Business Officer
fabian.buller@al-spharma.com 

Media Contact(s)

AL-S Pharma
Kathy Vincent
Corporate Affairs
kathy.vincent@al-spharma.com

MC Services AG
Brittney Sojeva
al-spharma@mc-services.eu

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¹Maier M et al., Sci Transl Med. 2018;10(470).

²Marlow TR et al., Neurobiol Dis. 2025;216:107124.