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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
New post-hoc analyses of the randomized, double-blind period of the AP101-02 clinical trial provide further insights into body-weight trajectories in patients with sporadic ALS and SOD1-ALS.

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

• Title: Learnings from the Phase 2 Study of AP-101 (AP101-02) and the Role of Weight as a Surrogate Marker of Clinical Response
• Presenter: Angela Genge, M.D., FRCP(C), eMBA, chief medical officer, AL-S Pharma
• Session: NEALS Affiliated Trial Session
• Date/Time: Wednesday, October 28, 2026, 11:05 a.m. ET
• Location: Ballroom

Planned NEALS-Affiliated Phase 3 Confirmatory Trial of AP-101 Featured in Poster Presentation
AL-S Pharma is advancing AP-101 toward AP101-03, a planned NEALS-affiliated, multicenter, randomized, double-blind, placebo-controlled Phase 3 clinical trial designed to evaluate the efficacy and safety of AP-101 in adults with ALS. The study is planned to enroll approximately 300 participants, randomized 2:1 to receive AP-101 at a dose of 2,500 mg intravenously every three weeks or placebo for 12 months, in addition to standard of care.

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

• Title: Design of a Planned Phase 3 Confirmatory Trial of AP-101, a Human Antibody Targeting Misfolded SOD1 in ALS
• Presenter: Angela Genge, M.D., FRCP(C), eMBA, chief medical officer, AL-S Pharma
• Poster/Session: Poster 204; Session 2
• Date/Time: Thursday, October 29, 2026, 4:50-6:30PM ET
• Location: Opal Sands, Ballroom

 About AP101-02
The AP101-02 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.

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
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.

Life sciences executive with more than 25 years of CFO and chairman experience to lead AP-101 into confirmatory Phase 3 trial

ZURICH, Switzerland – October 14, 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 the appointment of Harry Welten as chief executive officer (CEO). Mr. Welten succeeds Michael Salzmann, Ph.D. The Company recently reported positive topline results from a Phase 2 study of its lead program AP-101, a human monoclonal antibody targeting misfolded superoxide dismutase 1 (SOD1). AL-S Pharma is currently advancing AP-101 toward a confirmatory Phase 3 clinical trial in ALS which it aims to initiate in 2027.

“As AP-101 enters its confirmatory study, we are delighted to welcome Harry to AL-S Pharma. Harry has spent his career building life sciences companies, both as CFO and as chairman setting strategy at the board level, and has a proven record of securing the financing and partnerships needed to advance clinical programs. The board is confident that his strategic and financial leadership is what AL-S Pharma needs to take AP-101 through Phase 3 and toward regulatory approval, so that it can become available to people living with ALS,” said Hubert Birner, Ph.D., chairman of AL-S Pharma. “On behalf of the board, I would also like to thank Michael for his leadership. During his tenure, the Company advanced AP-101 from early development to a Phase 3-ready program.”

“AL-S Pharma has reached a pivotal stage in the development of AP-101, and I have great respect for what the team has built over the past decade. I look forward to working with the board, the leadership team, and our clinical and scientific partners to build on this strong foundation,” said Mr. Welten. “As AP-101 advances into Phase 3, my priorities will be to execute the Phase 3 program with rigor, on time and on budget, and to secure the financing and strategic partnerships required to bring AP-101 though regulatory approval to patients. Above all, I am committed to contributing to the well-being of people living with ALS and their families, who urgently need new treatment options.”

Mr. Welten is a board-level executive with more than 25 years of chief financial officer (CFO) and chairman experience in the life sciences industry across Europe and the United States. He has guided companies through growth, restructuring, financings, M&A, and commercialization, and has raised more than CHF 500 million through venture capital, IPOs, follow-on offerings, PIPE financings, and convertible instruments. He has led or supported more than eight M&A, public-market, and strategic transactions including the largest European IPO of 2005.

With more than 15 years of board experience, Mr. Welten served on 14 life sciences, medtech, and AI diagnostics boards in Switzerland, across Europe, and in the United States, seven of them as chairman, and has chaired both audit and finance committees and nomination and compensation committees. As chairman, he guided Biognosys, Aidence, and Proteomedix through to their sale or merger with Nasdaq-listed companies. He currently serves as chairman of b-rayZ AG and as a board member of Lumendi AG. Mr. Welton holds an MBA with honors from Columbia Business School, a degree in business administration and economics from the Zurich School of Management, as well as a federal degree in banking and finance from the KV Business School Zurich.

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.

AP-101 is a first-in-class Phase 3-ready antibody targeting misfolded SOD1 in SOD1-ALS and sporadic ALS 

Phase 2 findings further demonstrate clinically meaningful disease modification and prolonged survival in ALS as supported by key neuroaxonal injury biomarkers pNfH and NfL after six months of treatment

Company convened internationally recognized leaders in ALS research to examine how advances in disease biology are reshaping clinical investigation and therapeutic development

ZURICH, Switzerland — June 24, 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 clinical data from the global Phase 2 clinical trial (AP-101-02) evaluating the company’s Phase 3-ready lead program, AP-101, in patients with ALS. The AP-101-02 clinical trial results were featured today in a poster presentation at the European Network to Cure ALS (ENCALS) 2026 Congress.

AP-101 is an investigational human-derived antibody that selectively targets the misfolded, toxic form of superoxide dismutase 1 (SOD1) to disrupt the progressive spread of ALS. The goal of this approach is to help slow the spread of misfolded SOD1 in the nervous system of people living with ALS and support the body’s natural ability to clear it. ¹

“While ALS is a highly diverse disease, these data reinforce the central role of misfolded SOD1 as a therapeutic target in ALS,” said Angela Genge, M.D., FRCP(C), eMBA, chief medical officer of AL-S Pharma. “The consistency observed across multiple biomarker and clinical measures provide strong rationale for continued development of AP-101 as a potential disease-modifying therapy. We are focused on the continued advancement of the AP-101 clinical program and bringing a much-needed new treatment option to people living with ALS.”

The global AP-101 Phase 2 clinical trial met its primary endpoint related to safety and tolerability. Recent findings 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 found to be similar to those in the placebo group, with no reported antibody responses induced by AP-101.

The prespecified analysis of an exploratory composite endpoint indicated that early treatment with AP-101 prolonged survival and delayed ventilatory support when compared 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 advancing AP-101 into a confirmatory Phase 3 clinical trial of AP-101 in ALS aimed to initiate the first quarter of 2027. AP-101 received Orphan Drug designations from the U.S. Food and Drug Administration, the European Medicines Agency, and Swissmedic.

AL-S Pharma ENCALS Symposium: “Beyond “Sporadic” ALS: Genetics, Biomarkers, and the Misfolded SOD1 Pathway.”

Moderated by Dr. Genge, AL-S Pharma’s symposium brought together internationally recognized leaders in ALS research to examine how advances in disease biology are reshaping clinical investigation and therapeutic development. Designed for neurologists, physician-scientists, clinical trialists, and industry professionals in drug development, the session connected emerging insights in genetic architecture, biomarker interpretation, and protein misfolding biology to the practical realities of ALS diagnosis, stratification, and trial design.

• Ammar Al-Chalabi, Ph.D., FRCP, FMedSci, provided a contemporary reappraisal of so-called sporadic ALS, challenging the traditional familial/sporadic divide and highlighting a more nuanced view of genetic contribution and disease architecture.
• Dame Pamela Shaw, DBE, MBBS, M.D., FRCP, FAAN, FANA, FAAAS, discussed neurofilament biomarkers in ALS clinical trials, focusing on what these markers have taught us over time, where they are most useful today, and how they should be interpreted in the context of therapeutic studies.
• Philip Van Damme, M.D., Ph.D., reviewed the lessons learned from SOD1-ALS and discussed how misfolded SOD1 could play a role in the absence of SOD1 mutations.

A moderated panel discussion, “Translating ALS Biology into Better Trials,” featured Prof. Leonard van den Berg, Ph.D., Prof. Andrea Malaspina, M.D., Ph.D., and Prof. Julian Grosskreutz, M.D. They discussed how emerging insights in ALS genetics, biomarker interpretation, and misfolded protein biology can be translated into better trial design, patient stratification, and therapeutic development.

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.

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.

 Zurich, Switzerland, January 7, 2026 – AL-S Pharma AG, a Swiss-based, single-asset clinical-stage biopharmaceutical company founded and co-owned by Neurimmune and TVM Capital Life Science, is developing AP-101 for Amyotrophic Lateral Sclerosis (ALS). The company today announced that Dr. Michael Salzmann, Chief Executive Officer, and Prof. Angela Genge, MD, Chief Medical Officer, will present at the 44th Annual J.P. Morgan 2026 Healthcare Conference on Thursday, January 15, 2026, at 8:30 a.m. PT.

AL-S Pharma will present its recently announced positive results from the global Phase 2 study of AP-101 in 73 participants for the treatment of ALS, along with key data updates, including subgroup analyses, supporting plans for a confirmatory Phase 3 study.

About AP-101
AP-101 is an investigational human-derived antibody directed against misfolded superoxide dismutase 1 (SOD1) designed to inhibit the spread of SOD1 pathology in the CNS of ALS patients. AP-101 received orphan drug designations from FDA, EMA and Swissmedic. The Phase 2 study evaluated safety, tolerability, pharmacodynamic (PD) markers, and pharmacokinetics (PK) of AP-101 in patients with sporadic ALS and in patients with mutations in the SOD1 gene.

About AL-S Pharma AG
AL-S Pharma is a Swiss-based single-asset company founded and co-owned by Neurimmune and TVM Capital Life Science to develop AP-101. The company is fully financed by TVM Life Science Innovation I and Neurimmune. AL-S Pharma brings together a seasoned team of biotech and pharmaceutical leaders with expertise spanning drug discovery, translational research, and clinical development. Based on the successful Phase 2 data, AL-S Pharma is currently advancing AP-101 into late-stage development.

• Primary safety and tolerability endpoint met
• AP-101 prolonged survival and delayed need for ventilatory support after 12 months of treatment
• Functional stabilization and reduced neurofilament biomarkers observed in study participants with elevated misfolded SOD1

 Zurich, Switzerland, December 5, 2025 – AL-S Pharma, a clinical-stage biotech company dedicated to the development of AP-101 for the treatment of amyotrophic lateral sclerosis (ALS) today presented its Phase 2 clinical study results on AP-101 at the 36^th International Symposium on ALS/MND in San Diego.

AP-101 is an investigational human antibody therapeutic selectively targeting misfolded SOD1 to disrupt the progressive spread of pathology in ALS.¹ The global phase 2 study with 73 participants met its primary endpoint related to safety and tolerability. Adverse events were comparable to placebo, and no AP-101-induced antibody responses were reported.

The 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 6 months followed by 6 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. In addition, AP-101 treatment resulted in favorable neurofilament biomarker changes aligned with clinical benefit.

“We are very excited by the positive Phase 2 results for AP-101,” said Angela Genge, MD, Chief Medical Officer of AL-S Pharma. “We are observing very consistent results across survival, function, and biomarkers in two independent cohorts within our study. Despite the statistical limitations of a Phase 2 study, the exploratory outcome data indicates that treatment with AP-101 results in a clinically meaningful treatment effect in both sporadic ALS as well as SOD1-ALS patients.”

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 AL-S Pharma is currently preparing for a confirmatory Phase 3 clinical study.

“We are deeply committed to advancing an urgently needed new treatment option for ALS. Together with our investigators, advisors, collaborators, and their clinical teams, we are pioneering a new disease-modifying approach by targeting misfolded SOD1 with a therapeutic antibody,” said Michael Salzmann, PhD, Chief Executive Officer of AL-S Pharma. “We are grateful to all the trial participants and their families whose dedication made this study possible. We plan to engage with regulators on next steps in the coming months while we prepare for the confirmatory Phase 3 study.”

The AP-101-02 study was a global, randomized, double-blind, placebo-controlled study evaluating safety, tolerability, pharmacodynamic markers, and pharmacokinetics of AP-101 in study participants with sporadic ALS (N=52) and mutant SOD1-ALS (N=21) over 6 months followed by a 6-months open-label extension and a safety follow-up period. Further information on the clinical study of AP-101 for ALS can be accessed on ClinicalTrials.gov (study number NCT05039099).

¹Maier M et al., Sci Transl Med. 2018;10(470).
²Marlow TR et al., Neurobiol Dis. 2025;216:107124.

 About AP-101
AP-101 is an investigational human-derived antibody directed against misfolded superoxide dismutase 1 (SOD1) designed to inhibit the spread of SOD1 pathology in the CNS of ALS patients. AP-101 received orphan drug designations from FDA, EMA and Swissmedic. The Phase 2 study evaluated safety, tolerability, pharmacodynamic (PD) markers, and pharmacokinetics (PK) of AP-101 in patients with sporadic ALS and in patients with mutations in the SOD1 gene.

About AL-S Pharma AG
AL-S Pharma is a Swiss-based single-asset company founded and co-owned by Neurimmune and TVM Capital Life Science to develop AP-101. The company is fully financed by TVM Life Science Innovation I and Neurimmune. AL-S Pharma brings together a seasoned team of biotech and pharmaceutical leaders with expertise spanning drug discovery, translational research, and clinical development. Based on the successful Phase 2 data, AL-S Pharma is currently advancing AP-101 into late-stage development.

Zurich, Switzerland, September 25, 2025 – AL-S Pharma announced today that it has appointed Angela Genge, MD, Professor of Neurology at McGill University in Montreal, as Chief Medical Officer (CMO). With a distinguished record in amyotrophic lateral sclerosis (ALS) research and the Head of ALS Global Centre of Excellence for Research and Patient Care at McGill, Dr. Genge will join the company’s executive team to further develop AP-101 for the treatment of ALS. Since 2016, Dr. Genge has served as the global principal investigator for the Phase 1 and Phase 2 clinical development of AP-101, a first-in-class investigational antibody targeting misfolded SOD1. The company recently reported positive topline Phase 2 study results of AP-101 for the treatment of ALS (press release Sept 4, 2025).

“We are very happy to welcome Prof. Dr. Genge in an expanded role at this exciting time for AL-S Pharma,” said Michael Salzmann, PhD, Chief Executive Officer of AL-S Pharma. “As an internationally recognized leader in ALS, Prof. Dr. Genge’s expertise will be pivotal to guide our AP-101 late-stage development. Together, we look forward to advancing this potential first-in-class, disease-modifying therapy for ALS.”

Angela Genge, MD, FRCP, is a recognized leader in ALS clinical research and drug development. She has served as global principal investigator on pivotal ALS trials, advised regulatory authorities, and participated in more than 80 global clinical advisory boards and trial steering committees. Her contributions have been recognized with prestigious awards such as the Forbes Norris Award, the PACTALS Excellent Research Award, and the Wings Over Wall Street Diamond Award.

“AL-S Pharma’s commitment to develop AP-101 as a first-in-class therapy targeting misfolded SOD1, a key driver of disease-progression, has always impressed me,” said Dr. Genge. “I am very much looking forward to working together with the leadership team to further advance the clinical development in an effort to bring this innovative therapy to patients.”

In 2016, Neurimmune and TVM Capital Life Science co-created AL-S Pharma AG to develop AP-101. AL-S Pharma has executed its innovative clinical plan for AP-101 in collaboration with an international network of ALS experts. The company recently completed a global, randomized, double-blind, placebo-controlled Phase 2 study evaluating safety, tolerability, pharmacodynamic markers, and pharmacokinetics of AP-101 in both patients with sporadic ALS (N=52) and patients with mutant SOD1-ALS (N=21) over 24 weeks followed by a 24-week open-label extension and a safety follow-up period. Further information on the clinical study of AP-101 for ALS can be accessed on ClinicalTrials.gov (study number NCT05039099).

About AP-101
AP-101 is an investigational human antibody directed against misfolded superoxide dismutase 1 (SOD1) and was discovered by Neurimmune’s Reverse Translational Medicine^TM (RTM^TM) technology. The antibody has been designed to inhibit the spread of SOD1 pathology in the cerebrospinal fluid and spinal cord of ALS patients. AP-101 received orphan drug designations from FDA, EMA and Swissmedic. The Phase 2 study completed by AL-S Pharma evaluated the safety, tolerability, pharmacodynamic (PD) markers, and pharmacokinetics (PK) of AP-101 in patients with sporadic ALS and patients with mutations in the SOD1 gene.

About AL-S Pharma AG
AL-S Pharma is a Swiss-based single-asset company founded and co-owned by Neurimmune and TVM Capital Life Science to develop AP-101. The company is fully financed by TVM Life Science Innovation I and Neurimmune. AL-S Pharma is led by an experienced management team and the Board of Directors comprises Guy Rouleau, director of McGill University’s Montreal Neurological Institute (MNI) and Hospital, Chair Department of Neurology and Neurosurgery, McGill University, Hubert Birner, Managing Partner, TVM Capital Life Science, Munich, Marc Riviere, General Partner, TVM Capital Life Science, Montreal, Christoph Hock, CMO of Neurimmune, and Jan Grimm, CSO of Neurimmune.

Follow AL-S Pharma on LinkedIn.

About Neurimmune
Neurimmune is a biopharmaceutical company translating human immune memory into antibody therapeutics. Neurimmune develops drug candidates for CNS and related protein aggregation diseases including Alzheimer’s disease, amyotrophic lateral sclerosis and ATTR cardiomyopathy. With its Reverse Translational Medicine^TM technology, Neurimmune discovered the anti-ATTR antibody cliramitug (formerly NI006, ALXN2220) for ATTR cardiomyopathy, the anti-SOD1 antibody AP-101 for ALS and the anti-NogoA antibody NG004 for spinal cord injury, programs being currently evaluated in clinical trials.

For further information, please visit www.neurimmune.com. Follow Neurimmune on LinkedIn.

About TVM Capital Life Science
TVM is a leading international venture capital firm focused on investing in life science innovations. The company has a highly experienced transatlantic investment team and approximately $900 million under management. TVM’s portfolio focuses on therapeutics and medical technologies from North America and the EU that represent differentiated first-in-class or best-in-class assets with the potential to transform standard of care. With its early-stage investments, TVM follows several key principles, which include increasing capital efficiency, streamlining development timelines and soliciting buyer input at the time of investment. This strategy has been validated through successful exits such as AurKa Pharma, Inc. and Acanthas Pharma, Inc.

For further information, please visit www.tvm-capital.com. Follow TVM on LinkedIn.

• Phase 2 of AP-101 in sporadic ALS and mutant SOD1-ALS met its primary safety and tolerability endpoint
• Clinically meaningful changes in outcome measures and stabilization of biomarkers were observed
• Data will be presented at upcoming scientific conferences and AL-S Pharma plans to engage with regulatory authorities later this year
• AP-101 is a first-in-class investigational human antibody targeting misfolded SOD1, a pathological hallmark of ALS

Zurich, Switzerland, September 4, 2025 – AL-S Pharma today announced positive topline results from the Phase 2 clinical study evaluating AP-101, a potential first-in-class, disease-modifying treatment for amyotrophic lateral sclerosis (ALS). AP-101 is a monoclonal antibody targeting misfolded SOD1, a key pathology in both sporadic ALS and mutant SOD1-ALS.¹ AP-101, in addition to standard of care, met its primary endpoint related to safety and tolerability. Clinically meaningful changes in exploratory clinical outcome measures related to survival and non-invasive ventilation, as well as stabilization of clinical disease-staging and neurofilament biomarkers, were observed following 12 months of treatment. Results will be shared with regulatory authorities, presented at upcoming ALS conferences, and submitted for publication in a peer-reviewed scientific journal.

“We are excited by the topline Phase 2 results of AP-101 for ALS, a devastating degenerative disease for which new treatments are urgently needed,” said Michael Salzmann, PhD, Chief Executive Officer, AL-S Pharma. “We are grateful to the participants, their families and the international network of ALS experts who made this trial possible, and we look forward to sharing results from the study at the International Symposium on ALS/MND.”

“This is the first Phase 2 study to assess a SOD1-targeted therapeutic in both sporadic ALS and in ALS patients with mutations in the SOD1 gene. The study allowed us to rigorously assess patient safety, pharmacokinetics, and early signals of biological activity,” said Peter Andersen, Professor and Senior Consultant Neurologist, Umea University, Sweden, and Principal Investigator for the AP-101 Phase 2 study. “The completion of the trial marks an important step in evaluating the therapeutic potential of AP-101 for broad use in patients with ALS. The results support the hypothesis that misfolded SOD1 protein plays a more general role in ALS”.

ALS is a fatal neurodegenerative disease characterized by loss of both upper and lower motor neurons. Symptoms typically begin focally with spread in a contiguous anatomical pattern, leading to muscle weakness, respiratory issues, swallowing and speaking difficulties, and ultimately to death. Very limited therapeutic options are available.

SOD1 pathology has been proposed to be a disease driver in both genetically determined SOD1-ALS as well as in sporadic ALS.^2,3 Pathological, genetic and preclinical evidence suggest that SOD1 misfolding and the formation of neurotoxic SOD1 species drive cell death in the motor system. AP-101 is a first-in-class antibody therapeutic targeting misfolded SOD1.

AL-S Pharma’s Phase 2 study is a multicenter, randomized, double-blind, placebo-controlled study evaluating safety, tolerability, pharmacodynamic markers, and pharmacokinetics of AP-101 in both patients with sporadic ALS (N=52) and patients with mutant SOD1-ALS (N=21) over 24 weeks followed by a 24-week open-label extension and a safety follow-up period. Further information on the clinical study of AP-101 for ALS can be accessed on ClinicalTrials.gov (study number NCT05039099).

In 2016, Neurimmune and TVM Capital Life Science co-created AL-S Pharma to develop AP-101. AL-S Pharma has executed its innovative clinical plan for AP-101 in collaboration with an international network of ALS experts.

Presentation Details

36^th International Symposium on ALS/MND
Title: Top-line results of the Phase 2 proof-of-concept study of AP-101, a first-in-class human antibody targeting misfolded SOD1 in amyotrophic lateral sclerosis
Presenter(s): Prof. Dr. Angela Genge (McGill University – Montreal, Canada)
Session Type: Oral Presentation
Date: December 5, 2025

2025 Annual NEALS Meeting
Title: Baseline characteristics and analysis of misfolded SOD1 target levels in a Phase 2 proof-of-concept study to evaluate the human antibody AP-101 in sporadic ALS and genetically determined SOD1-ALS
Presenter: Prof. Dr. Angela Genge (McGill University – Montreal, Canada)
Session Type: Poster at Science Symposium
Date: October 2025 (additional details to be announced)

¹Maier M et al., Sci Transl Med. 2018;10(470).
²Forsberg K et al., PLoS One. 2010;5(7):e11552.
³Trist BG et al., Brain. 2022;145(9):3108-3130.

About AP-101
AP-101 is an investigational human antibody directed against misfolded superoxide dismutase 1 (SOD1) and was discovered by Neurimmune’s Reverse Translational Medicine^TM (RTM^TM) technology. The antibody has been designed to inhibit the spread of SOD1 pathology in the cerebrospinal fluid and spinal cord of ALS patients. AP-101 received orphan drug designations from FDA, EMA and Swissmedic. The Phase 2 study completed by AL-S Pharma evaluated the safety, tolerability, pharmacodynamic (PD) markers, and pharmacokinetics (PK) of AP-101 in patients with sporadic ALS and patients with mutations in the SOD1 gene.

About AL-S Pharma AG
AL-S Pharma is a Swiss-based single-asset company founded and co-owned by Neurimmune and TVM Capital Life Science to develop AP-101. The company is fully financed by TVM Life Science Innovation I and Neurimmune. AL-S Pharma is led by an experienced management team and the Board of Directors comprises Guy Rouleau, director of McGill University’s Montreal Neurological Institute (MNI) and Hospital, Chair Department of Neurology and Neurosurgery, McGill University, Hubert Birner, Managing Partner, TVM Capital Life Science, Munich, Marc Riviere, General Partner, TVM Capital Life Science, Montreal, Christoph Hock, CMO of Neurimmune, and Jan Grimm, CSO of Neurimmune.

Follow AL-S Pharma on LinkedIn.

About Neurimmune
Neurimmune is a biopharmaceutical company translating human immune memory into antibody therapeutics. Neurimmune develops drug candidates for CNS and related protein aggregation diseases including Alzheimer’s disease, amyotrophic lateral sclerosis and ATTR cardiomyopathy. With its Reverse Translational Medicine^TM technology, Neurimmune discovered the anti-ATTR antibody cliramitug (formerly NI006, ALXN2220) for ATTR cardiomyopathy, the anti-SOD1 antibody AP-101 for ALS and the anti-NogoA antibody NG004 for spinal cord injury, programs being currently evaluated in clinical trials.

For further information, please visit www.neurimmune.com. Follow Neurimmune on LinkedIn.

About TVM Capital Life Science
TVM is a leading international venture capital firm focused on investing in life science innovations. The company has a highly experienced transatlantic investment team and approximately $900 million under management. TVM’s portfolio focuses on therapeutics and medical technologies from North America and the EU that represent differentiated first-in-class or best-in-class assets with the potential to transform standard of care. With its early-stage investments, TVM follows several key principles, which include increasing capital efficiency, streamlining development timelines and soliciting buyer input at the time of investment. This strategy has been validated through successful exits such as AurKa Pharma, Inc. and Acanthas Pharma, Inc.

For further information, please visit www.tvm-capital.com. Follow TVM on LinkedIn.