Birch triterpenes

Your support is helping move important science forward. Give today to sustain our critical research. Dear ALS Community, Earlier this month, at the International Symposium on ALS/MND, I watched a presentation from AL-S Pharma announcing that AP-101—a therapeutic antibody targeting misfolded SOD1—had succeeded in Phase 2b and is now advancing toward Phase 3 clinical trials. Our team at ALS TDI helped make that molecule what it is. Years ago, we partnered with a European biotech called Neurimmune and executed key parts of the preclinical characterization work that supported research-to-development transition for AP-101 to initiate human trials. It was painstaking, unglamorous science - the kind that rarely makes it into newsletters. SOD1 mutations cause some of the most aggressive forms of familial ALS. They ravaged my late best friend, Sean Scott, and generations of his family. When our scientists were running those experiments, we had Sean’s family and families like his at top of mind. We held hope that the approach could benefit people with sporadic ALS, as well. AL-S Pharma maintains that hope and intends to advance AP101 into human trials in both SOD1 familial ALS and in sporadic ALS. We don’t know if AP-101 will succeed in its Phase 3 development just like we couldn't know when we began collaborating with Neurimmune whether this program would ever advance this far. We only knew it was worth trying. Here's what I want you to understand: if you gave to ALS TDI between 2013 and 2018, you helped fund this work. Not because we asked you to support a specific antibody program - we rarely mentioned it - but because your generosity gave us the flexibility to pursue promising science wherever it led. That is the power of the support you provide. Progress in research is nonlinear. We cannot predict which programs will break through or when. But we can build a pipeline worth believing in, and right now, ours includes another lead program called TDI-1831 that may help people with rare ALS-causing mutations, and a versatile platform for delivering precision mRNA therapeutics directly to the brain and spinal cord. I don't know which of these will advance to Phase 3 clinical development or beyond. But I know the work is worth trying. And I know it's only possible because of you. If you're able to make a gift this year, it will go directly toward the science that could one day reach someone's best friend, someone's parent, someone's child. Make a year-end gift With deep gratitude, Fernando Vieira, M.D. CEO & Chief Scientific Officer ALS Therapy Development Institute

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, Dec. 5, 2025 /PRNewswire/ -- 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 36th 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. Follow AL-S Pharma on LinkedIn. Contact for Media John Capodanno (US) [email protected] +1 917 750-8649 Anne Hennecke (EU) [email protected] +49 211 529 252 14 Contact for AL-S Pharma Dr. Michael Salzmann [email protected] SOURCE AL-S Pharma AG 21% more press release views with Request a Demo