AL-S Pharma, a Swiss clinical-stage biotech company, has unveiled encouraging Phase 2 results for its investigational therapy for amyotrophic lateral sclerosis (ALS), a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord.

 

AP-101, a human antibody therapeutic designed to selectively target misfolded SOD1 and disrupt the progression of ALS pathology, met its primary safety and tolerability endpoint in a global study of 73 participants. Adverse events were similar to placebo, and no AP-101-induced antibody responses were detected.

 

Exploratory analyses revealed that early treatment with AP-101 extended survival and delayed the need for ventilatory support compared to participants who received placebo for six months before switching to AP-101. 

 

Positive effects were observed in both sporadic ALS patients (p = 0.013) and SOD1 mutation carriers (p = 0.036). Disease stabilization, measured by King's staging, accompanied improvements in survival, and functional decline measured by ALSFRS-R was reduced in participants with elevated misfolded SOD1 at baseline and in SOD1 mutation carriers. Favorable neurofilament biomarker changes also aligned with clinical benefits.

 

"We are very excited by the positive Phase 2 results for AP-101," said Angela Genge, 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 results support the company’s hypothesis that targeting misfolded SOD1 may be a disease-modifying approach for ALS. AL-S Pharma is now preparing for a confirmatory Phase 3 trial.

 

"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, 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 trial evaluating safety, tolerability, pharmacodynamic markers, and pharmacokinetics in 52 sporadic ALS and 21 SOD1-ALS participants over six months, followed by a six-month open-label extension and safety follow-up.