Positive top-line Phase IIa results for tegoprubart

US clinical-stage biotech Eledon Pharmaceuticals (Nasdaq: ELDN) saw its share gain 8% to $3.92 pre-market, after it announced positive top-line results from a Phase IIa clinical trial of tegoprubart (formerly AT-1501) in patients with amyotrophic lateral sclerosis (ALS).

Tegoprubart is an investigational humanized monoclonal antibody that inhibits CD40 Ligand (CD40L), a membrane protein linked to increased peripheral immune responses and neuroinflammation in ALS. The 12-week trial included 54 patients with ALS at 13 treatment sites in the USA and Canada. The primary objectives of the study were to assess the safety and tolerability of multiple doses of tegoprubart in four sequential, ascending dose cohorts (1, 2, 4, and 8mg/kg). Secondary outcome measures included pharmacokinetic assessment of multiple intravenous doses of tegoprubart on target engagement and on pro-inflammatory biomarkers associated with ALS. Each subject served as their own control, with changes being compared to baseline.

Tegoprubart successfully met the primary endpoints of safety and tolerability. Adverse events were equally distributed across dose levels. Tegoprubart was well-tolerated, and no drug-related serious adverse events were observed. Anti-drug antibodies (ADAs) were present in less than 5% of samples. All ADAs were of low titer and did not impact tegoprubart drug levels.