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AL-S Pharma is planning a registrational study for its amyotrophic lateral sclerosis (ALS) monoclonal antibody (mAb), AP-101, after the drug met its efficacy and safety endpoints in a mid-stage trial.

During the Phase II study (NCT05039099), ALS patients treated early with AP-101 displayed prolonged survival and delayed ventilatory support after 12 months of treatment compared to those who received placebo for six months then AP-101 for six months. This benefit was observed across both the sporadic ALS and superoxide dismutase 1 (SOD1) mutation carrier cohorts.

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The SOD1-targeting mAb also triggered disease stabilisation, as measured by King’s staging, which qualitatively categorises disease progression based on the condition of the bulbar, upper and lower limb and diaphragm regions.

Patients receiving AP-101 also experienced reduced functional decline in patients with elevated, misfolded SOD1 levels at baseline, as well as those with SOD1 mutations.

Alongside the drug’s impact on disease progression and stabilisation, patients receiving AP-101 experienced changes to neurofilament biomarkers, which AL-S claims “aligns with AP-101’s clinical benefit”.

While AL-S did not provide specifics on the drug’s safety profile, the Swiss biotech did note that adverse events (AEs) were similar between the placebo and treatment groups. There were no antibody responses to AP-101 observed during the 12-month study.

AL-S presented the data at the 36th International Symposium on ALS/MND, which was held from 5 to 7 December in San Diego; however, the company had previously confirmed the drug met the trial endpoints.

According to the CEO of AL-S Pharma, Michael Salzmann, the biotech now plans to discuss next steps for AP-101 with regulators in the coming months, as the company prepares to initiate a confirmatory Phase III study.

Homing in on SOD1

While ALS has typically been a challenging indication to treat, there are now four therapies that have gained approval from the US Food and Drug Administration (FDA).

This includes Biogen and Ionis Pharmaceuticals’ SOD1-targeting antisense oligonucleotide, Qalsody (tofersen), which gained accelerated approval from the FDA in 2023, despite the VALOR trial failing to meet its primary endpoint of improved ALSFRS-R scores over 28 weeks. The FDA granted the drug approval based on changes in plasma levels of neurofilament light (NfL) protein.

While Qalsody’s results have been mixed thus far, SOD1 has been a target of interest across the ALS research space, with four drugs, including AP-101, in active development, according to GlobalData’s Pharmaceutical Intelligence Center.

GlobalData is the parent company of Clinical Trials Arena.

The ALS Association currently estimates that SOD1 gene mutations are observed in 10-20% of familial ALS cases, while 1-2% of sporadic ALS cases are linked to this mutation.

If AL-S’s SOD1-directed AP-101 were to get the FDA go-ahead, it would offer an alternative dosing option to Qalsody, as AP-101 can be administered intravenously, while Qalsody requires patients to undergo an intrathecal injection.

According to GlobalData’s patient-based forecast, Qalsody will make $68m for Biogen and Ionis in 2029.

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