Enrollment Complete in Phase 2 Trial Testing Temelimab

GeNeuro has completed patient enrollment in its ProTEct-MS Phase 2 clinical trial evaluating temelimab as a treatment for multiple sclerosis (MS), the company announced.

The study (NCT04480307), taking place at the Karolinska Institutet’s Academic Specialist Center (ASC) in Stockholm, Sweden, has enrolled 42 people with relapsing MS whose disability progressed in the absence of relapses after treatment with rituximab. Top-line results are expected by March 2022.

“MS disease progression continues to be a major unmet need and we are eager to explore the therapeutic potential of temelimab in patients progressing without relapses. This constitutes a key unmet medical need in MS and will allow us to push the boundaries of current therapeutic possibilities,” Fredrik Piehl, the study’s lead investigator, said in a press release.

Rituximab works by depleting B-cells — a type of immune cell known to drive inflammation in MS — via targeting a protein on their surface called CD20. The therapy often is used off-label in MS and other autoimmune disorders.

Temelimab is a monoclonal antibody designed to neutralize a viral protein —  called pHERV-W — known to play a role in the inflammation and myelin destruction that characterize MS. (Myelin is the fatty protective coat surrounding nerve cells that is lost in MS.)

“Modern MS therapies strongly suppress relapse activity, but have little effect on the long-term course of disability. ProTEct-MS pursues a novel therapeutic concept where an anti-neurodegenerative compound, temelimab, is administered to patients already on a high-efficacy anti-inflammatory therapy,” said David Leppert, chief medical officer of GeNeuro.

Participants in the ProTEct-MS study were assigned randomly to receive one of three monthly doses of intravenous (into-the-vein) temelimab (18, 36 or 54 mg/kg), or a placebo, for 48 weeks.

The trial’s main goal is to assess the safety and tolerability of temelimab. Additional goals include assessing the effects of the treatment on brain atrophy (shrinkage) and lesion volume, both assessed by neuroimaging.

The study follows the promising results seen with temelimab in the Phase 2b ANGEL-MS trial (NCT03239860), in which treatment for 96 weeks with temelimab reduced brain atrophy (shrinkage), helped maintain the integrity of myelin, and reduced disease progression.

“We are very grateful to the patients who have agreed to participate in this important study and we also thank the team at Karolinska Institutet’s Stockholm Academic Specialist Center for their great efforts that have allowed to dramatically cut the delay due to the COVID-19 pandemic from the initial 3 month estimate down to 6 weeks,” Leppert said.

“Temelimab has already been shown to have a neuroprotective effect in MS and it has the potential to address the key unmet medical need of disability progression, so we look forward to the results of this important study in [the first quarter of] 2022,” Leppert concluded.