Clinical Trials

By targeting the fundamental underlying mechanisms of neurodegeneration in MS, i.e. neutralizing microglial-mediated damage, as well as restoring oligodendrocyte precursor cells (OPC) remyelination capacity, temelimab may address the critical unmet medical need of blocking disability progression independent of relapses in MS.

Current studies

The ProTEct-MS clinical study

GeNeuro’s ProTEct-MS study, which has tested monthly doses of 18, 36 and 54 mg/kg of temelimab on 41 patients, was conducted at the Academic Specialist Center (ASC) of the Karolinska Institutet, Stockholm, Sweden, in a double-blind, placebo-controlled fashion and was designed to evaluate the safety, tolerability and efficacy of the product based on the latest biomarkers associated with disease progression. The topline results of the ProTEct-MS study were announced on March 21, 2022, and showed that the primary endpoint of the ProTEct-MS study was met, with results confirming the excellent safety profile and tolerability of higher doses of temelimab administered concomitantly with a high-efficacy anti-inflammatory drug; in addition, efficacy data, obtained in this patient group already effectively treated against inflammation, showed that temelimab has a favorable impact on key MRI measures of neurodegeneration; the observed effect sizes in this new patient population were consistent with the ones shown in the previous CHANGE-MS and ANGEL-MS studies.

The study was presented at the MSVirtual2020

Visit for more information on clinical trials of temelimab (previously known as GNbAC1).

Completed studies

Multiple Sclerosis Phase II: CHANGE-MS and its ANGEL-MS extension

In March 2019, GeNeuro published the top-line 96-week results of its extension Phase 2b clinical trial, ANGEL-MS, which demonstrated that the 18mg/kg dose of temelimab had remarkably consistent benefits over all other groups on key MRI measures linked to MS disease progression, thereby confirming and extending the results of CHANGE-MS at Week 48.

The data showed that, after two years of treatment, the patients originally randomized to 18 mg/kg temelimab showed evidence of continued improvements in MRI-based neurodegenerative outcomes, such as brain volumes, magnetization transfer ratio (MTR) and black holes during ANGEL-MS up to 96 weeks compared to all other groups. Importantly, these effects were not driven by an anti-inflammatory effect. These data were presented at ECTRIMS 2019 in Stockholm, Sweden. 

As the study demonstrated, temelimab continued to be safe and well tolerated over this extended treatment period, which allows new therapeutic solutions in combination with anti-inflammatory drugs or as a monotherapy to be considered, with the objective to bring new benefits against disease progression across all forms of MS.

ANGEL-MS (Assessing the HERV-W Env ANtagonist GNbAC1 (temelimab) for Evaluation in an open label Long-term Safety Study in patients with Multiple Sclerosis) was a 2-year safety and efficacy extension study to CHANGE-MS (a 270 RRMS patients Phase II clinical trial in 50 clinical centers in 12 European countries), which offered continued treatment to those patients who had completed the 12-month primary study. Ninety-four percent of eligible patients (n=219) entered ANGEL-MS and continued treatment at the same dose of temelimab they were receiving at the end of CHANGE-MS. Across the two studies, a total of 154 patients received temelimab treatment for 96 weeks or more.

Type 1 Diabetes (T1D)

In May 2019, the Company announced the results from the six-month extension of its Phase 2a study of temelimab in T1D, which confirmed all previously observed positive observations during the trial, which has met its primary objective (safety of temelimab administration in combination with T1D treatments).

GeNeuro believes these data open the door to further development in early-onset T1D pediatric patient population but, based on its current strategy, has decided to temporarily pause development of temelimab in T1D in order to prioritize the development for MS.

The Phase IIa randomized, placebo-controlled, safety and signal finding study (RAINBOW) evaluated temelimab in 64 adult patients diagnosed with T1D for up to 4 years, in 12 centers in Australia. The primary endpoint of the study was safety of temelimab administration in combination with T1D treatments. Secondary endpoints measured T1D biomarkers such as HbA1c, C-peptide levels, and other biomarkers associated with T1D, such as insulin consumption, glycaemia and production of diabetic auto-antibodies.