On June 30, at the 12th Congress of the European Academy of Neurology (EAN) 2026, Regeneron announced the results from an additional analysis of the Phase III NIMBLE study of cemdisiran for generalized myasthenia gravis (gMG). The positive results highlight the potential for use of cemdisiran in this patient segment, according to GlobalData, a leading intelligence and productivity platform.
Cemdisiran is an investigational RNA interference (RNAi) therapeutic designed to lower the production of the complement component 5 (C5) protein in the liver. The trial sought to evaluate subcutaneously administered cemdisiran and pozelimab as monotherapies or in combination in participants with gMG (Myasthenia Gravis Foundation of America [MGFA] Class II–V) who are acetylcholine receptor antibody-positive (AChR+) and/or low-density lipoprotein receptor-related protein 4-positive (LRP4+).
The trial met its primary endpoint, which was a change from baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) score at week 24, as well as key secondary endpoints, including a change in baseline in the Quantitative Myasthenia Gravis (QMG) score at week 24 and the cumulative rate of treatment-emergent adverse events (TEAEs). The data marks an exciting development for LRP4+ MG patients, as there are few effective treatments available in this space.
Cemdisiran is a disease-modifying treatment (DMT) that is thought to lower the levels of the C5 protein by targeting C5 messenger RNA (mRNA) in the liver, which helps prevent complement-mediated tissue damage. The NIMBLE trial sought to recruit patients who were LRP4+. Key opinion leaders (KOLs) previously interviewed by GlobalData noted that there is a significant unmet need for effective treatments targeting the LRP4+ patient population.
Jos Opdenakker, Neurology Analyst at GlobalData, comments: “Currently, there is no effective DMT on the market that targets the LRP4+ MG population, Cemdisiran’s promising results open the door for it to be used as a monotherapy, which means that Regeneron can position cemdisiran to meet this unmet need.”
Regeneron is also co-developing and investigating cemdisiran as a combination therapy with Alnylam Pharmaceuticals’ pozelimab, a C5 antibody, but GlobalData expects this combination therapy to be very expensive. A common issue that DMTs for MG encounter is that they are very expensive. This issue is not likely to be resolved if they are used in combination.
Opdenakker adds: “If cemdisiran were to be approved as a monotherapy, this could alleviate some of the financial constraints surrounding the use of this DMT. However, the approval of cemdisiran as a monotherapy would have to be carefully considered by Regeneron, as this could potentially mark the end of its strategic alliance with Alnylam to develop the potentially lucrative combination therapy of pozelimab and cemdisiran.”
GlobalData forecasts that the pozelimab and cemdisiran combination therapy could drive sales of approximately $1.9 billion by 2034 in the seven major pharmaceutical markets (7MM*), driven by its targeting of multiple MG subtypes, including AChR+ patients and the overlooked LRP4+ MG patient segment.
The Phase III clinical trial results for cemdisiran mark a significant milestone in the treatment of gMG. With clinically meaningful improvements in the MG-ADL score and QMG, as well as meaningful safety data, cemdisiran offers hope for both AChR+ and LRP4+ patients.
Opdenakker continues: “However, Regeneron will face challenges as it enters a highly competitive market filled with established DMTs. It must ensure that it opts for a lucrative marketing strategy to facilitate a successful launch, given that launching cemdisiran in combination with pozelimab may not garner the best results, both therapeutically and commercially, for patients and clinicians, based on this most recent trial data.”
Opdenakker concludes: “Cemdisiran monotherapy has the potential to become a valuable addition to the gMG treatment paradigm, offering new possibilities for disease management, if Regeneron can successfully navigate the complexities of market competition and opt for a congruent marketing strategy.”
*7MM-US, France, Germany, Italy, Spain, UK, and Japan
