Efgartigimod

Johnson & Johnson unveiled more details from a Phase III study of nipocalimab in generalised myasthenia gravis (gMG), adding another feather to its cap for the FcRn inhibitor programme. In addition to bolstering nipocalimab's potential in gMG, the results may also set the stage for a showdown with argenx's rival FcRn blocker Vyvgart (efgartigimod alfa), which was approved in 2021 for gMG patients who are positive for anti-AChR antibodies.Data from the VIVACITY-MG3 study, presented Friday at the European Academy of Neurology (EAN) conference, demonstrated superiority of nipocalimab over placebo in improving activities of daily living.The trial enrolled 199 adults with gMG, including 153 who were antibody-positive. Participants received either nipocalimab or placebo, both in addition to standard of care. Efficacy results showed that nipocalimab-treated patients achieved a 4.70-point improvement from baseline in the Myasthenia Gravis - Activities of Daily Living (MG-ADL) score over weeks 22, 23, and 24, compared to a 3.25-point improvement seen with placebo. The gap was statistically significant."For someone living with gMG, a one- to two-point change on MG-ADL may be the difference between normal eating and frequent choking on food, or shortness of breath at rest and being on a ventilator," the company said in its release.Key secondary endpoints were also met, including improvements in muscle strength and function as measured by the Quantitative Myasthenia Gravis (QMG) score over weeks 22 and 24. Further, a significantly greater proportion of nipocalimab-treated patients experienced ≥2-point improvement from baseline in MG-ADL response.Safety data were consistent with previous nipocalimab studies, with similar rates of serious adverse events and discontinuations between the nipocalimab and placebo groups.'Sizeable opportunity'J&J, which acquired the anti-FcRn antibody through its $6.5-billion takeout of Momenta Pharmaceuticals back in 2020, plans to submit regulatory filings for nipocalimab in gMG later this year, potentially setting up a competitive challenge to Vyvgart. Argenx's product generated $1.2 billion in 2023 sales.However, J&J aims to differentiate nipocalimab by highlighting its efficacy across a broader range of antibody-positive gMG patients, including those with anti-MuSK and anti-LRP4 antibodies, in addition to the more common anti-AChR antibodies. A recent Morgan Stanley note cited one key opinion leader (KOL) as seeing a "sizable opportunity" for nipocalimab in gMG, potentially as a second-line option for patients with incomplete response to Vyvgart. The KOL estimated that about 50% of Vyvgart patients could fit that portrait.The gMG success adds to nipocalimab's growing portfolio of positive clinical data across autoimmune indications. Earlier this month, J&J reported that nipocalimab had met its primary endpoint in a Phase II trial for Sjögren's disease. The drug has also shown promise in rare maternal-foetal disorders.

Harbour BioMed today announced that it has formally resubmitted the Biologics License Application (BLA) for its core drug candidate, batoclimab (HBM9161), a fully human monoclonal antibody (mAb) targeting generalized myasthenia gravis (gMG), to the National Medical Products Administration (NMPA) of China.   Batoclimab (HBM9161), as a fully human monoclonal antibody, works by blocking the interaction between FcRn and IgG, thereby accelerating the clearance of pathogenic IgG, providing an effective treatment for autoimmune diseases mediated by pathogenic IgG. In the Phase III clinical study for generalized myasthenia gravis, batoclimab has demonstrated positive therapeutic effects, rapidly and significantly relieving patients' clinical symptoms and improving their quality of life, with a favorable safety profile.   Based on the rigorous execution of the clinical trial protocol, Harbour BioMed has successfully completed the extension study of the Phase III clinical trial without recruiting new patients and collected more long-term safety data. Therefore, the company has proactively included these safety data and resubmitted the BLA for batoclimab as planned.   Dr. Jingsong Wang, Founder, Chairman and CEO of Harbour BioMed, said, "We are pleased to resubmit the BLA as planned and will continue to maintain close communication with NMPA to facilitate the review of this innovative therapy. The results of the Phase III clinical trial have fully demonstrated the significant efficacy of batoclimab in both primary and secondary endpoints, and we are confident that this innovative drug will bring a new treatment option for patients with generalized myasthenia gravis and benefit more patients."   Recalling the previous announcement, Harbour BioMed had announced on December 1, 2023, the withdrawal of the marketing application for batoclimab (HBM9161) for the treatment of generalized myasthenia gravis (gMG), and planned to resubmit it in the first half of 2024. The company stated that this withdrawal was due to the delay in the Phase III study of batoclimab, which required the collection of more long-term safety data.   It is worth noting that batoclimab was granted the "Breakthrough Therapy Designation" by the China National Medical Products Administration in 2021 and completed the proof-of-concept study for Chinese patients with generalized myasthenia gravis in August of the same year. In March 2023, the company announced positive results from its Phase III clinical trial. In addition, Harbour BioMed has reached a collaboration agreement with NBP Pharmaceutical Co., Ltd., a wholly-owned subsidiary of CSPC, to jointly develop batoclimab in Greater China, committed to providing more treatment options for patients with this disease.   In the field of FcRn antibody drugs, the current market is still in the early stage of development, with relatively few participants. Currently, the FcRn antibodies that have been marketed include Efgartigimod α co-developed by Argenx and Zai Lab, and Rozanolixizumab from UCB, while Nipocalimab from Johnson & Johnson is in Phase III clinical trials. Harbour BioMed's batoclimab will bring new hope and vitality to this field.