Everest Medicines’ EVER001 shows promising Phase 1b/2a trial results for autoimmune kidney disease treatment

Everest Medicines has announced promising preliminary results from its ongoing Phase 1b/2a clinical trial of EVER001, a next-generation Bruton’s tyrosine kinase (BTK) inhibitor, for the treatment of primary membranous nephropathy (pMN). The results, derived from data collected up until September 13, 2024, indicate that the drug may offer an effective treatment option for patients suffering from this rare autoimmune kidney disease.

In the low-dose cohort of the trial, 9 out of 11 patients (81.8%) achieved overall clinical remission after 36 weeks of treatment, with 10 out of 11 (91%) reaching immunological complete remission. In the high-dose cohort, 6 out of 7 patients (85.7%) achieved overall clinical remission, and all patients reached immunological complete remission by week 24. These results suggest that EVER001 may be a powerful tool in addressing the substantial treatment gap in pMN management.

The drug also demonstrated significant efficacy in reducing proteinuria, a key marker of kidney damage. In the low-dose group, proteinuria was reduced by 78.3% by week 36, while the high-dose cohort saw a 73.8% reduction by week 24. Additionally, EVER001 effectively reduced anti-PLA2R antibodies, which are often implicated in pMN, with more than a 90% decrease observed in the low-dose group by week 24 and in the high-dose group by week 12.

Potential of EVER001 as a next-generation BTK inhibitor

EVER001, formerly known as XNW1011, is a covalent reversible BTK inhibitor designed to address the shortcomings of earlier-generation BTK inhibitors. These older treatments have been associated with significant side effects such as bleeding, arrhythmia, and severe infections. However, the results from this trial suggest that EVER001 may be safer and more effective, offering a more selective approach to treating autoimmune renal diseases.

Experts have praised EVER001 for its pharmacokinetic properties and selectivity, which are expected to differentiate it from other drugs in development. By targeting BTK, a key enzyme involved in the immune response, EVER001 could offer a new therapeutic approach to treating diseases like pMN, where B cell-mediated inflammation plays a central role.

The need for effective treatments in primary membranous nephropathy treatment

Primary membranous nephropathy, a leading cause of nephrotic syndrome in adults, continues to pose a major challenge for healthcare systems worldwide. In countries like China, pMN has become increasingly prevalent, with an estimated 2 million patients affected. The United States, Europe, and Japan also report significant numbers of cases. Despite this, there are currently no approved treatments for pMN, leaving many patients with limited options.

While current treatments aim to manage symptoms and improve remission rates, relapse remains common, and side effects from existing drugs often limit their use. With results from this trial showing high remission rates and a favorable safety profile, EVER001 could provide a much-needed alternative to current therapies.

Future prospects for EVER001 and Everest Medicines

The Phase 1b/2a trial, which is being conducted in China, has delivered strong evidence of EVER001’s potential in the treatment of primary membranous nephropathy. Everest Medicines is focused on advancing the clinical development of EVER001, with plans to present more detailed data at upcoming medical conferences and publications.

Rogers Yongqing Luo, Chief Executive Officer of Everest Medicines, expressed confidence in the results, noting that they represent the first public release of data from the company’s global pipeline. He added that Everest Medicines is committed to progressing the development of EVER001, aiming to meet the urgent needs of patients with autoimmune renal diseases.

As the company continues to explore the full potential of EVER001, the drug’s early success could pave the way for a new standard in the treatment of autoimmune renal diseases, with an emphasis on both efficacy and safety.