Vera Therapeutics is a clinical-stage biotechnology company focused on developing treatments for serious immunological diseases.
What obstacles have hindered progress in treating autoimmune diseases? What has Vera Therapeutics discovered to address these challenges?
The main challenge has been understanding the complex biology of autoimmune diseases and translating that knowledge into precise treatments. Autoimmune diseases involve a variety of immune system cells, and while we have learned much about B cells—the cells responsible for producing protective antibodies—their potential as a therapeutic target has been underutilized. At Vera, we focused on IgA nephropathy, an autoimmune kidney disease where the root cause, a molecule called galactose-deficient IgA1, is well understood. This clarity allowed us to design a targeted trial to test whether reducing IgA1 levels could effectively treat the disease.
Our lead candidate, Atacicept, works by dialing down the levels of IgA1, the initial trigger for IgA nephropathy. Data from 2024 has shown that patients taking Atacicept in the Phase 2b ORIGIN trial maintain normal kidney function over two years—an unprecedented result for this disease. IgA nephropathy, which often leads to end-stage kidney disease and dialysis, has outcomes comparable to lung or colorectal cancer in terms of mortality. The ability of Atacicept to prevent kidney function decline offers hope for transforming the treatment landscape for autoimmune kidney diseases and beyond.
What does Atacicept's success mean for patients?
IgA nephropathy is the most common primary glomerulonephritis, typically affecting young individuals around the age of 35. In the United States, approximately 160,000 people are diagnosed with the disease, and globally, it affects millions. Without effective treatment, most patients face kidney failure, requiring dialysis or transplantation, with a mortality rate comparable to certain cancers. Atacicept’s success in maintaining normal kidney function over two years marks a paradigm shift, providing a potential functional cure for these patients.
The data, presented at the American Society of Nephrology Conference and published in the Journal of the American Society of Nephrology, showed an annual kidney function decline of less than one GFR unit, which is virtually unheard of in kidney disease research. Atacicept is a patient-friendly, injectable therapy administered once weekly, similar to other well-known treatments like Ozempic. Over 90% of patients in the Phase 2b trial adhered to this regimen, demonstrating its feasibility and potential to improve long-term outcomes for those suffering from IgA nephropathy.
What are the broader implications of your work for treating autoimmunity?
While IgA nephropathy is our initial focus, the success of Atacicept opens the door to treating a range of autoimmune diseases. By addressing the overactivity of B cells, our approach has broad applications across nephrology, hematology, dermatology, and neurology. We are now expanding our research into additional autoimmune kidney diseases and other conditions that could benefit from this mechanism of action.
One of our promising areas of research is systemic lupus erythematosus (lupus). We already have phase 2 data showing that Atacicept can lead to full or near resolution of the disease in about 40% of patients. While IgA nephropathy has been our primary focus, we plan to conduct a pivotal trial in lupus, aiming to deliver similarly transformative outcomes in this challenging disease area.
What is the meaning of the FDA Breakthrough Therapy Designation Vera has received?
The FDA Breakthrough Therapy Designation is a recognition reserved for the most promising medicines under development. It provides an expedited pathway to approval, granting access to senior FDA members and streamlining the review process. We were honored to receive this designation earlier this year, as it accelerates our efforts to bring Atacicept to patients. Our Phase 3 trial, which began over a year ago, has already completed enrollment for the pivotal study. We expect to have Phase 3 results in the first half of next year and plan to file a Biologics License Application (BLA) with the FDA in the second half, aiming for a commercial launch in 2026.
Are you considering collaborations as you expand beyond kidney-related diseases?
Yes, collaborations are a significant focus for us, especially as we expand into broader autoimmune conditions. We recently announced a partnership with the University of Michigan to identify new patients outside of our pivotal Phase 3 trial. This initiative aims to match Atacicept with patients suffering from IgA nephropathy as well as other autoimmune kidney diseases, such as membranous nephropathy and FSGS. These collaborations enhance our ability to reach more patients and validate the therapeutic potential of our approach.
Currently, we do not have partnerships with other biopharmaceutical companies, as Atacicept’s unique mechanism of action makes it a disruptive medicine in this field. Unlike standard care, which typically involves ACE inhibitors to manage symptoms, Atacicept directly targets the root cause of autoimmune kidney diseases. This sets it apart and positions it as a standalone innovation in addressing these conditions.
Your stock recently hit an all-time high. How do you plan to manage growth and sustain this momentum?
Growth is an exciting and vital part of Vera’s journey. We view financial success as "frozen energy," a means to capitalize on our future and fulfill our promise to patients. Our strong market position, bolstered by successful fundraising efforts in 2024, ensures we are well-capitalized to deliver our first product launch in 2026 and transition to a commercial-stage company. This financial stability is critical for executing our vision and scaling efficiently.
Managing rapid growth requires discipline, a lean approach, and a strong team culture. At this stage, we focus on maintaining efficiency while expanding our capabilities. Biotech success depends on addressing unmet medical needs through innovative science and sound business practices. Vera is at a sweet spot for growth, leveraging our momentum to drive a transformative impact on patient care while building a sustainable business framework for long-term success.
What do you hope to achieve by this time next year?
By this time next year, we hope to have positive pivotal trial data from our Phase 3 study, confirming the results we achieved in Phase 2. This is a critical milestone in the regulatory process, and we aim to file our BLA by year-end. Additionally, we plan to have preliminary data showing Atacicept’s efficacy in treating new autoimmune diseases, both within and beyond kidney-related conditions.
Success in these areas will not only validate our lead program but also pave the way for expansion into broader opportunities. Our ultimate goal is to transform how autoimmune diseases are treated, delivering precise, science-driven solutions to patients in need. We are excited to continue building on our progress and bringing meaningful advancements to the field of medicine.
