Vera Therapeutics Receives U.S. FDA Breakthrough Therapy Designation for Atacicept in Immunoglobulin A Nephropathy (IgAN)

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BRISBANE, Calif., May 28, 2024 (GLOBE NEWSWIRE) -- Vera Therapeutics, Inc. (Nasdaq: VERA), a late clinical-stage biotechnology company focused on developing and commercializing transformative treatments for patients with serious immunological diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to atacicept for the treatment of IgAN. The designation reflects the FDA’s determination that, based on an assessment of data from the Phase 2b ORIGIN clinical trial of atacicept for IgAN, atacicept may demonstrate substantial improvement on a clinically significant endpoint over available therapies for patients with IgAN.

“We are pleased that the FDA has determined that the accumulated clinical data of atacicept in IgAN may demonstrate substantial improvement on kidney function as measured by eGFR over available therapies, and we believe that atacicept has the potential to be a transformative treatment for patients,” said Marshall Fordyce, M.D., Founder and CEO of Vera Therapeutics. “Later this year we plan to announce long-term 96-week results from the Phase 2b ORIGIN trial, and in the first half of 2025 we expect the primary endpoint results from our pivotal Phase 3 ORIGIN 3 trial, which are anticipated to support submission for regulatory approval of atacicept in IgAN.”

The FDA’s Breakthrough Therapy Designation is designed to expedite the development and review of drugs that are intended to treat a serious condition and for which preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapies on one or more clinically significant endpoints.

As part of the Breakthrough Therapy Designation request, the FDA reviewed data from the Phase 2b ORIGIN trial of atacicept in IgAN which demonstrate the stabilization of eGFR over 72 weeks of treatment. Participants first received atacicept in a 36-week double-blind period, and then continued to receive atacicept 150 mg self-administered subcutaneously once weekly at home during 36 additional weeks of open-label follow-up. Importantly, the stabilization of eGFR with atacicept reflects an eGFR profile more consistent with that of the general population versus those presenting with IgAN. Vera believes the stability of eGFR with atacicept represents a substantial potential improvement over currently available therapies.

About the Phase 2b ORIGIN clinical trialThe Phase 2b ORIGIN clinical trial (NCT04716231) is a global, multicenter, randomized, double-blind, placebo-controlled trial evaluating the safety and efficacy of atacicept in 116 patients with IgAN who continue to have persistent proteinuria and remain at high risk of disease progression despite being on a stable prescribed regimen of a renin-angiotensin-aldosterone system inhibitor (RAASi) for at least 12 weeks that is the maximum labeled or tolerated dose. The Phase 2b ORIGIN clinical trial evaluated three dose strengths of atacicept versus placebo, administered weekly by prefilled syringe. Patients were randomized 2:2:1:2 to atacicept 150 mg, atacicept 75 mg, atacicept 25 mg, or matching placebo. Upon completion of the 36-week blinded treatment period, all patients were offered open-label atacicept 150 mg for an additional 60 weeks.

The primary endpoint was the change in proteinuria as evaluated by urine protein to creatinine ratio (UPCR) at week 24 and the key secondary endpoint was the change in proteinuria as evaluated by UPCR at week 36. Additional exploratory endpoints include change in proteinuria as evaluated by UPCR at weeks 12, 48, and 96; change in estimated glomerular filtration rate (eGFR); change in serum immunoglobulin levels, and serum galactose-deficient IgA1 (Gd-IgA1) levels; safety and tolerability; and serum pharmacokinetics (PK).

The trial met its primary and key secondary endpoints, with statistically significant and clinically meaningful proteinuria reductions and stabilization of eGFR versus placebo through week 36. The safety profile was comparable between atacicept and placebo.

For more information about the Phase 2b ORIGIN clinical trial, please visit www.clinicaltrials.gov.

About the Phase 3 clinical trial (ORIGIN 3)The ORIGIN 3 clinical trial (NCT04716231) is a global, multicenter, randomized, double-blind, placebo-controlled Phase 3 trial evaluating the safety and efficacy of atacicept 150 mg in patients with IgAN who continue to have persistent proteinuria and remain at high risk of disease progression despite being on a stable prescribed regimen of renin-angiotensin system inhibitors (RASi) (ACEi or ARB) for at least 12 weeks that is the maximum labeled or tolerated dose. The objectives of the trial are to determine the effect of atacicept on proteinuria and preservation of kidney function compared to placebo.

The Phase 3 trial is composed of up to a 4-week screening period, a 104-week double-blind treatment period, a 52-week open-label extension and 26 weeks of follow-up. Participants will be randomized 1:1 to atacicept 150 mg once weekly subcutaneous injections (N=188) or placebo once weekly subcutaneous injections (N=188) for 104 weeks, followed by a 52-week open-label extension. The primary endpoint is the change from baseline in proteinuria as evaluated by urine protein to creatinine ratio (UPCR) at week 36. The key secondary endpoint is annualized rate of change in estimated glomerular filtration rate (eGFR) up to week 104. Additional secondary endpoints are the change in Gd-IgA1, change in eGFR up to week 52, and time from randomization to first occurrence of composite kidney failure endpoint event.

For more information about the ORIGIN 3 clinical trial, please visit www.clinicaltrials.gov.

About IgA nephropathy (IgAN), or Berger’s diseaseIgAN, also known as Berger’s disease, is a serious and progressive autoimmune disease of the kidney, for which there remains a high unmet medical need. IgAN is driven by the production of immunogenic Gd-IgA1, which triggers autoantibodies that lead to the formation of pathogenic immune complexes, which become trapped in the kidney’s glomeruli, causing inflammation and progressive damage. In up to 50 percent of patients, IgAN can lead to end-stage kidney disease (ESKD) or kidney failure, which has considerable morbidity and impact on patients’ lives.

About AtaciceptAtacicept is an investigational recombinant fusion protein that contains the soluble transmembrane activator and calcium-modulating cyclophilin ligand interactor (TACI) receptor that binds to the cytokines B-cell activating factor (BAFF) and A proliferation-inducing ligand (APRIL). These cytokines are members of the tumor necrosis factor family that promote B-cell survival and autoantibody production associated with certain autoimmune diseases, including IgAN and lupus nephritis. Vera believes atacicept is positioned for best-in-class potential, targeting B cells and plasma cells to reduce autoantibodies and having been administered to more than 1,500 patients in clinical studies across different indications.

About VeraVera Therapeutics is a late clinical-stage biotechnology company focused on developing treatments for serious immunological diseases. Vera’s mission is to advance treatments that target the source of immunological diseases in order to change the standard of care for patients. Vera’s lead product candidate is atacicept, a fusion protein self-administered as a subcutaneous injection once weekly that blocks both B-cell Activating Factor (BAFF) and A PRoliferation-Inducing Ligand (APRIL), which stimulate B cells and plasma cells to produce autoantibodies contributing to certain autoimmune diseases, including IgAN, also known as Berger’s disease, and lupus nephritis. In addition, Vera is evaluating additional diseases where the reduction of autoantibodies by atacicept may prove medically useful. Vera is also developing MAU868, a monoclonal antibody designed to neutralize infection with BK virus (BKV), a polyomavirus that can have devastating consequences in certain settings such as kidney transplant. Vera retains all global developmental and commercial rights to atacicept and MAU868. For more information, please visit www.veratx.com.

Forward-looking StatementsStatements contained in this press release regarding matters, events or results that may occur in the future are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, atacicept’s potential to be a best-in-class therapy for patients with IgAN, Vera’s expectations regarding presenting 96-week data from the Phase 2b ORIGIN trial in 2024, Vera’s plans to receive and share topline data from the pivotal Phase 3 trial in the first half of 2025, Vera’s expectations regarding its submission for regulatory approval of atacicept for IgAN and Vera’s product candidates, strategy, and regulatory matters. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as “potential,” “will,” “may,” “expected,” “plan,” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Vera’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks related to the regulatory approval process, results of earlier clinical trials may not be obtained in later clinical trials, preliminary results may not be predictive of topline results, risks and uncertainties associated with Vera’s business in general, the impact of macroeconomic and geopolitical events, and the other risks described in Vera’s filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management’s assumptions and estimates as of such date. Vera undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

For more information, please contact:

Investor Contact:Joyce AllaireLifeSci Advisors212-915-2569jallaire@lifesciadvisors.com

Media Contact:Mari PurpuraLifeSci Advisorsmpurpura@lifesciadvisors.com

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Source: Vera Therapeutics

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