Vera Therapeutics Receives U.S. FDA Breakthrough Therapy Designation for Atacicept in Immunoglobulin A Nephropathy (IgAN)
Vera Therapeutics announced that the FDA has granted Breakthrough Therapy Designation to its drug atacicept for the treatment of Immunoglobulin A Nephropathy (IgAN). This decision is based on data from a Phase 2b ORIGIN trial, showing atacicept's potential to significantly improve kidney function as measured by eGFR over existing treatments. The company plans to release long-term results from this trial later in 2024 and expects Phase 3 trial results in the first half of 2025, which will support its regulatory approval submission.
- FDA granted Breakthrough Therapy Designation for atacicept in IgAN.
- Phase 2b ORIGIN trial data shows stabilization of eGFR over 72 weeks.
- Potential for atacicept to significantly improve kidney function compared to existing treatments.
- Positive long-term clinical trial results expected later in 2024.
- Phase 3 trial results anticipated in the first half of 2025.
- Regulatory approval for atacicept is still pending, with final data expected in 2025.
- Initial treatment period in the trial was only 36 weeks, with additional open-label follow-up.
Insights
The FDA granting Breakthrough Therapy Designation to Vera Therapeutics' atacicept for IgAN is a significant milestone. This designation is reserved for drugs that show substantial improvement over existing treatments for serious conditions, suggesting that atacicept has shown promising results in its Phase 2b trials.
IgA Nephropathy (IgAN) is a kidney disease that can lead to end-stage renal disease. Current treatments mainly manage symptoms, but they do not address the underlying cause or significantly alter the disease's progression. The stabilization of eGFR (estimated Glomerular Filtration Rate) is a critical endpoint because it indicates how well the kidneys are filtering blood. Atacicept’s ability to stabilize eGFR over 72 weeks suggests it could offer a meaningful improvement over existing therapies.
For retail investors, the news implies that Vera Therapeutics is making progress towards a potentially lucrative treatment in a niche market. However, it's important to note that the pivotal Phase 3 trial results, expected in the first half of 2025, will be the determining factor for regulatory approval. Investors should keep an eye on these upcoming results and any potential hurdles in the regulatory process.
The designation also means that atacicept will benefit from expedited development and review processes, potentially reducing time to market. This can lead to faster revenue generation if the drug is approved, but investors should remain cautious until later-stage trial results confirm these early findings.
The announcement that Vera Therapeutics has received Breakthrough Therapy Designation for atacicept is a positive development for the company’s financial outlook. This designation often leads to increased investor confidence, which may result in a rise in the company's stock price in the short term.
From a financial perspective,
However, investors should also consider the financial risks involved. The costs of continued clinical trials, potential delays and the competitive landscape could impact the company’s financial health. It's worth noting that while the Breakthrough Therapy Designation expedites the review process, it does not guarantee approval.
Long-term investors should monitor the company’s burn rate and funding needs, especially as they approach the Phase 3 trial's primary endpoint results. The outcome of these results will be pivotal in determining future financial stability and growth prospects for Vera Therapeutics.
The Breakthrough Therapy Designation for atacicept in treating IgAN positions Vera Therapeutics favorably within the biopharmaceutical market. This designation signals the FDA's recognition of the drug's potential, which can enhance Vera's credibility and attract strategic partnerships or acquisition interest.
IgAN is a niche yet significant market, with limited effective treatments currently available. The reported stabilization of eGFR with atacicept could meet a significant unmet medical need, potentially capturing a substantial market share upon approval. Vera's strategy to release long-term trial results later this year and Phase 3 trial results in 2025 will be critical milestones for market positioning and competitive advantage.
Investors should consider the competitive landscape: other biotech firms are also developing treatments for IgAN. The ability of atacicept to stand out will depend on subsequent trial outcomes and how the drug’s efficacy and safety profile compare to existing and emerging therapies.
In summary, the designation strengthens Vera’s market position, but the final market impact will hinge on future clinical trial results and competitive dynamics.
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 trial
The 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 disease
IgAN, 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 Atacicept
Atacicept 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 Vera
Vera 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 Statements
Statements 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 Allaire
LifeSci Advisors
212-915-2569
jallaire@lifesciadvisors.com
Media Contact:
Mari Purpura
LifeSci Advisors
mpurpura@lifesciadvisors.com
FAQ
What is the FDA Breakthrough Therapy Designation for Vera Therapeutics' atacicept?
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