Everest Medicines' Partner Calliditas Therapeutics Announces Nefecon® the Only FDA-approved Treatment for IgA Nephropathy to Significantly Slow Kidney Function Decline

Rhea-AI Summary

Everest Medicines (HKEX 1952.HK) licensing partner Calliditas Therapeutics AB (Nasdaq: CALT, Nasdaq Stockholm: CALTX) announced that the U.S. FDA has fully approved Nefecon® delayed release capsules for adults with primary immunoglobulin A nephropathy (IgAN) at risk for disease progression. Nefecon® demonstrated a significant benefit over placebo in reducing kidney function loss, with a 50% less deterioration in kidney function compared to placebo over a 2-year period. The drug is also approved in China and showed a greater treatment effect in the Chinese subpopulation compared to the global population. Nefecon® is considered a revolutionary therapeutic option for IgAN patients, offering a disease-modifying treatment with sustained and clinically meaningful reduction in eGFR declines and proteinuria. The drug's approval represents a significant milestone for Everest Medicines and its partner Calliditas Therapeutics AB, with plans for a China launch in 2024.

Positive

• Full FDA approval for Nefecon® is a significant milestone, confirming the drug's capability to preserve kidney function and significantly delay disease progression.
• Nefecon® demonstrated a 50% less deterioration in kidney function compared to placebo over a 2-year period, indicating a significant benefit for patients with primary IgAN.
• The drug's approval in China and the greater treatment effect observed in the Chinese subpopulation compared to the global population indicate a promising outlook for addressing the urgent need for effective treatment options for IgAN patients in China.
• The drug offers a revolutionary therapeutic option for IgAN patients, providing a disease-modifying treatment with sustained and clinically meaningful reduction in eGFR declines and proteinuria.

Negative

• None.

Insights

Medical Research Analyst

The FDA's full approval of Nefecon® for primary IgAN patients marks a significant milestone in the treatment of a chronic kidney disease with limited therapeutic options. The robust clinical data indicating a 50% reduction in kidney function deterioration compared to placebo sets a new standard in IgAN management. This approval could potentially lead to increased demand and market share for Everest Medicines, as they prepare to launch the treatment in China, which has the highest IgAN patient population. The impact on Everest Medicines' financials could be substantial, as the drug addresses a previously unmet medical need.

From an investment perspective, the full FDA approval enhances the credibility and market potential of Nefecon®, possibly leading to positive investor sentiment. The long-term benefits for stakeholders include sustained revenue growth from a novel treatment in a high-prevalence market. However, investors should monitor the post-market safety profile and adoption rate among healthcare providers.

Pharmaceutical Industry Analyst

The recent FDA approval of Nefecon® represents a strategic advantage for Everest Medicines in the pharmaceutical industry, particularly within the nephrology sector. The drug's ability to modulate B-cell immunity and reduce pathogenic antibodies is a novel approach in IgAN treatment. The statistically significant eGFR benefit over placebo, observed in clinical trials, underscores the drug's efficacy.

For industry stakeholders, this development could signal a shift towards more innovative treatments for chronic kidney diseases. The drug's performance in the Chinese subpopulation suggests a potentially higher efficacy in this demographic, which may influence marketing strategies and competitive positioning. Everest Medicines' stock could see increased volatility as the market reacts to this news and anticipates the drug's commercial performance in China.

Healthcare Economist

With the full FDA approval of Nefecon®, Everest Medicines is poised to address a significant economic burden associated with IgAN, particularly in China. IgAN, being a leading cause of end-stage kidney disease, incurs considerable healthcare costs due to dialysis and transplantation. The introduction of Nefecon® may reduce these long-term costs by preserving kidney function and delaying disease progression.

Economic implications for the healthcare system include potential savings from reduced chronic kidney disease progression and its associated treatments. However, the cost-effectiveness of Nefecon® will be a critical factor in its adoption. Payers and healthcare providers will need to balance the drug's price with its clinical benefits to determine its value proposition in the treatment of IgAN.

SHANGHAI, Dec. 20, 2023 /PRNewswire/ -- Everest Medicines (HKEX 1952.HK, "Everest", or the "Company")'s licensing partner Calliditas Therapeutics AB (Nasdaq: CALT, Nasdaq Stockholm: CALTX) ("Calliditas") announced that the U.S. Food and Drug Administration (FDA) had approved Nefecon^® delayed release capsules to reduce the loss of kidney function in adults with primary immunoglobulin A nephropathy (IgAN) at risk for disease progression. Nefecon^® was first approved in December 2021 under the FDA accelerated approval pathway and is now the first fully FDA-approved treatment for IgAN based on a measure of kidney function.

• The FDA approval is for adults with primary IgAN who are at risk for disease progression, irrespective of proteinuria levels.
• Nefecon^® is now approved by the FDA with a confirmed and statistically significant benefit over placebo (p<0.0001) in estimated glomerular filtration rate (eGFR) over the two-year period that consisted of 9 months of treatment with Nefecon^® or optimized RASi and a 15-month follow-up off drug.
• At 2 years, there was a 6.11 mL/min/1.73 m² decline in eGFR in the Nefecon^® group compared with a 12.0 mL/min/1.73 m² decline in the placebo group (p<0.0001), representing 50% less deterioration of kidney function in Nefecon^® -treated patients compared to placebo-treated patients over the 2-year period.
• Nefecon^® is a B-cell immunomodulator designed to target the origin of the disease and reduce the production of pathogenic galactose-deficient IgA1 antibodies, which cause IgAN.
• Nefecon^® was approved by China's National Medical Products Administration in November 2023. Results from Chinese subpopulation analysis demonstrated a numerically greater magnitude of Nefecon^® treatment effect over placebo in kidney function, proteinuria and microhematuria compared to the global population.

"We congratulate our partner on the full FDA approval of Nefecon^®, which confirms the drug's capability to preserve kidney function and significantly delay disease progression. This first-ever fully FDA-approved treatment for IgAN represents a beacon of hope for the entire IgAN community globally," said Rogers Yongqing Luo, Chief Executive Officer of Everest Medicines.  "On the back of this important milestone, we are excited to be launching this first-in-disease treatment in China in the first quarter of 2024. China has the largest population of IgAN patients in the world with more rapid disease progression, and we are very happy to have the solution to address this urgent need."

"Nefecon^® represents a revolutionary therapeutic option for Chinese patients who have more rapid disease progression in IgAN with no effective treatment options. This medicine offers patients a truly disease modifying treatment with sustained and clinically meaningful reduction in eGFR declines along with durable proteinuria reduction. The FDA full approval and the China approval allow physicians to take more proactive measures in managing the impact of the disease to preserve kidney function," said Professor Zhang Hong at Peking University First Hospital, a member of the global steering committee for the Phase 3 NefIgArd clinical trial. "Based on the exciting results in both efficacy and safety in the Chinese population observed from the NefIgArd trial, we believe Nefecon^® will bring significant changes to Chinese patients with IgAN."

In the global Phase 3 trial, Nefecon^® demonstrated a highly statistically significant and clinically relevant benefit compared to placebo in eGFR over the two-year period. The reduction in UPCR observed with Nefecon^® treatment was also durable and the proportion of patients with microhematuria in the Nefecon group declined. Nefecon^® was also generally well-tolerated in the Phase 3 clinical trial.

Further subpopulation analysis from the NefIgArd clinical trial showed a numerically greater treatment effect in kidney function prevention, proteinuria reduction and microhematuria improvement in the Chinese subpopulation compared to the global population. The mean absolute change from baseline in eGFR at 24 months showed approximately 66% less deterioration in kidney function with 9-month Nefecon^® treatment compared with a smaller preservation of kidney function (50%) in the global population. Patients treated with Nefecon^® in China showed a 43% greater reduction (95% CI 8%, 65%) in UPCR compared with placebo at 24 months and a 31% greater reduction (95% CI 0, 53) at 9 months.  In the global population, Nefecon^® treatment provided an approximately 30% greater reduction in UPCR at both 24 months and 9 months compared to placebo. The proportion of Chinese patients without microhematuria in the Nefecon^® group increased from 26.9% at baseline to 57.7% during observational follow-up, while it was maintained at 14.3% in the placebo group.

About Nefecon^®

Nefecon^® is a patented oral, delayed release formulation of budesonide, a corticosteroid with potent glucocorticoid activity and weak mineralocorticoid activity that undergoes substantial first pass metabolism. The formulation is designed as a delayed release capsule that is enteric coated so that it remains intact until it releases budesonide to the distal ileum. Each capsule contains coated beads of budesonide that target mucosal B-cells present in the ileum where the disease originates, as per the predominant pathogenesis models.

In June 2019, Everest Medicines entered into an exclusive, royalty-bearing license agreement with Calliditas, which gives Everest Medicines exclusive rights to develop and commercialize Nefecon^® in Mainland China, Hong Kong, Macau, Taiwan and Singapore. The agreement was extended in March 2022 to include South Korea as part of Everest Medicine's territories.

About Everest Medicines

Everest Medicines is a biopharmaceutical company focused on developing, manufacturing and commercializing transformative pharmaceutical products and vaccines that address critical unmet medical needs for patients in Asian markets. The management team of Everest Medicines has deep expertise and an extensive track record from both leading global pharmaceutical companies and local Chinese pharmaceutical companies in high-quality discovery, clinical development, regulatory affairs, CMC, business development and operations. Everest Medicines has built a portfolio of potentially global first-in-class or best-in-class molecules in the company's core therapeutic areas of renal diseases, infectious diseases and autoimmune disorders. For more information, please visit its website at www.everestmedicines.com.

Forward-Looking Statements:

This news release may make statements that constitute forward-looking statements, including descriptions regarding the intent, belief or current expectations of the Company or its officers with respect to the business operations and financial condition of the Company, which can be identified by terminology such as "will," "expects," "anticipates," "future," "intends," "plans," "believes," "estimates," "confident" and similar statements. Such forward-looking statements are not guarantees of future performance and involve risks and uncertainties, or other factors, some of which are beyond the control of the Company and are unforeseeable. Therefore, the actual results may differ from those in the forward-looking statements as a result of various factors and assumptions, such as future changes and developments in our business, competitive environment, political, economic, legal and social conditions. The Company or any of its affiliates, directors, officers, advisors or representatives has no obligation and does not undertake to revise forward-looking statements to reflect new information, future events or circumstances after the date of this news release, except as required by law.

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SOURCE Everest Medicines

FAQ

What is the significance of the FDA approval of Nefecon® for Everest Medicines (HKEX 1952.HK) and Calliditas Therapeutics AB (Nasdaq: CALT, Nasdaq Stockholm: CALTX)?

The FDA approval of Nefecon® is a significant milestone for Everest Medicines and Calliditas Therapeutics AB, confirming the drug's capability to preserve kidney function and significantly delay disease progression.

What is the benefit of Nefecon® over placebo in reducing kidney function loss?

Nefecon® demonstrated a 50% less deterioration in kidney function compared to placebo over a 2-year period, indicating a significant benefit for patients with primary IgAN.

What is the significance of Nefecon®'s approval in China?

The approval of Nefecon® in China indicates a promising outlook for addressing the urgent need for effective treatment options for IgAN patients in China, with plans for a China launch in 2024.

What is the treatment effect of Nefecon® in the Chinese subpopulation compared to the global population?

Nefecon® showed a greater treatment effect in the Chinese subpopulation compared to the global population, indicating a promising outlook for addressing the urgent need for effective treatment options for IgAN patients in China.

What does Nefecon® offer for IgAN patients?

Nefecon® offers a revolutionary therapeutic option for IgAN patients, providing a disease-modifying treatment with sustained and clinically meaningful reduction in eGFR declines and proteinuria.