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Novartis (NVS) Presents Positive Data on Rare Kidney Disease Drug

Novartis NVS presented encouraging results from a pre-specified interim analysis of the phase III APPLAUSE-IgAN study of Fabhalta (iptacopan).

Fabhalta, an oral, Factor B inhibitor of the alternative complement pathway, is already approved by the FDA for the treatment of adults with the rare blood disorder paroxysmal nocturnal hemoglobinuria.

Novartis AG Price and Consensus

Novartis AG Price and Consensus
Novartis AG Price and Consensus

Novartis AG price-consensus-chart | Novartis AG Quote

Novartis is looking to expand the drug’s label for the treatment of immunoglobulin A nephropathy (IgAN), a heterogeneous, progressive, rare kidney disease.

APPLAUSE-IgAN is a phase III multicenter, randomized, double-blind, placebo-controlled, parallel-group study to evaluate the efficacy and safety of twice-daily oral Fabhalta (200 mg) in 518 adults with IgAN.


The primary endpoint of the study for the interim analysis and the final analysis is proteinuria reduction at 9 months as measured by UPCR and the annualized total eGFR slope over 24 months, respectively.

This pre-specified interim analysis included 250 patients for the efficacy analysis and 443 for the safety analysis. Interim analysis data showed that patients treated with Fabhalta achieved a 38.3% proteinuria reduction (as measured by 24-hour UPCR) at nine months when compared to placebo on top of supportive care.

The proteinuria reduction of 38.3% was clinically meaningful and statistically significant.  Novartis stated that proteinuria reduction is an increasingly recognized surrogate marker correlating with progression to kidney failure and has been used as an endpoint in IgAN clinical trials to support accelerated approvals.

Results were presented at a late-breaking clinical trials session at the World Congress of Nephrology in Buenos Aires, Argentina.

APPLAUSE-IgAN is the first and only phase III study to demonstrate significant proteinuria reduction by targeting the complement system in patients with IgAN. The study also showed that Fabhalta was well tolerated with a favorable safety profile consistent with the previously reported data.

Novartis has submitted its application to the FDA for a possible accelerated approval of the drug in this indication. The regulatory body accepted the same and granted priority review.

The primary endpoint evaluating Fabhalta's ability to slow IgAN progression by measuring the annualized total eGFR slope over 24 months is expected at the study’s completion in 2025.

A potential approval of Fabhalta for IgAN should increase the sales potential of the drug.

Fabhalta is currently being evaluated for a range of rare diseases, including C3G, atypical hemolytic uremic syndrome, immune complex membranoproliferative glomerulonephritis and lupus nephritis.

Shares of Novartis have lost 6.5% in the year-to-date period against the industry’s growth of 9.3%.

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Zacks Investment Research

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With the successful spin-off of the Sandoz business in 2023, Novartis is now a pure-play innovative medicine company with a focus on these core therapeutic areas — cardiovascular, renal and metabolic, immunology, neuroscience and oncology.

Earlier in 2023, Novartis acquired Chinook Therapeutics for $3.5 billion to strengthen its renal pipeline. The acquisition added two late-stage candidates, atrasentan and zigakibart, for the treatment of IgAN to NVS’ pipeline.

In December 2023, Calliditas Therapeutics CALT obtained FDA approval for Tarpeyo delayed-release capsules to reduce the loss of kidney function in adults with primary IgAN at risk for disease progression. The drug was first approved in December 2021, under accelerated approval, based on the surrogate marker of proteinuria.

Last month, Calliditas Therapeutics announced that the FDA has granted an orphan drug exclusivity period of seven years for Tarpeyo, expiring in December 2030, based on when the company obtained full approval with a new indication for this drug.

The exclusivity period reflects the new indication covering all adult patients with primary IgAN at risk of disease progression, based on a confirmed reduction of kidney loss reflecting a clinical benefit on kidney function for adult patients with primary IgAN.

Zacks Rank & Stocks to Consider

NVS currently carries a Zacks Rank #3 (Hold). A couple of better-ranked stocks from the biotech sector are ADMA Biologics, Inc. ADMA and ANI Pharmaceuticals, Inc. ANIP, both sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.

In the past 60 days, estimates for ADMA Biologics’ 2024 earnings per share (EPS) have improved from 22 to 30 cents. In the past year, shares of ADMA have surged 88.5%.

ADMA Biologics’ earnings beat estimates in three of the trailing four quarters and met once, delivering an average surprise of 85.00%.

In the past 60 days, estimates for ANI Pharmaceuticals’ 2024 EPS have improved from $4.06 to $4.43. In the past year, shares of ANIP have surged 75.5%.

ANI Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 109.06%.

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