Novartis Touts Encouraging Data From Late-Stage Study For Fabhalta In Rare Type Of Blood Disorder

On Friday, Novartis AG NVS revealed topline results from the APPULSE-PNH Phase 3B study of Fabhalta (iptacopan) in adult patients with paroxysmal nocturnal hemoglobinuria (PNH) who were switched from anti-C5 therapies (Hb ≥10g/dL following treatment with eculizumab or ravulizumab).

Ultomiris (ravulizumab) and Soliris (eculizumab) are commonly used anti-C5 therapies, and they are made by AstraZeneca Plc AZN.

Also Read: Positive Outlook For Novartis As Company Capitalizes On Growing Treatment Opportunities, Analyst Highlights

PNH is a rare complement-mediated blood disorder. People with PNH have an acquired mutation in some of their hematopoietic stem cells, which are located in the bone marrow and can grow and develop into red blood cells, white blood cells, and platelets.

This mutation causes them to produce RBCs that are susceptible to premature destruction by the complement system.

After 24 weeks of treatment with Fabhalta, the average hemoglobin (Hb) level improved compared to baseline. The study’s safety profile of Fabhalta monotherapy was consistent with previously reported data.

Data will be presented at an upcoming medical meeting in 2025.

The FDA recently granted accelerated approval for Fabhalta to reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression, and development is ongoing in multiple complement-mediated diseases.

Last month, Novartis raised its mid-term sales guidance, increasing its compound annual growth rate (CAGR) target to 6% from 5% for 2023-2028.

The upgrade reflects performance from current growth drivers and anticipated launches, many of which are expected to retain U.S. exclusivity into the 2030s.

Earlier this week, PTC Therapeutics PTCT signed an exclusive global license and collaboration agreement with Novartis for its PTC518 Huntington’s disease program, which includes related molecules.

Under the agreement, PTC will receive an upfront payment of $1.0 billion, up to $1.9 billion in development, regulatory and sales milestones, a profit share in the U.S., and double-digit tiered royalties on ex-U.S. sales.

Is Novartis A Good Stock To Buy?

Wall Street analysts view Novartis on the whole as a Neutral, given the history of coverage over the past three months. Graham Perry from B of A Securities in Novartis is the most bearish, expecting a 3.7% fall in the stock in the coming year.

But looking at how the market as a whole thinks of the stock, you can reference historical price action for views on whether investors feel strongly about the stock one way or another. In the past 3 months, Novartis fell 12.4%, which indicates that opinion soured on the business and how attractive it is to own based on either its stock price, or underlying fundamentals, like revenue, which rose 8.84% over the past year.

A complete overview of how Wall Street views individual stocks is available here, while real time updates on the latest analyst actions will be delivered via Benzinga PRO. Try it for free.

Price Action: At the last check on Friday, NVS stock was up 0.18% to $102.34.

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