Novartis Unlocks Potential with Fabhalta: Encouraging Results from PNH Study
Novartis (NVS) has announced positive top-line results from a late-stage study on Fabhalta (iptacopan), aimed at adult patients suffering from paroxysmal nocturnal hemoglobinuria (PNH).
Fabhalta is an oral medication that acts as a Factor B inhibitor targeting the alternative complement pathway. The APPULSE-PNH study, a phase IIIB multicenter, single-arm, open-label trial, assessed the efficacy and safety of taking Fabhalta as a monotherapy (200mg twice daily) for adults with PNH who had previously undergone treatment with anti-C5 therapies (eculizumab or ravulizumab).
Year to date, shares of Novartis have risen 0.7%, while the industry has seen a robust growth of 9.5%.
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Insights from the APPULSE-PNH Trial
The APPULSE-PNH study enrolled 52 participants who received Fabhalta for a duration of 24 weeks.
Results indicated that oral Fabhalta improved average hemoglobin (Hb) levels compared to baseline in adults who switched from anti-C5 therapies. These positive outcomes highlight Fabhalta’s effectiveness for both patients transitioning from anti-C5 treatments and those who had not previously received complement-inhibiting therapies.
Moreover, the safety profile of Fabhalta monotherapy aligns with earlier reports. Notably, it was granted FDA approval in December 2023 for adult PNH treatment, alongside European Medicines Agency approval in May 2024 for adults with PNH-related hemolytic anemia.
Furthermore, Fabhalta recently received accelerated FDA approval for reducing proteinuria in adults facing rapid progression of primary immunoglobulin A nephropathy (IgAN). This medication’s potential spans a wide range of rare kidney diseases, including C3 glomerulopathy and atypical hemolytic uremic syndrome, among others.
Encouraging Developments for Scemblix
Additionally, Novartis has released promising long-term findings from the phase III ASC4FIRST study regarding its leukemia treatment, Scemblix (asciminib).
This drug showed a significant major molecular response (MMR) compared to all selected tyrosine kinase inhibitors (TKIs) and was notably 15.1% more effective than second-generation TKIs with an MMR rate of 72.0% versus 56.9% after 96 weeks.
Strategic Pipeline Expansion at NVS
Novartis remains committed to innovation, focusing on therapeutic areas such as cardiovascular health, renal treatments, immunology, neuroscience, and oncology.
During the impressive third quarter, Novartis exceeded earnings and sales estimates, prompting an increase in their guidance. The company now anticipates a compound annual growth rate (CAGR) of 6% for total sales from 2023 to 2028, up from the previous estimate of 5%.
This uptick in guidance stems from strong sales of key drugs and anticipated product launches. While organic growth fuels the business, NVS is also pursuing strategic acquisitions to enhance its pipeline.
A notable move was made with a global license and collaboration agreement with PTC Therapeutics (PTCT) for developing a Huntington’s disease candidate, which involves an upfront payment of $1.0 billion, with potential development milestones worth up to $1.9 billion.
In addition, Novartis is collaborating with Olema Pharmaceuticals, Inc. (OLMA) to provide its breast cancer drug Kisqali (ribociclib) for a phase III trial of Olema’s palazestrant in earlier stages of ER+/HER2- breast cancer.
NVS Stock Analysis & Industry Outlook
Currently, Novartis holds a Zacks Rank #3 (Hold).
A stronger alternative in the large-cap pharma sector is Pfizer (PFE), carrying a Zacks Rank #2 (Buy). Over the past two months, Pfizer’s earnings projections for 2024 rose from $2.62 to $2.91 per share, with 2025 estimates also climbing from $2.84 to $2.91. Despite their shares declining by 10% year to date, Pfizer has consistently surpassed earnings expectations.
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