last month, Novartis (NVS -3.20%) has revealed promising results from a Phase 3 clinical trial of an orphan drug candidate known as iptacopan for adult patients diagnosed with a blood disorder known as paroxysmal nocturnal hemoglobinuria (PNH).
What do these results mean for PNH patients and the future of Novartis? To find out, let’s dig into data from clinical trials and the global PNH market.
A treatment that can change your life
PNH is a rare and serious blood disorder estimated to affect 10-20 per million people worldwide. The disease causes the breakdown of red blood cells (RBCs) and can lead to symptoms such as muscle cramps, fatigue, shortness of breath and unintended weight loss.
As with other medical conditions, the severity of PNH varies. Worst-case scenarios can include potentially fatal blood clots. For this reason, it is of utmost importance to treat the condition as early as possible for optimal health.
The most common treatment for PNH is a class of drugs called anti-C5 antibodies, including: AstraZenecaof (AZN -0.96%) Soliris and Ultomiris. These treatments have produced dramatically better results than previous treatments. ) and are dependent on blood transfusions as part of their treatment.
As such, iptacopan may be the next step for patients who do not have optimal results with anti-C5 antibodies. Novartis iptacopan helped his 96.8% (60 of 62) of patients remain transfusion-free after her 6 months of treatment. This far exceeds 40% (14 of 35) of patients on anti-C5 therapy who did not need a blood transfusion during her 6 months of treatment.
The safety profile of iptacopan was as robust as the anti-C5 antibody. No patient discontinued iptacopan or anti-C5 antibody therapy due to adverse events during the clinical trial.
breakthrough sales potential
Iptacopan has the potential to dramatically change the prognosis of many PNH patients. As more treatments like iptacopan eventually hit the market, market research firm ReportLinker predicts that the global PNH treatment market will grow from $3 billion in 2020 to $3 billion by 2027. is expected to grow to $5.7 billion.
Anti-C5 therapy has been incorporated as first-line therapy. However, iptacopan’s efficacy could replace anti-C5 therapy, so I believe this drug could represent 30% of the $3.8 billion market. This will increase Novartis annual sales by $1.1 billion.
That alone is impressive enough when compared to the $52.2 billion in total revenue analysts expect from the company in 2023. Novartis also expects the drug to generate peak annual sales of $3 billion, along with other rare disease indications in clinical trials among a total of 149 projects in its pipeline. .
great business for sale now
Novartis is a fundamentally strong pharmaceutical company. Analysts believe the company will achieve annual earnings growth of 4.2% over the next five years, which may be low given its deep drug pipeline.
Still, the 14.2 price/earnings ratio is just below the pharmaceutical industry average of 15. For a world-class company like Novartis, I think discounts relative to peers are a buying opportunity.
Cody Kester does not have a position in any of the stocks mentioned.The Motley Fool does not have a position in any of the stocks mentioned. The Motley Fool’s U.S. headquarters has a disclosure policy.