Novartis shares fell by as much as 3.9% in early Zurich trading on September 7, following the failure of its heart drug, pelacarsen, in a late-stage trial. The drug, which aimed to reduce cardiovascular events like heart attacks and strokes in patients with elevated lipoprotein(a) (Lp(a)), did lower Lp(a) levels but did not translate into a significant reduction in these events compared to a placebo. This setback marked the biggest decline for Novartis in over four months, although its shares had gained 14% year-to-date. The news also negatively impacted Amgen, a competitor developing a similar heart drug, which saw its stock fall almost 7% in post-market trading.
The trial's outcome has led analysts to question the broader therapeutic approach for Lp(a)-lowering treatments. Michael Leuchten, an analyst at Jefferies, commented, "This questions whether any Lp(a)-lowering therapies can ultimately show a cardiovascular benefit." Despite the disappointment, some analysts suggested the negative reaction might be overstated, as even a successful trial would have posed questions regarding patient benefits and market competition. Analysts had previously forecasted pelacarsen to be a potential blockbuster, with peak sales estimates ranging from $1.5 billion (UBS) to $5.4 billion (Jefferies), and Novartis itself had projected an overall market opportunity exceeding $5 billion.
This failure puts more pressure on Novartis's pipeline, as the company relies on new medicines to sustain growth amid patent expirations of older products. CEO Vas Narasimhan had indicated that successful late-stage trials could allow the company to raise its forecast for 5% to 6% annual sales growth until 2030. The pelacarsen disappointment follows a mixed week for Novartis; while the heart drug failed, its multiple sclerosis pill, remibrutinib, had succeeded in two late-stage trials, leading to a more than 6% increase in shares on September 1. Novartis is now looking ahead to other key pipeline tests, including del-desiran, an experimental injection for the muscle-wasting disease DM1, which was central to its $12 billion acquisition of Avidity Biosciences. Another significant upcoming data release is for del-desiran for the muscular dystrophy drug, for which success is crucial to justify the $12 billion acquisition price. Furthermore, delpacibart braxlosiran (del-brax) from the same acquisition, showed promise in a mid-stage trial for facioscapulohumeral muscular dystrophy (FSHD), meeting primary and secondary endpoints and potentially becoming the first disease-modifying treatment for the condition. Del-brax is currently in Phase III development, with a randomized, double-blind, placebo-controlled study enrolling 200 patients. While del-brax for FSHD is promising, the failure of pelacarsen amplifies the importance of these other pipeline developments for Novartis's future growth.
