FDA Formally Investigates Cancer Risk From Both Novartis SMA Gene Therapies After GLI1 Oncogene Activation Confirmed in Zolgensma Cases

Novartis' pelacarsen reduced lipoprotein(a) by 78% in a 7-year trial but failed to prevent heart attacks, strokes, or cardiovascular deaths in 8,323 adults with existing heart disease. The study, presented at the American Heart Association's Scientific Sessions, showed no significant differences in outcomes between the treatment and placebo groups. Serious adverse events were slightly higher in the pelacarsen group, with 19 cases of anaphylaxis reported.

Novartis's pelacarsen failed in Phase III trials, dimming prospects for Lp(a)-lowering drugs. Data published in NEJM and presented at AHA show no commercial potential for pelacarsen. Analysts are skeptical about other candidates, like Amgen's olpasiran, which is in Phase III trials.

Novartis and Novo Nordisk's late-stage heart drug trials failed to show benefits, according to results presented at the American Heart Association meeting. Both drugs, pelacarsen and an anti-inflammatory targeting IL-6, showed no reduction in heart attacks or strokes. The failures highlight the risks of over-relying on AI and new technologies in drug development, costing billions. Experts caution against hype surrounding AI's potential to cure diseases quickly.

Novartis confirmed that 9 tumors were reported in patients treated with its Zolgensma SMA gene therapy. Two cases showed evidence of vector integration into DNA, while the other seven were not causally linked. Over 6,000 patients have been treated with the therapy. Patient advocates raised concerns on social media, highlighting the importance of transparent communication. Regulators are assessing the evidence, according to SMA Europe.

Novartis and Ionis reported that pelacarsen failed to reduce cardiovascular events in the HORIZON trial, despite lowering lipoprotein(a) levels. Analysts see a challenging path for Lp(a)-targeting therapies, including Amgen's olpasiran and Eli Lilly's lepodisiran. Novartis' stock was impacted, and analysts lowered success probabilities for competitors' drugs. The trial results raise questions about the viability of Lp(a) targeting in cardiovascular disease.

Novartis is discontinuing an experimental heart drug after a study showed it did not reduce heart attacks or cardiovascular deaths. The drug targets lipoprotein(a), a genetic risk factor. Other companies, like Amgen and Eli Lilly, are also developing Lp(a) drugs. The findings were presented at the American Heart Association meeting.