Novartis Lp(a) Drug Fails Phase 3 Trial, Shaking Cardiovascular Market
Novartis announced that its experimental drug pelacarsen failed to reduce major cardiovascular events in a large Phase 3 trial, despite successfully lowering Lp(a) levels. The setback impacts the broader Lp(a) drug market, though competitors like Amgen and Eli Lilly continue their own trials.
Key points
- Novartis reported that pelacarsen, an antisense oligonucleotide, did not meet its primary endpoint in the Lp(a)HORIZON trial, which enrolled 8,323 patients with elevated Lp(a) and established cardiovascular disease.
- While the drug lowered Lp(a) levels by approximately 72%, it did not translate into a reduction in cardiovascular death, non-fatal heart attack, stroke, or urgent coronary revascularization.
- The failure has shocked cardiologists and analysts, but experts suggest the mechanism may not be dead, as competitors Amgen and Eli Lilly are developing RNA interference therapies that lower Lp(a) by more than 90%.
- Novartis plans to present full trial data at an upcoming medical meeting, noting that the results provide evidence on the relationship between Lp(a) lowering and cardiovascular outcomes.
- Stocks for Novartis, Ionis, and Amgen dropped after the announcement, reflecting market concerns about the Lp(a) drug pipeline.
Background
Lp(a) is a genetic risk factor affecting about 20% of the global population, with no approved targeted treatments. Previous setbacks in cardiovascular drug trials, such as Novo Nordisk's failed inflammation drug, have raised questions about the efficacy of new approaches. The Lp(a) drug race was seen as a potential multi-billion market, with Novartis leading the field before this setback.
How outlets are covering it
Fierce Biotech and Novartis emphasize that while pelacarsen failed, the results provide valuable scientific understanding and may inform future approaches. The New York Times highlights the shock to the cardiology field, with Dr. David Maron calling it a 'huge blow.' Yahoo Finance notes the market reaction, with stocks for Novartis, Ionis, and Amgen dropping. Analysts from William Blair and Citi suggest that deeper Lp(a) inhibition, as seen in Amgen's and Eli Lilly's drugs, might be necessary to see cardiovascular benefits, and that the mechanism is not necessarily dead.
Why it matters
The failure of pelacarsen, a leading candidate in the Lp(a) drug market, raises questions about the efficacy of current approaches to lowering Lp(a) for cardiovascular risk reduction. However, it may also push competitors to develop more potent therapies, potentially accelerating the development of new treatments for a significant portion of the global population.
What to watch
Novartis will present full trial data at an upcoming medical meeting. Amgen and Eli Lilly are expected to continue their own trials, with their RNA interference therapies showing deeper Lp(a) reduction. The market will watch for any further developments in the Lp(a) drug pipeline and the potential impact on cardiovascular risk management.
- Novartis Lp(a) drug fails to lower cardiovascular risk in closely watched ph. 3 trial Fierce Biotech
- The Heart Drug Pelacarsen Fails, Shocking Novartis and Cardiologists The New York Times
- Novartis Says Closely Watched Cholesterol Study Failed to Meet Goal WSJ
- NVS, IONS, AMGN Stocks Drop After-Hours — What’s Driving The Selloff? Yahoo Finance
- Novartis announces Lp(a)HORIZON Phase III topline results for pelacarsen in patients with elevated Lp(a) and established cardiovascular disease (CVD) Novartis
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