The Phase 3 failure of Novartis's cardiovascular drug Pella Carson triggered a massive sell-off not just for Novartis, but also for competitors Amgen and Eli Lilly. This indicates investor fear that the entire LP(a) drug target class is flawed, rather than it being an issue with a single company's molecule.
Novartis's LP(a) drug failed in patients with established cardiovascular disease, suggesting that the underlying vascular damage from a lifetime of exposure may be irreversible. This implies effective treatment might require intervention decades earlier, a timeline that poses significant, perhaps insurmountable, challenges for clinical trials and regulatory approval.
If Amgen's siRNA drug succeeds where Novartis's antisense oligo (ASO) failed for the same LP(a) target, it could be a decisive blow to ASO technology. This would bolster the view that siRNA's superior target knockdown and less frequent dosing make it the preferred modality for large-population diseases, relegating ASOs to niche orphan indications.
While its massive Phase 3 trial for an antisense drug was ongoing, Novartis licensed an siRNA targeting the same LP(a) pathway from a Chinese company. This proactive move suggests Novartis was either building a next-generation follow-on or, more likely, hedging its bets due to a lack of internal confidence in its lead asset's modality.
Big Pharma is increasingly licensing assets from Chinese biotechs, putting the region on par with Europe as a source of innovation. However, outright M&A of Chinese firms is a complete non-starter. This reflects a bifurcated strategy: pharma will partner for assets but avoids the geopolitical and logistical headaches of acquisitions, creating a 'license-only' pipeline from China.
Companies like Bridge Bio are publicly agreeing to the White House's 'Most Favored Nation' pricing while privately securing critical carve-outs. Bridge Bio obtained a forward-looking exclusion for orphan drugs, which covers its entire pipeline. This suggests companies are trading symbolic compliance for political goodwill while ensuring the deals have minimal financial impact.