Late on Friday, Novartis said its drug pelacarsen did not cut heart attack and stroke risk in a large, late-stage study of patients with high Lp(a). Analysts had forecast peak annual sales between $3 billion and $6 billion if the drug proved successful.
Rival Lp(a) drugs face higher scrutiny
HORIZON's failure raises the burden of proof for rival Lp(a)-lowering drugs from Amgen and Eli Lilly. Both drugs from rivals are being tested in trials similar to pelacarsen, which are dependent on confirmed heart-related events and do not have a defined end date.
Still, some analysts noted that rival therapies could work where Novartis' failed. Amgen and Lilly's drugs have been known to lower cholesterol more effectively than pelacarsen in previous studies, and their trials are testing more targeted patients with especially high levels of Lp(a), which some analysts said could make a difference.
The Netherlands-based NewAmsterdam Pharma KH6.F, whose drug obicetrapib works mainly by lowering standard "bad" cholesterol LDL, not the harder-to-treat Lp(a) type, would not be as directly hit by the Novartis trial failure.
Biotechs CRISPR Therapeutics CRSP.BN and Silence Therapeutics are also developing treatments that target Lp(a). CRISPR's drug works by editing genes rather than blocking the protein temporarily. For Silence, which is looking for a partner to fund the drug's larger trials, the setback could make dealmaking at a desired value harder until full data is understood, William Blair analysts said.
Novartis plans to present the full results for pelacarsen at a medical meeting later this year.
Focus shifts to del-desiran and remibrutinib
The focus will now be on results from a late-stage study of its RNA therapy del-desiran in the fourth quarter. The drug, which treats a rare muscle disease, was acquired through the purchase of Avidity and Barclays analysts said success in that trial "is needed to justify" the high price tag.
Data from another study of Novartis' anti-inflammatory drug remibrutinib in patients with a skin condition called hidradenitis suppurativa is also expected this year. The drug succeeded in a trial of multiple sclerosis patients last week.
Jefferies analysts said the multiple sclerosis drug's recent success "should make the conclusion of the pelacarsen study more palatable" for investors.
As of Friday's market close, Novartis shares have gained around a fifth in value so far this year, buoyed by optimism over its pipeline. Barclays analysts said that Novartis' second drug, DII235, targeted at Lp(a) now seems "unlikely to provide any meaningful benefit."
Novartis shares fall after pelacarsen failure
Shares of Novartis NOVN.S fell more than 3% on Monday after its cholesterol drug failed in a closely watched study, dealing a blow to what investors had seen as a blockbuster treatment and raising pressure on upcoming data from a muscle-wasting therapy acquired through the Swiss drugmaker's $12 billion purchase of Avidity.
Investors had been counting on pelacarsen, anti-inflammatory drug remibrutinib and RNA therapy del-desiran to drive growth as Novartis prepares for patent expiries on older blockbusters, including heart drug Entresto.
The setback also casts uncertainty over the wider industry effort to target lipoprotein(a), or Lp(a), an inherited cardiovascular risk factor with no approved treatments.
Amgen AMGN.O and Eli Lilly LLY.N are running late-stage trials of rival experimental medicines, olpasiran and lepodisiran.
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