On Tuesday, Novartis AG (NYSE:NVS) stock witnessed one of the sharpest single-day declines for the company in recent history.
Phase 3 HARBOR Trial Misses Primary Endpoint
On Tuesday, the company shared data from the global Phase 3 HARBOR study evaluating del-desiran for myotonic dystrophy type 1 (DM1).
The study did not demonstrate statistically significant improvement versus placebo on the primary endpoint of video hand opening time (vHOT), a novel measure of hand myotonia.
Myotonia is a neuromuscular condition where muscles are unable to relax right away after a voluntary contraction or strong effort.
Safety findings from HARBOR were generally consistent with previously reported data.
Novartis is evaluating the full HARBOR dataset and will engage with health authorities to determine the most appropriate development path for del-desiran.
Del-desiran is one of three antibody-oligonucleotide conjugate (AOC) therapies added to the Novartis neuromuscular pipeline through the acquisition of Avidity Biosciences for a whopping $12 billion.
Status of $12 Billion Avidity Pipeline Acquisition
Novartis is advancing delpacibart zotadirsen (del-zota) in patients with Duchenne muscular dystrophy with mutations amenable to exon 44 skipping (DMD44).
The company filed del-zota for accelerated approval and received FDA priority review designation.
Novartis is planning to meet with the FDA on next steps for delpacibart braxlosiran (del-brax) in facioscapulohumeral muscular dystrophy (FSHD) based on recent positive Phase 1/2 biomarker data.
Pelacarsen Cardiovascular Trial Fails
On Friday, Novartis also shared another trial disappointment after it released data from the pelacarsen phase 3 Lp(a)HORIZON trial, a cardiovascular outcomes study.
The study did not meet its primary endpoint of reducing the risk of cardiovascular events, a composite of cardiovascular death, non-fatal myocardial infarction, non-fatal stroke, and urgent coronary revascularization requiring hospitalization, compared to placebo.
Lower lipoprotein (a) (Lp(a)) levels were achieved with pelacarsen in this study population, which was receiving guideline-directed treatments, including lipid-lowering and antihypertensive therapies.
Elevated Lp(a) is an inherited cardiovascular risk factor affecting approximately one in five people worldwide with no approved targeted treatment.
Ripple Effect Hits Partners
After the update, Ionis Pharmaceuticals Inc. (NASDAQ:IONS) stock also tanked as Novartis obtained global rights to develop, manufacture, and commercialize pelacarsen under a 2019 license and collaboration agreement.
In 2023, Royalty Pharma plc (NASDAQ:RPRX) acquired an interest in Ionis’ royalty in Biogen’s SPINRAZA (nusinersen) and Novartis’ pelacarsen for up to $1.125 billion, including an upfront payment of $500 million and up to $625 million in additional pelacarsen milestone payments.
After the disappointing trial data, Royalty Pharma stock is also trading lower on Tuesday.
NVS/IONS/RPRX Stock Price Activity: Novartis shares were down 12.57% at $139.88, Ionis Pharmaceuticals shares were down 9.80% at $52.40, and Royalty Pharma shares were down 6.29% at $59.94 during premarket trading Tuesday, according to Benzinga Pro data.
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