DII235

During its Q1 2026 earnings conference call, held April 28, 2026, Novartis framed remibrutinib’s summer MS readouts as the most important near-term swing factor for potential upside to its 2030 growth outlook, while using Q1 to defend Pluvicto’s peak-sales case after withdrawing a European pre-taxane filing and to temper expectations around unresolved accelerated-approval discussions for votoplam. Remibrutinib (Rhapsido) — MS and hidradenitis suppurativa (HS) as franchise-defining catalysts: CEO Vasant Narasimhan said a superior-to-anti-CD20 result in the two replicate Phase III relapsing MS trials, expected this summer, would represent a “massive” opportunity, while a non-inferior result would still position HS as a comparably sized indication. The HS Phase III program enrolled ahead of schedule and was accelerated into H2 2026, with management describing an oral-first positioning strategy ahead of biologics if data support it. Avidity-derived neuromuscular assets — first-to-market framing across DM1 and FSHD: Following the close of the Avidity acquisition, Novartis said del-desiran’s Phase III HARBOR study in myotonic dystrophy type 1 (DM1) is on track for an H2 2026 readout, and that del-zota for Duchenne muscular dystrophy (DMD) exon 44 skipping is tracking toward an NDA submission in H1 2026 pending chemistry, manufacturing, and controls (CMC) resolution. Management explicitly framed both assets as first-to-market opportunities and said the Antibody Oligonucleotide (AOC) modality acquired through Avidity would be applied across internal pipeline targets beyond the neuromuscular franchise. Pluvicto — EMA pre-taxane filing withdrawn, European path rerouted: Novartis withdrew its European pre-taxane metastatic castration-resistant prostate cancer (mCRPC) submission after failing to resolve the European Medicines Agency’s (EMA) objections to the comparator arm design, and said it would now wait for overall survival data from the hormone-sensitive prostate cancer (HSPC) setting before pursuing a new European filing. Management said the withdrawal had no impact on the $5 billion-plus peak sales forecast, noting that Japan and China retained access to both the PSMAfore and PSMA VISION populations. Remibrutinib HS efficacy bar — Phase II data difficult to interpret: Analyst Sachin Jain of Bank of America pressed management on the target efficacy profile for remibrutinib in HS relative to existing biologics, noting that the Phase II data featured an inverse dose response and low placebo-adjusted response rates. Narasimhan acknowledged the heterogeneity of the HS population and said the positioning relative to biologics would depend on the Phase III data profile, declining to specify a target efficacy threshold. Votoplam — Phase III dose confirmed but FDA engagement unresolved: Analyst Thibault Boutherin of Morgan Stanley asked whether new data from the open-label extension had increased confidence in an accelerated approval pathway. Management confirmed the 10-milligram dose for the pivotal Phase III study based on in-house data but said discussions with partner PTC Therapeutics on the optimal FDA interaction strategy, including the potential for accelerated approval, remained incomplete, with no further timeline provided. Summer 2026 — Remibrutinib Phase III MS dual readout: The two replicate Phase III studies in relapsing MS are expected to read out this summer. Management confirmed blinded safety data show no drug-induced liver injury or alanine transaminase (ALT) elevations attributable to remibrutinib, but said no visibility into annualized relapse rate data was available ahead of the readout. Narasimhan said a positive MS outcome was the single largest potential driver of upside to the company’s guided 5% to 6% growth outlook to 2030. Early H2 2026 — Pelacarsen HORIZON cardiovascular outcomes readout: The HORIZON study is powered for a 20% relative risk reduction in patients with lipoprotein(a) [Lp(a)] of 70 milligrams per deciliter or higher, and a 25% relative risk reduction in the 90-milligram-per-deciliter subgroup, with a median enrolled Lp(a) level of 108 milligrams per deciliter. Management said the follow-on once-yearly siRNA asset DII-235 is already Phase III-ready, signaling a deliberate two-asset Lp(a) franchise build regardless of the primary trial outcome, and noted that the American College of Cardiology and American Heart Association have now recommended universal Lp(a) testing once per lifetime in the United States. This article was generated with AI assistance and reviewed and edited by the AllSci editorial team Explore more at AllSci News: https://allsci.com/news/ Your email address will not be published. Required fields are marked * Comment * Name * Email * Website