Novartis Reports Positive Data for Neuromuscular Disorder Drug

Novartis NVS announced positive data from the biomarker cohort of the FORTITUDE phase I/II study.  The FORTITUDE phase I/II study is a randomized, double-blind, placebo-controlled study evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics and exploratory efficacy of delpacibart braxlosiran (del-brax) in patients with facioscapulohumeral muscular dystrophy (FSHD).Del-brax is an investigational antibody oligonucleotide conjugate (AOC) therapy with the potential to become the first disease-modifying treatment for FSHD, a rare progressive neuromuscular disorder that leads to irreversible muscle weakness and disability.Cohort C, a biomarker cohort, evaluated del-brax at a dose of 2 mg/kg administered every six weeks versus placebo over 12 months in 51 patients aged 16-70 with FSHD.  The primary endpoint of the study cohort was the change in plasma concentration of KHDC1L, a DUX4-regulated circulating biomarker. The key secondary endpoint was the change from baseline in the levels of creatine kinase, a marker of muscle damage.The stock is up in pre-market trading. Year to date, shares of Novartis have gained 7.5% compared to the industry’s decline of 4.5%.Image Source: Zacks Investment ResearchMore on NVS’ Data From FORTITUDE StudyThe study met its primary and key secondary endpoints, demonstrating reductions in KHDC1L (cDUX) and creatine kinase biomarker levels, indicating both strong target engagement and reduction in muscle damage in FSHD patients.The safety profile is consistent with the previous results.The results support the dosing regimen being used in the ongoing phase III trial.Del-brax is the only investigational therapy to demonstrate disease-modifying potential in FSHD and is currently being evaluated in a phase III study. The candidate has received Orphan Drug and Fast Track designations from the FDA as well as Orphan Drug designation from the European Medicines Agency.Novartis is reviewing the full biomarker and clinical dataset and plans to engage with global regulators for the same.The candidate was added to Novartis' pipeline through its 2026 acquisition of Avidity Biosciences and is one of three late-stage disease-modifying AOC therapies acquired.In addition to del-brax, the Avidity acquisition added phase III candidate delpacibart-etedesiran (del-desiran) for myotonic dystrophy type 1 and phase II candidate delpacibart-zotadirsen (del-zota) for Duchenne muscular dystrophy. Together, these programs expand Novartis’ neuromuscular portfolio and strengthen its leadership in rare neurological diseases.NVS’ neuromuscular portfolio include Zolgensma (onasemnogene abeparvovec)/Itvisma (onasemnogene abeparvovec-brve), a one-time gene therapy designed to address the genetic root cause of spinal muscular atrophy (SMA) by replacing the function of the missing or nonworking SMN1 gene.NVS Looks to Strengthen Pipeline/Portfolio2026 is a pivotal year for Novartis as it navigates the largest patent expiry in its history.Novartis is now banking on key growth drivers — Kisqali, Kesimpta, Pluvicto, Scemblix and Leqvio — to support top-line growth.NVS is also focusing on strategic acquisitions to strengthen its pipeline.The company is all set to acquire SNV4818 from Synnovation Therapeutics, a pan-mutant selective PI3Kα inhibitor currently in phase I/II for HR+/HER2- breast cancer and other advanced solid tumors. The program aligns with Novartis’ breast cancer strategy and could be combined with CDK inhibitors and endocrine therapies. The deal is expected to be closed in the first half of 2026, subject to customary conditions.In addition, Novartis agreed to acquire Excellergy, including Exl-111, a phase I anti-IgE antibody with extended half-life and high affinity. Its differentiated mechanism may enable faster and deeper receptor downregulation, potentially improving symptom control and dosing convenience in allergic diseases. The transaction is expected to be closed in the second half of 2026, subject to customary conditions.NVS’ Zacks Rank & Stocks to ConsiderNovartis currently carries a Zacks Rank #3 (Hold). A couple of better-ranked large cap pharma stocks are Bayer AG BAYRY and Roche RHHBY, both carrying a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.Shares of Bayer have gained 26.6% in the past year. Estimates for BAYRY’s 2026 earnings per share (EPS) have increased from $1.20 to $1.25 over the past 60 days.Roche’s EPS estimates for 2026 have increased from $3.27 to $3.30 over the past 60 days, while that for 2027 has increased from $3.45 to $3.51 over the same time frame. Shares of RHHBY have gained 20.2% over the past year.  7 Best Stocks for the Next 30 DaysJust released: Experts distill 7 elite stocks from the current list of 220 Zacks Rank #1 Strong Buys. They deem these tickers "Most Likely for Early Price Pops."Since 1988, the full list has beaten the market more than 2X over with an average gain of +23.7% per year. So be sure to give these hand picked 7 your immediate attention. See them now >>This article originally published on Zacks Investment Research (zacks.com).Zacks Investment ResearchWeiter zum vollständigen Artikel bei Zacks Weiter zum vollständigen Artikel bei Zacks