Regeneron Pharmaceuticals reported positive topline results from the Phase 3 NIMBLE trial evaluating investigational cemdisiran, an siRNA targeting complement factor 5 (C5), as monotherapy for adults with generalized myasthenia gravis (gMG). The trial, involving patients with anti-acetylcholine receptor antibodies, randomized participants to receive cemdisiran (600 mg subcutaneously every 12 weeks), a cemdisiran-pozelimab combination (200 mg each every 4 weeks), or placebo.
Ionis Pharmaceuticals, Inc. received FDA approval on August 21, 2025, for DAWNZERA™ (donidalorsen), the first RNA-targeted therapy for prophylaxis against hereditary angioedema (HAE) attacks in adults and pediatric patients aged 12 and older. HAE, a rare genetic condition affecting approximately 7,000 Americans, causes recurrent, potentially life-threatening swelling in various body parts. DAWNZERA targets plasma prekallikrein (PKK), a key protein in HAE attack pathways, and is administered via subcutaneous autoinjector every four (Q4W) or eight weeks (Q8W), offering the longest dosing interval among HAE prophylactics. This approval marks Ionis’ second independent product launch within nine months, following TRYNGOLZA® for familial chylomicronemia syndrome.
Metabolic dysfunction-associated steatotic liver disease (MASLD) is a leading cause of chronic liver disease, particularly in patients with type 2 diabetes (T2D). The IMAGIN Study, a single-center prospective observational trial, investigated the impact of empagliflozin as an add-on to metformin versus metformin monotherapy on MASLD progression in SGLT2 inhibitor-naïve T2D patients with an estimated glomerular filtration rate (eGFR) ≥45 mL/min/1.73 m². The study aimed to assess changes in liver steatosis, fibrosis, and metabolic parameters, alongside exploring potential microRNA (miRNA) biomarkers for treatment response.
Heart failure with improved ejection fraction (HFimpEF) represents an understudied subgroup of heart failure patients who, despite LVEF improvement, face residual risks comparable to those with consistently higher LVEF (>40%). The prognostic implications of the degree of LVEF improvement and its impact on treatment response remain poorly understood. The DELIVER trial (NCT03619213) addresses this gap by evaluating dapagliflozin, a sodium-glucose cotransporter 2 inhibitor, in patients with heart failure and LVEF >40%, including those with HFimpEF. This study explores whether the extent of LVEF improvement influences clinical outcomes and the therapeutic benefits of dapagliflozin.
A randomized, double-blind, placebo-controlled trial, published in JAMA, evaluated metformin’s efficacy for knee osteoarthritis (OA) in 108 overweight or obese adults (BMI ≥25 kg/m², mean age ~60 years) with symptomatic knee OA (Kellgren-Lawrence grade 2-3). Conducted over 18 months, patients received metformin (up to 2g daily) or placebo alongside standard care. Primary outcomes were changes in knee pain (WOMAC pain score, 0-20 scale) and cartilage volume loss (via MRI). Secondary outcomes included physical function (WOMAC function score), weight loss, and safety.
