Therapeutic Areas

Data from the RAINBOWFISH trial shows early treatment with Evrysdi (risdiplam) in pre-symptomatic infants with spinal muscular atrophy (SMA) leads to significant motor milestone achievements, such as standing and walking, as well as cognitive development typical of non-SMA children, without the need for permanent ventilation.

Tremfya (guselkumab) demonstrated superior endoscopic remission rates in both Crohn disease and ulcerative colitis, including biologic-naïve and biologic-refractory patients, according to Phase III clinical trial data.

Twice-yearly lenacapavir, a long-acting HIV-1 capsid inhibitor, reduced HIV infections by 96% in the Phase III PURPOSE 2 trial while showing superior efficacy over daily Truvada.

Findings from the BATURA Phase IIIb trial demonstrated that Airsupra (albuterol/budesonide) significantly reduced the risk of severe asthma exacerbations in patients with intermittent or mild persistent asthma compared to albuterol alone.

In the Phase II PATH-HHT trial, pomalidomide significantly reduced the severity of epistaxis and improved quality of life in patients with hereditary hemorrhagic telangiectasia, offering a potential treatment for the bleeding disorder, which currently lacks FDA-approved therapies.

Ziresovir is a potent, selective, orally bioavailable RSV F protein inhibitor, that has been shown to reduce viral load and the Wang Respiratory Score with a favorable safety profile in hospitalized infant patients with respiratory syncytial virus infection.

Proof-of-concept Phase II ARTEMIS-UC trial shows efficacy of investigational humanized monoclonal antibody tulisokibart in treating inflammatory bowel disease compared with placebo.

Webinar Date/Time: Tuesday, November 12th, 2024 at 10am EST | 7am PST | 3pm GMT | 4pm CET

Phase III REGENCY trial results highlight potential of Gazyva/Gazyvaro (obinutuzumab) to improve outcomes in lupus nephritis patients, reducing progression to end-stage kidney disease.

Ebglyss was approved by the FDA earlier this month to treat moderate-to-severe atopic dermatitis in patients aged 12 years and older who weigh at least 88 lbs.

Niktimvo (axatilimab) gained FDA approval in August 2024 to treat graft-versus-host-disease in patients who did not achieve a response to at least two prior lines of systemic therapy.

Kesimpta (ofatumumab) demonstrates efficacy lowering disability progression and disease activity in different populations of patients with relapsing multiple sclerosis.

Study finds nearly 75% of patients with heterozygous familial hypercholesterolemia achieved a ≥50% drop in serum LDL-C concentration from baseline to about 12 months of therapy with a PCSK9 inhibitor.

Lu AG09222 shows promise as first-in-class medication that prevents neurogenic inflammation, vasodilation, and parasympathetic lacrimation, which are considered to be surrogate markers of migraine attacks.

Edoxaban monotherapy lowered the risk of adverse outcomes, including death from any cause, myocardial infarction, stroke, and systemic embolism at 12 months compared to the dual antithrombotic therapy approach in patients with atrial fibrillation and stable coronary artery disease.

The first-in-class, investigational RNA interference drug plozasiran was found to lower triglyceride levels and incidence of pancreatitis compared to placebo in patients with persistent chylomicronemia, including patients with familial chylomicronemia syndrome.

Crinecerfont was granted previously granted FDA Priority Review designation to treat children, adolescents, and adults with classic congenital adrenal hyperplasia.

Investigators conclude that policies seeking to lower rates of diabetes may also contribute to the goal of ending tuberculosis.

Elinzanetant was found to significantly lower the frequency and severity of moderate to severe vasomotor symptoms with statistically significant improvements in sleep disturbances and menopause-related quality of life.

Mild or moderate acute kidney injury has been linked to increased morbidity and mortality, with a greater risk of chronic kidney disease in patients who undergo cardiac surgery with cardiopulmonary bypass.

Phase III REST-ON trial data show higher rates of weight loss in patients administered Lumryz to treat both narcolepsy type 1 (with cataplexy) and narcolepsy type 2 (without cataplexy) compared with placebo.

Patients administered tirzepatide (Zepbound and Mounjaro; Eli Lilly) also achieved sustained weight loss in Phase III trial, with individuals administered the 15 mg dose showing a 22.9% average reduction in body weight compared with 2.1% among those receiving placebo.

Results show nipocalimab could address the high unmet need of patients with severe hemolytic disease of the fetus and newborn, which is a life-threatening and rare condition with no other therapies currently in clinical development.

Phase IV study finds antihypertensive therapy guided by ambulatory blood pressure monitoring improves target organ damage in patients with masked hypertension.

Webinar Date/Time: Tuesday, August 27, 2024 at 11:00 AM EDT | 8:00 AM PDT | 4:00 PM BST | 5:00 PM CEST

Randomized trial finds once daily oral semaglutide 50 mg lowered energy intake, appetite, and food cravings, with improved control of eating and clinically meaningful reductions in body weight after 20 weeks.

Phase IIb trial indicates liraglutide (Novo Nordisk) may reduce cognitive decline associated with Alzheimer disease by protecting against shrinkage in areas of the brain controlling memory, learning, language, and decision-making.

Randomized trial shows Farxiga improved right ventricular pulsatile afterload and myocardial performance during exercise in patients with heart failure with preserved ejection fraction.

Results from an induction trial and maintenance trial show Skyrizi (risankizumab) improved endoscopic and histological secondary outcomes characterized by endoscopic improvement, remission, and histological, endoscopic, and mucosal improvements in patients with moderately to severely active ulcerative colitis.

Antibody-mediated rejection is the primary cause of kidney allograft failure following kidney transplantation.