Mirikizumab Benefit in UC Extends Into Maintenance Phase
The investigational monoclonal antibody mirikizumab was more effective in inducing and maintaining clinical remission in patients with moderate to severe ulcerative colitis (UC) compared with placebo, according to two phase III randomized trials. Among over 1,200 patients in the LUCENT-1 induction trial, 24.2% of those who received mirikizumab experienced clinical remission at week 12 compared […]
Novel Triple Agonist Nabs Biggest Weight Loss Yet in Obesity Trial
SAN DIEGO — An investigational triple-hormone receptor agonist yielded major weight loss in people with obesity, a phase II trial showed. By 24 weeks, patients on any of the four doses of once-weekly injectable retatrutide lost a significantly greater amount of weight than those on placebo — achieving the trial’s primary endpoint — reported Ania […]
Once-Weekly Insulin Icodec Bests Daily Insulin Options for Type 2 Diabetes
SAN DIEGO — Investigational once-weekly insulin icodec outperformed once-daily insulin glargine U100 when it came to glycemic control in type 2 diabetes, the phase IIIa ONWARDS 1 trial found. Over the course of 52 weeks, people with type 2 diabetes saw a greater average reduction in HbA1c with icodec compared with glargine U100 (estimated between-group […]
Near 15% Weight Loss With Novel Oral GLP-1 RA in Phase II Obesity Trial
SAN DIEGO — Orforglipron, an investigational nonpeptide GLP-1 receptor agonist, helped people with obesity lose a significant amount of weight in a phase II trial. Tested at four once-daily doses — 12, 24, 36, or 45 mg — patients on orforglipron lost between 8.6% to 12.6% of body weight compared with a 2% change seen […]
‘Game Changer’ in NASH: Resmetirom Hits Endpoints in Pivotal Study
VIENNA — The investigational agent resmetirom resolved liver biopsy findings in nonalcoholic steatohepatitis (NASH) more often than placebo, according to the phase III MAESTRO-NASH trial. While 10% of placebo patients achieved resolution of inflammation, ballooning, and disease activity, the rate was 26% among people treated with the 80-mg dose of resmetirom and 30% with the […]
Duchenne Muscular Dystrophy Treatment Narrowly Captures Support of FDA Advisors
FDA advisors narrowly supported accelerated approval for Sarepta Therapeutics’ investigational gene therapy for Duchenne muscular dystrophy (DMD) on Friday. In a 8-to-6 vote, the agency’s Cellular, Tissue, and Gene Therapies Advisory Committee said the overall benefits and risks supported SRP-9001 (delandistrogene moxeparvovec) for ambulatory Duchenne patients, using expression of Sarepta’s micro-dystrophin as a surrogate endpoint. […]
Sarepta’s Duchenne Gene Therapy Does Not Have ‘Unambiguous Evidence,’ FDA Staff Says
Clinical studies to date “do not provide unambiguous evidence” for SRP-9001 (delandistrogene moxeparvovec), an investigational gene therapy for ambulatory Duchenne muscular dystrophy patients, FDA reviewers said in briefing documents released ahead of an advisory committee meeting. The agency also raised safety concerns about the possibility of administering an ineffective gene therapy. Duchenne muscular dystrophy is […]
Novel Chemo Delivery System Achieves High Response Rate in Bladder Cancer
CHICAGO — An investigational intravesical delivery system designed to provide sustained local release of gemcitabine into the bladder produced high rates of complete response (CR) in difficult-to-treat non-muscle-invasive bladder cancer (NMIBC), data from an ongoing phase IIb study showed. Preliminary results from the SunRISe-1 trial demonstrated that monotherapy with the novel treatment, dubbed TAR-200, led […]
Perioperative Toripalimab Prolongs EFS in Stage III Lung Cancer
Adding an investigational anti-PD-1 drug to perioperative chemotherapy improved event-free survival (EFS) in patients with resectable, stage III non-small cell lung cancer (NSCLC), an interim analysis of a phase III study found. Among 404 patients receiving perioperative chemotherapy in the so-called Neotorch trial, the median investigator-assessed EFS was not reached in patients assigned to toripalimab, […]
Robust PSA Drops With Radionuclide Therapy in Castration-Resistant Prostate Cancer
ORLANDO — An investigational radionuclide therapy targeting prostate-specific membrane antigen (PSMA) achieved biochemical responses in 70% of patients with castration-resistant prostate cancer (CRPC), and prostate-specific antigen (PSA) reductions in all but one patient, enrolled in a small preliminary clinical trial. Overall, 22 of 23 patients had some degree of PSA decline in response to fractionated […]