Clinical Trial News

Rhythm Pharmaceuticals announces FDA approval of Imcivree for children aged 2 and above, targeting syndromic or monogenic obesity, including Bardet-Biedl syndrome and POMC/PCSK1/LEPR deficiencies. Imcivree is the first precision medicine for the MC4R pathway, showing significant weight and hunger reduction in trials.

FDA halts RSV vaccine trials involving infants under 2 years and RSV-naive children aged 2-5 years due to increased severe illness reported in a trial.

FDA approves Lilly's Zepbound for moderate-to-severe obstructive sleep apnoea in obese adults, the first prescription medication for the condition. Zepbound, used with diet and exercise, significantly reduces breathing disruptions and aids long-term weight loss, with 42-50% of patients achieving remission or mild OSA.

This study, part of the Precision Diets for Diabetes Prevention trial, recruited 36 participants aged 30-70 with BMI 23-40 kg/m². 32 completed metabolic tests, forming the main cohort. An independent validation cohort of 24 and a combined CGM cohort of 29 were also used. Metabolic tests included OGTT, at-home OGTT with CGM, IIGI, insulin suppression test, and plasma glucose measurement. Calculations assessed muscle insulin resistance, β-cell function, incretin effect, and hepatic insulin resistance. A machine-learning framework identified metabolic subphenotypes from OGTT glucose time series, validated on an independent cohort.

2024 saw significant medical breakthroughs, including gene therapy restoring hearing in children with hereditary deafness, the first genetically-edited pig kidney transplant into a human, discovery of a cause of lupus, FDA approval of the first new class of schizophrenia drug in over 30 years, and authorization of the first over-the-counter combo flu and COVID test outside of emergency use.

The REMED trial was a multicenter, randomized, open-label study in Czech Republic evaluating dexamethasone in COVID-19 ARDS patients. It included 222 patients, randomized 1:1 to receive high-dose (20 mg/day for 5 days, then 10 mg/day for 5 days) or standard-dose (6 mg/day for 10 days) dexamethasone. Primary outcome was ventilator-free days at day 28. Recruitment ended early due to futility, with 69.6% power to detect a 3 VFD difference.

Sanofi and SK bioscience expand collaboration to develop next-gen PCVs, including PCV21, the first 21-valent vaccine in phase 3 for infants & toddlers, aiming to address unmet needs in pneumococcal disease.

Ionis Pharmaceuticals announced FDA approval of Tryngolza (olezarsen), the first treatment for familial chylomicronemia syndrome (FCS), reducing triglycerides and acute pancreatitis risk. Tryngolza, self-administered monthly, showed significant reductions in triglyceride levels and AP events in a Phase 3 study, with a favorable safety profile. This marks a pivotal moment for Ionis, transitioning to a commercial-stage biotech company.

Merck announced positive phase 3 trial results for doravirine/islatravir, a once-daily, oral, two-drug regimen for HIV-1, showing non-inferiority to current therapies. The company plans to present findings and file with regulators, continuing its commitment to advancing HIV treatment options.

Bavdegalutamide (ARV-110), a PROTAC protein degrader, selectively degrades wild-type and mutant AR with high potency, showing superior activity over enzalutamide in cell-based systems and AR-expressing xenograft models. It effectively inhibits tumor growth in enzalutamide- and abiraterone-resistant models and enhances abiraterone activity. Bavdegalutamide is the first PROTAC degrader in clinical trials for mCRPC.