Clinical Trial News

FDA advisors will assess obeticholic acid's (Ocaliva) clinical benefit in PBC, despite null post-approval evidence. Intercept Pharmaceuticals seeks an indication for reducing hepatic decompensation, liver transplant, and death in PBC patients with compensated cirrhosis, using the drug with or without UDCA. FDA reviewers doubt the drug's efficacy for PBC patients intolerant or unresponsive to UDCA, citing failed postmarketing trials. The advisory committee will vote on whether the drug's benefits outweigh risks in non-contraindicated PBC patients.

Sally Lau, MD, discusses her role in the DeLLphi-304 trial, focusing on the accelerated approval of Imdelltra for relapsed small-cell lung cancer. She highlights the importance of nurse navigators and diversity grants in supporting patient enrollment and retention in trials, emphasizing the need for early involvement and responsive sponsors. Lau's passion for clinical trials stems from witnessing direct patient benefits and innovative treatment approaches.

The FDA granted 510(k) clearance to the Sleep Rx Mat, an under-mattress device aiding in OSA diagnosis by tracking respiratory rate, body movement, heart rate, sleep cycles, and interruptions. It provides a sleep quality score and requires a prescription.

Medidata and EORTC extend partnership for four years to enhance patient access and experience in cancer trials, leveraging 13 Medidata solutions for seamless data management and patient engagement, aiming to deliver new treatments faster and explore AI-driven research collaborations.

Bria-IMT (SV-BR-1-GM) combined with retifanlimab-dlwr or pembrolizumab showed improved overall survival (OS) and progression-free survival (PFS) in heavily pretreated metastatic breast cancer patients, with median OS of 15.6 months and PFS of 4.1 months in the 2022 cohort, and 13.4 months OS and 3.9 months PFS since study onset. These results support Bria-IMT's potential as a new standard of care.

Obesity, linked to 5 million deaths globally, increases risks of T2DM, heart disease, and cancers. GLP1R agonists like semaglutide (WEGOVY) emerged as effective treatments, with tirzepatide (ZEPBOUND) and setmelanotide (IMCIVREE) also approved. The article updates on current obesity treatments and ongoing clinical trials.

The TGA approved tirzepatide (Mounjaro) for weight management in adults with BMI ≥30 kg/m2 or ≥27 kg/m2 plus comorbidities. Mounjaro, already used for type 2 diabetes, now supports weight loss and maintenance with a reduced-calorie diet and increased physical activity. Eli Lilly Australia aims to boost production by 150% in H2 2024 to meet demand, despite current shortages until August 2024. Mounjaro remains on private prescription, not PBS, starting at $395/month.

Basking Biosciences has initiated the Phase II RAISE trial of BB-031 for acute ischaemic stroke, aiming to assess safety, tolerability, and preliminary efficacy. The trial will enrol 156 patients within 24 hours of stroke onset, with a Data Safety Monitoring Committee overseeing the study. BB-031 targets von Willebrand Factor using RNA aptamer technology, aiming to expand therapeutic options for stroke patients.

Incendia Therapeutics has enrolled the first subject in a Phase Ic trial of PRTH-101, a DDR1 inhibitor for advanced solid tumours. The open-label trial will assess PRTH-101's safety, tolerability, and anti-tumour activity, both alone and with pembrolizumab. Up to 270 patients will be enrolled in the US, with the study aiming to determine optimal dosing for future Phase II programmes and explore DDR1 as a biomarker for patient responses.

China's Drug Administration Law reforms, including the nationwide Market Authorization Holder (MAH) system, aim to boost foreign investment and streamline drug approvals. Challenges persist in cross-border manufacturing, IP, and regulatory compliance. The MAH system allows partnerships with local manufacturers or CMOs for quicker market access. Cross-border manufacturing is legally feasible but practically limited. Foreign companies have options to either hold the MAH or have a local entity do so. Recent regulations support local manufacturing of imported drugs, with priority review for originator drugs, addressing concerns like IPR protection and regulatory compliance.