Clinical Trial News

Capricor Therapeutics' CAP-1002 trial for Duchenne muscular dystrophy continues after positive DSMB review. Plans to discuss expedited FDA approval in 2024. CAP-1002, derived from heart cells, aims to improve arm and heart function in DMD patients. Recruitment ongoing; top-line data expected late 2024.

A study conducted in Trieste, Italy, evaluated the feasibility of a self-sampling-based cervical cancer screening strategy during the COVID-19 pandemic. The study aimed to reduce screening delays caused by lockdowns by offering home-collection kits to women. Results showed a high return rate of 80%, with the HPV Selfy test proving effective for self-sampling-based screening. The study supports the use of self-sampling as a complementary method to enhance cervical cancer screening coverage.

Phase 2 trial results for odronextamab showed a 52% ORR and 31% CR in DLBCL patients, with durable responses. Phase 1 data indicated a 48% ORR and 30% CR post-CAR-T progression. ctDNA negativity linked to longer PFS. Odronextamab is under FDA and EMA review for R/R DLBCL and FL.

Solid Biosciences Inc. received FDA Fast Track designation for SGT-003, a next-generation gene therapy for Duchenne muscular dystrophy (DMD), using AAV-SLB101 capsid. This designation aims to expedite development and review, following encouraging pre-clinical data. The company's stock surged 68.4% post-announcement. A phase I/II study will assess SGT-003's safety and efficacy in pediatric DMD patients.

The U.S. Food and Drug Administration (FDA) has granted priority review for the New Drug Application (NDA) of elafibranor, a potential novel second-line treatment for primary biliary cholangitis (PBC), a rare cholestatic liver disease. The European Medicines Agency (EMA) and the UK Medicines and Healthcare products Regulatory Agency (MHRA) have also validated the Marketing Authorization Application (MAA) for review. The target FDA PDUFA date is set for June 10, 2024.

Jacobio Pharma to present clinical results of KRAS G12C inhibitor glecirasib at 2024 ASCO GI. Glecirasib, granted breakthrough therapy designation for pancreatic cancer, shows promise in ongoing trials for solid tumors with KRAS G12C mutation, aiming to expedite treatment access.

Novartis announced FDA approval of Fabhalta® (iptacopan) as the first oral monotherapy for adults with PNH, showing superior hemoglobin level increases and transfusion avoidance in trials. Fabhalta, a Factor B inhibitor, addresses unmet needs in PNH treatment, offering a new option for patients previously treated with anti-C5 therapies.

Neurocrine Biosciences announced FDA Breakthrough Therapy designation for crinecerfont in congenital adrenal hyperplasia, with a new drug application planned for 2024. The company also updated its R&D portfolio, including advancements in gene therapy and muscarinic compounds, and anticipates Phase 2 data readouts for several programs in 2024.

Seattle Children's Hospital launched BrainChild Bio, a biotech startup focusing on CAR-T cell therapies for pediatric brain tumors, leveraging technology and funding from the hospital. With prior clinical testing and plans for future trials, BrainChild aims for accelerated approval, despite FDA scrutiny into CAR-T therapy's safety risks.

In Chinese patients with mHSPC, DARO combined with ADT + DOC improved overall survival, delayed progression to CRPC and PSA, with a safety profile similar to placebo. DARO showed significant benefits in PSA responses and treatment duration, consistent with the ARASENS study's overall findings.