Clinical Trial News

The article provides an update on CRISPR-based therapies, highlighting the first-ever approval of a CRISPR-based medicine, Casgevy, for sickle cell disease and transfusion-dependent beta thalassemia. It discusses the progress, challenges, and future directions of CRISPR clinical trials across various diseases, including blood disorders, chronic bacterial infections, protein-folding diseases, inflammatory diseases, cancers, cardiovascular disease, HIV/AIDS, diabetes, and autoimmune diseases.

Clearside Biomedical discussed its Q4 2023 financial results and corporate updates, highlighting progress in its CLS-AX clinical development program for wet AMD, including the initiation and completion of the Phase IIb trial, Odyssey. The company emphasized its leadership in suprachoroidal drug delivery technology, collaborations, and anticipated data readouts in 2024. Financial updates included a cash balance sufficient to fund operations into Q3 2025.

Tyra Biosciences Inc reported a Q4 net loss of $22.8M, up from $12.9M last year, with R&D expenses doubling to $20.7M. The company's cash position is strong at $403.5M, supporting operations through 2026. Progress includes advancing TYRA-300 for mUC treatment and SURF301 Phase 1 study, with key milestones expected in 2024.

PMV Pharmaceuticals ended 2023 with $228.6M in cash, reporting a $69.0M net loss, improved from $73.3M. R&D expenses rose to $55.9M for rezatapopt development, while G&A costs fell to $24.2M. The company progresses in the PYNNACLE trial, with Phase 2 starting Q1 2024, and strengthens leadership for clinical advancements.

Amylyx Pharmaceuticals' shares fell after its ALS treatment failed in a phase III study. Acadia Pharmaceuticals also saw a decline due to a failed schizophrenia treatment study. Regulus Therapeutics reported positive results for a kidney disease treatment, boosting its shares. Moderna initiated a cancer therapy study, increasing its stock value. Regeneron received FDA approval to extend its cholesterol drug's label to pediatric patients.

BioNTech reported strong 2023 financials with €3.8B in revenues and advanced its oncology and infectious disease pipelines, aiming for multiple product approvals. The company plans to initiate 10+ registration trials by 2024 end, targeting a first oncology product launch by 2026. Despite COVID-19 vaccine revenue challenges, BioNTech remains profitable, focusing on innovative therapies and strategic collaborations to drive long-term growth.

Vaxart discussed its 2023 achievements, including progress in oral vaccine development for norovirus and COVID-19, highlighted by a $9.27M BARDA contract for a Phase IIb COVID-19 trial. The company emphasized the potential of its oral pill vaccines to offer advantages over injectables, such as ease of administration and mucosal immunity. Vaxart also announced the appointment of a new CEO, Steve Lo, and shared updates on financial results and future clinical trial plans.

A phase 1 clinical trial evaluating a new CAR-T therapy approach for glioblastoma has shown dramatic tumor regression in patients, with one achieving near-complete regression. Despite eventual tumor progression, the results are promising for treating this deadly brain cancer.

Vir Biotechnology's tobevibart and elebsiran received FDA Breakthrough Therapy and EMA PRIME designations for chronic hepatitis delta treatment, based on Phase 2 SOLSTICE trial data showing rapid virus suppression. The Phase 3 ECLIPSE program starts in 2025, aiming to expedite development for this serious condition with unmet medical needs.

Phase 2b trial of izokibep for hidradenitis suppurativa showed significant clinical improvements, with a third of patients achieving HiSCR100 by week 16. Patients switching from placebo to izokibep at week 16 matched the response speed and magnitude of those starting treatment earlier. Izokibep demonstrated a favorable safety profile and potential for differentiation with higher clinical responses. Long-term data indicated no increased safety risks and further clinical improvements over time.