BRISTOL MYERS SQUIBB

CAR T cell therapy for autoimmune diseases, including lupus, is advancing with positive Phase 1 trial results showing potential to reset immune systems, reduce disease activity, and improve quality of life.

2024 saw significant clinical successes and failures in biopharma, with Vertex, BMS, and Alnylam achieving notable wins, while AbbVie, Sage Therapeutics, Pfizer, and Gilead faced setbacks in neurological and other indications. Despite high failure rates in drug trials, particularly in CNS diseases, some companies advanced new treatments, while others faced challenges in their pipelines.

The global Sickle Cell Disease (SCD) treatment market, valued at US$ 2.7 billion in 2023, is expected to grow at a CAGR of 18.5% to surpass US$ 17.7 billion by 2034. Growth is driven by increasing SCD prevalence, medical research advancements, and innovative treatments like gene therapy. North America leads due to strong healthcare infrastructure and significant patient population, while Sub-Saharan Africa and Asia are key due to high disease prevalence. Challenges include high treatment costs and limited healthcare access in developing regions.

Avidity Biosciences, leveraging its AOC platform, excels in RNA therapies for muscle diseases, showing a 276% return over the past year. With a $3.9B market cap, it focuses on precision cardiology and muscle-related treatments, boasting a robust pipeline including del-desiran for DM1 and del-brax for FSHD. Despite negative EPS, its strong financials and upcoming milestones signal growth potential.

Real-world analysis of ide-cel in r/r MM with CNS involvement showed 70% had no CNS relapse post-treatment, with median PFS of 10.5 months and OS of 12.9 months. CAR T-cell therapy demonstrated efficacy and feasibility in this rare form of MM.

Cendakimab, under development for eosinophilic esophagitis and gastroenteritis, is expected to generate $143 mn in annual revenue by 2037 in the US. Bristol-Myers Squibb, a biopharmaceutical company, reported revenues of $45,006 mn for FY2023, with a net margin of 17.8%.

Editas Medicine lays off 65% of staff, shelving its sickle cell disease treatment Reni-Cel; Ionis Pharmaceuticals shifts to in-house drug commercialization; GLP-1 drugs spark debate in Trump's circle; investors sue Bristol Myers Squibb over Padlock Therapeutics milestone payments.

The FDA emphasizes the need for diversity in clinical trials to ensure drugs work across all populations. Despite recognizing the importance, practical steps to achieve this diversity remain unclear. Community engagement and collaboration are key to bridging the gap between theory and practice.

2024 saw significant medical advances, including FDA approval of Orlynvah for resistant UTIs, Cobenfy for schizophrenia, lenacapavir for HIV prevention, and Miplyffa/Aqneursa for Niemann-Pick disease type C. Elinzanetant showed promise for hot flashes, and future innovations include potential non-opioid pain treatments, improved flu vaccines, and gene therapy for vision restoration.

Tasca Therapeutics announced a $52 million Series A financing to advance its drug discovery platform, progress CP-383 into Phase 1/2 clinical studies, and expand its drug candidate pipeline. The company identifies auto-palmitoylated proteins as unique therapeutic targets across various diseases. CP-383, a first-in-class molecule, has shown robust anti-cancer activity. The executive team brings extensive experience in drug discovery and development.