BRISTOL MYERS SQUIBB

More than 250 branded drugs from pharmaceutical giants, including Bristol Myers Squibb, Sanofi-Pasteur, Merck, Pfizer, and others, will see price hikes in the U.S. starting Jan. 1. Pfizer alone is raising prices on over 60 drugs, with a median increase of 4.5%. The price hikes, which do not include rebates or discounts, are necessary to support investments in new medicines and address increased costs. U.S. prescription drug prices are significantly higher compared to those in similarly wealthy countries, and while annual increases have been scaled back, drug launch prices continue to rise, leading to restricted access and higher out-of-pocket costs for patients. Additional price increases are expected to be announced this month.

The FDA concluded 2024 with significant drug approvals, including novel treatments for schizophrenia, COPD, Alzheimer's, and gene therapies for hemophilia B and L-amino acid decarboxylase deficiency. Key decisions in 2025 include verdicts on Biogen and Eisai’s Leqembi for Alzheimer's, AstraZeneca and Daiichi Sankyo’s Dato-DXd for breast cancer, Vertex’s Suzetrigine for acute pain, Alnylam’s Amvuttra for ATTR-CM, and Sanofi’s Fitusiran for hemophilia A and B.

Bristol Myers Squibb's phase 3 trial of Reblozyl showed significant improvement in red blood cell transfusion independence for MDS patients, meeting primary and secondary endpoints. The COMMANDS study demonstrated Reblozyl's efficacy and safety, offering a new first-line treatment option for transfusion-dependent MDS patients.

Bayer advances asundexian to phase 3 trials despite missing efficacy in phase 2, focusing on atrial fibrillation and stroke prevention. Asundexian showed safety without increased bleeding. Competing with BMS and J&J's milvexian, which also demonstrated safety and potential efficacy in stroke prevention, both aim to innovate anticoagulant therapy.

FDA approved Ferring Pharmaceuticals' Adstiladrin, a gene therapy for high-risk BCG-unresponsive non-muscle-invasive bladder cancer. It's the first gene therapy for this condition, showing a 51% complete response rate. Adstiladrin will be available in the US by late 2023, offering a new treatment option.

Autolus secures FDA approval for Aucatzyl, a CD19-targeting CAR-T therapy for relapsed or refractory B-cell precursor ALL, with no REMS requirement. Aucatzyl achieved 42% complete remission in the FELIX trial, with low rates of CRS and neurotoxicity. Competing with Novartis' Kymriah and Gilead/Kite's Tecartus, Aucatzyl aims to capture market share in the US, targeting around 3,000 patients.

FDA approved subcutaneous nivolumab for solid tumors, supported by CheckMate-67T trial. Tislelizumab approved for gastric/GEJ cancers, based on RATIONALE-305 trial. 2024 saw breast cancer advancements, including inavolisib and ribociclib approvals. ctDNA emerged as a biomarker in HER2+ breast cancer, per DAPHNe trial findings.

In 2024, CGTLive® tracked cell and gene therapy advancements, covering major medical meetings and FDA decisions. Highlights included Epic Bio's EPI-321 for FSHD, CAR-T therapy for autoimmune diseases by Georg Schett, MD, Tessera Therapeutics' RNA gene writers, Kriya Therapeutics' AAV vector-based gene therapy for common diseases, and Arbor Biotechnologies' ABO-101 for primary hyperoxaluria type 1.

General Proximity, a biotech company, emerged from stealth with its OmniTAC platform for induced proximity medicines, raising $16M. Backed by notable investors and a team of top scientists, it aims to target undruggable proteins in diseases like cancer and neurodegeneration, leveraging biological proximity for drug discovery.

Sanofi's Sarclisa shows promise in phase 3 IKEMA trial for earlier multiple myeloma treatment, aiming to compete with J&J's Darzalex. Despite Darzalex's market lead and new subcutaneous formulation, Sarclisa offers a simpler infusion and potential for patients resistant to Darzalex. Sanofi plans to extend Sarclisa's label based on IKEMA results.