Following U.S. Pfizer and U.K. GlaxoSmithKline (GSK), Japan's Takeda Pharmaceutical has also decided to pull out of developing cell and gene therapies (CGT).
Takeda said on the 1st on its website that it is not currently conducting clinical trials for cell therapies and is seeking a partner to transfer its preclinical-stage research programs. The company said it will focus its research and development (R&D) capabilities on three areas: small molecules, biologics, and antibody-drug conjugates (ADC).
Cell and gene therapies are next-generation biopharmaceuticals that fundamentally treat diseases by modifying a patient's cells or genes. Representative examples include therapies that regulate or alter gene expression, CAR (chimeric antigen receptor)-T cells, and stem cell therapies. They were once called the "dream therapy" that could fundamentally cure diseases with a single injection, but corporations are increasingly walking away due to massive development expense and growing safety concerns.
Takeda also plans to sell the T-cell therapy platform of GammaDelta Therapeutics, which it acquired in 2021. It will halt phase 1/2 clinical trials of a new drug candidate for acute myeloid leukemia that was being developed with this technology.
The direct backdrop to the pullout is deteriorating profitability. As sales plunged due to the patent expiration of its flagship attention-deficit/hyperactivity disorder (ADHD) drug Vyvanse and the spread of generics, Takeda last year alone trimmed pipeline (new drug development projects) worth 1.2615 trillion won. Earlier in May this year, it also cut half of its oncology pipeline, halting development of small-molecule candidates and T-cell therapies.
Takeda is not the only pharmaceutical company stepping away from cell and gene therapies. The previous day, Denmark's Novo Nordisk also terminated a joint development agreement for a heart failure cell therapy with a Japanese bio company. The termination fee is up to $598 million (837.7 billion won). Pfizer and GSK also halted CGT clinical trials last year and moved to large-scale job cuts, and Kite, a subsidiary of U.S. Gilead, and Genentech of Switzerland's Roche likewise scrapped projects worth billions of dollars in succession.
Reports of patient deaths in recent clinical trials have also fueled the exit trend. A death occurred in the clinical trial of Elevidys, a gene therapy for Duchenne muscular dystrophy (DMD) being co-developed by Roche and U.S. Sarepta Therapeutics, and a patient also died in a clinical trial of a CAR-T cell therapy by U.S. Allogene Therapeutics.
Cell and gene therapies had been expected to be an innovative alternative for rare and intractable diseases, but their limitations—namely the difficulty of predicting immune responses and long-term toxicity—are resurfacing. That does not mean, however, that their value as new drugs has disappeared.
Experts say that as basic research into how cells and genes work advances, therapy development could regain momentum. A person in the pharmaceutical industry said, "Obesity treatments also had many side effects in early drugs, but as basic research newly clarified the mechanisms, candidates that resolve side effects have recently been developed one after another."
In particular, CAR-T cell therapy, dubbed the "dream anticancer drug," is a prime example. Global pharmaceutical companies around the world are accelerating efforts to develop products to follow Kymriah from Switzerland's Novartis, currently the only CAR-T cell therapy. Among domestic corporations, Curocell has completed a phase 3 clinical trial of a CAR-T cell therapy for lymphoma, a type of blood cancer, and is awaiting approval from the Ministry of Food and Drug Safety, while AbClon is also conducting late-stage clinical trials for a lymphoma treatment.