Precision and personalised medicine has a significant impact on how pharma companies and other stakeholders engage with patients, providing treatments that better fit specific patient populations, rather than generating inconsistent results in a broader patient population. These medicines will change treatment paradigms for chronic, life-long conditions that severely impact people’s quality of life such as Type 1 diabetes.
The approval of CellTrans’s Lantidra (donislecel), the first cell therapy for the treatment of Type 1 diabetes, in June 2023 could lend hope to the development and approval of other cell therapies for this patient population. In fact, the day following Lantidra’s approval, Eli Lilly revealed its $300 million acquisition of Sigilon Therapeutics, a drug developer with a cell therapy candidate in preclinical trials, also for the treatment of Type 1 diabetes.
According to GlobalData’s Epidemiology Database, diagnosed prevalent cases of Type 1 diabetes in the 16 major pharmaceutical markets (16MM) are expected to increase from 13 million in 2022, to over 16 million in 2029.
Personalised treatments such as cell therapies could help healthcare services to cope with the expected increase in patient population size by improving the quality of care, potentially reducing the cost and burden of lifelong insulin treatment, and decreasing the chance of hospitalisation.
Lantidra is an allogeneic cell therapy consisting of insulin-producing cells, collected from deceased donors, which help to control the insulin levels of adults with Type 1 diabetes who experience recurrent or severe hypoglycemia because of their inability to detect falling insulin levels.
Initial Phase I/II trials for Lantidra began in 2004; the drug received orphan drug designation from the Food and Drug Administration in 2017, and is expected to generate sales of $200 million by 2029, according to GlobalData patient-based forecasts. For some patients, the treatment could prove effective for years.
Exciting candidates in development all use different mechanisms of action. For example, Throne Biotechnologies’s autologous stem cell therapy, Educator Therapy, could become available to patients soon, as the only candidate currently in Phase III trials for Type 1 diabetes. Educator Therapy aims to correct the body’s autoimmune destruction of cells fundamental in metabolic control, and is also being trialed for alopecia areata and Covid-19 pneumonia.
Meanwhile, further down the pipeline, Avotres’s AVT-001 autologous cell therapy acts by targeting CD8+ regulatory T-cells as an immunomodulatory therapy and is expected to complete Phase I/II trials in December 2023.
Alternatively, Prokidney’s REACT autologous cell therapy acts as a regenerative therapy by regenerating renal cells in the kidney and is expected to launch commercially in the US by 2026, according to GlobalData estimates.
While most drugs in development are non-modified cell therapies, there are also several gene-modified cell therapies to watch out for. In particular, clustered regularly interspaced palindromic repeats (CRISPR) Therapeutics’s VCTX-210 is a gene-modified allogeneic beta-cell replacement therapy, created with CRISPR-Cas9 gene editing technology.
Developed in collaboration with ViaCyte, VCTX-210 is expected to launch commercially in the US by 2027 and will generate sales of over $400 million by 2029, according to GlobalData estimates.
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Given the rising prevalence of Type 1 diabetes worldwide, especially in developing countries, effective cell therapies that treat the cause of the disease and have longer-lasting effects, such as CellTrans’s Lantidra, will be in greater demand and therefore generate increased investment in innovation within this field.
However, to realise the potential of these treatments, and to offer hope to this patient population, the pharma industry needs to overcome some of the largest barriers to adoption such as costly manufacturing, complex logistics in moving biologics quickly between facilities, changing regulatory landscapes, and drug pricing and reimbursement.
*16 MM = the US, France, Germany, Italy, Spain, the UK, Japan, Australia, Brazil, Canada, China, India, Mexico, Russia, South Africa, and South Korea.