High Stakes: Seven Key Drug Trial Failures in 2024
The cost of developing a new drug – a process that can take between 10 and 15 years from concept to market – has risen to approximately $2.3 billion, and there are many opportunities for failure along the way. Around 90% of drug candidates in clinical trials fail, despite significant investments in time and money.
Failure can be the result of poor trial design, safety issues, a lack of efficacy, or a lack of funding, failure to maintain good manufacturing protocols, failing to follow FDA guidance, or difficulty with patient recruitment and retention. An MIT School of Management research report found that even at Phase 3, the probability of a clinical trial having a successful outcome sat at just 59%.
Behind every drug trial failure is an army of people: researchers, clinicians, professionals and patients who have invested countless hours, resources and funding. These failures are disappointing for companies, particularly smaller biotechs who may not survive, and they can be devastating for patients desperate for new treatments.
2024 has already seen a number of clinical setbacks. PRISM MarketView took a look at seven high-profile failures in 2024:
Sage Therapeutics (SAGE) reported on Wednesday that its double-blind, placebo-controlled Phase 2 study of dalzanemdor in Parkinson’s Disease (PD) did not meet its primary endpoint, crushing the hopes of both the company’s dedicated researchers and clinicians, and the Parkinson’s disease community. Sage’s shares rallied +4.30% in intraday trading on Thursday after dipping on Wednesday. Based on the data, the company says it does not plan any further development of dalzanemdor in PD, but expects to deliver topline data from its studies in Huntington’s disease and Alzheimer’s disease during 2024.
Acadia Pharmaceuticals’ (ACAD) highly anticipated pimavanserin for the treatment of negative symptoms of schizophrenia did not demonstrate a statistically significant improvement over placebo in the company’s Phase 3 ADVANCE-2 trial. The decision followed two previous attempts by Acadia to secure regulatory approval for the expanded use of pimavanserin in the treatment of psychosis related to dementia and Alzheimer’s disease in 2021 and 2022.
Allakos Inc. (ALLK) announced in January 2024 that it would lay off half its workforce after its Phase 2 clinical trial in atopic dermatitis and its Phase 2b clinical trial in chronic spontaneous urticaria did not meet their primary endpoints.
Amylyx Pharmaceuticals’ (AMLX) voluntary withdrawal of RELYVRIO® dealt a significant blow to the amyotrophic lateral sclerosis (ALS) community after the company’ announced in March 2024 that its Phase 3 PHOENIX study had not met primary or secondary endpoints. Following the withdrawal, Amylyx has made the drug available free of charge to ALS patients who believe it is helping them, a decision applauded by The ALS Association. In April, the company announced it would cut 70% of its workforce, many of whom were engaged in research, in response to the PHOENIX study’s results.
Bristol Myers Squibb (BMY) announced in March 2024 that its Phase 3 YELLOWSTONE clinical trial program evaluating Zeposia (ozanimod) in moderate to severe active Crohn’s disease failed to meet its primary endpoint of clinical remission at Week 12. Zeposia had previously attracted attention as a treatment for multiple sclerosis, for which it received FA approval in 2020, and ulcerative colitis which was approved in 2021.
Australian pharma company, CSL Behring (CSL.AX) embarked on the ‘most ambitious’ clinical trial in its history, evaluating CSL112 in 17,400 heart attack patients across 49 countries. However, in February 2024, CSL announced that the trial failed to meet its primary endpoint of a reduction in the risk of major adverse cardiovascular events after 90 days when compared to placebo.
Gilead Sciences (GILD) missed its endpoint of overall survival in metastatic non-small cell lung cancer patients in a Phase 3 trial of Trodelvy versus standard-of-care chemotherapy, docetaxel. Gilead was granted FDA approval for Trodelvy in February 2023 as a treatment for breast cancer, and the drug has shown promise in treating two different types of metastatic breast cancer and has demonstrated improved clinical outcomes in metastatic urothelial cancer.
