[비즈한국] Recently, there has been a series of cases where pharmaceutical and biotech companies have "voluntarily discontinued" clinical trials or "voluntarily withdrawn" clinical trial plans. Last month, LG Chem051910 announced that it was voluntarily discontinuing its global Phase 3 clinical trial for the gout treatment Tigulixostat. While clinical trials are sometimes halted in late-stage Phase 3 due to unexpected side effects, a "voluntary discontinuation" is rare, drawing significant attention from the market. However, looking at the cases of "voluntary discontinuation" or "voluntary withdrawal" of major clinical trials over the past six months, it appears that such decisions are not due to simple factors like concerns over low clinical success rates.
Clinical trial 'voluntary discontinuation' occurring once a month
On the 27th of last month, LG Chem announced through a "Major Management Matter Related to Investment Judgment" that it would voluntarily discontinue the global Phase 3 clinical trial for its gout treatment, 'Tigulixostat.' Tigulixostat is a once-daily oral gout medication with a mechanism that inhibits the expression of 'xanthine oxidase,' the enzyme that produces uric acid, the primary cause of gout. It was highly anticipated as it was the fastest-developing drug in the company's pipeline and the first gout treatment to prove its efficacy in a global Phase 3 trial. However, LG Chem stated, "Although the clinical results confirmed the safety and superiority of Tigulixostat compared to a placebo, we decided to terminate the trial due to a strategic reallocation of resources, considering its commercial value."
LG Chem plans to focus on its "oncology pipeline." According to the company's website, as of this month, 10 out of its 21 total pipelines—about half—are 'oncology pipelines.' These include two preclinical, six Phase 1, and two Phase 3 trials. This decision is interpreted as a reaction to the company’s operating loss of 252 billion KRW in the fourth quarter of last year, which marked its first annual deficit in five years. Previously, Vice Chairman Shin Hak-cheol stated at the annual shareholders' meeting that same month, "We will continuously secure financial soundness by thoroughly analyzing all costs from scratch, improving internal efficiency, adjusting priorities for efficient investment, and optimally allocating resources."
There have been a total of 7 cases of "voluntary discontinuation" or "voluntary withdrawal" of major clinical trials in the past six months, occurring at a rate of once a month. Last year, companies like GC Cell144510 and Curacle365270, and this year, Korea United Pharm033270, Genome & Company314130, LG Chem, Aprogen, and Peptron have made such decisions. While some voice concerns that voluntary discontinuation during a trial might stem from fears of failure, recent cases show that the reasons are varied, including market environments, strategic modifications, economic feasibility, and changes in regulatory policies. Discontinuations due to market environments and strategic changes have been the most common.
'Market response' is the most frequent reason
For GC Cell and Genome & Company, the reason was the "market environment." Last November, GC Cell announced the voluntary withdrawal of its Australian Phase 1 clinical trial for 'AB-201,' a cell therapy used for solid cancers like breast and stomach cancer. The company explained, "Unlike the 2022–2023 period when the Phase 1 trial was planned, the treatment environment has changed, leading us to believe that patient enrollment would be limited. We intend to choose and focus on domestic clinical trials to accelerate patient enrollment." It appears that the growth of a competing product, 'Enhertu,' affected patient recruitment.
Genome & Company early-terminated its domestic Phase 2 trial for the microbiome immuno-oncology drug 'GEN-001' for bile duct cancer patients last month. This was because of market changes, such as the approval of the combination therapy of Keytruda and chemotherapy as a first-line treatment for bile duct cancer. The company stated, "As a result of reviewing the feasibility of R&D and commercial viability against investment due to changes in the new drug development environment, we decided to terminate the clinical trial early as we revised our new drug development strategy."
'Strategic modification' is also a common reason. In December last year, Curacle voluntarily withdrew its Phase 2 clinical trial plan application for 'CU104,' an indication-extension pipeline of 'CU06,' which is being developed as a vascular endothelial function disruptor. This decision was based on the judgment that it would be more strategic to focus on the development of 'CU06,' for which safety and efficacy have already been proven. LG Chem also voluntarily discontinued the global Phase 3 trial for its gout treatment 'Tigulixostat' last month for the same reason. The company stated that, based on U.S. market research, it would stop developing the gout treatment and strengthen its investment in the oncology pipeline.
Affected by 'shrunken investment sentiment'?
Other reasons include 'economic feasibility' and 'changes in regulatory policies.' Korea United Pharm decided to voluntarily discontinue the Phase 3 clinical trial for the functional dyspepsia treatment 'UI028' in January because it failed to achieve its target drug price. The company announced, "We consulted with regulatory agencies such as the Health Insurance Review & Assessment Service and the Ministry of Food and Drug Safety to review economic feasibility, but judged that obtaining our target price would be difficult. Therefore, we decided to discontinue the Phase 3 trial, judging that continuing the development of UI028 would provide no real benefit to the company."
In some cases, companies benefit from changes in regulatory policies. Aprogen, which is developing the Herceptin biosimilar 'AP063,' announced the voluntary discontinuation of its Phase 3 trial after the European Medicines Agency (EMA) pushed for a policy that allows for biosimilar marketing authorization without Phase 3 clinical trial data. The company stated, "With the decision to voluntarily discontinue, we expect to reduce the development costs for the AP063 Phase 3 trial. During pre-consultations with the EMA, there is a possibility we may be asked to supplement data, such as results from a small additional Phase 1 trial to verify immunogenicity."
The pharmaceutical and biotech industry cites "shrunken investment sentiment" as a reason for the rise in voluntary clinical trial discontinuations. Since raising funds is difficult, the consensus is that it is a rational decision to terminate trials before they become too long if market conditions change. A source at a pharmaceutical company explained, "While there is a problem that credibility could take a hit upon discontinuation, choosing to focus might be better. This is because costs continue to accumulate the longer the clinical trial lasts."
In the "2024 Bio-Industry Assessment and 2025 Outlook" survey conducted by the Korea Biotechnology Industry Organization last year, 71.2% of companies identified "shrunken bio-investment sentiment" as the top major domestic and international issue for the bio-industry in 2024. This was followed by U.S.-China geopolitical conflicts such as the Biosecure Act (28.8%) and a slowdown in the listing of biopharmaceutical companies (32.2%). When asked about the role of the government in revitalizing the bio-industry, "financial support" was the most common response at 40.7%.