Merck's Keytruda Faces Setback in Advanced Liver Cancer Trial but Maintains Strong Growth Outlook

VCBeat (WeChat ID: biobeat1) has learned that Merck recently announced the results of a pivotal Phase III study evaluating the efficacy of its PD-1 inhibitor Keytruda in the treatment of advanced hepatocellular carcinoma (HCC), the most common type of liver cancer. The assessment showed that the drug failed to meet its co-primary endpoints of overall survival (OS) and progression-free survival (PFS).

KEYNOTE-240 was a comparative study evaluating Keytruda plus best supportive care versus placebo plus best supportive care for the treatment of patients with advanced hepatocellular carcinoma (HCC) who had previously received systemic therapy. Although the study demonstrated improvements in overall survival (OS) and progression-free survival (PFS) in the Keytruda group compared with the placebo group, the results were not statistically significant.

“We are disappointed that KEYNOTE-240 did not meet its co-primary endpoints, but the results for overall survival (OS), progression-free survival (PFS), and objective response rate (ORR) were consistent with those from the Phase 2 study (KEYNOTE-224), which supported the accelerated approval of Keytruda for the treatment of patients with hepatocellular carcinoma who had previously been treated with sorafenib,” said Dr. Roy Baynes, Chief Medical Officer and Senior Vice President and Head of Global Clinical Development at Merck Research Laboratories.

Keytruda is currently approved for the treatment of patients with hepatocellular carcinoma (HCC) who have previously been treated with Bayer’s/Amgen’s Nexavar (sorafenib). This approval is based on data from the KEYNOTE-224 study. If the data from the KEYNOTE-240 study are positive and ultimately lead to an expansion of Keytruda’s labeled indications, it will broaden the drug’s target patient population.

Keytruda, as a monotherapy for second-line hepatocellular carcinoma, is being evaluated in several other studies, including the Phase III KEYNOTE-394 study.

In another press release, Merck announced that the FDA had approved Keytruda as an adjuvant therapy for the treatment of patients with high-risk stage III melanoma.

In the United States, Keytruda has been approved as a monotherapy for adult patients with advanced melanoma. Its recent approval as an adjuvant therapy will enable Merck to reach a broader population of melanoma patients in the U.S., marking the first time Keytruda has received such approval for adjuvant use in the country. Last December, Keytruda was also approved in the European Union for the adjuvant treatment of melanoma.

The FDA’s approval was based on data from the pivotal Phase III KEYNOTE-054 study, which was conducted in collaboration with the European Organisation for Research and Treatment of Cancer (EORTC). The study data demonstrated that treatment with Keytruda significantly reduced the risk of cancer recurrence after surgery in patients with melanoma, a population typically at high risk for disease recurrence.

Merck & Co.’s stock rose 45.3% over the past year, compared with a 7.8% gain for the industry.

Merck’s outstanding performance over the past year was primarily driven by strong financial results and regulatory updates related to Keytruda. In a short period, Keytruda has become Merck’s flagship product. It is currently approved in the United States for 15 indications across 10 different tumor types. The drug generated $7.17 billion in sales in 2018, representing a substantial year-over-year increase of 88%. Currently, Keytruda continues to expand globally and penetrate new markets.

The development program for Keytruda is also progressing smoothly, with the drug currently being investigated in more than 900 clinical trials across over 30 types of cancer, including more than 500 combination studies. Furthermore, Merck is collaborating with multiple companies, including Amgen, Incyte, GlaxoSmithKline, and Pfizer, to evaluate the combined efficacy of Keytruda and other therapeutic regimens.

Foreign media have stated that Keytruda has strong growth prospects, driven by its significantly increased utilization, recent approvals for new indications, and potential additional approval opportunities worldwide.

(Compiled by Cheng Xiaoqin)